Medicus Pharma (NASDAQ:MDCX) announced that it has submitted Protocol SKNJCT-005 to the US Food and Drug Administration (FDA) under its existing Investigational New Drug (IND) application for SkinJect, advancing the therapy into registrational development for patients with Gorlin Syndrome, also known as Nevoid Basal Cell Carcinoma Syndrome (NBCCS).
The company said the protocol outlines a Phase 2b, open-label study intended to generate efficacy and safety data that could support a future New Drug Application (NDA) for SkinJect in the rare inherited disorder.
According to Medicus, the submission expands the SkinJect clinical program into an orphan disease indication with limited treatment options and no FDA-approved lesion-directed therapies specifically developed for Gorlin Syndrome.
Gorlin Syndrome is characterized by the development of multiple basal cell carcinomas and recurrent skin cancers, often requiring repeated surgical procedures throughout a patient's life.
The condition is estimated to affect approximately one in 30,000 to 60,000 individuals worldwide, which Medicus estimates translates to roughly 6,000 to 12,000 patients in the United States.
"This FDA submission marks an important milestone in our SkinJect strategy to develop what we believe could become the first FDA-approved lesion-directed therapy for patients with Gorlin Syndrome," Medicus CEO Dr Raza Bokhari said in a statement.
“Patients with Gorlin Syndrome frequently face repeated surgeries throughout their lifetimes. The ability to treat multiple lesions simultaneously using a localized precision therapy, while potentially preserving cosmetic outcomes represents an important advancement and what we believe could become a new standard of care for this patient population.”
The proposed SKNJCT-005 trial will enroll up to 50 patients with Gorlin Syndrome and multiple basal cell carcinomas. The study's primary endpoint is the proportion of patients achieving complete clinical clearance in at least 50% of selected target lesions by Week 10, based on blinded independent central review of standardized clinical photographs.
Under the study design, patients may receive treatment across two to four target lesions simultaneously. Each lesion will receive three applications of SkinJect administered on Days 1, 8 and 15, with an optional fourth treatment on Day 22 if visual clearance has not been achieved. Participants will be monitored through Week 24 to assess durability of response.
The company said the protocol includes blinded central review, lesion-level assessments, standardized photography, Reflectance Confocal Microscopy (RCM), Line-Field Optical Coherence Tomography (LF-OCT), and follow-up evaluations intended to support potential regulatory registration.
As part of its regulatory strategy, Medicus said it applied for FDA Orphan Drug Designation for SkinJect in April 2026 and plans to submit an application for a Rare Pediatric Disease Priority Review Voucher before the end of the second quarter of 2026.
The company believes the treatment may qualify for both programs because Gorlin Syndrome affects fewer than 200,000 people in the United States and often manifests during childhood.
If granted, Orphan Drug Designation could provide regulatory incentives including fee waivers and market exclusivity following approval. A Rare Pediatric Disease Priority Review Voucher, if awarded, could potentially accelerate FDA review timelines.
The registrational study builds on results from the company's completed Phase 2 SKNJCT-003 trial in nodular basal cell carcinoma.
Medicus previously reported that the 200-microgram doxorubicin-containing microneedle array (D-MNA) cohort achieved 64% clinical clearance and 55% histological clearance at Day 57, with no treatment-related serious adverse events reported.
Shares of Medicus were up nearly 11% on Wednesday morning in New York.