Medicus Pharma Receives FDA Clearance To Launch Registrational Phase 2b SkinJect® Trial For Gorlin Syndrome

Medicus Pharma Ltd., a biotechnology and life sciences company focused on developing innovative therapeutic products, has announced that the U.S. Food and Drug Administration (FDA) has completed its safety review of the Investigational New Drug (IND) application for SKNJCT-005 and issued a "Study May Proceed" letter. The decision authorises the company to begin its registrational Phase 2b clinical trial evaluating SkinJect® (D-MNA) 200 mcg in patients with Gorlin Syndrome who develop multiple basal cell carcinomas (BCCs), marking an important step towards a potential regulatory submission.

The FDA's authorisation follows Medicus' submission of the SKNJCT-005 study protocol and its response to the agency's information requests issued in June 2026. The Phase 2b, open-label, multicentre trial is designed to enrol up to 50 patients with Gorlin Syndrome and aims to generate the safety and efficacy data needed to support a future New Drug Application (NDA). The study will evaluate SkinJect®, a proprietary dissolvable microneedle patch that delivers the chemotherapy drug doxorubicin directly into basal cell carcinoma lesions, allowing targeted treatment while minimising exposure to the rest of the body.

Commenting on the milestone, Dr. Raza Bokhari, Executive Chairman and Chief Executive Officer of Medicus Pharma, said the FDA's clearance represents a significant regulatory achievement for the SkinJect® programme. He noted that the agency's recommendations have strengthened the clinical study by improving the scientific assessment of treatment efficacy and extending the evaluation of long-term durability. He added that these refinements are expected to generate a stronger clinical data package as the company works towards a potential NDA submission for patients with Gorlin Syndrome, many of whom undergo repeated surgeries throughout their lives because of recurring skin cancers.

Previous Phase 1 and Phase 2 studies of SkinJect® demonstrated a favourable safety profile along with encouraging treatment results. Data from the completed 90-patient SKNJCT-003 study showed clinical clearance in 64% of treated lesions and histological clearance in 55% of lesions after three treatments using the 200 mcg dose. These findings provided the basis for advancing the therapy into its registrational Phase 2b study.

As part of its review, the FDA recommended several protocol enhancements to improve the quality of the study. These include requiring biopsy confirmation of nodular basal cell carcinoma before enrolment, revising the primary efficacy endpoint to include both clinical and histological clearance, conducting in-person investigator assessments, extending patient follow-up from 24 weeks to at least two years to assess long-term treatment durability, and refining statistical analyses. The agency also recommended additional pharmacokinetic assessments, genetic confirmation of Gorlin Syndrome, establishment of an independent Data Safety Monitoring Board and consideration of future randomised, blinded comparator-controlled studies. Medicus confirmed that these recommendations do not constitute clinical hold issues and plans to incorporate them into an amended study protocol before initiating the trial.

Gorlin Syndrome is a rare inherited genetic disorder that causes patients to develop multiple basal cell carcinomas from childhood or early adulthood, often resulting in repeated surgeries, permanent scarring and a significant impact on quality of life. The condition affects an estimated 1 in 30,000 to 60,000 people worldwide, with approximately 6,000 to 12,000 patients in the United States alone. SkinJect® is being developed as a minimally invasive, lesion-directed treatment designed to reduce the need for repeated surgical procedures while preserving surgery as an option when necessary. If approved, it has the potential to become the first FDA-approved lesion-directed therapy specifically developed for Gorlin Syndrome and the first microneedle-based chemotherapy platform for the treatment of basal cell carcinoma lesions.