Abeona Therapeutics Inc 10-K for FY2022

Abeona Therapeutics Inc. (ABEO)

10-K 2023-03-29 For: 2022-12-31

Added on April 08, 2026

About this 10-K

Abeona Therapeutics Inc filed this 10-K on March 29, 2023, covering the fiscal year ended December 31, 2022 (FY2022).

Headline figures

Fiscal year ended December 31, 2022, as reported in this filing, compared with the same period a year earlier where the filing states it.

Revenue: $1.41M; down 52.9% from $3M a year earlier
Net loss: $39.7M; compared with a net loss of $84.94M a year earlier

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(Mark One)
☒	ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934
For the fiscal year ended December 31, 2022

Or

☐	TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934
For the transition period from _______ to _______

Delaware		83-0221517
(State or Other Jurisdiction of incorporation or Organization		(I.R.S. Employer Identification No.)

Title of each class		Trading Symbol(s)		Name of each exchange on which registered
Common Stock, $0.01 par value		ABEO		Nasdaq Capital Market

Large accelerated filer ☐	Accelerated filer ☐
Non-accelerated filer ☒	Smaller reporting company ☒
Emerging growth company ☐

		Page
Part I
Item 1.	Business	3
Item 1A.	Risk Factors	25
Item 1B.	Unresolved Staff Comments	54
Item 2.	Properties	54
Item 3.	Legal Proceedings	54
Item 4.	Mine Safety Disclosures	54

Part II
Item 5.	Market for Registrant’s Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities	55
Item 7.	Management’s Discussion and Analysis of Financial Condition and Results of Operations	56
Item 7A.	Quantitative and Qualitative Disclosures About Market Risk	65
Item 8.	Financial Statements and Supplementary Data	65
Item 9.	Changes in and Disagreements With Accountants on Accounting and Financial Disclosure	65
Item 9A.	Controls and Procedures	65
Item 9B.	Other Information	66
Item 9C.	Disclosure Regarding Foreign Jurisdictions that Prevent Inspections	66

Part III
Item 10.	Directors, Executive Officers and Corporate Governance	67
Item 11.	Executive Compensation	67
Item 12.	Security Ownership of Certain Beneficial Owners and Management and Related Stockholder Matters	67
Item 13.	Certain Relationships and Related Transactions, and Director Independence	67
Item 14.	Principal Accounting Fees and Services	67

Part IV
Item 15.	Exhibits, Financial Statement Schedules	68
Item 16.	Form 10-K Summary	69

Signatures		70

	●	sufficient scale to support commercial manufacturing requirements for EB-101
	●	processes related to biopsy, cell collection, storage and transportation as part of manufacturing for EB-101
	●	processes related to product release testing for EB-101
	●	processes related to the manufacture and release testing of retroviral supernatant
	●	establishing transportation and packaging processes and materials for finished EB-101 product
	●	proprietary AAV vector manufacturing processes and techniques that produce a highly purified product candidate
	●	AAV serum-free suspension technology that is readily scalable
	●	multiple assays to accurately characterize our process and the AAV vectors we produce
	●	a series of purification processes, which may be adapted and customized for multiple different AAV capsids, with a goal of higher concentrations of active vectors, and that are essentially free of empty capsids.

	●	completion of preclinical laboratory tests and in vivo studies in accordance with the FDA’s current Good Laboratory Practice (“GLP”) regulations and applicable requirements for the humane use of laboratory animals or other applicable regulations;
	●	submission to the FDA of an application for an IND, which allows human clinical trials to begin unless the FDA objects within 30 days;
	●	approval by an independent institutional review board (“IRB”), reviewing each clinical site before each clinical trial may be initiated;
	●	performance of adequate and well-controlled human clinical trials according to the FDA’s Good Clinical Practice (“GCP”) regulations, and any additional requirements for the protection of human research subjects and their health information, to establish the safety and efficacy of the proposed biologic product candidate for its intended use;
	●	development of manufacturing processes to ensure the product candidate’s identity, strength, quality, purity, and potency;
	●	preparation and submission to the FDA of a BLA for marketing approval that includes substantial evidence of safety, purity and potency from results of nonclinical testing and clinical trials;
	●	satisfactory completion of an FDA pre-approval inspection of the manufacturing facility or facilities where the biologic product candidate is produced to assess compliance with cGMP and to assure that the facilities, methods and controls are adequate to preserve the biologic product candidate’s identity, safety, strength, quality, potency and purity;
	●	potential FDA audit of the nonclinical and clinical trial sites that generated the data in support of the BLA; and
	●	payment of user fees and the FDA review and approval, or licensure, of the BLA. BLA application fees for products designated as orphan drugs by the FDA are waived.

