Grace Therapeutics, Inc 10-K for FY2026
Grace Therapeutics, Inc. (GRCE)
10-K
2026-06-18
For: 2026-03-31
View Original
Added on
June 18, 2026
About this 10-K
Grace Therapeutics, Inc filed this 10-K on June 18, 2026, covering the fiscal year ended March 31, 2026 (FY2026).
Headline figures
Fiscal year ended March 31, 2026, as reported in this filing, compared with the same period a year earlier where the filing states it.
- Net loss
- $7.79M
- compared with a net loss of $9.57M a year earlier
- Diluted EPS
- -$0.47
- compared with -$0.79 a year earlier
UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549
FORM 10-K
(Mark One)
ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934
For the fiscal year ended March 31 ,
or
TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934
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For the transition period from
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Commission file number: 001-35776
(Exact name of registrant as specified in its charter)
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(Address of principal executive offices, including zip code)
(Registrant’s telephone number, including area code)
Securities registered pursuant to Section 12(b) of the Act:
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Securities registered pursuant to Section 12(g) of the Act: None
Indicate by check mark if the registrant is a well-known seasoned issuer, as defined in Rule 405 of the Securities Act. Yes ☐ No ☒
Indicate by check mark if the registrant is not required to file reports pursuant to Section 13 or 15(d) of the Act. Yes ☐ No ☒
Indicate by check mark whether the registrant: (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange Act of 1934 during the preceding 12 months (or for such shorter period that the registrant was required to file such reports), and (2) has been subject to such filing requirements for the past 90 days. Yes ☒ No ☐
Indicate by check mark whether the registrant has submitted electronically every Interactive Data File required to be submitted pursuant to Rule 405 of Regulation S-T (§232.405 of this chapter) during the preceding 12 months (or for such shorter period that the registrant was required to submit such files). Yes ☒ No ☐
Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, a smaller reporting company, or an emerging growth company. See definitions of “large accelerated filer,” “accelerated filer,” “smaller reporting company,” and “emerging growth company” in Rule 12b-2 of the Exchange Act.
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If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐
Indicate by check mark whether the registrant has filed a report on and attestation to its management’s assessment of the effectiveness of its internal control over financial reporting under Section 404(b) of the Sarbanes-Oxley Act (15 U.S.C. 7262(b)) by the registered public accounting firm that prepared or issued its audit report. ☐
If securities are registered pursuant to Section 12(b) of the Act, indicate by check mark whether the financial statements of the registrant included in the filing reflect the correction of an error to previously issued financial statements. ☐
Indicate by check mark whether any of those error corrections are restatements that required a recovery analysis of incentive-based compensation received by any of the registrant’s executive officers during the relevant recovery period pursuant to §240.10D-1(b). ☐
Indicate by check mark whether the registrant is a shell company (as defined in Rule 12b-2 of the Securities Exchange Act of 1934). Yes ☐ No ☒
The aggregate
market value of the voting and non-voting shares of common stock held by
non-affiliates of the registrant, based on the closing sale price of the
registrant’s common stock on September 30, 2025, the last business day of its
most recently completed second fiscal quarter, as reported on the Nasdaq Stock
Market, was $35,051,405 .
The number of outstanding shares of common stock of the registrant, par value $0.0001 per share, as of June 15, 2026, was 16,274,026 .
DOCUMENTS INCORPORATED BY REFERENCE
Certain portions of the registrant’s definitive proxy statement for its 2026 annual meeting of stockholders, which the registrant intends to file pursuant to Regulation 14A with the Securities and Exchange Commission no later than 120 days after the end of the registrant’s fiscal year to which this Annual Report on Form 10-K relates, are incorporated by reference into Part III of this Annual Report on Form 10-K.
1
GRACE THERAPEUTICS, INC.
(Formerly ACASTI PHARMA INC.)
FORM 10-K
For the Fiscal Year Ended March 31, 2026
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PART I
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Item 1.
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Item 1A.
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Item 1B.
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| Item 1C. | Cybersecurity | 58 | |
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Item 2.
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Item 3.
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Item 4.
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PART II
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Item 5.
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Item 6.
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Item 7.
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Item 7A.
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Item 8.
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Item 9.
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Item 9A.
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Item 9B.
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Item 9C.
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PART III
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Item 10.
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Item 11.
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Item 12.
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Item 13.
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Item 14.
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PART IV
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Item 15.
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Item 16.
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SPECIAL NOTE REGARDING FORWARD-LOOKING STATEMENTS
This Annual Report on Form 10-K contains information that may be forward-looking statements within the meaning of U.S. federal securities laws and forward-looking information within the meaning of Canadian securities laws, both of which we refer to in this Annual Report on Form 10-K as forward-looking statements. Forward-looking statements can be identified by the use of terms such as “may,” “will,” “should,” “expect,” “plan,” “anticipate,” “believe,” “intend,” “estimate,” “predict,” “potential,” “continue,” or other similar expressions concerning matters that are not statements about the present or historical facts. Forward-looking statements in this Annual Report on Form 10-K include, among other things, information or statements about:
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our ability to build a late-stage pharmaceutical company focused in rare and orphan diseases and, on developing and commercializing products that improve clinical outcomes using our novel drug delivery technologies;
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our ability to apply new proprietary formulations to existing pharmaceutical compounds to achieve enhanced efficacy, faster onset of action, reduced side effects, and more convenient drug delivery that can result in increased patient compliance;
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the potential for our drug candidates to receive exclusivity from the U.S. Food and Drug Administration (“FDA”) or regulatory approval under the Section 505(b)(2) regulatory pathway under the Federal Food, Drug and Cosmetic Act (“FDCA”);
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our ability, plan and timing to address the items referenced in the Complete Response Letter (“CRL”) from the FDA related to GTx-104; the timing and outcome of the
Type A meeting scheduled with the FDA related to GTx-104; our a resubmission of a new drug application (“NDA”) for GTx-104 under Section 505(b)(2) of the FDCA; the acceptance of such resubmission of an NDA by the FDA; and the timing and ability to receive FDA approval for marketing GTx-104;
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the future prospects of our GTx-104 drug candidate, including but not limited to GTx-104’s potential to be administered to improve the management of hypotension in patients with aneurysmal subarachnoid hemorrhage (“aSAH”); the ability of GTx-104 to achieve a pharmacokinetic (“PK”) and safety profile similar to the oral capsule form of nimodipine; GTx-104’s potential to provide improved bioavailability; and GTx-104’s potential to achieve pharmacoeconomic benefit over the oral capsule form of nimodipine;
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our plan to maximize the value of our de-prioritized drug candidates, GTx-102 and GTx-101, including through potential licensing or sale of those drug candidates;
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the future prospects of our GTx-102 drug candidate, including but not limited to GTx-102’s potential to provide clinical benefits to decrease symptoms associated with Ataxia Telangiectasia; GTx-102’s potential ease of drug administration; the timing and outcomes of a Phase 3 efficacy and safety study for GTx-102; the timing of an NDA filing for GTx-102 under Section 505(b)(2) of the FDCA; and the timing and ability to receive FDA approval for marketing GTx-102;
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the future prospects of our GTx-101 drug candidate, including but not limited to GTx-101’s potential to be administered to postherpetic neuralgia (“PHN”) patients to treat the severe nerve pain associated with the disease; assumptions about the biphasic delivery mechanism of GTx-101, including its potential for rapid onset and continuous pain relief for up to eight hours; and the timing and outcomes of single ascending dose/multiple ascending dose and PK bridging studies, and a Phase 2 and Phase 3 efficacy and safety study; the timing of an NDA filing for GTx-101 under Section 505(b)(2) of the FDCA; and the timing and ability to receive FDA approval for marketing GTx-101;
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the quality of our clinical data, the cost and size of our development programs, expectations and forecasts related to our target markets and the size of our target markets; the cost and size of our commercial infrastructure and manufacturing needs in the United States, European Union, and the rest of the world; and our expected use of a range of third-party contract research organizations (“CROs”) and contract manufacturing organizations (“CMOs”) at multiple locations;
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expectations and forecasts related to our intellectual property portfolio, including but not limited to the probability of receiving orphan drug exclusivity from the FDA for our leading pipeline drug candidates; our patent portfolio strategy; and outcomes of our patent filings and extent of patent protection;
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our intellectual property position and duration of our patent rights;
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our strategy, future operations, prospects and the plans of our management with a goal to enhance shareholder value;
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our need for additional financing, and our estimates regarding our operating runway and timing for future financing and capital requirements;
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our expectations regarding our financial performance, including our costs and expenses, liquidity, and capital resources;
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our projected capital requirements to fund our anticipated expenses; and
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our ability to commercialize GTx-104 in the United States or establish strategic partnerships or commercial collaborations or obtain non-dilutive funding.
Although the forward-looking statements in this Annual Report on Form 10-K are based upon what we believe are reasonable assumptions, you should not place undue reliance on those forward-looking statements since actual results may vary materially from them.
In addition, the forward-looking statements in this Annual Report on Form 10-K are subject to a number of known and unknown risks, uncertainties and other factors, many of which are beyond our control, that could cause our actual results and developments to differ materially from those that are disclosed in or implied by the forward-looking statements, including, among others:
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we are heavily dependent on the success of our lead drug candidate, GTx-104;
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we may not be able to sufficiently address the issues set forth in the FDA’s Complete Response Letter for GTx-104;
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we may not be able to resubmit the NDA for GTx-104 in a timely manner or otherwise, or resubmission may not result in approval by the FDA;
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our contract manufacturer may not be able to remediate the deficiencies identified during a current Good Manufacturing Practice (“cGMP”) inspection that were cited in the FDA’s Complete Response Letter for GTx-104 in a timely or satisfactory manner or demonstrate ongoing compliance with applicable regulatory requirements, including cGMP;
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clinical development is a lengthy and expensive process with an uncertain outcome, and results of earlier studies and trials may not be predictive of future trial results. Failure can occur at any stage of clinical development;
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we are subject to uncertainty relating to healthcare reform measures and reimbursement policies that, if not favorable to our drug candidates, could hinder or prevent our drug candidates’ commercial success;
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if we are unable to establish sales and marketing capabilities or enter into agreements with third parties to market and sell our drug products, if approved, we may be unable to generate any revenue;
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if we are unable to differentiate our drug products from branded reference drugs or existing generic therapies for similar treatments, or if the FDA or other applicable regulatory authorities approve products that compete with any of our drug products, our ability to successfully commercialize our drug products would be adversely affected;
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our success depends in part upon our ability to protect our intellectual property for our drug candidates;
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intellectual property rights do not necessarily address all potential threats to our competitive advantage;
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we do not have internal manufacturing capabilities, and if we fail to develop and maintain supply relationships with various third-party manufacturers, or if such third parties fail to provide us with sufficient quantities of active pharmaceutical ingredients, excipients or drug products, or fail to do so at acceptable quality levels or prices or fail to maintain or achieve satisfactory regulatory compliance, we may be unable to develop or commercialize our drug candidates;
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the design, development, manufacture, supply, and distribution of our drug candidates are highly regulated and technically complex; and
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the other risks and uncertainties identified in Item 1A. Risk Factors and Item 7. Management’s Discussion and Analysis of Financial Condition and Results of Operations included in this Annual Report on Form 10-K.
All of the forward-looking statements in this Annual Report on Form 10-K are qualified by this cautionary statement. There can be no guarantee that the results or developments that we anticipate will be realized or, even if substantially realized, that they will have the consequences or effects on our business, financial condition, or results of operations that we anticipate. As a result, you should not place undue reliance on forward-looking statements. Except as required by applicable law, we do not undertake to update or amend any forward-looking statements, whether as a result of new information, future events or otherwise. All forward-looking statements are made as of the date of this Annual Report on Form 10-K.
We express all amounts in this Annual Report on Form 10-K in U.S. dollars, except where otherwise indicated. References to “$” and “U.S.$” are to U.S. dollars.
Except as otherwise indicated, references in this Annual Report on Form 10-K to “Grace,” “Grace Therapeutics,” “Acasti,” “the Company,” “we,” “us,” and “our” refer to Grace Therapeutics, Inc. (formerly known as Acasti Pharma, Inc.) and its consolidated subsidiary.
RISK FACTOR SUMMARY
The risk factors summarized below could materially harm our business, operating results and/or financial condition, impair our future prospects and/or cause the price of shares of our common stock to decline. For more information, see “Item 1A. Risk Factors” in this Form 10-K. Material risks that may affect our business, operating results and financial condition include, but are not necessarily limited to, the following:
Risks Related to our Business
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We received a Complete Response Letter from the FDA citing certain manufacturing deficiencies and other non-clinical issues, which may delay or prevent approval of GTx-104.
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We may not achieve our publicly announced milestones on time, or at all.
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We are heavily dependent on the success of our lead drug candidate, GTx-104.
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We may not be able to maintain our operations and advance our research and development and if approved, commercialization of our GTx-104 lead drug candidate without additional funding.
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We have no history of commercializing drugs, which may make it difficult for you to evaluate the success of our business to date and to assess our future viability.
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Disruptions at the FDA, the SEC and other government agencies could hinder their ability to hire and retain key leadership and other personnel, prevent or delay new products and services from being developed, approved, or commercialized in a timely manner or otherwise prevent those agencies from performing normal business functions on which the operation of our business may rely, which could negatively impact our business.
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Unfavorable global geopolitical events and economic conditions, including military conflicts and trade tensions between the U.S. and its global trading partners, including China, could adversely affect our business, financial condition or results of operations, including conduct of our clinical trials and our manufacturing activities.
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We may not be able to maximize value from our de-prioritized drug candidates, GTx-102 and GTx-101, through either out-licensing or sale.
Risks Related to Development, Testing and Commercialization of Our Products
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The regulatory approval processes of the FDA and comparable foreign authorities are lengthy, time consuming and inherently unpredictable, and if we are ultimately unable to obtain regulatory approval for our drug candidates, our business will be substantially harmed.
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If a product candidate causes undesirable side effects, then we may be unable to receive regulatory approval or market acceptance for such product candidate.
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Our drug development strategy relies heavily upon the 505(b)(2) regulatory pathway, which requires us to certify that we do not infringe upon third-party patents covering approved drugs. Such certifications often result in third-party claims of intellectual property infringement, the defense of which will be costly and time consuming, and an unfavorable outcome in any litigation may prevent or delay our development and commercialization efforts which would harm our business.
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If the market opportunities for GTx-104 are smaller than we believe they are, our results of operations may be adversely affected, and our business may suffer.
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We are subject to uncertainty relating to healthcare reform measures and reimbursement policies which, if not favorable to our drug candidates, could hinder or prevent our drug candidates’ commercial success.
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Recent and future changes in healthcare legislation and regulations may increase the difficulty and cost to obtain marketing approval for a drug candidate, increase the costs to commercialize an approved product, and adversely affect the price set for such product.
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Our commercial success depends upon attaining significant market acceptance of our drug candidates and drug products, if approved, among physicians, nurses, pharmacists, patients, the medical community, and third-party payors.
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If we are unable to establish sales and marketing capabilities or enter into agreements with third parties to market and sell our drug candidates, we may be unable to generate any revenue.
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Even if GTx-104 receives regulatory approval, we will still face regulatory difficulties.
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Even if our drug candidates receive regulatory approval in the United States, we may never obtain regulatory approval or successfully commercialize our products outside of the United States.
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If we are unable to differentiate our drug candidates from branded reference drugs or existing generic therapies for similar treatments, or if the FDA or other applicable regulatory authorities approve generic products that compete with any of our drug candidates, our ability to successfully commercialize our drug candidates would be adversely affected.
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We face significant competition from other biotechnology and pharmaceutical companies, and our operating results will suffer if we fail to compete effectively.
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We could incur substantial costs and disruption to our business and delays in the launch of our drug candidates if our competitors and/or collaborators bring legal action against us, which could harm our business and operating results.
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We are subject to numerous complex regulatory requirements and failure to comply with these regulations, or the cost of compliance with these regulations, may harm our business, or
subject us to civil or criminal proceedings, investigations, or penalties and
may limit our ability to commercialize our products.
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Our employees, independent contractors, principal investigators, consultants, commercial partners and vendors may engage in misconduct or other improper activities, including non-compliance with regulatory standards and requirements.
Risks Related to Our Intellectual Property
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If we are sued for infringing intellectual property rights of third parties, it will be costly and time consuming, and an unfavorable outcome in that litigation would have a material adverse effect on our business.
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We may be subject to claims that our employees, consultants, or independent contractors have wrongfully used or disclosed alleged confidential information or trade secrets of their other clients or former employers to us.
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Our success depends in part upon our ability to protect our intellectual property for our branded products and drug candidates.
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If we fail to comply with our obligations in the agreements under which we license rights to technology from third parties, or if the license agreements are terminated for other reasons, we could lose license rights that are important to our business.
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We may be subject to claims challenging the inventorship or ownership of our patents and other intellectual property.
Risks Related to Our Dependence on Third Parties
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If our current CMO is unable to timely resolve the deficiencies identified by the FDA in the CRL related to GTx‑104, we may be required to engage an alternative manufacturer, which could result in significant delays, increased costs, or our inability to obtain regulatory approval or commercialize GTx‑104 at all.
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We rely on third parties to manufacture commercial and clinical supplies of our drug candidates, and we intend to rely on third parties to manufacture commercial supplies of any approved drug products. The commercialization of any of our drug products could be stopped, delayed, or made less profitable if those third parties fail to provide us with sufficient quantities of active pharmaceutical ingredients, excipients, or drug products, or fail to do so at acceptable quality levels or prices or fail to maintain or achieve satisfactory regulatory compliance.
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We may not be successful in establishing or maintaining development and commercialization collaborations, and any partner may not devote sufficient resources to the development or commercialization of our drug candidates or may otherwise fail in development or commercialization efforts, which could adversely affect our ability to develop certain of our drug candidates and our financial condition and operating results.
PART I
Overview
We are focused on developing and commercializing products for rare and orphan diseases that have the potential to improve clinical outcomes by using our novel drug delivery technologies. We seek to apply new proprietary formulations to approved and marketed pharmaceutical compounds to achieve enhanced efficacy, faster onset of action, reduced side effects, more convenient drug delivery and increased patient compliance; all of which could result in improved patient outcomes. The active pharmaceutical ingredients used in the drug candidates under development by us may be already approved in a target indication or could be repurposed for use in new indications.
Our therapeutic pipeline consists of three unique clinical-stage drug candidates supported by an intellectual property portfolio of more than 79 granted and pending patents in various jurisdictions worldwide. These drug candidates aim to improve clinical outcomes in the treatment of rare and orphan diseases by applying proprietary formulation and drug delivery technologies to existing pharmaceutical compounds to achieve improvements over the current standard of care, or to provide treatment for diseases with no currently approved therapies.
The existing well understood efficacy and safety profiles of these marketed compounds provide the opportunity for us to utilize the Section 505(b)(2) regulatory pathway under the Federal Food, Drug and Cosmetic Act (“FDCA”) for the development of our reformulated versions of these drugs and therefore may provide a potentially shorter path to regulatory approval. Under Section 505(b)(2), if sufficient support of a product’s safety and efficacy either through previous FDA experience or sufficiently within the existing and accepted scientific literature, can be established, it may eliminate the need to conduct some of the pre-clinical studies and clinical trials that new drug candidates might otherwise require.
We believe rare disorders represent an attractive area for drug development, and there remains an opportunity for us to utilize already approved drugs that have established safety profiles and clinical experience to potentially address significant unmet medical needs. A key advantage of pursuing therapies for rare disorders is the potential to receive orphan drug designation (“ODD”) from the FDA. Our three drug candidates have received ODD status and, provided certain conditions are met at new drug application approval, those candidates, if approved, will be entitled to orphan drug exclusivity (“ODE”), which blocks FDA from approving for seven years any other application for a product that is the same drug for the same orphan indication, except in limited circumstances, such as a showing of clinical superiority to the product with ODE. ODD status can also result in tax credits of up to 25% of clinical development costs conducted in the United States upon marketing approval and a waiver of the NDA fees, which we estimate can translate into savings of approximately $4.3 million for our lead drug candidate, GTx-104. Developing drugs for rare diseases can often allow for clinical trials that are more manageably scaled and may require a smaller, more targeted commercial infrastructure.
The specific diseases targeted for drug development by us are well understood, although the patient populations suffering from such diseases may remain poorly served by available therapies or, in some cases, approved therapies do not yet exist. We aim to effectively treat debilitating symptoms that result from these underlying diseases.
Our management team possesses significant experience in drug formulation, drug delivery research and development, manufacturing, regulatory affairs, business development, as well as late-stage drug development and commercialization. Importantly, our team is comprised of industry professionals with deep expertise and knowledge, including a world-renowned practicing neurosurgeon-scientist and respected authority in aneurysmal subarachnoid hemorrhage, as well as product development, chemistry, manufacturing and controls (“CMC”), planning, implementation, management, and drug commercialization.
Our Pipeline
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GTx-104 is a clinical stage, novel, injectable formulation of nimodipine being developed for IV infusion in aSAH patients to address significant unmet medical needs. The unique nanoparticle technology of GTx-104 facilitates aqueous formulation of insoluble nimodipine for a standard peripheral IV infusion. GTx-104 provides a convenient IV delivery of nimodipine in the Intensive Care Unit potentially eliminating the need for nasogastric tube administration in unconscious or dysphagic patients. Intravenous delivery of GTx-104 also has the potential to lower food effects, drug-to-drug interactions, and eliminate potential dosing errors. Further, GTx-104 has the potential to better manage hypotension in aSAH patients. GTx-104 has been administered in over 200 patients and healthy volunteers and was well tolerated with significantly lower inter- and intra-subject pharmacokinetic variability compared to nimodipine oral capsules.
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GTx-102 is targeted for the treatment of ataxia-telangiectasia (“A-T”) in a pediatric population. A-T is caused by mutations in the ataxia telangiectasia mutated gene. Children with A-T experience cerebellar ataxia and other motor dysfunctions, oculomotor apraxia, dysarthria, and dysphagia. A Phase-1 pharmacokinetic study was successfully completed and GTx-102 was well tolerated with no serious events reported.
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GTx-101 is a topical bio adhesive film-forming bupivacaine spray for Postherpetic Neuralgia (“PHN”), which can be persistent and often causes debilitating pain following infection by the shingles virus. Four single-dose Phase 1 trials to evaluate the PK, safety, dose proportionality and tolerability of GTx-101 have been performed. In these trials, no serious adverse events were reported and GTx-101 was well tolerated. We believe that GTx-101 could be administered to patients with PHN to treat pain associated with the disease.
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In May 2023, we implemented a strategic realignment plan that resulted in engaging a new management team, streamlining our research and development activities, and greatly reducing our workforce. Following the realignment, we are concentrating on the development of our lead product candidate GTx-104. In June 2026, we decided to not resume
internal development funding for GTx-102 or GTx-101 under our current operating
plan.
GTx-104
GTx-104 is a clinical stage, novel, injectable formulation of nimodipine being developed for IV infusion in aSAH patients to address significant unmet medical needs. The unique nanoparticle technology of GTx-104 facilitates aqueous formulation of insoluble nimodipine for a standard peripheral IV infusion.
About aneurysmal Subarachnoid Hemorrhage (aSAH)
aSAH is bleeding over the surface of the brain in the subarachnoid space between the brain and the skull, which contains blood vessels that supply the brain. A primary cause of such bleeding is the rupture of an aneurysm in the brain. aSAH is characterized by high mortality (up to 25% early mortality) and significant potential of neurological decline (approximately 40%). The result is a relatively uncommon type of stroke that accounts for about 5% of all strokes and an estimated 42,500 U.S. hospital treated patients per year. Patients are typically hospitalized for two to four weeks following aSAH, with the most severe cases extending to a month or more. Due to the length of hospital stay and disproportionately high mortality and morbidity, aSAH has significant cost of care impact.
In contrast to more common types of ischemic stroke in elderly individuals, aSAH often occurs at a relatively young age, with approximately half the affected patients younger than 60 years old. Approximately 10% to 15% of aSAH patients die before reaching the hospital, and those who survive the initial hours post hemorrhage are admitted or transferred to tertiary care centers with high risk of complications, including rebleeding and systemic manifestations affecting cardiovascular, pulmonary, and renal function.
Unmet Needs with Oral Nimodipine
Nimodipine was granted FDA approval in 1988 and is the only approved drug that has been clinically shown to improve neurological outcomes in aSAH patients. It is only available in the United States as a generic oral capsule, generic oral liquid solution and as a branded oral liquid solution called NYMALIZE™, which is manufactured and sold by Arbor Pharmaceuticals (acquired in September 2021 by Azurity Pharmaceuticals). Nimodipine has poor water solubility and high permeability characteristics because of its high lipophilicity. Additionally, orally administered nimodipine has dose-limiting side-effects such as hypotension, poor absorption and low bioavailability resulting from high first-pass metabolism, and a narrow administration window as food effects lower bioavailability significantly. Due to these issues, blood levels of orally administered nimodipine can be highly variable, making it difficult to manage blood pressure in aSAH patients, often leading to frequent dose interruptions. Nimodipine capsules are also difficult to administer, particularly to unconscious patients or those with impaired ability to swallow, while the oral liquid solution has tolerability challenges due to solubility limitations of nimodipine.
GTx-104 Technology
Our lead drug candidate, GTx-104, is a novel injectable formulation of nimodipine for the treatment of aSAH. This formulation offers several potential advantages over oral administration of nimodipine that is the current Standard of Care (SoC) in the United States.
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Novel injectable formulation of nimodipine
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Overcomes solubility limitations of nimodipine
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A patented formulation that uses non-ionic surfactant micelles as the drug carrier to solubilize nimodipine
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Simple to prepare in a pharmacy and stable at room temperature
Value Proposition
GTx-104 provides a convenient IV delivery of nimodipine in the Intensive Care Unit potentially eliminating the need for nasogastric tube administration in unconscious or dysphagic patients. Intravenous delivery of GTx-104 also has the potential to lower food effects, drug-to-drug interactions, and eliminate potential dosing errors. Further, GTx-104 has the potential to better manage hypotension in aSAH patients. GTx-104 has been administered in over 200 patients and healthy volunteers and was well tolerated with significantly lower inter- and intra-subject pharmacokinetic variability compared to nimodipine oral capsules.
GTx-104 is designed to address significant unmet medical needs for patients with aSAH. We believe that with this novel nimodipine IV formulation may offer a potential value to physicians, hospitals, and their patients.
GTx-104 Market Opportunity
Approximately 42,500 patients in the United States are affected by aSAH per year. Company sponsored third party market research including claims analysis suggests that incidence may be as high as approximately 70,000 in the United States. Outside of the United States, annual cases of aSAH are estimated at approximately 60,000 in the European Union, and approximately 150,000 in China.
The unmet needs in the treatment of aSAH and the potential of GTx-104 to address the limitations of the current standard of care were the subject of a Key Opinion Leader event we hosted in November 2024. In an independent market research survey we conducted of hospital administrators and critical and neuro intensive care physicians at institutions with Comprehensive or Advanced Stroke Center certification who are involved in purchasing decisions for their institutions/units, respondents reported 80% likelihood of adopting an IV formulation of nimodipine, assuming 100% bioavailability, better safety, no food effects, effective hypotension management, potential hospital value and patient value.
Clinical Data
Pivotal Phase 3 STRIVE-ON Randomized Safety Trial
The STRIVE-ON trial was a prospective,
randomized open-label trial of GTx-104 compared with nimodipine oral capsules
in patients hospitalized with aSAH. 50 patients were administered GTx-104 and
52 patients received nimodipine oral capsules. The primary endpoint was the
number of patients with at least one episode of clinically significant
hypotension reasonably considered to be caused by the drug, and additional
endpoints included safety, clinical, and pharmacoeconomic outcomes. Each patient
was evaluated for up to 90 days inclusive of the 21-day treatment period. There
was a higher proportion of the most severe cases of aSAH (Hunt & Hess Grade
V) with the worst prognosis in the GTx-104 arm (8%) compared to the nimodipine
oral capsule arm (2%).
On September 25, 2024, we announced
the completion of enrollment in our Phase 3 STRIVE-ON trial for GTx-104. On
February 10, 2025, we announced the trial met its primary endpoint and provided
evidence of clinical benefit for GTx-104 compared to nimodipine oral capsules.
Patients receiving GTx-104 were observed to have a 19% reduction in at least
one incidence of clinically significant hypotension compared to nimodipine oral
capsules (28% versus 35%). Other measures also favored or were comparable to
GTx-104, including:
•
54% of patients who received GTx-104 had a relative dose intensity of 95% or higher of the prescribed dose compared to only 8% on nimodipine oral
capsules.
•
29% relative increase in the number of patients receiving GTx-104 compared to nimodipine oral
capsules with favorable outcomes at 90 days follow up on the modified Rankin scale. Quality of life as measured by EQ-5D-3L also favored patients receiving GTx-104 versus nimodipine oral
capsules.
•
Fewer intensive care unit (ICU) readmissions, ICU days, and ventilator days for patients receiving GTx-104 versus nimodipine oral
capsules.
•
Adverse events were comparable between the two arms and no new safety issues were identified with patients receiving GTx-104. All deaths in both arms of the trial were due to severity of the patient’s underlying disease. There were eight deaths on the GTx-104 arm compared to four deaths on the nimodipine oral capsule arm. The survival status of one patient on the nimodipine oral capsule arm was unknown. No deaths were determined to be related to GTx-104 or nimodipine oral capsules.
Furthermore, pharmacoeconomic measures favored the use of GTx-104 for patients with aSAH.
We believe these data validate the GTx-104 value proposition. If approved, GTx-104 has the potential to address significant challenges with oral nimodipine administration and may transform the standard of care for patients with aSAH.
In April 2026, an abstract
highlighting the results from our STRIVE-ON Phase 3 Safety Trial was presented
at the 2026 American Academy of Neurology (AAN) annual meeting in
Chicago, IL. The AAN is a leading voice in brain health, and the world’s
largest association of neurologists and neuroscience professionals with 44,000+
members. In September 2025, results from the STRIVE-ON trial were presented at
the 2025 Neurocritical Care Society annual meeting, held in Montreal,
Quebec, Canada. The Neurocritical Care Society is the only professional society
representing multi-disciplinary teams of neurocritical care providers around
the world whose mission is to improve outcomes for patients with
life-threatening neurological illnesses.
GTx-104 Phase 1 PK Trial
In September 2021, we initiated our pivotal pharmacokinetic (“PK”) bridging trial to evaluate the relative bioavailability of GTx-104 compared to currently marketed nimodipine oral capsules in approximately 50 healthy subjects. This PK trial established the 505(b)(2) regulatory pathway for GTx-104.
Final results from this pivotal PK
trial were reported in May 2022, and showed that the bioavailability of GTx-104
compared favorably with nimodipine oral capsules in all subjects, and no
serious adverse events were observed for GTx-104.
All endpoints indicated that
statistically there was no difference in exposures between GTx-104 and
nimodipine oral capsules over the defined time periods for both maximum
exposure and total exposure. Plasma concentrations obtained following IV
administration showed significantly less variability between subjects as
compared to nimodipine oral capsules because IV administration is not as
sensitive to some of the physiological processes that affect oral
administration, such as taking the drug with and without meals, variable
gastrointestinal transit time, variable drug uptake from the gastrointestinal
tract into the systemic circulation, and variable hepatic blood flow and
hepatic first pass metabolism. Previous studies have shown these processes
significantly affect the oral bioavailability of nimodipine, and therefore
cause oral administration to be prone to larger inter- and intra-subject
variability.
The bioavailability of nimodipine oral
capsules observed was only approximately 7% compared to 100% for GTx-104.
Consequently, about one-twelfth of the amount of nimodipine is delivered with
GTx-104 to achieve comparable PKs as with nimodipine oral capsules. This data
is presented in the chart below.
Regulatory
In April 2025, we announced details of a Type C written meeting response with the FDA. The purpose of this meeting was to obtain feedback on the completed Phase 3 STRIVE-ON safety trial of GTx-104 and our planned NDA submission, including clinical, non-clinical, and CMC requirements.
In June 2025, we submitted to the FDA
an NDA for GTx-104 for the treatment of aSAH, which was accepted for review by
the FDA in August 2025 with an April 23, 2026, Prescription Drug User Fee Act
(“PDUFA”) target date for completing FDA’s review of our submission. The NDA
included clinical results from our STRIVE-ON trial for GTx-104.
In
April 2026, we announced the FDA had issued a CRL in response to our NDA
submission for GTx-104. In the CRL, the FDA referenced certain items in the CMC
and non-clinical sections of the application, which we believe can be addressed
in a resubmission of the NDA. The cited items relate to additional leachable
data time points for commercial product, non-clinical product toxicology risk
assessments, and cGMP deficiencies at our CMO. The CRL did not raise any issues
with respect to the safety or efficacy of GTx-104. A Type A meeting with the
FDA has been scheduled to potentially clarify the path forward and determine
the appropriate next steps. We expect to provide a regulatory update after the
receipt of official meeting minutes.
GTx-102
GTx-102 is a novel, concentrated oral-mucosal spray of betamethasone intended to improve neurological symptoms of A-T for which there are currently no FDA-approved therapies. GTx-102 is a stable, concentrated oral spray formulation comprised of the gluco-corticosteroid betamethasone that, together with other excipients, can be sprayed conveniently over the tongue of the A-T patient and is rapidly absorbed.
We have licensed the data from the multicenter, double-blinded, randomized, placebo-controlled crossover trial from Azienda Ospedaliera Universitaria Senese, Siena, Italy, where Dr. Zannolli et. al. studied the effect of oral liquid solution of betamethasone to reduce ataxia symptoms in patients with A-T. This oral liquid solution is not marketed in the United States, and therefore is not available for clinical use. Currently, betamethasone is only available in the United States as an injectable or as a topical cream. This license gives us the right to reference the trial’s data in our NDA filing. On November 12, 2015, we submitted the data from the Zannolli trial to the FDA’s Division of Neurology at a pre-Investigational New Drug (“IND”) meeting and received guidance from the agency on the regulatory requirements to seek approval.
About Ataxia Telangiectasia
A-T is a rare genetic progressive autosomal recessive neurodegenerative disorder that affects children, with the hallmark symptoms of cerebellar ataxia and other motor dysfunction, and dilated blood vessels (telangiectasia) that occur in the sclera of the eyes. A-T is caused by mutations in the ataxia telangiectasia gene, which is responsible for modulating cellular response to stress, including breaks in the double strands of DNA.
Children with A-T begin to experience balance and coordination problems when they begin to walk (toddler age), and ultimately become wheelchair-bound in their second decade of life. In pre-adolescence (between ages 5 and 8), patients experience oculomotor apraxia, dysarthria, and dysphagia. They also often develop compromised immune systems and are at increased risk of developing respiratory tract infections and cancer (typically lymphomas and leukemia).
Unmet Needs
There is no known treatment to slow disease progression, and treatments that are used are strictly aimed at controlling the symptoms (or conditions secondary to the disease. There are no FDA-approved therapeutic options currently available. Patients typically die by age 25 from complications of lung disease or cancer.
Market Opportunity
According to a third-party report we commissioned in 2018, A-T affects approximately 4,300 patients per year in the United States and has a potential total addressable market of $150 million, based on the number of treatable patients in the United States.
Clinical Data
In a multicenter, double-blind, randomized, placebo-controlled crossover trial conducted in Italy, Dr. Zannolli et al. studied the effect of an oral liquid solution of betamethasone on the reduction of ataxia symptoms in 13 children (between ages 2 to 8 years) with A-T. The primary outcome measure was the reduction in ataxia symptoms as assessed by the International Cooperative Ataxia Rating Scale (“ICARS”).
In the trial, oral liquid betamethasone reduced the ICARS total score by a median of 13 points in the intent-to-treat population and 16 points in the per-protocol population (the median percent decreases of ataxia symptoms of 28% and 31%, respectively). Adverse events in the trial were minimal, with no compulsory withdrawals and only minor side effects that did not require medical intervention. Clinical trial results in A-T patients administered oral betamethasone indicated that betamethasone significantly reduced ICARS total score relative to placebo (P = 0.01). The median ICARS change score (change in score with betamethasone minus change in score with placebo) was -13 points (95% confidence interval for the difference in medians was -19 to -5.5 points).
Based on the Zannolli data, we believe that our GTx-102 concentrated oral spray has the potential to provide clinical benefits in decreasing A-T symptoms, including assessments of posture and gait disturbance and kinetic, speech and oculomotor functions. In addition, GTx-102 may ease drug administration for patients experiencing A-T given its application of 1-3x/day of 140µL of concentrated betamethasone liquid sprayed onto the tongue using a more convenient metered dose delivery system, as these A-T patients typically have difficulty swallowing.
Pharmacokinetic Data
GTx-102 administered as a concentrated oral spray achieves similar blood levels at only 1/70th the volume of an oral solution of betamethasone. This more convenient mode of administration will be important for A-T patients who have difficulties swallowing large volumes of liquids.
We initiated a PK bridging trial of GTx-102 as compared to the oral liquid solution of betamethasone used in the Zannolli trial and against the injectable form of betamethasone that is approved in the U.S. in the third calendar quarter of 2022. The primary objectives of the PK bridging trial were to evaluate the bioavailability, pharmacokinetics, and safety of GTx-102. In December 2022, we reported that the topline results of this trial met all primary outcome measures.
GTx-102 update
As previously disclosed, in May 2023 we
made a strategic decision to defer clinical development of GTx-102 to
prioritize resources toward GTx-104 and its advancement toward an NDA filing.
In June 2026, following continued
evaluation of strategic priorities and focus on GTx-104, we determined we will
not resume internal development funding for GTx-102 under our current operating
plan. Accordingly, we determined that the remaining carrying value of the GTx-102
IPR&D asset is no longer recoverable on an internal-development basis.
As of March 31, 2026, the remaining
capitalized carrying value of GTx-102 was $9.2 million. We expect to recognize an impairment charge for the
full remaining carrying value in the first quarter of fiscal year 2027.
Consistent with our stated position
since 2023, we retain all intellectual property and patent rights associated
with GTx-102 and continue to evaluate business development opportunities,
including out-licensing, partnerships, or non-dilutive arrangements, that would
enable advancement of the program without requiring our direct capital
investment. We continue to believe GTx-102 may provide significant value
through such a transaction. There can be no assurance that any such transaction
will be completed on favorable terms or at all.
GTx-101
GTx-101 is a non-narcotic, topical bio-adhesive film-forming bupivacaine spray designed to ease the symptoms of patients suffering with PHN. GTx-101 is administered via a metered-dose of bupivacaine spray and forms a thin bio-adhesive topical film on the surface of the patient’s skin, which enables a touch-free, non-greasy application. It also comes in convenient, portable 30 ml plastic bottles. Unlike oral gabapentin and lidocaine patches which are used for the treatment of PHN, we believe that the biphasic delivery mechanism of GTx-101 has the potential for rapid onset of action and continuous pain relief for up to eight hours. No skin sensitivity was reported in a Phase 1 trial.
