Abeona Therapeutics Announces Completion of Patient Follow-up in Pivotal Phase 3 VIITAL™ Study of EB-101 in Patients with Recessive Dystrophic Epidermolysis Bullosa (RDEB)

Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced that the last patient has completed their 6-month follow-up visit in Abeonas pivotal Phase 3 VIITAL study of its investigational autologous, engineered cell therapy, EB-101, in patients with recessive dystrophic epidermolysis bullosa (RDEB).

Completion of the last patients 6-month follow-up visit marks a key milestone that enables us to report key findings from our Phase 3 VIITAL study of EB-101 in RDEB, said Vish Seshadri, Chief Executive Officer of Abeona. We thank the patients, their families, and the clinical investigators, and plan to report topline results from this pivotal study within the next month.

Abeona has been verifying and preparaing the data in real time to ensure that an efficient database lock can be completed within two to three weeks from the last patients final visit. Abeona expects the VIITAL study, if positive, to serve as the basis for seeking approval by the U.S. Food and Drug Administration (FDA) of EB-101 for the treatment of patients with RDEB.

About 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.

About EB-101

EB-101 is an autologous, engineered cell therapy currently being investigated in Abeonas pivotal Phase 3 VIITAL study for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), a rare connective tissue disorder without an approved therapy. The EB-101 VIITAL study is a randomized clinical trial with target enrollment of at least 10 to 15 RDEB patients with approximately 36 large, chronic wound sites treated in total. Treatment with EB-101 involves using gene transfer to deliver the COL7A1 gene into a patients 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. The U.S. FDA has granted Rare Pediatric Disease Designation for EB-101. 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.

About Abeona Therapeutics

Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing cell and gene therapies for serious diseases. Abeonas lead clinical program is EB-101, its investigational autologous, engineered cell therapy for recessive dystrophic epidermolysis bullosa is in Phase 3 development. The Companys development portfolio also features AAV-based gene therapies for ophthalmic diseases with high unmet medical need. Abeonas novel, next-generation AAV capsids are being evaluated to improve tropism profiles for a variety of devastating diseases. Abeonas fully integrated cell and gene therapy cGMP manufacturing facility produces EB-101 for the pivotal Phase 3 VIITAL study and is capable of clinical and potential commercial production of AAV-based gene therapies.