	●	Phase 1: The biologic product candidate initially is introduced into healthy human subjects and tested for safety, dosage tolerance, absorption, metabolism, distribution, excretion and, if possible, to gain an early understanding of its effectiveness. In the case of some product candidates for severe or life-threatening diseases, especially when the product candidate may be too inherently toxic to ethically administer to healthy volunteers, the initial human testing is often conducted in patients.

	●	Phase 2: The biologic product candidate is evaluated in a limited patient population to identify possible adverse effects and safety risks, to preliminarily evaluate the efficacy of the product candidate for specific targeted diseases and to determine dosage tolerance, optimal dosage and dosing schedule.

	●	Phase 3: The biologic product candidate is administered to an expanded patient population at geographically dispersed clinical trial sites in adequate and well-controlled clinical trials to generate sufficient data to statistically confirm the efficacy and safety of the product for approval. These clinical trials are intended to establish the overall risk/benefit ratio of the product candidate and provide an adequate basis for product labeling. Typically, two Phase 3 trials are required by the FDA for product approval. Under some limited circumstances, however, the FDA may approve a BLA based upon a single Phase 3 clinical study plus confirmatory evidence or a single large multicenter trial without confirmatory evidence.

	●	Priority review: A product candidate is eligible for priority review if it treats a serious condition and, if approved, it would be a significant improvement in the safety or effectiveness of the treatment, diagnosis or prevention of a serious condition compared to marketed products. The FDA aims to complete its review of priority review applications within six months as opposed to 10 months for standard review.

	●	Accelerated approval: Drug or biologic products studied for their safety and effectiveness in treating serious or life-threatening illnesses and that provide meaningful therapeutic benefit over existing treatments may receive accelerated approval. Accelerated approval means that a product candidate may be approved on the basis of adequate and well-controlled clinical trials establishing that the product candidate has an effect on a surrogate endpoint that is reasonably likely to predict a clinical benefit, or on the basis of an effect on a clinical endpoint other than survival or irreversible morbidity or mortality or other clinical benefit, taking into account the severity, rarity and prevalence of the condition and the availability or lack of alternative treatments. As a condition of approval, the FDA may require that a sponsor of a drug or biologic product candidate receiving accelerated approval perform adequate and well-controlled post-marketing clinical trials. In addition, the FDA currently requires as a condition for accelerated approval pre-approval of promotional materials. Failure to conduct required post-approval studies, or confirm a clinical benefit during post-marketing studies, will allow the FDA to withdraw the drug or biologic from the market on an expedited basis.

	●	The second applicant can establish in its application that its medicinal product, although similar to the orphan medicinal product already authorized, is safer, more effective or otherwise clinically superior;
	●	The holder of the marketing authorization for the original orphan medicinal product consents to a second orphan medicinal product application; or
	●	The holder of the marketing authorization for the original orphan medicinal product cannot supply sufficient quantities of orphan medicinal product.