About Postherpetic Neuralgia (PHN)
PHN is neuropathic pain due to damage caused by the varicella zoster virus (“VZV”). Infection with VZV causes two distinct clinical conditions. Primary VZV infection causes varicella (i.e., chickenpox), a contagious rash illness that typically occurs among young children. Secondary VZV can reactivate clinically, decades after initial infection, to cause herpes zoster (“HZ”), otherwise known as shingles. Acute HZ arises when dormant virus particles, persisting within an affected sensory ganglion from the earlier, primary infection with VZV become reactivated when cellular immunity to varicella decreases. Viral particles replicate and may spread to the dorsal root, into the dorsal horn of the spinal cord, and through peripheral sensory nerve fibers down to the level of the skin. Viral particles also may circulate in the blood. This reactivation is accompanied by inflammation of the skin, immune response, hemorrhage, and destruction of peripheral and central neurons and their fibers. Following such neural degeneration, distinct types of pathophysiological mechanisms involving both the central and peripheral nervous systems may give rise to the severe nerve pain associated with PHN. While the rash associated with HZ typically heals within two to four weeks, the pain may persist for months or even years, and this PHN manifestation is the most common and debilitating complication of HZ. There is currently no consensus definition for PHN, but it has been suggested by the Centers for Disease Control and Prevention that PHN is best defined as pain lasting at least three months after resolution of the rash.
Unmet Need
PHN is associated with significant loss of function and reduced quality of life, particularly in the elderly. It has a detrimental effect on all aspects of a patient’s quality of life. The nature of PHN pain varies from mild to severe, constant, intermittent, or triggered by trivial stimuli. Approximately half of patients with PHN describe their pain as “horrible” or “excruciating,” ranging in duration from a few minutes to constant on a daily or almost daily basis. Pain can disrupt sleep, mood, work, and activities of daily living, adversely impacting the quality of life and leading to social withdrawal and depression. PHN is the foremost cause of intractable, debilitating pain in the elderly, and has been cited as the leading cause of suicide in chronic pain patients over the age of 70.
Current treatment of PHN most often consists of oral gabapentin (first line) and prescription lidocaine patches or antidepressants (second line), and refractory cases may be prescribed opioids to address persistent pain. Gabapentin and opioid abuse have continued to proliferate, and lidocaine patches are suboptimal for many reasons. An independent third-party market research firm we commissioned interviewed more than 250 physicians who regularly treat PHN patients and found that approximately 40% of patients using lidocaine patches experience insufficient pain relief. Lidocaine patches are difficult to use, fall off, and look unsightly with possible skin sensitivity and irritation. Additionally, lidocaine patches can only be used for 12 hours and then need to be removed for 12 hours before being reapplied. Prescription lidocaine patches are only approved for PHN, and the market is currently made up of both branded and generic offerings.
Market Potential
It is estimated that PHN affects approximately 120,000 patients per year in the United States. According to a third-party report, the total addressable market for GTx-101 could be as large as $2.5 billion, consisting of approximately $200 million for PHN pain and $2.3 billion for non-PHN pain indications.
Clinical Data
To date, we have conducted four Phase I trials in healthy volunteers to assess the PK, safety, and tolerability of GTx-101 and to determine the plasma levels of bupivacaine hydrogen chloride administered as a single dose in various concentrations between 30 mg (three sprays) and 2100 mg (twenty sprays).
These trials confirmed that GTx-101 is well absorbed through the skin, as demonstrated in the graph below, while very little is absorbed systemically.
In all four trials, the administration of GTx-101 to healthy volunteers was safe and well tolerated. In addition, no evidence of skin irritation was observed at the application site following the spray administrations. The data below is from two separate trials superimposed on each other – one trial of GTx-101 and one trial of the Lidoderm patch.
Regulatory
The data from the single dose Phase 1 clinical trial for GTx-101 was submitted to the FDA’s Division of Anesthesiology and feedback was received at a pre-IND meeting that informed the design of pre-clinical toxicology studies and a clinical and regulatory pathway to approval under section 505(b)(2). We completed a minipig skin sensitivity study in the second calendar quarter of 2022, and we initiated a single dose PK trial in healthy human volunteers in July 2022. Topline results from this single dose PK trial were reported in December 2022, and the results met all primary outcome measures.
GTx-101 update
As previously disclosed, in May 2023 we made a strategic decision to defer clinical development of GTx-101 to prioritize resources toward GTx-104 and our advancement toward an NDA filing.
In June 2026, following
continued evaluation of strategic priorities and focus on GTx-104, we
determined we will not resume internal development funding for GTx-101 under our
current operating plan. Accordingly, we determined that the remaining carrying
value of the GTx-101 IPR&D asset is no longer recoverable on an
internal-development basis.
As of March 31, 2026, the
remaining capitalized carrying value of GTx-101 was $4.3 million. We expect to
recognize an impairment charge for the full remaining carrying value in the
first quarter of fiscal year 2027.
Consistent with our stated
position since 2023, we retain all intellectual property and patent rights
associated with GTx-101 and continues to evaluate business development
opportunities, including out-licensing, partnerships, or non-dilutive
arrangements, that would enable advancement of the program without requiring our
direct capital investment. We continue to believe GTx-101 may provide
significant value through such a transaction. There can be no assurance that
any such transaction will be completed on favorable terms or at all.
Commercialization Strategy
We
have worldwide commercialization rights for all our pipeline drug candidates
and plan to maximize the value of each of our drug candidates over time. If we
receive regulatory approval for GTx-104 in the U.S., we plan to commercialize
GTx-104 with a highly experienced and targeted hospital-based sales force. We
may further seek commercial partnerships to fully exploit the market potential
of GTx-104 in territories outside the U.S. We do not plan to resume internal
development funding for GTx-102 or GTx-101 under our current operating plan. It
is possible that we out-license or sell GTx-102 and/or GTx-101 for the U.S.
and/or global markets.
Manufacturing and Supply
We currently do not own any manufacturing facilities. The manufacture of our pipeline of drug candidates is highly reliant on complex techniques and personnel aseptic techniques, which present significant challenges and require specialized expertise. Further, these processes undergo a high level of scrutiny by regulatory agencies. Consequently, we utilize a network of third-party CMOs for manufacturing our drug candidates. All CMOs are monitored and evaluated by us to assess compliance with regulatory requirements.
We work with independent consultants
to perform periodic quality audits of our manufacturers to review the
manufacturing process for our drug candidates and to provide input on quality
issues. All lots of the drug substance and drug product used in clinical supply
are manufactured under current good manufacturing practices. We plan to
continue to rely upon CMOs to manufacture clinical and commercial quantities if
a product is approved. We have development agreements in place with these CMOs
and we have personnel with pharmaceutical development and manufacturing
experience who are responsible for the relationships with our CMOs. In April
2026, we received a CRL from the FDA in response to our NDA for GTx‑104
identifying certain cGMP deficiencies at our current CMO. Our ability to obtain
regulatory approval for GTx‑104 is dependent, in significant part, on our CMO’s
ability to timely and satisfactorily remediate the deficiencies identified in
the CRL and demonstrate ongoing compliance with applicable regulatory
requirements, including cGMP. For additional information regarding risks
related to our use of third-party CMOs, please see the risk factors captioned “If
our current CMO is unable to timely resolve the deficiencies identified by the
FDA in its CRL for GTx‑104, we may be required to engage an alternative
replacement manufacturer, which could result in significant delays, increased
costs, or our inability to obtain regulatory approval or commercialize GTx‑104
at all.” and “We rely on third parties to manufacture commercial and
clinical supplies of our drug candidates, and we intend to rely on third
parties to manufacture commercial supplies of any approved drug products. The
commercialization of any of our drug products could be stopped, delayed, or
made less profitable if those third parties fail to provide us with sufficient
quantities of active pharmaceutical ingredients, excipients, or drug products,
or fail to do so at acceptable quality levels or prices or fail to maintain or
achieve satisfactory regulatory compliance.”
Intellectual Property Portfolio
We have a multi-layered intellectual
property protection strategy, which we believe will create barriers to entry
and solidify our position in the market. All of our clinical pipeline drug
candidates have received orphan status designation from the FDA, which could
result in 7 years of marketing exclusivity in the United States. Orphan status
in Europe could result in 10 years of final marketing exclusivity. Such
marketing exclusivity is dependent on the candidates receiving final marketing
authorizations from the applicable government agencies, and the conditions for
receiving such marketing exclusivity. In addition, we protect our drug
candidates through a well-defined patent filing strategy. Our patent estate
includes 79 granted and pending applications in various global jurisdictions,
including 10 U.S. issued patents and 3 pending U.S. patent applications. We
believe that our intellectual property portfolio, consisting primarily of
composition and method-of-use patents, will protect the market value of our
products by extending exclusivity beyond what is granted through the orphan
designation. We intend to continue to build our patent portfolio by filing for
patent protection on new developments with respect to our product candidates.
We expect that these patents will, if and when issued, will be listable in the
US FDA Orange Book: Approved Drug Products with Therapeutic Equivalence, to
which potential competitors will be required to certify upon submission of
their applications referencing our drug products, if approved.
We strive to protect and enhance the proprietary technology, inventions, and improvements that are commercially important to the development of our business, including seeking, maintaining, and defending patent rights, whether developed internally or licensed from third parties. We also rely on trade secrets relating to manufacturing know-how, continuing technological innovation and in-licensing opportunities to develop, strengthen, and maintain our proprietary position. We may also rely on regulatory protections afforded through orphan drug status, data exclusivity, market exclusivity, and patent term extensions, where available.
We are actively seeking U.S. and international patent protection for a variety of technologies and intend to seek patent protection or rely upon trade secret rights to protect other technologies that may be used to discover and validate targets and that may be used to identify and develop novel pharmaceutical products. We seek these protections, in part, through confidentiality and proprietary information agreements.
Individual patents extend for varying periods depending on the date of filing or the date of issuance, and the legal term of patents in the countries in which they are obtained. Generally, utility patents issued for applications filed in the United States are granted a term of 20 years from the earliest effective filing date of a non-provisional patent application. In addition, in certain instances, a patent term can be extended to recapture a portion of the U.S. Patent and Trademark Office delay in issuing the patent as well as a portion of the term effectively lost as a result of the FDA regulatory review period. However, as to the FDA component, the restoration period cannot be longer than 5 years and the total patent term including the restoration period must not exceed 14 years following FDA approval. The duration of foreign patents varies in accordance with provisions of applicable local law but typically is also 20 years from the earliest effective filing date. The actual protection afforded by a patent may vary on a product-by-product basis from country to country and can depend upon many factors, including the type of patent, the scope of its coverage, the availability of regulatory-related extensions, the availability of legal remedies in a particular country and the validity and enforceability of the patent.
We have numerous issued U.S. patents
and pending U.S. patent applications, as well as issued patents and patenting
patent applications in other jurisdictions. Eight patents for GTx-104 have been
granted in the United States. One patent for GTx-104 has been granted in each
of Europe, Australia, Canada, United Kingdom, India, and Japan. One patent for
GTx-101 has been granted in each of Japan, Europe, Australia, Canada, China,
Mexico, United Kingdom, and India. One patent for GTx-101 has been granted in Europe,
China, Mexico, Japan and South Africa. One patent for GTx-102 has been granted
in each of the United States, Japan, Australia and Canada. One patent for
GTx-201 has been granted in each of the United States, Japan and Korea.
Government Regulation
We are subject to extensive regulation by the FDA and other federal, state, and local regulatory agencies. The FDCA and the FDA's implementing regulations set forth, among other things, requirements for the testing, development, manufacture, quality control, safety, effectiveness, approval, labeling, storage, record-keeping, reporting, distribution, import, export, sale, advertising and promotion of our product candidates. Although we focus primarily on regulation in the U.S., because that is currently our primary focus, we may seek approval for, and market, our products candidates in other countries in the future. Generally, our activities in other countries will be subject to regulation that is similar in nature and scope as that imposed in the U.S., although there can be important differences.
Development and Approval
Under the FDCA, FDA approval of an NDA is required before any new drug can be marketed in the U.S. NDAs require extensive studies and submission of a large amount of data by the applicant.
Preclinical Testing. Before testing any compound in human patients in the U.S., a company must generate extensive preclinical data. Preclinical testing generally includes laboratory evaluation of product chemistry and formulation, as well as toxicological and pharmacological studies in several animal species to assess the toxicity and dosing of the product. Certain animal studies must be performed in compliance with the FDA's Good Laboratory Practice (“GLP”) regulations and the U.S. Department of Agriculture's Animal Welfare Act.
IND Application. Human clinical trials in the U.S. cannot commence until an IND application is submitted and becomes effective. A company must submit preclinical testing results to the FDA as part of the IND, and the FDA must evaluate whether there is an adequate basis for testing the drug in initial clinical studies in human volunteers. Unless the FDA raises concerns, the IND becomes effective 30 days following its receipt by the FDA, and the clinical trial proposed in the IND may begin. Once human clinical trials have commenced, the FDA may temporarily or permanently stop a clinical trial by placing it on "clinical hold" at any time because of concerns about the safety of the product being tested, or for other reasons.
Clinical Trials. Clinical trials involve the administration of a drug to healthy human volunteers or to patients, under the supervision of a qualified investigator. The conduct of clinical trials is subject to extensive regulation, including compliance with the FDA's bioresearch monitoring regulations and current Good Clinical Practice (“cGCP”) requirements, which establish standards for conducting, recording data from, and reporting the results of, clinical trials, and are intended to assure that the data and reported results are credible and accurate, and that the rights, safety, and well-being of study participants are protected. Clinical trials must be conducted under protocols that detail the study objectives, parameters for monitoring safety, and the efficacy criteria, if any, to be evaluated. Each protocol is reviewed by the FDA as part of the IND. In addition, each clinical trial must be reviewed and approved by, and conducted under the auspices of, an Institutional Review Board (“IRB”) for each clinical site. Companies sponsoring the clinical trials, investigators, and IRBs also must comply with, as applicable, regulations and guidelines for obtaining informed consent from the study patients, following the protocol and investigational plan, adequately monitoring the clinical trial, and timely reporting of adverse events. The FDA enforces good clinical practices through periodic inspections of trial sponsors, clinical investigators, and trial sites. Foreign studies conducted under an IND must meet the same requirements that apply to studies being conducted in the U.S. Data from a foreign study not conducted under an IND may be submitted in support of an NDA if the study was conducted in accordance with cGCP and the FDA is able to validate the data.
A study sponsor is required to publicly post specified details about certain clinical trials and clinical trial results on government or independent websites (e.g., https://clinicaltrials.gov). Human clinical trials typically are conducted in three sequential phases, although the phases may overlap, be combined, or be subdivided in some cases:
•
Phase 1 clinical trials involve the initial administration of the investigational drug to humans, typically to a small group of healthy human subjects, but occasionally to a group of patients with the targeted disease or disorder. Phase 1 clinical trials generally are intended to evaluate the safety, metabolism and pharmacologic actions of the drug, the side effects associated with increasing doses, and, if possible, to gain early evidence of effectiveness.
•
Phase 2 clinical trials generally are controlled studies that involve a relatively small sample of the intended patient population and are designed to develop initial data regarding the product's effectiveness, to determine dose response and the optimal dose range, and to gather additional information relating to safety and potential adverse events.
•
Phase 3 clinical trials are conducted after preliminary evidence of effectiveness has been obtained and are intended to gather the additional information about safety and effectiveness necessary to evaluate the drug's overall risk-benefit profile, and to provide a basis for physician labeling. Generally, Phase 3 clinical development programs consist of expanded, multi-site, large-scale studies of patients with the target disease or disorder to obtain statistical evidence of the efficacy and safety of the drug at the proposed dosing regimen. Phase 3 data often form the core basis on which the FDA evaluates a drug’s safety and effectiveness when considering the product application.
The sponsoring company, the FDA, or the IRB may suspend or terminate a clinical trial at any time on various grounds, including a finding that the patients are being exposed to an unacceptable health risk. Further, success in early-stage clinical trials does not assure success in later-stage clinical trials. Data obtained from clinical activities are not always conclusive and may be subject to alternative interpretations that could delay, limit or prevent regulatory approval.
NDA Submission and Review. The FDCA provides two pathways for the approval of new drugs through an NDA. An NDA under Section 505(b)(1) of the FDCA is a comprehensive application to support approval of a product candidate that includes, among other things, data and information to demonstrate that the proposed drug is safe and effective for its proposed uses, that manufacturing methods and controls are adequate to ensure its identity, strength, quality, and purity of the drug, and that proposed labeling is appropriate and contains all necessary information. A 505(b)(1) NDA contains results of the full set of preclinical studies and clinical trials conducted by or on behalf of the applicant to characterize and evaluate the product candidate including negative or ambiguous results as well as positive findings.
Section 505(b)(2) of the FDCA provides an alternate regulatory pathway to obtain FDA approval that permits the filing of an NDA where at least some of the information required for approval comes from studies not conducted by or for the applicant and for which the applicant has not obtained a right of reference. The applicant may rely to some extent upon the FDA's findings of safety and effectiveness for an approved product that acts as the reference drug and submit its own product-specific data — which may include data from preclinical studies or clinical trials conducted by or on behalf of the applicant — to address differences between the product candidate and the reference drug. We are pursuing the Section 505(b)(2) regulatory approval pathway for GTx-104, with NIMOTOP oral capsules as the reference drug.
The submission of an NDA under either Section 505(b)(1) or Section 505(b)(2) generally requires payment of a substantial user fee to the FDA. However, such fees can be waived by the FDA for orphan drugs such as GTx-104.
When an NDA is submitted, the FDA makes an initial determination as to whether the application is sufficiently complete to be accepted for review. The FDA may refuse to file an application and/or request additional information before acceptance. A refusal to file, which requires resubmission of the NDA with the requested additional information, delays review of the application. Once accepted for filing, FDA begins an in-depth review of the application. The FDA reviews applications to determine, among other things, whether a product is safe and effective for its intended use and whether the manufacturing controls are adequate to assure and preserve the product's identity, strength, quality, and purity. For some NDAs, the FDA may convene an advisory committee to seek insights and recommendations on issues relevant to approval of the application. Although the FDA is not bound by the recommendation of an advisory committee, the agency considers such recommendations carefully when making decisions.
The FDA may determine that a Risk Evaluation and Mitigation Strategy (“REMS”) is necessary to ensure that the benefits of a new product outweigh its risks, and the product can therefore be approved. A REMS may include various elements, ranging from a medication guide or patient package insert to limitations on who may prescribe or dispense the drug, depending on what the FDA considers necessary for the safe use of the drug. Under the Pediatric Research Equity Act, certain applications for approval must also include an assessment, generally based on clinical study data, of the safety and effectiveness of the subject drug in relevant pediatric populations. Before approving an NDA, the FDA will inspect the facility or facilities where the product is manufactured. The FDA will not approve an application unless it determines that the manufacturing processes and facilities are compliant with cGMP requirements and adequate to assure consistent production of the product within required specifications.
Once the FDA accepts an NDA submission which occurs, if at all, within 60 days after submission of the NDA –– under the current goals and policies agreed to by the FDA under the Prescription Drug User Fee Act (“PDUFA”), the FDA's standard goal for a review of an NDA is ten months from the filing decision, or six months from the filing decision for a priority application. The FDA does not always meet its PDUFA goal dates, and in certain circumstances, the PDUFA goal date may be extended by FDA requests for additional information, studies, or clarification. After reviewing an NDA and the facilities where the product is manufactured, the FDA either issues an approval letter or a complete response letter (“CRL”) outlining the deficiencies in the submission. The CRL may require additional testing or information, including additional preclinical or clinical data. Even if such additional information and data are submitted, the FDA may decide the NDA still does not meet the standards for approval. Data from clinical trials are not always conclusive and the FDA may interpret data differently than the sponsor. As discussed elsewhere in this Annual Report on Form 10-K, on April 23, 2026, we received a CRL in response to our NDA for GTx-104 referencing certain items in the chemistry, manufacturing, and controls and non-clinical sections of the application, which we believe we can address in a potential resubmission of the NDA. If, and when, those deficiencies have been addressed to the FDA’s satisfaction in a potential resubmission of an NDA, the FDA may issue an approval letter.
Obtaining regulatory approval may take several years, involves the expenditure of substantial resources, and depends on several factors, including the severity of the disease in question, the availability of alternative treatments, and the risks and benefits demonstrated in clinical trials. Additionally, as a condition of approval, the FDA may impose restrictions that could affect the commercial success of a drug or impose post-approval commitments and requirements, including the completion within a specified time of additional clinical studies or trials, which often are referred to as "Phase 4" or "post-marketing" studies.
Post-approval modifications to the drug, such as changes in indications, labeling, or manufacturing processes or facilities, may require a sponsor to develop additional data or conduct additional preclinical studies or clinical trials, to be submitted in a new or supplemental NDA, which would require FDA approval.
Post-Approval Regulation
Once approved, drug products are subject to continuing regulation by the FDA. If ongoing regulatory requirements are not met or if safety or manufacturing problems occur after the product reaches the market, the FDA may at any time withdraw product approval or take actions that would limit or suspend marketing. Additionally, the FDA may require post-marketing studies or clinical trials, changes to a product’s approved labeling, including the addition of new warnings and contraindications, or the implementation of other risk management measures, including distribution-related restrictions, if there are new safety information developments.
Good Manufacturing Practices. Companies engaged in manufacturing drug products, or their components, must comply with applicable cGMP requirements and product-specific regulations enforced by the FDA and other regulatory agencies. Compliance with cGMP includes adhering to requirements relating to organization and training of personnel, buildings and facilities, equipment, control of components and drug product containers and closures, production and process controls, quality control and quality assurance, packaging and labeling controls, holding and distribution, laboratory controls, and records and reports. The FDA regulates and inspects equipment, facilities, and processes used in manufacturing pharmaceutical products prior to approval. If, after receiving approval, a company makes a material change in manufacturing equipment, location, or process (all of which are, to some degree, incorporated in the NDA), additional regulatory review and approval may be required. The FDA also conducts regular, periodic visits to re-inspect equipment, facilities, and processes following the initial approval of a product. Failure to comply with applicable cGMP requirements and conditions of product approval may lead the FDA to take enforcement action or seek sanctions, including fines, issuance of warning letters, civil penalties, injunctions, suspension of manufacturing operations, operating restrictions, withdrawal of FDA approval, seizure or recall of products, and criminal prosecution. Although we periodically monitor the FDA compliance of our third-party manufacturers, we cannot be certain that our present or future third-party manufacturers will consistently comply with cGMP and other applicable FDA regulatory requirements.
Advertising and Promotion. The FDA and other federal regulatory agencies closely regulate the marketing and promotion of drugs through, among other things, standards and regulations for direct-to-consumer advertising, advertising and promotion to healthcare professionals, communications regarding unapproved uses, industry-sponsored scientific and educational activities, and promotional activities involving the Internet. A product cannot be commercially promoted before it is approved. After approval, product promotion can include only those claims relating to safety and effectiveness that are consistent with the labeling approved by the FDA. Healthcare providers are permitted to prescribe drugs for "off-label" uses –– that is, uses not approved by the FDA and not described in the product's labeling –– because the FDA does not regulate the practice of medicine. However, FDA regulations impose restrictions on manufacturers' communications regarding off-label uses. Broadly speaking, a manufacturer may not promote a drug for off-label use, but under certain conditions may engage in non-promotional, balanced, scientific communication regarding off-label use. In addition to FDA restrictions on the marketing of pharmaceutical products, state and federal fraud and abuse laws have been applied to restrict certain marketing practices in the pharmaceutical industry. Failure to comply with applicable FDA requirements and restrictions in this area may subject a company to adverse publicity and enforcement action by the FDA, the Department of Justice, or the Office of the Inspector General of the Department of Health and Human Services (“HHS”), as well as state authorities. This could subject a company to a range of penalties that could have a significant commercial impact, including civil and criminal fines and agreements that materially restrict the way a company promotes or distributes a drug.
Other Requirements. NDA holders must comply with other regulatory requirements, including submitting annual reports, reporting information about adverse drug experiences, and maintaining certain records.
Additionally, on December 20, 2019, the Further Consolidated Appropriations Act for 2020 was signed into law, which includes bipartisan legislation called the Creating and Restoring Equal Access to Equivalent Samples Act of 2019 or the “CREATES Act.” The CREATES Act aims to address the concern articulated by both the FDA and others in the industry that some brand manufacturers have improperly restricted the distribution of their products, including by invoking the existence of a REMS for certain products to deny generic product developers access to samples of brand products. Because generic product developers need samples to conduct certain comparative testing required by the FDA, some have attributed the inability to timely obtain samples as a cause of delay in the entry of generic products. To remedy this concern, the CREATES Act establishes a private cause of action that permits a generic product developer to sue the brand manufacturer to compel it to furnish the necessary samples on “commercially reasonable, market-based terms.”
Hatch-Waxman Act
The Drug Price Competition and Patent Term Restoration Act of 1984 (the “Hatch-Waxman Act”) establishes two abbreviated approval pathways for pharmaceutical products that are in some way follow-on versions of already approved products.
Generic Drugs. A generic version of an approved drug is approved by means of abbreviated NDA (“ANDA”), by which the sponsor demonstrates that the proposed product is the same as the approved, brand-name drug, which is referred to as the reference listed drug (“RLD”). Generally, an ANDA must contain data and information showing that the proposed generic product and RLD (i) have the same active ingredient, in the same strength and dosage form, to be delivered via the same route of administration, (ii) are intended for the same uses, and (iii) are bioequivalent. This is instead of independently demonstrating the proposed product's safety and effectiveness, which are inferred from the fact that the product is the same as the RLD, which the FDA previously found to be safe and effective.
505(b)(2) NDAs. As discussed above, if a product is similar, but not identical, to an already approved product, it may be submitted for approval via an NDA under section 505(b)(2) of the FDCA. Unlike an ANDA, this does not excuse the sponsor from demonstrating the proposed product's safety and effectiveness. Rather, the sponsor is permitted to rely to some degree on information from investigations that were not conducted by or for the applicant and for which the applicant has not obtained a right of reference and must submit its own product-specific data of safety and effectiveness to an extent necessary because of the differences between the products. An NDA approved under 505(b)(2) may in turn serve as an RLD for subsequent applications from other sponsors.
RLD Patents. In an NDA, a sponsor must identify patents that claim the drug substance or drug product or a method of using the drug. When the drug is approved, those patents are among the information about the product that is listed in the FDA publication, “Approved Drug Products with Therapeutic Equivalence Evaluations,” which is referred to as the Orange Book. The sponsor of an ANDA or 505(b)(2) application seeking to rely on an approved product as the RLD must make one of several certifications regarding each listed patent. A “Paragraph I” certification is the sponsor’s statement that patent information has not been filed for the RLD. A “Paragraph II” certification is the sponsor’s statement that the RLD’s patents have expired. A "Paragraph III" certification is the sponsor's statement that it will wait for the patent to expire before obtaining approval for its product. A "Paragraph IV" certification is an assertion that the patent does not block approval of the later product, either because the patent is invalid or unenforceable or because the patent, even if valid, is not infringed by the new product.
Regulatory Exclusivities. The Hatch-Waxman Act provides periods of regulatory exclusivity for products that would serve as RLDs for an ANDA or 505(b)(2) application. If a product is a "new chemical entity," or “NCE” — generally meaning that the active moiety has never before been approved in any drug — there is a period of five years from the product's approval during which the FDA may not accept for filing any ANDA or 505(b)(2) application for a drug with the same active moiety. An ANDA or 505(b)(2) application may be submitted after four years, however, if the sponsor of the application makes a Paragraph IV certification.
A product that is not an NCE (i.e., the active moiety has been previously approved) may qualify for a three-year period of exclusivity if the NDA, including a 505(b)(2), application contains new clinical data — other than bioavailability studies — derived from studies conducted by or for the sponsor that were necessary for approval. In that instance, the exclusivity period does not preclude filing or review of an ANDA or 505(b)(2) application; rather, the FDA is precluded from granting final approval to the ANDA or 505(b)(2) application until three years after approval of the RLD. Additionally, the exclusivity applies only to the conditions of approval that require submission of the clinical data.
Once the FDA accepts for filing an ANDA or 505(b)(2) application containing a Paragraph IV certification, the applicant must within 20 days provide notice to the RLD or listed drug NDA holder and patent owner that the application has been submitted and provide the factual and legal basis for the applicant's assertion that the patent is invalid or not infringed. If the NDA holder or patent owner files suit against the ANDA or 505(b)(2) applicant for patent infringement within 45 days of receiving the Paragraph IV notice, the FDA is prohibited from approving the ANDA or 505(b)(2) application for a period of 30 months or the resolution of the underlying suit, whichever is earlier. If the RLD has NCE exclusivity and the notice is given and suit filed during the fifth year of exclusivity, the regulatory stay extends until 7.5 years after the RLD approval. The FDA may approve the proposed product before the expiration of the regulatory stay if a court finds the patent invalid or not infringed or if the court shortens the period because the parties have failed to cooperate in expediting the litigation.
Patent Term Restoration. A portion of the patent term lost during product development and FDA review of an NDA, including a 505(b)(2) application, may be restored if approval of the application is the first permitted commercial marketing of a drug product containing an active ingredient. The patent term restoration period is generally one-half the time between the effective date of the IND or the date of patent grant (whichever is later) and the date of submission of the NDA, plus the time between the date of submission of the NDA and the date of FDA approval of the product. The maximum period of restoration is five years, and the patent cannot be extended to more than 14 years from the date of FDA approval of the product. Only one patent claiming each approved product is eligible for restoration and the patent holder must apply for restoration within 60 days of approval. The U.S. Patent and Trademark Office in consultation with the FDA, reviews and approves the application for patent term restoration.
Other Exclusivities
Pediatric Exclusivity. Section 505A of the FDCA provides for a six-month extension of exclusivity or patent protection if an NDA sponsor submits pediatric data that fairly responds to a written request from the FDA for such data. The data does not need to show that the product is effective in the pediatric population studied; rather, if the clinical trial is deemed to fairly respond to the FDA's written request, additional protection is granted. If reports of requested pediatric studies are submitted to and accepted by FDA within the statutory time limits, whatever existing statutory or regulatory periods of exclusivity or Orange Book listed patent protection that cover the drug are extended by six months. This is not a patent term extension, but it effectively extends the regulatory period during which the FDA cannot approve an ANDA or 505(b)(2) application owing to regulatory exclusivity or listed patents. When any product candidate is approved, we will evaluate seeking pediatric exclusivity as appropriate.
Orphan Drug Exclusivity. The Orphan Drug Act provides incentives for the development of drugs intended to treat rare diseases or conditions, which is one that affects fewer than 200,000 individuals in the U.S. or more than 200,000 individuals, but for which there is no reasonable expectation that the cost of developing the product and making it available in the U.S. for the disease or condition will be recovered from U.S. sales of the product. If a sponsor demonstrates that a drug product qualifies for ODD, the FDA grants ODD to the product for that use. The benefits of ODD include research and development tax credits and exemption from user fees. A drug that is approved for the orphan drug designated indication generally is granted seven years of orphan drug exclusivity. During that period, the FDA generally may not approve any other application for the same drug for the same indication, although there are exceptions, most notably when the later product is shown to be clinically superior to the product with exclusivity. Clinical superiority can be established by way of greater efficacy, greater safety, or making a major contribution to patient care. The FDA can revoke a product’s orphan drug exclusivity under certain circumstances, including when the product sponsor is unable to assure the availability of sufficient quantities of the product to meet patient needs. Orphan drug exclusivity does not prevent the FDA from approving a different drug for the same disease or condition, or the same drug for a different disease or condition.
All our clinical-stage product candidates – GTx-104, GTx-102, and GTx-101 – have an ODD.
U.S. Healthcare Reform
The Patient Protection and Affordable Care Act, as amended (the "Affordable Care Act"), is a sweeping measure to expand healthcare coverage within the U.S., primarily through the imposition of health insurance mandates on employers and individuals and expansion of the Medicaid program. This law substantially changed the way healthcare is financed by both governmental and private insurers and significantly impacts the pharmaceutical industry. Changes that may affect our business include those governing enrollment in federal healthcare programs, reimbursement changes, benefits for patients, rules regarding prescription drug benefits under the health insurance exchanges, changes to the Medicaid Drug Rebate program, expansion of the Public Health Service Act's 340B drug pricing program (340B Program), fraud and abuse, and enforcement. These changes impact existing government healthcare programs and have resulted in the development of new programs, including Medicare payment for performance initiatives.
Certain provisions of the Affordable Care Act have been subject to judicial challenges, as well as efforts to modify them or to alter their interpretation and implementation.
It is unclear how efforts to modify or invalidate the Affordable Care Act or its implementing regulations, or portions thereof, will affect the Affordable Care Act or our business.
Legislative changes relating to reimbursement have been adopted in the U.S. since the Affordable Care Act was enacted. For example, the Budget Control Act of 2011, among other things, created the Joint Select Committee on Deficit Reduction to recommend to Congress proposals for spending reductions. The Joint Select Committee did not achieve a targeted deficit reduction, which triggered the legislation’s automatic reductions. In concert with subsequent legislation, this has resulted in aggregate reductions to Medicare payments to providers. Under current legislation, sequestration is currently set at 2%. As long as these cuts remain in effect, they could adversely impact payment for any products we may commercialize in the future.
Further, the Inflation Reduction Act (the “IRA”), among other things, established a Medicare Part B and Part D inflation rebate scheme, under which, generally, manufacturers owe rebates if the average sales price of certain Part B drugs or annual average manufacturer price of certain covered Part D drugs increases faster than the pace of inflation. The IRA also created a drug price negotiation program under which the prices for certain Medicare units of certain high Medicare spend drugs and biologics without generic or biosimilar competition are capped by reference to, among other things, a specified non-federal average manufacturer price, starting in 2026. The program excludes drugs with an orphan drug designation that are not approved for any indications unrelated to that designation. The IRA further made several changes to the Medicare Part D benefit, including a limit on annual out-of-pocket costs, and replacement of the coverage gap discount program with a new manufacturer discount program that began in 2025.
Additionally, on July 4, 2025, the One Big Beautiful Bill Act (the “OBBA”) was signed into law. The OBBBA is projected to decrease federal health care spending by approximately $1 trillion by reducing Medicaid spending and enrollment and making changes to federal Medicare spending. The law also made changes to Affordable Care Act marketplace enrollment that are projected to decrease the number of individuals with marketplace coverage.
Additional legislative changes, regulatory changes, or guidance could be adopted, which may impact the marketing approvals and reimbursement for our product candidates. For example, there has been increasing legislative, regulatory, and enforcement interest in the United States with respect to drug pricing practices, and there has been recent interest in incorporating so-called Most Favored Nation pricing into the U.S. healthcare system, under which prices for drugs in the United States could be tied to foreign reference prices through a mechanism that is not yet defined. There have been several Congressional inquiries and proposed and enacted federal and state legislation and regulatory initiatives designed to, among other things, bring more transparency to product pricing, evaluate the relationship between pricing and manufacturer patient programs, and reform government healthcare program reimbursement methodologies for drug products. If healthcare policies or reforms intended to curb healthcare costs are adopted or if we experience negative publicity with respect to pricing of our products, if approved, or the pricing of pharmaceutical drugs generally, the prices that we charge for any approved products may be limited, our commercial opportunity may be limited and/or our revenues from sales of our products, if approved, may be negatively impacted.
Healthcare Privacy Laws
We may be subject to laws and regulations covering data privacy and the protection of health-related and other personal information. The legislative and regulatory landscape for privacy and data protection continues to evolve, and there has been an increasing focus on privacy and data protection issues which may affect our business. Numerous federal and state laws and regulations, including state security breach notification laws, state health information and/or privacy laws and federal and state consumer protection laws and consumer privacy laws, govern the collection, use, disclosure, and protection of personal information. Such laws and regulations include the Health Insurance Portability and Accountability Act of 1996, the Health Information Technology for Economic and Clinical Health Act, and their implementing regulations (collectively, “HIPAA”), Section 5 of the Federal Trade Commission Act (the “FTC Act”), and the California Consumer Privacy Act, as amended by the California Privacy Rights Act (the “CCPA”). Other jurisdictions have enacted or proposed legislation and/or regulations similar to the CCPA. Health-specific consumer privacy laws were also passed in multiple states. Many of these laws differ from each other in significant ways and have different effects. Many of the state laws enable a state attorney general or other regulator to bring actions and provide private rights of action to consumers as enforcement mechanisms. There is also heightened sensitivity around certain types of health data, which may be subject to additional protection.
HIPAA also imposes requirements relating to the privacy, security and transmission of individually identifiable health information. HIPAA imposes privacy and security obligations on covered entity health care providers, health plans, and health care clearinghouses, as well as their “business associates” (i.e., certain persons or entities that create, receive, maintain, or transmit protected health information in connection with providing a specified service or performing a function on behalf of a covered entity). HIPAA has four tiers of civil monetary penalties and grants state attorneys enforcement authority. The Department of Justice also may impose criminal penalties. Additionally, certain states have adopted comparable privacy and security laws and regulations, some of which may be more stringent than HIPAA.
Although we are not directly subject to the privacy, security, and transmission requirements of HIPAA, other than potentially with respect to providing certain employee benefits, we may obtain health information from third parties that are subject to privacy and security requirements under HIPAA, and other privacy and data security and consumer protection laws, and we could potentially be subject to criminal penalties if we, our affiliates, or our agents knowingly receive individually identifiable health information maintained by a HIPAA-covered entity in a manner that is not authorized or permitted by HIPAA, and could also potentially be subject to other civil and/or criminal penalties if we obtain, use or disclose information in a manner not permitted by other privacy and data security and consumer protection laws.
Outside the United States, our clinical trial programs and other processing activities implicate international data protection laws, including the EU General Data Protection Regulation 2016/679 (the “GDPR”). The GDPR has increased our responsibility and liability in relation to the processing of personal data of individuals located in the EU. The GDPR, together with the national legislation of the EU member states governing the processing of personal data, impose strict obligations and restrictions on the ability to collect, analyze, and transfer personal data, including health data and samples from clinical trials and adverse event reporting. For example, these obligations and restrictions may require obtaining explicit consent of the individuals to whom the personal data relate, providing transparency notices to individuals, sharing personal data with third parties, transferring personal data out of the EU, reporting personal data breaches with data protection authorities and affected individuals, and ensuring the security and confidentiality of personal data. Violations of EU data protection laws may result in significant financial penalties (including possible fines of up to 4% of global annual turnover for the preceding financial year or €20 million (whichever is higher)). The GDPR also confers a private right of action on data subjects and consumer associations to lodge complaints with data protection authorities, seek judicial remedies, and obtain compensation for damages resulting from violations of the GDPR. EU data protection laws also require us to implement appropriate transfer mechanisms in order to lawfully transfer personal from the EU to countries or organizations outside the EU.
Moreover, as a result of the broad scale release and availability of Artificial Intelligence (“AI”) technologies such as generative AI, there is a global trend towards more regulation (e.g., the EU AI Act and AI laws passed in U.S. states) to ensure the ethical use, privacy, and security of AI and the data that it processes. Compliance with such laws will likely be an increasing and substantial cost in the future.
Failure to comply with these laws and regulations could result in government enforcement actions and create liability for us (including the imposition of significant penalties), private litigation and/or adverse publicity that could negatively affect our business.
