Krystal Biotech Announces Completion of the GEM-3 Pivotal Phase 3 Study Evaluating B-VEC for the Treatment of Dystrophic Epidermolysis Bullosa
- Topline results anticipated in the fourth quarter of 2021
- Of the 31 patients enrolled in the trial, 29 patients completed the study with no missing visits, including the three primary endpoint assessment visits
PITTSBURGH, Oct. 26, 2021 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc., (“Krystal”) (NASDAQ: KRYS), the leader in redosable gene therapies for rare diseases, today announced that the last participant has completed the 26-week dosing period and 30-day safety follow up visit in the GEM-3 study, Krystal’s pivotal Phase 3 clinical trial evaluating investigational beremagene geperpavec (B-VEC) as a topical gene therapy for the treatment of dystrophic epidermolysis bullosa (DEB). DEB is a rare, genetic skin disease that causes skin to tear or blister from minor contact and is caused by a lack of functional type VII collagen (COL7) protein in the skin.
“We want to express our gratitude to the trial participants, their families, the clinical investigators and their coordinators for their participation and commitment that enabled us to complete this study in a timely manner during such a challenging year,” said Suma Krishnan, Chief Operating Officer of Krystal Biotech. “We remain on track to announce topline data from the trial this quarter, and if positive, we will work expeditiously to complete regulatory filings with the FDA and EMA with the goal of bringing a topical, genetically corrective therapy to patients living with DEB.”
About the GEM-3 Pivotal StudyThe GEM-3 trial is a randomized, double-blind, intra-patient placebo-controlled study designed to evaluate the efficacy and safety of B-VEC for the treatment of DEB. The trial enrolled 31 patients ranging in ages from one (1) year to forty-four (44) years old. Sixty-one percent (61%) of the patients enrolled were pediatric patients (18 years old or younger). Less than ten percent (10%) of enrolled patients have the dominant form of dystrophic epidermolysis bullosa (DDEB). Of the 31 patients enrolled in the trial, 29 patients completed the study with no missing visits, including the three primary endpoint assessment visits.
In each patient, a primary wound pair was identified by the investigator; one wound was randomized to receive a weekly topical application of B-VEC and the other a placebo. Primary wound pairs selected in the study included all three wound area segments of
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