Clinical Data Krystal Biotech says GEM-3 trial met primary endpoint, secondary endpoint 07:16 KRYS Krystal Biotech announced topline results from the pivotal GEM-3 trial of investigational beremagene geperpavec, now known as VYJUVEK, for the treatment of dystrophic Epidermolysis Bullosa. The primary endpoint of the trial evaluated complete wound healing of topical VYJUVEK compared to placebo at six-month timepoints and met statistical significance. VYJUVEK 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 were enrolled and evaluable for safety and efficacy per the primary intent-to-treat analysis; 67% of wounds treated with VYJUVEK achieved the primary endpoint of investigator assessed complete wound healing at the six-month timepoints as compared to 22% of wounds treated with placebo. 71% of wounds treated with VYJUVEK achieved the secondary endpoint of investigator assessed complete wound healing at the three-month timepoints as compared to 20% of wounds treated with placebo: 51.0%. In an ad-hoc analysis, the trial also demonstrated a statistical difference between the active and placebo groups for wounds that demonstrated complete wound healing at both the three- and six-month timepoints VYJUVEK was well tolerated. No drug-related serious adverse events or discontinuations due to treatment were reported. One mild drug-related adverse event was reported during the trial. The immunogenicity profile of VYJUVEK was consistent with the prior GEM-1/2 study where we observed no meaningful change in anti-HSV-1 or anti-COL7 antibodies. Based on these results, Krystal intends to file a Biologics License Application with the U.S. Food and Drug Administration in the first half of 2022 as the first step in executing its global regulatory and commercialization strategy to bring this investigational therapy to patients in need. The Company expects to submit a Marketing Authorization Application in Europe shortly after the BLA. Exploration of the potential regulatory path forward in other geographies, including Japan, is underway. Krystal will continue to manufacture VYJUVEK using the commercial scale process at its in-house cGMP manufacturing facility, ANCORIS, which was designed to support potential launch. The Company is currently constructing its second, larger, facility ASTRA, which is expected to come on-line in 2022 to help support a potential global launch and the pipeline.