Coverage and Reimbursement
Sales of any pharmaceutical product depend, in part, on the extent to which such product will be covered by third-party payors, such as federal, state, and foreign government healthcare programs, commercial insurance and managed healthcare organizations, and the level of reimbursement for such product by third-party payors. Significant uncertainty exists as to the coverage and reimbursement status of any newly approved product. Decisions regarding the extent of coverage and amount of reimbursement to be provided are made on a payor-by-payor basis. One third-party payor’s decision to cover a product does not ensure that other payors will also provide coverage for the product. As a result, the coverage determination process can require manufacturers to provide scientific and clinical support for the use of a product to each payor separately, and can be a time-consuming process, with no assurance that coverage and adequate reimbursement will be applied consistently or obtained in the first instance.
In order to secure coverage and reimbursement for any products, if approved, we may need to conduct expensive pharmacoeconomic studies to demonstrate the medical necessity and cost-effectiveness of the product, in addition to the costly studies required to obtain FDA or other comparable regulatory approvals. Even if we conduct pharmacoeconomic studies, our products, if approved, may not be considered medically necessary or cost-effective by payors. Further, a payor’s decision to provide coverage for a product does not guarantee that an adequate reimbursement rate will be set, including as commercial payors may establish different rates for different health care providers.
In addition, third-party payors are increasingly reducing reimbursements for pharmaceutical products and services. The U.S. government and state legislatures have continued implementing cost-containment programs, including price controls, restrictions on coverage and reimbursement, and requirements for substitution of generic products. Third-party payors are increasingly challenging the prices charged, examining the medical necessity, and reviewing the cost effectiveness of pharmaceutical products, in addition to questioning their safety and efficacy. Increasingly, third-party payors are requiring that drug companies provide them with predetermined discounts from list prices and are challenging the prices charged for medical products. Further, such payors are increasingly challenging the price, examining the medical necessity and reviewing the cost effectiveness of medical drug candidates. Adoption of price controls and cost-containment measures, and adoption of more restrictive policies in jurisdictions with existing controls and measures, could further limit or delay sales of any product. Decreases in third-party reimbursement for any product or a decision by a third-party payor not to cover a product could reduce physician usage and patient demand for the product.
Government Price Reporting
A manufacturer that participates in the Medicaid Drug Rebate Program is required to pay a rebate to each state Medicaid program for its covered outpatient drugs that are dispensed to Medicaid beneficiaries and paid for by a state Medicaid program, as a condition of having federal funds available for that manufacturer’s drugs under Medicaid and Medicare Part B. Those rebates are based on pricing data the manufacturer reports on a monthly and quarterly basis to the Centers for Medicare & Medicaid Services (“CMS”), the federal agency that administers the Medicare and Medicaid programs. These data include the average manufacturer price and, in the case of innovator products, the best price for each drug, which best price, in general, represents the lowest price available from the manufacturer to any wholesaler, retailer, provider, health maintenance organization, nonprofit entity, or governmental entity in the U.S. in any pricing structure, calculated to include all sales and associated rebates, discounts, and other price concessions. Where the average manufacturer price of a drug increases faster than the pace of inflation, the drug may be subject to an additional rebate paid by its manufacturer in the amount that the average manufacturer price has exceeded the pace of inflation. In September 2024, CMS further modified the regulations governing the Medicaid Drug Rebate Program, which could increase manufacturer costs and the complexity of compliance, impact rebate liabilities, and be time-consuming to implement.
Federal law requires that each manufacturer that participates in the Medicaid Drug Rebate Program also participate in the 340B drug pricing program in order for federal funds to be available for the manufacturer’s drugs under Medicaid and Medicare Part B. The 340B program, which is administered by the Health Resources and Services Administration (“HRSA”), requires participating manufacturers to agree to charge statutorily defined covered entities no more than the 340B "ceiling price" for the manufacturer’s covered outpatient drugs. These 340B covered entities include a variety of community health clinics and other entities that receive health services grants from the Public Health Service, as well as hospitals that serve a disproportionate share of low-income patients, certain free-standing cancer hospitals, critical access hospitals, rural referral centers and sole community hospitals. The 340B ceiling price is calculated using a statutory formula, which is based on the average manufacturer price and rebate amount for the covered outpatient drug as calculated under the Medicaid Drug Rebate Program, and, in general, products subject to Medicaid price reporting and rebate liability are also subject to the 340B ceiling price calculation and discount requirement. Changes to the Medicaid Drug Rebate amount also could affect a manufacturer’s 340B ceiling price calculations and negatively impact results of operations.
HRSA issued a final regulation regarding the calculation of the 340B ceiling price and the imposition of civil monetary penalties on manufacturers that are found to have knowingly and intentionally overcharged covered entities, which became effective on January 1, 2019. It is not fully clear how the government will apply its enforcement authority under the regulation. Manufacturers also are required to report 340B ceiling prices to HRSA on a quarterly basis, and HRSA then publishes them to covered entities. Moreover, under a final regulation effective January 13, 2021, HRSA established an administrative dispute resolution (“ADR”) process for claims by covered entities that a manufacturer has engaged in overcharging, and by manufacturers that a covered entity violated the prohibitions against diversion or duplicate discounts. Such claims are to be resolved through an ADR panel of government officials rendering a decision that could be appealed in federal court. HRSA issued a final rule, effective June 2024, that modified aspects of the ADR process, which could impact the procedures that are used to determine whether a manufacturer owes additional 340B discounts. An ADR proceeding could subject a manufacturer to onerous procedural requirements and result in additional liability.
Manufacturers are required to report the average sales price for certain products paid for under Medicare Part B. Manufacturers calculate the average sales price based on a statutorily defined formula as well as regulations and interpretations of the statute by CMS. CMS may use these submissions to determine payment rates for drugs under Medicare Part B. Since 2023, manufacturers must pay refunds to Medicare for single source drugs or biologics, or biosimilar biological products, reimbursed under Medicare Part B and packaged in single-dose containers or single-use packages, for units of discarded drug reimbursed by Medicare Part B in excess of ten percent of total allowed charges under Medicare Part B for that drug. Manufacturers that fail to pay refunds could be subject to civil monetary penalties of 125 percent of the refund amount. In addition, as noted previously, a manufacturer may be liable for Part B inflation rebates for utilization in quarters starting with the first quarter of 2023. Manufacturers may be liable for civil monetary penalties for violations of this program.
Statutory or regulatory changes or CMS guidance could affect the average sales price calculations for approved products and the resulting Medicare payment rate, and could negatively impact results of operations. For example, the IRA, among other things, requires the Secretary of HHS to negotiate, with respect to Medicare units and subject to a specified cap, the price of a set number of certain high Medicare spend drugs and biologicals per year with the first negotiated prices taking effect in 2026. The IRA established a Medicare Part B inflation rebate scheme, under which, generally speaking, manufacturers owe rebates if the average sales price of a Part B drug increases faster than the pace of inflation. Failure to pay a Part B inflation rebate timely is subject to a civil monetary penalty.
These or any other public policy changes could impact the market conditions for our product candidates. We further expect continued scrutiny on government price reporting and pricing more generally from Congress, agencies, and other bodies, and are seeing an increase in state interest in price reporting, transparency, and other policies to address drug pricing concerns.
In order to be eligible to have products paid for with federal funds under the Medicaid and Medicare Part B programs, as applicable, and purchased by certain federal agencies and grantees, a manufacturer is required to participate in the U.S. Department of Veterans Affairs (the “VA”) Federal Supply Schedule (“FSS”) pricing program. Under this program, the manufacturer is obligated to make its covered drugs available for procurement on an FSS contract and charge a price to four federal agencies — the VA, U.S. Department of Defense (DoD), Public Health Service and U.S. Coast Guard — that is no higher than the statutory Federal Ceiling Price (the “FCP”). The FCP is based on the non-federal average manufacturer price (“Non-FAMP”), which the manufacturer calculates and reports to the VA on a quarterly and annual basis. A manufacturer would also need to participate in the Tricare Retail Pharmacy program, under which it will pay quarterly rebates on utilization of innovator products that are dispensed through the Tricare Retail Pharmacy network to Tricare beneficiaries. The rebates are calculated as the difference between the annual Non-FAMP and FCP.
Pricing and rebate calculations vary across products and programs, are complex, and are often subject to interpretation by the manufacturer, governmental or regulatory agencies, and the courts. Significant civil monetary penalties can be applied if a manufacturer is found to have knowingly submitted any false average manufacturer price, best price, or Non-FAMP information to the government or fails to submit the required price data on a timely basis. Such conduct also could be grounds for CMS to terminate the Medicaid drug rebate agreement, in which case federal payments may not be available under Medicaid or Medicare Part B for covered outpatient drugs. Civil monetary penalties could also be applied if a manufacturer is found to have charged 340B covered entities more than the statutorily mandated ceiling price. In addition, claims submitted to federally-funded healthcare programs, such as Medicare and Medicaid, for drugs priced based on incorrect pricing data provided by a manufacturer can implicate the False Claims Act.
Healthcare Fraud and Abuse Laws
In addition to FDA restrictions on marketing of pharmaceutical products, our business is subject to healthcare fraud and abuse regulation and enforcement by both the federal government and the states in which we conduct our business. These laws include, but are not limited to, the following:
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The federal Anti-Kickback Statute prohibits, among other things, knowingly and willfully offering, paying, soliciting or receiving remuneration, directly or indirectly, in cash or in kind, to induce or in return for purchasing, leasing, ordering or arranging for or recommending the purchase, lease or order of any healthcare item or service reimbursable, in whole or in part, under Medicare, Medicaid or other federally financed healthcare programs. This statute has been interpreted to apply to arrangements between pharmaceutical manufacturers on one hand and prescribers, purchasers, and formulary managers on the other. A violation of the Anti-Kickback Statute may be established without proving actual knowledge of the statute or specific intent to violate it. In addition, the government may assert that a claim including items or services resulting from a violation of the federal Anti-Kickback Statute constitutes a false or fraudulent claim for purposes of the federal civil False Claims Act. Violations of the federal Anti-Kickback Statute are punishable by imprisonment, criminal fines, damages, civil monetary penalties, and exclusion from participation in federal healthcare programs. Although there are a number of statutory exceptions and regulatory safe harbors protecting certain common activities from prosecution, the exceptions and safe harbors are drawn narrowly and practices that involve remuneration intended to induce prescribing, purchasing, or recommending pharmaceuticals, including certain discounts, or engaging such individuals as consultants, speakers or advisors, may be subject to scrutiny if they do not fit squarely within the exception or safe harbor. Federal enforcement agencies also have shown increased interest in pharmaceutical companies’ product and patient assistance programs, including reimbursement and co-pay support services, and a number of investigations into these programs have resulted in significant civil and criminal settlements. Our practices may not in all cases meet all of the criteria for safe harbor protection from anti-kickback liability. Moreover, there are no safe harbors for many common practices, such as educational and research grants, charitable donations, product support and patient assistance programs. Arrangements that implicate the Anti-Kickback Statute and do not fit within an exception or safe harbor are reviewed on a case-by-case basis to determine whether, based on the facts and circumstances, they violate the statute.
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The federal civil False Claims Act prohibits any person from, among other things, knowingly presenting, or causing to be presented, a false or fraudulent claim for payment of government funds, or knowingly making, using, or causing to be made or used, a false record or statement material to an obligation to pay money to the government, or knowingly concealing or knowingly and improperly avoiding, decreasing, or concealing an obligation to pay money to the U.S. federal government. Actions under the False Claims Act may be brought by private individuals known as qui tam relators in the name of the government, and who may share in any monetary recovery. In recent years, several pharmaceutical and other healthcare companies have faced enforcement actions under the False Claims Act for, among other things, providing free product to customers with the expectation that the customers would bill federal programs for the product and distributing guidance to prescribers and other customers that affected customers' billing or coding practices on claims submitted to the federal government. Other companies have faced enforcement actions for causing false claims to be submitted because of the company's marketing of the product for unapproved uses.
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The healthcare fraud provisions under HIPAA prohibit, among other things, knowingly and willfully executing a scheme to defraud any healthcare benefit program, including private third-party payors. The HIPAA fraud provisions also prohibit knowingly and willfully falsifying, concealing or covering up a material fact or making any materially false, fictitious or fraudulent statement or representation, or making or using any false writing or document knowing the same to contain any materially false, fictitious or fraudulent statement or entry in connection with the delivery of or payment for healthcare benefits, items or services. Violations of HIPAA fraud provisions may result in criminal, civil, and administrative penalties, fines and damages, including exclusion from participation in federal healthcare programs.
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The majority of states, as well as many of the non-US jurisdictions where we may operate, also have statutes or regulations similar to the federal anti-kickback and false claims laws, which apply to items and services reimbursed under Medicaid and other state programs, or, in several states, apply regardless of the payor. Several states now require pharmaceutical companies to report expenses relating to the marketing and promotion of pharmaceutical products in those states and to report gifts and payments to individual health care providers in those states. Some of these states also prohibit certain marketing-related activities including the provision of gifts, meals, or other items to certain health care providers. Other states have laws requiring pharmaceutical sales representatives to be registered or licensed, and still others impose limits on co-pay assistance that pharmaceutical companies can offer to patients. In addition, several states require pharmaceutical companies to implement compliance programs or marketing codes.
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The Physician Payments Sunshine Act, implemented as the Open Payments program, and its implementing regulations, requires manufacturers of drugs, devices, biologics and medical supplies for which payment is available under Medicare, Medicaid or the Children’s Health Insurance Program (with certain exceptions) to report annually to CMS information related to direct or indirect payments and other transfers of value to physicians (defined to include doctors, dentists, optometrists, podiatrists and chiropractors), other healthcare professionals (such as physician assistants and nurse practitioners) and teaching hospitals, as well as ownership and investment interests held in the company by physicians and their immediate family members.
Compliance with such laws and regulations requires substantial resources. Because of the breadth of these various fraud and abuse laws, it is possible that some of our business activities could be subject to challenge under one or more of such laws. Such a challenge could have material adverse effects on our business, financial condition and results of operations. In the event governmental authorities conclude that our business practices do not comply with current or future statutes, regulations or case law involving applicable fraud and abuse or other healthcare laws and regulations, they may impose sanctions under these laws, which are potentially significant and may include civil monetary penalties, damages, exclusion of an entity or individual from participation in government health care programs, criminal fines and imprisonment, additional reporting requirements if we become subject to a corporate integrity agreement or other settlement to resolve allegations of violations of these laws, as well as the potential curtailment or restructuring of our operations. Even if we are not determined to have violated these laws, government investigations into these issues typically require the expenditure of significant resources and generate negative publicity.
Foreign Corrupt Practices Act
In addition, the U.S. Foreign Corrupt Practices Act of 1997 prohibits corporations and their intermediaries from engaging in certain activities to obtain or retain business or to influence a person working in an official capacity. It is illegal to pay, offer to pay or authorize the payment of anything of value to any official of another country, government staff member, political party or political candidate to obtain or retain business or to otherwise influence a person working in that capacity.
Human Capital Resources
As of March 31, 2026, we had a total of five full-time employees, all of whom are located in the United States. Our employees have significant prior experience within the pharmaceutical, and biotech industries. We continue to focus on building a high performing organization through our emphasis on accountability for results as measured by our performance development process. To help ensure that employees fully understand our long-term strategy, and how their work contributes to our success, we utilize a variety of channels to facilitate open and direct communication, including: regular calls with all employees, and ongoing update communications as needed.
Corporate Structure
We were incorporated on February 1,
2002 under Part 1A of the Companies Act (Québec) under the name
“9113-0310 Québec Inc.” On August 7, 2008, pursuant to a Certificate of
Amendment, we changed our name to “Acasti Pharma, Inc.” We became a reporting
issuer in the Province of Québec on November 17, 2008. On February 14, 2011, the
Business Corporations Act (Québec) came into effect and replaced the Companies
Act (Québec). On August 27, 2021, Acasti Pharma, Inc. completed its
acquisition of Grace Therapeutics Inc., a privately held emerging
biopharmaceutical company focused on developing innovative drug delivery
technologies for the treatment of rare and orphan diseases via a merger.
Following completion of the merger, Grace Therapeutics Inc. became our wholly
owned subsidiary and was renamed Acasti Pharma U.S. Inc.
On October 1, 2024, we changed our jurisdiction of incorporation from the Province of Québec in Canada to the Province of British Columbia in Canada (“Acasti British Columbia”) pursuant to a “continuance” effected in accordance with Chapter XII of the Business Corporations Act (Québec) (the “Continuance”). On October 7, 2024, we changed our jurisdiction of incorporation from the Province of British Columbia in Canada to the State of Delaware (“Acasti Delaware”) in the United States pursuant to a “continuance” effected in accordance with Section 308 of the Business Corporations Act (British Columbia) and a “domestication” (the “Domestication”) under Section 388 of the General Corporation Law of the State of Delaware (the “DGCL”). Both the Continuance and the Domestication were approved by our shareholders at our Annual and Special Meeting of Shareholders held on September 30, 2024. Effective on October 28, 2024, we changed our corporate name to “Grace Therapeutics, Inc.”. Effective on October 28, 2024, we changed the corporate name of our subsidiary to “Grace Therapeutics U.S., Inc.” (“Grace U.S.”).
Prior to the Continuance and Domestication, our Class A common shares, without par value per share (“common shares”), were listed on The Nasdaq Stock Market LLC (“Nasdaq”) under the symbol “ACST.” Upon the effectiveness of the Continuance, each of our outstanding common shares at the time of the Continuance remained issued and outstanding as a common share, without par value per share, of Acasti British Columbia. Upon effectiveness of the Domestication, each outstanding common share of Acasti British Columbia at the time of the Domestication automatically became one outstanding share of common stock, par value $0.0001 per share, of Acasti Delaware (“common stock”).
Information regarding stockholder tax consequences of the Domestication and potential tax elections is available in our Registration Statement on Form S-4 originally filed with the SEC on June 27, 2024, as amended on July 31, 2024.
YOU SHOULD CONSULT YOUR TAX ADVISORS WITH RESPECT TO THE APPLICATION OF U.S. FEDERAL TAX LAWS TO YOUR PARTICLAR SITUATION, AS WELL AS ANY TAX CONSEQUENCES ARISING UNDER THE LAWS OF ANY STATE, LOCAL OR NON-U.S. JURISDICTION.
Available Information
This Annual Report on Form 10-K, our quarterly reports on Form 10-Q, our current reports on Form 8-K, and any amendments to these reports are filed, or will be filed, as applicable, with the SEC, and the Canadian Securities Administrators (“CSA”). These reports are available free of charge on our website, https://www.gracetx.com/, as soon as reasonably practicable after we electronically file such reports with or furnish such reports to the SEC and the CSA. Information contained on, or accessible through, our website is not a part of this Annual Report on Form 10-K, and the inclusion of our website address in this document is an inactive textual reference.
Additionally, our filings with the SEC may be accessed through the SEC’s website at https://www.sec.gov and our filings with the CSA may be accessed through the CSA’s System for Electronic Document Analysis and Retrieval at https://www.sedarplus.ca/.
Risks Factors Relating to our Business
We received a Complete Response Letter (CRL) from the FDA citing certain manufacturing deficiencies and other non-clinical issues, which may delay or prevent approval of GTx-104.
In
April 2026, we received a CRL from the FDA in response to our NDA for GTx-104.
The CRL referenced certain items related to the CMC and other non-clinical
sections of the application, including items related to additional leachable
data time points for commercial product, non-clinical product toxicology risk
assessments, and cGMP deficiencies at our CMO. A Type A meeting with the FDA
has been scheduled to potentially clarify the path forward and determine the
appropriate next steps.
We rely in large part on our CMO to remediate the identified deficiencies, including implementing corrective and preventive actions, and our ability to resolve these issues is subject to factors outside our control. Remediation may require facility upgrades, quality system enhancements, equipment requalification, and additional validation studies or testing, any of which could be costly and time-consuming. We also may be required to generate additional data to support manufacturing changes. If our CMO is unable to adequately or timely remediate the identified deficiencies, we may need to transfer manufacturing operations to an alternative facility, which would involve significant time, expense and regulatory risk. We also may be unable to identify or establish manufacturing at an adequate alternative facility. For additional information on these risks, please see the risk factors captioned “If our current CMO is unable to timely resolve the deficiencies identified by the FDA in its CRL for GTx‑104, we may be required to engage an alternative replacement manufacturer, which could result in significant delays, increased costs, or our inability to obtain regulatory approval or commercialize GTx‑104 at all.” and “We rely on third parties to manufacture commercial and clinical supplies of our drug candidates, and we intend to rely on third parties to manufacture commercial supplies of any approved drug products. The commercialization of any of our drug products could be stopped, delayed, or made less profitable if those third parties fail to provide us with sufficient quantities of active pharmaceutical ingredients, excipients, or drug products, or fail to do so at acceptable quality levels or prices or fail to maintain or achieve satisfactory regulatory compliance.”
If we cannot sufficiently address the issues set forth in the CRL, we may not be able to resubmit the NDA, or resubmission may not result in approval. If we do resubmit the NDA, the FDA may conduct a re-inspection of the applicable CMO, and there can be no assurance that the facility will be found compliant or that additional deficiencies will not be identified. As a result, approval of GTx-104 may be significantly delayed, limited, or may not be obtained at all, any of which could materially and adversely affect our business, financial condition, results of operations and prospects.
We may not achieve our publicly announced milestones on time, or at all.
From time to time, we may publicly announce the timing of certain events that we expect to occur, such as the anticipated timing of our NDA resubmission for GTx-104. These statements are forward-looking and are based on the best estimate of management at the time relating to the occurrence of the events. However, the actual timing of these events may differ from what has been publicly disclosed. The timing of events such as completion of a clinical trial, discovery of a new product candidate, filing or resubmission of an application to obtain regulatory approval, beginning of commercialization of products, completion of a strategic partnership, or announcement of additional clinical trials for a product candidate may ultimately vary from what is publicly disclosed. These variations in timing may occur as a result of different events, including the outcome of CRL remediation efforts or the results obtained during a clinical trial or during a research phase, problems with a supplier or a distribution partner (such as the items identified in CRL we received in April 2026) or any other event having the effect of delaying the publicly announced timeline. We undertake no obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as otherwise required by law. Any variation in the timing of previously announced milestones could have a material adverse effect on our business, financial condition, operating results or the trading price of our common stock.
We are heavily dependent on the success of our lead drug candidate, GTx-104.
Our business and future success are substantially dependent on our ability to successfully and timely develop, obtain regulatory approval for, and commercialize our lead product candidate, GTx-104. On April 23, 2026, we received a CRL from the FDA in response to our NDA for GTx‑104. The CRL will delay any potential approval of GTx‑104 unless and until we adequately address the deficiencies to the satisfaction of FDA, and there can be no assurance that we will be able to do so on a timely basis, or at all. Even if we resubmit the NDA, there can be no assurance that the FDA will determine our response adequately addresses the deficiencies identified in the CRL, will not identify additional deficiencies upon resubmission, or will approve the NDA.
Any delay or setback in our NDA resubmission, any requirement to conduct additional work (including new studies or manufacturing activities), any adverse findings from an FDA inspection of our CMO, or any additional FDA requests following resubmission would extend the period before which we could potentially obtain approval for GTx-104, commercialize GTx‑104, and generate any product revenue. Failure to obtain approval of GTx-104 would adversely affect our business, financial condition, operating results and prospects, including by limiting our ability to execute our strategy, potentially requiring us to scale back operations, and increasing our need for additional financing on terms that may be dilutive or otherwise unfavorable.
Our planned development, approval and commercialization of GTx-104 may fail to be completed in a timely manner or at all. As part of our strategic realignment plan, we determined to focus primarily on the development of GTx-104, which concentrates the level of our drug development risk on one drug candidate. We cannot provide assurance that we will be able to obtain approval for GTx-104 or any other of our drug candidates from the FDA or any foreign regulatory authority or that we will obtain such approval in a timely manner.
We may not be able to maintain our operations and advance our research and development and if approved, commercialization of our GTx-104 lead drug candidate without additional funding.
We have incurred operating losses and negative cash flows from operations since our inception. To date, we have financed our operations through public offerings and private placements of securities, proceeds from exercises of warrants, rights and options, and receipt of research tax credits and research grant programs. Our cash and cash equivalents were $17.0 million as of March 31, 2026, and $22.1 million as of March 31, 2025.
Our current assets, as of March 31, 2026, are projected to support our current liabilities as at that date when combined with the projected level of our expenses through at least twelve months from the issuance date of the audited consolidated financial statements included with this Annual Report on Form 10-K, including expenses in connection with the potential resubmission of the NDA for GTx-104, working capital and other general corporate purposes. Additional capital will be required by us to support the commercial launch of GTx-104, if approved. To fully execute our business plan, we plan to raise the necessary capital primarily through additional securities offerings and multiple sources of non-dilutive capital, such as grants, loans and strategic alliances.
We cannot be certain that additional funding will be available when needed on acceptable terms, or at all. If we are unable to raise additional capital in sufficient amounts, when required or on acceptable terms, we also could be required to:
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seek out-licensing or commercialization partners to assist in the commercialization of GTx-104, if approved;
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delay, limit or reduce the commercialization of GTx-104, if approved; or
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significantly curtail our operations.
We have no history of commercializing drugs, which may make it difficult for you to evaluate the success of our business to date and to assess our future viability.
Our operations to date have been largely focused on raising capital and developing products for rare and orphan diseases, including undertaking preclinical studies and conducting clinical trials. We have not yet received FDA approval of GTx-104, and as such we have not yet demonstrated our ability to successfully supply GTx-104 for commercial sale or conduct sales, marketing and distribution activities necessary for successful product commercialization. Consequently, any predictions about our future success or viability may not be as accurate as they could be if we had a longer history of successfully developing and commercializing drugs.
Our future success depends on our ability to retain key executives and to attract, retain and motivate qualified personnel.
We are highly dependent on the principal members of our executive team. While members of our executive team have significant industry experience, they have not been with the Company for long. Any of our executive officers could leave our employment at any time, as all of our employees are “at will” employees. Recruiting and retaining qualified employees for our business, including scientific and technical personnel, will also be critical to our success. There is currently a shortage of skilled executives and other personnel in our industry, which is likely to continue. As a result, competition for skilled personnel is intense and the turnover rate can be high. As we build our organization, we may not be able to attract and retain personnel on terms that are favorable to us given the competition among numerous pharmaceutical companies for individuals with similar skill sets. In addition, failure to succeed in clinical studies or regulatory approvals may make it more challenging to recruit and retain qualified personnel. The inability to recruit key executives or the loss of the services of any executive or key employee might impede the progress of our development and commercialization objectives.
We may need to expand our organization, and we may experience difficulties in managing this growth, which could disrupt our operations and our ability to compete.
If our drug development efforts are successful, we expect to expand our employee base to increase our managerial, scientific, engineering, operational, sales, marketing, financial and other resources and to hire more consultants and contractors. Future growth would impose significant additional responsibilities on our management, including the need to identify, recruit, maintain, motivate and integrate additional employees, consultants and contractors. We may not be able to effectively manage the expansion of our operations, which may result in weaknesses in our infrastructure, or give rise to operational mistakes, loss of business opportunities, loss of employees and reduced productivity among remaining employees. Future growth could require significant capital expenditures and may divert financial resources from other projects, such as the development of our existing or future product candidates. Our future financial performance and our ability to sell and commercialize our product candidates, if approved, and compete effectively will depend, in part, on our ability to effectively manage any future growth.
We face potential product liability, and if claims are brought against us, we may incur substantial liability.
The use of our product candidates in clinical trials, and the sale of any drug candidates for which we obtain marketing approval, exposes us to the risk of product liability claims. Product liability claims might be brought against us by consumers, healthcare providers, pharmaceutical companies or others selling or otherwise coming into contact with our product candidates. If we cannot successfully defend against product liability claims, we could incur substantial liability and costs. In addition, regardless of merit or eventual outcome, product liability claims may result in:
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impairment of our business reputation;
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withdrawal of clinical study participants;
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costs due to related litigation;
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distraction of management’s attention from our primary business;
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substantial monetary awards to patients or other claimants; and
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the inability to commercialize our product candidates.
Our current product liability insurance coverage may not be sufficient to reimburse us for any expenses or losses we may suffer. Moreover, insurance coverage is becoming increasingly expensive, and, in the future, we may not be able to maintain insurance coverage at a reasonable cost or in sufficient amounts to protect us against losses due to liability. Any successful product liability claims or series of claims brought against us could cause our stock price to decline and, if judgments exceed our insurance coverage, could adversely affect our results of operations and business.
We rely on information technology and any failure, inadequacy, interruption, or security lapse of that technology, including any cybersecurity incidents, could harm our ability to operate our business effectively.
We rely on information
technology systems and third-party service providers to support our business,
including our research and development activities, clinical trials, regulatory
submissions, manufacturing operations, and corporate functions. Despite the
implementation of security measures, our information technology systems, and
those of third parties on which we rely, are vulnerable to damage or
interruption from events including cyber-attacks (including ransomware, malware
attacks, unauthorized access attempts, and denial of service and other
unintentional intrusions or malicious cyberattacks), social engineering
(including phishing) or other fraudulent schemes, and other cybersecurity
incidents, as well as natural disasters, terrorism, war and telecommunication
and electrical failures. These threats may arise from persons inside our
organization, authorized persons with access to systems inside our organization
or those with whom we do business, or unauthorized individuals. System
failures, accidents or cybersecurity incidents impacting us or our contractors,
consultants, vendors, or other business partners could cause interruptions in
our operations and could result in a material disruption of our drug product
development and clinical activities and business operations, in addition to
possibly requiring substantial expenditures of resources to address. The loss
of drug product development or clinical trial data could result in delays in
our regulatory approval efforts and significantly increase our costs to recover
or reproduce the data. To the extent that any disruption or cybersecurity
incident were to result in unauthorized access to, loss or modification
of, or damage to, our data or
applications, or unauthorized disclosure of confidential or proprietary
information, we could incur liability and our development programs, and the
development of our product candidates, could be delayed, which could adversely
affect our financial position, results of operations and business. A
significant cybersecurity incident may also deter clinical trial participants
from participating in any future trials. Furthermore, if our information
technology systems, or those of third parties on which we rely, suffer severe
damage, disruption or shutdown and our business continuity plans do not
effectively resolve the issues in a timely manner, we could experience delays
in reporting our financial results or miss reporting deadlines.
In addition, we depend on third parties, including CROs and CMOs, and other vendors, to conduct significant aspects of our operations. Any cybersecurity incident affecting these third parties could have similar adverse effects on us, and we may have limited ability to monitor or control their cybersecurity measures.
We have implemented
security measures and continue to invest in cybersecurity, but such measures
may not be sufficient to prevent all cybersecurity incidents. Cyber threats are
evolving in frequency and sophistication, including using artificial intelligence
(“AI”), and we may not be able to anticipate or adequately mitigate these
risks. For example, threat actors may use AI tools to automate and enhance
cybersecurity attacks against us. We use software and platforms designed to
detect such cybersecurity threats, including AI-based tools, but these threats
could become more sophisticated and harder to detect and counteract, which may
pose significant risks to our data security and systems. Any actual or
perceived cybersecurity incident could also result in regulatory scrutiny or
investigations, litigation, reputational harm, and significant costs.
Additionally, while we have implemented security measures that we believe are
appropriate and continue to enhance cybersecurity protections, a regulator
could deem our security measures not to be appropriate given the lack of
prescriptive measures in certain laws. Increased regulation of data collection,
use and retention practices, including self-regulation and industry standards,
changes in existing laws and regulations, enactment of new laws and
regulations, increased enforcement activity, and changes in interpretation of
laws, could increase our cost of compliance and operation, limit our ability to
grow our business or otherwise harm our business.
Disruptions at the FDA, the SEC and other government agencies could hinder their ability to hire and retain key leadership and other personnel, prevent or delay new products and services from being developed, approved, or commercialized in a timely manner or otherwise prevent those agencies from performing normal business functions on which the operation of our business may rely, which could negatively impact our business.
The ability of the FDA to review and approve new products or review other regulatory submissions can be affected by a variety of factors, including government budget and funding levels, a reduction in the FDA’s workforce and its ability to hire and retain key personnel and accept the payment of user fees, shifting policy priorities, and statutory, regulatory and policy changes. In addition, government funding of the FDA, SEC and other government agencies on which our operations may rely, including those that fund research and development activities, is subject to the political process, which is inherently fluid and unpredictable.
Disruptions at the FDA and other agencies may also increase the time to meet with and receive agency feedback, accept, review and/or approve our submissions, conduct inspections, issue regulatory guidance, or take other actions that facilitate the development, approval and marketing of regulated products, which would adversely affect our business. In addition, government proposals to reduce or eliminate budgetary deficits may include reduced allocations to the FDA and other related government agencies. For example, over the last several years, the U.S. government has shut down several times and certain regulatory agencies, such as the FDA and the SEC, have had to furlough critical FDA, SEC and other government employees and stop critical activities. It is unclear how certain executive actions or other potential actions by the U.S. presidential administration or other parts of the federal government will impact the FDA or other regulatory authorities that oversee our business. These budgetary pressures may reduce the FDA’s ability to perform its responsibilities. If a significant reduction in the FDA’s workforce occurs, the FDA’s budget is significantly reduced or a prolonged government shutdown occurs, or if global health concerns prevent the FDA or other regulatory authorities from conducting business as usual or conducting inspections, reviews or other regulatory activities, it could significantly impact the ability of the FDA to timely review and process our regulatory submissions or take other actions critical to the development or marketing of our product candidates, which could have a material adverse effect on our business.
Unfavorable global geopolitical events and economic conditions, including military conflicts and trade tensions between the U.S. and its global trading partners, including China, could adversely affect our business, financial condition or results of operations, including conduct of our clinical trials and our manufacturing activities.
Our results of operations could be adversely affected by general conditions in the global economy and in the global financial markets. The financial markets and the global economy may also be adversely affected by the current or anticipated impact of military conflict, including the ongoing conflicts in the Middle East and between Russia and Ukraine, terrorism or other political events, including trade tensions between the U.S. and its global trading partners, including China. Sanctions imposed by the U.S. and other countries in response to conflicts may also adversely impact the financial markets and the global economy, and any economic countermeasures by the affected countries or others could exacerbate market and economic instability. We have conducted business outside of the U.S. in the past and may do so in the future. Clinical trials for our product candidates outside of the U.S. and unfavorable economic conditions resulting in the weakening of the U.S. dollar would make those clinical trials more costly to operate. Furthermore, a severe or prolonged economic downturn, higher inflation and interest rates, political disruption or other geopolitical events, including an expansion of current global military conflicts, could result in a variety of risks to our business, including weakened demand for our product candidates or any future product candidates, if authorized or approved, and our ability to raise additional capital when needed on acceptable terms, if at all.
A weak or declining economy or political disruption, including any international trade disputes, or changes in laws or policies governing the terms of international trade, and in particular increased trade restrictions, tariffs or taxes on imports from countries where we manufacture products could strain our manufacturers or suppliers, possibly resulting in supply disruption or increased manufacturing and distribution costs. For example, the U.S. has imposed or threatened tariffs on certain imports from Canada, Mexico and China. Historically, tariffs have led to increased trade and political tensions. In response to tariffs, other countries have implemented retaliatory tariffs on U.S. goods. Political tensions as a result of trade policies and tariff uncertainty could reduce trade volume, investment, technological exchange and other economic activities between major international economies, resulting in a material adverse effect on global economic conditions and the stability of global financial markets.
We face risks related to the personal data we collect, process, and share.
Our ability to conduct our business is dependent on the data that we collect, process, and share in discovering, developing, and commercializing drug products. These data are often considered personal data and are therefore regulated by data privacy laws in the United States and abroad.
Several U.S. states have proposed and passed consumer privacy laws. For example, the California Consumer Privacy Act of 2018, as amended by the California Privacy Rights Act, includes certain transparency and other requirements to protect personal data and grants California consumers with certain rights regarding their personal data. In addition, California consumers have the right to bring a private right of action in connection with data security incidents involving certain elements of personal data. Additionally, other jurisdictions have enacted similar legislation and/or regulations. Health-specific consumer privacy laws have also passed in multiple states.
At the federal level, the Federal Trade Commission (the “FTC”) sets expectations for taking appropriate steps to safeguard consumers’ personal information and providing a level of privacy or security commensurate to promises made to individuals. The FTC expects a company’s data privacy and security measures to be reasonable and appropriate in light of the sensitivity and volume of consumer information it holds, the size and complexity of its business, and the cost of available tools to help manage risks. Failure to meet these standards may constitute unfair or deceptive acts or practices in violation of Section 5 of the FTC Act. Enforcement by the FTC under the FTC Act can result in civil penalties or enforcement actions. The FTC also has the power to enforce the Health Breach Notification Rule, which imposes notification obligations on companies for breaches of certain health information contained in personal health records. The FTC has brought enforcement actions under both Section 5 of the FTC Act and the Health Breach Notification Rule.
Additionally, we have conducted business outside the U.S. in the past and may do so in the future. These activities subject us to additional data protection authority oversight and require us to comply with stringent local and regional data privacy laws, including the GDPR. The GDPR has a wide range of compliance obligations relating to the processing and protection of personal data, including obligations to having a lawful basis for processing personal data (which may in certain situations require explicit consent of data subjects), providing detailed information about the processing activities, dealing with restrictions on sharing of personal data with third parties and the transferring of personal data out of the EU, having contractual arrangements in place where required (such as with clinical trial sites and vendors), reporting in certain instances personal data breaches to data protection authorities and/or affected individuals, appointing data protection officers, conducting data protection impact assessments, responding to privacy rights requests and keeping records of processing activities. Violations of the GDPR carry significant financial penalties for noncompliance (including possible fines of up to 4% of global annual turnover for the preceding financial year or €20 million, whichever is higher). The GDPR also confers a private right of action on data subjects and consumer associations to lodge complaints with data protection authorities, seek judicial remedies, and obtain compensation for damages resulting from violations of the GDPR.
In addition to the GDPR, EU Member States have issued their own implementation legislation and other privacy, data protection and ePrivacy legislation. Moreover, other countries are issuing privacy and data protection legislation, generally modeled after the GDPR. These privacy and data protection laws and regulations increase our responsibility and liability in relation to personal data that we process, and compliance has been and is expected to continue to be difficult, constantly evolving, costly and time consuming. We may be required to expend significant capital and other resources to ensure ongoing compliance with applicable privacy and data protection laws, to protect against security incidents, or to alleviate issues caused by such incidents. In addition, if our efforts to comply with applicable privacy and data protection laws and regulations are not successful, it could adversely affect our business. Failure to comply with such laws and regulations could result in government enforcement actions and create liability for us, including but not limited to imposition of significant penalties, private litigation (including class actions) and/or adverse publicity that could negatively affect our business.
The landscape of federal, state, local and foreign privacy laws regulating personal data is constantly evolving, and compliance with these laws requires a flexible privacy framework and substantial resources. Compliance efforts will likely be an increasing and substantial cost in the future and may impose limitations on our business activities. If we fail to comply with applicable requirements, we could be subject to a range of regulatory actions that could affect our ability to commercialize our products candidates. We may also be subject to fines, penalties or private actions in the event of non-compliance with such laws. Any threatened or actual government enforcement action could also generate adverse publicity and could result in additional regulatory oversight. Moreover, despite our efforts, the possibility of security incidents occurring cannot be eliminated entirely and there can be no assurance that any measures we take will prevent such events that could adversely affect our business due, in part, to the ever-changing threat landscape. Additionally, we rely on third party vendors and service providers to support various aspects of our business operations. These third parties may pose risks related to data security, compliance, and contractual obligations. A failure by a third party to adequately protect our data could have adverse consequences for our business and reputation.
