Abeona Therapeutics Inc. announced that additional data from its pivotal Phase 3 VIITAL™ study of EB-101 for recessive dystrophic epidermolysis bullosa (RDEB) has been accepted for an oral presentation at the inaugural International Societies for Investigative Dermatology (ISID) Meeting, being held May 10-13, 2023 in Tokyo, Japan. Abeona previously reported positive top-line efficacy and safety data from the VIITAL study in November 2022. Abstract number 806 entitled, “Results from VIITAL: A phase 3, randomized, intrapatient-controlled trial of an investigational collagen type VII gene–corrected autologous cell therapy, EB-101, for the treatment of recessive dystrophic epidermolysis bullosa (RDEB),” will be presented by Jean Tang, M.D., Ph.D., Professor of Dermatology, Stanford University School of Medicine and Principal Investigator of the VIITAL study during a session between 1:15-3:45 p.m. Japan Standard Time on May 11, 2023.

Recessive Dystrophic Epidermolysis Bullosa: Recessive dystrophic epidermolysis bullosa (RDEB) is a rare connective tissue disorder characterized by severe skin wounds that cause pain and can lead to systemic complications impacting the length and quality of life. People with RDEB have a defect in the COL7A1 gene, leaving them unable to produce functioning type VII collagen, which is necessary to anchor the dermal and epidermal layers of the skin. There is currently no approved treatment for RDEB.

EB-101: EB-101 is an autologous, engineered cell therapy currently being developed for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), a rare connective tissue disorder without an approved therapy. The pivotal Phase 3 VIITAL™ study is a randomized clinical trial that evaluated the efficacy, safety and tolerability of EB-101 in 43 large chronic wound pairs in 11 subjects with RDEB. Treatment with EB-101 involves using gene transfer to deliver the functional COL7A1 gene into a patient's own skin cells (keratinocytes and its progenitors) and transplanting those cells back to the patient.

EB-101 is being investigated for its ability to enable normal Type VII collagen expression and to facilitate wound healing. EB-101 has been granted Regenerative Medicine Advanced Therapy, Breakthrough Therapy, Orphan Drug and Rare Pediatric Disease designations by the U.S. FDA. Abeona produces EB-101 for the VIITAL study at its fully integrated gene and cell therapy manufacturing facility in Cleveland, Ohio.

EB-101 is an investigational product not yet approved by the FDA.