Phase III trials underway to treat severe skin disease Gorlin Syndrome

PellePharm, Inc.,announced it has dosed the first patient in a pivotal Phase III clinical trial of Patidegib Topical Gel, 2%, vs. vehicle gel in patients with Gorlin Syndrome . The Phase III study is intended to assess the safety and efficacy of patidegib topical gel for the reduction of disease burden of persistently developing basal cell carcinomas (BCCs) in Gorlin Syndrome, a rare genetic disease that leads to the chronic formation of multiple BCCs. Patients with this severe skin disease, for which there is no FDA-approved therapy, can suffer from tens to hundreds of BCC tumors during their lifetime and often must undergo repetitive and disfiguring surgeries to remove the tumors.

The randomized, double-blind, vehicle-controlled Phase III trial is evaluating the safety and efficacy of Patidegib Topical Gel 2% applied topically, twice daily to the face over 12 months vs. vehicle gel. Approximately 150 adults with Gorlin Syndrome will be randomized 1:1 to patidegib topical gel or vehicle. The primary efficacy endpoint is the number of new surgically eligible BCCs (nSEBs) that develop over the 12-month period in the two treatment arms. Secondary endpoints include the total number of new BCCs over 12 months, percentage of study participants developing greater than >2 facial nSEBs over 12 months; the percentage of participants developing greater than 1 facial nSEBs over 12 months; the number of nSEBs per participant over six and nine months; and change in the Advanced Basal Cell Carcinoma Index (aBCCdex) lesion scale score. An open-label safety and tolerability extension study is planned for at least 12 months for Phase III study completers following the end of the study.

Comment: In November 2018, PellePharm entered into a strategic development and commercialization collaboration with Danish dermatology specialists LEO Pharma to address unmet medical needs across various skin diseases with no approved treatments, including Gorlin Syndrome and High Frequency Basal Cell Carcinoma (HF-BCC), two rare forms of skin cancer. Under the agreement, LEO Pharma is providing resources to PellePharm to fund its pivotal Phase III trial of patidegib topical gel in patients with Gorlin Syndrome.