	●	the federal Anti-Kickback Statute, which prohibits, among other things, persons, and entities from knowingly and willfully soliciting, receiving, offering or paying remuneration, directly or indirectly, in cash or kind, in exchange for, or to induce, either the referral of an individual for, or the purchase, order or recommendation of, any good or service for which payment may be made under federal healthcare programs such as the Medicare and Medicaid programs. This statute has been interpreted to apply to arrangements between pharmaceutical manufacturers, on the one hand, and prescribers, purchasers, and formulary managers on the other. Although a number of statutory exemptions and regulatory safe harbors exist to protect certain common activities from falling under the Anti-Kickback Statute, these are narrow, and practices may not fall under the applicable safe harbors and exemptions. For example, the United States Department of Health and Human Services recently promulgated a regulation that is effective in two phases. First, the regulation excludes from the definition of “remuneration” limited categories of (a) PBM rebates or other reductions in price to a plan sponsor under Medicare Part D or a Medicaid Managed Care Organization plan reflected in point-of sale reductions in price and (b) PBM service fees. Second, effective January 1, 2023, the regulation expressly provides that rebates to plan sponsors under Medicare Part D either directly to the plan sponsor under Medicare Part D, or indirectly through a pharmacy benefit manager will not be protected under the anti-kickback discount safe harbor. The PPACA amended the intent requirement of the federal Anti-Kickback Statute. A person or entity no longer needs to have actual knowledge of this statute or specific intent to violate it in order to commit a violation;

	●	the federal false claims and civil monetary penalties laws, including the civil False Claims Act (the “FCA”), which prohibit, among other things, individuals, or entities from knowingly presenting, or causing to be presented, claims for payment from Medicare, Medicaid or other third-party payors that are false or fraudulent, or making a false statement to avoid, decrease, or conceal an obligation to pay money to the federal government. Certain marketing practices, including off-label promotion, also may implicate the FCA. FCA claims may be pursued by whistleblowers through qui tam actions, even if the government declines to intervene and civil liability may be predicated on reckless disregard for the truth. The PPACA also codified case law that a claim including items or services resulting from a violation of the federal Anti-Kickback Statute constitutes a false or fraudulent claim for purposes of the FCA. Separately, the criminal federal False Claims Act imposes criminal fines or imprisonment against individuals or entities who make or present a claim to the government knowing such claim to be false, fictitious, or fraudulent;

	●	the federal Physician Payments Sunshine Act, which requires certain manufacturers of drugs, devices, biologics and medical supplies for which payment is available under Medicare, Medicaid, or the Children’s Health Insurance Program, with specific exceptions, to report annually to the Centers for Medicare & Medicaid Services (“CMS”), information related to payments and other transfers of value made to or at the request of covered recipients, such as, but not limited to, physicians, physician assistants, nurse practitioners, clinical nurse specialists, certified registered nurse anesthetists and teaching hospitals, as well as ownership and investment interests held by physicians and their immediate family. Payments made to physicians and certain research institutions for clinical trials are included within the ambit of this law. Reported information is made publicly available in searchable formats by CMS;

	●	additional federal false statements and fraud and abuse statutes prohibit knowingly and willfully executing, or attempting to execute, a scheme to defraud or to obtain, by means of false or fraudulent pretenses, representations or promises, any of the money or property owned by, or under the custody or control of, a healthcare benefit program, regardless of whether the payor is public or private, in connection with the delivery or payment for health care benefits, knowingly and willfully embezzling or stealing from a health care benefit program, willfully obstructing a criminal investigation of a health care offense and knowingly and willfully falsifying, concealing, or covering up by any trick or device a material fact or making any materially false statements in connection with the delivery of, or payment for, healthcare benefits, items, or services relating to healthcare matters. PPACA amended the intent requirement of certain of these criminal statutes under the Health Insurance Portability and Accountability Act of 1996 (“HIPAA”) so that a person or entity no longer needs to have actual knowledge of the statute, or the specific intent to violate it, to have committed a violation; and

	●	HIPAA, as amended by the Health Information Technology for Economic and Clinical Health Act of 2009, or HITECH Act, and similar state laws impose obligations on certain entities with respect to safeguarding the privacy, security and transmission of protected health information. HIPAA’s security and certain privacy standards are directly applicable to persons or organizations of covered entities, other than members of the covered entity’s workforce, that create, receive, maintain or transmit protected health information on behalf of a covered entity for a function or activity regulated by HIPAA. The HITECH Act strengthened the civil and criminal penalties that may be imposed against covered entities, business associates and individuals, and gave state attorneys general new authority to file civil actions for damages or injunctions in federal courts to enforce the federal HIPAA laws and seek attorneys’ fees and costs associated with pursuing federal civil actions. In addition, other federal and state laws, such as the California Consumer Privacy Act, may regulate the privacy and security of information that we maintain, many of which may differ from each other in significant ways and may not be preempted by HIPAA; and

	●	the General European Data Protection Regulation (“GDPR”), which became applicable May 25, 2018, harmonizes data privacy laws across Europe. The GDPR sets forth rules relating to the protection with regard to the processing and transfer of personal data as well as an individual’s right to the protection of personal data, including medical information and clinical trial related data. In addition, there are rules relating to the export of personal data outside the European Union and in particular there are certain challenges in relation to export to the United States.