We face risks related to the growth of artificial intelligence and data analytics.
AI is being adopted rapidly by the biotechnology industry. Companies like ours have been using AI, machine learning, and data analytics tools in aspects of their operations, including target identification, clinical trial design, and regulatory and operational support. Should we continue to adopt these tools, they may not perform as intended and may produce inaccurate, incomplete, or biased outputs. Any such deficiencies could adversely affect our development decisions, clinical trial execution, regulatory submissions, or commercialization strategy, and could result in increased costs, delays, or failure to obtain or maintain regulatory approval for our product candidates.
The regulatory framework governing the use of AI and automated tools in drug development is evolving and uncertain. Regulatory authorities, including the FDA, may impose new requirements or expectations regarding the validation, transparency, or use of such technologies, including in connection with NDAs. Any failure to comply with applicable requirements or to adequately document and validate our use of these tools could delay or adversely affect regulatory review or approval of our product candidates.
In addition, our use of AI depends on third-party data, software, and infrastructure. We may be subject to risks relating to data quality, data access restrictions, intellectual property claims, and cybersecurity incidents. Any disruption, limitation, or compromise of these inputs could impair our ability to use such tools effectively and could adversely affect our business.
Finally, our competitors may adopt AI technologies more effectively than we do, which could enhance their development efficiency and competitive position relative to ours.
We may not
be able to maximize value from our de-prioritized drug candidates, GTx-102 and
GTx-101, through either out-licensing or sale.
Our GTx-102 and GTx-101 drug candidates are at an earlier development stage than GTx-104 and will require additional time and resources to develop. As part of our strategic realignment plan, we determined to focus primarily on the development of GTx-104 and to de-prioritize the development of GTx-102 and GTx-101. While we will continue to seek ways to maximize the value of GTx-102 and GTx-101, through out-licensing or sale, we may not be successful in doing so.
Risks Related to Development, Testing and Commercialization of Our Products
Clinical development is a lengthy and expensive process with an uncertain outcome, and results of earlier studies and trials may not be predictive of future trial results. Failure can occur at any stage of clinical development.
Clinical testing, even when utilizing the 505(b)(2) pathway, is expensive and can take many years to complete, and its outcome is inherently uncertain. Failure can occur at any time during the clinical trial process, even with active ingredients that have previously been approved by the FDA as safe and effective. The results of preclinical studies and early clinical trials of our drug candidates may not be predictive of the results of later stage clinical trials. A number of companies in the biopharmaceutical industry have suffered significant setbacks in advanced clinical trials due to lack of efficacy or adverse safety profiles, notwithstanding promising results in earlier trials.
Our drug candidates are in various stages of development. Clinical trial failures may occur at any stage and may result from a multitude of factors both within and outside our control, including flaws in formulation, adverse safety or efficacy profile and flaws in trial design, among others. If the trials result in negative or inconclusive results, we or our collaborators may decide, or regulators may require us, to discontinue trials of our drug candidates or conduct additional clinical trials or preclinical studies. In addition, data obtained from trials and studies are susceptible to varying interpretations, and regulators may not interpret our data as favorably as we do, which may delay, limit or prevent regulatory approval. For these reasons, our future clinical trials may not be successful.
Delays in clinical trials are common and have many causes, and any delay could result in increased costs to us and could jeopardize or delay our ability to obtain regulatory approval and commence drug product sales. We may also find it difficult to enroll patients in our clinical trials, which could delay or prevent development of our drug candidates.
We may experience delays in clinical trials of our drug candidates. Our planned clinical trials may not begin on time, have an effective design, enroll a sufficient number of patients or be completed on schedule, if at all. Our clinical trials can be delayed for a variety of reasons, including:
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inability to raise or delays in raising funding necessary to initiate or continue a trial;
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delays in obtaining regulatory approval to commence a trial;
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delays in reaching agreement with the FDA on final trial design;
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imposition of a clinical hold for safety reasons or following an inspection of our clinical trial operations or trial sites by the FDA or other regulatory authorities;
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delays in reaching agreement on acceptable terms with prospective CMOs, CROs, and clinical trial sites, or failure by such CMOs to complete the manufacturing of clinical trial materials or CROs to follow and carry out the clinical study protocol at each site in accordance with the terms of our agreements with them;
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delays in obtaining required IRB, approval at each site;
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difficulties or delays in having patients’ complete participation in a trial or return for post-treatment follow-up;
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clinical sites electing to terminate their participation in one of our clinical trials, which would likely have a detrimental effect on subject enrollment;
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time required to add new clinical sites; or
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delays by our CMOs to produce and deliver sufficient supply of clinical trial materials.
If initiation or completion of any clinical trials is delayed for any of the above reasons or other reasons, our development costs may increase, the regulatory approval process could be delayed and our ability to commercialize and commence sales of our drug candidates could be materially harmed, which could have a material adverse effect on our business.
In addition, identifying and qualifying patients to participate in clinical trials of our drug candidates is critical to our success. The timing of our clinical trials depends on the speed at which we can recruit patients to participate in testing our drug candidates as well as completion of required follow-up periods. We may not be able to identify, recruit and enroll a sufficient number of patients, or those with required or desired characteristics or to complete our clinical trials in a timely manner. Patient enrollment is, and completion of the trials are affected by a variety of factors, including:
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severity and prevalence of the disease under investigation;
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design of the trial protocol;
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size of the patient population;
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eligibility criteria for the trial in question;
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perceived risks and benefits of the drug candidate under trial;
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proximity and availability of clinical trial sites for prospective patients;
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availability of competing therapies and clinical trials;
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efforts to facilitate timely enrollment in clinical trials;
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patient referral practices of physicians; and
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ability to monitor patients adequately during and after treatment.
The regulatory approval processes of the FDA and comparable foreign authorities are lengthy, time consuming and inherently unpredictable, and if we are ultimately unable to obtain regulatory approval for our drug candidates, our business will be substantially harmed.
The time required to obtain approval by the FDA and comparable foreign authorities is unpredictable but typically takes many years following the commencement of clinical trials and depends upon numerous factors, including the substantial discretion of the regulatory authorities. In addition, approval policies, regulations or the type and amount of clinical data necessary to gain approval may change during the course of a drug candidate’s clinical development and may vary among jurisdictions. It is possible that none of our existing drug candidates or any drug candidates we may seek to develop will ever obtain regulatory approval in the United States or other jurisdictions.
Our drug candidates could fail to receive regulatory approval for many reasons, including the following:
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the FDA or comparable foreign regulatory authorities may disagree that our changes to branded reference drugs meet the criteria for the 505(b)(2) regulatory pathway or foreign regulatory pathways;
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we may be unable to demonstrate to the satisfaction of the FDA or comparable foreign regulatory authorities that a drug candidate is safe and effective or comparable to its branded reference product for its proposed indication;
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the results of any clinical trials we conduct may not meet the level of statistical significance required by the FDA or comparable foreign regulatory authorities for approval;
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we may be unable to demonstrate that a drug candidate’s clinical and other benefits outweigh its safety risks;
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the FDA or comparable foreign regulatory authorities may fail to approve the manufacturing processes or facilities of third-party manufacturers with which we contract for clinical and commercial supplies; and
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the approval policies or regulations of the FDA or comparable foreign regulatory authorities may change significantly in a manner rendering our clinical data insufficient for approval.
This
lengthy approval process as well as the unpredictability of future clinical
trial results may result in us failing to obtain regulatory approval to market
our drug candidates, which would harm our business, results of operations and
prospects significantly. For example, in April 2026, we received a CRL from the
FDA in response to our NDA for GTx-104. The CRL referenced certain items
related to the CMC and other non-clinical sections of the application,
including items related to additional leachable data time points for commercial
product, non-clinical product toxicology risk assessments, and cGMP
deficiencies at our CMO. The development of our product candidates also may be
delayed by other events beyond our control. For example, actions to limit
federal agency budgets or personnel, may result in reductions to the FDA’s
budget, employees, and operations, as well as changes to FDA regulatory
programs, all of which may lead to slower response times and longer review
periods, potentially affecting our ability to progress development of our
product candidates, undergo regulatory inspections or obtain regulatory
approval for our product candidates.
We have limited experience using the 505(b)(2) regulatory pathway to submit an NDA or any similar drug approval filing to the FDA, and we cannot be certain that any of our drug candidates will receive regulatory approval. If we do not receive regulatory approvals for our drug candidates, we may not be able to continue our operations. Even if we successfully obtain regulatory approvals to market one or more of our drug candidates, our revenue will be dependent, to a significant extent, upon the size of the markets in the territories for which we gain regulatory approval. If the markets for patients or indications that we are targeting are not as significant as we estimate, we may not generate significant revenue from sales of such drug products, if approved.
If a product candidate causes undesirable side effects, then we may be unable to receive regulatory approval or market acceptance for such product candidate.
We may experience numerous unforeseen events during, or as a result of, the testing process that could delay or prevent the commercialization of any of our product candidates, including the occurrence of significant adverse events in clinical trials. Such significant adverse events could lead to clinical trial challenges, such as difficulties in patient recruitment, retention, and adherence, potential product liability claims, and possible trial termination by us, regulatory authorities, and/or an IRB or ethics committees. These types of adverse events and clinical trial challenges could delay or prevent regulatory approval of our product candidates. Significant adverse events may also lead regulatory authorities to require additional warnings on the label for such product, require us to conduct additional costly post-marketing studies or trials, require us to develop a REMS, among other possible requirements. If the product candidate has already been approved, such approval may be withdrawn. Any delay in, denial, or withdrawal of marketing approval for one of our product candidates will adversely affect our financial position. Even if our product candidates receive marketing approval, undesirable side effects may limit the product’s commercial viability. Patients may not wish to use our product, physicians may not prescribe our product, and our reputation may suffer. Any of these events may significantly harm our business and financial prospects.
Our drug development strategy relies heavily upon the 505(b)(2) regulatory pathway, which requires us to certify that we do not infringe upon third-party patents covering approved drugs. Such certifications often result in third-party claims of intellectual property infringement, the defense of which will be costly and time consuming, and an unfavorable outcome in any litigation may prevent or delay our development and commercialization efforts which would harm our business.
Litigation or other proceedings to enforce or defend intellectual property rights are often complex in nature, may be very expensive and time-consuming, may divert our management’s attention from other aspects of our business and may result in unfavorable outcomes that could adversely impact our ability to launch and market our drug candidates, or to prevent third parties from competing with our drug products and drug candidates.
In particular, our commercial success depends in large part on our avoiding infringement of the patents and proprietary rights of third parties for existing approved drug products. Because we intend to utilize the 505(b)(2) regulatory pathway for the approval of our drug products and drug candidates, we rely in whole or in part on studies conducted by third parties related to those approved drug products.
Because patent applications can take many years to issue, there may be currently pending or subsequently filed patent applications which may later result in issued patents that may be infringed by our drug products or drug candidates. If any third-party patents were held by a court of competent jurisdiction to cover aspects of our drug candidates, including the formulation, method of use, any method or process involved in the manufacture of any of our drug candidates, any molecules or intermediates formed during such manufacturing process or any other attribute of the final product itself, the holders of any such patents may be able to block our ability to commercialize our drug candidates unless we obtain a license under the applicable patents, or until such patents expire. In either case, such a license may not be available on commercially reasonable terms or at all.
Parties making claims against us may request and/or obtain injunctive or other equitable relief, which could effectively block our ability to further develop and commercialize one or more of our drug candidates on a temporary or permanent basis. Defense of these claims, regardless of their merit, would involve substantial litigation expense and would be a substantial diversion of employee resources from our business. In the event of a successful claim of infringement against us, we may have to pay substantial damages, including treble damages and attorneys’ fees for willful infringement, obtain one or more licenses from third parties, pay royalties or redesign our infringing products or manufacturing processes, which may be impossible or require substantial time and monetary expenditure. We cannot predict whether any such license would be available at all or whether it would be available on commercially reasonable terms. Furthermore, even in the absence of litigation, we may need to obtain licenses from third parties to advance our research, manufacture clinical trial supplies or allow commercialization of our drug candidates. We may fail to obtain any of these licenses at a reasonable cost or on reasonable terms, if at all. In that event, we would be unable to further develop and commercialize one or more of our drug candidates, which could harm our business significantly. We cannot provide any assurances that third-party patents do not exist which might be enforced against our products, resulting in either an injunction prohibiting our sales, or, with respect to our sales, an obligation on our part to pay royalties and/or other forms of compensation to third parties.
If the market opportunities for GTx-104 are smaller than we believe they are, our results of operations may be adversely affected, and our business may suffer.
We are focused on developing and commercializing products for rare and orphan diseases that have the potential to improve clinical outcomes by using our novel drug delivery technologies. Our projections of both the number of people who have these conditions, as well as the subset of people with these conditions who have the potential to benefit from treatment with GTx-104, are based on estimates. These estimates may prove to be incorrect and new studies or clinical trials may change the estimated incidence or prevalence of these conditions. The number of patients in the United States and elsewhere may turn out to be lower than expected, may not be otherwise amenable to treatment with GTx-104, or new patients may become increasingly difficult to identify or gain access to, all of which would adversely affect our results of operations and our business.
We are subject to uncertainty relating to healthcare reform measures and reimbursement policies which, if not favorable to our drug candidates, could hinder or prevent our drug candidates’ commercial success.
Our ability to commercialize our drug candidates successfully will depend in part on the extent to which governmental authorities, private health insurers and other third-party payors establish appropriate coverage and reimbursement levels for our drug candidates and related treatments. As a threshold for coverage and reimbursement, third-party payors generally require that drug products have been approved for marketing by the FDA. Third-party payors are increasingly imposing additional requirements and restrictions on coverage and limiting reimbursement levels for medical products. These restrictions and limitations influence the purchase of healthcare services and products. The cost containment measures that healthcare payors and providers are instituting, and the effect of any healthcare reform could significantly reduce our revenues from the sale of any approved drug. We cannot provide any assurances that we will be able to obtain third-party coverage or reimbursement for our drug candidates in whole or in part.
No uniform policy for coverage and reimbursement for products exists among third-party payors in the United States. The Medicare and Medicaid programs increasingly are used as models in the United States for how private payors and other governmental payors develop their coverage and reimbursement policies for drugs and biologics. Some payors may require pre-approval of coverage for new or innovative devices or drug therapies before they reimburse healthcare providers who use such therapies. Coverage and reimbursement for products can differ significantly from payor to payor, and one payor’s decision to cover a product does not ensure that other payors will also provide similar coverage. It is difficult to predict at this time what payors will decide with respect to the coverage and reimbursement for our products, if approved and commercialized.
Additionally, the process for determining whether a payor will provide coverage for a product is typically separate from the process for setting the price of such product or establishing the reimbursement rate that the payor will pay for the product once coverage is approved. As a result, the determination of coverage and reimbursement is often a time-consuming and costly process that will require the seller to provide scientific and clinical support for the use of the drug candidates to each payor separately, with no assurance that coverage and adequate reimbursement will be applied consistently or obtained in the first instance. Furthermore, rules and regulations regarding reimbursement change frequently, in some cases at short notice, and we believe that changes in these rules and regulations are likely.
In the United States, there have been a number of legislative and regulatory changes to the healthcare system in ways that could affect our future revenues and profitability and the future revenues and profitability of our potential customers. Under the prescription drug benefit, Medicare beneficiaries can obtain prescription drug coverage from private sector plans that are permitted to limit the number of prescription drugs that are covered in each therapeutic category and class on their formularies. If our products are not widely included on the formularies of these plans, our ability to market our products to the Medicare population could be harmed.
There also have been, and likely will continue to be, legislative and regulatory proposals at the federal and state levels directed at containing or lowering the cost of healthcare. We cannot predict the initiatives that may be adopted in the future. The continuing efforts of the government, insurance companies, managed care organizations and other payors of healthcare costs to contain or reduce costs of healthcare may adversely affect one or more of the following:
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our ability to set a price that we desire for our drug products, if any are approved;
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our ability to generate revenues and achieve profitability;
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the future revenues of our potential customers, suppliers and collaborators; and
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the availability of capital to us.
Any of these scenarios could harm our ability to market our products and generate revenues. It is also possible that other proposals having a similar effect will be adopted.
Recent and future changes in healthcare legislation and regulations may increase the difficulty and cost to obtain marketing approval for a drug candidate, increase the costs to commercialize an approved product, and adversely affect the price set for such product.
In the United States and other jurisdictions, there have been, and we expect there will continue to be, a number of legislative and regulatory changes to the healthcare system that could impact the future results of our operations. In particular, there have been and continue to be a number of initiatives at the U.S. federal and state levels with the stated objective to reduce healthcare costs and improve the quality of healthcare. For example, the Affordable Care Act substantially changed the way healthcare is financed by both governmental and private insurers. The Affordable Care Act and its implementation continue to evolve as a result of legislative, administrative, and judicial developments. Further changes remain possible, which may potentially negatively affect pricing, coverage, or reimbursement for any products, if approved.
In addition to the Affordable Care Act, U.S. governments continue to seek to adopt healthcare policies and reforms intended to curb healthcare costs, such as federal or state controls on payment for drugs (including under Medicare, Medicaid, and commercial health plans). For example, the Budget Control Act of 2011 resulted in aggregate reductions, or sequestration, of Medicare payments to providers.
The IRA requires, among other things, the U.S. Secretary of HHS to negotiate, with respect to Medicare units and subject to a specified cap, the price of a set number of certain high spend Medicare Part B and D drugs and biologicals per year, with prices taking effect starting in 2026. Failures to comply with requirements under the drug price negotiation program could subject us to an excise tax and/or a civil monetary penalty. The program excludes drugs with an orphan drug designation that are not approved for any indications unrelated to that designation. In addition, the IRA established a Medicare Part B inflation rebate scheme, under which manufacturers owe rebates to Medicare if, generally speaking, the average sales price of a Part B drug increases faster than the pace of inflation. The failure to timely pay an inflation rebate may result in a civil monetary penalty. The IRA and any other similar laws introduced in the future may result in additional reductions in Medicare and other healthcare funding, which could negatively affect our future revenues and results of operations.
The One Big Beautiful Bill Act, signed into law on July 4, 2025, is projected to decrease federal health care spending by approximately $1 trillion by reducing Medicaid spending and enrollment and making changes to federal Medicare spending.
Individual states in the United States have also become increasingly aggressive in seeking to pass legislation and implementing regulations designed to control pharmaceutical and biological product pricing. Such measures could harm our business, results of operations, financial condition, and prospects. For example, an emerging trend at the state level is the establishment of prescription drug affordability boards, some of which will prospectively permit certain states to establish upper payment limits for drugs that the state has determined to be “high-cost”. Additionally, some state legislatures have passed laws that regulate how manufacturers make the 340B ceiling price available on the market. We expect that additional state reform measures will be adopted in the future, any of which could limit the amounts that state governments will pay for healthcare products and services, which could result in reduced demand or lower pricing for any approved product, or additional pricing pressures.
We cannot predict the likelihood, nature, or extent of government regulation that may arise from future legislation or administrative action in the United States or any other jurisdiction. For example, there has been recent interest in incorporating so-called Most Favored Nation pricing into the U.S. healthcare system, under which prices for drugs in the United States could be tied to foreign reference prices through a mechanism that is not yet defined. There have been several Congressional inquiries and proposed and enacted federal and state legislation and regulatory initiatives designed to, among other things, bring more transparency to product pricing, evaluate the relationship between pricing and manufacturer patient programs, and reform government healthcare program reimbursement methodologies for drug products. If healthcare policies or reforms intended to curb healthcare costs are adopted or if we experience negative publicity with respect to pricing of our products or the pricing of pharmaceutical drugs generally, the prices that we charge for any approved products may be limited, our commercial opportunity may be limited and/or our revenues from sales of our products may be negatively impacted.
If we fail to comply with reporting and payment obligations under the Medicaid Drug Rebate program or other governmental pricing programs, if we participate in these programs, we could be subject to additional rebate requirements, penalties, or other sanctions, which could have a material adverse effect on our business, financial condition, results of operations, and growth prospects.
Under the Medicaid Drug Rebate program, a participating manufacturer is required to pay a rebate to each state Medicaid program for its covered outpatient drugs that are dispensed to Medicaid beneficiaries and paid for by the state Medicaid program as a condition of having federal funds being made available for our drugs under Medicaid and Medicare Part B. Those rebates are based on pricing data reported by the manufacturer on a monthly and quarterly basis to CMS, the federal agency that administers the Medicaid Drug Rebate program. If we fail to pay the required rebate amount or report pricing data on a timely basis, if we are found to have knowingly submitted any false pricing or product information to the government, if we fail to submit the required pricing data on a timely basis, or if we misclassify or misreport product information, we may be subject to civil monetary penalties. CMS could also decide to terminate our Medicaid drug rebate agreement, in which case federal payments may not be available under Medicaid or Medicare Part B for our covered outpatient drugs.
Federal law requires that a manufacturer also participate in the 340B Drug Pricing program in order for federal funds to be available for the manufacturer’s drugs under Medicaid and Medicare Part B. The 340B program requires participating manufacturers to agree to charge no more than the 340B “ceiling price” for the manufacturer’s covered outpatient drugs to specified “covered entities,” including community health centers and other entities that receive certain federal grants, as well as certain hospitals that serve a disproportionate share of low-income patients. The 340B ceiling price is calculated based on the information reported under the Medicaid Drug Rebate program. If we are found to have knowingly and intentionally charged 340B covered entities more than the statutorily mandated ceiling price, we could be subject to significant civil monetary penalties and/or such failure also could be grounds for HRSA to terminate our agreement to participate in the 340B program, in which case our covered outpatient drugs would no longer be eligible for federal payment under the Medicaid or Medicare Part B program.
Federal law also requires that manufacturers report to CMS, on a quarterly basis, average sales price information for certain categories of drugs that are paid under the Medicare Part B program. Manufacturers calculate average sales price based on a statutorily defined formula as well as regulations and guidance. CMS may use the reported information to determine payment rates for drugs under Medicare Part B. If we are found to have made a misrepresentation in the reporting of our average sales price, we may be subject to civil monetary penalties. In addition, if we fail to provide timely information or knowingly provide false information, then we may also be subject to significant civil monetary penalties.
In addition, manufacturers must pay refunds to Medicare for single source drugs or biologicals, or biosimilar biological products, reimbursed under Medicare Part B and packaged in single-dose containers or single-use packages for units of discarded drug reimbursed by Medicare Part B in excess of 10 percent of total allowed charges under Medicare Part B for that drug. A failure to pay refunds for discarded drugs under the discarded drug refund program could subject us to civil monetary penalties of 125 percent of the refund amount.
Pricing and rebate calculations are complex, vary across products and programs, and are often subject to interpretation by the manufacturer, governmental agencies, and courts. A manufacturer that becomes aware that its Medicaid reporting for a prior quarter was incorrect, or has changed as a result of recalculation of the pricing data, is obligated to resubmit corrected data up to three years after those data originally were due. Restatements and recalculations increase the costs for complying with the laws and policies governing the Medicaid Drug Rebate program and could result in an overage or underage in our rebate liability for past quarters. They also may affect the 340B ceiling price and therefore liability under the 340B program.
In order to be eligible to have its products paid for with federal funds under the Medicaid and Medicare Part B programs and purchased by the Department of Veterans Affairs (the “VA”), Department of Defense (“DoD”), Public Health Service, and Coast Guard (the Big Four agencies), and certain federal grantees, a manufacturer is required to participate in the VA Federal Supply Schedule pricing program, established under Section 603 of the Veterans Health Care Act of 1992. Under this program, the manufacturer is obligated to make its covered drugs available for procurement on an FSS contract and charge a price to the Big Four agencies that is no higher than the FCP, which is a price calculated pursuant to a statutory formula. The FCP is derived from a calculated price point called the Non FAMP, which the manufacturer calculates and reports to the VA on a quarterly and annual basis. Pursuant to applicable law, knowing provision of false information in connection with a Non-FAMP filing can subject a manufacturer to significant penalties for each item of false information. The FSS contract also contains extensive disclosure and certification requirements.
Under Section 703 of the National Defense Authorization Act for FY 2008, a manufacturer is required to pay quarterly rebates to DoD on utilization of its innovator products that are dispensed through DoD’s Tricare network pharmacies to Tricare beneficiaries. The rebates are calculated as the difference between the annual Non-FAMP and FCP for the calendar year that the product was dispensed.
A manufacturer that overcharges the government in connection with the FSS contract or Tricare Retail Pharmacy Rebate Program, whether due to a misstated FCP or otherwise, is required to refund the difference to the government. Failure to make necessary disclosures and/or to identify contract overcharges can result in allegations against us under the FCA and other laws and regulations.
Our commercial success depends upon attaining significant market acceptance of our drug candidates and drug products, if approved, among physicians, nurses, pharmacists, patients, the medical community and third-party payors.
Even if we obtain regulatory approval for our drug product candidates, our drug product candidates may not gain market acceptance among physicians, nurses, pharmacists, patients, the medical community or third-party payors, which is critical to commercial success. Market acceptance of our drug candidates and any drug product for which we receive approval depends on a number of factors, including:
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the timing of market introduction of the drug candidate or drug product as well as competitive products;
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the clinical indications for which the drug product is approved;
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the convenience and ease of administration to patients of the drug candidate or drug product;
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the potential and perceived advantages of such drug candidate over alternative treatments;
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the cost of treatment in relation to alternative treatments, including any similar generic treatments;
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the availability of coverage and adequate reimbursement and pricing by third-party payors and government authorities;
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relative convenience and ease of administration;
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any negative publicity related to our or our competitors’ drug products that include the same active ingredient;
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the prevalence and severity of adverse side effects, including limitations or warnings contained in a drug product’s FDA-approved labeling; and
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the effectiveness of our sales and marketing efforts.
If our drug candidates or drug products, if approved, fail to achieve an adequate level of acceptance by physicians, nurses, pharmacists, patients, and the medical community, we will be unable to generate significant revenues, and we may not become or remain profitable.
If we are unable to establish sales and marketing capabilities or enter into agreements with third parties to market and sell our drug candidates, we may be unable to generate any revenue.
Although we intend to establish a small, focused, specialty sales and marketing organization to promote GTx-104, if approved for marketing in the United States, we currently have no such organization and the cost of establishing and maintaining such an organization may exceed the benefit of doing so. We believe that GTx-102 could also be marketed by a small, focused, specialty sales and marketing organization if and when we decide to resume development of GTx-102. Given the size of its potential market, we anticipate that commercializing GTx-101 would require entering into a strategic partnership with a larger marketing partner, if GTx-101 is approved by the FDA for marketing, and the ability to find any such strategic partnership would be uncertain. If we are unable to establish adequate sales, marketing and distribution capabilities, whether independently or with third parties, we may not be able to generate sufficient product revenue and may not become profitable. We will be competing with many companies that currently have extensive and well-funded marketing and sales operations. Without an internal team or the support of a third-party to perform marketing and sales functions, we may be unable to compete successfully against more established companies.
Even if GTx-104 receives regulatory approval, we will still face regulatory difficulties.
Even if we obtain regulatory approval for GTx-104, it would be subject to ongoing requirements by the FDA and comparable foreign regulatory authorities governing the manufacture, quality control, further development, labeling, packaging, storage, distribution, safety surveillance, patient registry, import, export, advertising, promotion, recordkeeping and reporting of safety and other post-market information. The safety profile of GTx-104 will continue to be closely monitored by the FDA and comparable foreign regulatory authorities after approval. If new safety information becomes available after approval of GTx-104, the FDA or comparable foreign regulatory authorities may require labeling changes or establishment of a REMS or similar strategy, impose significant restrictions on GTx-104’s indicated uses or marketing, or impose ongoing requirements for potentially costly post-approval trials or post-market surveillance. We will also be subject to continued compliance with cGMP and cGCP requirements for any clinical trials that we conduct post-approval.
In addition, manufacturers of drug products and their facilities are subject to continual review and periodic inspections by the FDA and other regulatory authorities for compliance with cGMP and other regulations. If we or a regulatory authority discover previously unknown problems with a product, such as adverse events of unanticipated severity or frequency, or problems with the facility where the product is manufactured, a regulatory authority may impose restrictions on that product, the manufacturing facility or us, including requiring recall or withdrawal of the product from the market or suspension of manufacturing. If we, GTx-104 or the manufacturing facilities for GTx-104 fail to comply with applicable regulatory requirements, a regulatory authority may, among other things:
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issue warning letters or untitled letters;
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mandate modifications to promotional materials or require us to provide corrective information to healthcare practitioners;
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require us to enter into a consent decree, which can include imposition of various fines, reimbursements for inspection costs, required due dates for specific actions and penalties for noncompliance;
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seek an injunction or impose civil or criminal penalties or monetary fines;
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suspend or withdraw regulatory approval;
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suspend any ongoing clinical trials;
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refuse to approve pending applications or supplements to applications filed by us;
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suspend or impose restrictions on operations, including costly new manufacturing requirements; or
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seize or detain products, refuse to permit the import or export of products, or require us to initiate a product recall.
The occurrence of any event or penalty described above may inhibit or preclude our ability to commercialize GTx-104 and generate revenue.
The FDA’s and other regulatory authorities’ policies may change, and additional government regulations may be enacted. We cannot predict the likelihood, nature or extent of government regulation that may arise from future legislation or administrative action, either in the United States or abroad. If we are slow or unable to adapt to changes in existing requirements or the adoption of new requirements or policies, or if we are not able to maintain regulatory compliance, we may lose any marketing approval for GTx-104 that we may have obtained, and we may not achieve or sustain profitability, which would adversely affect our business, prospects, financial condition and results of operations.
Advertising and promotion of GTx-104, if approved by the FDA, will be heavily scrutinized by, among others, the FDA, the Department of Justice, the HHS OIG, state attorneys general, members of Congress and the public. The FDA and other federal regulatory agencies closely regulate the marketing and promotion of drugs through, among other things, standards and regulations for direct-to-consumer advertising, advertising and promotion to healthcare professionals, communications regarding unapproved uses, industry-sponsored scientific and educational activities, and promotional activities involving the Internet. A product cannot be promoted before it is approved. After approval, product promotion can include only those claims relating to safety and effectiveness that are consistent with the labeling approved by the FDA. Healthcare providers are permitted to prescribe drugs for “off-label” uses — that is, uses not approved by the FDA and not described in the product’s labeling — because the FDA does not regulate the practice of medicine. However, FDA regulations impose restrictions on manufacturers’ communications regarding off-label uses. Broadly speaking, a manufacturer may not promote a drug for off-label use, but under certain conditions may engage in non-promotional, balanced, scientific communication regarding off-label use. Failure to comply with applicable FDA requirements and restrictions in this area may subject a company to adverse publicity and enforcement action, including enforcement letters, inquiries and investigations, and civil and criminal sanctions by the FDA or other government agencies. In addition, advertising and promotion of GTx-104, if approved outside of the United States, will be heavily scrutinized by comparable foreign regulatory authorities.
In the United States, promoting GTx-104 for unapproved indications can also subject us to false claims litigation under federal and state statutes, and other litigation and/or investigation, which can lead to civil and criminal penalties and fines and agreements that materially restrict the manner in which we promote or distribute our drug products. These false claims statutes include the FCA, which allows any individual to bring a lawsuit against a pharmaceutical company on behalf of the federal government alleging submission of false or fraudulent claims, or causing to present such false or fraudulent claims, for payment by a federal program such as Medicare or Medicaid. If the government prevails in the lawsuit, the individual will share in any fines or settlement funds. If we do not lawfully promote our approved products, we may become subject to such litigation and/or investigation and, if we are not successful in defending against such actions, those actions could adversely affect our business prospects, financial condition and results of operations.
In the European Union, strict requirements and restrictions regarding advertising and promotion apply, the details of which may vary per European Union Member States. Violation of those rules could subject us to litigation, investigations and/or civil and criminal penalties, which could adversely affect our business, prospects, financial condition and results of operations.
Even if our drug candidates receive regulatory approval in the United States, we may never obtain regulatory approval or successfully commercialize our products outside of the United States.
Our business plan is highly dependent upon our ability to obtain regulatory approval to market and commercialize our lead drug candidate, GTx-104 in the United States. As GTx-104 is currently the focus of our drug development program, the failure to commercialize it would have a material adverse effect on our ability to execute on our business plan and generate revenue. In addition, even if we obtain U.S. regulatory approvals to commercialize GTx-104, we may not be able to do so in other international jurisdictions.
If we obtain approval to commercialize any approved drug products outside of the United States, a variety of risks associated with international operations could materially adversely affect our business.
If any of our drug candidates are approved for commercialization, we may enter into agreements with third parties to market these drug products outside the United States. We expect that we will be subject to additional risks related to entering into international business relationships, including:
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different regulatory requirements for drug approvals in foreign countries;
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reduced protection for intellectual property rights;
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unexpected changes in tariffs, trade barriers and regulatory requirements;
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economic weakness, including inflation, or political instability in particular foreign economies and markets;
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compliance with tax, employment, immigration and labor laws for employees living or traveling abroad;
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foreign taxes, including withholding of payroll taxes;
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foreign currency fluctuations, which could result in increased operating expenses and reduced revenue, and other obligations incident to doing business in another country;
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workforce uncertainty in countries where labor unrest is more common than in the United States;
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ability to secure third-party marketing and selling agreements outside of the United States;
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production shortages resulting from any events affecting raw material supply or manufacturing capabilities abroad; and
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business interruptions resulting from geopolitical actions, including war and terrorism, or natural disasters including earthquakes, typhoons, floods and fires.
We are required to obtain regulatory approval for each of our drug candidates in each jurisdiction in which we intend to market such products, and the inability to obtain such approvals would limit our ability to realize their full market potential.
To market drug products outside of the United States, we must comply with numerous and varying regulatory requirements of other countries regarding safety and efficacy. Clinical trials conducted in one country may not be accepted by regulatory authorities in other countries, and regulatory approval in one country does not mean that regulatory approval will be obtained in any other country. However, the failure to obtain regulatory approval in one jurisdiction may adversely impact our ability to obtain regulatory approval in another jurisdiction. Approval processes vary among countries and can involve additional product testing and validation and additional administrative review periods. Seeking foreign regulatory approval could result in difficulties and costs for us and require additional non-clinical studies or clinical trials which could be costly and time consuming. Regulatory requirements can vary widely from country to country and could delay or prevent the introduction of our drug products in those countries. If we fail to comply with regulatory requirements in international markets or to obtain and maintain required approvals, or if regulatory approval in international markets is delayed, our target market will be reduced and our ability to realize the full market potential of our drug products will be harmed.
If we are unable to differentiate our drug candidates from branded reference drugs or existing generic therapies for similar treatments, or if the FDA or other applicable regulatory authorities approve generic products that compete with any of our drug candidates, our ability to successfully commercialize our drug candidates would be adversely affected.
Although we believe that our drug candidates will be clinically differentiated from branded reference drugs and their generic counterparts, if any, it is possible that such differentiation will not impact our market position. If we are unable to achieve significant differentiation for our product candidates against other drugs, the opportunity for our product candidates to achieve premium pricing and be commercialized successfully would be adversely affected.
In addition to existing branded reference drugs and the related generic products, the FDA or other applicable regulatory authorities may approve generic products that compete directly with our drug candidates, if approved. Once an NDA, including a 505(b)(2) application, is approved, the product covered thereby becomes a “listed drug” which can, in turn, be cited by potential competitors in support of approval of an abbreviated new drug application (“ANDA”). The FDCA, FDA regulations and other applicable regulations and policies provide incentives to manufacturers to create modified, non-infringing versions of a drug to facilitate the approval of an ANDA for generic substitutes. These manufacturers might only be required to conduct a relatively inexpensive study to show that their product has the same active ingredient(s), dosage form, strength, route of administration and conditions of use or labeling as our product candidate and that the generic product is bioequivalent to ours, meaning it is absorbed in the body at the same rate and to the same extent as our drug product. These generic equivalents, which must meet the same quality standards as branded pharmaceuticals, would be significantly less costly than ours to bring to market and companies that produce generic equivalents are generally able to offer their drug products at lower prices. After the introduction of a generic competitor, a significant percentage of the sales of any branded drug product is typically lost to the generic drug product. Accordingly, competition from generic equivalents of our drug candidates would materially adversely impact our ability to successfully commercialize our drug candidates.
We face significant competition from other biotechnology and pharmaceutical companies, and our operating results will suffer if we fail to compete effectively.
The biopharmaceutical industry is intensely competitive and subject to rapid and significant technological change. We expect to have competitors both in the United States and internationally, including major multinational pharmaceutical companies, biotechnology companies and universities and other research institutions.
Many of our competitors have substantially greater financial, technical and other resources, such as larger research and development staff and experienced marketing and manufacturing organizations. If our competitors market products that are more effective, safer or less expensive than our drug products, if any, or that reach the market sooner than our drug products, if any, we may enter the market too late in the cycle and may not achieve commercial success. In addition, the biopharmaceutical industry is characterized by rapid technological change. If we fail to stay at the forefront of technological change, we may be unable to compete effectively. Technological advances or drug products developed by our competitors may render our drug products, if any, or drug candidates obsolete, less competitive or not economical.
We could incur substantial costs and disruption to our business and delays in the launch of our drug candidates if our competitors and/or collaborators bring legal actions against us, which could harm our business and operating results.
We cannot predict whether our competitors or potential competitors, may bring legal actions against us based on our research, development, and commercialization activities, as well as any drug candidates or drug products resulting from these activities, claiming, among other things, infringement of their intellectual property rights, breach of contract or other legal theories. If we are forced to defend any such lawsuits, whether they are with or without merit or are ultimately determined in our favor, we may face costly litigation and diversion of technical and management personnel. These lawsuits could hinder our ability to enter the market early with our drug candidates and thereby hinder our ability to influence usage patterns when fewer, if any, of our potential competitors have entered such market, which could adversely impact our potential revenue from such drug candidates. Some of our competitors have substantially greater resources than we do and could be able to sustain the cost of litigation to a greater extent and for longer periods of time than we could. Furthermore, an adverse outcome of a dispute may require us: to pay damages, potentially including treble damages and attorneys’ fees, if we are found to have willfully infringed a party’s patent or other intellectual property rights; to cease making, licensing or using products that are alleged to incorporate or make use of the intellectual property of others; to expend additional development resources to reformulate our products or prevent us from marketing a certain drug; and to enter into potentially unfavorable royalty or license agreements in order to obtain the rights to use necessary technologies. Royalty or licensing agreements, if required, may be unavailable on terms acceptable to us, or at all.
We are subject to numerous complex regulatory requirements and failure to comply with these regulations, or the cost of compliance with these regulations, may harm our business, or subject us to civil or criminal proceedings, investigations, or penalties and may limit our ability to commercialize our products.
The research, testing, development, manufacturing, quality control, approval, labeling, packaging, storage, record-keeping, promotion, advertising, marketing, distribution, possession and use of our drug candidates, among other things, are subject to regulation by numerous governmental authorities in the United States and elsewhere. The FDA regulates drugs under the FDCA, and implementing regulations. Non-compliance with any applicable regulatory requirements can result in refusal of the governmental authority to approve products for marketing, criminal prosecution and fines, warning letters, product recalls or seizure of products, total or partial suspension of production, prohibitions or limitations on the commercial sale of products or refusal to allow the entering into of federal and state supply contracts. FDA and comparable governmental authorities have the authority to withdraw product approvals that have been previously granted. In addition, the regulatory requirements relating to our drug candidates and drug products, if any, may change from time to time and it is impossible to predict what the impact of any such changes may be.
