Abeona announces publication of long-term data from Phase 1/2a trial of EB-101
Abeona Therapeutics announced long term efficacy and safety results from its Phase 1/2a clinical trial evaluating EB-101, a gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa. Treatment with EB-101 resulted in sustained wound healing with a favorable safety profile at three years post-treatment. These long-term follow-up data in seven RDEB participants with 42 treated wounds were published in the peer-reviewed journal JCI Insight. Key Study findings include: Wounds selected for treatment were present for a mean of 11.2 years. Three years after treatment with EB-101, a majority of RDEB patients had sustained wound healing, with 80% of wounds achieving 50% healing, and 70% achieving greater than or equal to75%. Two years after treatment, only 1 of 6 untreated, prospectively selected control wounds, had greater than or equal to50% healing. 50% or greater wound healing was associated with no pain and no itch at treated sites three years post-treatment, compared with presence of pain in 53% and itch in 61% of wound sites at baseline. EB-101 was associated with long-term molecular expression of type VII collagen protein, which plays an important role in anchoring the dermal and epidermal layers of the skin. No serious treatment-related adverse events were observed during the three-year observation period. No replication competent virus was present at any time point. Researchers from Stanford University School of Medicine conducted the Phase 1/2a single-center, open-label clinical trial to evaluate the long-term wound healing and safety of EB-101 in seven adult patients with severe generalized RDEB and to assess patient-reported outcomes following treatment. Chronic open wounds, defined as wounds present and unhealed for at least 12 weeks, with a total area of at least 100 cm2, were required for enrollment. In the trial, gene-corrected EB-101 skin grafts were transplanted onto six wound sites in each of the seven adult participants and wounds selected for treatment had been present for a mean of 11.2 years. Participants were followed for two to five years after transplantation of EB-101 and received standard of care therapies including iron supplementation and esophageal dilations during the study. Abeona is currently continuing preparations for the pivotal Phase 3 VIITALTM Study evaluating EB-101 for the treatment of RDEB pending the anticipated receipt of Chemical, Manufacturing and Controls clearance from the U.S. Food and Drug Administration expected in Q4 2019.