ABEONA THERAPEUTICS INC.

Current report

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

Current report

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.

Current report

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.

Current report

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.

Current report

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

Current report

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.

Current report

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.

Current report

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.

Current report

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.

Current report

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.

Current report

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.

Current report

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.

Current report

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.

Current report

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.