Opus Genetics, Inc 10-Q for FY2025 Q3
Opus Genetics, Inc. (IRD)
10-Q
2025-11-12
For: 2025-09-30
View Original
Added on
April 09, 2026
About this 10-Q
Opus Genetics, Inc filed this 10-Q on November 12, 2025, covering the quarter ended September 30, 2025 (FY2025 Q3).
Headline figures
Quarter ended September 30, 2025, as reported in this filing, compared with the same period a year earlier where the filing states it.
- Revenue
- $3.08M
- down 20.4% from $3.87M a year earlier
- Net loss
- $17.45M
- compared with a net loss of $7.53M a year earlier
- Diluted EPS
- -$0.25
- compared with -$0.29 a year earlier
UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, DC 20549
Form 10-Q
(Mark One)
For the Quarterly Period Ended September 30, 2025
OR
For the transition period from to ________
Commission File Number: 001-34079
(Exact name of Registrant as specified in its charter)
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(State or other jurisdiction of
incorporation or organization)
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(I.R.S. Employer
Identification No.)
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(Address of principal executive offices)
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(Zip Code)
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Registrant’s telephone number, including area code: (984 ) 884-6030
N/A
(Former name or former address, if changed since last report)
Securities registered pursuant to Section 12(b) of the Act:
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Title of each class
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Trading Symbol
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Name of each exchange on which registered
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The
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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 (section 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 the 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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Large accelerated filer
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Accelerated filer
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Smaller reporting company
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Emerging growth company
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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 is a shell company (as defined in Rule 12b-2 of the Exchange Act). Yes ☐
No ☒
The number of outstanding shares of the registrant’s common stock as of November 10, 2025 was 68,964,208 .
OPUS GENETICS, INC.
FORM 10-Q
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Page
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PART 1 – FINANCIAL INFORMATION
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Item 1.
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Item 2.
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Item 3.
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Item 4.
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PART II – OTHER INFORMATION
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Item 1.
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Item 1A.
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47
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Item 2.
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Item 3.
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Item 4.
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Item 5.
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Item 6.
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Opus Genetics, Inc.
(in thousands, except share amounts and par value)
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As of
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September 30,
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December 31,
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Assets
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(Unaudited) |
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Current assets:
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Cash and cash equivalents
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$
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$
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Prepaids and other current assets
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Short-term investments
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Total current assets
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Property and equipment, net
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Total assets
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$
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Liabilities and stockholders’ equity
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Current liabilities:
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Accounts payable
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$
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Accrued expenses and other liabilities
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Total liabilities
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Commitments and contingencies (Note 4 and Note 10)
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Series A preferred stock, par value $
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Stockholders’ equity:
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Preferred stock, par value $
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Common stock, par value $
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Additional paid-in capital
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Accumulated deficit
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Total stockholders’ equity
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Total liabilities, Series A preferred stock and stockholders’ equity
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$
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$
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See accompanying notes
Opus Genetics, Inc.
(in thousands, except share and per share amounts)
(Unaudited)
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For the Three Months Ended
September 30,
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For the Nine Months Ended
September 30,
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2025
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2024
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Operating expenses:
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General and administrative
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Research and development
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Total operating expenses
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Other income, net
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Loss before income taxes
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Benefit (provision) for income taxes
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| Other comprehensive loss, net of tax |
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| Comprehensive loss |
$
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$
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Basic and diluted
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$
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Number of shares used in per share calculations:
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Basic and diluted
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See accompanying notes.
Opus Genetics, Inc.
(in thousands, except share amounts)
(Unaudited)
Series A Preferred Stock
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Common Stock
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Additional
Paid–In
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Accumulated
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Total
Stockholders’
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||||||||||||||||||||||||
| Shares | Amount |
Shares
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Amount
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Capital
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Deficit
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Equity
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Balance at December 31, 2023
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Issuance of common stock in connection with the at-the-market program and purchase agreement
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Stock-based compensation
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Share repurchases for the payment of employee taxes
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Net and comprehensive loss
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Balance at March 31, 2024
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Issuance of common stock in connection with the at-the-market program and purchase agreement
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Stock–based compensation
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Net and comprehensive loss
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—
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Balance at June 30, 2024
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Issuance of common stock in connection with the at-the-market program
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Issuance costs
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Stock-based compensation
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Net and comprehensive loss
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Balance at September 30, 2024
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Balance at December 31, 2024
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$
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Issuance of common stock and pre-funded warrants in connection with the March 2025 offering and private placement
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Issuance of common stock in connection with at-the-market program
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Stock-based compensation
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Share repurchases for the payment of employee taxes
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Exercise of stock options
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Net and comprehensive loss
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Balance at March 31, 2025
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Conversion of preferred stock
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Stock-based compensation
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Issuance cost, net credit
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Net and comprehensive loss
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Balance at June 30, 2025
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Issuance of common stock in connection with private placement and at-the-market program
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Issuance costs
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Stock-based compensation
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Exercise of stock options
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Net and comprehensive loss
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Balance at September 30, 2025
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See accompanying notes.
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For the Nine
Months Ended
September 30,
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2025
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2024
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Operating activities
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Net loss
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$
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Adjustments to reconcile net loss to net cash used in operating activities:
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Stock-based compensation
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Depreciation
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Fair value change in warrant and other derivative liabilities
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Non-cash interest
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Unrealized loss from short-term investments
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Warrant financing costs
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Change in assets and liabilities:
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Accounts receivable
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Contract assets and unbilled receivables
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Prepaids and other current assets
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Accounts payable
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Accrued expenses
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Net cash used in operating activities
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Investing activities
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Net cash used in investing activities
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Financing activities
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Proceeds from issuance of common stock and pre-funded warrants in connection with the March 2025 offering and March
2025 private placement
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Proceeds from issuance of warrants in connection with the March 2025 offering and March 2025 private placement
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Proceeds from issuance of common stock in connection with the at-the-market program, private placement and purchase
agreement
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| Proceeds from funding agreement, related party |
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Issuance costs
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(
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Exercise of stock options
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Share repurchases for the payment of employee taxes
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Net cash provided by financing activities
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Net increase (decrease) in cash and cash equivalents
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Cash and cash equivalents at beginning of period
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Cash and cash equivalents at end of period
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$
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$
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Supplemental disclosure of cash flow information:
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Cash paid for income taxes
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$
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$
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Cash paid for interest
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$
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$
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Supplemental non-cash financing transactions:
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Conversion of Series A preferred stock into common stock
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$ | $ | ||||||
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Change in unpaid issuance costs
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$
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$
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See accompanying notes.
Nature of Business and Basis of
Presentation
Opus Genetics, Inc. (the “Company” or “Opus”), a Delaware corporation formerly known as Ocuphire Pharma, Inc. (the “Company” or
“Opus”), is a clinical-stage biopharmaceutical company developing gene therapies for the treatment of inherited retinal diseases (“IRDs”) and small molecule therapies for other ophthalmic disorders. The Company’s headquarters is
located in Durham, North Carolina.
On October 22, 2024, the Company acquired a private corporation then operating under the name of “Opus Genetics,
Inc.” (“Private Opus”) pursuant to the terms of an Agreement and Plan of Merger, dated as of October 22, 2024 (such agreement, the “Merger Agreement” and the transaction consummated via the Merger Agreement, the “Opus
Acquisition”), by and among the Company, Private Opus, and certain merger subsidiaries party thereto.
The Company’s pipeline includes assets from the adeno-associated virus (“AAV”) based gene therapy portfolio of Private Opus that
address mutations in genes that cause different forms of Leber congenital amaurosis (“LCA”), bestrophinopathy, and retinitis pigmentosa. The Company’s most advanced gene therapy program is designed to address mutations in the LCA5
gene, which encodes the lebercilin protein. More specifically, we are developing OPGx-LCA5 to treat LCA5-associated inherited retinal disease (“IRD”), an early-onset retinal degeneration, and an open-label, dose-escalation Phase
1/2 clinical trial is ongoing. OPGx-BEST1 is another gene therapy candidate in the Company’s portfolio. This asset is being developed for the treatment of IRDs associated with mutations in the BEST1 gene, which can lead to legal blindness. Apart from gene
therapies, the Company’s pipeline also includes Phentolamine Ophthalmic Solution 0.75%, a non-selective alpha-1 and alpha-2 adrenergic antagonist to reduce pupil size as well as APX3330, a novel small-molecule inhibitor of Ref-1
designed to slow the progression of non-proliferative diabetic retinopathy.
In November 2022, the Company entered into a license and collaboration agreement (as
amended, the “Viatris License Agreement”) with Viatris, Inc. (“Viatris”), pursuant to which it granted Viatris an exclusive license to develop, manufacture, import, export and commercialize its refractive product candidate
Phentolamine Ophthalmic Solution 0.75% (“PS”). PS is a once-daily eye drop formulation of phentolamine mesylate designed to reduce pupil diameter and improve visual acuity. PS was approved by the FDA for the treatment for
pharmacologically induced mydriasis produced by adrenergic agonists (e.g., phenylephrine) or parasympatholytic (e.g., tropicamide) agents, or a combination thereof under the brand name RYZUMVI® in September 2023 and was launched commercially in April 2024. The Company is also developing PS for the treatment of presbyopia, an ophthalmic disorder that involves the progressive
loss of ability to focus on close objects that results in blurred near vision, difficulty seeing in dim light, and eye strain. Additionally, the Company is currently developing PS for decreased vision under mesopic (low) light
conditions following keratorefractive surgery, pursuant to a received FDA agreement under Special Protocol Assessment (“SPA”).
Basis of Presentation
The accompanying condensed consolidated financial statements have been prepared by the Company, without audit, pursuant to
the rules and regulations of the Securities and Exchange Commission (“SEC”) and include the accounts of the Company’s subsidiary. All intercompany transactions and balances have been
eliminated in consolidation. Certain information and footnote disclosures normally included in financial statements prepared in accordance with U.S. generally accepted accounting principles (“GAAP”) have been condensed or
omitted pursuant to such rules and regulations. The Company’s fiscal year begins on January 1 and ends on December 31.
The December 31, 2024 condensed consolidated balance sheet was derived from audited financial
statements and may not include all disclosures required by GAAP; however, the Company believes that the disclosures are adequate to make the information presented not misleading. The accompanying condensed consolidated financial statements
should be read in conjunction with the audited financial statements and related notes thereto for the year ended December 31, 2024 included in the Company’s Annual Report on Form 10-K filed with the SEC on March 31, 2025.
In the opinion of management, all adjustments, consisting of only normal recurring adjustments
that are necessary to present fairly the financial position, results of operations, and cash flows for the interim periods, have been made. The results of operations for the interim periods are not necessarily indicative of the operating
results for the full fiscal year or any future periods.
The derivative liability line item reflected on the December 31, 2024 consolidated balance
sheet in the prior year was reclassified to the accrued expenses and other liabilities line item in the amount of $2,000
Liquidity
Since its inception, the Company has devoted substantially all of its resources to drug development and clinical trials.
The Company expects that its cash and cash equivalents as of September 30, 2025 of $30.8 million will be sufficient to fund its operations for at least the next 12 months from the date of issuance of these financial statements.
In the future, the Company may need to raise additional funds until it is able to generate sufficient revenues to fund its
development activities. The Company’s future operating activities, coupled with its plans to raise capital or issue debt financing, may provide additional liquidity in the future, however these actions are not solely within the
control of the Company and the Company is unable to predict the outcome of these actions to generate the liquidity ultimately required.
Use of Estimates
The preparation of financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the
amounts reported in the condensed consolidated financial statements and accompanying notes. Actual results could differ from those estimates.
Segment Information
Operating segments are components of an enterprise for which separate financial
information is available and are evaluated regularly by the Company’s chief operating decision maker in deciding how to allocate resources and assessing performance. The Company’s chief operating decision maker is its Chief
Executive Officer. The Company’s Chief Executive Officer views the Company’s operations and manages its business in one operating segment, which is the business of development of products related to vision performance and health.
Accordingly, the condensed consolidated financial statements and accompanying notes contained herein include the measure of profit or loss, categories of expenses and other financial information that is evaluated by the Company’s
Chief Executive Officer.
Summary of Significant Accounting Policies
The Company’s significant accounting policies are described in Note 1, “Company Description and Summary of Significant Accounting Policies” to the consolidated financial statements in its Annual Report on Form 10-K for the year ended December 31, 2024, that was filed
with the SEC, on March 31, 2025. Since the date of those financial statements, except as set forth below under “Warrant liabilities”, there have been no material changes to the Company’s significant accounting policies.
Warrant liabilities
The
Company issued warrants to purchase equity securities in connection with the March 2025 financings and are recorded under the warrant liabilities line item in the accompany condensed consolidated balance sheets (See Note 8 –
Financings). The Company accounts for these warrants as a liability at fair value when the valuation inputs are not fixed and determinable. Additionally, issuance costs associated with the warrant liability were expensed as incurred
and reflected as financing costs in the accompanying condensed consolidated statements of comprehensive loss. The Company adjusts the liability for changes in fair value until the earlier of; 1) the exercise or 2) expiration of the
warrants. Any future change in fair value of the warrant liabilities, when outstanding, is recognized in the condensed consolidated statements of comprehensive loss under the fair value change in warrant and other derivative
liabilities line item.
Recent Accounting Pronouncements
In December 2023, the Financial Accounting Standards Board (“FASB”) issued ASU 2023-09 Income
Taxes (Topic 740): Improvements to Income Tax Disclosures, which enhances income tax disclosures primarily related to the rate reconciliation and income taxes paid information. This guidance also includes certain other
amendments to improve the effectiveness of income tax disclosures. This ASU is effective for fiscal years beginning after December 15, 2024, including interim periods within those fiscal years and should be applied on a prospective
basis, with retrospective application permitted. The Company has determined that the adoption of this guidance will augment its income tax disclosures related mainly to categorical detail in the rate reconciliation and jurisdictional
detail associated with income taxes paid.
In November 2024, the
FASB issued ASU 2024-03 Income Statement - Reporting Comprehensive Income - Expense Disaggregation Disclosures (Subtopic 220-40): Disaggregation of Income Statement Expenses. This ASU is intended to improve the disclosures related to expenses and provide investors
more detailed information about certain types of expenses. This ASU is effective for annual periods beginning after December 15, 2026, and interim reporting periods beginning after December 15, 2027, with early adoption permitted.
The Company is currently evaluating the potential impact that this new standard will have on our consolidated financial statements and related disclosures.
| 2. |
Mergers
|
Acquisition of Opus Genetics
As described in Note 1 – Company Description and Summary of Significant Accounting Policies, on October 22, 2024, the Company completed the stock purchase of Private Opus. Under the terms of the Merger Agreement, at the closing of the Opus Acquisition, the Company issued to the security holders of Private Opus
Merger with Rexahn
On November 5, 2020, the Company completed a merger transaction with Rexahn (“Rexahn Merger”). In connection with the Rexahn Merger, the
Company, Shareholder Representatives Services LLC, as representative of the Rexahn stockholders prior to the Merger, and Olde Monmouth Stock Transfer Co., Inc., as the rights agent, entered into the Contingent Value Rights Agreement (the
“CVR Agreement”).
Pursuant to the terms of the Rexahn Merger and the CVR Agreement, Rexahn stockholders of record as of immediately prior to the effective time of the Rexahn Merger received one contingent value right (“CVR”) for each share of Rexahn common stock held.
The CVRs are not transferable, except in certain limited circumstances, will not be
certificated or evidenced by any instrument, will not accrue interest and will not be registered with the SEC or listed for trading on any exchange. The CVR Agreement will continue in effect until the later of the end of the CVR Term (as
defined in the CVR Agreement) and the payment of all amounts payable thereunder. As of September 30, 2025, no payments subject
to the CVR Agreement had been received beyond those
previously reported in the second and third quarters of calendar year 2021. In addition, no milestones had been accrued as of
September 30, 2025, as there were no
| 3. |
Fair Value Measurements
|
The Company follows accounting guidance that emphasizes that fair value is a market-based
measurement, not an entity-specific measurement. Fair value is defined as “the price that would be received to sell an asset or paid to transfer a liability in an orderly transaction between market participants at the measurement date.”
Fair value measurements are defined on a three-level hierarchy:
| • |
Level 1 inputs: Unadjusted quoted prices for identical assets or liabilities in active markets;
|
| • |
Level 2 inputs: Quoted prices for similar assets and liabilities in active markets, quoted prices in
markets that are not active, or inputs which are observable, whether directly or indirectly, for substantially the full term of the asset or liability; and
|
| • |
Level 3 inputs: Unobservable inputs in which there is little or no market data available, which
requires management to develop its own assumptions in pricing the asset or liability.
|
As of September 30, 2025 and December 31, 2024, the fair values of cash and cash equivalents, accounts receivable,
contract assets and unbilled receivables, prepaid and other assets, accounts payable and accrued expenses approximated their carrying values because of the short-term nature of these assets or liabilities. The fair value of the long-term
funding agreement, related party line item was determined on an amortized cost basis, based on an effective rate of interest.
The fair value of the short-term investments, while
outstanding, were based on observable Level 1 inputs in the form of quoted market prices from a major stock exchange. The fair value of the liabilities associated with the equity line financing facility and the March 2025 Warrants and
March 2025 Private Placement Warrants (as defined below) are based on cash flow models discounted at current implied market rates representing expected returns by market participants for similar instruments and are based on Level 3 inputs
as well the Company’s underlying stock price and associated volatility, expected term and market interest rates (See Note 8 – Financings). There were no
The fair value of financial instruments measured
on a recurring basis is as follows (in thousands):
|
|
As of September 30, 2025
|
|||||||||||||||
|
Description
|
Total
|
Level 1
|
Level 2
|
Level 3
|
||||||||||||
|
Assets:
|
||||||||||||||||
|
Short-term investments
|
$
|
|
* |
$
|
|
* |
$
|
|
$
|
|
||||||
|
Total assets at fair value
|
$
|
|
* |
$
|
|
* |
$
|
|
$
|
|
||||||
|
Liabilities:
|
||||||||||||||||
|
Warrant liabilities
|
$
|
|
$
|
|
$
|
|
$
|
|
||||||||
| Other derivative liabilities |
||||||||||||||||
|
Total liabilities at fair value
|
$
|
|
$
|
|
$
|
|
$
|
|
||||||||
|
*
|
|
|
|
As of December 31, 2024
|
|||||||||||||||
|
Description
|
Total
|
Level 1
|
Level 2
|
Level 3
|
||||||||||||
|
Assets:
|
||||||||||||||||
|
Short-term investments
|
$
|
|
$
|
|
$
|
|
$
|
|
||||||||
|
Total assets at fair value
|
$
|
|
$
|
|
$
|
|
$
|
|
||||||||
|
Liabilities:
|
||||||||||||||||
|
Other derivative liabilities
|
$
|
|
$
|
|
$
|
|
$
|
|
||||||||
|
Total liabilities at fair value
|
$
|
|
$
|
|
$
|
|
$
|
|
||||||||
The following table provides a roll-forward of
short-term investments measured at fair value on a recurring basis using observable level 1 inputs for the nine months ended September 30, 2025 and 2024 (in thousands):
|
Nine Months Ended
September 30,
|
||||||||
|
|
2025
|
2024
|
||||||
|
Short-term investments
|
||||||||
|
Balance as of beginning of period
|
$
|
|
$
|
|
||||
|
Unrealized loss
|
(
|
)
|
(
|
)
|
||||
|
Balance as of end of period
|
$
|
|
* |
$
|
|
|||
|
*
|
De minimis value
|
The following table provides a
roll-forward of liabilities measured at fair value on a recurring basis using unobservable level 3 inputs for the nine months ended September 30, 2025 and 2024 (in thousands):
|
Nine Months Ended
September 30,
|
||||||||
|
2025
|
2024
|
|||||||
|
Warrant liabilities
|
||||||||
|
Balance as of beginning of period
|
$
|
|
$
|
|
||||
|
Issuance of March 2025 Warrants and March 2025 Private Placement Warrants
|
||||||||
|
Change in fair value
|
||||||||
|
Balance as of end of period
|
$
|
|
$
|
|
||||
|
Nine Months Ended
September 30,
|
||||||||
|
2025
|
2024
|
|||||||
|
Other derivative liabilities
|
||||||||
|
Balance as of beginning of period
|
$
|
|
$
|
|
||||
|
Change in fair value
|
( |
) | ||||||
|
Balance as of end of period
|
$
|
|
$
|
|
||||
There were no
|
4.
|
Commitments and Contingencies
|
Apexian Sublicense Agreement
On January 21, 2020, the Company entered into a sublicense agreement
with Apexian Pharmaceuticals, Inc., pursuant to which it obtained exclusive worldwide patent and other intellectual property rights. In exchange for the patent and other intellectual rights, the Company agreed to certain milestone
payments and royalty payments on future sales (See Note 10 – Apexian Sublicense Agreement). As of September 30, 2025, there was sufficient uncertainty with regard to any future cash milestone payments under the sublicense agreement that no liabilities were recorded related to the
sublicense agreement.
