Krystal Biotech, Inc. announced that results from the Phase 1 and 2 study of topical beremagene geperpavec (B-VEC) for the treatment of dystrophic epidermolysis bullosa (DEB) were published in Nature Medicine. The publication provides a comprehensive analysis of the data from the Phase 1 and 2 study showing that repeat topical applications of B-VEC were associated with durable wound closure, full-length cutaneous type VII collagen (COL7) expression, and anchoring fibril assembly with minimal reported adverse events. DEB is a rare and severe disease that affects the skin and mucosal tissues. It is caused by one or more mutations in the COL7A1 gene, which is responsible for the production of the protein COL7 that forms anchoring fibrils that bind the dermis (inner layer of the skin) to the epidermis (outer layer of the skin).

The lack of functional anchoring fibrils in DEB patients leads to extremely fragile skin that blisters and tears from minor friction or trauma. DEB patients suffer from open wounds, leading to skin infections, fibrosis that can cause fusion of fingers and toes, and ultimately an increased risk of developing an aggressive form of squamous cell carcinoma that, in severe cases, can be fatal. In the Phase 1 and 2 study, matched wounds were evaluated in nine RDEB patients receiving topical B-VEC or placebo repeatedly over 12 weeks.

Primary and secondary mechanistic and clinical endpoints were met. No Grade 2 or above B-VEC-related adverse events, vector shedding, or systemic drug exposure were noted.