	●	Our cell and gene therapy product candidates are based on proprietary methodologies, which makes it difficult to predict the time and cost of product candidate development and regulatory approval. Additionally, regulatory requirements governing cell and gene therapy products have evolved and may continue to change in the future.
	●	If we do not obtain the necessary U.S. or worldwide regulatory approvals to commercialize EB-101, we will not be able to sell EB-101.
	●	Even if we receive regulatory approval for EB-101, our lead drug candidate, we may not be able to successfully commercialize the product and the revenue that we generate from its sales, if any, may be limited.
	●	We may encounter substantial delays in our clinical studies or we may fail to demonstrate safety and efficacy to the satisfaction of applicable regulatory authorities. Additionally, we may find it difficult to enroll patients in our clinical studies, which could delay or prevent clinical studies of our product candidates.
	●	We have received and may apply for additional designations such as breakthrough therapy designation, RMAT designation, fast track designation, and rare pediatric disease designation from the FDA intended to facilitate or encourage product candidate development. We may not receive any such designations or be able to maintain them. Moreover, any such designations may not lead to faster development or regulatory review or approval and it does not increase the likelihood that our product candidates will receive marketing approval.
	●	While certain of our product candidates have received orphan drug designation from the FDA, there is no guarantee that we will be able to maintain this designation, receive this designation for any of our other product candidates, or receive or maintain any corresponding benefits, including periods of exclusivity.
	●	Even if we obtain regulatory approval for a product candidate, our products will remain subject to regulatory scrutiny.
	●	The COVID-19 pandemic and efforts to reduce its spread have affected our operations and significantly impacted worldwide economic conditions, and could continue to have a material effect on our operations, business, and financial condition.
	●	We could experience production problems in our manufacturing facilities that result in delays in our development or commercialization programs. We might also experience delays in manufacturing if any of our vendors, contract laboratories or suppliers are found to be out of compliance with current Good Manufacturing Practice.
	●	If we fail to comply with applicable regulations, the relevant regulatory authority may require remedial measures that may be costly or time-consuming to implement and that may include the suspension of a clinical trial or commercial sales or the closure of a manufacturing facility.
	●	The widespread outbreak of an illness, communicable disease, or any other public health crisis could adversely affect our business, results of operations and financial condition.
	●	We expect to rely on third parties, and these third parties may not perform satisfactorily. Additionally, our reliance on third parties requires us to share our trade secrets, which increases the possibility that a competitor will discover them or that our trade secrets will be misappropriated.
	●	Our drug candidates are subject to the risks of failure inherent in the development of pharmaceutical products based on new technologies, and our failure to develop safe and commercially viable drugs would severely limit our ability to become profitable or to achieve significant revenues.

