Krystal Biotech (KRYS) : First gene therapy for skin

• The first gene therapy that can be applied to the skin was able to heal the blistering wounds endured by children with a structural skin deficiency. In addition to being the first topical gene therapy, the treatment from Krystal Biotech would be the first gene therapy that can be repeated because it doesn’t trigger an immune response.

 

 

 

 

Krystal Biotech announces positive topline results from GEM-3 pivotal trial for VYJUVEK for treatment of dystrophic Epidermolysis Bullosa

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.

• 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.
• 71% of wounds treated with VYJUVEKTM achieved the secondary endpoint of investigator assessed complete wound healing at the three-month timepoints as compared to 20% of wounds treated with placebo.
• 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 .
• VYJUVEKTM 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 VYJUVEKTM (as measured by anti-HSV-1 and anti-COL7 antibodies) 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 (BLA) with the U.S. Food and Drug Administration (FDA) 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 (MAA) 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 VYJUVEKTM 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.