Pivotal Phase 3 clinical trial is planned to commence in early 2018
EB-101 gene therapy for patients with Recessive Dystrophic Epidermolysis Bullosa (RDEB) has demonstrated promising efficacy and safety in the Phase 1/2 clinical trial
EB-101 has received Orphan Drug and Rare Pediatric Disease Designations in the USA and Orphan Drug Designation in the EU
NEW YORK and CLEVELAND, July 18, 2017 (GLOBE NEWSWIRE) — Abeona Therapeutics Inc. (NASDAQ:ABEO), a leading clinical-stage biopharmaceutical company focused on developing novel gene therapies for life-threatening rare diseases, announced today guidance from a recent Type-C meeting with the FDA which has recommended accelerating the EB-101 program into a pivotal Phase 3 trial. The Company continues to engage the FDA on the final Phase 3 clinical trial design, planned to commence early 2018, and will provide an update on the program in the coming months.
“The FDA guidance is an important milestone in our clinical development plan for EB-101, and we are pleased to be moving forward into a registrational Phase 3 clinical study in 2018. Abeona is committed to advancing innovative gene therapies that address the unmet needs of patients suffering with dystrophic epidermolysis bullosa, a devastating rare skin disease. We are grateful that the FDA has recognized EB-101 as a rare disease product that addresses the underlying disease pathology to offer significant therapeutic benefit for RDEB patients, and we look forward to the collective work ahead in advancing this therapy,” stated Timothy J. Miller, Ph.D., President and CEO of Abeona Therapeutics Inc.
Abeona’s EB-101 product is an autologous, ex-vivo gene therapy in which the COL7A1 gene is inserted into a patient’s own skin cells (keratinocytes) for the treatment of the underlying disease in Recessive Dystrophic Epidermolysis Bullosa. The EB-101 program has been granted Orphan Drug and Rare Pediatric Disease Designations from the US Food and Drug Administration (FDA) and Orphan Drug Designation from the European Medicines Agency (EMA).
“EB Research Partnership (EBRP), along with EB Medical Research Foundation, are honored to have helped support EB-101 development by the dedicated researchers at Stanford University. Their tireless efforts in combination with Abeona’s leadership is driving real progress in the RDEB patient community. EBRP is encouraged by clinical results to date and looks forward to realizing the promise of EB-101 in addressing the devastating effects on RDEB patients’ quality of life and disease burden,” stated Alexander Silver, co-founder and chairman EB Research Partnership.
About EB-101 Phase 1/2 Clinical Trial:
In the Phase 1/2 …