University of Pennsylvania
LCA5/RDH12 License Agreement
On June 15, 2022, Opus entered into an amended and restated license agreement (the “LCA5/RDH12 Agreement”) with the Trustees of the University of Pennsylvania
(“Penn”) pursuant to which it was granted an exclusive, royalty-bearing license to certain patents and a non-exclusive license to certain information relating to products directed towards treatment or correction of mutation of the
LCA5 or RDH12 genes. In return for these rights, the Company is obligated to make certain development, regulatory and commercial milestone payments up to a maximum potential aggregate amount of $2.6 million and royalty payments on future net sales of such products. Until the Company is required to pay royalties under the LCA5/RDH12
Agreement, the Company must pay a de minimis annual license maintenance fee to Penn. The Company is also obligated to make payments on any sublicense income, with such percentage depending on
the stage of product development, which there was no sublicense income for any of the periods presented. During the quarter ended September 30, 2025, the first development milestone under the LCA5/RDH12 Agreement was satisfied and the
related amount payable was included in accrued expenses and other liabilities as of September 30, 2025 and research and development expense for the three and nine months ended September 30, 2025.
Iveric Asset Purchase Agreement –
BEST1 and RHO Programs
On December 23, 2022, Opus entered into an asset purchase agreement (the “Iveric Agreement”) with a subsidiary of Iveric Bio, Inc. (“Iveric”) pursuant to which
the Company acquired certain assets, including the BEST1 License (as defined below), relating to the BEST1 and RHO products. In return for these rights, the Company is obligated to make payments to Iveric upon the achievement of
specified development and commercial milestones, the maximum potential aggregate amount of such payments being $111.7
million. As of September 30, 2025, the Company determined that none of the future obligations under the agreement were probable and therefore no liabilities were recorded related to the
agreement.
Penn and University of Florida BEST1
License Agreement
On April 10, 2019, Iveric entered into an exclusive patent license agreement (as amended, the “BEST1 License”) with Penn and the University of Florida Research
Foundation (“UF”), which agreement was assigned to Opus under the terms of the Iveric Agreement. Under the BEST1 License, Opus received exclusive patent rights and non-exclusive knowhow and data rights with regard to products to
treat diseases associated with mutations in the BEST1 gene. In return for these rights, the Company is obligated to make payments to Penn upon the achievement of certain clinical, regulatory and commercial milestones, the maximum
potential aggregate amount of such payments being $76.4 million. The Company is also obligated to make royalty payments on
future net sales of licensed BEST1 products. Until the Company is required to pay royalties under the BEST1 License, the Company must pay a de minimis annual license maintenance fee to UF and
Penn. The Company must also make payments on any sublicense income, with such percentage depending on the stage of product development, which there was no sublicense income during any of the periods presented. In consideration for
Penn and UF’s consent to the assignment of the BEST1 License to us under the Iveric Agreement, the Company will also pay Penn a percentage of each milestone payment that we are required to pay to Iveric under the Iveric Agreement. As
of September 30, 2025, the Company determined that none of the future obligations under the agreement were probable and therefore no liabilities were recorded related to the
agreement.
Penn and UF RHO License Agreement
On June 6,
2018, Iveric entered into an exclusive patent license agreement (the “RHO License”) by and between Penn and UF pursuant to which the Company has exclusive patent rights and non-exclusive knowhow and data rights with regard to products
to treat rhodopsin-mediated diseases as a result of the Iveric Agreement as defined above. In return for these rights, the Company is obligated to make development and commercial milestone payments, the maximum potential aggregate
amount of such payments being $93.5 million and royalty payments on future sales of such products. As of September 30, 2025, the Company determined that none of the future obligations under the agreement were probable and therefore no liabilities were
recorded related to the agreement.
Massachusetts Eye and Ear
Infirmary License Agreement
On November
9, 2021, Opus entered into a license agreement with the Massachusetts Eye and Ear Infirmary (“MEEI”), granting an exclusive worldwide license of MEEI patents for use in the NMNAT1 program for all products and processes including the
treatment of retinal disease in humans, and a non-exclusive worldwide license to technological information. In return for these rights, the Company is obligated to make development milestone payments, the maximum potential aggregate
amount of such payments being $0.4 million and royalty payments on future sales of such products. As of September 30, 2025, the Company determined that none of the future obligations under the agreement were probable and therefore no liabilities were recorded related to the
agreement.
Facility and Other Leases
On January 1, 2025, the Company relocated its headquarters to Durham, North Carolina. On July 1st, 2025, the Company amended the lease for its headquarters in Durham, North Carolina for three months through September 30, 2025 , and the lease is currently in place on a month-to-month basis. The headquarters lease qualifies for the short-term
exception under ASC 842, Leases.
The
Company also leases additional laboratory space in Durham, North Carolina on a month-to-month basis. Upon the Opus Acquisition, the Company assumed a number of equipment leases that expired in and are now on a month-to-month basis. Both the laboratory space and equipment leases qualify for the short-term exception under ASC 842, Leases.
The rent
expense associated with all leases amounted to $0.1 million and less than $0.1 million during the three months ended September 30, 2025 and 2024, respectively and $0.2 million and less than $0.1 million during the
nine months ended September 30, 2025 and 2024, respectively.
Other
In the ordinary course of business, from time to time, the Company may be subject to a broad range of claims and legal proceedings that
relate to contractual allegations, patent infringement and other claims. In addition, the Company from time to time may be potentially committed to reimburse third parties for costs incurred associated with business development related
transactions upon the achievement of certain milestones. The Company establishes accruals when applicable for matters and commitments for which it believes losses are probable and can be reasonably estimated.
| 5. |
Supplemental Balance Sheet Information
|
Accrued expenses and other liabilities
Accrued expenses and other liabilities consist of the following (in thousands):
| As of |
||||||||
|
September 30,
|
December 31,
|
|||||||
|
2025
|
2024
|
|||||||
|
Research and development services and supplies
|
$
|
|
$
|
|
||||
| Compensation and benefits |
|
|
||||||
| Professional services | ||||||||
|
Other
|
|
|
||||||
|
Total
|
$
|
|
$
|
|
||||
| 6. |
Related Party Transactions
|
Consulting Agreements with Dr. Pepose
On April 11, 2024, the Company entered into a consulting agreement (the “Pepose Consulting Agreement”) with Dr. Pepose, a former director of the Company. Pursuant to the Pepose Consulting Agreement, Dr. Pepose was
paid a monthly consulting fee and received an award of 32,000 RSUs, as well as stock options to purchase 48,000 shares of the Company’s common stock. The RSUs vested in 12 equal monthly installments that began on May 11, 2024 and concluded on April 11, 2025. On November 21, 2024, the Pepose Consulting Agreement was amended to continue through
April 11, 2026.
For the agreements with Dr. Pepose described above, the Company incurred related consulting expenses of $0.1 million and $0.4 million during the three and nine months ended September 30, 2025, respectively, and the Company incurred related consulting expenses of $0.1 million and $0.3
million during the three and nine months ended September 30, 2024,
respectively.
March 2025 Subscription Agreements with Dr. George Magrath and Cam Gallagher
On March 21, 2025, the Company entered into a subscription agreement with each of Dr. George Magrath, the Company’s Chief Executive Officer, and Cam Gallagher, the chairman of the Company’s board of directors (the “Board”), in connection with a private offering of our securities. For more
information, see Note 8 – Financings.
August 2025 Subscription Agreements with Cam Gallagher and Sean Ainsworth
On August 25, 2025, the Company entered into subscription agreements pursuant to which the Company agreed to issue and sell in
a private placement (the “August 2025 Private Placement”) to certain investors 3,138,338 shares of its common stock for
gross proceeds of approximately $3.5 million.
The August 2025 Private Placement was led by Cam Gallagher, Chair of the Company’s Board, with an investment of $1.0 million, along with participation by Sean Ainsworth, the lead independent director of the Board, in the amount of $0.1 million, and other investors for the balance of the Private Placement. For more information, see Note 8 – Financings.
Consulting Agreement with Dr. Jean Bennett
In connection with Dr. Jean Bennett’s appointment as a member of the Board, effective October 22, 2024, she and the Company entered into a consulting
agreement (the “Bennett Consulting Agreement”), pursuant to which Dr. Bennett will provide consulting services to the Company for a one-year
period. Pursuant to the Bennett Consulting Agreement, Dr. Bennett was granted a restricted stock unit award with respect to 100,000
shares of the Company’s common stock, which award vested on October 22, 2025. The Company incurred no
Letter Agreement and Strategic Partnership—FFB
On
August 25, 2022, Private Opus entered into a binding letter of agreement (“2022 Binding Letter Agreement”) with Foundation Fighting Blindness (“FFB”) and the Jaeb Center for Health Research (the “JCHR”) to collaborate on natural
history studies involving individuals with retinal dystrophies associated with mutations in multiple genes of interest. Under the terms of the 2022 Binding Letter Agreement, FFB and the JCHR had the sole responsibility and authority
to design and conduct the study, with input from the Company. Subject to certain conditions, the 2022 Binding Letter Agreement required that the Company provide FFB with a total of $2.0 million of funding to support the study, such amount being payable in an initial installment of $0.4 million at the time of submission of the final study protocol to the Institutional Review Board of the JCHR and, subject to certain conditions, in four annual installments of $0.4
million on the anniversaries of such submission. On May 27, 2025, the Company entered into a binding letter of agreement (“2025 Letter Agreement”) with FFB and the JCHR, which superseded and canceled the 2022 Binding Letter Agreement. As of September 30, 2025 a total of $0.4 million was paid by the Company under the 2022 Binding Letter Agreement and no more payments were due under the 2022 Binding Letter Agreement.
Under the 2025 Letter Agreement, the Company will collaborate with FFB and the JCHR on portions of a study involving individuals with retinal
dystrophies associated with mutations in the RDH12 or BEST1 genes (the “Study”). The term of this 2025 Letter Agreement ends on the date that is two months from the Study’s completion. FFB and the JCHR, as its designee, shall have the sole responsibility and authority to design and conduct the Study, with input from the Company. Under the
2025 Letter Agreement, the Company is obligated to make two payments to FFB: (1) $0.3 million on or before June 30, 2025 and (2) $0.3 million on or before January 31, 2027 upon receipt of semi-annual reports from FFB outlining the progress being made in the Study, including visit completion status and publication plans, and
the ability for the Company to provide ongoing comments and suggestions regarding possible changes to the Study. Such payments shall constitute the sole compensation paid to FFB in return for Company access to research materials
and datasets.
The Company paid the initial $0.3
million due on or before June 30, 2025 under the 2025 Letter Agreement which was recorded as research and development expense. As of September 30, 2025, the Company is required to fund one additional installment in the aggregate
of $0.3 million upon receipt of future semi-annual reports.
RDF Agreement
On June 13, 2025, the Company entered into a funding agreement (the “RDF Agreement”) with the Foundation Fighting Blindness Retinal Degeneration Fund
(“RDF”), whose sole member is FFB, a significant stockholder of the Company, relating to the Company’s program to develop gene therapies to treat patients impacted by retinitis pigmentosa caused by pathogenic variants in the Mer
proto-oncogene tyrosine kinase (MERTK) gene (the “MERTK Program”). The RDF Agreement provides for nondilutive funding by RDF of up to $2.0
million to support the development of the MERTK Program, $1.0 million of which was disbursed to the Company in June
2025 and up to $1.0 million of which may be disbursed to the Company upon achievement of a specified development
milestone subject to RDF’s receipt of eligible funds.
Under the RDF Agreement, the Company is subject to certain diligence obligations to develop and commercialize a product under the MERTK Program. If
the Company is unable to achieve certain milestones by certain dates, or otherwise fails to meet its diligence obligations, the Company will be obligated to collaborate with RDF to out-license or otherwise make applicable rights
available to a third party.
In addition, the Company will pay a milestone payment equal to the total amounts funded by RDF under the RDF Agreement upon the achievement of a regulatory milestone. The Company will also make tiered
royalty payments to RDF in low-to-mid single percentages until RDF has received aggregate royalty payments equal to 300 %
of the amounts funded by RDF under the Agreement. In the event of a change of control of the Company or a sale or exclusive license of the MERTK Program, RDF will have the option to require the Company to buy out RDF’s
interest under the Agreement for an amount equal to 100 % of the funds disbursed to the Company under the
Agreement. The Agreement may be terminated by either party for cause, including material breach or bankruptcy, subject to a cure period.
The RDF Agreement was accounted for as debt under ASC 470, Debt. ASC 470 requires interest expense to be recorded under an effective interest rate method.
During the three and nine months ended September 30, 2025, interest expense was $0.1 22.3 % and was recorded as interest expense in the accompanying condensed
consolidated statements of comprehensive loss. The accreted liability in connection with the RDF Agreement was $1.1
million as of September 30, 2025 and was recorded under the long-term funding agreement, related party line item in the accompanying condensed consolidated balance sheets.
| 7. |
Series A Preferred Stock
|
On October 22, 2024, the Company filed a Certificate of Designation of Preferences, Rights and
Limitations of the Series A Non-Voting Convertible Preferred Stock with the Secretary of State of the State of Delaware (the “Certificate of Designation”) in connection with the Opus Acquisition. The Certificate of Designation provides for
the authorization of 14,146 shares of Series A preferred stock, of which 14,145.374 Series A preferred stock were issued upon close of the Opus Acquisition. On April 30, 2025, the Company held its 2025 Annual Meeting of Stockholders. During the
2025 Annual Meeting, the Company’s stockholders voted to approve the conversion of each share of Series A preferred stock into 1,000
shares of common stock. Subsequently, on May 5, 2025, all shares of Series A preferred stock were converted into 14,145,374 shares
of common stock.
| 8. |
Financings
|
August 2025 Private Placement
On
August 25, 2025, the Company entered into subscription agreements pursuant to which the Company agreed to issue and sell in the August 2025 Private Placement to certain investors 3,138,338 shares of its common stock for gross, and net, proceeds of approximately $3.5 million.
The
Company intends to use the net proceeds of the August 2025 Private Placement to expedite manufacturing process development, including scale-up of clinical and commercial production and testing, to ensure sufficient supply of cGMP
material for its gene therapy candidates, OPGx-LCA5 and OPGx-BEST1. No underwriting discounts or commissions were paid
with respect to the August 2025 Private Placement.
The
August 2025 Private Placement was led by Cam Gallagher, Chair of the Board, along with participation by Sean Ainsworth, the lead independent director of the Board, and other investors. See Note 6 – Related Party Transactions.
March 2025
Financings
On
March 21, 2025, the Company entered into an underwriting agreement with Craig-Hallum Capital Group, LLC, as the sole underwriter. Pursuant to the underwriting agreement, the Company agreed to issue and sell, in an underwritten
public offering (the “March 2025 Offering”), 12,219,736 shares of common stock and warrants to purchase up to 21,052,631 shares of common stock (the “March 2025 Warrants”). Each share of common stock was sold together with one March 2025 Warrant
to purchase one share of common stock, at a price to the public of $0.95 per share and related March 2025 Warrant. The Company also issued 8,832,895 pre-funded warrants (“Pre-Funded Warrants”) at a price to the public of $0.9499 per Pre-funded Warrant.
On
March 21, 2025, the Company entered into a subscription agreement (the “Subscription Agreement”) with each of Dr. George Magrath, the Company’s Chief Executive Officer, and Cam Gallagher, the chairman of the Board. Pursuant to the
Subscription Agreement, the Company agreed to issue and sell, in a private offering (the “March 2025 Private Placement”), a total of 392,157
shares of common stock to Mr. Magrath and 784,314 shares of common stock to Mr. Gallagher, as well as 392,157 warrants to purchase shares of common stock to Mr. Magrath and 784,314 warrants to purchase shares of common stock to Mr. Gallagher (“March 2025 Private Placement Warrants”). Each March 2025 Private Placement Warrant has an
initial exercise price of $1.15 , expires on the five-year anniversary of the original issuance date and may be called by
the Company 30 days following the release of the Company’s OPGx-BEST1 DUO-1001 Cohort 1 data upon achievement of a volume weighted average price of our common stock for 30 consecutive trading days of over $1.725 per share and the
trading average daily volume for such 30 day period exceeds $150,000 per trading day. See Note 6 – Related Party Transactions.
The
combined gross proceeds from the March 2025 Offering and the March 2025 Private Placement, which both closed on March 24, 2025 (the “Closing Date”), were approximately $21.5 million, before deducting underwriting discounts and commissions and offering expenses payable by the Company in the amount of $1.8 million.
March
2025 Warrants
The
March 2025 Warrants have an initial exercise price equal to $0.95 per share of common stock and are exercisable for five years from the date of issuance. The exercise prices and numbers of shares of common stock issuable upon exercise are subject to
appropriate adjustment in the event of stock dividends, stock splits, reorganizations or similar events affecting the common stock and also upon any distributions of assets, including cash, stock or other property to our
stockholders. A holder may not exercise the March 2025 Warrant if, after giving effect to such exercise, the holder (together with its affiliates) would beneficially own (as determined in accordance with the terms of the March 2025
Warrants) more than 4.99 % (or, at the election of the holder, 9.99 %) of the outstanding common stock immediately after giving effect to the exercise. Lastly, certain volatility provisions in the event of a fundamental transaction
precluded the March 2025 Warrants from being considered indexed to the Company’s own stock, and as such, were classified on the condensed consolidated balance sheets as warrant liabilities.
The
March 2025 Warrants are callable by the Company in certain circumstances. Subject to certain exceptions, in the event that the March 2025 Warrants are outstanding, if, after the Closing Date, (i) the Company has announced OPGx-BEST1
DUO-1001 Cohort 1 data, (ii) the volume weighted average price of the common stock for 30 consecutive trading days
(“Warrant Measurement Period”), which 30 consecutive trading day period shall not have commenced until after the initial
exercise date) exceeds $1.425 (subject to adjustment), (iii) the trading average daily volume for such Warrant
Measurement Period exceeds $150,000 per trading day and (iv) the March 2025 Warrant holder is not in possession of any
information that constitutes or might constitute material non-public information which was provided by the Company, its subsidiaries or any of its officers, directors, employees, agents or affiliates, then the Company may, within
one trading day of the end of such Warrant Measurement Period, upon notice, call for cancellation of all or any portion of the March 2025 Warrants for which a notice of exercise has not yet been delivered for consideration equal to
$0.001 per March 2025 Warrant share.
In the event of a fundamental transaction, as defined in the Form of Warrant, the
holders of the March 2025 Warrants will be entitled to receive upon exercise the kind and amount of securities, cash or other property that the holders would have received had they exercised immediately prior to such fundamental
transaction. Additionally, as more fully described in the Form of Warrant, in the event of certain fundamental transactions, the holders of the March 2025 Warrants will be entitled to receive consideration in an amount equal to
the Black Scholes Value of the remaining unexercised portion of the March 2025 Warrants on the date of consummation of such fundamental transaction.