Our employees, independent contractors, principal investigators, consultants, commercial partners and vendors may engage in misconduct or other improper activities, including non-compliance with regulatory standards and requirements.
We are exposed to the risk that our employees, independent contractors, principal investigators, consultants, commercial partners, and vendors may engage in fraudulent conduct or other illegal activity. Misconduct by these parties could include intentional, reckless and/or negligent conduct that violates (1) the laws of the FDA and similar foreign regulatory bodies, including those laws requiring the reporting of true, complete, and accurate information to such regulatory bodies; (2) healthcare fraud and abuse laws of the United States and similar foreign fraudulent misconduct laws; and (3) laws requiring the reporting of financial information or data accurately. Specifically, the promotion, sales and marketing of health care items and services, as well as certain business arrangements in the healthcare industry are subject to extensive laws designed to prevent misconduct, including fraud, kickbacks, self-dealing and other abusive practices. These laws may restrict or prohibit a wide range of pricing, discounting, marketing, structuring and commission(s), certain customer incentive programs and other business arrangements generally. Activities subject to these laws also involve the improper use of information obtained in the course of patient recruitment for clinical trials. It is not always possible to identify and deter employee and other third-party misconduct. The precautions we take to detect and prevent inappropriate conduct may not be effective in controlling unknown or unmanaged risks or losses or in protecting us from governmental investigations or other actions or lawsuits stemming from a failure to comply with these laws. If any such actions are instituted against us, and we are not successful in defending ourselves, those actions could have a significant impact on our business, including the imposition of civil, criminal and administrative penalties, damages, monetary fines, possible exclusion from participation in Medicare, Medicaid and other federal healthcare programs, contractual damages, reputational harm, diminished profits and future earnings, and curtailment of our operations, any of which could adversely affect our ability to operate our business and our results of operations.
Risks Relating to Our Intellectual Property
If we are sued for infringing intellectual property rights of third parties, it will be costly and time consuming, and an unfavorable outcome in that litigation would have a material adverse effect on our business.
Our commercial success also depends upon our ability and the ability of our future collaborators to develop, manufacture, market and sell our drug candidates and to use our proprietary technologies without infringing the proprietary rights of third parties. Numerous U.S. and foreign issued patents and pending patent applications, which are owned by third parties, exist in the fields in which we are developing drug candidates. Because patent applications can take many years to issue, there may be currently pending applications, which may later result in issued patents that our product candidates or proprietary technologies may infringe. Similarly, there may be issued patents relevant to our drug candidates of which we are not aware.
There is a substantial amount of litigation involving patent and other intellectual property rights in the biotechnology and biopharmaceutical industries generally. In particular, the generic drug industry is characterized by frequent litigation between generic drug companies and branded drug companies. If a third-party claims that we infringe its intellectual property rights, we may face a number of issues, including, but not limited to:
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infringement and other intellectual property claims which, with or without merit, may be expensive and time-consuming to litigate and may divert our management’s attention from our core business;
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substantial damages for infringement, including, but not limited to, treble damages, punitive damages, loss of profits and attorneys’ fees, which we may have to pay if a court decides that the drug product or proprietary technology at issue infringes on or violates the third-party’s rights;
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if a license is available from the third-party, we may have to pay substantial royalties, fees and/or grant cross licenses to our technology; and
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redesigning our drug candidates or processes so they do not infringe, which may not be possible or may require substantial funds and time.
We have not conducted an extensive search of patents issued to third parties, and no assurance can be given that third-party patents containing claims covering our drug candidates, technology or methods do not exist, have not been filed, or could not be filed or issued. Because of the number of patents issued and patent applications filed in our technical areas or fields, we believe there is a significant risk that third parties may allege they have patent rights encompassing our products, technology, or methods. Other drug candidates that we may in-license or acquire could be subject to similar risks and uncertainties.
We may be subject to claims that our employees, consultants, or independent contractors have wrongfully used or disclosed alleged confidential information or trade secrets of their other clients or former employers to us.
As is common in the biotechnology and pharmaceutical industry, certain of our employees were formerly employed by other biotechnology or pharmaceutical companies, including our competitors or potential competitors. Moreover, we engage the services of consultants to assist us in the development of our drug candidates, many of whom were previously employed at or may have previously been or are currently providing consulting services to, other biotechnology or pharmaceutical companies, including our competitors or potential competitors. We may be subject to claims that these employees and consultants or we have inadvertently or otherwise used or disclosed trade secrets or other proprietary information of their former employers or their former or current customers. Litigation may be necessary to defend against these claims. Even if we are successful in defending against these claims, litigation could result in substantial costs and be a distraction to management. Any such litigation would be protracted, expensive, and potentially subject to an unfavorable outcome.
Our success depends in part upon our ability to protect our intellectual property for our branded products and drug candidates.
Our commercial success with respect to our drug products and drug candidates, depends on obtaining and maintaining patent protection in the United States and in other countries and trade secret protection for our drug candidates, proprietary technologies and their uses. Our ability to protect our drug products from unauthorized or infringing use by third parties depends in substantial part on our ability to obtain and maintain valid and enforceable patents.
Due to evolving legal standards relating to patentability, validity and enforceability of patents covering pharmaceutical inventions and the scope of claims made under these patents, our ability to maintain, obtain and enforce patents is uncertain and involves complex legal and factual questions. The patent positions of pharmaceutical and biotechnology companies can be highly uncertain and involve complex legal and factual questions for which important legal principles remain unresolved. Changes in either the patent laws or in the interpretations of patent laws in the United States and other countries may diminish the value and the scope of our intellectual property. Accordingly, we cannot predict the breadth of claims that may be allowed or enforced in our patents or in third-party patents.
The degree of future protection for our proprietary rights is uncertain. Only limited protection may be available and may not adequately protect our rights or permit us to gain or keep our competitive advantage. For example:
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we might not have been the first to file patent applications for these or similar inventions;
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we might not have been the first to make the inventions covered by each of our pending patent applications and issued patents;
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others may independently develop similar or alternative technologies or duplicate any of our technologies;
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it is possible that none of our or our licensors’ pending patent applications will result in issued patents;
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any patents we obtain, or our licensors’ issued patents may not encompass commercially viable products, may not provide us with any competitive advantages, or may be challenged by third parties for lack of novelty, obviousness, lack of demonstrated or predicted utility, or other technical reasons related to the drafting of the patent itself;
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any patents we obtain, or our in-licensed issued patents may not be valid or enforceable; or
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we may not develop additional proprietary technologies that are patentable.
Proprietary trade secrets and unpatented know-how are also very important to our business. Although we have taken steps to protect our trade secrets and unpatented know-how, including entering into confidentiality agreements with third parties, and confidential information and inventions agreements with certain of our employees, consultants, and advisors, third parties may still obtain this information, or we may be unable to protect our rights. Enforcing a claim that a third-party illegally obtained and is using our trade secrets or unpatented know-how is expensive and time consuming, and the outcome is unpredictable. In addition, courts outside the United States may be less willing to protect trade secret information. Moreover, our competitors may independently develop equivalent knowledge, methods, and know-how, and we would not be able to prevent their use.
If we fail to comply with our obligations in the agreements under which we license rights to technology from third parties, or if the license agreements are terminated for other reasons, we could lose license rights that are important to our business.
We may be a party to a number of technology licenses that are important to our business and expect to enter into additional licenses in the future. Our existing license agreements impose, and we expect that future license agreements will impose, on us, various development, regulatory and/or commercial diligence obligations, payment of milestones and/or royalties and other obligations. Under these agreements, we must rely on our licensor to comply with their obligations under the primary license agreements under which such third-party obtained rights in the applicable intellectual property, where we may have no relationship with the original licensor of such rights. If our licensors fail to comply with their obligations under these upstream license agreements, the original third-party licensor may have the right to terminate the original license, which may terminate our sublicense. If this were to occur, we would no longer have rights to the applicable intellectual property unless we are able to secure our own direct license with the owner of the relevant rights, which we may not be able to do at a reasonable cost or on reasonable terms, which may impact our ability to continue to develop and commercialize our drug candidates and companion diagnostic incorporating the relevant intellectual property. If we fail to comply with our obligations under our license agreements, or we are subject to a bankruptcy or insolvency, the licensor may have the right to terminate the license. In the event that any of our important technology licenses were to be terminated by the licensor, we would likely cease further development of the related program or be required to spend significant time and resources to modify the program to not use the rights under the terminated license.
We may be subject to claims challenging the inventorship or ownership of our patents and other intellectual property.
We may also be subject to claims that former employees, collaborators or other third parties have an ownership interest in our patents or other intellectual property. We may be subject to ownership disputes in the future arising, for example, from conflicting obligations of consultants or others who are involved in developing our drug candidates and companion diagnostic. Litigation may be necessary to defend against these and other claims challenging inventorship or ownership. If we fail in defending any such claims, in addition to paying monetary damages, we may lose valuable intellectual property rights, such as exclusive ownership of, or right to use, valuable intellectual property. Such an outcome could have a material adverse effect on our business. Even if we are successful in defending against such claims, litigation could result in substantial costs and be a distraction to management and other employees.
Intellectual property rights do not necessarily address all potential threats to our competitive advantage.
The degree of future protection afforded by our intellectual property rights is uncertain because intellectual property rights have limitations and may not adequately protect our business or permit us to maintain our competitive advantage. The following examples are illustrative:
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others may be able to make compounds that are similar to our drug candidates but that are not covered by the claims of the patents that we own or have exclusively licensed;
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we or our licensors or future collaborators might not have been the first to make the inventions covered by the issued patent or pending patent application that we own or have exclusively licensed;
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we or our licensors or future collaborators might not have been the first to file patent applications covering certain of our inventions;
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others may independently develop similar or alternative technologies or duplicate any of our technologies without infringing our intellectual property rights;
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it is possible that our pending patent applications will not lead to issued patents;
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issued patents that we own or have exclusively licensed may be held invalid or unenforceable as a result of legal challenges by our competitors;
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we may not develop additional proprietary technologies that are patentable; and
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the patents of others may have an adverse effect on our business.
Should any of these events occur, they could significantly harm our business, results of operations and prospects.
We may not be able to protect our intellectual property rights throughout the world.
Many companies have encountered significant problems in protecting and defending intellectual property rights in foreign jurisdictions. The legal systems of some countries, particularly certain developing countries, do not favor the enforcement of patents, trade secrets and other intellectual property protection, which could make it difficult for us to stop the infringement of our patents or marketing of competing products in violation of our proprietary rights generally. Proceedings to enforce our patent rights in foreign jurisdictions could result in substantial costs and divert our efforts and attention from other aspects of our business, could put our patents at risk of being invalidated or interpreted narrowly and our patent applications at risk of not issuing and could provoke third parties to assert claims against us. We may not prevail in any lawsuits that we initiate, and the damages or other remedies awarded, if any, may not be commercially meaningful. Accordingly, our efforts to enforce our intellectual property rights around the world may be inadequate to obtain a significant commercial advantage from the intellectual property that we develop or license.
If our estimates or judgments relating to our critical accounting policies for intangible assets prove to be incorrect, further impairment charges could result.
We carry a significant amount of
intangible assets on our consolidated balance sheet, associated with acquired
in process research and development. In the ordinary course of business,
circumstances may arise, including manifestation of any of the risks identified
in this section, that could result in further recognition that the carrying
values of our assets may not be recovered from future operations. Under such
circumstances, it is possible we may be required to further impair our asset
values to the extent that their remaining value after any such impairment can
be recovered by our business going forward. Intangible assets with an
indefinite useful life are subject to an impairment review at least annually. For
example, in June 2026, following continued evaluation of strategic priorities
and focus on GTx-104, we determined that we will not resume internal
development funding for GTx-102 or GTx-101 under our current operating plan.
Accordingly, we determined that the remaining carrying values of the GTx-102
IPR&D asset and the GTx-101 IPR&D asset are no longer recoverable on an
internal-development basis.
Risks Related to Our Dependence on Third Parties
If our current CMO is unable to timely resolve the deficiencies identified by the FDA in the CRL related to GTx‑104, we may be required to engage an alternative manufacturer, which could result in significant delays, increased costs, or our inability to obtain regulatory approval or commercialize GTx‑104 at all.
In April 2026, we received a CRL from the FDA in response to our NDA for GTx‑104 identifying certain cGMP deficiencies at our current CMO. Our ability to obtain regulatory approval for GTx‑104 is dependent, in significant part, on our CMO’s ability to timely and satisfactorily remediate the deficiencies identified in the CRL and demonstrate ongoing compliance with applicable regulatory requirements, including cGMP.
We do not control the day‑to‑day operations of our CMO, and the remediation of the deficiencies identified in the CRL may be complex, costly and time‑consuming and may require substantial cooperation and investment by our CMO. There can be no assurance that our CMO will be able to implement the corrective actions required by the FDA within a timeframe acceptable to the FDA, or at all.
If our current CMO is unable or unwilling to satisfactorily resolve the deficiencies identified in the CRL, we may be required to identify, engage and qualify an alternative CMO. Transitioning manufacturing to a new CMO would require significant time and resources, including technology transfer, validation activities, potential changes to manufacturing processes, and additional regulatory submissions and FDA review. The FDA may require additional studies, data or inspections before approving GTx‑104 that is manufactured by a new CMO, and any such requirements could further delay approval.
These activities could take a prolonged period of time or may not be successfully completed. Any such delays or failures could materially and adversely affect the timing or likelihood of regulatory approval of GTx‑104 and, if approved, our ability to commercially launch or supply GTx‑104 at all. As GTx‑104 is our lead product candidate, any such delay or failure would have a material adverse effect on our business, financial condition, results of operations and prospects.
We rely on third parties to manufacture commercial and clinical supplies of our drug candidates, and we intend to rely on third parties to manufacture commercial supplies of any approved drug products. The commercialization of any of our drug products could be stopped, delayed, or made less profitable if those third parties fail to provide us with sufficient quantities of active pharmaceutical ingredients, excipients, or drug products, or fail to do so at acceptable quality levels or prices or fail to maintain or achieve satisfactory regulatory compliance.
We do not own any manufacturing facilities, and we do not currently, and do not expect in the future, to independently conduct any aspects of our product manufacturing and testing, or other activities related to the clinical development and commercialization of our drug candidates. We currently rely, and expect to continue to rely, on third parties with respect to these items, and control only certain aspects of their activities.
Any of these third parties may terminate their engagements with us at any time. If we need to enter into alternative arrangements, it could delay our drug candidate development and commercialization activities. Our reliance on these third parties reduces our control over these activities but does not relieve us of our responsibility to ensure compliance with all required legal, regulatory, and scientific standards such as the requirement for our product candidates and any future commercialized products to be manufactured according to cGMP and any applicable product specifications. Additionally, the facilities used by any contract manufacturer to manufacture any of our product candidates must be the subject of a satisfactory inspection before the FDA approves the product candidate manufactured at that facility. As previously noted, we received a CRL from the FDA in April 2026 in response to our NDA for GTx-104, which identified certain cGMP deficiencies at our CMO. If our CMO or other third parties do not successfully carry out their contractual duties, meet expected deadlines or conduct our manufacturing in accordance with regulatory requirements or contractual requirements, we may not be able to complete, or may be delayed in completing, clinical trials required to support future regulatory submissions and approval, which would have a material adverse effect on our ability to commercialize of our drug candidates, if approved.
Certain changes in the manufacturing process or procedure, including a change in the location where the drug candidate is manufactured or a change of a third-party manufacturer, generally require prior FDA, or foreign regulatory authority, review and/or approval of the manufacturing process and procedures in accordance with current cGMP. We may need to conduct additional preclinical studies and clinical trials to support approval of such changes. This review may be costly and time-consuming and could delay or prevent the launch of a drug candidate.
More generally, manufacturers of pharmaceutical products often encounter difficulties in production, particularly in scaling up and validating initial commercial production. These problems include difficulties with production costs and yields, quality control, including stability of the product, quality assurance testing, shortages of qualified personnel, as well as compliance with strictly enforced federal, state, and foreign regulations. Additionally, our manufacturers may experience manufacturing difficulties due to resource constraints or as a result of labor disputes or unstable political environments. If our manufacturers were to encounter any of these difficulties, or otherwise fail to comply with their contractual obligations, our ability to make product candidates available for clinical trials and development purposes or to commercialize any of our product candidates in the United States would be jeopardized. If our contract manufacturers cannot successfully manufacture material that conforms to our specifications and the FDA’s cGMP requirements, and other requirements of any governmental agency whose jurisdiction to which we are subject, our product candidates will not be approved or, if already approved, may be subject to recalls or other negative actions. Additionally, any delay or interruption in our ability to meet commercial demand may result in the loss of potential revenues and could adversely affect our ability to gain market acceptance for approved products. In addition, any delay or interruption in the supply of clinical trial supplies could delay the completion of clinical trials, increase the costs associated with maintaining clinical trial programs and, depending upon the period of delay, require us to commence new clinical trials at additional expense or terminate clinical trials completely. Additionally, if supply from one approved manufacturer is interrupted, there could be a significant disruption in commercial supply. Regulatory agencies may also require additional studies if a new manufacturer is relied upon for commercial production. Accordingly, switching manufacturers or adding a new manufacturing site/manufacturer may involve substantial costs and is likely to result in a delay in our desired clinical and commercial timelines.
We rely on third parties to conduct our preclinical studies and clinical trials. If these third parties do not successfully carry out their contractual duties or meet expected deadlines, we may not be able to obtain regulatory approval for or commercialize our drug candidates, and our business could be substantially harmed.
We have relied upon and plan to continue to rely upon third-party CROs to monitor and manage data for our preclinical and clinical programs. We rely on these parties for execution of our preclinical studies and clinical trials, and control only certain aspects of their activities. Nevertheless, we are responsible for ensuring that each of our trials is conducted in accordance with the applicable protocol, legal, regulatory, and scientific standards and our reliance on the CROs does not relieve us of our regulatory responsibilities. We and our CROs are required to comply with FDA laws and regulations regarding cGCP, which are also required by the Competent Authorities of the Member States of the European Economic Area and comparable foreign regulatory authorities in the form of International Conference on Harmonization, guidelines for all of our drug candidates in clinical development. Regulatory authorities enforce cGCP through periodic inspections of trial sponsors, principal investigators, and trial sites. If we or any of our CROs fail to comply with applicable cGCP, the clinical data generated in our clinical trials may be deemed unreliable and the FDA or comparable foreign regulatory authorities may require us to perform additional clinical trials before approving our marketing applications. We cannot assure you that upon inspection by a given regulatory authority, such regulatory authority will determine that any of our clinical trials comply with cGCP regulations. In addition, our clinical trials must be conducted with product produced under cGMP regulations. While we have agreements governing the activities of our CROs and CMOs, we have limited influence over their actual performance. In addition, portions of the clinical trials for our drug candidates are expected to be conducted outside of the United States, which will make it more difficult for us to monitor CROs and perform visits of our clinical trial sites and will force us to rely heavily on CROs to ensure the proper and timely conduct of our clinical trials and compliance with applicable regulations, including cGCP. Failure to comply with applicable regulations in the conduct of the clinical trials for our drug candidates may require us to repeat clinical trials, which would delay the regulatory approval process.
We may not be successful in establishing or maintaining development and commercialization collaborations, and any partner may not devote sufficient resources to the development or commercialization of our drug candidates or may otherwise fail in development or commercialization efforts, which could adversely affect our ability to develop certain of our drug candidates and our financial condition and operating results.
Because developing pharmaceutical products, conducting clinical trials, obtaining regulatory approval, establishing manufacturing capabilities and marketing approved products are expensive, we are exploring collaborations with third parties outside of the United States that have more resources and experience. In situations where we enter into a development and commercial collaboration arrangement for a drug candidate, we may also seek to establish additional collaborations for development and commercialization in territories outside of those addressed by the first collaboration arrangement for such drug candidate. There are a limited number of potential partners, and we expect to face competition in seeking appropriate partners. If we are unable to enter into any development and commercial collaborations and/or sales and marketing arrangements on acceptable terms, if at all, we may be unable to successfully develop and seek regulatory approval for our drug candidates and/or effectively market and sell future approved drug products, if any, in all of the territories outside of the United States where it may otherwise be valuable to do so.
Even if we are able to establish collaboration arrangements, any such collaboration may not ultimately be successful, which could have a negative impact on our business, results of operations, financial condition and prospects. If we partner with a third-party for development and commercialization of a drug candidate, we can expect to relinquish some or all of the control over the future success of that drug candidate to the third-party. It is possible that a partner may not devote sufficient resources to the development or commercialization of our drug candidate or may otherwise fail in development or commercialization efforts, in which event the development and commercialization of such drug candidate could be delayed or terminated, and our business could be substantially harmed. In addition, the terms of any collaboration or other arrangement that we establish may not prove to be favorable to us or may not be perceived as favorable, which may negatively impact the trading price of our common stock. In some cases, we may be responsible for continuing the development of a product candidate or research program under a collaboration, and the payment we receive from our partner may be insufficient to cover the cost of this development. Moreover, collaborations and sales and marketing arrangements are complex, and time-consuming to negotiate, document and implement, and they may require substantial resources to maintain.
We are subject to a number of additional risks associated with our dependence on collaborations with third parties, the occurrence of which could cause our collaboration arrangements to fail. Conflicts may arise between us and our partners, such as conflicts concerning the interpretation of clinical data, the achievement of milestones, the interpretation of financial provisions or the ownership of intellectual property developed during the collaboration. If any such conflicts arise, a partner could act in its own self-interest, which may be adverse to our interests. Any such disagreement between us and a partner could result in one or more of the following, each of which could delay or prevent the development or commercialization of our drug candidates and harm our business:
●
reductions in the payment of royalties or other payments we believe are due pursuant to the applicable collaboration arrangement;
●
actions taken by a partner inside or outside our collaboration which could negatively impact our rights or benefits under our collaboration; and
●
unwillingness on the part of a partner to keep us informed regarding the progress of its development and commercialization activities or to permit public disclosure of the results of those activities.
Risks
Related to Tax
There is a significant risk that we may have been classified as a PFIC
for U.S. federal income tax purposes and that such PFIC status may taint our
common shares owned by U.S. Holders prior to the Domestication.
Following
the Domestication, we are now a U.S. corporation for U.S. federal income tax
purposes and thus, we generally cannot be treated as PFIC in taxable years
following the Domestication. However, investors who owned our common shares
prior to the Domestication and who are U.S. Holders (as defined below) should
be aware that, based on our financial statements and projections and given
uncertainty regarding the composition of our income and assets, there is a
significant risk that we may have been classified as a “passive foreign
investment company” or “PFIC” in prior years, including for the portion of our 2025 taxable year prior to the Domestication. Any U.S. Holder who held our
common shares prior to the Domestication may continue to be treated as holding
common stock in a PFIC even subsequent to the Domestication if such U.S. Holder
has not made certain elections to mitigate the adverse U.S. federal income tax
consequences of holding shares of a PFIC.
The
rules governing PFICs can have adverse tax effects on U.S. Holders, which
effects may be mitigated by making certain elections for U.S. federal income
tax purposes, which elections may or may not be available. If we were a PFIC in
any year, a U.S. Holder in such year would be required to file an annual
information return with the IRS on IRS Form 8621 to report distributions
received on their common shares, any gain realized on disposition of such
common shares and any other information required by such form. Additionally, if
we were classified as a PFIC in any taxable year with respect to which a U.S.
Holder owned common shares, we generally would continue to be treated as a PFIC
with respect to such U.S. Holder in all succeeding taxable years, unless the
U.S. Holder makes a “deemed sale election.”
Each
investor who owned our common shares prior to the Domestication and who is a
U.S. Holder should consult his, her or its own tax advisor regarding the U.S.
federal, state and local, and non-U.S. tax consequences of the acquisition,
ownership, and disposition of our common stock, the U.S. federal tax
consequences of the PFIC rules, and the availability of any election that may
be available to the holder to mitigate adverse U.S. federal income tax
consequences of holding shares of a PFIC, including on a go-forward basis.
For
purposes of this disclosure, a “U.S. Holder” is a beneficial owner of shares of
our Common Stock that, for U.S. federal income tax purposes, is (a) an
individual who is a citizen or resident of the United States, (b) a company, or
other entity classified as a company for U.S. federal income tax purposes, that
is created or organized in or under the laws of the United States, any state in
the United States or the District of Columbia, (c) an estate if the income of
such estate is subject to U.S. federal income tax regardless of the source of
such income, or (d) a trust if (i) such trust has validly elected to be treated
as a U.S. person for U.S. federal income tax purposes or (ii) a U.S. court is
able to exercise primary supervision over the administration of such trust and
one or more U.S. persons have the authority to control all substantial
decisions of such trust.
We may not be able to use our net operating loss carry forwards to
offset future taxable income for U.S. and Canadian federal income tax purposes.
At March 31,
2026, Grace U.S. (formerly Acasti Pharma U.S.) and Grace Therapeutics, Inc. had
net operating loss carry forwards (“NOLs”) for U.S. federal income tax purposes
of approximately $16.4 million and $24.1 million, respectively, which have no
expiry.
Grace
U.S. underwent an “ownership change” within the meaning of Section 382 of the U.S.
Internal Revenue Code of 1986, as amended (the “Code”) as a result of the 2021
merger with Grace Therapeutics, the privately held company, and therefore we
and/or Grace U.S. may be subject to an annual limit on the amount of NOLs that
may be used to offset our future taxable income or the future taxable income of
Grace U.S. for U.S. federal income tax purposes. Such annual limit is generally
equal to the product of (i) the total value of the loss company’s (in this
case, Grace U.S.) outstanding equity immediately prior to an “ownership change”
(subject to certain adjustments); and (ii) the applicable federal long-term
tax-exempt interest rate for the month that includes the “ownership change.”
At
March 31, 2026, we had NOLs for Canadian federal income tax purposes of
approximately $143.0 million, which
expire at various dates through 2043. The extent to which we can utilize any or
all of our Canadian NOLs will depend on many factors, including the
jurisdiction applicable to any of our future taxable revenue. In connection
with our shift of our operations to the United States, we may not be able to
justify the allocation of revenue to Canada sufficient to recover the tax
benefits arising from NOLs and other tax credits.
Our
ability to use NOLs will also depend on the amount of taxable income generated
in future periods. The NOLs may expire before we can generate sufficient
taxable income to use the NOLs.
The IRS may not agree that we should be treated as a foreign
corporation for U.S. federal tax purposes prior to the Domestication.
Although
prior to the Domestication, we were incorporated in Quebec, Canada, the IRS may
assert that since August 27, 2021, we should have been treated as a U.S.
corporation (and, therefore, a U.S. tax resident) for U.S. federal tax purposes
pursuant to Section 7874 of the Code. For U.S. federal tax purposes, a
corporation generally is considered a tax resident in the jurisdiction of its
organization or incorporation. Because prior to the Domestication we were an
entity incorporated in Canada, we generally have been classified as a foreign
corporation (and, therefore, not a U.S. tax resident) for U.S. federal tax
purposes at all times prior to the Domestication). However, Section 7874 of the
Code provides an exception under which a foreign corporation may, in certain
circumstances, be treated as a U.S. corporation for U.S. federal tax purposes.
Under Section 7874, if (1) in the 2021 merger with Grace Therapeutics, former Grace Therapeutics stockholders owned (within the meaning of Section 7874) 80% or more (by vote or value) of our ordinary shares after the merger by reason of holding Grace Therapeutics common stock (such ownership percentage, the "Section 7874 ownership percentage"), and (2) our "expanded affiliated group" did not have "substantial business activities" in Canada ("the substantial business activities test"), we would have been treated as a U.S. corporation for U.S. federal tax purposes following the 2021 merger with Grace Therapeutics. If the Section 7874 ownership percentage of the former Grace Therapeutics stockholders after the merger was less than 80% but greater than or equal to 60%, and the substantial business activities test was not met, we and our U.S. affiliates may have been in some circumstances, subject to certain adverse U.S. federal income tax provisions (which, among other things, could have limited their ability to utilize certain U.S. tax attributes such as NOLs to offset U.S. taxable income or gain resulting from certain transactions). Following the 2021 merger and prior to the Domestication, the application of these rules could have resulted in significant additional U.S. tax liability and limited our ability to restructure or access cash earned by certain of our non-U.S. subsidiaries, in each case, without incurring substantial U.S. tax liabilities.
Based on the terms of the merger, the rules for determining share ownership under Section 7874 and certain factual assumptions, we believe that former Grace Therapeutics stockholders owned (within the meaning of Section 7874) less than 60% (by both vote and value) of our ordinary shares after the merger by reason of holding shares of Grace Therapeutics common stock. Therefore, we believe that we should not have been treated as a U.S. corporation for U.S. federal tax purposes prior to the Domestication and that Section 7874 should otherwise not have applied to us or our affiliates as a result of the 2021 merger with Grace Therapeutics.
Risks Relating to Our Common Stock
We do not expect to pay any cash dividends for the foreseeable future.
The continued operation and expansion of our business will require substantial funding. Accordingly, we do not anticipate that we will pay any cash dividends on our common stock for the foreseeable future. Any determination to pay dividends in the future will be at the discretion of our board of directors and will depend upon our results of operations, financial condition, contractual restrictions, restrictions imposed by applicable law and other factors our board of directors deems relevant.
The price of our common stock may be volatile.
Market prices for securities of pharmaceutical companies can fluctuate significantly. Factors such as the announcement to the public or in various scientific or industry forums of technological innovations; new commercial products; patents or exclusive rights obtained by us or others; disputes or other developments relating to proprietary rights, including patents, litigation matters and our ability to obtain patent protection for our technologies; the commencement, enrollment or announcement of results of clinical trials we conduct, or changes in the development status of our drug candidates; results or delays of preclinical and clinical studies by us or others; any delay in our regulatory filings for our drug candidates and any adverse development or perceived adverse development with respect to the applicable regulatory authority’s review of such filings; a change of regulations; additions or departures of key scientific or management personnel; overall performance of the equity markets; general political and economic conditions; publications; failure to meet the estimates and projections of the investment community or that we may otherwise provide to the public; research reports or positive or negative recommendations or withdrawal of research coverage by securities analysts; actual or anticipated variations in quarterly operating results; announcements of significant acquisitions, strategic partnerships, joint ventures or capital commitments by us or our competitors; public concerns over the risks of pharmaceutical products; unanticipated serious safety concerns related to the use of our drug candidates or drug products; our access to financial resources; future sales of securities by us or our stockholders; and many other factors, many of which are beyond our control, could have considerable effects on the price of our common stock. The price of our common stock has fluctuated significantly in the past and there can be no assurance that the market price of our common stock will not experience significant fluctuations in the future.
In addition, securities of pharmaceutical companies often experience extreme price and volume fluctuations that are unrelated or disproportionate to the operating performance of those companies. Broad market and industry factors may negatively affect the market price of our common stock, regardless of our actual operating performance. In the past, securities class action litigation often has been instituted against pharmaceutical companies following periods of volatility in the market price of their securities. This type of litigation, if instituted against us, could result in substantial costs and a diversion of management’s attention and resources, which would harm our business, operating results or financial condition.
Raising additional capital may cause dilution to our existing stockholders, restrict our operations, or require us to relinquish rights to our technologies or drug candidates.
We will need to raise additional capital in the future in order to fully execute on our business plan. We may seek additional capital through a combination of public and private equity offerings, debt financing, and non-dilutive strategic partnerships and alliances and licensing arrangements. To the extent that we raise additional capital through the sale of equity or convertible debt securities, the ownership interests of our stockholders will be diluted, and the terms may include liquidation or other preferences that adversely affect the rights of our stockholders. The incurrence of indebtedness by us would result in increased fixed payment obligations and could involve certain restrictive covenants, such as limitations on our ability to incur additional debt, limitations on our ability to acquire or license intellectual property rights and other operating restrictions that could adversely impact our ability to conduct our business. If we raise additional funds through strategic partnerships and alliances and licensing arrangements with third parties, we may have to relinquish valuable rights to our technologies or drug candidates, or grant licenses on terms unfavorable to us.
We may pursue opportunities or transactions that adversely affect our business and financial condition and the public announcement of any of these or similar strategic opportunities or transactions might have a significant effect on the price of our common stock.
Our management, in the ordinary course of our business, regularly explores potential strategic opportunities and transactions. These opportunities and transactions may include strategic joint venture relationships, significant debt or equity investments in us by third parties, the acquisition or disposition of material assets, the licensing, acquisition or disposition of material intellectual property, the development of new drug candidates, the sale of our common stock and other similar opportunities and transactions. The public announcement of any of these or similar strategic opportunities or transactions might have a significant effect on the price of our common stock. Our policy is to not publicly disclose the pursuit of a potential strategic opportunity or transaction unless we are required to do so by applicable law, including applicable securities laws relating to periodic disclosure obligations. There can be no assurance that investors who buy or sell common stock are doing so at a time when we are not pursuing a particular strategic opportunity or transaction that, when announced, would have a significant effect on the price of our common stock.
In addition, any such future corporate development may be accompanied by certain risks, including exposure to unknown liabilities of the strategic opportunities and transactions, higher than anticipated transaction costs and expenses, the difficulty and expense of integrating operations and personnel of any acquired companies, disruption of our ongoing business, diversion of management’s time and attention, and possible dilution to stockholders. We may not be able to successfully overcome these risks and other problems associated with any future acquisitions and this may adversely affect our business and financial condition.
Not applicable.
We are increasingly dependent on third-party provided software applications and computing infrastructure to conduct key operations. We depend on both our own procured systems, networks, and technology as well as the systems, networks and technology of our contractors, consultants, vendors and other business partners.
We assess and manage cybersecurity
risks associated with third-party service providers through a range of
processes, including performing risk-based due diligence during onboarding, and
conducting periodic reassessments of key and higher-risk vendors based on
defined criticality and risk criteria.
We also seek to obtain and review
cybersecurity audit reports (e.g., SOC reports) where available and incorporate
appropriate security, data protection, and incident notification provisions
into our contracts. While these measures are designed to mitigate third-party
risks, they may not prevent all adverse events.
Process for Assessing, Identifying and Managing Material Risks from Cybersecurity Threats
In the event of a cybersecurity
incident, we maintain a Cybersecurity Incident Response Plan. Pursuant to the
plan and its escalation protocols, designated personnel are responsible for
assessing the severity of a cybersecurity incident and associated threat,
containing the threat, remediating the threat, including recovery of data and
access to systems, analyzing any reporting obligations associated with the cybersecurity
incident, and performing post-incident analysis and program enhancements, as
appropriate. We have a relationship with various law firms to assist with
advisory on legal aspects of addressing cybersecurity incidents and
communicating accordingly.
Governance
Management Oversight
Board Oversight
Cybersecurity Risks
We maintain a Risk Management Policy that governs the process in which we identify cybersecurity risks, evaluate their associated impacts and risk levels, and document them accordingly in the Cybersecurity Risk Register.
Our
head office and operations are located at 103 Carnegie Center Suite 300
Princeton, New Jersey, 08540.
In
the ordinary course of business, we are at times subject to various legal
proceedings and disputes. We assess our liabilities and contingencies in
connection with outstanding legal proceedings utilizing the latest information
available. Where it is probable that we will incur a loss and the amount of the
loss can be reasonably estimated, we record a liability in our consolidated
financial statements. These legal contingencies may be adjusted to reflect any
relevant developments on a quarterly basis. Where a loss is not probable or the
amount of loss is not estimable, we do not accrue legal contingencies. While
the outcome of legal proceedings is inherently uncertain, based on information
currently available and available insurance coverage, our management believes
that it has established appropriate legal reserves. However, it is possible
that the ultimate resolution of these matters, if unfavorable, may be material
to our financial position, results of operations, or cash flows. We are not
currently a party to any legal proceedings that, in the opinion of management,
are likely to have a material effect on our business.
Not applicable.
PART II
Market for Registrant’s Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities
Market Information
Shares of our common stock are traded on the Nasdaq Capital Market under the symbol “GRCE.”
Holders
As of June 15, 2026, there were 27 holders of record of our common stock. The actual number of our stockholders is greater than this number of record holders because most of our stockholders are beneficial owners whose shares are held in street name by brokers and other nominees.
Dividends
We do not anticipate paying any cash dividend on our common stock in the foreseeable future. We presently intend to retain any future earnings to finance the expansion and growth of our business. Any future determination to pay dividends will be at the discretion of our board of directors and will depend on our financial condition, results of operations, capital requirements and other factors the board of directors deems relevant. In addition, the terms of any future debt or credit facility may preclude us from paying dividends.
Recent Sales of Unregistered Securities
None.
Issuer Repurchases of Equity Securities
None.
The following discussion should be read in conjunction with our consolidated financial statements and notes thereto found elsewhere in this Annual Report on Form 10-K. This Annual Report on Form 10-K contains forward-looking statements within the meaning of the U.S. Private Securities Litigation Reform Act of 1995. You should review our Special Note Regarding Forward-Looking Statements presented at the beginning of this Annual Report on Form 10-K. These statements are subject to risks and uncertainties that could cause actual results and events to differ materially from those expressed or implied by such forward-looking statements. For a detailed discussion of these risks and uncertainties, see Item 1A, “Risk Factors” of this Annual Report on Form 10-K. We caution readers not to place undue reliance on these forward-looking statements, which reflect management’s analysis only as of the date of this Annual Report on Form 10-K. We undertake no obligation to update forward-looking statements which reflect events or circumstances occurring after the date of this Annual Report on Form 10-K, unless required by applicable securities laws.
Overview
This management’s discussion and analysis (“MD&A”) is presented in order to provide the reader with an overview of the financial results and changes to our financial position as of March 31, 2026 and for the year then ended. This MD&A explains the material variations in our operations, financial position and cash flows for the years ended March 31, 2026 and 2025.
Market data, and certain industry data and forecasts included in this MD&A were obtained from internal surveys and market research conducted by third parties hired by us, publicly available information, reports of governmental agencies and industry publications, and independent third-party surveys. We have relied upon industry publications as our primary sources for third-party industry data and forecasts. Industry surveys, publications and forecasts generally state that the information they contain has been obtained from sources believed to be reliable, but that the accuracy and completeness of that information are not guaranteed. We have not independently verified any of the data from third-party sources or the underlying economic assumptions they have made. Similarly, internal surveys, industry forecasts and market research, which we believe to be reliable based upon management or contracted third parties’ knowledge of our industry, have not been independently verified. Our estimates involve risks and uncertainties, including assumptions that may prove not to be accurate, and these estimates and certain industry data are subject to change based on various factors, including those discussed in this Annual Report on Form 10-K.
This MD&A should be read in conjunction with our consolidated financial statements for the years ended March 31, 2026 and 2025 included elsewhere in this Annual Report on Form 10-K.
Our annual financial statements, which include the accounts of our wholly owned subsidiary, have been prepared in accordance with U.S. GAAP and the rules and regulations of the SEC related to reports filed in Form 10-K. All intercompany transactions and balances are eliminated on consolidation.
All amounts appearing in the MD&A for the period-by-period discussions are in thousands of U.S. dollars, except share and per share amounts or unless otherwise indicated.
