Medicus Pharma (NASDAQ:MDCX) said the US Food and Drug Administration (FDA) has issued a "Study May Proceed" letter authorizing the company to begin its NDA-enabling Phase 2b registrational study of SkinJect in patients with Gorlin syndrome who develop multiple basal cell carcinomas.
The authorization follows the FDA's review of the company's investigational new drug (IND) application for the SKNJCT-005 study and Medicus' response to information requests issued by the agency in June.
The Phase 2b, open-label, multicenter study will evaluate SkinJect 200 micrograms in up to 50 patients with Gorlin syndrome, a rare inherited disorder that causes the development of multiple basal cell carcinomas over a patient's lifetime. Medicus said the study is intended to generate efficacy and safety data to support a future New Drug Application.
SkinJect is a dissolvable microneedle-array patch designed to deliver doxorubicin directly into basal cell carcinoma lesions while limiting systemic drug exposure. According to Medicus, previous Phase 1 and Phase 2 studies showed a favorable safety profile and encouraging clinical and histological clearance rates.
The company cited results from its completed 90-patient SKNJCT-003 study, which reported 64% clinical clearance and 55% histological clearance after three treatments with the 200-microgram dose at Day 57.
The FDA recommended several changes to the study protocol, including requiring baseline biopsy confirmation of nodular basal cell carcinoma, revising the primary endpoint to require both clinical and histological clearance, conducting in-person investigator assessments alongside histological confirmation, updating the responder definition, and extending patient follow-up from 24 weeks to at least two years.
The agency also provided additional recommendations covering pharmacokinetic assessments, evaluation of used microneedle arrays, genetic confirmation of Gorlin syndrome, establishment of an independent Data Safety Monitoring Board, statistical methods, and future randomized, blinded comparator-controlled study designs. Medicus noted these recommendations were not identified as clinical hold issues and said it intends to submit an amended protocol incorporating the FDA's feedback.
Medicus CEO Dr Raza Bokhari described the FDA authorization as a significant regulatory milestone for the SkinJect program and said the agency's recommendations strengthen the registrational study and support the program's path toward a potential NDA submission.
"We believe these refinements position SkinJect to generate an even more robust data package as we advance toward a potential NDA submission for patients with Gorlin Syndrome, a population with substantial unmet medical need, who frequently face repeated surgeries throughout their lifetimes,” Dr Bokhari said.
“The ability to treat multiple lesions using a localized precision therapy represents an important advancement and what we believe could become a new standard of care for this patient population."
The company said it will provide further updates on study initiation and patient enrollment as development progresses.