PHILADELPHIA, PA — Medicus Pharma (Nasdaq: MDCX) has advanced its SkinJect® treatment into registrational development for Gorlin Syndrome, a rare genetic disorder that causes recurring skin cancers and often requires patients to undergo repeated surgical procedures throughout their lives.
The biotechnology company said it submitted a Phase 2b clinical trial protocol to the U.S. Food and Drug Administration under its existing investigational new drug application, a step intended to generate data supporting a future New Drug Application for the therapy.
The move expands Medicus’ development strategy beyond conventional basal cell carcinoma treatment and into an orphan disease market where no FDA-approved lesion-directed therapies currently exist.
Gorlin Syndrome, also known as Nevoid Basal Cell Carcinoma Syndrome, is a hereditary disorder linked to mutations affecting the Hedgehog signaling pathway. Patients can develop dozens, hundreds, or in some cases more than 1,000 basal cell carcinomas over a lifetime, with tumors often appearing during childhood or adolescence.
The company estimates the condition affects approximately 6,000 to 12,000 people in the United States and a larger patient population internationally.
The planned Phase 2b study, designated SKNJCT-005, will enroll up to 50 patients with Gorlin Syndrome and multiple basal cell carcinomas. Researchers will evaluate whether SkinJect can achieve complete visual clearance in at least half of selected target lesions by Week 10.
Patients will receive treatment on two to four lesions simultaneously, reflecting the multi-lesion nature of the disease. The study includes follow-up through Week 24 to assess durability of response.
Medicus is pursuing the program as a potential orphan-drug opportunity that could provide an initial regulatory and commercial pathway for broader SkinJect development across non-melanoma skin cancer indications.
The company has already applied for FDA Orphan Drug Designation and said it plans to seek a Rare Pediatric Disease Priority Review Voucher before the end of the second quarter.
If granted, orphan-drug status could provide seven years of U.S. market exclusivity following approval and exempt the company from FDA application fees that currently exceed $5 million. A Rare Pediatric Disease Priority Review Voucher could accelerate regulatory review timelines and carries potential financial value because such vouchers can be sold or transferred.
The registrational program builds on data from Medicus’ completed Phase 2 study in nodular basal cell carcinoma. According to the company, patients treated with the 200-microgram dose achieved 64% clinical clearance and 55% histological clearance, with no treatment-related serious adverse events reported.
“This FDA submission marks an important milestone in our SkinJect strategy,” Chief Executive Officer Raza Bokhari said, adding that patients with Gorlin Syndrome frequently face repeated surgeries over their lifetimes.
The study will use blinded central review of clinical photographs along with lesion-level assessments, Reflectance Confocal Microscopy and Line-Field Optical Coherence Tomography to evaluate treatment outcomes.
The protocol was developed with input from dermatologic oncology specialists, including principal investigator Dr. Babar Rao, and representatives from the Gorlin Syndrome Alliance, according to the company.
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