Phase III VIITAL study of EB 101 meets 2 primary endpoints in recessive dystrophic epidermolysis bullosa.- Abeona Therapeutics

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. In particular EB 101 significantly improved wound healing vs. control at six months, with wound healing level from baseline at 50% or greater seen in 81.4% Randomized Treated Wounds vs 16.3% Randomized Untreated Control Wounds. Other VIITAL study endpoints measuring proportion of wounds achieving 75% or greater wound healing and complete wound healing at six months also achieved statistical significance.

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). 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. Based on the positive topline results, Abeona intends to submit a Biologics License Application (BLA) for EB 101 to the FDA in the second quarter of 2023.