Our assets as
of March 31, 2026 include cash and cash equivalents of $16,977 and intangible
assets and goodwill of $49,266. Our current liabilities were $2,146 as of March
31, 2026 and were comprised primarily of amounts due to or accrued for
creditors.
In February 2025, we completed a
private placement of Company securities with certain institutional and
accredited investors. Net proceeds were $13,705. Refer to Note 7, Stockholders’
Equity - 2025 Private Placement, in the accompanying
consolidated financial statements elsewhere in this document for additional
information. We believe our existing cash and cash equivalents will be
sufficient to sustain planned operations through at least 12 months from the
issuance date of the consolidated financial statements included with this
Annual Report on Form 10-K.
Results of Operations
Comparison of the years ended March 31, 2026 and 2025
The following table summarizes our results of operations for the years ended March 31, 2026 and 2025:
|
Year ended
|
||||||||||||
|
March 31, 2026
|
March 31, 2025
|
Increase (Decrease)
| ||||||||||
| $ | $ | $ | ||||||||||
|
(in thousands)
|
||||||||||||
|
Operating expenses
|
||||||||||||
|
Research and
development expenses
|
2,405 | 9,511 | (7,106 | ) | ||||||||
|
General and
administrative expenses
|
8,672 | 7,168 | 1,504 | |||||||||
|
Loss from operating activities
|
(11,077 | ) | (16,679 | ) | (5,602 | ) | ||||||
|
Change in fair
value of derivative warrant liabilities
|
900 | 3,218 | (2,318 | ) | ||||||||
|
Interest and
other income, net
|
685 | 711 | (26 | ) | ||||||||
|
Foreign
exchange loss
|
(1 | ) | (17 | ) | (16 | ) | ||||||
|
Income tax
benefit
|
1,700 | 3,199 | (1,499 | ) | ||||||||
|
Net loss
|
(7,793 | ) | (9,568 | ) | (1,775 | ) | ||||||
Net Loss
The net loss
of $7,793 or $0.47 loss per share for the year ended March 31, 2026, decreased
by $1,775 from the net loss of $9,568 or $0.79 loss per share for the year
ended March 31, 2025. The decrease in net loss was primarily due to a $2,318
decrease in change in fair value of derivative warrant liabilities, a $7,106
decrease in research and development expenses, and a $1,499 decrease in income
tax benefit, partially offset by a $1,504 increase in general and
administrative expenses.
Research and development expenses
Research and development
expenses consist primarily of:
●
fees paid to external service providers such as CROs and CMOs related to clinical trials, including contractual obligations for clinical development, clinical sites, manufacturing and scale-up, and formulation of clinical drug supplies; and
●
salaries and related expenses for research and development personnel, including expenses related to stock options.
We
record research and development expenses as incurred.
Our research and development during the years ended March 31, 2026 and 2025 were focused primarily on our clinical development program for our GTx-104 drug candidate.
The following table summarizes our research and development expenses:
|
Research and development expenses
|
||||||||||||
|
Year ended
| ||||||||||||
|
March 31, 2026
|
March 31, 2025
|
Increase (Decrease)
| ||||||||||
| $ | $ | $ | ||||||||||
| (in thousands) | ||||||||||||
|
Total third-party research and development expenses1
|
938 | 8,486 | (7,548 | ) | ||||||||
|
Salaries and benefits
|
1,249 | 809 | 440 | |||||||||
|
Research and development expense before stock-based compensation and depreciation
|
2,187 | 9,295 | (7,108 | ) | ||||||||
|
Stock-based compensation
|
218 | 216 | 2 | |||||||||
|
Total
|
2,405 | 9,511 | (7,106 | ) | ||||||||
1Total third-party research and development expenses are calculated before salaries and benefits, depreciation, write-off of equipment and stock-based compensation.
Total research and development expenses for the year
ended March 31, 2026 were $2,405, compared to $9,511 for the year ended March
31, 2025. This decrease of $7,106 was primarily due to the decrease in research
activities for GTx-104 of $7,542 driven by the close-out of the GTx-104 pivotal
Phase 3 safety clinical trial during the first calendar quarter of 2026 offset
by a $436 increase in external consulting and data management costs incurred in
support of the NDA of GTx-104 which was submitted to the FDA in June 2025.
Salaries and benefits of $1,249 for the year ended March 31, 2026 increased by $440 compared to $809 for the year ended March 31, 2025. The increase was primarily due to salary and bonus increases of $350 as well as increased benefits costs of $55.
Stock-based compensation of $218 for the year ended March 31, 2026, increased by $2 compared to $216 for the year ended March 31, 2025.
General and administrative expenses
General and administrative expenses consist primarily of salaries and related benefits, including stock-based compensation, related to our executive, finance, legal, and support functions, including professional fees for auditing, tax, legal, consulting, rent and utilities and insurance.
|
General and administrative
expenses
|
||||||||||||
|
| Year ended | |||||||||||
|
March
31, 2026
|
March
31, 2025
|
Increase
(Decrease)
| ||||||||||
| $ | $ | $ | ||||||||||
|
(in
thousands)
|
||||||||||||
|
Professional
fees
|
4,254 | 3,455 | 799 | |||||||||
|
Salaries and
benefits
|
1,780 | 2,031 | (251 | ) | ||||||||
|
Other
|
2,051 | 1,161 | 890 | |||||||||
|
General and administrative expense before stock-based compensation and
depreciation1
|
8,085 | 6,647 | 1,438 | |||||||||
|
Stock-based
compensation
|
580 | 514 | 66 | |||||||||
|
Depreciation
and loss on disposal
|
7 | 7 | — | |||||||||
|
Total
|
8,672 | 7,168 | 1,504 | |||||||||
1 General and administrative sub-total expenses are calculated before stock-based compensation and depreciation.
General and
administrative expenses were $8,672 for the year ended March 31, 2026, an
increase of $1,504 from $7,168 for the year ended March 31, 2025. The increase
was primarily driven by $799 in
non-recurring legal and due diligence costs incurred in connection with
strategic initiatives evaluated during the period, as well as increased professional
fees and other general and administrative costs of $890 primarily related to
pre-commercial planning for GTx-104, offset in part by a decrease in salaries
and benefits of $251 primarily a result of decreased headcount. Stock-based
compensation of $580 for the year ended March 31, 2026, decreased by $66
compared to $514 for the year ended March 31, 2025. The increase was primarily
due to more stock option awards granted during the year ended March 31, 2026.
Change in fair value of derivative warrant liabilities
The decrease in the fair value of derivative warrant
liabilities for the year ended March 31, 2026 of $2,318 was mainly attributable
to the settlement of the warrant liability as the 2023 Common Warrants (defined
below) expired on October 21, 2025, which was the 60th day after the
date of the acceptance by the FDA of the NDA for our product candidate GTx-104.
Interest income
Interest and other income, net was $685 for the year ended March 31, 2026, compared to $711 for the year ended March 31, 2025. The $26 decrease in our interest and other income was due to a decrease in interest rates.
Income tax benefit
Income tax
benefit was $1,700 for the year ended March 31, 2026, compared to $3,199 for
the year ended March 31, 2025, reflecting a decrease of $1,499. The decrease
primarily resulted from updated drug commercialization timelines, which reduced
the projected future tax liability and generated a corresponding income tax
benefit of the same amount.
Liquidity and Capital Resources
Cash flows and financial condition for the years ended March 31, 2026 and March 31, 2025
Summary
As of March 31, 2026, cash and cash equivalents were $16,977, a net decrease of $5,156 compared to cash and cash equivalents of $22,133 at March 31, 2025.
In February 2025, we completed a private placement of our securities with certain institutional and accredited investors. Net proceeds to us were $13,705. Refer to Note 7, Stockholders’ Equity - 2025 Private Placement in the accompanying consolidated financial statements elsewhere in this document for additional information. We believe our existing cash and cash equivalents will be sufficient to sustain planned operations through at least 12 months from the issuance date of the consolidated financial statements included with this Annual Report on Form 10-K.
We will require additional capital to fund our daily operating needs beyond that time. We do not expect to generate revenue from product sales unless and until we successfully complete drug development and obtain regulatory approval, which is subject to significant uncertainty. To date, we have financed our operations primarily through public offerings and private placements of our common equity, warrants and convertible debt and the proceeds from research tax credits. Until such time that we can generate significant revenue from drug product sales, if ever, we will require additional financing, which is expected to be sourced from a combination of public or private equity or debt financing or other non-dilutive sources, including fees, milestone payments and royalties from collaborations with third parties. Arrangements with collaborators or others may require us to relinquish certain rights related to our technologies or drug product candidates. Adequate additional financing may not be available to us on acceptable terms, or at all. Our inability to raise capital as and when needed could have a negative impact on our financial condition and our ability to pursue our business strategy. We plan to raise additional capital in order to maintain adequate liquidity. Negative results from studies or trials, if any, or depressed prices of our Common Stock could impact our ability to raise additional financing. Raising additional equity capital is subject to market conditions that are not within our control.
Net cash used in operating activities
Net cash used
in operating activities for the year ended March 31, 2026 was $8,869, compared
to $14,904 for the year ended March 31, 2025, a decrease of $6,035. The
decrease in net cash used in operating activities was primarily due to a $7,106
decrease in research and development activities due to the completion of our
GTx-104 pivotal Phase 3 STRIVE-ON trial in the first fiscal quarter of 2026,
partially offset by a $1,504 increase in general and administrative expenses
for legal, consulting and other professional fees.
Net cash provided by investing activities
Net cash used in investing activities
for the year ended March 31, 2025, was $0 due to our purchase of short-term
investments of $15 and maturity of short-term investments of $15. There were no
investing activities for the year ended March 31, 2026.
Net cash provided by financing activities
Net cash
provided by financing activities for the year ended March 31, 2026 was $3,713,
compared to $14,032 for the year ended March 31, 2025, a decrease of $10,319.
The net cash provided by financing activities for the year ended March 31,
2026, was attributable to the $4,040 net proceeds received from the exercise of
the 2023 Common Warrants offset by payment of stock issuance costs $327 from
the 2023 Private Placement (defined below) which occurred in September 2023.
Net cash provided by financing activities of $14,032 for the year ended March
31, 2025, was primarily attributable to the $14,999 gross proceeds received
from the 2025 Private Placement (defined below) which occurred in February
2025, offset by stock issuance costs of $967.
2025 Private Placement
In February 2025, we agreed to offer
and sell in a private placement (the “2025 Private Placement”) an aggregate of
3,252,132 shares of Common Stock, at a purchase price of $3.395 per share of
Common Stock (the “2025 Private Placement Shares”), and pre-funded warrants to
purchase up to 1,166,160 shares of Common Stock, at a purchase price equal to
the purchase price per 2025 Private Placement Share less $0.0001 (the “2025
Pre-Funded Warrants”). Each 2025 Pre-Funded Warrant is exercisable for one
share of Common Stock at an exercise price of $0.0001 per share, is exercisable
immediately and will expire once exercised in full. For each 2025 Private
Placement Share and 2025 Pre-Funded Warrant issued, we agreed to issue to each
purchaser an accompanying warrant to purchase shares of Common Stock (or 2025
Pre-Funded Warrants in lieu thereof), exercisable for an aggregate of 4,418,292
shares of Common Stock (or 2025 Pre-Funded Warrants in lieu thereof) (the “2025
Common Warrants”). Each 2025 Common Warrant is exercisable for one share of
Common Stock at an exercise price of $3.395 per share, is immediately
exercisable and will expire on the earlier of (i) the 60th day after the date
the FDA approves the NDA for GTx-104 and (ii) September 25, 2028. The 2025
Private Placement closed on February 11, 2025. The net proceeds to us from the
2025 Private Placement were $13,705, after deducting fees and expenses.
2023 Private Placement
In September 2023, we entered into a
securities purchase agreement (the “Purchase Agreement”) with certain
institutional and accredited investors in connection with a private placement
offering of our securities (the “2023 Private Placement”). Pursuant to the
Purchase Agreement, we sold 1,951,371 Common Shares, at a purchase price of
$1.848 per Common Share and pre-funded warrants (the “2023 Pre-Funded
Warrants”) to purchase up to 2,106,853 Common Shares at a purchase price equal
to the purchase price per Common Share less $0.0001. Each 2023 Pre-Funded
Warrant is exercisable for one Common Share at an exercise price of $0.0001 per
Common Share, is immediately exercisable, and will expire once exercised in
full. Pursuant to the Purchase Agreement, we also issued to such institutional
and accredited investors common warrants (the “2023 Common Warrants”) to
purchase Common Shares, exercisable for an aggregate of 2,536,391 Common
Shares. Under the terms of the Purchase Agreement, for each Common Share and
each 2023 Pre-Funded Warrant issued in the 2023 Private Placement, an
accompanying five-eighths (0.625) of a Common Warrant was issued to the
purchaser thereof. Each whole Common Warrant was exercisable for one Common
Share at an exercise price of $3.003 per Common Share, was immediately
exercisable, and would expire on the earlier of (i) the 60th day after the date
of the acceptance by the FDA of an NDA for our product candidate GTx-104 and
(ii) five years from the date of issuance. The 2023 Private Placement closed on
September 25, 2023. The net proceeds to us from the 2023 Private Placement were
$7,338, after deducting fees and expenses. In October 2025, we received $4,040
in net proceeds from exercises of 1,345,464 2023 Common Warrants that were
issued in the 2023 Private Placement. for 1,345,464 shares of Common Stock. The
remaining 1,190,927 2023 Common Warrants issued in the 2023 Private Placement
expired on October 21, 2025, in accordance with their terms as the 60th day
after the FDA’s acceptance for review of our NDA for GTx-104 had passed.
Contractual Obligations and Commitments
Our contractual obligations and commitments primarily include trade payables, CMO and CRO agreements.
Research and development contracts and contract research organizations agreements
We utilize CMOs, for the development and production of clinical materials and CROs to perform services related to our clinical trials. Pursuant to the agreements with CMOs and CROs, we have either the right to terminate the agreements without penalties or under certain penalty conditions. As of March 31, 2026, we had $95 of commitments to CMOs and $28 of commitments to CROs for the next twelve months.
Contingencies
We evaluate contingencies on an ongoing basis and establish loss provisions for matters in which losses are probable, and the amount of the loss can be reasonably estimated.
Use of Estimates and Measurement of Uncertainty
The preparation of these consolidated financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets, liabilities, income, and expenses. Actual results may differ from these estimates.
Estimates are based on management’s best knowledge of current events and actions that management may undertake in the future. Estimates and underlying assumptions are reviewed on an ongoing basis. Revisions to accounting estimates are recognized in the period in which the estimates are revised and in any future periods affected.
Estimates and assumptions include the measurement of stock-based compensation, derivative warrant liabilities, accruals for research and development contracts and contract organization agreements, and valuation of intangibles and goodwill. Estimates and assumptions are also involved in determining which research and development expenses qualify for research and development tax credits and in what amounts. We recognize the tax credits once we have reasonable assurance that they will be realized.
While our significant accounting policies are described in the notes to our financial statements, we believe that the following critical accounting policies are most important for understanding and evaluating our reported financial results, as these policies relate to the more significant areas involving management’s judgments and estimates.
Critical Accounting Policies
Research and development costs
Research
and developments expenditures are expensed as incurred. These costs consist of
employees’ salaries and benefits related to research and development
activities, contractors and consultants that conduct our clinical trials,
laboratory material and small equipment, clinical trial materials, stock-based
compensation expense, and other non-clinical costs and regulatory fees. We
accrue research and development expenses based on work performed, which relies
on estimates of total costs incurred based on patient enrollment and completion
of patient studies, invoices received and contracted costs. Advance
payments for goods and services that will be used in future research and
development are recognized in prepaids or other assets and are expensed when
the services are performed, or the goods are used.
Valuation of Intangible Assets and Goodwill
In a business combination, the fair value of in-process research and development ("IPR&D") assets acquired is capitalized and accounted for as indefinite-lived intangible assets, and not amortized until the underlying project receives regulatory approval, at which point the intangible assets will be accounted for as definite-lived intangible assets or discontinued. If discontinued, the intangible assets will be written off. R&D costs incurred after the acquisition are expensed as incurred.
Our IPR&D and goodwill was $49,300
as of March 31, 2026, which represents 74% of total assets. Goodwill and
indefinite lived assets are not amortized but are subject to an impairment
review annually and more frequently when indicators of impairment exist. An
impairment of goodwill could occur if the carrying amount of a reporting unit
exceeds the fair value of that reporting unit. An impairment of
indefinite-lived intangible assets would occur if the fair value of the
intangible asset is less than the carrying value.
The nature of the assumptions in the intangible assets’ impairment tests are considered critical due to a high level of subjectivity and judgment necessary to account for highly uncertain matters, and the impact of the assumptions on our financial condition and our operating performance could be material.
We test goodwill for impairment by first assessing qualitative factors to determine whether it is more likely than not that the fair value is less than its carrying amount. If we conclude it is more likely than not that fair value of the reporting unit is less than its carrying amount, a quantitative impairment test is performed. We test indefinite lived intangible assets for impairment by first assessing qualitative factors to determine whether it is more likely than not that the fair value is less than its carrying amount. Events that could result in an impairment, or trigger an interim impairment assessment, include the decision to discontinue the development of a drug, the receipt of additional clinical or nonclinical data regarding our drug candidates or a potentially competitive drug candidates, changes in the clinical development program for a drug candidate, or new information regarding potential sales for the drug candidates and increases in our weighted average cost of capital.
Individual IPR&D projects and goodwill are tested for impairment on an annual basis in the fourth quarter, and in between annual tests if an event occurs or circumstances change that would more likely than not reduce the fair value of each technology or our reporting unit below its carrying value. No impairment of the identified intangible assets was recognized for the years ended March 31, 2026 and 2025.
Financial Instruments
Credit Risk
Financial instruments that potentially subject us to a concentration of credit risk consist primarily of cash and cash equivalents. Cash and cash equivalents are all invested in accordance with our investment policy with the primary objective being the preservation of capital and the maintenance of liquidity, which risk is managed by dealing only with highly rated U.S. and Canadian institutions. We maintain our cash and cash equivalents at accredited financial institutions in amounts that exceed federally insured limits. We do not believe that it is subject to unusual credit risk beyond the normal credit risk associated with commercial banking relationships.
Interest Rate Risk
Interest rate risk is the risk that the fair value or future cash flows of a financial instrument will fluctuate because of changes in market rates. Our exposure to interest rate risk as of March 31, 2026, was as follows:
|
Cash and cash equivalents
|
Short-term fixed interest rate
|
Our capacity to reinvest the short-term amounts with equivalent return will be impacted by variations in short-term fixed interest rates available on the market. Management believes the risk we will realize a loss as a result of the decline in the fair value of our short-term investments is limited because these investments have short-term maturities and are held to maturity.
Our contractual obligations related to financial instruments and other obligations and liquidity resources are presented in the liquidity and capital resources of this MD&A.
We expect to incur significant expenses and continued operating losses for the foreseeable future. We expect our expenses will increase substantially in connection with our ongoing activities, particularly as we advance clinical development for our drug candidates in our pipeline; continue to engage contract manufacturing organizations to manufacture our clinical study materials and to ultimately develop large-scale manufacturing capabilities in preparation for commercial launch; seek regulatory approval for our drug candidates; and add personnel to support our drug product development and future drug product launch and commercialization.
We believe our
existing cash and cash equivalents will be sufficient to sustain planned
operations through at least 12 months from the issuance date of the
consolidated financial statements included with this Annual Report on Form
10-K. We require additional capital to
fund our daily operating needs beyond that time. We plan to raise additional
capital prior to that time in order to maintain adequate liquidity. The
deficiencies identified in the CRL, negative results from studies, if any, and
depressed prices of our common stock could impact our ability to raise
additional financing. Raising additional equity capital is subject to market
conditions not within our control. If we do not raise additional funds in this
time period, we may not be able to realize our assets and discharge our
liabilities in the normal course of business.
Recent Accounting Pronouncements
In November 2024, the FASB issued ASU 2024-03, Income Statement—Reporting Comprehensive Income—Expense Disaggregation Disclosures (Subtopic 220-40) (“ASU 2024-03”), to improve the disclosures about a public business entity’s expenses and address requests from investors for more detailed information about the types of expenses (including purchases of inventory, employee compensation, depreciation, amortization and depletion) in commonly presented expense captions (such as cost of sales, SG&A and research and development).
ASU
2024-03 applies to all public business entities and is effective for annual
reporting periods beginning after December 15, 2026 and interim reporting
periods within annual reporting periods beginning after December 15, 2027. The
requirements will be applied prospectively with the option for retrospective
application. Early adoption is permitted. We are currently evaluating the
effect of adopting this new guidance on our consolidated financial statements
and disclosures.
We have considered recent accounting pronouncements and concluded that they are either not applicable to our business or that the effect is not expected to be material to our consolidated financial statements as a result of future adoption.
As a smaller reporting company, we are not required to provide this information.
See our consolidated financial statements beginning on page F-1 of this Annual Report on Form 10-K.
None.
As of the end of the period covered by
this Annual Report on Form 10-K, our management, with the participation of our
chief executive officer (“CEO”) and principal financial officer (“PFO”), has
performed an evaluation of the effectiveness of our disclosure controls and
procedures within the meaning of Rules 13a-15 (e) and 15d-15(e) of the Exchange
Act. Based upon this evaluation, our CEO and PFO concluded that, as of March
31, 2026, our existing disclosure controls and procedures were effective. It
should be noted that while the CEO and PFO believe that our disclosure controls
and procedures provide a reasonable level of assurance that they are effective,
they do not expect the disclosure controls and procedures to be capable of
preventing all errors and fraud. A control system, no matter how well conceived
or operated, can provide only reasonable, not absolute, assurance that the
objectives of the control system are met.
Management’s Report on Internal Controls over Financial Reporting
Our management, with the participation of our CEO and PFO, is responsible for establishing and maintaining adequate internal control over financial reporting. Our internal control system was designed to provide reasonable assurance regarding the reliability of financial reporting and the preparation and fair presentation of our consolidated financial statements. All internal control systems, no matter how well designed, have inherent limitations. Therefore, even those systems determined to be effective may not prevent or detect misstatements and can provide only reasonable assurance with respect to financial statement preparation and presentation. Also, projections of any evaluation of effectiveness to future periods are subject to the risk that controls may become inadequate because of changes in conditions, or that the degree of compliance with the policies or procedures may deteriorate. Our management conducted an assessment of the design and operation effectiveness of our internal control over financial reporting as of March 31, 2026. In making this assessment, we used the criteria established within the Internal Control—Integrated Framework (2013) issued by the Committee of Sponsoring Organizations of the Treadway Commission. Based on this assessment, our management has concluded that, as of March 31, 2026, our internal control over financial reporting was effective.
Changes in Internal Control over Financial Reporting
No changes were made to our internal controls over financial reporting that occurred during the fiscal quarter ended March 31, 2026 that have materially affected, or are reasonably likely to materially affect, our internal controls over financial reporting.
We are a non-accelerated filer under the Exchange Act and not required to comply with the auditor attestation requirements of Section 404(b) of the Sarbanes-Oxley Act of 2002. Therefore, this Annual Report on Form 10-K does not include an attestation report of our independent registered public accounting firm regarding our management’s assessment of internal control over financial reporting.
(a) On June 17, 2026, following continued
evaluation of strategic priorities and focus on GTx-104, our Board of Directors
determined that we will not resume internal development funding for GTx-102 or
GTx-101 under our current operating plan. Accordingly, we determined that the remaining carrying values of the GTx-102 IPR&D asset
and the GTx-101 IPR&D asset are no longer recoverable on an
internal-development basis.
As of March 31, 2026, the remaining capitalized carrying
value of GTx-102 IPR&D was $9.2 million and the remaining capitalized
carrying value of GTx-101 IPR&D was $4.3 million. We expect to recognize an
impairment charge for the full remaining carrying values of the GTx-102
IPR&D asset and the GTx-101 IPR&D asset in the first quarter of fiscal
year 2027. The Company does not expect the impairment charges to result in
future cash expenditures.
(b) During the fiscal quarter ended
March 31, 2026, none of our directors or officers adopted terminated
None.
PART III
Code of Conduct and Ethics
The Board has adopted a written Code of Conduct and Ethics (the “Code of Conduct”) within the meaning of Item 406(b) of Regulation S-K. This Code of Conduct applies to our directors, officers and employees. A current copy of the Code of Conduct is posted on the Governance Documents section of the Investors page of our website, which is located at www.gracetx.com. We intend to satisfy the disclosure requirement under Item 5.05 of Form 8-K regarding any substantive amendment to, or waiver from, a provision of the Code of Conduct by posting such information on the website address and location specified above.
The additional information required by this Item will be set forth under the sections entitled “Information Regarding the Board of Directors and Corporate Governance” and “Executive Officers” in our 2026 Proxy Statement to be filed with the SEC within 120 days of March 31, 2026 and is incorporated by reference into this Annual Report on Form 10-K.
The information required by this Item will be set forth under the sections entitled “Executive Compensation” and “Director Compensation” in our 2026 Proxy Statement to be filed with the SEC within 120 days of March 31, 2026 and is incorporated by reference into this Annual Report on Form 10-K.
Security Ownership of Certain Beneficial Owners and Management and Related Stockholder Matters
The information required by this Item will be set forth under the sections entitled “Securities Authorized for Issuance under Equity Compensation Plans” and “Security Ownership of Certain Beneficial Owners and Management” in our 2026 Proxy Statement to be filed with the SEC within 120 days of March 31, 2026 and is incorporated by reference into this Annual Report on Form 10-K.
The information required by this Item will be set forth under the sections entitled “Transactions with Related Persons and Indemnification” and “Information Regarding the Board of Directors and Corporate Governance” in our 2026 Proxy Statement to be filed with the SEC within 120 days of March 31, 2026 and is incorporated by reference into this Annual Report on Form 10-K.
The information required by this Item will be set forth under the section entitled “Ratification of Appointment of Independent Registered Public Accounting Firm” in our 2026 Proxy Statement to be filed with the SEC within 120 days of March 31, 2026 and is incorporated by reference into this Annual Report on Form 10-K.
PART IV
(a)(1) Financial Statements—The consolidated financial statements included in Item 8 are filed as part of this Annual Report on Form 10-K.
(a)(2) Financial Statement Schedules—All schedules have been omitted because they are not applicable or required, or the information required to be set forth therein is included in the consolidated financial statements or notes thereto included in Item 8 of this Annual Report on Form 10-K.
(a)(3) Exhibits—The exhibits required by Item 601 of Regulation S-K are listed in paragraph (b) below.
(b) Exhibits—The exhibits listed on the Exhibit Index below are filed herewith or are incorporated by reference to exhibits previously filed with the SEC.
EXHIBITS INDEX
|
Exhibit
No.
|
Description
|
|
|
2.1
| ||
|
3.1
| ||
|
3.2
| ||
|
3.3
| ||
|
4.1*
| ||
| 4.2 | Description of Securities (incorporated by reference to Exhibit 4.2 to the Company’s Annual Report on Form 10-K filed with the SEC on June 23, 2025) | |
|
4.3
| ||
|
4.4
| ||
|
4.5
| ||
|
10.1+
| ||
|
10.2+
|
|
10.3+
|
||
|
10.4+
|
||
|
10.5+
|
||
|
10.6+
|
||
|
10.7+
|
||
|
10.8+
|
||
|
10.9
|
||
|
10.10
|
||
|
10.11
|
||
|
10.12
|
||
|
10.13+
|
||
|
10.14+
|
||
|
10.15+
|
| 10.16+ | ||
| 10.17+ | ||
| 10.18+ | ||
| 10.19+ | ||
| 10.20+ | ||
| 10.21+ | ||
| 10.22+ | ||
| 10.23+ | ||
| 10.24+ | ||
| 10.25* |
Separation Agreement by and between Carrie D’Andrea andthe Company, dated June 17, 2026
| |
| 19.1 | ||
| List of
Subsidiary | ||
| Consent of
KPMG LLP, an Independent Registered Public Accounting Firm | ||
| Certification
of Chief Executive Officer pursuant to Rule 13a-14(a) or 15d-14(a) of the
Securities Exchange Act of 1934 | ||
| Certification
of Principal Financial Officer pursuant to Rule 13a-14(a) or 15d-14(a) of the
Securities Exchange Act of 1934 | ||
| Certification
of the Chief Executive Officer pursuant to 18 U.S.C. Section 1350, as adopted
pursuant to Section 906 of the Sarbanes-Oxley Act of 2002 | ||
| Certification
of the Principal Financial Officer pursuant to 18 U.S.C. Section 1350, as
adopted pursuant to Section 906 of the Sarbanes-Oxley Act of 2002 |
|
97.1
|
|
Grace Therapeutics, Inc. Incentive Compensation Recoupment Policy (incorporated by reference to Exhibit 97.1 to the Company’s Annual Report on Form 10-K filed with the SEC on June 23, 2025) |
|
|
|
|
|
101.INS*
|
|
Inline XBRL Instance Document
|
|
|
|
|
|
101.SCH*
|
|
Inline XBRL Taxonomy Extension Schema
Document
|
|
|
|
|
|
101.CAL*
|
|
Inline XBRL Taxonomy Extension
Calculation Linkbase Document
|
|
|
|
|
|
101.DEF*
|
|
Inline XBRL Taxonomy Extension
Definition Linkbase Document
|
|
|
|
|
|
101.LAB*
|
|
Inline XBRL Taxonomy Extension Label
Linkbase Document
|
|
|
|
|
|
101.PRE*
|
|
Inline XBRL Taxonomy Extension
Presentation Linkbase Document
|
|
|
|
|
|
104*
|
|
Cover Page Interactive Data File
(formatted as Inline XBRL and contained in Exhibit 101)
|
* Filed or furnished
herewith.
+ Management contract,
compensatory plan or arrangement.
None.
Pursuant to the requirements of Section 13 or 15(d) of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned, thereunto duly authorized.
|
Dated: June 18, 2026
|
|
|
|
|
GRACE THERAPEUTICS, INC.
|
|
|
|
|
|
|
|
By:
|
/s/ Prashant Kohli
|
|
|
Name:
|
Prashant Kohli
|
|
|
|
Title: Chief Executive Officer
|
|
|
|
(Principal Executive Officer)
|
Pursuant to the requirements of the Securities Exchange Act of 1934, this report has been signed below by the following persons on behalf of the registrant and in the capacities and on the dates indicated.
| Signature | Title | Date | ||
| /s/ Prashant Kohli |
Chief Executive Officer and Director
|
June 18, 2026
|
||
| Prashant Kohli |
(Principal Executive Officer)
|
|||
| /s/ Robert DelAversano |
Principal Financial Officer
|
June 18, 2026
|
||
| Robert DelAversano |
(Principal Financial Officer and Principal Accounting Officer)
|
|||
| /s/ Brian Davis |
Director
|
June 18, 2026
|
||
| Brian Davis | ||||
| /s/ Vimal Kavuru |
Director
|
June 18, 2026
|
||
| Vimal Kavuru | ||||
| /s/ Edward Neugeboren |
Director
|
June 18, 2026
|
||
| Edward Neugeboren |
|
|
|
|
|
|
|
/s/ George Kottayil
|
|
Director
|
|
June 18, 2026
|
|
George Kottayil
|
|
|
|
|
GRACE THERAPEUTICS, INC.
Consolidated Financial Statements
For the years ended March 31, 2026 and 2025
| Report of Independent Registered Public Accounting Firm (KPMG LLP, Philadelphia, PA, PCAOB | F-1 |
| F-3 | |
| F-4 | |
| F-5 | |
| F-6 | |
| F-7 |
To the Stockholders and Board of Directors
Grace Therapeutics, Inc.:
Opinion on the Consolidated Financial Statements
We have audited the accompanying consolidated balance sheets of Grace Therapeutics, Inc. and subsidiary (the Company) as of March 31, 2026 and 2025, the related consolidated statements of loss and comprehensive loss, stockholders’ equity, and cash flows for the years then ended, and the related notes (collectively, the consolidated financial statements). In our opinion, the consolidated financial statements present fairly, in all material respects, the financial position of the Company as of March 31, 2026 and 2025, and the results of its operations and its cash flows for the years then ended, in conformity with U.S. generally accepted accounting principles.
Basis for Opinion
These consolidated financial statements are the responsibility of the Company’s management. Our responsibility is to express an opinion on these consolidated financial statements based on our audits. We are a public accounting firm registered with the Public Company Accounting Oversight Board (United States) (PCAOB) and are required to be independent with respect to the Company in accordance with the U.S. federal securities laws and the applicable rules and regulations of the Securities and Exchange Commission and the PCAOB.
We conducted our audits in accordance with the standards of the PCAOB. Those standards require that we plan and perform the audit to obtain reasonable assurance about whether the consolidated financial statements are free of material misstatement, whether due to error or fraud. The Company is not required to have, nor were we engaged to perform, an audit of its internal control over financial reporting. As part of our audits, we are required to obtain an understanding of internal control over financial reporting but not for the purpose of expressing an opinion on the effectiveness of the Company’s internal control over financial reporting. Accordingly, we express no such opinion.
Our audits included performing procedures to assess the risks of material misstatement of the consolidated financial statements, whether due to error or fraud, and performing procedures that respond to those risks. Such procedures included examining, on a test basis, evidence regarding the amounts and disclosures in the consolidated financial statements. Our audits also included evaluating the accounting principles used and significant estimates made by management, as well as evaluating the overall presentation of the consolidated financial statements. We believe that our audits provide a reasonable basis for our opinion.
Critical Audit Matter
The critical audit matter communicated below is a matter arising from the current period audit of the consolidated financial statements that was communicated or required to be communicated to the audit committee and that: (1) relates to accounts or disclosures that are material to the consolidated financial statements and (2) involved our especially challenging, subjective, or complex judgments. The communication of a critical audit matter does not alter in any way our opinion on the consolidated financial statements, taken as a whole, and we are not, by communicating the critical audit matter below, providing a separate opinion on the critical audit matter or on the accounts or disclosures to which it relates.
F-1
Going concern analysis
As discussed in Note 1 to the consolidated financial statements, the Company has incurred operating losses and negative cash flows from operations in each period since its inception. As of March 31, 2026, the Company had an accumulated deficit of $228.5 million. To date, the Company has financed its operations primarily through public offerings and private placements of its common equity, warrants and convertible debt and the proceeds from research tax credits. Until such time that the Company can generate significant revenue from drug product sales, if ever, it will require additional financing, which is expected to be sourced from a combination of public or private equity or debt financing or other non-dilutive sources. In April 2026, the Company received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) regarding its New Drug Application (NDA) for GTx-104 requiring remediation of specified deficiencies and resubmission of the NDA for regulatory approval. The Company believes its existing cash and cash equivalents will be sufficient to sustain planned operations, through at least one year from the issuance of the consolidated financial statements. The Company plans to raise additional capital in order to maintain adequate liquidity. Adequate additional financing may not be available to the Company on acceptable terms, or at all. The Company’s inability to raise capital as and when needed could have a negative impact on its financial condition and its ability to pursue its business strategy.
We identified the assessment of liquidity and the Company’s ability to continue as a going concern as a critical audit matter. A high degree of subjective auditor judgment was required to evaluate the Company’s forecasted cash flows used in its liquidity analysis due to uncertainty in certain assumptions used to estimate the cash flows. Specifically, auditor judgment was required to evaluate management’s estimated expenses associated with its plans to remediate the FDA’s specified deficiencies and resubmit the NDA for regulatory approval.
The following are the primary procedures we performed to address this critical audit matter. We performed sensitivity analyses on estimated expenses by evaluating the impact of changes in forecasted cash flows on the Company’s going concern assessment. We compared the Company’s historical forecasted cash flows to actual results to assess the Company’s ability to accurately forecast. We evaluated management’s estimated expenses associated with its plans to remediate the FDA’s specified deficiencies and resubmit the NDA for regulatory approval by (1) conducting interviews with management to gain an understanding of the Company’s overall strategy and regulatory plans related to its remediation of deficiencies, (2) evaluating the consistency of information used in management’s analysis with management’s plans for expense and working capital management activities presented to the Board of Directors and other public information disseminated by the Company, and (3) comparing the information used in management’s analysis with evidence obtained in other areas of the audit to evaluate whether it supported or contradicted the conclusions reached by management.
/s/ KPMG LLP
We have served as the Company’s auditor since 2023.
June 18, 2026
GRACE THERAPEUTICS, INC.
(Formerly ACASTI PHARMA INC.)
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March 31, 2026 |
March 31, 2025 |
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(Expressed in thousands except share data)
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$ | $ | ||||||
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Assets
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Current assets:
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Cash and cash equivalents
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Receivables
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Prepaid expenses
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Total current assets
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Equipment, net
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Intangible assets
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Goodwill
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Total assets
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Liabilities and stockholders’ equity
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Current liabilities:
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Trade and other payables
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Total current liabilities
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Derivative warrant liabilities
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Deferred tax liability
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Total liabilities
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Commitments and contingencies (Note 12)
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Stockholders’ equity:
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| Preferred stock, $ |
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| Common stock, $ |
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Additional paid-in capital
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Accumulated other comprehensive loss
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( |
) | ( |
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Accumulated deficit
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( |
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Total stockholders' equity
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Total liabilities and stockholders’ equity
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The accompanying notes are an integral part of these consolidated financial statements
GRACE THERAPEUTICS, INC.
(Formerly ACASTI PHARMA INC.)
|
Year Ended
March 31, 2026 |
Year Ended
March 31, 2025 |
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(Expressed in thousands, except share and per share data)
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$ | $ | ||||||
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Operating expenses
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Research and development expenses
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General and administrative expenses
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Loss from operating activities
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( |
) | ( |
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Foreign exchange loss
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( |
) | ( |
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Change in fair value of derivative warrant liabilities
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Interest and other income, net
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Total other income, net
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Loss before income tax benefit
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( |
) | ( |
) | ||||
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Income tax benefit
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Net loss and total comprehensive loss
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( |
) | ( |
) | ||||
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Basic and diluted loss per share
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( |
) | ( |
) | ||||
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Weighted-average number of shares outstanding
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The accompanying notes are an integral part of these consolidated financial statements
GRACE THERAPEUTICS, INC.
(Formerly ACASTI PHARMA INC.)
| Common stock | ||||||||||||||||||||||||
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(Expressed in thousands except share data)
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Number | Amount | Additional paid-in capital |
Accumulated other comprehensive loss |
Accumulated deficit | Total stockholders' equity | ||||||||||||||||||
| $ | $ | $ | $ | $ | ||||||||||||||||||||
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Balance, March 31, 2025
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( |
) | ( |
) | ||||||||||||||||||||
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Net loss
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— | ( |
) | ( |
) | |||||||||||||||||||
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Stock-based compensation
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— | |||||||||||||||||||||||
| Issuance of common stock upon exercise of pre-funded warrants | ||||||||||||||||||||||||
| Issuance of common stock upon exercise of common warrants | ||||||||||||||||||||||||
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Balance at March 31, 2026
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( |
) | ( |
) | ||||||||||||||||||||
| Common stock | ||||||||||||||||||||||||
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(Expressed in thousands except share data)
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Number | Amount |
Additional
paid-in capital |
Accumulated other comprehensive loss |
Accumulated deficit |
Total stockholders' equity |
||||||||||||||||||
| $ | $ | $ | $ | $ | ||||||||||||||||||||
| Balance, March 31, 2024 | ( |
) | ( |
) | ||||||||||||||||||||
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Net loss
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— | ( |
)
|
( |
)
|
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Stock-based compensation
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— | |
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| Issuance of common stock upon cashless exercise of pre-funded warrants | ||||||||||||||||||||||||
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Issuance of common stock and warrants through private placement, net of offering costs
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Issuance of common stock upon exercise of common warrants
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Balance at March 31, 2025
|
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( |
)
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( |
)
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The accompanying notes are an integral part of these consolidated financial statements
GRACE THERAPEUTICS, INC.
