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Krystal Biotech Announces Positive Topline Results from GEM-3 Pivotal Trial of VYJUVEK™ in Patients with Dystrophic Epidermolysis Bullosa
• Pivotal GEM-3 trial met its primary endpoint of complete wound healing at six-month timepoints, and its secondary endpoint of complete wound healing at
About this update from Krystal Biotech, Inc.
[{"type":"text","content":"• Pivotal GEM-3 trial met its primary endpoint of complete wound healing at six-month timepoints, and its secondary endpoint of complete wound healing at three-month timepoints VYJUVEKTM was well tolerated, with no drug-related serious adverse events or discontinuations Biologics License Application (BLA) on track to be submitted to U.S. Food and Drug Administration (FDA) in 1H22Conference call to discuss results scheduled for today, Monday, November 29, 2021 at 8:00 a.m. EST PITTSBURGH, Nov. 29, 2021 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc., (“Krystal”) (NASDAQ: KRYS), the leader in redosable gene therapies for rare diseases, today announced positive topline results from the pivotal GEM-3 trial of investigational beremagene geperpavec (B-VEC), now known as VYJUVEKTM, for the treatment of dystrophic Epidermolysis Bullosa (dystrophic EB). The primary endpoint of the trial evaluated complete wound healing of topical VYJUVEKTM compared to placebo at six-month timepoints and met statistical significance. VYJUVEKTM is the first non-invasive, topical and redosable gene therapy in development, and the only genetically corrective approach to treat dystrophic EB that has successfully completed a double blinded Phase 3 trial. Highlights of Topline Results from the GEM-3 Trial 31 patients (31 primary matched-wound pairs) were enrolled and evaluable for safety and efficacy per the primary intent-to-treat (ITT) analysis 67% of wounds treated with VYJUVEKTM achieved the primary endpoint of investigator assessed complete wound healing at the six-month timepoints as compared to 22% of wounds treated with placebo (absolute difference (95% CI): 45.8% (23.6%-68.0%); p","length":2520,"tagName":"div"}]