The fair value of the March 2025 Warrants at the time of issuance was $14.7 million and were recorded in the warrant liabilities line item in the accompanying condensed consolidated balance sheets upon
issuance. The fair value change during the three and nine months ended September 30, 2025 was an expense of $9.0
million and $5.5 million, respectively. The fair value of these instruments were based on a Monte Carlo simulation incorporating a
volatility rate of 72.5 % and 80.0 % as of September 30, 2025 and March 24, 2025, respectively, a risk free rate of 3.6 %
and 4.0 % as of September 30, 2025 and March 24, 2025, respectively, the market price of the Company’s common stock at
$1.65 and $1.15
per share as of September 30, 2025 and March 24, 2025, respectively, and other factors over a simulated term of 4.5
years and 5 years at September 30, 2025 and March 24, 2025, respectively. As of September 30, 2025 the transaction
costs attributed to the March 2025 Warrants amounted to approximately $1.3 million and were recorded in the
accompanying condensed consolidated statements of comprehensive loss under financing costs.
March 2025 Private Placement Warrants
The March 2025 Private Placement Warrants have an initial exercise price equal to $1.15 per share of common stock and are
exercisable for five years from the date of issuance. The March 2025 Private Placement Warrants are callable by
the Company in certain circumstances. Subject to certain exceptions, in the event that the March 2025 Private Placement Warrants are outstanding, if, after the Closing Date, (i) the Company announced OPGx-BEST1 DUO-1001
Cohort 1 data, (ii) the volume weighted average price of the common stock for 30 consecutive trading days (the
“Private Placement Measurement Period”, which 30 consecutive trading day period shall not have commenced until
after the initial exercise date) exceeds $1.725 (subject to adjustment), (iii) the trading average daily volume
for such Private Placement Measurement Period exceeds $150,000 per trading day and (iv) the March 2025 Private
Placement Warrant holder is not in possession of any information that constitutes or might constitute material non-public information which was provided by the Company, its subsidiaries or any of its officers, directors,
employees, agents or affiliates, then the Company may, within one trading day of the end of such Private Placement Measurement Period, upon notice, call for cancellation of all or any portion of the March 2025 Private
Placement Warrants for which a notice of exercise has not yet been delivered for consideration equal to $0.001
per March 2025 Private Placement Warrant share. Other terms under the March 2025 Private Placement Warrants are generally identical to the terms of the March 2025 Warrants discussed above. Lastly, certain volatility
provisions in the event of a fundamental transaction precluded the March 2025 Private Placement Warrants from being considered indexed to the Company’s own stock, and as such, were classified on the condensed consolidated
balance sheets as warrant liabilities.
The March 2025 Private Placement Warrants have an initial exercise price equal to $
The fair value of the March 2025 Private Placement Warrants at the time of issuance was $0.8
million and were recorded in the warrant liabilities line item in the accompanying condensed consolidated balance sheets upon issuance. The fair value change during the three and nine months ended September 30, 2025 was an
expense of $0.5 million and $0.3 million, respectively. The fair value of these instruments were based on a Monte Carlo simulation incorporating a volatility rate of 72.5 % and 80.0 % as of September 30,
2025 and March 24, 2025, respectively, a risk free rate of 3.6 % and 4.0 % as of September 30, 2025 and March 24, 2025, respectively, the market price of the Company’s common stock at $1.65 and $1.15 per share as of September 30,
2025 and March 24, 2025, respectively, and other factors over a simulated term of 4.5 years and 5 years as of September 30, 2025 and March 24, 2025, respectively. As of September
30, 2025 transaction costs attributed to the March 2025 Private Placement Warrants were de minimis and were recorded in the accompanying condensed consolidated statements of comprehensive loss under financing costs.
Pre-Funded Warrants
The Pre-Funded Warrants have an exercise price of $0.0001 per share of common stock and are immediately exercisable and are
exercisable at any time until exercised in full. The exercise prices and numbers of shares of common stock issuable upon exercise are subject to appropriate adjustment in the event of stock dividends, stock splits,
reorganizations or similar events affecting the common stock. A holder may not exercise the Pre-Funded Warrant if, after giving effect to such exercise, the holder (together with its affiliates) would beneficially own (as
determined in accordance with the terms of the Pre-Funded Warrants) more than 4.99 % (or, at the election of the
holder, 9.99 %) of the outstanding common stock immediately after giving effect to the exercise. In the event of
a fundamental transaction, as defined in the Form of Pre-Funded Warrant, the holders of the Pre-Funded Warrants will be entitled to receive upon exercise of the Pre-Funded Warrants the kind and amount of securities, cash or
other property that the holders would have received had they exercised the Pre-Funded Warrants immediately prior to such fundamental transaction.
The Pre-Funded Warrants were recorded in the accompanying condensed consolidated balance sheets as Additional paid-in capital.
Pre-Funded Warrants
The Pre-Funded Warrants have an exercise price of $
The Pre-Funded Warrants were recorded in the accompanying condensed consolidated balance sheets as Additional paid-in capital.
At-The-Market Program
On January 13, 2025, the Company filed a prospectus supplement with the SEC in connection with the establishment of an at-the-market equity offering program (the “ATM Program”) for the
offer and sale, from time to time, of shares of its common stock having an aggregate offering price of up to $40.0 million.
On the same date, the Company entered into a Sales Agreement (the “Sales Agreement”) with Leerink Partners LLC (“Leerink”), pursuant to which the Company may offer and sell shares of its common stock through or to Leerink, acting as
sales agent, under the ATM Program. In connection with the entry into the Sales Agreement, the Company terminated its prior ATM program established under the Capital on DemandTM Sales Agreement dated March 11, 2021, between the Company
and JonesTrading Institutional Services LLC.
During the three and nine months ended September 30, 2025, the Company sold 1,472,703 and 1,825,656 shares of common stock, respectively, under the Leerink ATM program for gross proceeds of $1.7 million and $2.1 million, respectively, before deducting issuance expenses. Total issuance expenses, including sales agent fees and legal and accounting
expenses, were $0.1 million and $0.3
million for the three- and nine-month periods ended September 30, 2025, respectively. During the three and nine months ended September 30, 2024, the Company sold 219,406 and 1,608,522 shares
of common stock under the JonesTrading ATM program, respectively,
for aggregate gross proceeds of $0.5 million and $3.8 million, respectively, before deducting issuance expenses. Total issuance expenses, including sales agent fees and legal and accounting expenses, were $0.1 million and $0.2 million, for the three- and
nine-month periods ended September 30, 2024, respectively. As of September 30,
2025, the Company had sold an aggregate of 9,479,494 shares of common stock under the ATM programs since their inception,
resulting in gross proceeds of $28.5 million and total issuance costs of $1.4 million.
Registered Direct Offering
On June 4, 2021, the Company entered
into a placement agency agreement for a registered direct offering (“RDO”) with A.G.P./Alliance Global Partners (“AGP”). Pursuant to the terms of the placement agency agreement, AGP on June 8, 2021 sold an aggregate of 3,076,923 shares of the Company’s common stock and warrants to purchase 1,538,461 shares of the Company’s common stock (the “RDO Warrants”) at an offering price of $4.875 per one share and per of each RDO
Warrant. The RDO was made pursuant to the Company’s 2021 shelf registration.
The RDO Warrants have an exercise
price of $6.09 per share, are exercisable from the initial issuance date of June 8, 2021, and will expire five years following the initial issuance date. As of September 30, 2025, 1,538,461 RDO Warrants were outstanding and none have been exercised since issuance.
Subject to limited exceptions, a holder of a RDO Warrant will not have the right to exercise any portion of its RDO Warrants if the holder, together with its affiliates,
would beneficially own in excess of 4.99 % (or, at the election of a holder prior to the date of issuance, 9.99 %) of the number of shares of the Company’s common stock outstanding immediately after giving effect to such exercise; provided that upon
prior notice to the Company, the holder may increase or decrease the beneficial ownership limitation, provided further that in no event shall the beneficial ownership limitation exceed 9.99 %.
Pre-Merger Financing
On June 17,
2020, the Company, Rexahn and certain investors entered into a Securities Purchase Agreement, which was amended and restated in its entirety on June 29, 2020 (as amended and restated, the “Securities Purchase Agreement”). Pursuant to the
Securities Purchase Agreement, the investors invested a total of $21.15 million in cash, including $0.3 million invested by five
directors of the Company prior to the Rexahn Merger and one director of Rexahn upon closing of the Rexahn Merger (the
“Pre-Merger Financing”). The Pre-Merger Financing also included the issuance of Series A Warrants and Series B Warrants discussed further below.
Series A Warrants
The Series A
Warrants were issued on November 19, 2020 at an initial exercise price of $4.4795 per share, were immediately exercisable upon
issuance and have a term of five years from the date of issuance. The Series A Warrants are exercisable for 5,665,838 shares of common stock in the aggregate (without giving effect to any limitation on exercise contained therein) and were outstanding
as of September 30, 2025.
The Series A Warrants were accounted for and classified as equity on the accompanying condensed consolidated balance sheets.
Warrant
Activity and Summary
|
Warrants
|
Exercise
Price Per
Warrant
|
Weighted
Average
Exercise
Price
|
Weighted
Average
Term
(Years)
|
|||||||||||||
|
Outstanding and exercisable at December 31, 2024
|
|
$
|
|
$
|
|
|
||||||||||
|
Issued
|
|
$
|
|
$
|
|
|
||||||||||
|
Exercised
|
|
$
|
|
$
|
|
—
|
||||||||||
|
Expired
|
|
$
|
|
$
|
|
—
|
||||||||||
|
Outstanding and exercisable at September 30, 2025
|
|
$
|
|
$
|
|
|
||||||||||
The following table summarizes information about warrants outstanding at September 30, 2025:
|
Exercise Price
|
Number
Outstanding
|
Weighted Average
Remaining
Contractual
life (Years)
|
Number Exercisable
at
September 30,
2025
|
|||||||||||
|
$
|
|
|
|
|
* |
|||||||||
|
$
|
|
|
|
|
* |
|||||||||
|
$
|
|
|
|
|
||||||||||
|
$
|
|
|
|
|
||||||||||
|
Total
|
|
|
||||||||||||
|
*
|
|
The above tables exclude the 8,832,895
Pre-Funded Warrants issued in connection with the March 2025 Offering. The Pre-Funded Warrants were deemed outstanding common stock for net loss per share purposes (See Note 12 – Net Loss per Share).
| 9. |
Stock-based Compensation
|
Stock-based
compensation expense was included in general and administrative and research and development costs as follows in the accompanying condensed statements of comprehensive loss for the three- and nine-month periods indicated below (in thousands):
|
|
Three Months
Ended
September 30,
|
Nine Months
Ended
September 30,
|
||||||||||||||
|
|
2025
|
2024
|
2025
|
2024
|
||||||||||||
|
General and administrative
|
$ |
$
|
|
$
|
|
$
|
|
|||||||||
|
Research and development
|
|
|
|
|
||||||||||||
|
Total stock-based compensation
|
$
|
|
$
|
|
$
|
|
$
|
|
||||||||
Inducement Plan
On August 8, 2025, the Company amended the Opus Genetics, Inc. 2021 Inducement
Plan (the “Inducement Plan”) to adjust the reserve by 800,000 shares to a total of 4,125,258 shares of its common stock. The Inducement Plan is to be used exclusively for grants of awards to individuals who were
not previously employees or directors of the Company, as an inducement material to the individual’s entry into employment with the Company within the meaning of Rule 5635(c)(4) of Nasdaq’s continued listing requirements.
2020 Equity
Incentive Plan
In November 2020, the stockholders of the Company approved the 2020 Equity Incentive Plan
(the “2020 Plan”) for stock-based awards. Under the 2020 Plan, (i) 1,000,000 new shares of common stock were reserved for
issuance and (ii) up to 70,325 additional shares of common stock may be issued, consisting of (A) shares that remain
available for the issuance of awards under prior equity plans and (B) shares of common stock subject to outstanding stock options or other awards covered by prior equity plans that have been cancelled or expire on or after the date that
the 2020 Plan became effective. Under the 2020 Plan, the shares reserved automatically increase on January 1 of each year, for a period of not more than ten years from the date the 2020 Plan is approved by the stockholders of the Company, commencing on January 1, 2021 and ending on (and including) January 1, 2030, by an amount
equal to 5 % of the shares of common stock outstanding as of December 31st of the preceding calendar year. The 2020 Plan permits the grant of incentive and nonstatutory stock options, appreciation rights, restricted stock, restricted stock units, performance stock and
cash awards, and other stock-based awards. On January 1, 2025, 1,578,733 shares were added to the 2020 Plan as a
result of its annual increase provision.
2018
Equity Incentive Plan
Prior to the 2020 Plan, the Company had adopted a
2018 Equity Incentive Plan (the “2018 Plan”) in April 2018 under which 1,175,000 shares of the Company’s common stock were
reserved for issuance to employees, directors and consultants. Upon the effective date of the 2020 Plan, no additional
shares were available for issuance under the 2018 Plan.
Stock Options
During the three and nine months ended September 30, 2025, 733,448 and 2,793,277 stock
options were granted, respectively, to directors, officers, employees and consultants, generally vesting over a -
to four-year period with monthly, quarterly and annual vesting tranches. During the three and nine months ended September 30,
2024, zero and 1,021,166
stock options were granted, respectively, to directors, officers, employees and consultants, generally vesting over a - to four-year period with monthly, quarterly and annual vesting tranches.
The Company recognized $0.5 million and $0.5
million in stock-based compensation expense related to stock options during the three months ended September 30, 2025 and 2024, respectively, and $1.5 million and $1.4 million during the nine months ended
September 30, 2025 and 2024, respectively. There were 25,000 and 30,000 stock options exercised during the three and nine months ended September 30, 2025, respectively with an intrinsic value of less than $0.1 no
stock option exercises during the nine months ended September 30, 2024. As of September 30, 2025 and December 31, 2024, 7,218,064
and 5,073,736 stock options were outstanding, respectively. The following table provides a summary of stock option activity under the Company’s equity award plans:
|
Number of
Options
|
Weighted
Average
Exercise
Price
|
Weighted Average
Remaining
Contractual
Term (years)
|
Aggregate
Intrinsic
Value(1)
(in thousands)
|
|||||||||||||
|
|
||||||||||||||||
|
Outstanding at December 31, 2024
|
|
$
|
|
|
$
|
|
||||||||||
|
Granted
|
|
$
|
|
|||||||||||||
|
Exercised
|
(
|
)
|
$
|
|
||||||||||||
|
Forfeited/Cancelled
|
(
|
)
|
$
|
|
||||||||||||
|
Outstanding at September 30, 2025
|
|
$
|
|
|
$
|
|
||||||||||
|
Vested and expected to vest at September
30, 2025
|
|
$
|
|
|
$
|
|
||||||||||
|
Vested and exercisable at September 30,
2025
|
|
$
|
|
|
$
|
|
||||||||||
| (1) |
|
The weighted average fair value per share of options granted during the three and nine
months ended September 30, 2025 was $0.79 and $0.68 , respectively, during each of these periods. The weighted average fair value per share of options granted during the three and nine months ended September 30, 2024 was and $1.95 , respectively.
The Company measures the fair value of stock options with service-based vesting criteria to employees, directors, consultants and directors on the date of grant using the Black-Scholes option pricing model. The Company does not have
sufficient trading history of its common stock to support an internal calculation of volatility and expected term. As such, the Company has used a weighted average volatility considering the volatilities of several guideline companies.
For purposes of identifying similar entities, the
Company considered characteristics such as industry, length of trading history, and stage of life cycle. The assumed dividend yield was based on the Company’s expectation of not paying dividends in the foreseeable future. The average
expected life of the options was based on the contractual term for agreements that allow for exercise of vested options through the end of the contractual term upon termination of continuous service, and for all other agreements, was
based on the midpoint between the vesting date and the end of the contractual term according to the “simplified method” as described in Staff Accounting Bulletin 110. The risk-free interest rate is determined by reference to implied
yields available from U.S. Treasury securities with a remaining term equal to the expected life assumed at the date of grant. The Company records forfeitures when they occur.
The weighted average assumptions used
in the Black-Scholes option pricing model are as follows during the three and nine months ended September 30, 2025 and 2024:
|
|
Three Months
Ended
September 30,
|
Nine Months
Ended September 30, |
||||||||||||||
|
|
2025
|
2024
|
2025
|
2024
|
||||||||||||
|
Expected stock price volatility
|
|
%
|
|
%
|
|
%
|
|
%
|
||||||||
|
Expected life of options (years)
|
|
—
|
|
|
||||||||||||
|
Expected dividend yield
|
|
%
|
|
%
|
|
%
|
|
%
|
||||||||
|
Risk free interest rate
|
|
%
|
|
%
|
|
%
|
|
%
|
||||||||
During the three and nine months ended September 30, 2025,
During the three and nine months ended September 30, 2025, 145,319 and 618,949 options
were forfeited, respectively. During the three and nine months ended
September 30, 2024,
114,425 and 583,360
options were forfeited, respectively.
Restricted Stock Units
During the three and nine months ended September 30, 2025,
the Company granted an aggregate of 200,000 and 1,655,361 restricted stock units (“RSUs”), respectively, to members of the Board and certain officers, employees and consultants under
the 2020 Plan. The weighted average grant date per unit fair value of the RSUs granted during the three and nine months ended September 30, 2025 was $1.33 and $0.99 , respectively, during each of these periods. The vesting period of the RSUs range from a -year period to a four-year
period during which 25 % of the RSUs vest annually on each anniversary of the grant date, subject to the recipient’s
continued service on such dates.
During the three and nine months ended September
30, 2024, the Company granted an aggregate of zero and 592,222 RSUs, respectively, to the members of the Board and certain officers, employees and consultants under the 2020 Plan. The weighted average grant date per
unit fair value of the RSUs granted during the nine months ended September
30, 2024 was $2.24 . The vesting period of the RSUs range from a -year period to a four-year period during which 25 % of the RSUs vest annually on each anniversary of the grant date, subject to the recipient’s continued service on such dates.
During the three and nine months ended September 30, 2025, zero and 381,534 RSUs vested, respectively, and 26,038
and 257,826 were forfeited during the three and nine months ended
September 30, 2025, respectively.
During the three and nine months ended September 30, 2024, zero and 144,162 RSUs vested, respectively, and 36,660 and 119,330 RSUs were forfeited during the three and nine months ended September 30, 2024, respectively.
The total expense for the three and nine months ended September 30, 2025 related to RSUs was
$0.3 million and $1.1
million, respectively. The total expense for the three and nine months ended September 30, 2024 related to these RSUs was $0.3 million and $0.9
million, respectively. As of September 30, 2025 and December 31, 2024, there were 2,409,231 and 1,393,230 RSUs were outstanding, respectively.
The
following table summarized the Company’s RSU activity under the Company’s equity award plans:
|
|
Number of
Shares
|
|||
|
Non-vested at December 31, 2024
|
|
|||
|
Granted
|
|
|||
|
Forfeited
|
(
|
)
|
||
|
Vested
|
(
|
)
|
||
|
Non-vested at September 30, 2025
|
|
|||
Common Stock Issued for Services
The Company granted stock for services in the amount of and 84,243 common shares
during the three and nine month periods ended September 30, 2025, respectively, and and 81,234 common shares during the three and nine month periods ended September 30, 2024,
respectively. The awards were granted to members of the Board who elected to receive their Board compensation in the form of stock for services. The weighted average fair value of the shares granted was and $1.26 per share
during the three and nine month periods ended September 30, 2025, respectively, and and $3.01 per share during the three and nine month periods ended September 30, 2024,
respectively, and were 100 % vested upon issuance. The stock-based compensation related to these services amounted to and $0.1 million during
the three and nine month periods ended September 30, 2025, respectively, and
and $0.2 million during the three and nine month periods ended September 30, 2024, respectively.
