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New England Journal of Medicine Publishes Phase 3 Data on B-VEC in Patients with Dystrophic Epidermolysis Bullosa
PITTSBURGH, Dec. 14, 2022 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a biotechnology company focused on developing and
About this update from Krystal Biotech, Inc.
[{"type":"text","content":"PITTSBURGH, Dec. 14, 2022 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a biotechnology company focused on developing and commercializing genetic medicines for patients with rare diseases, announced today that data from the pivotal Phase 3 (GEM-3) trial of beremagene geperpavec (B-VEC) for dystrophic epidermolysis bullosa (DEB) have been published here in the New England Journal of Medicine (NEJM). The full manuscript, titled “Trial of Beremagene Geperpavec (B-VEC) for Dystrophic Epidermolysis Bullosa,” will appear in the December 15, 2022 issue of the NEJM. In this GEM-3 trial of 31 patients, complete wound healing at 6 months occurred in 67.4% of B-VEC wounds compared to 21.6% for placebo (difference, 45.8 percentage points; 95% confidence interval [CI], 23.6 to 68.0; p=0.002). Complete wound healing at 3 months occurred in 70.6% of the wounds exposed to B-VEC as compared with 19.7% of those exposed to placebo (difference, 51.0 percentage points; 95% CI, 29.3 to 72.6; p=0.0005). “The impressive phase 3 results with B-VEC are the best we have seen to date in patients with DEB and, if approved, B-VEC provides hope for these patients suffering through debilitating and potentially life-threatening symptoms associated with the disease, ” said Peter Marinkovich, M.D., Director of the Blistering Disease Clinic at Stanford Health Care, Associate Professor of Dermatology at the Stanford University School of Medicine, primary investigator of the GEM-3 trial and primary author of the manuscript. The GEM-3 trial was a randomized, double-blind, intra-patient placebo-controlled multi-center trial designed to evaluate the efficacy and safety of B-VEC for the treatment of DEB. In the trial, matched wounds receiving topical B-VEC or placebo were evaluated in 31 DEB patients over 26 weeks. The pivotal GEM-3 trial met its primary endpoint of complete wound healing at six-months and its secondary endpoint of complete wound healing at three-months. B-VEC was well tolerated, with no drug-related serious adverse events or discontinuations due to treatment. “B-VEC was developed by Krystal scientists as a potential first-in-class therapy for DEB,” said Suma Krishnan, President, Research & Development, Krystal Biotech. “We are working closely with the FDA to get B-VEC approved and deliver a meaningful benefit to patients wit...