Our Focus
PellePharm is focused on developing patidegib, an investigational topical treatment designed to mitigate the tumor burden in patients with Gorlin Syndrome and Basal Cell Carcinomas (BCCs), and other potential indications.
Our Approach
Patients suffering from Gorlin Syndrome and BCCs have a genetic mutation in the tumor suppressor gene encoding Patched1 (PTCH1), which acts as the primary inhibitor of the hedgehog signaling pathway.
Patidegib was developed to work by turning off the hedgehog signaling pathway. It is intended to chemically enhance a naturally occurring hedgehog pathway inhibitor by blocking the Smoothened signal, which allows cells to function normally and stop the overproduction of tumors found in patients with Gorlin Syndrome and BCCs.
Because oral formulations for this treatment typically have serious systemic side effects which cause many patients to discontinue treatment, patidegib is formulated as a topical gel to reduce its side effect profile due to its minimal systemic exposure.
