PHILADELPHIA, July 27, 2026
Medicus Pharma Ltd. announced that the U.S. Food and Drug Administration (FDA) has issued a “Study May Proceed” letter following completion of its safety review of the company’s Investigational New Drug (IND) application for SKNJCT-005. The authorization allows Medicus to initiate its NDA-enabling Phase 2b registrational clinical trial evaluating SkinJect® (D-MNA) 200 mcg in patients with Gorlin Syndrome who develop multiple basal cell carcinomas (BCCs). The FDA’s decision follows the company’s submission of the study protocol and its response to the agency’s information requests issued in June 2026. According to Medicus, the regulatory milestone advances the development of SkinJect® as a potential first FDA-approved lesion-directed therapy specifically designed for patients with Gorlin Syndrome, a rare genetic disorder characterized by the repeated development of basal cell carcinomas throughout life.
FDA Recommendations Strengthen Registrational Study Design
The SKNJCT-005 study is designed as an open-label, multicenter Phase 2b registrational trial expected to enroll up to 50 patients with Gorlin Syndrome, also known as Nevoid Basal Cell Carcinoma Syndrome. Following its review, the FDA recommended several protocol enhancements intended to strengthen the scientific rigor of the study and support future regulatory review. These include requiring baseline biopsy confirmation of nodular basal cell carcinoma before enrollment, revising the primary efficacy endpoint to require both clinical and histological clearance, incorporating investigator-confirmed clinical assessments, refining responder definitions, and extending patient follow-up from 24 weeks to at least two years to evaluate long-term treatment durability. The agency also provided additional non-clinical-hold recommendations regarding pharmacokinetic assessments, statistical analysis, genetic confirmation of Gorlin Syndrome, establishment of an independent Data Safety Monitoring Board, and consideration of future randomized, comparator-controlled studies. Medicus stated that it plans to submit an amended study protocol incorporating these recommendations before initiating the trial.
SkinJect Demonstrates Encouraging Early Clinical Results
SkinJect® is a proprietary dissolvable microneedle-array patch that delivers doxorubicin directly into basal cell carcinoma lesions, enabling localized chemotherapy while minimizing systemic drug exposure. Previous Phase 1 and Phase 2 clinical studies demonstrated a favorable safety profile together with encouraging efficacy results. Data from the completed 90-patient SKNJCT-003 study showed 64% clinical clearance and 55% histological clearance after three treatments using the 200-mcg dose by Day 57. Chief Executive Officer Dr. Raza Bokhari said the FDA’s recommendations further strengthen the registrational study by improving efficacy assessments and long-term evaluation of treatment durability. He added that the enhanced protocol is expected to generate a more robust clinical data package to support a future New Drug Application (NDA) while addressing a significant unmet need among patients who often undergo repeated surgeries throughout their lives.
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Program Targets Significant Unmet Need in Rare Genetic Disease
Gorlin Syndrome is a rare autosomal dominant genetic disorder primarily caused by abnormalities in the Hedgehog signaling pathway, leading to the lifelong formation of multiple basal cell carcinomas beginning in childhood or early adulthood. Many patients develop dozens or even hundreds of tumors, requiring repeated surgical excisions that can result in significant scarring, functional impairment, and reduced quality of life. The disorder is estimated to affect approximately 1 in 30,000 to 60,000 individuals worldwide, representing an estimated 6,000 to 12,000 patients in the United States. SkinJect® is being developed as a minimally invasive, lesion-directed therapy intended to reduce dependence on repeated surgery while preserving surgical intervention for lesions requiring definitive treatment. With FDA authorization now secured and protocol refinements underway, Medicus Pharma continues advancing the program toward potential regulatory submission while expanding its pipeline of innovative therapies targeting oncology, rare diseases, and other areas of significant unmet medical need.
Source: Medicus Pharma press release
