ABEONA THERAPEUTICS INC.

Our business and financial results are subject to numerous risks and uncertainties. There have been no material changes to the risk factors discussed in Part I, Item 1A. Risk Factors in our Annual Report on Form 10-K for the year ended December 31, 2025, except as set forth below.

Risks associated with the acquisition of product candidates could cause substantial delays in the preclinical and clinical development of our product candidates.

We acquired ABO-801 from Ellodi Pharmaceuticals, L.P. on November 6, 2026. Because we were not involved in the preclinical or clinical development of ABO-801 (then APT-1011) prior to such date, we may experience difficulties in the transition of certain development activities from Ellodi and its affiliates to us, which may result in delays in clinical trials, as well as problems in our development efforts, particularly if we do not receive all of the necessary information, reports and data from Ellodi and its affiliates in a timely manner. Further, we have had no involvement with or control over the preclinical and clinical development of ABO-801 to date. As a result, our assumptions about ABO-801’s development potential are based in large part on the data generated from trials conducted by Ellodi and we may observe materially and adversely different results in any ongoing or future clinical trials. In addition, results from preclinical and clinical trials can be interpreted in different ways. Even if we believe the nonclinical or clinical data for ABO-801 is promising, compliance or data integrity issues may later arise and even if not, the data may not be sufficient to support approval by the FDA or comparable foreign regulatory authorities. Approval of ABO-801 may be delayed by several years or may require us to expend significantly more resources than we have available.

We have relied on Ellodi’s having conducted research and development in accordance with the applicable protocol, legal, regulatory and scientific standards, having accurately reported the results of all clinical trials conducted prior to our agreement with Ellodi and having correctly collected and interpreted the data from these trials. To the extent any of these have not occurred, expected development time and costs may be increased, which could adversely affect any future revenue from this product candidate.

We may also acquire or in-license additional product candidates for pre-clinical or clinical development in the future as we continue to build our pipeline. The risks associated with acquiring or in-licensing such future product candidates could result in delays in the commencement or completion of our preclinical studies and clinical trials, and our ability to generate revenues from our product candidates may be delayed.

We can provide no assurance that ABO-801 will obtain regulatory approval or that the results of clinical trials will be favorable.

The testing, marketing and manufacturing of ABO-801 will require the approval of the FDA or regulatory agencies of other countries. We cannot predict with any certainty the amount of time necessary to obtain regulatory approvals, including from the FDA or other foreign regulatory authorities, and whether any such approvals will ultimately be granted. Preclinical and clinical trials may reveal that ABO-801 is ineffective or unsafe, in which event its further development could be seriously delayed or terminated. ABO-801 (then APT-1011) has had two completed Phase 3 studies and neither study met its prespecified co-primary patient-reported symptom endpoint. Although we plan to use the dysphagia symptom questionnaire (“DSQ”) in the next Phase 3 study for ABO-801, which has been used in connection with the approval of EoE products approved to-date in the United States, there is no guarantee that the FDA will agree with this approach or that the next Phase 3 study will be successful. In addition, we cannot guarantee that our planned clinical trials will be initiated or conducted as planned or completed on schedule, if at all.

Potential licensees, partners, or collaborators may view ABO-801 with more skepticism because of its history of failed clinical trials, thereby requiring additional data and further explanations to overcome this skepticism and obtain commercially reasonable terms for future development or collaboration.

Although the FDA approved FAYUVI™, also known as UX-111, which we have licensed to Ultragenyx, the royalties we expect to receive will depend on FAYUVI’s commercial performance and other factors that we do not control.

In 2022, we entered into a license agreement with Ultragenyx Pharmaceutical Inc. (“Ultragenyx”) granting Ultragenyx global development and commercialization rights to a gene therapy program (UX-111 or FAYUVI) for the treatment of Sanfilippo syndrome type A (MPS IIIA). The FDA approved FAYUVI™ (rebisufligene etisparvovec-hopf) in September 2026. Under the terms of our agreement with Ultragenyx, we are eligible to receive royalties on net sales and payments based on the achievement of certain sales milestones. There have as-yet been no commercial sales of FAYUVI, and we cannot predict the amount or timing of the royalties we will receive.

Ultragenyx has sole control over the commercialization of FAYUVI, including launch timing, pricing and discounting, payor contracting, and the sales and marketing resources it devotes to the product. Ultragenyx may prioritize other products over FAYUVI or change its commercial strategy. It may also sublicense or divest its rights in FAYUVI or undergo a change of control, and any successor in interest may have different commercial priorities.

We rely on Ultragenyx for sales information and for the accuracy of its royalty reports. Our audit rights are limited, audits are costly, and disputes over royalty calculations could delay or reduce payments. We are also exposed to Ultragenyx’s ability to pay amounts owed to us. If royalties from FAYUVI are lower or occur later than we expect, our results of operations and financial condition could be adversely affected.