General
As of September 30, 2025, 874,829 shares were available for future
issuance under the 2020 Plan and Inducement Plan, in the aggregate. No shares were available for future issuance under the
2018 Plan. Unrecognized stock-based compensation cost was $6.0 million as of September
30, 2025. The unrecognized stock-based expense is expected to be recognized over a weighted average period of 1.7 years.
| 10. |
Apexian Sublicense Agreement
|
On January
21, 2020, the Company entered into a sublicense agreement (as amended on June 4, 2020, the “Apexian Sublicense Agreement”) with Apexian, pursuant to which it obtained exclusive worldwide patent and other intellectual property rights that
constitute a Ref-1 Inhibitor program relating to therapeutic applications to treat disorders related to ophthalmic and diabetes mellitus conditions. The lead compound in the Ref-1 Inhibitor program is APX3330, which the Company intends to
develop as an oral tablet therapeutic to treat diabetic retinopathy initially, and potentially later to treat diabetic macular edema, geographic atrophy and age-related macular degeneration. In connection with the Apexian Sublicense
Agreement, the Company issued a total of 891,422 shares of its common stock to Apexian and to certain affiliates of Apexian in
calendar year 2020.
The Company
also agreed to make one-time milestone payments under the Apexian Sublicense Agreement for each of the first ophthalmic indication and the first diabetes mellitus indication for the development and regulatory milestones, and once for each
of several sales milestones. These milestone payments include (i) payments for specified developmental and regulatory milestones (including completion of the first Phase 2 trial and the first Phase 3 pivotal trial in the United States, and
filing and achieving regulatory approval from the FDA for the first New Drug Application for a compound) totaling up to $11
million in the aggregate and (ii) payments for specified sales milestones of up to $20 million in the aggregate, which net sales
milestone payments are payable once, upon the first achievement of such milestone. Lastly, the Company also agreed to make a royalty payment equal to a single-digit percentage of its net sales of products associated with the covered patents
under the Apexian Sublicense Agreement. If it is not terminated pursuant to its terms, the Apexian Sublicense Agreement shall remain in effect until expiration of the last to expire of the covered patents.
None of the milestone or royalty payments were triggered or
deemed probable as of September 30, 2025.
|
11.
|
License and Collaboration Revenue and Other Funding Agreements
|
Viatris License Agreement
On
November 6, 2022, the Company entered into the Viatris License Agreement, pursuant to which it granted Viatris an exclusive, perpetual, sub-licensable license to develop, manufacture, import, export and commercialize (i) PS, for treating
(a) reversal of mydriasis, (b) night vision disturbances or dim light vision, and (c) presbyopia, and (ii) PS and low dose pilocarpine for treating presbyopia (together, the “PS Products”) worldwide except for certain countries and
jurisdictions in Asia (the “Viatris Territory”). The Company retains the exclusive right to develop, manufacture, have manufactured, import, export and commercialize the PS Products outside of the Viatris Territory.
Under the terms of the Viatris License Agreement, the Company in partnership with Viatris, will develop the PS Products in the United States. Viatris will reimburse the Company for agreed-to budgeted costs related to the development of the PS Products through FDA approval and then share costs above the agreed upon threshold amount. Viatris will be responsible for developing the PS Products in countries and jurisdictions in the Viatris Territory outside of the United States. In addition, in August 2025, Opus and Viatris entered into a side letter to the Viatris License Agreement providing for the sharing of expenses arising in connection with a patent dispute.
Under the terms of the Viatris License Agreement, the Company in partnership with Viatris, will develop the PS Products in the United States. Viatris will reimburse the Company for agreed-to budgeted costs related to the development of the PS Products through FDA approval and then share costs above the agreed upon threshold amount. Viatris will be responsible for developing the PS Products in countries and jurisdictions in the Viatris Territory outside of the United States. In addition, in August 2025, Opus and Viatris entered into a side letter to the Viatris License Agreement providing for the sharing of expenses arising in connection with a patent dispute.
Pursuant to the Viatris License Agreement, the Company received a one-time non-refundable cash payment of $35 million in November 2022 for the exclusive, perpetual, sub-licensable license to develop, manufacture, import, export and commercialize the
PS Products in the Viatris Territory. In addition, with respect to the PS Products, the Company will be eligible to receive potential additional payments of up to $130 million upon achieving certain specified regulatory or net sales milestones, with the first milestone payment of $10 million already made following approval by the FDA of PS for reversal of mydriasis, which occurred during the third quarter of 2023. The Company will also receive tiered
royalties, starting at low double-digit royalties up to low 20 % royalties, based on the aggregate annual net sales of all PS
Products in the United States, and will receive low double-digit royalties based on all annual net sales in the Viatris Territory outside of the United States. The royalty payments will continue on a country-by-country basis from the date
of the first commercial sale of the first PS Product in a country of the Viatris Territory until December 31, 2040.
The Viatris
License Agreement was accounted for under the provisions of ASC 606. In accordance with the provisions under ASC 606, the Company identified two
distinct performance obligations at the effective date: (1) the license to its intellectual property and (2) research and development services.
The
Company determined that the licenses transferred represented functional intellectual property. As such, the revenue related to the licenses was recognized at the point in time in which the license/know-how was delivered to Viatris which
occurred during the fourth quarter of 2022. The Company determined that revenue related to the initial research and development services that were constrained to the 120-day non-cancellation period were to be recognized over time as the services were rendered based on an estimated percentage of completion input model. The initial research and
development services were completed in the first quarter of 2023. Revenue related to the on-going research and development services are based on activities completed during the period.
Recognition
of Revenue
Revenue
recognized under the Viatris License Agreement during the three and nine months ended September 30, 2025 was $3.1 million and $10.3 million, respectively. Revenue recognized under the Viatris License Agreement during the three and nine
months ended September
30, 2024 was $3.9 million and $6.7
million, respectively, related to the output of ongoing research and development services and to a much lesser extent royalty payments. The Company records a provision for credit losses, when
appropriate, based on historical experience, current conditions and reasonable supportable forecasts. The Company has not incurred any bad debt expense to date and no allowance for credit losses has been recorded during the periods
presented.
Regulatory Milestones under the Viatris License Agreement
The Company has
evaluated the regulatory milestones that may be received in connection with the Viatris License Agreement. There is uncertainty that the events to obtain the remaining regulatory milestones (aside from the approval by the FDA of RYZUMVI) will
be achieved given the nature of clinical development and the stage of the development of the PS Products. These remaining regulatory milestones will be constrained until it is probable that a significant revenue reversal will not occur.
Sales Milestone and Royalty Payments
Sales
milestones and royalties relate predominantly to a license of intellectual property granted to Viatris and are determined by sales or usage-based thresholds. The sales milestones and royalties are accounted for under the royalty
recognition constraint and are accounted for as constrained variable consideration. The Company applies the royalty recognition constraint for each commercial milestone and
only recognize revenues for each once a sale of a licensed product (achievement of each) occurs.
Each of the
remaining regulatory and sales milestone performance obligations (aside from the $10 million milestone payment related to the FDA’s
approval of PS in the third quarter of 2023) were constrained as of September 30, 2025 and no revenue was recognized related to these
milestones.
A reconciliation of the closing balance of the contract assets and unbilled receivables
associated with the Viatris License Agreement is as follows as of September 30, 2025 and 2024 (in thousands):
|
Nine Months Ended
September 30,
|
||||||||
| 2025 | 2024 |
|||||||
|
Contract assets and unbilled receivables
|
||||||||
|
Balance as of beginning of nine-month period
|
$
|
|
$ | |||||
|
Revenue recognized
|
|
|||||||
|
Reclassification to Accounts receivable related to costs billed under the Viatris License Agreement
|
(
|
)
|
( |
) | ||||
|
Balance as of end of nine-month period
|
$
|
|
$ | |||||
BioSense License and Assignment
On March 10, 2020, prior to the Rexahn Merger, Rexahn entered into an amendment to its collaboration and license agreement, (as amended, the “BioSense License and
Assignment Agreement”) with BioSense to advance the development and commercialization of the Rexahn RX-3117 drug compound (“RX-3117”) for all human uses in the Republic of Singapore, China, Hong Kong, Macau, and Taiwan (the “BioSense
Territory”).
Under the BioSense License and Assignment Agreement, the Company is
eligible to receive additional milestone payments in an aggregate of up to $84.5 million upon the achievement of development,
regulatory and commercial goals and will also be eligible to receive tiered royalties at low double-digit rates on annual net sales in the BioSense Territory. The Company determined that none of the milestone payments under the BioSense
License and Assignment Agreement were probable of payment as of September 30, 2025, and as a result, no revenue related to the milestones was recognized, as the achievement of
events entitling the Company to any milestone payments were highly susceptible to factors outside of the Company’s control. Future sales-based royalties related to the exclusive license to develop RX-3117, if any, will be recognized in the
period the underlying sales transaction occurs.
Payments received under the BioSense License and Assignment Agreement will be subject to the CVR Agreement described in
Note 2 – Mergers.
Processa License Agreement
On June 16, 2021, the Company entered into a license agreement (the “Processa License Agreement”) with Processa
Pharmaceuticals, Inc. (“Processa”), pursuant to which the Company agreed to grant Processa an exclusive license to develop, manufacture and commercialize RX-3117 globally, excluding the BioSense Territory.
Pursuant to the agreement, Processa is obligated to make future payments to the Company upon the achievement of certain development, regulatory and commercial milestones. In addition, Processa is obligated to pay the Company mid-single-digit percentage royalties based on annual sales.
Pursuant to the agreement, Processa is obligated to make future payments to the Company upon the achievement of certain development, regulatory and commercial milestones. In addition, Processa is obligated to pay the Company mid-single-digit percentage royalties based on annual sales.
On June 27, 2025, Processa notified the Company that it would not be developing RX-3117 and terminated the Processa License Agreement, effective October 25, 2025 (the “Termination Date”). No future payments will be received under the Processa License Agreement after the Termination Date and if any future payments are received prior to the Termination Date, they will be subject to the CVR Agreement described in Note 2 – Mergers.
The Company determined that none
of the milestone payments under the Processa License Agreement were probable of payment as of September 30, 2025, and as a result, no
revenue related to the milestones was recognized.
SBIR Grant Agreement
In September 2024, Private Opus received a Small Business Innovation Research (“SBIR”) grant through the Department of
Health and Human Services in the amount of $0.9 million to be used on the development of the RHO product. Direct and allocated
indirect costs for development activities are reimbursed on a draw-down basis as development activities are completed. For the three and nine months ended September 30, 2025, the Company recognized $0.1 million and $0.3 million of revenue related to the SBIR
grant, respectively, and was recorded as other income, net in the accompanying condensed consolidated statements of comprehensive loss. There was no
RDH12 Agreement
On July 22, 2025, the Company, together with its wholly owned subsidiary, OpusTX, LLC (collectively, “Opus”), entered into a
funding and license agreement (the “RDH12 Agreement”) with Eyes on the Future (“EOTF”), and RDH12 Fund for Sight (the “Fund,” and together with EOTF, the “Funding Parties”), charitable organizations, relating to Opus’ program to develop
gene therapies that treat patients with inherited retinal degeneration associated with mutations in the RDH12 gene (the “RDH12 Program”).The RDH12 Agreement provides for funding by the Funding Parties of up to $1.6 million to support the development of the RDH12 Program. Opus is required to use the funding to conduct development activities in
accordance with a mutually agreed development plan.
Under the RDH12 Agreement, Opus is subject to certain diligence obligations to develop a product under the RDH12 Program. If Opus is unable to achieve certain
milestones by the specified dates, or if certain other events occur (a “License Trigger Event”), then the Funding Parties may exercise their rights under a non-exclusive, global, royalty-free and fully paid-up license granted by Opus to the
Funding Parties to develop products under the RDH12 Program. If the Funding Parties exercise such license rights, then Opus will receive a non-exclusive license under the data and other intellectual property generated by the Funding
Parties to develop products under the RDH12 Program, and the right to negotiate an exclusive license to such data and intellectual property to commercialize products under the RDH12 Program. The RDH12 Agreement includes certain restrictions
on Opus’ ability to out-license rights to the RDH12 Program, and during the term of the RDH12 Agreement, Opus may not grant a third party an exclusive license to develop or commercialize products under the RDH12 Program in the United States
without the prior written consent of the Funding Parties.
The term of the RDH12 Agreement continues until the earlier of (a) dosing by Opus of three patients in a Phase 1a/2b clinical trial prior to a License Trigger
Event, and (b) the first commercial sale of a product under the RDH12 Program following receipt of regulatory approval in the United States or certain other European countries. The RDH12 Agreement will also terminate if an exclusive,
global licensee of Opus for the RDH12 Program assumes Opus’ obligations under the RDH12 Agreement. The RDH12 Agreement may be terminated by either party for cause, including material breach or bankruptcy, subject to a cure period, or by the
Funding Parties for convenience following a License Trigger Event.
Eligible research and development costs under the RDH12 Agreement, as approved by the Funding Parties, are
reimbursed on a draw-down basis as development activities are completed by the Company. For the three and nine months ended September 30, 2025, the Company recognized $0.1
| 12. |
Net Loss per
Share
|
Basic loss per share of common stock is computed by dividing net loss by the weighted average
number of shares of common stock outstanding during the period. Diluted earnings or loss per share of common stock is computed similarly to basic loss or earnings per share except the weighted average shares outstanding are increased to
include additional shares from the assumed exercise of any common stock equivalents, if dilutive. The Company’s Series A preferred stock, warrants, stock options and RSUs, while outstanding, are considered common stock equivalents for
this purpose. Diluted earnings is computed utilizing the treasury method for the warrants, stock options and RSUs. Diluted earnings with respect to the Series A preferred stock
utilizing the if-converted method was not applicable during the periods presented as no conditions required for conversion had occurred. No incremental common stock equivalents were included in calculating diluted loss per share because
such inclusion would be anti-dilutive given the net loss reported for the periods presented.
The following potential common shares were not considered in the computation of diluted net loss per share as their effect would have been anti-dilutive for the three- and nine-month periods presented below:
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September 30,
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| 2025 | 2024 | |||||||
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Warrants
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Stock options
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RSUs
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13.
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Income Taxes
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The effective tax rate for the three and nine months ended September 30, 2025 and 2024 was zero no
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14.
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Deferred Compensation Plan
|
Effective October 1st, 2021, the Company began offering a 401(k) plan (“401K Plan”) to its employees. All employees
are eligible to participate in the 401K Plan. The Company makes
matching contributions equal to 100 % on the first 3 % of compensation that is deferred as an elective deferral and an additional 50 % on the next 2 % of compensation. The Company’s matching
contributions are made on a payroll-by-payroll basis. During the three months ended September 30, 2025 and 2024, the Company contributed less than $0.1 million and less than $0.1 million to the 401K Plan, respectively. During the
nine months ended September 30, 2025 and 2024, the Company contributed $0.1 million and $0.1 million to the 401K Plan,
respectively.
| 15. |
Subsequent Events
|
November 2025 Registered Direct Offering
On November 5, 2025, the Company entered into a securities purchase agreement to sell securities in a registered direct offering for gross
proceeds of approximately $23.0 million, before deducting offering expenses.
In the offering, the Company sold an aggregate of 3,827,751
shares of its common stock at a price of $2.09 per share and, in lieu of common stock to certain investors, pre-funded warrants
to purchase up to an aggregate of 7,177,033 shares of common stock at a purchase price of $2.0899 per pre-funded warrant. Each pre-funded warrant has an exercise price of $0.0001 per share of common stock, will be immediately exercisable subject to certain conditions set forth in each pre-funded warrant, and will not expire. The
offering closed on November 7, 2025.
Opus Genetics, Inc.
Form 10-Q
| Item 2. |
Management’s Discussion and Analysis of Financial Condition and Results of Operations
|
The following discussion of our financial condition and results of operations should be read in conjunction with the unaudited financial statements and
notes included in Part I “Financial Information”, Item I “Financial Statements” of this Quarterly Report on Form 10-Q (the “Report”) and the audited financial statements and related footnotes included in our Annual Report on Form 10-K for the
year ended December 31, 2024.
Forward-Looking Statements
Certain statements contained in this Report are not statements of historical fact and are forward-looking statements within the meaning of Section 27A of the
Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended (the “Exchange Act”). Forward-looking statements give current expectations or forecasts of future events or our future financial or operating
performance. Such statements include, but are not limited to, statements concerning our strategic business plans, the applications of our product candidates, ongoing discussions with the U.S. Federal Drug Administration (the “FDA”) regarding
various of our drug products, and continued drug development and commercialization under our agreement with Viatris, Inc. (“Viatris”). In some cases, you can identify forward-looking statements by the following words: “anticipate,” “believe,”
“could,” “continue,” “estimate,” “expect,” “intend,” “may,” “ongoing,” “plan,” “potential,” “predict,” “project,” “should,” “will,” “would” or the negative of those terms, and similar expressions that convey uncertainty of future events or
outcomes to identify these forward-looking statements.
These forward-looking statements reflect our management’s beliefs and views with respect to future events, are based on estimates and assumptions as of the date of this
Report and are subject to risks and uncertainties, many of which are beyond our control, that could cause our actual results to differ materially from those in these forward-looking statements, including, without limitation:
| • |
Our clinical data related to gene therapies for the treatment of inherited retinal diseases (“IRDs”) is preliminary and related to a relatively small group of patients, and, as a result, data that initially appears promising may be
revised, updated, or invalidated at a later data readout and/or may ultimately not be capable of duplication in additional patients;
|
| • |
Failure to successfully integrate our businesses following our acquisition of former Opus Genetics Inc. (the “Opus Acquisition”) could have a material adverse effect on our business, financial condition and results of operations;
|
| • |
The Opus Acquisition significantly expanded our product pipeline and business operations and shifted our business strategies, which may not improve the value of our common stock;
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| • |
Our gene therapy product candidates are based on a novel technology that is difficult to develop and manufacture, which may result in delays and difficulties in obtaining regulatory approval;
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| • |
Our planned clinical trials may face substantial delays, result in failure, or provide inconclusive or adverse results that may not satisfy FDA requirements to further develop our therapeutic products;
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| • |
Delays or difficulties associated with patient enrollment in clinical trials may affect our ability to conduct and complete those clinical trials and obtain necessary regulatory approvals;
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| • |
Changes in regulatory requirements could result in increased costs or delays in development timelines;
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| • |
We depend heavily on the success of our product pipeline; if we fail to find strategic partners or fail to adequately develop or commercialize our pipeline products, our business will be materially harmed;
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| • |
Others may discover, develop, or commercialize products similar to those in our pipeline before or more successfully than we do or develop generic variants of our products even while our product patents
remain active, thereby reducing our market share and potential revenue from product sales;
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| • |
We do not currently have any sales or marketing infrastructure in place and we have limited drug research and discovery resources;
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| • |
The future commercial success of our products could significantly depend upon several uncertain factors, including third-party reimbursement practices and the existence of competitors with similar products;
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| • |
Product liability lawsuits against us or our suppliers or manufacturers could cause us to incur substantial liabilities and could limit commercialization of any product candidate that we may develop;
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| • |
Failure to comply with health and safety laws and regulations could lead to material fines;
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| • |
We have not generated significant revenue from sales of any products and expect to incur losses for the foreseeable future;
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| • |
Our future viability is difficult to assess due to our short operating history and our future need for substantial additional capital, access to which could be limited by any adverse developments that
affect the financial services markets;
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| • |
Raising additional capital may cause our stockholders to be diluted, among other adverse effects;
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| • |
We operate in a highly regulated industry and face many challenges adapting to sudden changes in legislative reform or the regulatory environment, which affects our pipeline stability and could impair our
ability to compete in international markets;
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| • |
We may not receive regulatory approval to market our developed product candidates within or outside of the U.S.;
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| • |
With respect to any of our product candidates that receive marketing approval, we may be subject to substantial penalties if we fail to comply with applicable regulatory requirements;
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| • |
Our potential relationships with healthcare providers and third-party payors will be subject to certain healthcare laws and regulations, which could expose us to extensive potential liabilities;
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| • |
We rely on third parties for material aspects of our business, such as conducting our nonclinical and clinical trials and supplying and manufacturing bulk drug substances, which exposes us to certain risks;
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| • |
We may be unsuccessful in entering into or maintaining licensing arrangements (such as the Viatris License Agreement (as defined below)) or establishing strategic alliances on favorable terms, which could
harm our business;
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| • |
Our current focus on the cash-pay utilization for future sales of RYZUMVI may limit our ability to increase sales or achieve profitability with this product;
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| • |
Inadequate patent protection for our product candidates may result in our competitors developing similar or identical products or technology, which would adversely affect our ability to successfully
commercialize;
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| • |
We may be unable to obtain full protection for our intellectual property rights under U.S. or foreign laws;
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| • |
We may become involved in lawsuits for a variety of reasons associated with our intellectual property rights, including alleged infringement suits initiated by third parties;
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| • |
We are dependent on our key personnel, and if we are not successful in attracting and retaining highly qualified personnel, we may not be able to successfully implement our business strategy;
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| • |
As we grow, we may not be able to operate internationally or adequately develop and expand our sales, marketing, distribution, and other corporate functions, which could disrupt our operations;
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| • |
The market price of our common stock is expected to be volatile;
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| • |
Our common stock may be subject to delisting from the Nasdaq Capital Market (“Nasdaq”) and delisting could adversely affect our ability to access capital markets;
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| • |
Factors out of our control related to our securities, such as securities litigation or actions of activist stockholders, could adversely affect our business and stock price and cause us to incur significant
expenses;
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| • |
Instability and operational disruptions at government agencies, such as the FDA, may adversely impact our development and commercialization plans by causing delays and requiring the use of additional, unforeseen resources to obtain
regulatory approval for trials and products in our pipeline; and
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| • |
Impact from current or proposed tariffs on imported goods we purchase.
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We discuss many of these risks in greater detail under Part I, Item 1A “Risk Factors” in our Annual Report on Form 10-K for the year ended December 31, 2024 and below under
the heading “Risk Factors,” and in subsequent reports filed with or furnished to the Securities and Exchange Commission (the “SEC”). Moreover, we operate in a very competitive and rapidly changing environment. New risks emerge from time to
time. It is not possible for our management to predict all risks, nor can we assess the impact of all factors on our business or the extent to which any factor, or combination of factors, may cause actual results to differ materially from those
contained in any forward-looking statements we may make. Given these uncertainties, you should not place undue reliance on these forward-looking statements.
Any forward-looking statement made by us in this Report speaks only as of the date hereof or as of the date specified herein. We undertake no obligation to publicly update any
forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by applicable laws or regulations.
Overview
Opus Genetics, Inc. (the “Company,” “Opus,” “we,” “us,” or “our”) is a clinical-stage biopharmaceutical company developing gene
therapies for the treatment of IRDs and small molecule therapies for other ophthalmic disorders.
On October 22, 2024, Opus Genetics, Inc., a Delaware corporation formerly known as Ocuphire Pharma, Inc. (the “Company,” “Opus,” “we,” “us” or “our”), acquired a private
corporation then operating under the name of “Opus Genetics Inc.” (“Private Opus”) pursuant to the terms of an Agreement and Plan of Merger, dated as of October 22, 2024 (such agreement, the “Merger Agreement” and the transaction consummated via
the Merger Agreement, the “Opus Acquisition”), by and among the Company, Private Opus, and certain merger subsidiaries party thereto. As consideration for the Opus Acquisition, the Company issued 5,237,063 shares of its common stock and
14,145.374 shares of Series A preferred stock, each of which is convertible into 1,000 shares of common stock.
Gene Therapy Programs
Our pipeline features a portfolio of seven adeno-associated virus (“AAV”) based gene therapies that address mutations in genes that cause different forms of Leber congenital amaurosis (“LCA”),
bestrophinopathy, and retinitis pigmentosa.
OPGx-LCA5
OPGx-LCA5 is designed to address a form of LCA due to biallelic mutations in the LCA5 gene, which encodes the lebercilin protein. LCA5-associated
IRD is an early-onset severe inherited retinal dystrophy. Studies in patients with this mutation have reported evidence for the dissociation of retinal architecture and visual function in this disease, suggesting an opportunity for therapeutic
intervention through gene augmentation. OPGx-LCA5 uses an adeno-associated virus 8 (AAV8) vector to precisely deliver a functional LCA5 gene to the outer retina via a single subretinal injection. The program has been granted Rare Pediatric
Disease, Regenerative Medicine Advanced Therapy (RMAT), and Orphan Drug designations from the FDA.
OPGx-LCA5 is currently being evaluated in an open-label, Phase 1/2 clinical trial. To date, six late-stage participants have been treated with
OPGx-LCA5, all of whom have experienced improvements in vision, providing evidence of biological activity with the potential for functional restoration of vision in individuals with advanced disease. In September 2025, we reported positive data
from the six participants. The three pediatric participants treated over three months demonstrated large gains in cone-mediated vision with improvements across multiple measures of visual function. In the three adult participants, responses have
been observed out to 18 months, underscoring the potential durability of the treatment response. OPGx-LCA5 has been well tolerated with no ocular serious adverse events or dose-limiting toxicities.
On November 6, 2025 the Company announced the successful completion of a Type B RMAT meeting with the FDA regarding OPGx-LCA5. The meeting
provided constructive feedback from the FDA on key elements of the Company’s registration strategy, including Chemistry, Manufacturing and Controls (“CMC”), and the pivotal trial design. The FDA acknowledged the significant unmet medical need
for individuals with LCA5-related blindness and reaffirmed its commitment to regulatory flexibility for rare genetic diseases.
The Company will incorporate the FDA’s feedback into its updated clinical development and CMC plans for the Phase 3 portion of the study to
include enrolling as few as 8 participants in a single arm, 12-month study utilizing an adaptive design, which provides flexibility on endpoints and number of participants, reflective of LCA5 as a rare condition with an urgent medical need.
The Company expects the Phase 3 portion of the trial will include a run-in period prior to dosing to evaluate the natural history of each
participant to serve as their own control in the study. The Company is actively identifying patients for this segment and has enrolled the first participant for ongoing disease monitoring. Following availability of validated clinical drug
supply manufactured with the intended commercial processes, dosing with OPGx-LCA5 is anticipated in the second half of 2026 with topline clinical data expected approximately one year later.
In September 2025, the FDA introduced the Rare Disease Evidence Principles (“RDEP”) review process to provide greater speed and predictability
in the review of therapies intended to treat rare diseases with very small patient populations with significant unmet medical need and that are driven by a known genetic defect that is the major driver of the pathophysiology. With a patient
population of fewer than 1,000 individuals, the Company believes that its LCA5 program meets the eligibility criteria for the RDEP process and plans to submit an application.
OPGx-BEST1
OPGx-BEST1 is being developed for the treatment of IRDs associated with mutations in the BEST1 gene. BEST1 disease, or vitelliform macular dystrophy, is a rare,
inherited retinal condition causing macular degeneration by mutations in the BEST1 gene, leading to progressive vision loss and, in some cases, blindness. In preclinical studies conducted in a naturally occurring canine model of BEST1 disease,
OPGx-BEST1 demonstrated restoration of the retinal pigment epithelium-photoreceptor interface using AAV-mediated gene delivery, providing evidence in
support of a first-in-man clinical trial. In August 2025, we announced FDA clearance of an Investigational New Drug (“IND”) application to initiate a clinical trial. An adaptive, open-label, dose-exploring Phase 1/2 trial, known as BIRD1, is
currently recruiting participants to study the safety and tolerability of subretinally injected OPGx-BEST1 in participants with Best Vitelliform Macular Dystrophy (BVMD) or Autosomal-Recessive Bestrophinopathy (ARB). Initial data is expected in
the first quarter of 2026.
Earlier Stage Programs
We also have five programs in pre-clinical development. Three programs are currently in IND-enabling studies and received grant/partner funding
to support their development: OPGx-RHO is being co-funded by the Foundation Fighting Blindness (“FFB”) and the National Institutes of Health (“NIH”); OPGx-RDH12 is being co-funded by the Global RDH12 Alliance; and OPGx-MERTK is being co-funded
by the Retinal Degeneration Fund (“RDF”) of the FFB. Our pre-IND programs include OPGx-NMNAT1 and OPGxCNGB1.
Phentolamine Ophthalmic Solution 0.75% (PS)
Our pipeline also includes Phentolamine Ophthalmic Solution 0.75% (PS), a relatively non-selective alpha-1 and alpha-2 adrenergic antagonist
designed to reduce pupil size, administered as an eye drop. It aims to work by uniquely blocking the alpha-1 receptors found on the radial iris dilator muscles, which are activated by the alpha-1 adrenergic receptors. PS is designed to reduce
pupil diameter through a sympatholytic mechanism of action that avoids engaging the ciliary muscle, potentially reducing risks such as retinal tears or detachment associated with older parasympathomimetic agents. PS is targeting three
different indications.
In November 2022, we entered into a license and collaboration agreement (as amended, the “Viatris License Agreement”) with Viatris, Inc. (“Viatris”), pursuant to which we granted Viatris an exclusive license to develop, manufacture, import, export and commercialize PS for treating (a) reversal of pharmacologically-induced mydriasis, (b) decreased vision under mesopic (low) light conditions after keratorefractive surgery, and (c) presbyopia; and (ii)
PS and low dose pilocarpine for treating presbyopia (together, the “PS Products”) worldwide except for certain countries and jurisdictions in Asia. For more information on the Viatris License Agreement, please refer to Note 11 – License and
Collaboration Agreements and Other Funding Agreements included in Part I, Item 1– Financial Statements and Supplementary Data of this Report.
RYZUMVI® (phentolamine ophthalmic solution) 0.75%: PS was approved by
the FDA for the treatment for pharmacologically-induced mydriasis under the brand name RYZUMVI® in September 2023, which triggered a $10
million milestone payment under the Viatris License Agreement. RYZUMVI was commercialized by Viatris in April 2024.
Presbyopia: In June 2025, we announced positive
results from VEGA-3, our second pivotal Phase 3 trial evaluating PS for the treatment of presbyopia, an ophthalmic disorder that involves the progressive loss of ability to focus on close objects that results in blurred near vision, difficulty
seeing in dim light, and eye strain. VEGA-3 met its primary endpoint, with a statistically significant 27.2% of participants treated with PS achieving a ≥15-letter improvement in binocular distance-corrected near visual acuity (DCNVA), with
less than a 5-letter loss in binocular best-corrected distance visual acuity (BCDVA) at 12 hours post-dose on Day 8, compared to 11.5% of patients on placebo (p<0.0001). The trial also met key secondary efficacy endpoints, reinforcing the
benefit observed. We intend to file a Supplemental New Drug Application (“sNDA”) for the treatment of presbyopia with PS in the fourth quarter of 2025.
Mesopic, Low-Contrast Conditions: We are conducting our second Phase 3 trial, known as LYNX-3, to treat significant, chronic night driving impairment in
keratorefractive patients with reduced mesopic vision. The program is being conducted under a Special Protocol Assessment (“SPA”) and has received Fast Track Designation from the FDA. The first Phase 3 trial, LYNX-2, met its primary endpoint of a
gain of three lines (or 15 letters) or more of distance vision improvement on a low contrast chart in low light conditions after 15 days of dosing. In the study, 17.3% of participants treated with PS achieved a ≥15-letter Early Treatment Diabetic
Retinopathy Study (ETDRS) (≥ 3-line) improvement in Mesopic Low Contrast Distance Visual Acuity (mLCVA) at Day 15, compared to 9.2% in the placebo group (p<0.05). We are currently enrolling participants in LYNX-3, with results expected in
2026.
APX3330
APX3330 is a selective small molecule that is designed to act on the dual-functioning Apurinic/Apyrimidinic Endonuclease 1/Redox Effector Factor-1 (APE1/Ref-1) protein, referred to as Ref-1. We
completed a Phase 2 clinical study of APX3330 in diabetic retinopathy and reached FDA agreement under a SPA for a Phase 3 program. We are currently seeking a strategic partner to advance the clinical development of this diabetic retinopathy
program and redirecting existing resources toward the acquired gene therapy programs.
Recent Developments
November 2025 Registered Direct Offering
On November 5, 2025, we entered into a securities
purchase agreement to sell securities in a registered direct offering for gross proceeds of approximately $23.0 million, before deducting offering expenses (the “November 2025 Registered Direct Offering”). The offering closed on November 7,
2025. See Historical Capital
Resources section below for further detail.
August 2025 Private Placement
On August 25, 2025, we entered into subscription agreements pursuant to which we agreed to issue and sell in a private placement (the “August 2025 Private Placement”) to certain
investors an aggregate of 3,138,338 shares of our common stock. The aggregate gross proceeds from the August 2025 Private Placement were $3.5 million. The August 2025 Private Placement closed on August 25, 2025. See Historical Capital Resources section below for further detail.
Global RDH12 Alliance Agreement
On July 22, 2025, the Company, together with its wholly owned
subsidiary, OpusTX, LLC (collectively, “Opus”), entered into a funding and license agreement (the “RDH12 Agreement”) with Eyes on the Future (“EOTF”), and RDH12 Fund for Sight (the “Fund,” and together with EOTF, the “Funding Parties”),
relating to Opus’ program to develop gene therapies that treat patients with inherited retinal degeneration associated with mutations in the RDH12 gene (the “RDH12 Program”). The RDH12 Agreement provides for funding by the Funding Parties of
up to $1.6 million to support the development of the RDH12 Program. For more information on the terms of the RDH12 Agreement, see Note 11 — License and
Collaboration Revenue and Other Funding Agreements in Part I, Item 1 – Financial Statements and Supplementary Data of this Report.
RDF Agreement
On June 13, 2025, we entered into a funding agreement (the
“RDF Agreement”) with the RDF, whose sole member is the FFB, a significant stockholder of the Company, relating to our program to develop gene therapies to treat patients impacted by retinitis pigmentosa caused by pathogenic variants in
the Mer proto-oncogene tyrosine kinase (MERTK) gene (the “MERTK Program”). The RDF Agreement provides for nondilutive funding by RDF of up to $2.0 million to support the development of the MERTK Program, $1.0 million of which was
disbursed to us in June 2025 and up to $1.0 million of which may be disbursed to us upon achievement of a specified development milestone subject to RDF’s receipt of eligible funds. For more information on the terms and related
obligations under the RDF Agreement, see Note 6 — Related Party Transactions in Part I, Item 1 – Financial Statements and Supplementary Data of this
Report.
Letter Agreement and Strategic Partnership—FFB
On May 27, 2025 a binding letter of agreement (“2025 Letter Agreement”) between the Company and FFB was executed that superseded and canceled the previous binding letter agreement between the
parties, executed on August 22, 2022.
Under the 2025 Letter Agreement, we will collaborate with FFB and the Jaeb Center for Health Research on portions of a study involving
individuals with retinal dystrophies associated with mutations in the RDH12 or BEST1 genes. For more information on the terms of the 2025 Letter Agreement and related payments thereunder, see Note 6 — Related Party Transactions in
Part I, Item 1 – Financial Statements and Supplementary Data of this Report.
Regenerative Medicine Advanced Therapy (RMAT) designation to OPGx-LCA5
On May 6, 2025, we announced that the FDA has granted RMAT designation to OPGx-LCA5, our investigational gene therapy for
the treatment of LCA due to genetic variations in the LCA5 gene. The RMAT designation for OPGx-LCA5 is based on early clinical evidence from our ongoing Phase 1/2 open-label, which is evaluating the safety and potential efficacy of
OPGx-LCA5 in patients with severe vision loss due to confirmed mutations in the LCA5 gene. The RMAT designation program offers the potential for expedited development and review of regenerative medicine therapies that demonstrate the
potential to address serious or life-threatening diseases based on preliminary clinical evidence. The designation provides sponsors with early interactions with the FDA, guidance on efficient development and manufacturing, and the
opportunity to discuss surrogate endpoints to support accelerated approval.
Conversion of Series A preferred stock to Common Stock
During the 2025 Annual Meeting, our stockholders voted to approve the conversion of each share of Series A preferred stock into 1,000 shares of common stock. Subsequently, on
May 5, 2025, all shares of Series A preferred stock were converted into 14,145,374 shares of common stock.
March 2025 Offering and Private Placement
On March 21, 2025, we entered into an underwriting agreement with Craig-Hallum Capital Group, LLC, as the sole underwriter (the “March 2025 Offering”). Under the March 2025
Offering and the March 2025 Private Placement (as defined below), we agreed to issue and sell common stock and warrants to purchase up to 13,396,207 shares of common stock, 8,832,895 pre-funded warrants to purchase common stock and 22,239,102
warrants to purchase common stock. The aggregate proceeds received from the March 2025 Offering and March 2025 Private Placement was 21.5 million. See Historical Capital Resources section below for
further detail.
Strategic Outlook
We intend to advance our current active pipeline and may explore opportunities to out-license from our portfolio or in-license other drug candidates. To date, our primary
activities have been conducting research and development activities, performing business and financial planning, recruiting personnel and raising capital. We have one product, RYZUMVI, approved for sale that is generating royalties based on
sales by Viatris, and we do not expect to consistently generate significant revenues, other than license and collaborations revenue, unless and until the FDA or other regulatory authorities approve, and we successfully commercialize, LCA5,
BEST1, other internally-developed assets or PS for other indications. Until such time, if ever, as we can consistently generate substantial product revenue, we expect to finance our cash needs through a combination of equity, debt and
alternative financings as well as through collaborations, strategic alliances and licensing arrangements.
Through September 30, 2025, we have funded our operations primarily through equity financings, the issuance of convertible notes in private placements, and license fee and
milestone payments in connection with the Viatris License Agreement.
Our net loss was $17.5 million and $33.1 million for the three and nine months ended September 30, 2025, respectively, as
compared to a net loss of $7.5 million and $22.4 million for the three and nine months ended September 30, 2024, respectively. As of September 30, 2025, we had an accumulated deficit of $172.1 million. We anticipate that our expenses will
continue to increase as we:
| • |
continue clinical trials for LCA5, BEST1, PS and for any other product candidate in our future pipeline;
|
| • |
continue nonclinical studies for our pipeline of gene therapies;
|
| • |
develop additional product candidates that we identify, in-license or acquire;
|
| • |
seek regulatory approvals for any product candidates that successfully complete clinical trials;
|
| • |
contract to manufacture our product candidates;
|
| • |
maintain, expand and protect our intellectual property portfolio;
|
| • |
hire additional staff, including clinical, scientific, operational and financial personnel, to execute our business plan;
|
| • |
add operational, financial and management information systems and personnel to support our product development and potential future commercialization efforts;
|
| • |
continue to operate as a public company; and
|
| • |
establish on our own or with partners, a sales, marketing and distribution infrastructure to commercialize any products for which we may obtain regulatory approval.
|
Our net loss will likely continue to fluctuate significantly from quarter to quarter and year to year, depending on the timing of our nonclinical studies, clinical trials,
expenditures on other research and development activities (and reimbursement thereof), and from potential milestone payments received from and revenue earned under the Viatris License Agreement or any other license and collaboration agreements
that we enter into.
Financial Operations Overview
License and Collaborations Revenue
License and collaborations revenue to date was derived from a one-time, non-refundable payment related to a license transfer, an additional milestone
payment and reimbursement of expenses earned under the Viatris License Agreement, and to a much lesser degree, from license agreements with BioSense Global LLC (“BioSense”) and Processa Pharmaceuticals, Inc. (“Processa”). We anticipate that we
will recognize revenue as we earn reimbursement for research and development services in connection with the Viatris License Agreement and we may earn additional revenues from potential milestone and royalty payments from the agreements with
Viatris or from other license agreements entered into the future; however, the attainment of milestones or level of sales required to earn significant royalty payments is highly uncertain for the reasons explained below. Until further notice,
we will report earned RYZUMVI royalties as a component of license and collaboration revenue listed in the condensed consolidated statements of comprehensive loss.
