PHILADELPHIA, PA — Medicus Pharma Ltd. (NASDAQ: MDCX) has submitted a registrational study protocol to the U.S. Food and Drug Administration for its SkinJect therapy in Gorlin syndrome, a rare genetic disorder that causes patients to develop recurring basal cell carcinomas throughout their lives, expanding the company’s development strategy into an orphan disease market with limited treatment options.
The protocol, designated SKNJCT-005, outlines a Phase 2b open-label study intended to generate data that could support a future New Drug Application for SkinJect in patients with Gorlin syndrome, also known as Nevoid Basal Cell Carcinoma Syndrome.
The submission marks a regulatory milestone for the company as it seeks to establish an initial commercial pathway for SkinJect through a rare-disease indication while pursuing broader applications in non-melanoma skin cancers.
Gorlin syndrome is an inherited disorder linked to abnormalities in the Hedgehog signaling pathway. Patients can develop dozens, hundreds or even thousands of basal cell carcinomas over their lifetimes, often beginning in childhood or adolescence.
Medicus estimates the condition affects approximately 6,000 to 12,000 people in the United States.
Unlike conventional treatment approaches that frequently rely on repeated surgical procedures, the proposed study is designed to evaluate whether SkinJect can clear multiple lesions simultaneously using a localized drug-delivery approach.
The trial is expected to enroll up to 50 patients with Gorlin syndrome and multiple basal cell carcinomas.
The primary endpoint will measure the proportion of patients achieving complete visual clearance in at least 50% of selected target lesions after 10 weeks of treatment. Patients will be followed through 24 weeks to assess durability of response.
The study incorporates blinded independent review of standardized clinical photography, along with lesion assessments using reflectance confocal microscopy and optical coherence tomography.
Medicus previously applied for FDA Orphan Drug Designation for SkinJect in April and plans to seek Rare Pediatric Disease designation before the end of the second quarter.
If granted, orphan designation could provide development incentives including waiver of certain FDA application fees and seven years of market exclusivity following approval. A Rare Pediatric Disease Priority Review Voucher could potentially accelerate review timelines or be sold to another drug developer.
Chief Executive Officer Raza Bokhari said the company believes SkinJect could become the first FDA-approved lesion-directed therapy developed specifically for Gorlin syndrome patients.
The registrational program builds on data from the company’s earlier Phase 2 study in nodular basal cell carcinoma. According to Medicus, the 200-microgram treatment cohort demonstrated 64% clinical clearance and 55% histological clearance, with no treatment-related serious adverse events reported.
For Medicus, success in Gorlin syndrome could provide an initial regulatory and commercial foothold for SkinJect while creating a foundation for expansion into broader skin cancer indications, a significantly larger market opportunity.
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