PellePharm reports Phase II data of patidegib for Gorlin Syndrome

PellePharm Inc. (Menlo Park, Calif.) reported top-line data from a Phase II trial in 17 patients with Gorlin Syndrome showing that twice-daily topical patidegib (saridegib, IPI-926) for 26 weeks led to a complete response in 12 tumors vs. 0 tumors for placebo.

The double-blind, U.K. trial’s co-primary endpoints are change from baseline to week 26 in diameter of basal cell carcinomas and safety. Secondary endpoints include size of central facial carcinomas, frequency of new carcinomas on the face and proportion of non-central facial carcinomas...