To date, outside of the license and collaborations revenue referenced above, we do not expect to generate significant
revenue unless or until RYZUMVI sales become material, or regulatory approval is obtained, and commercialization begins for LCA5, BEST1, other internally-developed assets or PS for additional indications. If we fail to complete the
development of LCA5, BEST1, PS, or any other product candidate we may pursue or fail to obtain regulatory approval, our ability to generate significant revenue will be compromised.
Operating Expenses
The Company’s operating expenses are classified into two categories: general and administrative and research and development.
General and Administrative Expenses
General and administrative expenses consist primarily of personnel-related costs, including salaries, benefits and stock-based compensation costs, for personnel in functions not
directly associated with research and development activities. Other significant costs include insurance coverage for directors and officers and other property and liability exposures, legal fees relating to intellectual property and corporate
matters, business development costs, professional fees for accounting and tax services, other services provided by business consultants and legal settlements.
Research and Development Expenses
To date, our research and development expenses have related primarily to the clinical stage development of our IRD programs, including LCA5 and BEST1, PS, and APX3330. Research
and development expenses consist of costs incurred in performing research and development activities, including compensation, benefits and stock-based compensation costs for research and development employees and costs for consultants, costs
associated with nonclinical studies and clinical trials, regulatory activities, manufacturing activities to support clinical activities, license fees, nonlegal patent costs, fees paid to external service providers that conduct certain research
and development, and an allocation of overhead expenses. We do not expect to incur meaningful research and development expenses in the future for APX3330, and we announced plans to seek a partner for the program to advance development.
Pursuant to the Viatris License Agreement, our research and development expenses related to the development of PS to date have been fully reimbursed by Viatris.
We expect that LCA5, BEST1, PS and other internally-developed assets will have higher development costs during the later stages of clinical development, as compared to costs
incurred during their earlier stages of development, primarily due to the increased size and duration of the later-stage clinical trials and associated nonclinical studies. We expect our research and development expenses to increase over the
next several years. However, it is difficult for us to determine with certainty the duration, costs and timing to complete our current or future nonclinical programs and clinical trials of LCA5, BEST1, PS and other internally-developed assets.
Fair value change in warrant and other derivative liabilities
The fair value change in warrant and other derivative liabilities consists of the fair value changes associated with the March 2025 Warrants and March 2025 Private Placement
Warrants are described further, below.
Financing costs
Financing costs consist of issuance costs attributed to our March 2025 Warrants and March 2025 Private Placement Warrants described below. There were no issuance costs
attributed to the equity line financing with Lincoln Park during the periods presented.
Interest Expense
Interest expenses consists of interest cost during the period ended September 30, 2025 incurred under the RDF Agreement, which is
accounted for as debt under ASC 470, Debt. ASC 470
Other Income, net
Other income, net includes interest earned from cash and cash equivalent investments, realized and unrealized gains (losses)
from equity investments, reimbursements in connection with grants and other sources when they occur. In addition, this line item includes payments made by the Company in connection with the Contingent Value Rights Agreement (the “CVR
Agreement”) discussed further below with former shareholders of Rexahn.
Provision for Income Taxes
Provision for income taxes consists of federal and state income taxes in the United States, as well as deferred income taxes and changes in related valuation allowance
reflecting the net tax effects of temporary differences between the carrying amounts of assets and liabilities for financial reporting purposes and the amounts used for income tax purposes. Currently, a full valuation allowance has been
provided on the net deferred tax assets as of September 30, 2025 and December 31, 2024 given the uncertainty of future taxable income and other related factors impacting the realizability or our remaining net deferred tax assets.
Results of Operations
Comparison of Three Months Ended September 30, 2025 and 2024
The following table summarizes Opus’s operating results for the periods indicated (in thousands):
|
For the Three Months Ended
|
||||||||||||
|
September 30,
|
||||||||||||
|
2025
|
2024
|
Change
|
||||||||||
|
License and collaborations revenue
|
$
|
3,079
|
$
|
3,867
|
$
|
(788
|
)
|
|||||
|
Operating expenses:
|
||||||||||||
|
General and administrative
|
4,981
|
2,894
|
2,087
|
|||||||||
|
Research and development
|
6,409
|
8,982
|
(2,573
|
)
|
||||||||
|
Total operating expenses
|
11,390
|
11,876
|
(486
|
)
|
||||||||
|
Loss from operations
|
(8,311
|
)
|
(8,009
|
)
|
(302
|
)
|
||||||
|
Fair value change in warrant and other derivative liabilities
|
(9,525
|
)
|
—
|
(9,525
|
)
|
|||||||
|
Interest expense
|
(68
|
)
|
—
|
(68
|
)
|
|||||||
|
Other income, net
|
450
|
483
|
(33
|
)
|
||||||||
|
Loss before income taxes
|
(17,454
|
)
|
(7,526
|
)
|
(9,928
|
)
|
||||||
|
Provision for income taxes
|
—
|
—
|
—
|
|||||||||
|
Net loss
|
$
|
(17,454
|
)
|
$
|
(7,526
|
)
|
$
|
(9,928
|
)
|
|||
License and Collaborations Revenue
License and collaborations revenue was $3.1 million and $3.9 million for the three months ended September 30, 2025 and 2024, respectively. Revenue during both quarterly
periods was derived from the Viatris License Agreement, largely for the reimbursement of research and development services. To a much lesser extent, revenue includes an earned royalty payment from the sales of RYZUMVI, indicated for the treatment
of pharmacologically-induced mydriasis produced by adrenergic agonists (e.g., phenylephrine) or parasympatholytic (e.g., tropicamide) agents by our commercial partner. The $0.8 million decrease in license and collaborations revenue during the
current three month period ended September 30, 2025 compared to the corresponding prior year period was due to a decrease in PS research and development services.
General and Administrative
General and administrative expenses for the three months ended September 30, 2025 were $5.0 million compared to $2.9 million for the three months ended September 30, 2024.
The increase of $2.1 million was primarily attributable to public company related costs of $0.4 million, general legal costs of $0.5 million, patent costs of $0.1 million, payroll related costs of $0.6 million, professional service fees of $0.2
million and other operating expenses of $0.3 million on a net basis when compared to the corresponding prior year period. General and administrative expenses included $0.6 million and $0.5 million in stock-based compensation expense during the
three months ended September 30, 2025 and 2024, respectively.
Research and Development
The following table illustrates the components of our research and development expenses for the periods presented (in thousands):
|
For the Three Months Ended
|
||||||||||||
|
September 30,
|
||||||||||||
|
2025
|
2024
|
Change
|
||||||||||
|
External costs:
|
||||||||||||
|
IRD programs
|
$
|
2,575
|
$
|
—
|
$
|
2,575
|
||||||
|
Phentolamine Ophthalmic Solution 0.75% (“PS”)
|
2,421
|
3,561
|
(1,140
|
)
|
||||||||
|
APX 3330
|
203
|
4,272
|
(4,069
|
)
|
||||||||
|
Unallocated
|
102
|
90
|
12
|
|||||||||
|
Total external cost
|
5,301
|
7,923
|
(2,622
|
)
|
||||||||
|
Internal costs:
|
||||||||||||
|
Employee related expenses
|
1,016
|
993
|
23
|
|||||||||
|
Facilities, supplies and other
|
92
|
66
|
26
|
|||||||||
|
Total internal costs
|
1,108
|
1,059
|
49
|
|||||||||
|
Total research and development expenses
|
$
|
6,409
|
$
|
8,982
|
$
|
(2,573
|
)
|
|||||
A greater percentage of research and development expense incurred has been allocated to IRD programs for the three months ended September 30, 2025 as compared to the three
months ended September 30, 2024 as the Company continues to focus on developing the IRD gene therapy programs acquired in connection with the Opus Acquisition. Conversely, a lesser percentage of research and development expense incurred has been
allocated to APX 3330 for the three months ended September 30, 2025 as compared to the three months ended September 30, 2024, as we do not expect to incur meaningful research and development expenses in the future for APX3330, and we are
currently seeking a strategic partner to advance the clinical development of this program.
Research and development expenses for the three months ended
September 30, 2025 were $6.4 million compared to $9.0 million for the three months ended September 30, 2024. The $2.6 million decrease was primarily attributable to lower APX 3330 expense of $4.1 million, including lower manufacturing
costs of $1.9 million, lower toxicology costs of $1.5 million, and lower regulatory costs of $0.2 million; and by lower PS expense of $1.1 million, driven by lower clinical research costs of $0.8 million. These decreases are partially offset by
increased IRD program expense of $2.6 million, including higher toxicology costs of $1.4 million and higher IRD clinical research costs of $0.8 million when compared to the corresponding prior year period. Pursuant to the Viatris License
Agreement, our research and development expenses related to the development of the PS Products have been fully reimbursed by Viatris to date. Research and development expenses included $0.2 million in stock-based compensation expense during
each of the three months ended September 30, 2025 and 2024.
Fair value change in warrant and other derivative liabilities
The fair value change in warrant and other derivative liabilities was attributed to the March 2025 Warrants and March 2025 Private Placement Warrants, as described further
below, and was $9.5 million for the three months ended September 30, 2025. The fair value changes are attributed to the fluctuations in our common stock fair value and underlying changes in volatility, expected term and interest rates.
Interest expense
During the three months ended September 30, 2025, Opus had interest expense in connection with the RDF Agreement in the amount of $0.1 million. There was no interest expense
during the comparable prior year period.
Other Income, net
During the three months ended September 30, 2025, Opus had other income, net of $0.4 million which primarily consisted of interest income in the amount of $0.3 million in
connection with our cash and cash equivalents on-hand and to a lesser extent grant revenue in the amount of $0.1 million.
During the three months ended September 30, 2024, Opus had other income, net of $0.5 million related primarily to interest income in connection with our cash and cash equivalents
on-hand.
Comparison of Nine Months Ended September 30, 2025 and 2024
The following table summarizes Opus’s operating results for the periods indicated (in thousands):
|
For the Nine Months Ended
|
||||||||||||
|
September 30,
|
||||||||||||
|
2025
|
2024
|
Change
|
||||||||||
|
License and collaborations revenue
|
$
|
10,331
|
$
|
6,690
|
$
|
3,641
|
||||||
|
Operating expenses:
|
||||||||||||
|
General and administrative
|
17,093
|
10,918
|
6,175
|
|||||||||
|
Research and development
|
20,384
|
19,817
|
567
|
|||||||||
|
Total operating expenses
|
37,477
|
30,735
|
6,742
|
|||||||||
|
Loss from operations
|
(27,146
|
)
|
(24,045
|
)
|
(3,101
|
)
|
||||||
|
Fair value change in warrant and other derivative liabilities
|
(5,803
|
)
|
—
|
(5,803
|
)
|
|||||||
|
Financing costs
|
(1,337
|
)
|
—
|
(1,337
|
)
|
|||||||
|
Interest expense
|
(68
|
)
|
—
|
(68
|
)
|
|||||||
|
Other income, net
|
1,286
|
1,648
|
(362
|
)
|
||||||||
|
Loss before income taxes
|
(33,068
|
)
|
(22,397
|
)
|
(10,671
|
)
|
||||||
|
Provision for income taxes
|
—
|
—
|
—
|
|||||||||
|
Net loss
|
$
|
(33,068
|
)
|
$
|
(22,397
|
)
|
$
|
(10,671
|
)
|
|||
License and Collaborations Revenue
License and collaborations revenue was $10.3 million and $6.7 million for the nine months ended September 30, 2025 and 2024, respectively. Revenue during the nine month
periods was derived primarily from the reimbursement of research and development services under the Viatris License Agreement. The $3.6 million increase in license and collaborations revenue during the current nine month period ended September
30, 2025 when compared to the corresponding prior year period was due to an increase in PS research and development services.
General and Administrative
General and administrative expenses for the nine months ended September 30, 2025 were $17.1 million compared to $10.9 million for the nine months ended September 30, 2024.
The increase of $6.2 million was primarily attributable to public company related costs of $2.0 million, legal fees of $1.7 million, patent fees of $0.7 million, professional service costs of $0.5 million, payroll related costs of $0.9 million
and business development activity and other operating costs on a net basis of $0.4 million when compared to the corresponding prior year period. General and administrative expenses totaled $1.8 million and $1.9 million in stock-based compensation
expense during the nine months ended September 30, 2025 and 2024, respectively.
Research and Development
The following table illustrates the components of our research and development expenses for the periods presented (in thousands):
|
For the Nine Months Ended
|
||||||||||||
|
September 30,
|
||||||||||||
|
2025
|
2024
|
Change
|
||||||||||
|
External costs:
|
||||||||||||
|
IRD programs
|
$
|
6,624
|
$
|
—
|
$
|
6,624
|
||||||
|
Phentolamine Ophthalmic Solution 0.75% (“PS”)
|
8,914
|
5,678
|
3,236
|
|||||||||
|
APX3330
|
325
|
10,959
|
(10,634
|
)
|
||||||||
|
Unallocated
|
340
|
238
|
102
|
|||||||||
|
Total external cost
|
16,203
|
16,875
|
(672
|
)
|
||||||||
|
Internal costs:
|
||||||||||||
|
Employee related expenses
|
3,880
|
2,776
|
1,104
|
|||||||||
|
Facilities, supplies and other
|
301
|
166
|
135
|
|||||||||
|
Total internal costs
|
4,181
|
2,942
|
1,239
|
|||||||||
|
Total research and development expenses
|
$
|
20,384
|
$
|
19,817
|
$
|
567
|
||||||
A greater percentage of research and development expense incurred has been allocated to IRD programs for the nine months ended September 30, 2025 as compared to the nine
months ended September 30, 2024 as the Company continues to focus on developing the IRD gene therapy programs acquired in connection with the Opus Acquisition. Conversely, a lesser percentage of research and development expense incurred has been
allocated to APX 3330 for the nine months ended September 30, 2025 as compared to the nine months ended September 30, 2024, as we do not expect to incur meaningful research and development expenses in the future for APX3330, and we are currently
seeking a strategic partner to advance the clinical development of this program.
Research and development expenses for the nine months ended September 30, 2025 were $20.4 million compared to $19.8 million for the nine months ended September 30, 2024. The
$0.6 million increase was primarily attributable to increased IRD program expense of $6.6 million, including higher toxicology costs of $4.2 million, higher IRD clinical research costs of $1.7 million, and higher CMC costs of $0.4 million; higher
PS expense of $3.2 million, driven by clinical research costs related to the PS Vega-3 and PS Lynx-2 trials; and higher research and development employee related compensation expense of $1.1 million. These increases are partially offset by
decreased APX3330 expense of $10.6 million, including lower manufacturing costs of $4.6 million, lower toxicology costs of $3.6 million, lower clinical costs of $1.5 million, and lower regulatory costs of $0.8 million when compared to the
corresponding prior year period. Pursuant to the Viatris License Agreement, our research and development expenses related to the development of PS are fully reimbursed by Viatris. Research and development expenses also included $0.8 million and
$0.7 million in stock-based compensation expense during the nine months ended September 30, 2025 and 2024, respectively.
Fair value change in warrant and other derivative liabilities
The fair value change in warrant and other derivative liabilities was attributed largely to the March 2025 Warrants and March 2025 Private Placement Warrants, as described
further below. The fair value change in the March 2025 Warrants and March 2025 Private Placement Warrants was $5.8 million for the nine months ended September 30, 2025. The fair value changes are attributed to the fluctuations in our common
stock fair value and underlying changes in volatility, expected term and interest rates. During the comparable prior year period, the fair value change in our other derivative liabilities was de minimis.
Financing costs
Financing costs for the nine months ended September 30, 2025 of $1.3 million was comprised of issuance costs attributed to the March 2025 Warrants and March 2025 Private
Placement Warrants. We did not have any financing costs during the nine months ended September 30, 2024.
Interest expense
During the nine months ended September 30, 2025, Opus had interest expense in connection with the RDF Agreement in the amount of $0.1 million. There was no interest expense
during the comparable prior year period.
Other Income, net
During the nine months ended September 30, 2025, Opus had other income, net of $1.3 million which primarily consisted of interest income in the amount of $1.0 million in
connection with our cash and cash equivalents on-hand and to a lesser extent grant revenue in the amount of $0.3 million.
During the nine months ended September 30, 2024, Opus had other income, net of $1.6 million related primarily to interest income in connection with our cash and cash
equivalents on-hand.
Liquidity and Capital Resources
Capital Resources
As of September 30, 2025, our principal sources of liquidity consisted of cash and cash equivalents of $30.8 million. We believe
that our cash on hand as of September 30, 2025 will be sufficient to fund our operations for at least twelve months beyond the date of this filing. As of September 30, 2025, our cash and cash equivalents were invested primarily in cash deposits
and cash equivalent investments at four large financial institutions.
Historical Capital Resources
Our primary source of cash to fund our operations has been various equity offerings in the amount of $94.9 million and the issuance of convertible notes in the amount of $8.5
million, inclusive of the promissory notes exchanged for Opus convertible notes. In addition, we received a one-time non-refundable cash payment of $35.0 million during the fourth quarter of 2023, a $10.0 million milestone payment during the
fourth quarter of 2023, and have received reimbursement for costs related to development since the fourth quarter of 2022, all in connection with the Viatris License Agreement. Lastly, we received funding in the amount of $1.3 million from the
RDF Agreement and various research and development grants.
November 2025 Registered Direct Offering
On November 5, 2025, we entered into a securities purchase agreement to sell securities in the November 2025 Registered Direct
Offering for gross proceeds of approximately $23.0 million, before deducting offering expenses. The financing was led by Perceptive Advisors and Balyasny Asset Management, with participation by new and existing institutional investors,
including Nantahala Capital. We intend to use the net proceeds to advance our LCA5 and BEST-1 gene therapy programs, as well as for working capital and general corporate purposes.
In the November 2025 Registered Direct Offering, we sold an aggregate of 3,827,751 shares of common stock at a price of $2.09
per share and, in lieu of common stock to certain investors, pre-funded warrants to purchase up to an aggregate of 7,177,033 shares of common stock at a purchase price of $2.0899 per pre-funded warrant. Each pre-funded warrant has an exercise
price of $0.0001 per share of common stock, will be immediately exercisable subject to certain conditions set forth in each pre-funded warrant, and will not expire. The offering closed on November 7, 2025.
August 2025 Private Placement
On August 25, 2025, we entered into subscription agreements pursuant to which we agreed to issue and sell in the August 2025
Private Placement to certain investors an aggregate of 3,138,338 shares of our common stock. The aggregate gross proceeds from the August 2025 Private Placement were approximately $3.5 million. The August 2025 Private Placement closed on
August 25, 2025.
We intend to use the net proceeds of the August 2025 Private Placement to expedite manufacturing process development, including scale-up of clinical and commercial production
and testing, to ensure sufficient supply of cGMP material for its gene therapy candidates, OPGx-LCA5 and OPGx-BEST1. No underwriting discounts or commissions were paid with respect to the August 2025 Private Placement.
The August 2025 Private Placement was led by Cam Gallagher, Chair of our board of directors (the “Board”), with an investment of $1.0 million, along with participation by Sean
Ainsworth, the lead independent director of the Board, and other investors.
March 2025 Financings
On March 21, 2025, we entered into an underwriting agreement with Craig-Hallum Capital Group, LLC, as the sole underwriter.
Pursuant to the underwriting agreement, we agreed to issue and sell, in an underwritten public offering, 12,219,736 shares of common stock and warrants to purchase up to 21,052,631 shares of common stock (the “March 2025 Warrants”). Each
share of common stock was sold together with one March 2025 Warrant to purchase one share of common stock, at a price to the public of $0.95 per share and related March 2025 Warrant. We also agreed to issue 8,832,895 pre-funded warrants
(“Pre-Funded Warrants”) at a price to the public of $0.9499 per Pre-Funded Warrant.
