PHILADELPHIA, PA — Medicus Pharma Ltd. (NASDAQ: MDCX) received FDA authorization to begin an NDA-enabling Phase 2b study of SkinJect in patients with Gorlin Syndrome, advancing the company’s lead program toward a potential regulatory submission while adding more stringent efficacy and follow-up requirements to the trial.
The Food and Drug Administration issued a “Study May Proceed” letter for the SKNJCT-005 study after completing its safety review of Medicus’ Investigational New Drug application, according to the company. The authorization followed Medicus’ response to information requests the agency issued June 18.
The open-label, multicenter study is expected to enroll as many as 50 patients with Gorlin Syndrome, also known as Nevoid Basal Cell Carcinoma Syndrome, who develop multiple basal cell carcinomas requiring repeated treatment.
SkinJect is a dissolvable microneedle-array patch designed to deliver 200 micrograms of doxorubicin directly into basal cell carcinoma lesions while limiting systemic exposure.
The FDA review also prompted Medicus to revise the registrational protocol, including changing the primary efficacy endpoint to require both clinical and histological clearance at a prespecified time point.
The amended protocol will also require baseline biopsy confirmation of nodular basal cell carcinoma, in-person investigator assessment of clinical clearance and a responder definition aligned with the combined clinical and histological endpoint.
Patient follow-up will be extended from 24 weeks to at least two years to assess durability of response, according to the company.
The FDA’s July 23 letter also included recommendations covering pharmacokinetic assessments, evaluation of used microneedle arrays, genetic confirmation of Gorlin Syndrome, creation of an independent Data Safety Monitoring Board and refinement of the study’s statistical methodology.
The agency additionally recommended a standalone Statistical Analysis Plan and future use of randomized, blinded and comparator-controlled study designs to support evidence of effectiveness. Medicus said those recommendations were not identified by the FDA as clinical-hold issues.
The company said it plans to submit an amended SKNJCT-005 protocol incorporating the agency’s recommendations.
“Importantly, the FDA’s recommendations strengthen the registrational study by enhancing the scientific rigor of the efficacy assessments while further supporting the long-term evaluation of treatment durability,” Executive Chairman and Chief Executive Officer Raza Bokhari said.
Medicus cited results from its completed 90-patient SKNJCT-003 study showing 64% clinical clearance and 55% histological clearance at Day 57 following three treatments with the 200-microgram dose.
Gorlin Syndrome is a rare genetic disorder associated with the development of repeated basal cell carcinomas beginning in childhood or early adulthood. The company said affected patients may develop dozens to more than 1,000 lesions over their lifetimes and often undergo repeated surgical excisions.
Medicus estimates the disorder affects roughly one in 30,000 to 60,000 people worldwide, corresponding to an estimated U.S. population of about 6,000 to 12,000 patients.
The company is developing SkinJect as a lesion-directed treatment intended to reduce reliance on repeated surgery while preserving surgery as an option for lesions requiring definitive excision.
Medicus said it expects to provide additional information on study initiation and enrollment timing as development progresses.
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