PHILADELPHIA, PA — Medicus Pharma Ltd. (Nasdaq: MDCX) received U.S. Food and Drug Administration authorization to begin a registrational Phase 2b study of its SkinJect treatment in patients with Gorlin syndrome, moving the company closer to generating data that could support a future drug application.
The FDA issued a “Study May Proceed” letter dated July 23 after completing its safety review of the company’s investigational new drug application and assessing Medicus’ response to agency information requests submitted June 18.
The open-label, multicenter study, designated SKNJCT-005, is expected to enroll as many as 50 patients with Gorlin syndrome who develop multiple basal cell carcinomas and often require repeated surgical treatment.
SkinJect is a dissolvable microneedle-array patch designed to deliver 200 micrograms of the chemotherapy drug doxorubicin directly into individual tumors while limiting systemic exposure.
The company plans to use the trial to collect efficacy and safety data that could support a new drug application. SkinJect remains investigational and has not been approved by the FDA.
Medicus agreed to revise the study protocol following the FDA’s review, including adopting a primary endpoint that requires both clinical and histological clearance at a predetermined assessment point.
The amended protocol will also require biopsy confirmation of nodular basal cell carcinoma before enrollment, in-person clinical assessments, a responder definition aligned with the combined endpoint and patient follow-up lasting at least two years.
The extended follow-up is intended to measure the durability of treatment response, compared with the 24-week period initially proposed.
The FDA also recommended pharmacokinetic testing, genetic confirmation of Gorlin syndrome, analysis of used microneedle patches, establishment of an independent data safety monitoring board and revisions to the trial’s statistical methods.
The agency further recommended a standalone statistical analysis plan and the eventual use of randomized, blinded and comparator-controlled trial designs to support evidence of effectiveness. Those recommendations were not identified as clinical-hold issues, according to Medicus.
The company is evaluating the additional guidance and plans to submit an amended study protocol before providing further details on enrollment and trial initiation.
“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 stated.
Medicus cited results from a completed 90-patient study in which three treatments at the 200-microgram dose produced clinical clearance in 64% of treated lesions and histological clearance in 55% at Day 57.
Those results came from an earlier trial and do not establish that SkinJect is effective in patients with Gorlin syndrome.
Gorlin syndrome is a rare inherited disorder associated with mutations affecting the Hedgehog signaling pathway. Patients can develop dozens or hundreds of basal cell carcinomas beginning in childhood or early adulthood, frequently requiring repeated surgical removal.
The study protocol estimates the condition affects about one in 30,000 to 60,000 people worldwide, including approximately 6,000 to 12,000 people in the United States.
Medicus is developing SkinJect as a localized treatment intended to reduce reliance on repeated surgery while preserving surgical excision for lesions that fail to respond or otherwise require removal.
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