Also on March 21, 2025, we entered into a subscription
agreement (the “Subscription Agreement”) with each of Dr. George Magrath, the Company’s Chief Executive Officer, and Cam Gallagher, the chairman our Board. Pursuant to the Subscription Agreement, the Company agreed to issue and
sell, in a private offering (the “March 2025 Private Placement”), a total of 392,157 shares of common stock to Mr. Magrath and 784,314 shares of common stock to Mr. Gallagher, as well as 392,157 warrants to purchase shares of common stock
to Mr. Magrath and 784,314 warrants to purchase shares of common stock to Mr. Gallagher (“March 2025 Private Placement Warrants”). Each March 2025 Private Placement Warrant has
an initial exercise price of $1.15, expires on the five-year anniversary of the original issuance date and may be called by the Company 30 days following the release of the Company’s OPGx-BEST1 DUO-1001 Cohort 1 data upon achievement of a
volume weighted average price of our common stock for 30 consecutive trading days of over $1.725 per share and the trading average daily volume for such 30 day period exceeds $150,000 per trading day.
The combined gross proceeds from the March 2025 Offering and the March 2025 Private Placement, which closed on March 24, 2025, were approximately $21.5 million, before
deducting underwriting discounts and commissions and offering expenses payable by us.
The March 2025 Offering (including the shares of common stock issuable from time to time upon exercise of the March 2025
Warrants and the Pre-Funded Warrants) was made pursuant to our Registration Statement on Form S-3 (File No. 333-276462) filed with the SEC on January 10, 2024, including the prospectus dated January 23, 2024 contained therein, as the same
has been supplemented.
March 2025 Warrants
The March 2025 Warrants have an initial exercise price equal to $0.95 per share of common stock and are exercisable for five
years from the date of issuance. The exercise prices and numbers of shares of common stock issuable upon exercise are subject to appropriate adjustment in the event of stock dividends, stock splits, reorganizations or similar events affecting
the common stock and also upon any distributions of assets, including cash, stock or other property to our stockholders. A holder may not exercise the March 2025 Warrant if, after giving effect to such exercise, the holder (together with its
affiliates) would beneficially own (as determined in accordance with the terms of the March 2025 Warrants) more than 4.99% (or, at the election of the holder, 9.99%) of the outstanding common stock immediately after giving effect to the
exercise.
The March 2025 Warrants are callable by us in certain circumstances. Subject to certain exceptions, in the event that the March
2025 Warrants are outstanding, if, after the closing date, March 24, 2025 (the “Closing Date”), (i) we have announced OPGx-BEST1 DUO-1001 Cohort 1 data, (ii) the volume weighted average price of the common stock for 30 consecutive trading days
(“Warrant Measurement Period”), which 30 consecutive trading day period shall not have commenced until after the initial exercise date) exceeds $1.425 (subject to adjustment), (iii) the trading average daily volume for such Warrant Measurement
Period exceeds $150,000 per trading day and (iv) the March 2025 Warrant holder is not in possession of any information that constitutes or might constitute material non-public information which was provided by the Company, its subsidiaries or
any of its officers, directors, employees, agents or affiliates, then the Company may, within one trading day of the end of such Warrant Measurement Period, upon notice, call for cancellation of all or any portion of the March 2025 Warrants for
which a notice of exercise has not yet been delivered for consideration equal to $0.001 per March 2025 Warrant share.
In the event of a fundamental transaction, as defined in the Form of Warrant, the holders of the March 2025 Warrants will be
entitled to receive upon exercise the kind and amount of securities, cash or other property that the holders would have received had they exercised immediately prior to such fundamental transaction. Additionally, as more fully described in the
Form of Warrant, in the event of certain fundamental transactions, the holders of the March 2025 Warrants will be entitled to receive consideration in an amount equal to the Black Scholes Value of the remaining unexercised portion of the March
2025 Warrants on the date of consummation of such fundamental transaction.
March 2025 Private Placement Warrants
The March 2025 Private Placement Warrants have an initial exercise price equal to $1.15 per share of common stock and are
exercisable for five years from the date of issuance. The March 2025 Private Placement Warrants are callable by us in certain circumstances. Subject to certain exceptions, in the event that the March 2025 Private Placement Warrants are
outstanding, if, after the Closing Date, (i) the Company announces OPGx-BEST1 DUO-1001 Cohort 1 data, (ii) the volume weighted average price of the common stock for 30 consecutive trading days (“Private Placement Measurement Period”), which
30 consecutive trading day period shall not have commenced until after the initial exercise date) exceeds $1.725 (subject to adjustment), (iii) the trading average daily volume for such Private Placement Measurement Period exceeds $150,000
per trading day and (iv) the March 2025 Private Placement Warrant holder is not in possession of any information that constitutes or might constitute material non-public information which was provided by the Company, its subsidiaries or any
of its officers, directors, employees, agents or affiliates, then the Company may, within one trading day of the end of such Private Placement Measurement Period, upon notice, call for cancellation of all or any portion of the March 2025
Private Placement Warrants for which a notice of exercise has not yet been delivered for consideration equal to $0.001 per March 2025 Private Placement Warrant share. All other terms under the March 2025 Private Placement Warrants are
identical to the terms of the March 2025 Warrants discussed above.
Pre-Funded Warrants
The Pre-Funded Warrants have an exercise price of $0.0001 per share of common stock and are immediately exercisable and are exercisable at any time until exercised in full. The
exercise prices and numbers of shares of common stock issuable upon exercise are subject to appropriate adjustment in the event of stock dividends, stock splits, reorganizations or similar events affecting the common stock. A holder may not
exercise the Pre-Funded Warrant if, after giving effect to such exercise, the holder (together with its affiliates) would beneficially own (as determined in accordance with the terms of the Pre-Funded Warrants) more than 4.99% (or, at the
election of the holder, 9.99%) of the outstanding common stock immediately after giving effect to the exercise. In the event of a fundamental transaction, as defined in the Form of Pre-Funded Warrant, the holders of the Pre-Funded Warrants will
be entitled to receive upon exercise of the Pre-Funded Warrants the kind and amount of securities, cash or other property that the holders would have received had they exercised the Pre-Funded Warrants immediately prior to such fundamental
transaction.
At-The-Market Program
On January 13, 2025, the Company filed a prospectus supplement with the SEC in connection with the establishment of an
at-the-market equity offering program (the “ATM program”) for the offer and sale, from time to time, of shares of its common stock, having an aggregate offering price of up to $40.0 million. On the same date, the Company also entered into a
sales agreement (the “Sales Agreement”) with Leerink Partners LLC (“Leerink”), pursuant to which the Company may offer and sell shares of its common stock through or to Leerink, acting as sales agent, under the ATM program. In connection with
the entry into the Sales Agreement, the Company terminated its prior ATM program established under the Capital on DemandTM Sales Agreement, dated March 11, 2021, between the Company and JonesTrading Institutional Services LLC.
Registered Direct Offering
On June 4, 2021, we entered into a placement agency agreement with A.G.P./Alliance Global Partners (“AGP”). Pursuant to the
terms of the placement agency agreement, AGP on June 8, 2021, sold an aggregate of 3,076,923 shares of our common stock and warrants to purchase 1,538,461 shares of our common stock (“RDO Warrants”) at an offering price of $4.875 per share
and 0.50 RDO Warrants, for gross proceeds of $15.0 million, before deducting AGP’s fees and related offering expenses in the amount of $1.1 million. The purchase agreement contains customary representations, warranties and agreements by the
Company, customary conditions to closing, indemnification obligations of the Company, other obligations of the parties and termination provisions.
The RDO Warrants have an exercise price of $6.09 per share, are exercisable upon the initial issuance date of June 8, 2021, and will expire five years following the initial
exercise date. Subject to limited exceptions, a holder of a RDO Warrant will not have the right to exercise any portion of its RDO Warrants if the holder, together with its affiliates, would beneficially own in excess of 4.99% (or, at the
election of a holder prior to the date of issuance, 9.99%) of the number of shares of common stock outstanding immediately after giving effect to such exercise; provided, however, that upon prior notice to us, the holder may increase or
decrease the beneficial ownership limitation, provided further that in no event shall the beneficial ownership limitation exceed 9.99%. As of September 30, 2024, 1,538,461 RDO Warrants were still outstanding. The offering of the securities was
made pursuant to our effective shelf registration statement on Form S-3.
Pre-Rexahn Merger Financing
Securities Purchase Agreement
On June 17, 2020, the Company, Rexahn and certain investors entered into a Securities Purchase Agreement, which was amended and
restated in its entirety on June 29, 2020 (as amended and restated, the “Securities Purchase Agreement”). Pursuant to the Securities Purchase Agreement, the investors invested a total of $21.15 million in cash, including $0.3 million invested
by directors of the Company, and one director of Rexahn, upon closing of the Rexahn Merger (the “Pre-Merger Financing”). The Pre-Merger Financing also included the issuance of Series A Warrants and Series B Warrants discussed further below.
Waiver Agreements
Effective February 3, 2021, each investor that invested in the Pre-Merger Financing (each, a “Holder”) entered into a Waiver Agreement with the Company
(collectively, the “Waiver Agreements”). Pursuant to the Waiver Agreements, the Holders and the Company agreed to waive certain rights, finalize the
exercise price and number of Series A Warrants and Series B Warrants, eliminate certain financing restrictions, extend the term of certain leak-out agreements, and, in the case of certain Holders, grant certain registration rights for the
shares underlying the warrants.
The Waiver Agreements provide for the permanent waiver of the full ratchet anti-dilution provisions, contained in the Series A Warrants (as certain of the anti-dilution
provisions had previously caused liability accounting treatment for the Series A Warrants). Upon the effective date of the Waiver Agreement, the Series A Warrants were reclassified to equity.
Pursuant to the Waiver Agreements, the number of shares underlying all of the Series B Warrants was fixed at 1,708,335 in the aggregate with respect to all Holders.
Series A Warrants
The Series A Warrants were issued on November 19, 2020 at an initial exercise price of $4.4795 per share, were immediately exercisable upon issuance and have a term of five
years from the date of issuance. The Series A Warrants are exercisable for 5,665,838 shares of common stock in the aggregate (without giving effect to any limitation on exercise contained therein). As of September 30, 2025, 5,665,838 Series A
Warrants were still outstanding.
At issuance, the Series A Warrants contained certain provisions that could have resulted in a downward adjustment of the initial exercise price and an upward
adjustment in the number of shares underlying the warrants if the Company were to have issued or sold, or made an agreement to issue or sell, any shares of
common stock for a price lower than the exercise price then in effect. Pursuant to the terms of the Waiver Agreements, these provisions are no longer in effect.
Cash Flows
The following table summarizes Opus’s cash flows for the periods indicated (in thousands):
|
For the Nine Months Ended
|
||||||||
|
September 30,
|
||||||||
|
2025
|
2024
|
|||||||
|
Net cash used in operating activities
|
$
|
(25,420
|
)
|
$
|
(18,138
|
)
|
||
|
Net cash provided by (used in) investing activities
|
—
|
—
|
||||||
|
Net cash provided by financing activities
|
25,914
|
4,269
|
||||||
|
Net increase (decrease) in cash and cash equivalents
|
$
|
494
|
$
|
(13,869
|
)
|
|||
Cash Flow from Operating Activities
For the nine months ended September 30, 2025, cash used in operating activities of $25.4 million was attributable to a net loss of $33.1 million, adjusted by a reclassification to financing
activities related to the March 2025 financings and by non-cash net operating income of approximately $9.9 million in the aggregate, and attributed to a net change cash use of approximately $2.2 million in Opus’s net operating assets and
liabilities. The non-cash expenses consisted principally of a fair value change in warrant and other derivative liabilities benefit of $5.8 million, stock-based compensation of $2.6 million, non-cash interest expense of $0.1 million, and
depreciation of less than $0.1 million. The reclassification to financing activities for issuance costs attributed to our liability classified warrants was $1.3 million. The change in operating assets and liabilities was primarily attributable to
a net decrease in our accounts payable and accrued expenses and by an increase in our prepaid expenses and other current assets, offset in part by a decrease in our accounts receivable, contract assets. All of the changes were attributed to
fluctuations in Opus’s operating expenses and collections under the normal course of business.
For the nine months ended September 30, 2024, cash used in operating activities of $18.1 million was attributable to a net loss
of $22.4 million, partially offset by $2.6 million in non-cash operating expenses and a net change cash source of $1.7 million in the Company’s net operating assets and liabilities. The non-cash expenses consisted principally of stock-based
compensation of $2.6 million. The change in operating assets and liabilities was primarily attributable to increases in our accrued expenses and to decreases in our prepaid expenses, all associated with the Company’s operating expenses under
the normal course of business. Our net sources of cash during the period were partially offset by a net increase in the Company’s accounts receivable attributed to the timing of payments from Viatris and to a net decrease in our accounts
payable due to the timing of payments under the normal course of business.
Cash Flow from Investing Activities
There were no sources or uses from investing activities during the periods presented.
Cash Flow from Financing Activities
Net cash provided by financing activities during the nine months ended September 30, 2025 was $25.9 million that consisted
principally of gross proceeds received from the March 2025 Offering and March 2025 Private Placement of $21.5 million in the aggregate, August 2025 Private Placement in the amount of $3.5 million and from the ATM in the amount of $2.1 million.
Both financings were offset by issuance costs of $2.2 million in the aggregate. Lastly, we received funding of $1.0 million in connection with the RDF Agreement.
Net cash provided by financing activities during the nine months ended September 30, 2024 was $4.3 million, consisting of net proceeds received from both the 2021 ATM and the
equity line financing in the aggregate of $4.3 million.
Liquidity and Capital Resource Requirements
As of September 30, 2025 we had cash and cash equivalents of $30.8 million. License and collaborations revenue inception to date was derived from a one-time non-refundable
payment of $35 million, a milestone payment of $10 million, reimbursement and expected reimbursement of expenses and royalties earned under the Viatris License Agreement and, to a much lesser degree, from license agreements with BioSense and
Processa in connection with the Rexahn RX-3117 drug compound, and other reimbursement from grants. We anticipate that we will recognize revenue as we earn reimbursement for research and development services in connection with the Viatris
License Agreement and we may earn additional revenues from future potential milestone and royalty payments from the agreement with Viatris, or from other license agreements entered into in the future; however, the attainment of milestones or
level of sales required to earn royalty payments is highly uncertain for the reasons explained below.
To date, outside of the license and collaborations revenue referenced above, we do not expect to generate significant revenue unless or until RYZUMVI sales become material,
or regulatory approval is obtained and commercialization begins for LCA5, BEST1, other internally-developed assets or PS for additional indications. If we fail to complete the development of LCA5, BEST1, other internally-developed assets, PS or
any other product candidate we may pursue in the future in a timely manner or fail to obtain regulatory approval for any of such product candidates, our ability to generate significant revenue would be compromised.
Through the ATM program with Leerink, we may offer and sell, from time to time at our sole discretion, to or through Leerink, acting as agent and/or principal, shares of
our common stock having an aggregate offering price of up to $40 million. As of September 30, 2025, the Company had sold an aggregate of 9,479,494 shares of common stock under the ATM programs since their inception, resulting in gross proceeds
of $28.5 million and total issuance costs of $1.4 million.
To the extent that we raise additional capital through the sale of equity or convertible debt securities, the ownership interest of our stockholders will be diluted, and the
terms of these securities may include liquidation, warrants or other preferences that adversely affect your rights as a common stockholder. Debt financing, if available, may involve agreements that include covenants limiting or restricting our
ability to take specific actions, such as incurring additional debt, making capital expenditures or declaring dividends. If we raise additional funds through future collaborations, strategic alliances or licensing arrangements with
pharmaceutical partners, we may have to relinquish valuable rights to our technologies, future revenue streams or grant licenses on terms that may not be favorable to us. If we are unable to raise additional funds through equity or debt
financings or through collaborations, strategic alliances or licensing arrangements when needed, we may be required to delay, limit, reduce or terminate our product development, future commercialization efforts, or grant rights to develop and
market our product candidates that we would otherwise prefer to develop and market ourselves.
Future Capital Requirements
Pursuant to the Viatris License Agreement, our budgeted research and development expenses related to the development of PS are fully reimbursed by Viatris. The development of
LCA5, BEST1 and other internally-developed assets is subject to numerous uncertainties, and we have based these estimates on assumptions that may prove to be substantially different than what we currently anticipate and could result in cash
resources being used sooner than what we currently expect. Additionally, the process of advancing early-stage product candidates and testing product candidates in clinical trials is costly, and the timing of progress in these clinical trials is
uncertain. Our ability to successfully transition to profitability will be dependent upon achieving a level of product sales adequate to support our cost structure. We cannot give any assurance that we will ever be profitable or generate
positive cash flow from operating activities.
Contractual Obligations and Commitments
Our contractual obligations in our Annual Report on Form 10-K for the year ended December 31, 2024, filed with the SEC on March 31, 2025, have not
materially changed since we filed that report, except as discussed below.
Facility Lease
On January 1, 2025, the Company relocated its headquarters to Durham, North Carolina. On July 1st, 2025, the Company amended the lease for its headquarters in Durham, North
Carolina for three months through September 30, 2025, and the lease is currently in place on a month-to-month basis.
The Company also leases additional laboratory space in Durham, North Carolina on a month-to-month basis. Upon the Opus Acquisition, the Company assumed a number of equipment
leases which expired in July 2025 and are now on a month-to-month basis.
Letter Agreement and Strategic Partnership—FFB
Under the 2025 Letter Agreement, we are required to make the remaining $0.3 million payment installment on or before January 31, 2027 upon
receipt of semi-annual reports from the FFB outlining the progress being made in the Study, including visit completion status and publication plans. For more information on the terms of the 2025 Letter Agreement and related payments
thereunder, see Note 6 — Related Party Transactions in Part I, Item 1 – Financial Statements and Supplementary Data” of this Report.
RDF Agreement
On June 13, 2025, we entered into the RDF Agreement with the RDF, whose sole member is FFB, a significant stockholder of the
Company, relating to our program to develop gene therapies to treat patients impacted by retinitis pigmentosa caused by pathogenic variants in the MERTK gene.
Under the RDF Agreement, we will pay a milestone payment equal to the total amounts funded by RDF under the RDF Agreement upon the achievement of a regulatory
milestone. We will also make tiered royalty payments to RDF in low-to-mid single percentages until RDF has received aggregate royalty payments equal to 300% of the amounts funded by RDF under the Agreement. In the event of a change of control
of the Company or a sale or exclusive license of the MERTK Program, RDF will have the option to require us to buy out RDF’s interest under the Agreement for an amount equal to 100% of the funds disbursed to us under the Agreement. For more
information on the terms and related obligations under the RDF Agreement, see Note 6 — Related Party Transactions in “Part I, Item 1 – Financial Statements and
Supplementary Data” of this Report.
As of the date of this Report, we determined that none of the future obligations under the agreement were probable.
Other Commitments
In the course of normal operations, we enter into cancelable purchase commitments from time to time with our suppliers for various key research, clinical and manufacturing
services. The purchase commitments covered by these arrangements are subject to change based on our research and development efforts.
Other Funding Requirements
As noted above, certain of our cash requirements relate to the funding of our ongoing research and development of our gene therapy product candidates, inclusive of any potential
milestone and royalty obligations under our intellectual property licenses. See “Part I, Item 1— Business— Pipeline— Sales and Marketing—Manufacturing— Apexian Sublicense Agreement— Review and Approval of Drugs and Biologics in the United
States” in our Annual Report on Form 10-K for the year ended December 31, 2024 for a discussion of design, development, pre-clinical and clinical activities that we may conduct in the future, including expected cash expenditures required for
some of those activities, to the extent we are able to estimate such costs.