Raising additional funds by issuing securities or through licensing or lending arrangements or through our at-the-market sale agreement would cause dilution to our existing stockholders, restrict our operations, or require us to relinquish rights to our technologies or product candidates and their future revenue streams.

We may need to raise additional capital to fund our future operations. Until such time, if ever, as we can generate substantial product revenues, we expect to finance our cash needs through equity offerings, debt financings, or other capital sources, including potential collaborations, strategic alliances, licensing arrangements, asset dispositions with third parties and other similar arrangements. In addition, we may seek additional capital due to favorable market conditions or strategic considerations, even if we believe we have sufficient funds for our current or future operating plans. If we raise additional capital by issuing equity securities, the share ownership of existing stockholders will be diluted. Any future debt financing may involve covenants that limit our ability to incur liens or additional debt, pay dividends, redeem, or repurchase our common stock, make certain investments or engage in certain merger, consolidation, or asset sale transactions. In addition, if we raise additional funds through collaborations, strategic alliances, licensing arrangements or asset dispositions with third parties, we may be required to relinquish valuable rights to our technologies, future revenue streams, research programs or product candidates, or grant licenses on terms that may not be favorable to us.

The terms of any financing may adversely affect the holdings or rights of our stockholders and the issuance of additional securities, whether equity or debt, or the possibility of such issuance, may cause the market price of our shares to decline. We may sell shares or other securities in other offerings, including under our open market sale agreement, at a price per share that is less than the prices per share paid by other investors, and investors purchasing shares of our common stock, preferred stock or other securities in the future could have rights superior to existing stockholders. The sale of additional equity or convertible securities would dilute all of our stockholders and the terms of these securities may include liquidation or other preferences that adversely affect our existing stockholders.

The manufacturing, testing and delivery of ZEVASKYN® present significant challenges for us, and we may not be able to produce ZEVASKYN®at the quality, quantities, or timing needed to support commercialization. Demand for ZEVASKYN may be lower than expected.

The manufacturing of ZEVASKYN® is complex and requires significant expertise. Even with the relevant experience and expertise, manufacturing cell therapy products often leads to difficulties in production, particularly in scaling out and validating initial production, managing the transition from clinical manufacturing to commercial manufacturing, and ensuring that the product meets required specifications. These problems include difficulties with production costs and yields, quality control, quality assurance testing, operator error, scarcity of qualified manufacturing and quality control testing personnel, shortages of any production raw materials as well as compliance with strictly enforced federal, state and foreign regulations.

We are susceptible to production interruptions that may impede our ability to manufacture cell and gene therapy products and produce an adequate product supply to support commercialization of ZEVASKYN®. Several factors could cause production interruptions, including equipment malfunctions, facility contamination, raw material shortages or contamination, natural disasters, public health emergencies, disruption in utility services, human error, or disruptions in the operations of our suppliers. ZEVASKYN® and product candidates are biologic drugs requiring processing steps that are more complex than those required for most chemical pharmaceuticals. We characterize our processes and products, and perform testing to ensure the safety, quality and efficacy of each product produced. While we take significant measures to fully understand and characterize each product, the steps we take may not be sufficient to ensure that a given lot will perform in the intended manner.

For example, we manufactured a full batch of ZEVASKYN®following patient biopsy collection in August 2025 that, despite being bonafide drug product, could not be released because a rapid sterility assay, mandated by the FDA as a release assay during the final stage of the BLA review, initially yielded a false positive result for sterility. Although we resumed biopsy collection in November 2025 upon completion of assay optimization and the necessary regulatory submission for its implementation, this false positive caused a manufacturing rejection, which caused a delay in our launch of ZEVASKYN®.

We also manufactured a batch of ZEVASKYN® following patient biopsy collection in July 2026 that, despite being bonafide drug product, could not be released because a cellular identity test, mandated by the FDA as a release assay during the final stage of BLA review, was out of specification. The FDA authorized release of this batch of ZEVASKYN® to the treatment site under a single-patient IND, but would not authorize release of this batch as commercial product. We are currently working with the FDA toward revising the specification for this test, but we cannot guarantee that the FDA will accept our proposed revision. Additional or similar issues associated with manufacturing and testing can have an adverse impact on our business, financial condition, cash flow, and results of operations.

There are several risks specific to the manufacturing process for ZEVASKYN® that require close attention. As an autologous product there are challenges associated with viability of biopsies as an incoming material. Due to variables such as the fragility of RDEB skin and site of the biopsy, initiation of autologous keratinocyte growth and expansion can be challenging or may be extended beyond the scheduled timing. Another concern during manufacturing is the slowing of cell proliferation, resulting in extended manufacturing time. If pre-release criteria are not met, the production process must be stopped, and a new biopsy must be obtained. If release criteria are out of range, epidermal sheets must be discarded and the manufacturing process must be repeated.

(c) The following table provides information about purchases of equity securities that are registered pursuant to Section 12 of the Exchange Act for the quarter ended September 30, 2026: