Krystal Biotech completes dosing in Phase 2 study of KB103
Krystal Biotech announces that four patients have been dosed in the GEM-Phase 2 study of KB103, a topical gene therapy for the treatment of dystrophic epidermolysis bullosa, or DEB. The study is being conducted in adult and pediatric populations to determine whether KB103 administered topically can safely and effectively promote healing of DEB patient wounds. In addition, the study will assess change from baseline in Investigator global assessments and patient reported outcomes, or PRO. This study, being conducted at Stanford University, is a single-center, randomized, placebo-controlled, intra-subject comparison of treatment and placebo wounds. Four patients are being treated in this study. Three chronic wounds are selected per patient; two wounds are randomized to KB103 and one to placebo. Patients will be on trial for approximately six months. The primary objective of this trial is to measure wound healing by imaging. Outcome measures include duration of wound closure relative to placebo, time to wound closure relative to placebo and change of wound surface area relative to baseline and placebo. Secondary outcome measures include change from baseline in the Investigator's Global Assessment score, change from baseline in PRO scales of severity and pain, level of collagen VII in KB103-administrated skin as measured by immunofluorescence and presence of anchoring fibrils as measured by immunoelectron microscopy.