Our other cash requirements within the next twelve months include accounts payable, accrued expenses, purchase commitments and other current liabilities. Our other cash
requirements greater than twelve months from various contractual obligations and commitments may include operating leases and contractual agreements with third-party service providers for clinical research, product development, manufacturing,
commercialization, supplies, payroll, equipment maintenance, and audits for periods into calendar year 2026. Refer to Note 4 – Commitments and Contingencies included in Part 1, Item 1 – “Financial Statements” of this Report for further detail
of our lease obligation and license agreements with regard to the timing of expected future payments.
We expect to satisfy our short-term and long-term obligations through cash on hand, from future equity and debt financings, and from reimbursement payments, potential milestone
and royalty payments under the Viatris License Agreement and any future collaborations and license agreements, until we generate an adequate level of revenue from commercial sales to cover expenses, if ever.
Critical Accounting Policies and Estimates
Our financial statements are prepared in accordance with U.S. generally accepted accounting principles (GAAP). These accounting
principles require us to make estimates and judgments that can affect the reported amounts of assets and liabilities as of the date of the financial statements as well as the reported amounts of revenue and expense during the periods presented.
We believe that the estimates and judgments upon which we rely are reasonably based upon information available to us at the time that we make these estimates and judgments. To the extent that there are material differences between these
estimates and actual results, our financial results will be affected. The accounting policies that reflect our more significant estimates and judgments and which we believe are the most critical to aid in fully understanding and evaluating our
reported financial results are described below.
Our significant accounting policies are discussed in Note 1 — Company Description and Summary of Significant Accounting Policies, included in “Part I,
Item 1 – Financial Statements and Supplementary Data” of this Report. We believe that the following accounting policies and estimates are the most critical to aid in fully understanding and evaluating
our reported financial results. These estimates require our most difficult, subjective, or complex judgments because they relate to matters that are inherently uncertain. We have reviewed these critical accounting policies and estimates and
related disclosures with the Audit Committee of our Board. We have not made any material changes to date, nor do we believe there is a reasonable likelihood of a material future change to the accounting methodologies for the areas described
below.
License and Collaborations Revenue
We account for license and collaborations revenue in accordance with the provisions of the Financial Accounting Standards
Board Accounting Standards Codification 606, Revenue from Contracts with Customers. The guidance provides a unified model to determine how revenue is
recognized. We have entered into license and collaboration agreements which have revenue recognition implications. We recognize license and collaborations revenue by first allocating the transaction price of a contract to each performance
obligation under the contract based on its stand-alone price. The stand-alone price of each performance obligation is based on its fair value utilizing a discounted cash flow
approach, taking into consideration assumptions, including projected worldwide net profit for each of the respective programs based on probability assessments, projections based on internal forecasts, industry data, and information from
other guideline companies within the same industry and other relevant factors. We do not expect to have in the future significant variable consideration adjustments related to our existing license and collaborations revenue
recognized. For discussion about the determination of license and collaborations revenue, see Note 11 — License and Collaboration Revenue and Other Funding Agreements
included in Part 1, Item 1 – “Financial Statements” of this Report.
Income Tax Assets and Liabilities
A full valuation allowance has been provided on our net deferred tax assets given the uncertainty of future taxable income and other related factors impacting
the realizability of our remaining net deferred tax assets. For additional information, see Note 13 — Income Taxes included in “Part II, Item 8 – Financial Statements and Supplementary Data” in our Annual Report filed on Form 10-K for the
year ended December 31, 2024, and see Note 13 — Income Taxes included in “Part 1, Item 1 – Financial Statements” of this Report.
Recent Accounting Pronouncements
Refer to Note 1— “Company Description and Summary of Significant Accounting Policies” to our condensed consolidated financial statements included in “Part 1, Item 1 – Financial Statements” in
this Report for a discussion of recently issued accounting pronouncements.
| Item 3. |
Quantitative and Qualitative Disclosures About Market Risk
|
Not applicable for smaller reporting companies.
| Item 4. |
Controls and Procedures
|
Evaluation of Disclosure Controls and Procedures
We maintain disclosure controls and procedures that are designed to ensure that information we are required to disclose in our Exchange Act reports is recorded, processed, summarized and reported
within the time periods specified in the SEC’s rules and forms, and that such information is accumulated and communicated to our management, including our principal executive officer and principal financial officer, as appropriate, to allow
timely decisions regarding required disclosure.
We designed and evaluated our disclosure controls and procedures recognizing that any controls and procedures, no matter how well designed and operated, can provide only reasonable assurance and
not absolute assurance of achieving the desired control objectives. Also, the design of a control system must reflect the fact that there are resource constraints and that the benefits of controls must be considered relative to their costs.
Because of the inherent limitations in all control systems, no evaluation of controls can provide absolute assurance that misstatements due to error or fraud will not occur or that all control issues and instances of fraud, if any, have been
detected. These inherent limitations include the realities that judgments in decision-making can be faulty and that breakdowns can occur because of simple error or mistake. The design of any system of controls is based, in part, upon certain
assumptions about the likelihood of future events and there can be no assurance that any design will succeed in achieving its stated goals under all potential future conditions.
Under the supervision of and with the participation of our management, including our principal executive officer and principal financial officer, we evaluated the effectiveness of our disclosure
controls and procedures, as such term is defined in Rules 13a-15(e) and 15(d)- 15(e) promulgated under the Exchange Act as of September 30, 2025. Based on this evaluation, our principal executive officer and principal financial officer concluded
that our disclosure controls and procedures were effective as of September 30, 2025.
Changes in Internal Control Over Financial Reporting
There were no changes in our internal control over financial reporting (as defined in Rule 13a-15(f) under the Exchange Act) during the quarter ended September 30, 2025, that have materially
affected, or are reasonably likely to materially affect, the Company’s internal control over financial reporting.
PART II – OTHER INFORMATION
| Item 1. |
Legal Proceedings
|
From time to time, we may be involved in various claims and legal proceedings relating to claims arising out of our operations. We are not currently a
party to any legal proceedings that, in the opinion of our management, are likely to materially affect our business or financial results. Regardless of outcome, litigation can have an adverse impact on us because of defense and settlement costs,
diversion of management resources and other factors.
| Item 1A. |
Risk Factors
|
Other than as set forth below, there have been no material changes in our risk factors previously disclosed in Part I, Item 1A “Risk Factors” in
our Annual Report on Form 10-K for the year ended December 31, 2024. You should carefully consider the risks and uncertainties described below and therein.
Our ability to utilize our common stock to finance future capital needs, or for other purposes, is limited by our authorized
shares available for issuance.
Following the November 2025 Registered Direct Offering, we had authority to issue a total of 125.0 million shares of common stock, of which approximately 69.0
million shares had been issued and 54.4 million shares were reserved for issuance pursuant to outstanding stock options, restricted stock units, and warrants.
With the limited shares of common stock presently available for issuance, our ability to secure additional funding through the sale of common stock is very limited.
Absent an increase in the shares of common stock authorized to be issued, we will be limited to other financing structures in the event additional financing is required. Such alternative structures may be less favorable or unavailable in which
case we may be forced to forego opportunities. We may also be limited in our ability to offer equity awards to incentive our employees if we do not have adequate shares available for issuance.
Instability and operational disruptions at government agencies, such as the FDA, may adversely impact our development and
commercialization plans by causing delays and requiring the use of additional, unforeseen resources to obtain regulatory approval for trials or products in our pipeline.
Our business depends largely on the successful clinical development, regulatory approval and commercialization of our product candidates. To
advance our candidates, we, along with many of the third‐parties we currently and may in the future work with, must work closely with the FDA and comparable regulatory agencies in foreign jurisdictions. Such authorities play a vital role in the
development of our product candidates by providing guidance on our clinical programs and reviewing and approving our regulatory submissions, including IND applications, requests for special designations, such as Orphan Drug Designation and Rare
Pediatric Disease Designation, and marketing applications.
If these agencies experience unexpected funding losses or mass layoffs or are otherwise affected by the current or any future shutdown of the U.S.
federal government, their oversight and review activities of existing and new submissions, such as IND applications, may be significantly disrupted or delayed. Such disruptions or delays could adversely impact our ability to develop and secure
timely approval of our product candidates.
There are also ongoing deliberations within the current presidential administration and Congress over potentially substantial proposed cuts to the
overall budget for the U.S. Department of Health and Human Services and funding of the FDA for the 2026 federal fiscal year. These budgetary decisions are unpredictable and remain subject to change. Such decisions could make it more difficult to
secure grant funding from government agencies and nongovernmental foundations. Further, there is also instability and uncertainty surrounding how the current U.S. administration will seek to modify or revise the requirements and policies of the
FDA and other regulatory agencies with jurisdiction over our product candidates. We may have to expend additional resources to comply with any new policies and to engage with the appropriate agencies.
Government shutdowns and funding cuts may also negatively affect the SEC. In the event of an extended shutdown, the SEC may operate with limited
staff or suspend certain functions altogether, which could delay the review or effectiveness of our filings, including registration statements or other financing-related disclosures. Such delays could adversely affect our ability to access the
public markets and obtain necessary capital in order to properly capitalize and continue to fund our operations.
If we fail to comply with the continued listing standards of Nasdaq, our common stock may be delisted, and our ability to
access the capital markets could be negatively impacted.
Our common stock is listed for trading on the Nasdaq Capital Market (“Nasdaq”). In order to maintain our listing, we must satisfy Nasdaq’s
continued listing requirements (the “Nasdaq Listing Rules”). The Nasdaq Listing Rules require that the closing price of our common stock generally remains at or above $1.00 per share (the “Minimum Bid Price Requirement”). The closing price of
our common stock has recently traded below $1.00, and may fall below $1.00 per share in the future. If our common stock closes at a price below $1.00 per share for 30 consecutive business days, we could be delisted from Nasdaq. In the event we
do receive notice of deficiency from Nasdaq, we expect that applicable Nasdaq Listing Rules would provide us with an initial period of 180 calendar days to regain compliance with the Minimum Bid Price Requirement, which may be extended by
Nasdaq in its discretion.
There are no assurances that we will be able to continue to comply with the Minimum Bid Price Requirement. We intend to monitor the closing
price of our common stock and may, if appropriate, consider available options to regain compliance with the Minimum Bid Price Requirement, which could include seeking to effect a reverse stock split. There are many factors outside of our
control that may adversely affect our minimum bid price, including those described in the “Risk Factors” section of our most recent Annual Report on Form 10-K for the year ended December 31, 2024 and in our other filings with the SEC.
Any potential delisting of our common stock from Nasdaq would likely result in decreased liquidity and increased volatility for our common stock
and may damage our reputation, adversely affecting our ability to raise additional capital or to pursue our strategic business plans. Additionally, delisting would make it more difficult for our stockholders to sell our common stock in the
public market. Further, if the Company seeks to implement a reverse stock split in order to remain listed, the announcement or implementation of such a reverse stock split could negatively affect the price of our common stock.
| Item 2. |
Unregistered Sales of Equity Securities and Use of Proceeds
|
None.
| Item 3. |
Defaults Upon Senior Securities
|
None.
| Item 4. |
Mine Safety Disclosures
|
Not applicable to our Company.
| Item 5. |
Other Information
|
During the quarter ended September 30, 2025, none of the Company’s directors or officers has adopted terminated
| Item 6. |
Exhibits
|
|
EXHIBIT
|
|
|
NUMBER
|
DESCRIPTION OF DOCUMENT
|
|
Fourth Amendment to Ocuphire Pharma, Inc. 2021 Inducement Plan (incorporated by reference to Exhibit 4.8 to Registrant’s Registration Statement on Form S-8, filed on September 10, 2025).
|
|
|
Funding and License Agreement, dated as of July 22, 2025, by and among the Company, OpusTX, LLC, Eyes on the Future and RDH12 Fund for Sight (incorporated by reference to Exhibit 10.2 to Registrant’s
Quarterly Report on Form 10-Q, filed on August 13, 2025).
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|
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Employment Agreement, dated as of August 29, 2025, by and between the Company and Robert Gagnon (incorporated by reference to Exhibit 10.1 to Registrant’s Current Report on Form 8-K, filed on September 2,
2025).
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|
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Side Letter to the License and Collaboration Agreement, dated as of August 13, 2025, by and between the Company and FamilyGen Life Sciences, Inc.
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Certification of Principal Executive Officer pursuant to Section 302 of the Sarbanes-Oxley Act of 2002.
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Certification of Principal Financial Officer pursuant to Section 302 of the Sarbanes-Oxley Act of 2002.
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Certification of Principal Executive Officer and Principal Financial Officer pursuant to Section 906 of the Sarbanes-Oxley Act of 2002.
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101.INS
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Inline XBRL Instance Document.
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101.SCH
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Inline XBRL Taxonomy Extension Schema Document.
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101.CAL
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Inline XBRL Taxonomy Extension Calculation Linkbase Document.
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101.DEF
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Inline XBRL Taxonomy Extension Definition Linkbase Document.
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101.LAB
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Inline XBRL Taxonomy Extension Label Linkbase Document.
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101.PRE
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Inline XBRL Taxonomy Extension Presentation Linkbase Document.
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104
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Cover Page Interactive Data File (formatted as inline XBRL and contained in Exhibit 101)
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| * |
Documents are furnished not filed.
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| ** |
Indicates exhibits that are being filed herewith.
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| + |
Portions of this exhibit have been omitted in compliance with Item 601 of Regulation S-K.
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SIGNATURES
Pursuant to the requirements 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: November 12, 2025
Opus Genetics, Inc.
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By:
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/s/ George Magrath
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George Magrath
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||
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Chief Executive Officer and Director
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||
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(Principal Executive Officer)
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||
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By:
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/s/ Robert Gagnon
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|
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Robert Gagnon
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||
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Chief Financial Officer
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||
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(Principal Financial Officer and Accounting Officer)
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50
Exhibit 10.3
CERTAIN IDENTIFIED INFORMATION HAS BEEN EXCLUDED FROM THE EXHIBIT BECAUSE IT IS BOTH NOT MATERIAL AND IS THE TYPE THAT THE REGISTRANT TREATS AS PRIVATE OR CONFIDENTIAL. OMITTED INFORMATION HAS BEEN
REPLACED WITH ASTERISKS [***].
Side Letter to the License and Collaboration Agreement
FamyGen Life Sciences, Inc. (“FamyGen”) and Opus Genetics, Inc. f/k/a Ocuphire Pharma Inc., (“Opus”) (each a “Party” and collectively, the “Parties”) have entered into a License and Collaboration Agreement dated November 6, 2022 (the “License Agreement”), defining certain rights and obligations between the parties regarding Third Party Infringement actions concerning the Licensed Intellectual Property Rights. In
accordance with Section 8.5 of the License Agreement, FamyGen and Opus have instituted Civil Action No. 25-1895 (D.N.J) against a Third Party asserting one or more patents listed in the Orange Book in connection with NDA No. 217064 for RYZUMVI® (the “Action”). This letter sets forth the terms and conditions upon which FamyGen and Opus shall share
the expenses associated with the Action only.
The Parties hereby agree that Opus shall pay Expenses1 up to a cap of $[***] in the Action as follows:
| 1. |
Opus shall pay the first $[***] in Expenses as they are billed.
|
| 2. |
Any Expenses in excess of the first $[***] incurred as of October 15, 2026, will be paid initially by FamyGen. The accrued balance of those Expenses, up to the cap of $[***], will then be paid by Opus to FamyGen in full by no later
than November 15, 2026.
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| 3. |
After October 15, 2026, any Expenses up to the cap of $[***] will be paid by Opus as they are billed.
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| 4. |
To the extent there are any outstanding Expenses (up to the cap of $[***] less Expenses already paid by Opus) owed to FamyGen by Opus upon achieving FDA Approval for Product 1B or Product 1C (as defined in the License Agreement),
FamyGen shall have the right to offset such amount of outstanding Expenses by withholding the equivalent amount from any Regulatory Milestone payment related to FDA Approval for either Product 1B or Product 1C under Schedule 3, Section
1.(b) of the License Agreement.
|
| 5. |
To the extent FDA Approval for Product 1B or Product 1C has not occurred, or occurred at a point in time where no Expenses were owed, FamyGen shall have the right to offset any outstanding Expenses (up to the cap of $[***] less
Expenses already paid by Opus) by withholding the equivalent amount from any royalties owed pursuant to the Royalty Payments owed under Schedule 3, Section 1.(c) of the License Agreement.
|
1 The term “Expenses” means all reasonable and necessary costs and expenses related to the Action, exclusive of attorneys’ fees of
Katten and Dechert and any other outside counsel retained by Opus and FamyGen, and includes without limitation court filing fees, third party document review, process server fees, expert witness fees and expenses, local counsel fees, deposition
costs, including court reporter fees and transcription costs, document and electronic data hosting and duplication costs, travel expenses, demonstrative exhibits costs, translation services and any other out-of-pocket expenses directly related to
the Action.
| 6. |
Opus agrees to pay any and all attorneys’ fees and costs of any law firm retained solely by Opus in connection with the Action.
|
Except as expressly provided in this letter, all terms and provisions of the License Agreement are and will remain in full force and effect.
[SIGNATURE PAGE FOLLOWS]
The foregoing accurately reflects the Parties’ understanding of the terms and conditions for the sharing of expenses for the Action brought pursuant to the License Agreement.
|
FAMYGEN LIFE SCIENCES, INC.
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||
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By:
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/s/ Kevin Macikowski | |
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Name:
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Kevin Macikowski | |
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Title:
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Assistant Secretary | |
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OPUS GENETICS, INC.
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||
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By:
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/s/ Bernhard Hoffmann | |
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Name:
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Bernhard Hoffmann | |
|
Title:
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SVP – Corporate Development | |
Page 3 of 3
Exhibit 31.1
CERTIFICATION PURSUANT TO EXCHANGE ACT RULE 13a-14(a) OR 15d-14(a), AS ADOPTED
PURSUANT TO SECTION 302 OF THE SARBANES OXLEY ACT OF 2002
I, George Magrath, certify that:
| 1. |
I have reviewed this Quarterly Report on Form 10-Q for the quarter ended September 30, 2025 of Opus Genetics, 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 consolidated 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 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.
|
|
Date: November 12, 2025
|
/s/ George Magrath
|
|
|
Name:
|
George Magrath
|
|
|
Title:
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Chief Executive Officer
(Principal Executive Officer)
|
|
Exhibit 31.2
CERTIFICATION PURSUANT TO EXCHANGE ACT RULE 13a-14(a) OR 15d-14(a), AS ADOPTED
PURSUANT TO SECTION 302 OF THE SARBANES OXLEY ACT OF 2002
I, Robert Gagnon, certify that:
| 1. |
I have reviewed this Quarterly Report on Form 10-Q for the quarter ended September 30, 2025 of Opus Genetics, 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 consolidated 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 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.
|
|
Date: November 12, 2025
|
/s/ Robert Gagnon
|
|
|
Name:
|
Robert Gagnon
|
|
|
Title:
|
Chief Financial Officer
|
|
|
(Principal Financial Officer and Principal Accounting Officer)
|
||
Exhibit 32.1
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 Quarterly Report on Form 10-Q for the quarter ended September 30, 2025 (the “Report”) of Opus Genetics, Inc., a Delaware corporation (the “Company”) as
filed with the Securities and Exchange Commission, George Magrath, as Chief Executive Officer of the Company, and Robert Gagnon, as Chief Financial Officer of the Company, each hereby certifies, pursuant to Section 906 of the Sarbanes-Oxley Act
of 2002, 18 U.S.C. Section 1350, that to the best of his knowledge and belief:
(1) The Report fully complies with the requirements of section 13(a) or 15(d) of the Securities Exchange Act of 1934 (15 U.S.C. 78m or 78o(d)); and
(2) The information contained in the Report fairly presents, in all material respects, the financial condition and result of operations of the Company.
|
Date: November 12, 2025
|
|
|
/s/ George Magrath
|
|
|
George Magrath
|
|
|
Chief Executive Officer
|
|
|
(Principal Executive Officer)
|
|
|
/s/ Robert Gagnon
|
|
|
Robert Gagnon
|
|
|
Chief Financial Officer
|
|
|
(Principal Financial Officer and Principal Accounting Officer)
|