Krystal Biotech Reports Topline Results from GEM-3 Trial
Nov 29, 2021 By MarketDepth
Krystal Biotech, Inc. (NASDAQ: KRYS) has reported topline results from its GEM-3 trial, investigating beremagene geperpavec, as known as VYJUVEK for treatment of dystrophic Epidermolysis Bullosa. The primary endpoint of the trial evaluated total wound healing of topical VYJUVEK compared to placebo at six-month time points with statistical significance. VYJUVEK is the first of its kind, non-invasive topical and re-dosable gene therapy in development, and sole genetically corrective approach in treating dystrophic EB that has a successful double blinded Phase 3 trial.
“Dystrophic Epidermolysis Bullosa is referred to as ‘the worst disease you’ve never heard of’ because of the incredibly devastating reality that patients with this genetic condition face, and we are thrilled to announce positive results from our pivotal GEM-3 trial of VYJUVEKTM which showed that this topical gene therapy led to durable wound healing in dystrophic EB wounds. With these results in hand, we look forward to advancing discussions with regulatory authorities and will work quickly to bring this potential first-ever treatment to patients with dystrophic EB and their families who are in desperate need.”Suma Krishnan, Founder and Chief Operating Officer of Krystal
“Today’s positive B-VEC results represent the culmination of years of study on the molecular basis and genetic correction of this disease. Finally, dystrophic EB patients may have an easily administered genetically targeted therapy which has been shown to promote durable wound healing in this clinical trial. This is a long overdue milestone for patients living with this disease, and one that has potential to drastically change the treatment paradigm,” said Dr. Peter Marinkovich, M.D., Bullous Disease Clinic Director and Associate Professor of Dermatology at Stanford University.
Krystal plans to file a Biologics License Application with the U.S. Food and Drug Administration in the first half of 2022.