	●	We may be unable to successfully develop, market, or commercialize our products or our product candidates without establishing new relationships and maintaining current relationships and our ability to successfully commercialize, and market our product candidates could be limited if a number of these existing relationships are terminated.
	●	We may incur substantial product liability expenses due to the use or misuse of our products for which we may be unable to obtain insurance coverage.
	●	Our ability to successfully develop and commercialize our drug candidates will substantially depend upon the availability of reimbursement funds for the costs of the resulting drugs and related treatments.
	●	The market may not accept any pharmaceutical products that we develop, and adverse public perception of gene therapy products may negatively affect demand for, or regulatory approval of, our product candidates.
	●	We may be subject to federal, state, and foreign healthcare laws and regulations, including fraud and abuse laws, false claims laws, health information privacy and security laws and data privacy laws. If we are unable to comply, or have not fully complied, with such laws, we could face substantial penalties.
	●	Our business could suffer if we lose the services of, or fail to attract, key personnel.
	●	Trends toward managed health care and downward price pressures on medical products and services may limit our ability to profitably sell any drugs that we may develop.
	●	Our rights to develop and commercialize our product candidates are subject to, in part, the terms and conditions of licenses granted to us by others.
	●	If we are unable to obtain and maintain patent protection for our product candidates and technology, or if the scope of the patent protection obtained is not sufficiently broad, our competitors could develop and commercialize products and technology similar or identical to ours.
	●	Our intellectual property licenses with third parties may be subject to disagreements over contract interpretation.
	●	We may not be successful in obtaining necessary rights to our product candidates through acquisitions and in-licenses.
	●	We may not be able to protect our intellectual property rights around the world.
	●	Issued patents covering our product candidates could be found invalid or unenforceable if challenged in court. We may not be able to protect our trade secrets in court, and intellectual property litigation could cause us to spend substantial resources.
	●	Third-parties may initiate legal proceedings alleging that we are infringing their intellectual property rights, the outcome of which would be uncertain and could harm our business.
	●	We may be subject to claims asserting that our employees, consultants or advisors have wrongfully used or disclosed alleged trade secrets of their current or former employers or claims asserting ownership of what we regard as our own intellectual property.
	●	If we do not obtain patent term extension and data exclusivity for our product candidates, our business may be harmed.
	●	We have experienced a history of losses; we expect to incur future losses and we may be unable to obtain necessary additional capital to fund operations in the future. We do not have significant operating revenue and may never achieve profitability.
	●	Failure to achieve and maintain effective internal controls could have a material adverse effect on our business.
	●	We expect to continue to need to raise additional capital to operate our business, and our failure to obtain funding when needed or on terms that are favorable to us may force us to delay, reduce or eliminate our development programs or aspects thereof.
	●	The market price of our common stock may be volatile and adversely affected by several factors.
	●	Raising additional funds by issuing securities or through licensing or lending arrangements or through our at-the-market sale agreement may cause dilution to our existing stockholders, restrict our operations or require us to relinquish proprietary rights.
	●	Our quarterly operating results may fluctuate significantly.
	●	Provisions of our charter documents could discourage an acquisition of our company.

	●	regulators or IRBs may not authorize us or our investigators to commence or continue a clinical trial, conduct a clinical trial at a prospective trial site, or amend trial protocols, or regulators or IRBs may require that we modify or amend our clinical trial protocols;
	●	we may experience delays in reaching, or fail to reach, agreement on acceptable clinical trial contracts or clinical trial protocols with prospective trial sites and our CROs;
	●	regulators may require us to perform additional or unanticipated clinical trials to obtain approval or we may be subject to additional post-marketing testing, surveillance, or REMS requirements to maintain regulatory approval;
	●	flaws in a clinical trial may not become apparent until the trial is well advanced;
	●	clinical trials of our product candidates may produce negative or inconclusive results, or our studies may fail to reach the necessary level of statistical significance, and we may decide, or regulators may require us, to conduct additional clinical trials or abandon product development programs;
	●	clinical trials of our product candidates may require us to provide follow-up patient visits for safety for a minimum of five years even if we were to terminate and/or abandon a product development program;
	●	our third-party contractors may fail to comply with regulatory requirements or the clinical trial protocol, or fail to meet their contractual obligations to us in a timely manner, or at all, or we may be required to engage in additional clinical trial site monitoring;
	●	we, the regulators, or IRBs may require the suspension or termination of clinical research for various reasons, including noncompliance with regulatory requirements or a finding that the participants are being exposed to unacceptable health risks, undesirable side effects, or other unexpected characteristics (alone or in combination with other products) of the product candidate, or due to findings of undesirable effects caused by a chemically or mechanistically similar therapeutic or therapeutic candidate;
	●	changes in marketing approval and regulatory review policies or changes in or the enactment of additional statutes or regulations;
	●	the cost of clinical trials of and marketing applications for our product candidates may be greater than we anticipate;
	●	the supply or quality of our product candidates or other materials necessary to conduct clinical trials may be insufficient or inadequate;
	●	we may decide, or regulators may require us, to conduct or gather, as applicable, additional clinical trials, analyses, reports, data, or preclinical trials, or we may abandon product development programs;
	●	we may fail to reach an agreement with regulators or IRBs regarding the scope, design, or implementation of our clinical trials. For instance, the FDA or comparable foreign regulatory authorities may require changes to our study design that make further study impractical or not financially prudent;
	●	we may have delays in adding new investigators or clinical trial sites, or we may experience a withdrawal of clinical trial sites;
	●	there may be regulatory questions or disagreements regarding interpretations of data and results, or new information may emerge regarding our product candidates;
	●	we may make changes to our product candidates or their manufacturing process that necessitate additional studies or that result in our product candidates not performing as expected;