(Formerly ACASTI PHARMA INC.)
|
Year Ended March 31, 2026 |
Year Ended
March 31, 2025 | |||||||
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(Expressed in thousands)
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$ | $ | ||||||
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Cash flows from operating activities:
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Net loss
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( | ) | ( | ) | ||||
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Adjustments:
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Depreciation expense
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Loss on disposal
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Stock-based compensation
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Change in fair value of derivative warrant liabilities
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( | ) | ( | ) | ||||
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Deferred income tax benefit
|
( | ) | ( | ) | ||||
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Changes in operating assets and liabilities:
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Receivables
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Prepaid expenses
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( | ) | ||||||
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Trade and other payables
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Net cash used in operating activities
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( | ) | ( | ) | ||||
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Cash flows from investing activities:
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Maturity of short-term investments
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Purchase of short-term investments
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( | ) | ||||||
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Net cash provided by investing activities
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Cash flows from financing activities:
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Gross proceeds from issuance of common stock and warrants from private placement
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Stock issuance costs
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( | ) | ( | ) | ||||
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Proceeds from issuance of common stock from common warrant exercise
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Net cash provided by financing activities
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Net decrease in cash and cash equivalents
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( | ) | ( | ) | ||||
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Cash and cash equivalents, beginning of year
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Cash and cash equivalents, end of year
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Cash and cash equivalents are comprised of:
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Cash
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Cash equivalents
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Supplemental schedule of non-cash financing activities are comprised of:
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Issuance costs in accounts payable
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Settlement of derivative warrant liability
|
GRACE THERAPEUTICS, INC.
(Formerly ACASTI PHARMA, INC.)
(Expressed in thousands except share and per share data)
1. Nature of Operations
General
Grace Therapeutics, Inc. (formerly known as Acasti Pharma Inc.) (“Acasti Delaware” or “the Company”), is a Delaware corporation that, as further described below, previously existed under the laws of the Province of Québec, Canada (“Acasti Québec”), before changing its jurisdiction on October 1, 2024 to the Province of British Columbia, Canada (“Acasti British Columbia”). On October 7, 2024, Acasti British Columbia changed its jurisdiction to the State of Delaware. Effective October 28, 2024, the Company changed its corporate name to Grace Therapeutics, Inc.
Continuance and Domestication
On October 1, 2024, Acasti Québec changed its jurisdiction of incorporation from the Province of Québec in Canada to the Province of British Columbia in Canada pursuant to a “continuance” effected in accordance with Chapter XII of the Business Corporations Act (Québec) (the “Continuance”). Subsequently on October 7, 2024 (the “Effective Date”), Acasti British Columbia changed its jurisdiction of incorporation from the Province of British Columbia in Canada to the State of Delaware in the United States of America pursuant to a “continuance” effected in accordance with Section 308 of the Business Corporations Act (British Columbia) and a “domestication” (the “Domestication”) under Section 388 of the General Corporation Law of the State of Delaware. Both the Continuance and the Domestication were approved by the Company’s shareholders at the Company’s Annual and Special Meeting of Shareholders held on September 30, 2024.
Prior to the Continuance and Domestication, the Company’s Class A common shares, without par value per share (“Common Shares”), were listed on The Nasdaq Stock Market LLC (“Nasdaq”) under the symbol “ACST.” Upon the effectiveness of the Continuance, each outstanding Class A common share of Acasti Québec at the time of the Continuance remained issued and outstanding as a common share, without par value per share, of Acasti British Columbia. Upon effectiveness of the Domestication, each outstanding common share of Acasti British Columbia at the time of the Domestication automatically became one outstanding share of common stock, par value $0.0001 per share, of Acasti Delaware (“Common Stock”). The Common Stock continues to be listed for trading on Nasdaq and in connection with its corporate name change to Grace Therapeutics, Inc., commenced trading under the symbol “GRCE” on October 28, 2024.
The Continuance and Domestication has been accounted for as an exchange of equity interest among entities under common control resulting in a change in reporting entity, and has been retroactively reflected in the accompanying consolidated financial statements and notes thereto. All assets and liabilities of Acasti British Columbia were deemed assumed by the Company at the Effective Date, resulting in the retention of the historical basis of accounting as if they had always been combined for accounting and financial reporting purposes. Any excess resulting from the automatic conversion of each outstanding Common Share of Acasti British Columbia into one outstanding share of Common Stock of Acasti Delaware, is presented as Additional Paid-in Capital in the equity section of the accompanying consolidated financial statements and notes thereto. All per share amounts for all periods presented in the accompanying consolidated financial statements and notes thereto have been adjusted retroactively, where applicable, to reflect the effect of the change in par value.
Liquidity and Financial Condition
The Company has incurred operating losses and negative cash flows from operations in each period since its inception. The Company expects to incur significant expenses and continued operating losses for the foreseeable future.
In May 2023, the Company implemented a
strategic realignment plan to enhance shareholder value that resulted in the
Company engaging a new management team, streamlining its research and
development activities, and greatly reducing its workforce. Following the
realignment, the Company is a smaller, more focused organization, based in the
United States, and concentrated on its development of its lead product
candidate GTx-104. In June 2026, the Company decided
to not resume internal development funding for GTx-102 or GTx-101 under its
current operating plan.
In February 2025, the Company
completed a private placement of Company securities with certain institutional
and accredited investors. Net proceeds to the Company were $13,705 . Refer to
Note 7, Stockholders’ Equity - 2025 Private Placement, for
additional information.
As a result of
the Complete Response Letter (“CRL”) received in April 2026 from the U.S. Food
and Drug Administration (“FDA”) regarding its New Drug Application (“NDA”) for
GTx-104, management has decided to pause and strategically delay
commercialization and marketing expenditures for GTx-104 until the regulatory
requirements are resolved. The Company plans to use its current cash, which was
$16,977 as of March 31, 2026, towards resolving the items cited in the FDA’s CRL, working capital and other general
corporate purposes. The Company believes its existing cash and cash equivalents
will be sufficient to sustain planned operations through at least 12 months
from the issuance date of these consolidated financial statements included in
the Company’s Annual Report on Form 10-K.
The Company will require additional capital to fund its daily operating needs. The Company does not expect to generate revenue from product sales unless and until it successfully completes drug development and obtains regulatory approval, which is subject to significant uncertainty. To date, the Company has financed its operations primarily through public offerings and private placements of its common equity, warrants and convertible debt and the proceeds from research tax credits. Until such time that the Company can generate significant revenue from drug product sales, if ever, it will require additional financing, which is expected to be sourced from a combination of public or private equity or debt financing or other non-dilutive sources, which may include fees, milestone payments and royalties from collaborations with third parties. Arrangements with collaborators or others may require the Company to relinquish certain rights related to its technologies or drug product candidates. Adequate additional financing may not be available to the Company on acceptable terms, or at all. The Company’s inability to raise capital as and when needed could have a negative impact on its financial condition and its ability to pursue its business strategy. The Company plans to raise additional capital in order to maintain adequate liquidity. Negative results from studies or trials, if any, the timing and ability to receive FDA approval for marketing our drug candidates or depressed prices of the Company’s stock could impact the Company’s ability to raise additional financing. Raising additional equity capital is subject to market conditions that are not within the Company’s control. If the Company is unable to raise additional funds, the Company may not be able to realize its assets and discharge its liabilities in the normal course of business.
The Company remains subject to risks similar to other development stage companies in the biopharmaceutical industry, including compliance with government regulations, protection of proprietary technology, dependence on third-party contractors and consultants and potential product liability, among others.
2. Summary of significant accounting policies
Basis of presentation
These consolidated financial statements of Grace Therapeutics, Inc., which include the accounts of its subsidiary, have been prepared in accordance with generally accepted accounting principles in the United States of America ("U.S. GAAP"). All intercompany transactions and balances are eliminated on consolidation.
Use of estimates
The preparation of the consolidated financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets, liabilities, income, and expenses. Actual results may differ from these estimates.
Estimates are based on management’s best knowledge of current events and actions that management may undertake in the future. Estimates and underlying assumptions are reviewed on an ongoing basis. Revisions to accounting estimates are recognized in the period in which the estimates are revised and in any future periods affected.
Estimates and assumptions include the measurement of stock-based compensation, derivative warrant liabilities, accruals for research and development contracts and contract organization agreements, and valuation of intangibles and goodwill. Estimates and assumptions are also involved in determining the extent to which research and development expenses qualify for research and development tax credits. The Company recognizes tax credits once it has reasonable assurance that they will be realized.
Cash equivalents
Cash equivalents is comprised of highly liquid investments purchased with original maturities of 90 days or less. Cash equivalents consist of United States Treasury bills.
Equipment
Equipment is measured at cost less accumulated depreciation and accumulated impairment losses, if any. Cost includes expenditures that are directly attributable to the acquisition of the asset, including all costs incurred in bringing the asset to its present location and condition. Gains and losses on disposal of equipment are determined by comparing the proceeds from disposal with the carrying amount of the equipment.
Depreciation is recognized on a declining basis over the estimated useful lives of equipment, since this most closely reflects the expected pattern of consumption of the future economic benefits embodied in the asset. Items of equipment are depreciated from the date that they are available for use or, in respect of assets not yet in service, from the date they are ready for their intended use.
Intangible assets - acquired in-process research and development
In a business combination, the fair
value of in-process research and development (“IPR&D”) acquired is
capitalized and accounted for as indefinite-lived intangible assets, and not
amortized until the underlying project receives regulatory approval, at which
point the intangible assets will be accounted for as definite-lived intangible
assets and amortized over the remaining useful life or discontinued. If
discontinued, the intangible asset will be written off. The Company expects to
recognize an impairment charge for the full remaining carrying value of the
GTx-102 and GTx-101 IPR&D asset of $9,196 and $4,337 , respectively, in the
first quarter of fiscal year 2027. See Note 13 for further information. Research
and development (“R&D”) costs incurred after the acquisition are expensed
as incurred.
Impairment of long-lived assets
The Company reviews the recoverability of its finite long-lived assets whenever events or changes in circumstances indicate that it is carrying amount may not be recoverable. The carrying amount is first compared with the undiscounted cash flows. If the carrying amount is higher than the sum of undiscounted cash flows, then the Company determines the fair value of the underlying asset group. Any impairment loss to be recognized is measured as the difference by which the carrying amount of the asset group exceeds the estimated fair value of the asset group.
Goodwill and indefinite-lived assets are not amortized but are subject to an impairment review annually and more frequently when indicators of impairment exist. An impairment of goodwill could occur if the carrying amount of a reporting unit exceeds the fair value of that reporting unit. An impairment of indefinite-lived intangible assets would occur if the fair value of the intangible asset is less than the carrying value.
The Company tests its goodwill for impairment by first assessing qualitative factors to determine whether it is more likely than not that the fair value is less than its carrying amount. If the Company concludes it is more likely than not that fair value of the reporting unit is less than its carrying amount, a quantitative impairment test is performed.
The Company tests indefinite-lived intangible assets for impairment by first assessing qualitative factors to determine whether it is more likely than not that the fair value is less than its carrying amount. If the Company concludes it is more likely than not that the fair value is less than it's carrying amount, a quantitative impairment test is performed. The Company's annual impairment test is performed in the fourth quarter of the fiscal year.
Research and development costs
Research and developments expenditures are expensed as incurred. These costs consist of employees’ salaries and benefits related to research and development activities, contractors and consultants that conduct the Company’s clinical trials, laboratory material and small equipment, clinical trial materials, stock-based compensation expense, and other non-clinical costs and regulatory fees. Advance payments for goods and services that will be used in future research and development are recognized in prepaids or other assets and are expensed when the services are performed, or the goods are used.
Stock-based compensation
The Company has in place a stock option plan for directors, officers, employees, and consultants of the Company, with grants under the stock option plan approved by the Company’s Board of Directors. The plan provides for the granting of options to purchase Common Stock and the exercise price of each option equals the closing trading price of Common Stock on the day prior to the grant. The Company accounts for stock-based compensation arrangements in accordance with provisions of Accounting Standards Codification (“ASC”) 718, Compensation—Stock Compensation (“ASC 718”). ASC 718 requires the recognition of compensation expense, using a fair-value based method, for costs related to all share-based payments including stock options. ASC 718 requires companies to estimate the fair value of share-based payment awards on the date of grant using an option-pricing model. The Company measures the cost of such awards based on the fair value of the award at grant date and recognizes stock-based compensation expense in the consolidated statements of operations and comprehensive loss on a tranche by tranche basis. The fair value of options is estimated for each tranche of an award that vests on a graded basis. The fair value of options is estimated using the Black-Scholes option pricing model, which uses various inputs including fair value of the Common Stock at the grant date, expected term, historical volatility, risk-free interest rate and expected dividend yields of the Common Stock. The Company applies an estimated forfeiture rate derived from historical employee termination behavior in determining compensation expense. If the actual forfeitures differ from those estimated by management, adjustment to compensation expense may be required in future periods.
Government grants
Government grants are recorded as a reduction of the related expenses or costs of the asset acquired. Government grants are recognized when there is reasonable assurance that the Company has met the requirements of the approved grant program and there is reasonable assurance that the grant will be received.
Income taxes
Income taxes comprise of current and deferred taxes. The provision for income taxes is computed using the asset and liability method.
Current tax is the expected tax payable or receivable on the taxable income or loss for the year, using tax rates enacted at the reporting date, and any adjustment to tax payable in respect of previous years.
Deferred tax is recognized in respect of temporary differences between the carrying amounts (tax base) of assets and liabilities for financial reporting purposes and the amounts used for taxation purposes. Deferred tax assets and liabilities are measured at the tax rate expected to apply when the underlying asset or liability is realized (settled) based on the rates that are enacted at the reporting date. Deferred tax assets and liabilities are offset if the Company has the right to set off the amount owed by with the amount owed by the other party, the Company intends to set off and the offset right is enforceable at law. A deferred tax asset is recognized for unused tax losses, and tax credits, reduced by a valuation allowance. A valuation allowance is recorded to reduce the carrying amount of deferred income tax assets when it is more likely than not that these assets will not be realized. Tax benefits related to tax positions not deemed to meet the “more-likely-than-not” threshold are not permitted to be recognized in the consolidated financial statements.
Additionally, the Company accrues interest and penalties, if any, related to unrecognized tax benefits as a component of income tax expense. The unrecognized tax benefits, including accrued interest and penalties, if any, are included in income taxes payable in the accompanying Consolidated Balance Sheets.
Earnings per share
The Company presents basic and diluted
earnings per share ("EPS") data for its Common Stock. Basic EPS is
calculated by dividing the net income or loss by the weighted average number of
Common Stock outstanding during the year. Diluted EPS is determined by
adjusting the net income or loss and the weighted average number of Common
Stock outstanding adjusted for the effects of all dilutive potential Common
Stock, which comprise warrants and share options granted to employees. The
basic and diluted EPS are the same due to loss position.
Segment reporting
Operating segments are identified as components of an entity for which separate discrete financial information is available and that is regularly reviewed by the chief operating decision maker, the Company’s Chief Executive Officer, in deciding how to allocate resources to an individual segment and in assessing performance. The Company has determined it operates in a single operating segment and has one reporting segment. See Note 11 for further information.
Derivative warrant liabilities
Derivative warrant liabilities are recognized initially at fair value. Subsequent to initial recognition, derivative warrant liabilities are measured at fair value, with changes in fair value recognized in the consolidated statement of operations and comprehensive loss.
Fair value measurements
Certain of the Company’s accounting policies and disclosures require the determination of fair value, for both financial assets and liabilities.
In establishing fair value, the Company uses a fair value hierarchy based on levels as defined below:
●
Level 1: defined as observable inputs such as quoted prices in active markets.
●
Level 2: defined as inputs other than quoted prices in active markets that are either directly or indirectly observable.
●
Level 3: defined as inputs that are based on little or no observable market data, therefore requiring entities to develop their own assumptions.
The Company has determined that the carrying values of its short-term financial assets and liabilities (cash and cash equivalents and trade and other payables) approximate their fair value given the short-term nature of these instruments. The Company measured its derivative warrant liabilities at fair value on a recurring basis using level 3 inputs.
Financial Instruments
Concentration of credit risk
Financial instruments that potentially subject the Company to a concentration of credit risk consist primarily of cash and cash equivalents. Cash and cash equivalents are all invested in accordance with the Company’s Investment Policy with the primary objective being the preservation of capital and the maintenance of liquidity, which risk is managed by dealing only with highly rated U.S. and Canadian institutions. The Company maintains its cash and cash equivalents at accredited financial institutions in amounts that exceed federally insured limits. The Company does not believe that it is subject to unusual credit risk beyond the normal credit risk associated with commercial banking relationships.
Recent accounting pronouncements
On November 4, 2024, the FASB issued ASU 2024-03, Income Statement—Reporting Comprehensive Income—Expense Disaggregation Disclosures (Subtopic 220-40) (“ASU
2024-03”), to improve the disclosures about a public business entity’s expenses and address requests from investors for more detailed information about the types of expenses (including purchases of inventory, employee compensation, depreciation, amortization and depletion) in commonly presented expense captions (such as cost of sales, SG&A and research and development).
ASU
2024-03 applies to all public business entities and is effective for annual
reporting periods beginning after December 15, 2026 and interim reporting
periods within annual reporting periods beginning after December 15, 2027. The
requirements will be applied prospectively with the option for retrospective
application. Early adoption is permitted. The Company does not expect that the
adoption of ASU 2024-03 will have a material impact on its consolidated
financial statements and disclosures
In December 2023, the FASB issued ASU
No. 2023-09, Improvements to Income Tax Disclosures (Topic 740) (“ASU
2023-09”). The ASC 2023-09 requires disaggregated information about a reporting
entity’s effective tax rate reconciliation as well as additional information on
income taxes paid. The ASC 2023-09 is effective on a prospective basis for
annual periods beginning after December 15, 2024 and allows for adoption on a
prospective or retrospective basis. The Company adopted this ASC 2023-09 for the
2026 fiscal period on a prospective basis. Refer to Note 10 to the consolidated
financial statements for the inclusion of the required disclosures.
The Company has considered all other recent accounting pronouncements and concluded that they are either not applicable to the Company’s business or that the effect is not expected to be material to the consolidated financial statements as a result of future adoption.
3. Fair value measurements
Assets and liabilities measured at fair value on a recurring basis as of March 31, 2026 are as follows:
| Total |
Quoted
prices in active markets (Level 1) |
Significant
other observable inputs (Level 2) |
Significant
unobservable inputs (Level 3) | |||||||||||||
| $ | $ | $ | $ | |||||||||||||
|
Assets
|
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|
Treasury bills classified as cash equivalents
|
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|
Total assets
|
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|
Total liabilities
|
Assets and liabilities measured at fair value on a recurring basis as of March 31, 2025 are as follows:
| Total |
Quoted
prices in active markets (Level 1) |
Significant
other observable inputs (Level 2) |
Significant
unobservable inputs (Level 3) | |||||||||||||
| $ | $ | $ | $ | |||||||||||||
|
Assets
|
||||||||||||||||
|
Treasury bills classified as cash equivalents
|
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|
Total assets
|
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|
Liabilities
|
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|
Derivative warrant liabilities
|
||||||||||||||||
|
Total liabilities
|
There were no changes in valuation techniques or transfers between Levels 1, 2 or 3 during the years ended March 31, 2026 and 2025. The Company’s derivative warrant liabilities are measured at fair value on a recurring basis using unobservable inputs that are classified as Level 3 inputs. Refer to Note 7, Stockholders’ Equity, for the valuation techniques and assumptions used in estimating the fair value of the derivative warrant liabilities.
4. Equipment
The following is a summary of equipment, net:
|
March 31, 2026
|
Cost |
Accumulated
depreciation |
Write-off |
Net book
value |
||||||||||||
| $ | $ | $ | $ | |||||||||||||
|
Furniture and office equipment
|
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|
Computer equipment
|
( |
) | ||||||||||||||
|
Software
|
( |
) | ||||||||||||||
| ( |
) |
|
March 31, 2025
|
Cost |
Accumulated
depreciation |
Write-off |
Net book
value |
||||||||||||
| $ | $ | $ | ||||||||||||||
|
Furniture and office equipment
|
( |
) | ( |
) | ||||||||||||
|
Computer equipment
|
( |
) | ( |
) | ||||||||||||
|
Software
|
( |
) | ||||||||||||||
| ( |
) | ( |
) |
Depreciation expense was $7
5. Intangible assets and goodwill
Individual IPR&D projects and goodwill are tested for impairment on an annual basis in the fourth quarter, and in between annual tests if an event occurs or circumstances change that would more likely than not reduce the fair value of each technology or our reporting unit below its carrying value.
The impairment assessments resulted in the following activity during the years ended March 31, 2026 and 2025:
| GTx-104 | GTx-102 | GTx-101 | Total | |||||||||||||
| $ | $ | $ | $ | |||||||||||||
|
Intangible assets – in-process research and development
|
||||||||||||||||
|
Balance, March 31, 2024
|
||||||||||||||||
|
Impairment
|
||||||||||||||||
|
Balance, March 31, 2025
|
||||||||||||||||
|
Impairment
|
||||||||||||||||
|
Balance, March 31, 2026
|
| $ | ||||
|
Goodwill
|
||||
|
Balance, March 31, 2024
|
||||
|
Impairment
|
||||
|
Balance, March 31, 2025
|
||||
|
Impairment
|
||||
|
Balance, March 31, 2026
|
The Company's IPR&D projects, consistent with others in our industry, have risks and uncertainties associated with the timely and successful completion of the development and commercialization of product candidates, including our ability to confirm safety and efficacy based on data from clinical trials, our ability to obtain necessary regulatory approvals and our ability to successfully complete these tasks within budgeted costs. It is not permitted to market a human therapeutic without obtaining regulatory approvals, and such approvals require the completion of clinical trials that demonstrate that a product candidate is safe and effective. In addition, the availability and extent of coverage and reimbursement from third-party payers, including government healthcare programs and private insurance plans as well as competitive product launches, affect the revenues a product can generate. Consequently, the eventual realized values, if any, of acquired IPR&D projects may vary from their estimated fair values.
6. Trade and other payables
| March 31, 2026 | March 31, 2025 | |||||||
| $ | $ | |||||||
|
Trade payables
|
||||||||
|
Accrued research and development expenses
|
||||||||
|
Accrued liabilities and other payables
|
||||||||
|
Employee salaries and benefits payable
|
||||||||
|
Total trade and other payables
|
7. Stockholders' equity
Preferred Stock
The Company is authorized to issue up to 10,000,000 shares of preferred stock, par value $0.0001 per share. No
Common Stock
In connection with the consummation of the Domestication, on October 7, 2024, the Company adopted a Certificate of Incorporation (as amended, the “Charter”) and Bylaws (as amended, the “Bylaws”). The rights of holders of the Company’s Common Stock are now governed by the Charter, the Bylaws, and the General Corporation Law of the State of Delaware. The Company is authorized to issue up to 100,000,000 shares of Common Stock, par value $0.0001 per share.
2025 Private Placement
In February 2025, the Company agreed
to offer and sell in a private placement (the “2025 Private Placement”) an
aggregate of 3,252,132 shares of Common Stock at a purchase price of $3.395 per
share of Common Stock (the “2025 Private Placement Shares”) and pre-funded
warrants (the “2025 Pre-Funded Warrants”) to purchase up to 1,166,160 shares of
Common Stock at a purchase price equal to the purchase price per 2025 Private
Placement Share less $0.0001 (the “2025 Pre-Funded Warrant Shares”). Each 2025
Pre-Funded Warrant is exercisable for one share of Common Stock at an exercise
price of $0.0001 per share, exercisable immediately and will expire once
exercised in full.
Pursuant to the purchase agreement
related to the 2025 Private Placement, for each 2025 Private Placement Share
and each 2025 Pre-Funded Warrant issued, the Company agreed to issue to each
purchaser an accompanying common warrant (the “2025 Common Warrants” and,
together with the 2025 Pre-Funded Warrants, the “2025 Warrants”) to purchase
shares of Common Stock (or 2025 Pre-Funded Warrants in lieu thereof),
exercisable for an aggregate of 4,418,292 shares of Common Stock (or 2025
Pre-Funded Warrants in lieu thereof). Each 2025 Common Warrant is exercisable
for one share of Common Stock at an exercise price of $3.395 per share (or one
2025 Pre-Funded Warrant at an exercise price of $3.3949 per share in lieu
thereof), is immediately exercisable and will expire on the earlier of (i) the
60th day after the date the FDA approves the new drug application (“NDA”) for
GTx-104 and (ii) September 25, 2028. The 2025 Common Warrants were offered and
sold at a purchase price of $0.125 per 2025 Common Warrant, which purchase
price is included in the offering price per 2025 Private Placement Share and
2025 Pre-Funded Warrant issued in the 2025 Private Placement.
The 2025 Private Placement included the issuance of Common Stock, 2025 Pre-Funded Warrants, and 2025 Common Warrants to related parties namely (i) Shore Pharma LLC, an entity held in a trust for the benefit of immediate family members of Vimal Kavuru, the Chair of the Company’s Board of Directors and (ii) ADAR1 Partners, LP, AIGH Investment Partners, LP, and SS Pharma LLC, each a beneficial owner of more than 5 5,694 in 2025.
The Company paid TD Securities (USA) LLC, the placement agent, customary placement fees in its capacity as placement agent for the sale of the Company’s securities to certain of the investors in the 2025 Private Placement.
The 2025 Private Placement closed on February 11, 2025. The net proceeds to the Company were $13,705 , after deducting fees and expenses.
Both 2025 Pre-funded Warrants and 2025 Common Warrants are presented under additional paid-in capital in the equity section of the consolidated balance sheet as of March 31, 2026.
During
the twelve months ended March 31, 2026, 233,710 of the 2025 Pre-Funded Warrants were exercised
into 233,706 shares of Common Stock (the difference between the two figures
being the result of certain 2025 Pre-Funded Warrants exercised on a “cashless”
basis).
2023 Private Placement
In
September 2023, the Company entered into a securities purchase agreement
(the “Purchase Agreement”) with certain institutional and accredited investors
in connection with a private placement of its securities (the “2023 Private
Placement”). Pursuant to the Purchase Agreement, the Company offered and sold
1,951,371 Common Shares, at a purchase price of $1.848 per Common Share and
pre-funded warrants (the “2023 Pre-Funded Warrants”) to purchase up to
2,106,853 Common Shares at a purchase price equal to the purchase price per
Common Share less $0.0001 . Each 2023 Pre-Funded Warrant is exercisable for one
Common Share at an exercise price of $0.0001 per Common Share, is immediately
exercisable, and will expire once exercised in full. Pursuant to the Purchase
Agreement, the Company also issued, to such institutional and accredited
investors, warrants to purchase Common Shares exercisable for an aggregate of
2,536,391 Common Shares (the “2023 Common Warrants” and, together with the 2023
Pre-Funded Warrants, the “2023 Warrants”). Under the terms of the Purchase
Agreement, for each Common Share and each 2023 Pre-funded Warrant issued in the
2023 Private Placement, an accompanying five-eighths (0.625 ) of a 2023 Common
Warrant was issued to the purchaser thereof. Each whole 2023 Common Warrant was
exercisable for one Common Share at an exercise price of $3.003 per Common
Share, was immediately exercisable, and would expire on the earlier of (i) the
60th day after the date of the acceptance by the FDA of an NDA
for the Company’s product candidate GTx-104 and (ii) five years from the date
of issuance. The 2023 Private Placement closed on September 25, 2023. The net
proceeds to the Company from the 2023 Private Placement were $7,338 , after
deducting fees and expenses.
The 2023 Private Placement included the issuance of Common Shares, 2023 Pre-Funded Warrants, and 2023 Common Warrants to related parties namely (i) Shore Pharma LLC, an entity that was controlled by Vimal Kavuru, the Chair of the Company’s Board of Directors, at the time of the 2023 Private Placement and (ii) SS Pharma LLC, the beneficial owner of 5.5 % of Common Shares outstanding prior to the 2023 Private Placement, resulting in proceeds of $2,500 in 2023.
In
October 2025, the Company received $4,040 in gross proceeds from exercises of
1,345,464 2023 Common Warrants for 1,345,464 shares of Common Stock. The
remaining 1,190,927 2023 Common Warrants expired on October 21, 2025 in
accordance with the terms of the 2023 Common Warrant as the 60th day after the
FDA’s acceptance for review of the Company’s NDA for GTx-104 had passed.
The following table summarizes the Company’s outstanding warrants as of March 31, 2026, all of which are exercisable for shares of Common Stock:
| March 31, 2026 | ||||||||||||
|
No. of
warrants |
Exercise
Price ($) |
Expiration
Date | ||||||||||
|
Liability classified warrants (Derivative Warrant Liabilities)
|
||||||||||||
|
Common warrants issued in connection with 2023 Private Placement (2023 Common Warrants)
|
(1 | ) | ||||||||||
|
Equity classified warrants
|
||||||||||||
|
Pre-funded warrants issued in connection with 2023 Private Placement (2023 Pre-Funded Warrants)
|
No expiration | |||||||||||
|
Common warrants issued in connection with 2025 Private Placement (2025 Common Warrants)
|
(2 | ) | ||||||||||
|
Pre-funded warrants issued in connection with 2025 Private Placement (2025 Pre-Funded Warrants)
|
No expiration | |||||||||||
1. Following the exercise of 1,345,464 2023 Common Warrants in October
2025, the remaining 1,190,927 2023 Common Warrants expired on October 21, 2025 ,
which was the 60th day after the date of the acceptance by the FDA of the NDA
for the Company’s product candidate GTx-104.
2. The 2025 Common Warrants will expire on the earlier of: (i) the 60th
day after the date the FDA approves the NDA for GTx-104 and (ii) September 25,
2028 .
In connection with the Continuance and the Domestication, the Company continues its obligations under the 2023 Purchase Agreement and the 2023 Warrants. Upon the effectiveness of the Continuance, each outstanding 2023 Warrant exercisable for Common Shares remained exercisable for an equivalent number of common shares of Acasti British Columbia for the equivalent exercise price per share without any action by the holder. Upon the effectiveness of the Domestication, each outstanding 2023 Warrant exercisable for common shares of Acasti British Columbia remained exercisable for an equivalent number of shares of Common Stock for the equivalent exercise price per share without any action by the holder.
During the twelve months period March 31, 2026, 726,750 of the 2023 Pre-Funded Warrants were exercised into 726,750 shares of Common Stock.
Derivative Warrant Liabilities
The 2023 Common Warrants issued as a part of the 2023 Private Placement were derivative warrant liabilities given that the 2023 Common Warrants did not meet the fixed-for-fixed criteria and that the 2023 Common Warrants were not indexed to the Company's own stock.
The derivative warrant liabilities are measured at fair value at each reporting period and the reconciliation of changes in fair value is presented in the following table:
| Year ended | Year ended | |||||||
| March 31, 2026 | March 31, 2025 | |||||||
| $ | $ | |||||||
|
Beginning balance
|
||||||||
|
Issued during the year
|
||||||||
|
Change in fair value
|
) | ) | ||||||
|
Settlement of liability
|
( |
) | ||||||
|
Ending balance
|
The warrant liability was determined
based on the fair value of the 2023 Common Warrants at the issue date and the
reporting dates using the Black-Scholes model with the following assumptions.
The 2023 Common Warrants expired on October 21, 2025 , which was the 60th day
after the date of the acceptance by the FDA of the NDA for the Company's
product candidate GTx-104, which resulted in a $241 settlement of the
derivative warrant liability on the Company’s consolidated balance sheet as of March
31, 2026. The settlement fair value of the warrant liability was determined
using the Black-Scholes model with the following assumptions:
| October 21, 2025 | March 31, 2025 | |||||||
| Risk-free interest rate | % | % | ||||||
| Share price | $ | $ | ||||||
| Expected warrant life | ||||||||
| Dividend yield | % | % | ||||||
| Expected volatility | % | % |
As of March 31, 2026 and March 31, 2025, the balance of derivative warrant liabilities from related parties was $0 and $1,141 , respectively.
8. Stock-based compensation
2024 Equity Incentive Plan
At the Annual and Special Meeting of Shareholders on September 30, 2024, the Company’s shareholders approved the Grace Therapeutics, Inc. 2024 Equity Incentive Plan (the “2024 Plan”) which became effective on the date of the Domestication. The 2024 Plan replaced the Acasti Pharma Inc. Stock Option Plan and the Acasti Pharma Inc. Equity Incentive Plan (the “Prior Plans”). The 2024 Plan provides for the grant of awards of stock options, stock appreciation rights, restricted stock, restricted stock units, deferred stock units, unrestricted stock, dividend equivalent rights, performance-based awards and other equity-based awards to eligible persons as defined under the 2024 Plan. Any of these awards may, but need not, be made as performance incentives to reward the holders of such awards for the achievement of performance goals in accordance with the terms of the 2024 Plan. Stock options granted under the 2024 Plan may be non-qualified stock options or incentive stock options, as provided in the 2024 Plan.
In connection with the Continuance and the Domestication, the Company continues its obligations under the Prior Plans and all of the outstanding equity awards under the Prior Plans. Upon effectiveness of the Continuance, each outstanding option exercisable for and restricted share unit settleable into Common Shares remained exercisable for or able to be settled into, as applicable, an equivalent number of common shares of Acasti British Columbia for the equivalent exercise price per share (if applicable), without any action by the holder. Upon effectiveness of the Domestication, each outstanding option exercisable for and restricted share unit settleable into common shares of Acasti British Columbia remained exercisable for or able to be settled into, as applicable, an equivalent number of shares of Common Stock for the equivalent exercise price per share (if applicable), without any action by the holder.
Following the Effective Date of the 2024 Plan, no awards shall be made under the Prior Plans. However, Common Shares reserved under the Prior Plans to settle awards which were made under the Prior Plans may be issued and delivered following the Effective Date to settle such awards.
The 2024 Plan is administered by a committee designated from time to time, by resolution of the Company’s Board of Directors. The committee will also be responsible for determining, among others, the key terms of the awards including their grant dates, pricing, basis for fair value determination, vesting terms, restrictions, and terminations. The Board has designated its Compensation Committee to administer the 2024 Plan. The 2024 Plan authorizes a total of 1,350,000 shares of Common Stock available for issuance. As of March 31, 2026, there were 924,470 shares available for issuance under the 2024 Plan.
The 2024 Plan will terminate automatically ten years after the Effective Date and may be terminated on any earlier date as provided by the 2024 Plan.
The following table summarizes information about activities within the 2024 Plan and Prior Plans for the year ended March 31, 2026:
| Number of options | Weighted-average exercise price | Weighted-average Remaining Contractual Term (years) | Aggregate Intrinsic Value (in thousands) | |||||||||||||
| $ | ||||||||||||||||
| Outstanding, March 31, 2025 | ||||||||||||||||
| Granted | ||||||||||||||||
| Forfeited/Cancelled | ( | ) | ||||||||||||||
| Outstanding, March 31, 2026 | ||||||||||||||||
| Exercisable, March 31, 2026 |
The weighted-average grant date fair value of awards for options granted during the year ended March 31, 2026 was $1.85 . The fair value of options granted was estimated using the Black-Scholes option pricing model, resulting in the following weighted-average assumptions for the options granted:
| March 31, 2026 | March 31, 2025 | |||||||
| Weighted-
average | Weighted-
average | |||||||
| Exercise price | $ | $ | ||||||
| Share price | $ | $ | ||||||
| Dividend | ||||||||
| Risk-free interest | % | % | ||||||
| Estimated life (years) | ||||||||
| Expected volatility | % | % |
Compensation expense recognized under the 2024 Plan is summarized as follows:
| March 31, 2026 | March 31, 2025 | |||||||
| $ | $ | |||||||
|
Research and development expenses
|
||||||||
|
General and administrative expenses
|
||||||||
As of March 31, 2026, there was $319 of total unrecognized compensation cost, related to non-vested stock options, which is expected to be recognized over a remaining weighted-average vesting period of 1.1 years.
9. Loss per share
The Company has generated a net loss for all periods presented. Therefore, diluted loss per share is the same as basic loss per share since the inclusion of potentially dilutive securities would have had an anti-dilutive effect. All currently outstanding options and warrants could potentially be dilutive in the future.
The Company excluded the following potential Common Stock, presented based on amounts outstanding at each period end, from the computation of diluted net loss per share attributable to stockholders for the periods indicated because including them would have had an anti-dilutive effect:
| March 31, 2026 | March 31, 2025 | |||||||
|
Options outstanding
|
||||||||
|
2023 Common Warrants
|
||||||||
|
2025 Common Warrants
|
Basic and diluted net loss per share is calculated based upon the weighted-average number of shares of Common Stock outstanding during the year. Common Stock underlying the 2025 Pre-Funded Warrants and 2023 Pre-Funded Warrants are included in the calculation of basic and diluted earnings per share.
10. Income taxes
Income taxes are provided for the tax effects of transactions reported in the financial statements and consist of taxes currently due. Deferred taxes relate to differences between the basis of assets and liabilities for financial and income tax reporting which will be either taxable or deductible when the assets or liabilities are recovered or settled.
For the years
ended March 31, 2026 and 2025, the loss before income tax benefit was $9,493
and $12,767 , respectively
The provision for income taxes consisted of the following:
| March 31, 2026 | March 31, 2025 | |||||||
| $ | $ | |||||||
|
Current Provision
|
||||||||
|
Federal
|
||||||||
|
State
|
||||||||
|
Total Current Provision
|
||||||||
|
Deferred Provision
|
||||||||
|
Federal
|
( | ) | ( | ) | ||||
|
State
|
( | ) | ( | ) | ||||
|
Total Deferred Provision
|
( | ) | ( | ) | ||||
|
Total Provision for Income Taxes
|
( | ) | ( | ) |
The Company had an effective tax rate of 17.92 % and
25.06 % for the years ended March 31, 2026 and 2025, respectively.
During the current fiscal year
the Company adopted ASU 2023-09 prospectively. See Note 2 for additional
details on the adoption of ASU 2023-09. A reconciliation of the U.S. federal
statutory income tax rate to the Company’s effective tax rate pursuant to the
disclosure requirements of ASU 2023-09 for the year ended March 31, 2026 is as
follows (in thousands, except for percentages):
|
For the year ended
|
||||||||||||||||
|
March 31,
2026 |
March 31,
2026 |
March 31,
2025 |
March 31,
2025 |
|||||||||||||
|
$
|
$
|
|||||||||||||||
|
U.S. federal statutory
rate
|
( |
) | % | ( |
) | % | ||||||||||
|
State taxes and local
tax1, net of federal income tax effect
|
( |
) | % | ( |
) | % | ||||||||||
|
Difference in foreign
tax rates
|
% | ( |
) | % | ||||||||||||
|
Tax credits
|
( |
) | % | ( |
) | % | ||||||||||
|
Change in valuation
allowance
|
( |
)% | ( |
)% | ||||||||||||
|
Nontaxable or
nondeductible items:
|
||||||||||||||||
|
Non-deductible
stock-based compensation
|
( |
)% | ( |
)% | ||||||||||||
|
Change in fair value of
warrant liabilities
|
( |
) | % | ( |
) | % | ||||||||||
|
Other
|
( |
)% | ( |
)% | ||||||||||||
|
Other adjustments
|
( |
)% | ( |
) | % | |||||||||||
|
Total tax (benefit)
expense
|
( |
) | % | ( |
) | % | ||||||||||
1 account for 100% of the tax
effect in this category.
