PHILADELPHIA, PA — Medicus Pharma Ltd. received FDA clearance to begin a registrational Phase 2b trial of its SkinJect microneedle patch in patients with Gorlin Syndrome, advancing the treatment toward a potential marketing application while requiring stricter efficacy measures and at least two years of follow-up.
The Food and Drug Administration completed its safety review of the company’s investigational new drug application and issued a “Study May Proceed” letter dated July 23, Medicus stated.
The decision allows Medicus (Nasdaq: MDCX) to initiate SKNJCT-005, an open-label, multicenter study expected to enroll as many as 50 patients with Gorlin Syndrome who develop multiple basal cell carcinomas.
The FDA action permits the clinical trial to proceed but does not constitute approval of SkinJect or a determination that the treatment is effective.
SkinJect is a dissolvable microneedle-array patch designed to deliver 200 micrograms of doxorubicin directly into basal cell carcinoma lesions, limiting systemic exposure to the chemotherapy drug.
The study is intended to generate safety and efficacy data supporting a potential new drug application for patients who often undergo repeated surgeries to remove tumors.
Following its review, the FDA recommended that Medicus require biopsy confirmation of nodular basal cell carcinoma before enrollment and revise the trial’s primary endpoint to require both clinical and histological clearance at a predetermined time.
The agency also called for in-person assessments of clinical clearance, an updated responder definition and an extension of patient monitoring from 24 weeks to at least two years to evaluate whether responses are durable.
“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 stated.
The company agreed to incorporate those changes through an amended trial protocol.
The FDA also recommended additional pharmacokinetic testing, analysis of used microneedle arrays, genetic confirmation of Gorlin Syndrome, creation of an independent data safety monitoring board and revisions to the study’s statistical methodology.
Other recommendations included a standalone statistical analysis plan and the possible future use of randomized, blinded and comparator-controlled trials to establish substantial evidence of effectiveness.
The agency determined that those recommendations did not warrant placing the study on clinical hold, according to Medicus.
The protocol cites results from the company’s completed 90-patient SKNJCT-003 study, in which three treatments using the 200-microgram dose produced a 64% clinical clearance rate and a 55% histological clearance rate by Day 57. Those earlier findings have not established whether SkinJect will meet the revised composite endpoint in the new trial.
Gorlin Syndrome, also known as Nevoid Basal Cell Carcinoma Syndrome, is a rare inherited disorder associated with the development of multiple basal cell carcinomas beginning in childhood or early adulthood.
Affected patients may develop dozens or, in some cases, more than 1,000 tumors over their lifetimes, resulting in repeated surgical procedures, scarring and functional impairment, according to the trial protocol.
Medicus estimated the condition affects about one in 30,000 to 60,000 people worldwide, including roughly 6,000 to 12,000 patients in the United States.
The company has not disclosed when enrollment will begin. Medicus said it expects to provide additional information about the study’s initiation and recruitment schedule as development progresses.
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