	●	the FDA or comparable foreign regulatory authorities may disagree with our study design, including endpoints, or our interpretation of data from preclinical studies and clinical trials or find that a product candidate’s benefits do not outweigh its safety risks;
	●	the FDA or comparable foreign regulatory authorities may not accept data from studies with clinical trial sites in foreign countries;
	●	the FDA or comparable regulatory authorities may disagree with our intended indications;
	●	the FDA or comparable foreign regulatory authorities may fail to approve or subsequently find fault with the manufacturing processes or our contract manufacturer’s manufacturing facility for clinical and future commercial supplies;
	●	the data collected from clinical trials of our product candidates may not be sufficient to the satisfaction of the FDA or comparable foreign regulatory authorities to support the submission of a marketing application, or other comparable submission in foreign jurisdictions or to obtain regulatory approval in the United States or elsewhere;
	●	if one of our product candidates does not receive marketing approval in one country, it may impact our ability to receive marketing approval in other countries;
	●	the FDA or comparable regulatory authorities may take longer than we anticipate to make a decision on our product candidates; and
	●	we may not be able to demonstrate that a product candidate provides an advantage over current standards of care or current or future competitive therapies in development.

	●	severity of the disease under investigation;
	●	design of the study protocol;
	●	size and nature of the patient population;
	●	eligibility criteria for and design of the study in question;
	●	perceived risks and benefits of the product candidate under study, including as a result of adverse effects observed in similar or competing therapies;
	●	proximity and availability of clinical study sites for prospective patients;
	●	availability of competing therapies and clinical studies;
	●	efforts to facilitate timely enrollment in clinical studies;
	●	ability to compensate patients for their time and effort;
	●	risk that enrolled patients will drop out before completion or not return for post-treatment follow-up;
	●	inability to obtain or maintain patient informed consents;

	●	effectiveness of publicity created by clinical trial sites regarding the trial;
	●	patient referral practices of physicians; and
	●	ability to monitor patients adequately during and after treatment.

	●	restrictions on manufacturing, distribution, marketing, or labeling of such products, including restrictions on the indication or approved patient population, and required additional warnings, such as black box warnings, contraindications, and precautions;
	●	requirements to conduct post-marketing studies or clinical trials, or to institute risk mitigation strategies, such as REMS;
	●	issuance of corrective information;
	●	the product may become less competitive, we may face reputational harm, or we may face liability for any harm caused to patients or subjects;
	●	modifications on the way the product is administered;
	●	modifications on promotional pieces;
	●	issuance of warning, untitled, or cyber letters asserting that we are in violation of the law, or of safety alerts, Dear Healthcare Provider letters, press releases, or other communications containing warnings or other safety information about the product;
	●	injunction or imposition civil or criminal penalties or monetary fines, restitution, or disgorgement of profits or revenues;
	●	suspension or withdrawal of regulatory approval;
	●	suspension or termination of any ongoing clinical studies;
	●	refusal to approve a pending marketing application, such as a BLA or supplements to a BLA submitted by us;
	●	seizure, detention, or recall of product;
	●	refusal to permit the import or export of our products; or
	●	refusal to allow us to enter into supply contracts, including government contracts, exclusion from federal healthcare programs, FDA debarment, consent decrees, or corporate integrity agreements.

