Abeona Therapeutics Inc. announced positive topline data from its pivotal Phase 3 VIITAL study assessing the safety and efficacy of EB-101 for the treatment of patients with recessive dystrophic epidermolysis bullosa (RDEB). The VIITAL study met its two co-primary efficacy endpoints demonstrating statistically significant, clinically meaningful improvements in wound healing and pain reduction in large chronic RDEB wounds. All Evaluated Endpoints Successfully Achieved The pivotal Phase 3 VIITAL study evaluated the efficacy, safety and tolerability of EB-101 in 43 large chronic wound pairs in 11 subjects with RDEB.

The large chronic wounds randomized and treated in VIITAL measured greater than 20 cm(2) of surface area and had remained open for a minimum of six months and a maximum of 21 years (mean 6.2 years). The co- primary endpoints of the study were: 1) the proportion of RDEB wound sites with greater than or equal to 50% healing from baseline, comparing randomized treated with matched untreated (control) wound sites at the six-month timepoint, as determined by direct investigator assessment; and 2) pain reduction associated with wound dressing change assessed by the mean differences in scores of the Wong-Baker FACES scale between randomized treated and matched untreated (control) wounds at the six-month timepoint. The study allowed for wounds not included in the randomized primary efficacy analysis to receive EB-101 treatment (n=14 non-randomized wounds).

The tables below summarize the topline primary efficacy results: Post-Hoc Analysis of Pain Data In addition to meeting the co-primary pain endpoint, in a post-hoc analysis of the EB-101 treated severe wounds (baseline pain score of 6 or greater), including randomized and non-randomized (n=27), a mean pain reduction from baseline at six months of 5.70 was observed, as compared to a mean pain reduction of 3.51 for all treated randomized and non-randomized wounds for which pain was evaluated (n=53). Safety Results EB-101 was shown to be well-tolerated with no serious treatment-related adverse events observed, consistent with past clinical experience. There were no deaths or instances of positive replication-competent retrovirus (RCR) results, and no systemic immunologic responses were reported during the study, as well as no squamous cell carcinoma (SCC) at treatment sites after application of EB-101.

Two subjects reported at least one serious adverse event (SAE) unrelated to EB-101. Four subjects reported related treatment emergent adverse events (TEAEs), including procedural pain, muscle spasms and pruritis. Infections unrelated to EB-101 were observed in eight patients.

Abeona anticipates submitting results from this study, including further details with additional exploratory endpoints and the Week 12 results, for presentation at future medical meetings and for publication in a peer-reviewed journal. The Week 12 results are similar to Week 24 results, achieving statistical significance for pain reduction and wound healing at all levels. Jean Tang, M.D., Ph.D., Professor of Dermatology, Stanford University School of Medicine and Principal Investigator of the EB-101 pivotal Phase 3 VIITAL study said, "Large chronic RDEB wounds are the toughest to treat and often associated with intense chronic pain that significantly impacts the quality of life of RDEB patients, necessitating frequent use of opioids.

In the Phase 3 VIITAL study, EB-101 has been shown to both heal such large chronic wounds and significantly reduce pain. And continue to see durable clinical benefit of EB-101 with up to eight years of follow-up in Phase 1/2a study. Next Steps Based on the positive topline results, Abeona intends to submit a Biologics License Application (BLA) for EB-101 to the U.S. Food and Drug Administration (FDA) in the second quarter of 2023.

EB-101 has been granted Orphan Drug and Rare Pediatric Disease (RPD) designations by the FDA. Among the benefits of Orphan Drug designation are seven years of market exclusivity following FDA approval, potentially preventing FDA approval of another product deemed to be the same as the approved product for the same indication, waiver of application fees, and tax credits for clinical testing expenses conducted after orphan designation is received. A sponsor who receives an approval for a BLA with RPD designation may qualify for a Priority Review Voucher (PRV), subject to final determination by the FDA.

The PRV can be used to receive an expedited review process of a subsequent marketing application for a different product or sold to another company.