681 Development of a topical Hedgehog inhibitor for prevention of new surgically eligible basal cell carcinomas in patients with Gorlin Syndrome: Phase 3 trial

Identification of PTCH1 gene mutations in the Gorlin syndrome two decades ago led to the development of small molecule Hedgehog (HH) inhibitors that replace the function of the mutant gene. These drugs have remarkable oral efficacy against advanced BCCs and off-label vs. the multiple BCCs in patients with Gorlin syndrome. Unfortunately, on target, extra-cutaneous adverse effects cause patients to stop these drugs, after which BCCs in complete clinical and histologic remission generally recur. Therefore, PellePharm is developing a small molecule HH inhibitor for topical use.
Source: Journal of Investigative Dermatology - Category: Dermatology Authors: Tags: Pharmacology and Drug Development Source Type: research