	●	reduced control for certain aspects of manufacturing activities;
	●	reduced control over the protection of our trade secrets and know-how from misappropriation or inadvertent disclosure;
	●	inability to negotiate manufacturing agreements with third parties under commercially reasonable terms;
		reliance on the third party for regulatory compliance and quality assurance;
	●	termination or nonrenewal of manufacturing and service agreements with third parties in a manner or at a time that is costly or damaging to us; and
	●	disruptions to the operations of our third-party manufacturers and service providers caused by conditions unrelated to our business or operations, including the bankruptcy of the manufacturer or service provider.

	●	delay commercialization of, and our ability to derive product revenues from, EB-101 or any other product candidate;
	●	impose costly procedures on us; and
	●	diminish any competitive advantages that we may otherwise enjoy.

	●	actual and perceived efficacy and safety of EB-101;
	●	relative convenience, dosing burden and ease of administration;
	●	potential or perceived advantages or disadvantages over alternative treatments;
	●	potential post-marketing commitments imposed by regulatory authorities, such as patient registries;
	●	strength of sales, marketing and distribution support;
	●	price of our future products, both in absolute terms and relative to alternative treatments;
	●	the effect of current and future healthcare laws on EB-101; and
	●	availability of coverage and reimbursement from government and other third party payers.

	●	third-party-payors’ increasing challenges to the prices charged for medical products and services, including by limiting coverage and reimbursement and requiring payment of increased manufacturer rebates;
	●	the trend toward managed health care in the U.S. and the concurrent growth of Health Maintenance Organizations (“HMOs”) and similar organizations that can control or significantly influence the purchase of healthcare services and products; and
	●	state, federal, and foreign legislative proposals to control drug prices, reform healthcare or reduce government insurance programs.

	●	the scope of rights granted under the license agreement and other interpretation-related issues;
	●	the extent to which our technology and processes infringe intellectual property rights of the licensor that are not subject to the licensing agreement;
	●	the sublicensing of patent and other rights under our collaborative development relationships;
	●	our diligence obligations under the license agreement and what activities satisfy those diligence obligations;
	●	the inventorship or ownership of inventions and know-how resulting from the joint creation or use of intellectual property by our licensors and us and our partners; and
	●	the priority of invention of patented technology.

	●	others may be able to make gene therapy products that are similar to our product candidates but that are not covered by the claims of the patents that we license or may own in the future;
	●	we, or our license partners or current or future collaborators, might not have been the first to make the inventions covered by the issued patent or pending patent application that we license or may own in the future;
	●	we, or our license partners or current or future collaborators, might not have been the first to file patent applications covering certain of our or their inventions;
	●	others may independently develop similar or alternative technologies or duplicate any of our technologies without infringing our owned or licensed intellectual property rights;
	●	it is possible that our pending patent applications or those that we may own in the future will not lead to issued patents;
	●	issued patents that we hold rights to may be held invalid or unenforceable, including as a result of legal challenges by our competitors;
	●	our competitors might conduct research and development activities in countries where we do not have patent rights and then use the information learned from such activities to develop competitive products for sale in our major commercial markets;
	●	we may not develop additional proprietary technologies that are patentable;
	●	the patents of others may have an adverse effect on our business; and
	●	we may choose not to file a patent application for certain trade secrets or know-how, and a third party may subsequently file a patent application covering such intellectual property.

	●	seek regulatory and marketing approvals for our product candidates that successfully complete clinical studies;
	●	continue our research and preclinical and clinical development of our product candidates;
	●	further develop the manufacturing process for our vectors or our product candidates;
	●	expand the scope of our current clinical studies for our product candidates;
	●	change or add additional manufacturers or suppliers;
	●	seek to identify and validate additional product candidates;
	●	acquire or in-license other product candidates and technologies;
	●	make milestone or other payments under any license agreements;
	●	maintain, protect and expand our intellectual property portfolio;

	●	establish a sales, marketing and distribution infrastructure in the United States and Europe to commercialize any products for which we may obtain marketing approval;
	●	attract and retain skilled personnel;
	●	build additional infrastructure to support our operations as a larger public company and our product development and planned future commercialization efforts, including manufacturing capacity; and
	●	experience any delays or encounter issues with any of the above.