Cash paid for income taxes, net of refunds received, by
jurisdiction pursuant to the disclosure requirements of ASU 2023-09 for the
year ended March 31, 2026 is as follows (in thousands):
|
March
31, 2026
| ||||
|
Federal
|
||||
|
State
|
||||
|
New Jersey
|
||||
|
California
|
||||
|
Pennsylvania
|
||||
|
Other States
|
||||
|
Cash paid for income
taxes, net of refunds received
|
The table below presents the effects of temporary differences that gave rise to significant portions of deferred tax assets and liabilities as of March 31, 2026 and 2025:
|
March 31, 2026
|
March 31, 2025
| |||||||
|
$
|
$
| |||||||
|
Deferred income tax assets
|
||||||||
|
Tax losses carried forward
|
||||||||
|
Research and development expenses
|
||||||||
|
Property, plant and equipment
|
||||||||
|
Intangible assets
|
||||||||
|
Net federal investment tax credits
|
||||||||
|
Other accruals and other
|
||||||||
|
Orphan drug credit
|
||||||||
|
Total deferred income tax assets
|
||||||||
|
Valuation allowance
|
( | ) | ( | ) | ||||
|
Total deferred income tax assets, net of valuation allowance
|
||||||||
|
Deferred income tax liabilities
|
||||||||
|
Intangible assets
|
( | ) | ( | ) | ||||
|
Total deferred tax liabilities
|
( | ) | ( | ) | ||||
|
Net deferred tax liabilities
|
( | ) | ( | ) | ||||
The Company has U.S. Federal and State net operating loss carryforwards (“NOLs”) of approximately $40,438 and $67,873 and Canadian Federal and Quebec NOLs of $143,346 and $141,851 , respectively, as of March 31, 2026. The Company’s U.S. Federal NOLs, generated after December 31, 2017, are available for indefinite carryforward; however, their utilization is limited to 80 % of taxable income. State NOLs are subject to a 20-year carryforward period, with expirations beginning in 2038. The Company also has Orphan drug tax credits totaling $1,900 , which may be carried forward for 20 years and are set to begin expiring in 2045. Canadian and Quebec NOLs are scheduled to begin expiring in 2028, while scientific research and experimental development expenditure investment tax credits totaling $3,922 are set to begin expiring in 2029. Additionally, the Company has research and development expenses of $25,708 in Canada and $27,887 in Quebec, both of which are available for indefinite carryforward.
The utilization of the Company’s net operating loss carryforwards and research tax credit carryovers could be subject to annual limitations under Section 382 and 383 of the Internal Revenue Code of 1986, as amended (the “Code”), and similar state tax provisions, due to ownership change limitations that may have occurred previously or that could occur in the future. These ownership changes limit the amount of net operating loss carryforwards and other deferred tax assets that can be utilized to offset future taxable income and tax, respectively. In general, an ownership change, as defined by Section 382 and 383 of the Code, results from transactions increasing ownership of certain stockholders or public groups in the stock of the corporation by more than 50 percent points over a three-year period. The Company has not completed an analysis of an ownership change under Section 382 of the Code. To the extent that a study is completed and an ownership change is deemed to occur, the Company’s net operating losses and tax credits could be limited.
In assessing the realization of deferred tax assets, management considers whether it is more likely than not that some portion or all the deferred income tax assets will not be realized. The ultimate realization of deferred income tax assets is dependent upon the generation of future taxable income during the periods in which those temporary differences become deductible. Management considers the scheduled reversal of deferred income tax liabilities, projected future taxable income, and tax planning strategies in making this assessment. Based on consideration of these items, management has determined that enough uncertainty exists relative to the realization of the deferred income tax asset balances to warrant the application of a partial valuation allowance as of March 31, 2026 and March 31, 2025.
The Company’s
policy is to recognize interest and penalties associated with uncertain tax
benefits as part of the income tax provision and include accrued interest and
penalties with the related income tax liability on the Company’s Consolidated Balance Sheets. To date, the
Company has no nor no
On July 4, 2025, the One Big Beautiful Bill Act (“OBBBA”) was enacted in the U.S. The OBBBA includes significant provisions, such as the permanent extension of certain expiring provision of the Tax Cuts and Jobs Act, modification to the international tax framework and the restoration of favorable tax treatment for certain business provisions. The legislation has multiple effective dates, with certain provisions effective in 2025 and other implemented through 2027. Under OBBBA, the Company is permitted to claim 100 % bonus depreciation and to immediately expense Section 174 costs. These provisions accelerate tax deductions but does not create a permanent tax difference; therefore, the impact is timing‑related only and does not materially affect the Company’s income tax provision.
11. Segment Information
An operating segment is a component of an entity whose operating results are regularly reviewed by its chief operating decision maker (“CODM”) to make decisions about resources to be allocated to the segment and assess its performance. Factors used by the Company in determining the reportable segment include the nature of the Company's operating activities, the organizational and reporting structure and the type of information reviewed by the CODM to allocate resources and evaluate financial performance.
The Company has one reportable operating segment: the development and commercialization of pharmaceutical applications of its patents and licensed rights. The Company’s CODM is its Chief Executive Officer. The accounting policies of the segment are those described in the summary of significant accounting policies within Note 2.
The CODM assesses the performance of the segment based on net loss, which is reported on the income statement as consolidated net loss. The measure of segment assets is reported on the consolidated balance sheet as total consolidated assets.
The Company has not generated any revenue and expects to continue to incur significant expenses and operating losses as it advances product candidates through all stages of development, and ultimately, receive regulatory approval. Accordingly, the CODM utilizes the cash budget and forecasts in assessing the entity-wide operating results and performance, and in deciding how to allocate resources across the organization and its segment. Net loss is used to monitor budgets against actual results, which then is used in assessing the performance of the segment.
The table below summarizes the significant expenses, by category regularly review by the CODM, for the years ended March 31, 2026 and 2025:
|
March 31, 2026
$ |
March 31, 2025
$ | |||||||
|
Clinical development programs
|
( | ) | ( | ) | ||||
|
Professional fees
|
( | ) | ( | ) | ||||
|
Salaries and benefits
|
( | ) | ( | ) | ||||
|
Stock-based compensation
|
( | ) | ( | ) | ||||
|
Other general and administrative expenses (1)
|
( | ) | ( | ) | ||||
|
Other segment income (expense) (2)
|
||||||||
|
Interest income, net
|
||||||||
|
Income tax benefit
|
||||||||
|
Segment and consolidated net loss
|
( | ) | ( | ) |
1)
2)
12. Commitments and contingencies
Research and development contracts and contract research organizations agreements
The Company utilizes contract manufacturing organizations (“CMOs”) for the development and production of clinical materials and contract research organizations (“CROs”) to perform services related to its clinical trials. Pursuant to the agreements with these CMOs and CROs, the Company has either the right to terminate the agreements without penalties or under certain penalty conditions. As of March 31, 2026, the Company has $95 of commitments to CMOs and $30 of commitments to CROs for the next twelve months.
Legal proceedings and disputes
In the ordinary course of business, the Company is at times subject to various legal proceedings and disputes. The Company assesses its liabilities and contingencies in connection with outstanding legal proceedings utilizing the latest information available. Where it is probable that the Company will incur a loss and the amount of the loss can be reasonably estimated, the Company records a liability in its consolidated financial statements. These legal contingencies may be adjusted to reflect any relevant developments on a quarterly basis. Where a loss is not probable or the amount of loss is not estimable, the Company does not accrue legal contingencies. While the outcome of legal proceedings is inherently uncertain, based on information currently available, management believes that it has established appropriate legal reserves. No reserves or liabilities have been accrued as at March 31, 2026.
13. Subsequent Events
On
April 23, 2026, the Company received a CRL from the FDA in response to their
NDA for GTx-104. The CRL referenced certain items related to the chemistry,
manufacturing, and controls (CMC) and other non-clinical sections of the
application, including items related to additional leachable data time points for
commercial product, non-clinical product toxicology risk assessments, and cGMP
deficiencies at their CMO. A Type A meeting with the FDA has been scheduled to
potentially clarify the path forward and determine the appropriate next steps.
In order to prioritize resolving the items cited in the FDA’s CRL, the Company
does not plan to resume internal development funding for GTx-102 or GTx-101
under its current operating plan. Accordingly, the Company determined that the
remaining carrying value of the GTx-102 and GTx-101 IPR&D assets of $9,196
and $4,337 , respectively, are no longer recoverable on an internal-development
basis. The Company expects to recognize an impairment charge for the full
remaining carrying value in the first quarter of fiscal year 2027.
Exhibit 4.1
Exhibit 10.25
SEPARATION AND RELEASE OF CLAIMS AGREEMENT
This Separation and Release of Claims Agreement (the “Agreement”) is made as of the Agreement Effective Date (as defined below) between Grace Therapeutics, Inc. (the “Company”) and Carrie D’Andrea (“Employee”) (together, the “Parties”).
RECITALS
WHEREAS, the Company and Employee are parties to that certain Letter Agreement dated November 12, 2025, as amended by Amendment No. 1 dated January 10, 2026 (the “Letter Agreement”);
WHEREAS, the Parties are ending their employment relationship and wish to establish mutually agreeable terms for Employee’s orderly transition and separation from the Company effective on the Separation Date (as defined below); and
WHEREAS, the Parties agree that the payments, benefits, and rights set forth in this Agreement shall be the exclusive payments, benefits, and rights due Employee.
NOW, THEREFORE, in consideration of the mutual covenants and agreements contained herein, and for other good and valuable consideration, the receipt and sufficiency of which is hereby acknowledged, the Parties hereby agree as follows:
1.
Separation Date. Employee’s effective date of separation from employment with the Company and, as may be applicable, any of its subsidiaries, will be June 5, 2026 (the “Separation Date”). Employee will be separated without cause, as of the Separation Date, from any and all of her positions as an officer and employee of the Company and each of its subsidiaries, and Employee further agrees to execute and deliver any documents reasonably necessary to effectuate such resignations, provided nothing in any such document is inconsistent with any terms set forth in this Agreement. As of the Separation Date, all salary payments from the Company will cease and any benefits Employee had as of such date under Company-provided benefit plans, programs, or practices will terminate, except as required by applicable law or as otherwise specifically set forth in this Agreement or the Consulting Agreement.
2.
Severance Benefits; Equity Plans; Consulting Agreement.
(a)
In return for Employee’s timely signing and not revoking this Agreement as set forth below, and subject to Employee’s compliance with all terms hereof, (i) the Company shall continue to pay Employee, in accordance with the Company’s regularly established payroll procedure, Employee’s Base Salary for a period of six (6) months, with the first payment being made on the first regular payroll following the Agreement Effective Date (defined below); and (ii) provided Employee timely elects continued coverage under the Company’s health and welfare plans pursuant to COBRA, the Company shall pay or reimburse Employee premiums for the six (6) months of such coverage on terms no less favorable than those terms in effect as of the Agreement Effective Date (provided that such COBRA payment or reimbursement shall terminate on such earlier date as you are no longer eligible for COBRA coverage or you are eligible for coverage under another employer’s health plan) (collectively, the “Severance Benefits”). Employee acknowledges that, except as expressly provided herein or in any Consulting Agreement (as defined below), she is not entitled to any incentive compensation, bonus, additional severance, or other post-employment compensation.
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(b)
To the extent that, as of the Separation Date, Employee has vested into any or all portion of any equity award previously granted to Employee under the Company’s former or current equity incentive plans (the “Equity Plans”) and the applicable award agreements and grant notices thereunder (collectively, the “Equity Award Documents”), Employee will retain rights to such vested equity grant in accordance with, and subject to, the terms of the applicable Equity Award Documents. For the avoidance of doubt, and notwithstanding anything to the contrary in the Equity Award Documents, all of Employee’s outstanding unvested awards under the Equity Plans cease vesting as of the Separation Date and shall be forfeited, unless extended pursuant to the Consulting Agreement, as provided for in Section 2(c) below.
(c)
Employee and the Company have entered into a consulting agreement effective as of the Separation Date whereby Employee will perform certain services for the Company as an independent contractor after the Separation Date, which is attached hereto as Exhibit A (the “Consulting Agreement”). The Consulting Agreement describes, in addition to the fees for post-employment services, any continuing post-employment vesting and exercise rights Employee has with respect to any equity awards awarded to Employee prior to the Separation Date. Any compensation for Employee’s work as an independent contractor is governed by the Consulting Agreement.
(d)
Other than the Severance Benefits, Employee will not be eligible for, nor shall she have a right to receive, any payments or benefits from the Company or any of its subsidiaries following the Separation Date, other than reimbursement for any outstanding business expenses in accordance with Company policy.
3.
Release of Claims. In exchange for the consideration set forth in this Agreement, which Employee acknowledges she would not otherwise be entitled to receive, Employee hereby fully, forever, irrevocably and unconditionally releases, remises and discharges the Company, subsidiaries, predecessors, and successors, and all of its and their respective past and present officers, directors, stockholders, investors, partners, members, managers, employees, agents, representatives, plan administrators, attorneys, insurers and fiduciaries (each in their individual and corporate capacities) (collectively, the “Released Parties”) from any and all claims, complaints, demands, actions, causes of action, suits, rights, debts, sums of money, costs, accounts, reckonings, covenants, contracts, agreements, promises, doings, omissions, executions, damages, obligations, liabilities, and expenses (including attorneys’ fees and costs), of every kind and nature that Employee ever had or now has against any or all of the Released Parties, whether known or unknown, including, but not limited to, any and all claims arising out of or relating to Employee’s employment with and/or separation from the Company, including, but not limited to, all claims under Title VII of the Civil Rights Act of 1964, 42 U.S.C. § 2000e et seq., the Americans With Disabilities Act of 1990, 42 U.S.C. § 12101 et seq., the Age Discrimination in Employment Act, 29 U.S.C. § 621 et seq., the Genetic Information Nondiscrimination Act of 2008, 42 U.S.C. § 2000ff et seq., the Family and Medical Leave Act, 29 U.S.C. § 2601 et seq., the Worker Adjustment and Retraining Notification Act (“WARN”), 29 U.S.C. § 2101 et seq., the Rehabilitation Act of 1973, 29 U.S.C. § 701 et seq., Employee Order 11246, Executive Order 11141, the Fair Credit Reporting Act, 15 U.S.C. § 1681 et seq., the Uniformed Services Employment and Reemployment Rights Act of 1994 (“USERRA”), the False Claims Act, 31 U.S.C. § 3729 et seq., 38 U.S.C. § 4301 et seq., and the Employee Retirement Income Security Act of 1974 (“ERISA”), 29 U.S.C. § 1001 et seq., the New Jersey Law Against Discrimination (“NJLAD”), N.J.S.A. 10:5-1 et seq., the New Jersey Conscientious Employee Protection Act (“CEPA”), N.J.S.A. 34:19-1 et seq., the New Jersey Family Leave Act (“NJFLA”), N.J.S.A. 34:11B-1 et seq., the New Jersey Civil Rights Act, N.J.S.A. 10:6-1 et seq., the New Jersey Equal Pay Act, N.J.S.A. 10:5-12(t), the New Jersey Wage and Hour Law (“NJWHL”), N.J.S.A. 34:11-4.1 et seq., the New Jersey Wage Payment Law, N.J.S.A. 34:11-2 et seq., the New Jersey Earned Sick Leave Law, N.J.S.A. 34:11D-1 et seq., the New Jersey Workers’ Compensation Law’s anti-retaliation provisions, N.J.S.A. 34:15-39.1 et seq., all as amended; all claims arising out of any applicable state law and regulations; all common law claims including, but not limited to, actions in defamation, intentional infliction of emotional distress, fraud, misrepresentation, wrongful discharge, and breach of contract (including, without limitation, all claims arising out of or related to the Letter Agreement); all claims to any non-vested ownership interest in the Company, contractual or otherwise; all state and federal whistleblower claims to the maximum extent permitted by law; and any claim or damage arising out of Employee’s employment with and/or separation from the Company and/or its subsidiaries (including a claim for retaliation) under any common law theory or any federal, state or local statute or ordinance not expressly referenced above.
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Notwithstanding the foregoing, nothing in this release of claims prevents Employee from filing a charge with, cooperating with, or participating in any investigation or proceeding before, the Equal Employment Opportunity Commission or a state fair employment practices agency (except that Employee acknowledges that she may not recover any monetary benefits in connection with any such charge, investigation, or proceeding, and Employee further waives any rights or claims to any payment, benefit, attorneys’ fees or other remedial relief in connection with any such charge, investigation or proceeding). Further, nothing herein shall prevent Employee from bringing claims to enforce this Agreement, or release (i) any rights Employee may have under the Company’s certificate of incorporation, by-laws, insurance and/or any indemnification agreement between Employee and the Company (and/or otherwise under law) for indemnification and/or defense as an employee, officer or director of the Company for her service to the Company (recognizing that such indemnification and/or defense is not guaranteed by this Agreement and shall be governed by the instrument or law, if any, providing for such indemnification and/or defense), (ii) any rights Employee may have to vested or earned compensation or benefits, including pension or 401(k) benefits or interests under any ERISA-Covered benefit plan (excluding severance) provided by the Company, (iii) Employee’s right to communicate or cooperate with any government agency, (iv) claims for unemployment compensation or any state disability insurance benefits pursuant to the terms of applicable state law; (v) claims for workers’ compensation insurance benefits under the terms of any worker’s compensation insurance policy or fund of the Company; and (vi) any rights or claims that cannot be waived by law, including claims for unemployment benefits.
4.
Section 1542 Waiver. Employee understands and agree the claims released in Section 3 above include not only claims presently known to Employee, but also include all unknown or unanticipated claims, rights, demands, actions, obligations, liabilities, and causes of action of every kind and character that would otherwise come within the scope of the released claims as described in Section 3. Employee understands she may hereafter discover facts different from what she now believes to be true, which if known, could have materially affected this Agreement, but Employee nevertheless waives any claims or rights based on different or additional facts.
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Continuing Obligations. Employee acknowledges and reaffirms her obligation, to the extent permitted by law and except as otherwise permitted by Section 9 below, to keep confidential and not to use or disclose any and all non-public information concerning the Company and/or its subsidiaries that Employee acquired during the course of her employment with the Company and/or its subsidiaries, including, but not limited to, any non-public information concerning the Company’s and/or its subsidiaries’ business affairs, business prospects, and financial condition. Employee further acknowledges and reaffirms her non-competition and non-solicitation provisions set forth in Letter Agreement and the Confidentiality of Information and Ownership of Proprietary Property Agreement that she previously executed for the benefit of the Company (the “Restrictive Covenants Agreements”), which remain in full force and effect and which survive her separation from employment with the Company.
6.
Non-Disparagement. Employee understands and agrees that, to the extent permitted by law and except as otherwise permitted by Section 9 below, she will not, in public or private, make any disparaging, derogatory or defamatory statements to any person or entity, including, but not limited to, any media outlet, industry group, financial institution or current or former employee, board member, consultant, client or customer of the Company, regarding the Company or its subsidiaries, and all of their respective officers, directors or employees or regarding the Company’s business affairs, business prospects, or financial condition. The Company agrees that, to the extent permitted by law, none of their respective officers or directors will, in public or private, make any disparaging, derogatory or defamatory statements to any person or entity, including, but not limited to, any media outlet, industry group, financial institution or current or former employee, board member, consultant, client or customer of the Company, regarding Employee. Nothing herein prevents Employee from discussing or disclosing information about unlawful acts in the workplace, such as harassment or discrimination or any other conduct that Employee has reason to believe is unlawful. Notwithstanding the foregoing, nothing herein shall be construed as preventing any Party from making truthful disclosures in any litigation or arbitration.
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7.
Return of Company Property. At the Company’s request, Employee will return to the Company or, with the written permission of the Company, destroyed, all keys, files, records (and copies thereof), equipment (including, but not limited to, computer hardware, software and printers, flash drives and other storage devices, wireless handheld devices, cellular phones, tablets, etc.), Company identification and any other tangible or intangible Company-owned property in her possession or control and, other than with the written permission of the Company, has left intact, and has otherwise not destroyed, deleted or made inaccessible to the Company, all electronic Company documents, including but not limited to those that she developed or helped to develop during her employment or service with the Company. Employee further confirms that, to the extent requested by the Company, she has canceled all accounts for her benefit, if any, in the Company’s name, including but not limited to, credit cards, telephone charge cards, cellular phone and/or wireless data accounts and computer accounts.
8. Confidentiality. Employee understands and agrees that, to the extent permitted by law and except as otherwise permitted by Section 9 below, the contents of the negotiations and discussions resulting in this Agreement shall be maintained as confidential by Employee and her agents and representatives and shall not be disclosed except as otherwise agreed to in writing by the Company; provided, however, that nothing herein shall be construed as preventing Employee from making truthful disclosures in any litigation or arbitration or from having confidential conversations with her agents and representatives and family members on the condition that any individuals so informed must hold the above information in strict confidence.
9. Scope of Disclosure Restrictions. Nothing in this Agreement or elsewhere prohibits Employee from communicating with government agencies about possible violations of federal, state, or local laws or otherwise providing information to government agencies, filing a complaint with government agencies, or participating in government agency investigations or proceedings. Employee is not required to notify the Company of any such communications; provided, however, that nothing herein authorizes the disclosure of information Employee obtained through a communication that was subject to the attorney-client privilege. Further, notwithstanding Employee’s confidentiality and nondisclosure obligations, Employee is hereby advised as follows pursuant to the Defend Trade Secrets Act: “An individual shall not be held criminally or civilly liable under any Federal or State trade secret law for the disclosure of a trade secret that (A) is made (i) in confidence to a Federal, State, or local government official, either directly or indirectly, or to an attorney; and (ii) solely for the purpose of reporting or investigating a suspected violation of law; or (B) is made in a complaint or other document filed in a lawsuit or other proceeding, if such filing is made under seal. An individual who files a lawsuit for retaliation by an employer for reporting a suspected violation of law may disclose the trade secret to the attorney of the individual and use the trade secret information in the court proceeding, if the individual (A) files any document containing the trade secret under seal; and (B) does not disclose the trade secret, except pursuant to court order.”
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10. Cooperation. Employee agrees that, to the extent permitted by law, she shall cooperate fully with the Company in the investigation, defense or prosecution of any claims or actions which already have been brought, are currently pending, or which may be brought in the future against the Company by a third party or by or on behalf of the Company against any third party, whether before a state or federal court, any state or federal government agency, or a mediator or arbitrator, that relate in any manner to Employee’s conduct or duties at the Company or that are based on facts about which Employee obtained personal knowledge while employed at or providing services to the Company. Employee’s full cooperation in connection with such claims or actions shall include, but not be limited to, being available to meet with the Company’s counsel, at reasonable times and locations designated by the Company, to investigate or prepare the Company’s claims or defenses, to prepare for trial or discovery or an administrative hearing, mediation, arbitration or other proceeding and to act as a witness when requested by the Company. Any reasonable out-of-pocket expenses incurred by Employee associated with such cooperation will be paid for or reimbursed by the Company. Employee further agrees that, to the extent permitted by law, she will notify the Company promptly in the event that she is served with a subpoena (other than a subpoena issued by a government agency), or in the event that she is asked to provide a third party (other than a government agency) with information concerning any actual or potential complaint or claim against the Company.
11. Business Expenses and Final Compensation. Employee acknowledges the Company has reimbursed her for all business expenses incurred in conjunction with the performance of her employment and she is owed no other reimbursements. Employee acknowledges that she has received all compensation due to her from the Company, including, but not limited to, all wages, bonuses and accrued, unused vacation time, and that she is not eligible or entitled to receive any additional payments or consideration from the Company beyond that provided for in Section 2 of this Agreement.
12. Time for Consideration and Revocation. Employee acknowledges she was initially presented with this Agreement on June 2, 2026 (the “Receipt Date”). Employee understands that this Agreement shall be of no force or effect, and that she shall not be eligible for the consideration described herein, unless she signs and returns this Agreement no later than the twenty-second (22nd) day after the Receipt Date and does not revoke her acceptance in the subsequent seven (7) day period (the day immediately following expiration of such revocation period (the “Agreement Effective Date”). Employee acknowledges that she will not be entitled to the Severance Benefits unless the Agreement Effective Date occurs within sixty (60) days following the Separation Date (or such shorter period as may be directed by the Company at the time of separation).
13. Acknowledgments. Employee acknowledges that she has been given at least twenty-one (21) days to consider this Agreement, and that the Company is hereby advising her to consult with an attorney of her own choosing prior to signing this Agreement. Employee further acknowledges and agrees that any changes made to this Agreement following her initial receipt of this Agreement, whether material or immaterial, shall not restart or affect in any manner the twenty-one (21) day consideration period. Employee understands that she may revoke this Agreement for a period of seven (7) days after she signs it by notifying the Company in writing, and the Agreement shall not be effective or enforceable until the expiration of this seven (7) day revocation period. Employee understands and agrees that by entering into this Agreement she will be waiving any and all rights or claims she might have under the Age Discrimination in Employment Act, as amended by the Older Workers Benefit Protection Act, and that she has received or will receive consideration beyond that to which she was previously entitled.
14. Voluntary Assent. Employee states and represents that Employee has had an opportunity to fully discuss and review all the terms of this Agreement with an attorney of Employee’s own choosing. Employee further states and represents that Employee has carefully read this Agreement, understands the contents herein, freely, and voluntarily assents to all the terms and conditions hereof, and signs Employee’s name of Employee’s own free act.
15. Notice. Any notice delivered under this Agreement shall be deemed duly delivered three (3) business days after it is sent by registered or certified mail, return receipt requested, postage prepaid, one (1) business day after it is sent for next-business day delivery via a reputable nationwide overnight courier service, or immediately upon hand delivery, in each case addressed to the Company at its principal executive offices and to Employee at her most recent address set forth in the Company’s personnel records. Either Party may change the address to which notices are to be delivered by giving notice of such change to the other Party in the manner set forth in this Section 15.
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16. Arbitration. The parties agree all disputes, claims and causes of action, in law or equity, arising from or relating to this Agreement, the Letter Agreement or their enforcement, performance, breach, or interpretation, or Employee’s termination of employment or service, shall be subject to arbitration before the American Arbitration Association.
17. Applicable Law; Jury Trial Waiver. This Agreement shall be governed by and construed in accordance with the laws of the State of Delaware (without reference to the conflict of laws provisions thereof).
18. Successors and Assigns. This Agreement shall be binding upon and inure to the benefit of both Parties and their respective successors and assigns, including any corporation with which or into which the Company may be merged or which may succeed to its assets or business; provided, however, the obligations of Employee are personal and shall not be assigned by Employee. Notwithstanding the foregoing, in the event of Employee’s death, her rights to any payments or benefits hereunder to which she is or becomes entitled to at the time of her death may be assigned or transferred by the laws of descent and distribution and any such payments or benefits will be provided to Employee’s beneficiaries or estate, as applicable.
19. No Oral Modification, Waiver, Cancellation or Discharge. This Agreement may be amended or modified only by a written instrument executed by each Party. No delay or omission by the Company in exercising any right under this Agreement shall operate as a waiver of that or any other right. A waiver or consent given by the Company on any one occasion shall be effective only in that instance and shall not be construed as a bar to or waiver of any right on any other occasion.
20. Captions and Pronouns. The captions of the sections of this Agreement are for convenience of reference only and in no way define, limit, or affect the scope or substance of any section of this Agreement. Whenever the context may require, any pronouns used in this Agreement shall include the corresponding masculine, feminine or neuter forms, and the singular forms of nouns and pronouns shall include the plural, and vice versa.
21. Interpretation. The Parties agree this Agreement will be construed without regard to any presumption or rule requiring construction or interpretation against the drafting Party. References in this Agreement to “include” or “including” should be read as though they said “without limitation” or equivalent forms.
22. Severability. Each provision of this Agreement must be interpreted in such manner as to be effective and valid under applicable law, but if any provision of this Agreement is held to be prohibited by or invalid under applicable law, such provision will be ineffective only to the extent of such prohibition or invalidity, without invalidating the remainder of such provision or the remaining provisions of this Agreement. Moreover, if a court of competent jurisdiction determines any of the provisions contained in this Agreement to be unenforceable because the provision is excessively broad in scope, whether as to duration, activity, geographic application, subject or otherwise, it will be construed, by limiting or reducing it to the extent legally permitted, so as to be enforceable to the extent compatible with then applicable law to achieve the intent of the Parties.
23. Entire Agreement. This Agreement and the Restrictive Covenant Agreements contain and constitute the entire understanding and agreement between the Parties hereto with respect to Employee’s separation from the Company, severance benefits and the settlement of claims against the Company, and cancels all previous oral and written negotiations, agreements, commitments and writings in connection therewith, including without limitation, the Letter Agreement; provided, however, nothing in this Section 23 shall modify, cancel or supersede Employee’s obligations set forth in Section 5 above or under the Restrictive Covenants Agreements.
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24.
Tax Acknowledgement. In connection with the Severance Benefits provided to Employee pursuant to this Agreement, the Company shall withhold and remit to the tax authorities the amounts required under applicable law, and Employee shall be responsible for all applicable taxes owed by her with respect to such Severance Benefits under applicable law. Employee acknowledges that she is not relying upon the advice or representation of the Company with respect to the tax treatment of any of the Severance Benefits.
25.
Section 409A. The Section 409A provisions of the Letter Agreement are hereby incorporated by reference and shall apply to this Agreement as if set forth herein in full.
26.
Counterparts; Facsimile or .pdf Signatures. This Agreement may be executed in any number of counterparts, each of which when so executed and delivered will be deemed an original, and all of which together shall constitute one and the same agreement. This Agreement may be executed and delivered by facsimile or by .pdf file and upon such delivery the facsimile or .pdf signature will be deemed to have the same effect as if the original signature had been delivered to the other party.
[Signatures on Page Following]
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IN WITNESS WHEREOF, the Parties hereto have executed this Agreement as of the date(s) written below.
GRACE THERAPEUTICS, INC.
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By:
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/s/ Prashant Kohli
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Name:
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Prashant Kohli
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Title:
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Chief Executive Officer
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Date:
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June 17, 2024
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EMPLOYEE
I hereby agree to the terms and conditions set forth above. I have been given at least twenty-one (21) days to consider this Agreement, and I have chosen to execute this on the date below. I intend that this Agreement will become a binding agreement if I do not revoke my acceptance within seven (7) days. I have also been advised to consult with counsel with regards to this Agreement.
| /s/ Carrie D’Andrea | |
| Name: Carrie D’Andrea | |
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Date:
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June 17, 2026
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EXHIBIT A
Consulting Agreement
(See attached)
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Exhibit 21.1
Grace Therapeutics, Inc.
LIST OF SUBSIDIARY
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Name
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Jurisdiction of Incorporation
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Percent Owned
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Grace Therapeutics U.S., Inc. (formerly Acasti Pharma U.S., Inc.)
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Delaware, United States
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100%
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Exhibit 23.1
Consent of Independent Registered Public Accounting Firm
We consent to the incorporation by reference in the registration statements (Nos. 333-282537, 333-191383, 333-227476, and 333-274228) on Form S-8 and (Nos. 333-274899 and 333-285661) on Form S-3 of our report dated June 18, 2026, with respect to the consolidated financial statements of Grace Therapeutics, Inc. and subsidiary.
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Philadelphia, Pennsylvania
June 18, 2026
Exhibit 31.1
CERTIFICATION
PURSUANT TO SECTION 302 OF THE SARBANES-OXLEY ACT OF 2002
I, Prashant Kohli, certify that:
1. I have reviewed this Annual Report on Form 10-K of Grace Therapeutics, Inc.;
2. Based on my knowledge, this report does not contain any untrue statement of a material fact or omit to state a material fact necessary to make the statements made, in light of the circumstances under which such statements were made, not misleading with respect to the period covered by this report;
3. Based on my knowledge, the financial statements, and other financial information included in this report, fairly present in all material respects the financial condition, results of operations and cash flows of the registrant as of, and for, the periods presented in this report;
4. The registrant’s other certifying officer(s) and I are responsible for establishing and maintaining disclosure controls and procedures (as defined in Exchange Act Rules 13a-15(e) and 15d-15(e)) and internal control over financial reporting (as defined in Exchange Act Rules 13a-15(f) and 15d-15(f)) for the registrant and have:
(a) Designed such disclosure controls and procedures, or caused such disclosure controls and procedures to be designed under our supervision, to ensure that material information relating to the registrant, including its consolidated subsidiaries, is made known to us by others within those entities, particularly during the period in which this report is being prepared;
(b) Designed such internal control over financial reporting, or caused such internal control over financial reporting to be designed under our supervision, to provide reasonable assurance regarding the reliability of financial reporting and the preparation of financial statements for external purposes in accordance with generally accepted accounting principles;
(c) Evaluated the effectiveness of the registrant’s disclosure controls and procedures and presented in this report our conclusions about the effectiveness of the disclosure controls and procedures, as of the end of the period covered by this report based on such evaluation; and
(d) Disclosed in this report any change in the registrant’s internal control over financial reporting that occurred during the registrant’s most recent fiscal quarter (the registrant’s fourth fiscal quarter in the case of an annual report) that has materially affected, or is reasonably likely to materially affect, the registrant’s internal control over financial reporting; and
5. The registrant’s other certifying officer(s) and I have disclosed, based on our most recent evaluation of internal control over financial reporting, to the registrant’s auditors and the audit committee of the registrant’s board of directors (or persons performing the equivalent functions):
(a) All significant deficiencies and material weaknesses in the design or operation of internal control over financial reporting which are reasonably likely to adversely affect the registrant’s ability to record, process, summarize and report financial information; and
(b) Any fraud, whether or not material, that involves management or other employees who have a significant role in the registrant’s internal control over financial reporting.
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Date: June 18, 2026
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/s/ Prashant Kohli
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Prashant Kohli
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Chief Executive Officer
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Exhibit 31.2
CERTIFICATION
PURSUANT TO SECTION 302 OF THE SARBANES-OXLEY ACT OF 2002
I, Robert DelAversano, certify that:
1. I have reviewed this Annual Report on Form 10-K of Grace Therapeutics, Inc.;
2. Based on my knowledge, this report does not contain any untrue statement of a material fact or omit to state a material fact necessary to make the statements made, in light of the circumstances under which such statements were made, not misleading with respect to the period covered by this report;
3. Based on my knowledge, the financial statements, and other financial information included in this report, fairly present in all material respects the financial condition, results of operations and cash flows of the registrant as of, and for, the periods presented in this report;
4. The registrant’s other certifying officer(s) and I are responsible for establishing and maintaining disclosure controls and procedures (as defined in Exchange Act Rules 13a-15(e) and 15d-15(e)) and internal control over financial reporting (as defined in Exchange Act Rules 13a-15(f) and 15d-15(f)) for the registrant and have:
(a) Designed such disclosure controls and procedures, or caused such disclosure controls and procedures to be designed under our supervision, to ensure that material information relating to the registrant, including its consolidated subsidiaries, is made known to us by others within those entities, particularly during the period in which this report is being prepared;
(b) Designed such internal control over financial reporting, or caused such internal control over financial reporting to be designed under our supervision, to provide reasonable assurance regarding the reliability of financial reporting and the preparation of financial statements for external purposes in accordance with generally accepted accounting principles;
(c) Evaluated the effectiveness of the registrant’s disclosure controls and procedures and presented in this report our conclusions about the effectiveness of the disclosure controls and procedures, as of the end of the period covered by this report based on such evaluation; and
(d) Disclosed in this report any change in the registrant’s internal control over financial reporting that occurred during the registrant’s most recent fiscal quarter (the registrant’s fourth fiscal quarter in the case of an annual report) that has materially affected, or is reasonably likely to materially affect, the registrant’s internal control over financial reporting; and
5. The registrant’s other certifying officer(s) and I have disclosed, based on our most recent evaluation of internal control over financial reporting, to the registrant’s auditors and the audit committee of the registrant’s board of directors (or persons performing the equivalent functions):
(a) All significant deficiencies and material weaknesses in the design or operation of internal control over financial reporting which are reasonably likely to adversely affect the registrant’s ability to record, process, summarize and report financial information; and
(b) Any fraud, whether or not material, that involves management or other employees who have a significant role in the registrant’s internal control over financial reporting.
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Date: June 18, 2026
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/s/ Robert DelAversano
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Robert DelAversano
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Principal Financial Officer
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Exhibit 32.1
SECTION 906 CERTIFICATION
Pursuant to section 906 of the Sarbanes-Oxley Act of 2002 (subsections (a) and (b) of section 1350, chapter 63 of title 18, United States Code) in connection with the Annual Report on Form 10-K of Grace Therapeutics, Inc. for the annual period ended March 31, 2026, as filed with the Securities and Exchange Commission on the date hereof (the “Report”), the undersigned officer hereby certifies, to such officer’s knowledge, that:
(1) The Report fully complies with the requirements of Section 13(a) or 15(d) of the Securities Exchange Act of 1934; and
(2) The information contained in the Report fairly presents, in all material respects, the financial condition and results of operations of Grace Therapeutics, Inc.
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/s/ Prashant Kohli
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| Name: | Prashant Kohli | |
| Title: | Chief Executive Officer | |
| Date: | June 18, 2026 | |
This certification accompanies the Report pursuant to §906 of the Sarbanes-Oxley Act of 2002 and shall not, except to the extent required by the Sarbanes-Oxley Act of 2002, be deemed “filed” by Grace Therapeutics, Inc. for purposes of §18 of the Securities Exchange Act of 1934, as amended, or otherwise subject to the liability of that section.
Exhibit 32.2
SECTION 906 CERTIFICATION
Pursuant to section 906 of the Sarbanes-Oxley Act of 2002 (subsections (a) and (b) of section 1350, chapter 63 of title 18, United States Code) in connection with the Annual Report on Form 10-K of Grace Therapeutics, Inc. for the annual period ended March 31, 2026, as filed with the Securities and Exchange Commission on the date hereof (the “Report”), the undersigned officer hereby certifies, to such officer’s knowledge, that:
(1) The Report fully complies with the requirements of Section 13(a) or 15(d) of the Securities Exchange Act of 1934; and
(2) The information contained in the Report fairly presents, in all material respects, the financial condition and results of operations of Grace Therapeutics, Inc.
| /s/ Robert DelAversano | ||
| Name: | Robert DelAversano | |
| Title: | Principal Financial Officer | |
| Date: | June 18, 2026 | |
This certification accompanies the Report pursuant to §906 of the Sarbanes-Oxley Act of 2002 and shall not, except to the extent required by the Sarbanes-Oxley Act of 2002, be deemed “filed” by Grace Therapeutics, Inc. for purposes of §18 of the Securities Exchange Act of 1934, as amended, or otherwise subject to the liability of that section.