The authorization lets the company begin an NDA-enabling trial in up to 50 Gorlin Syndrome patients after the FDA completed its safety review of IND SKNJCT-005.
Medicus Pharma said the FDA (U.S. Food and Drug Administration) has completed its safety review of the IND (Investigational New Drug application) for SKNJCT-005 and issued a "Study May Proceed" letter, clearing the company to start an NDA-enabling registrational Phase 2b study of SkinJect 200 mcg in patients with Gorlin Syndrome who present with multiple basal cell carcinomas. The open-label, multicenter trial is expected to enroll up to 50 patients and is aimed at generating efficacy and safety data to support registration in the rare disease population. SkinJect is a dissolvable microneedle-array patch that delivers doxorubicin directly into basal cell carcinoma lesions to provide localized treatment while limiting systemic exposure. Medicus said earlier Phase 1 and Phase 2 studies showed a favorable safety profile and encouraging clearance rates. The SKNJCT-003 study, which enrolled 90 patients, showed 64% clinical clearance and 55% histological clearance after three treatments with the 200-mcg dose at Day 57. The FDA's review led to protocol changes that Medicus said will strengthen the study, including mandatory baseline biopsy confirmation of nodular basal cell carcinoma, a revised primary endpoint requiring both clinical and histological clearance, in-person investigator assessment, an aligned responder definition, and follow-up extended from 24 weeks to at least two years to assess durability. In its July 23, 2026 "Study May Proceed" letter, the agency also recommended work on pharmacokinetic assessments, used microneedle-array evaluation, genetic confirmation of Gorlin Syndrome, a Data Safety Monitoring Board, statistical methods, a standalone Statistical Analysis Plan, and future randomized, blinded, comparator-controlled designs. Medicus said those recommendations were identified as not constituting clinical hold issues and that it plans to submit an amended protocol. The company said it will provide further updates on study initiation and enrollment timing as development advances. Medicus is positioning SkinJect for a disorder marked by lifelong development of multiple basal cell carcinomas, repeated surgeries and significant unmet need, and said the therapy could become the first FDA-approved lesion-directed treatment specifically developed for patients with Gorlin Syndrome if approved.