	●	our ability to integrate operations, technology, products, and services;
	●	our ability to execute our business plan;
	●	operating results below expectations;
	●	announcements concerning product development results, including clinical trial results;
	●	regulatory or legal developments in the U.S. or EU, including decisions from regulatory agencies relating to our product candidates;
	●	litigation or public concern about the safety of our potential products;
	●	our issuance of additional securities, including debt or equity or a combination thereof, which will be necessary to fund our operating expenses;
	●	announcements of technological innovations or new products by us or our competitors;
	●	loss of any strategic relationship;
	●	industry developments, including, without limitation, changes in healthcare policies or practices or third-party reimbursement policies;
	●	economic and other external factors;
	●	period-to-period fluctuations in our financial results; and
	●	whether an active trading market in our common stock develops and is maintained.

	●	variations in the level of expenses related to our development programs;
	●	addition or termination of clinical trials;
	●	any intellectual property infringement lawsuit or arbitration in which we are, or may become, involved;
	●	regulatory developments affecting our product candidates; and
	●	our execution of any collaborative, licensing or similar arrangements, and the timing of payments we may make or receive under these arrangements.

Plan Category	Number of securities to be issued upon exercise of outstanding options, warrants and rights		Weighted average exercise price of outstanding options warrants and rights		Number of securities remaining available for future issuance under equity compensation plans (excluding securities reflected in column (a))
	(a)		(b)		(c)
Equity compensation plans approved by security holders:
2015 Equity Incentive Plan		237,570	$	37.11		109,544
2005 Equity Incentive Plan		3,200	$	32.00		—
Equity compensation plans not approved by security holders:		—	$	—		—
Total equity compensation plans		240,770	$	37.04		109,544

	Number of securities to Total number of shared (our units) purchased		Weighted average Average price paid per share (or unit)
	(a)
Shares delivered or withheld pursuant to restricted stock awards
October 1, 2022 - October 31, 2022		207	$	4.30
November 1, 2022 - November 30, 2022		—	$	—
December 1, 2022 - December 31, 2022		—	$	—
		207	$	4.30

	For the year ended December 31,						Change
($ in thousands)	2022			2021			$			%

Revenues:
License and other revenues	$	1,414		$	3,000		$	(1,586	)		(53	)%

Expenses:
Royalties		450			—			450			N/A
Research and development		28,965			38,726			(9,761	)		(25	)%
General and administrative		17,256			21,644			(4,388	)		(20	)%
Impairment of goodwill		—			32,466			(32,466	)		N/A
Impairment of licensed technology		1,355			—			1,355			N/A
Impairment of right-of-use lease assets		2,511			—			2,511			N/A
Impairment of construction-in-progress		1,792			—			1,792			N/A
Total expenses		52,329			92,836			(40,507	)		(44	)%

Loss from operations		(50,915	)		(89,836	)		38,921			(43	)%

Gain on settlement with licensor		—			6,743			(6,743	)		N/A
PPP loan payable forgiveness income		—			1,758			(1,758	)		N/A
Interest income		431			40			391			978	%
Interest expense		(736	)		(3,656	)		2,920			(80	)%
Change in fair value of warrant liabilities		11,383			—			11,383			N/A
Other income		141			15			126			840	%
Net loss	$	(39,696	)	$	(84,936	)	$	45,240			(53	)%

	●	decreased clinical and development work for our cell and gene therapy product candidates and other related costs of $5.7 million which primarily relates to the license out/discontinuation of our MPSIII programs;
	●	decreased non-cash stock compensation expenses of $3.2 million; and
	●	decreased salary and related costs of $1.0 million; partially offset by
	●	increased other costs of $0.1 million.

	●	employee and consultant-related expenses;
	●	preclinical and developmental costs;
	●	clinical trial costs;
	●	the cost of acquiring and manufacturing clinical trial materials; and
	●	costs associated with regulatory approvals.

	●	decreased professional fees of $3.9 million;
	●	decreased non-cash stock-based compensation of $2.7 million; partially offset by
	●	increased other costs of $0.8 million; and
	●	increased salary and related costs of $1.4 million.