Medicus Pharma clears FDA review for SkinJect Gorlin Syndrome trial

Medicus Pharma has received FDA authorisation to initiate a registrational Phase 2b study of its SkinJect microneedle patch in patients with Gorlin Syndrome, a rare genetic disorder characterised by the lifelong development of multiple basal cell carcinomas. The "Study May Proceed" letter, issued on 23 July 2026, follows the agency's safety review of the company's IND application for the SKNJCT-005 protocol.

SkinJect (D-MNA) is a dissolvable microneedle-array patch that delivers doxorubicin directly into BCC lesions, aiming to achieve localised tumour clearance while minimising systemic exposure. Earlier studies, including the 90-patient SKNJCT-003 study, reported 64% clinical clearance and 55% histological clearance after three treatments with the 200-mcg dose at day 57. The registrational study is expected to enrol up to 50 patients across multiple centres.

Protocol changes following FDA feedback

The FDA used its review to request several protocol amendments before issuing clearance. Medicus agreed to require baseline biopsy confirmation of nodular BCC for all participants, to revise the primary efficacy endpoint to demand both clinical and histological clearance at a pre-specified timepoint, and to extend patient follow-up from 24 weeks to at least two years to assess durability of response. In-person investigator assessment of clearance will also be mandatory alongside histological confirmation.

The agency also flagged additional recommendations covering pharmacokinetic assessments, genetic confirmation of Gorlin Syndrome, independent data safety monitoring, and the future incorporation of randomised, blinded comparator-controlled designs to support substantial evidence of effectiveness. The company said these were not classified as clinical hold issues and that it intends to address them through a further protocol amendment.

Raza Bokhari, executive chairman and chief executive of Medicus, said the FDA's input "strengthens the registrational study by enhancing the scientific rigor of the efficacy assessments while further supporting the long-term evaluation of treatment durability."

Rare disease context and competitive landscape

Gorlin Syndrome is an autosomal dominant condition driven primarily by mutations in the Hedgehog signalling pathway. Affected individuals can develop dozens to more than 1,000 BCCs across a lifetime, frequently requiring repeated surgical excisions that accumulate scarring, functional impairment and psychosocial burden. Medicus estimates the US patient population at 6,000 to 12,000 individuals, with a substantially larger addressable population globally.

The standard of care for Gorlin-related BCCs currently relies heavily on surgery and field-directed treatments. Hedgehog pathway inhibitors such as vismodegib and sonidegib carry approval for advanced or metastatic BCC, and have been used off-label or under compassionate access in Gorlin patients, but tolerability and systemic side-effect profiles limit long-term use. A localised, lesion-directed approach such as SkinJect occupies a different therapeutic niche: it targets individual lesions without systemic drug load, which could make it complementary rather than directly competitive with oral Hedgehog inhibitors. If regulatory approval follows, the company is positioning SkinJect as potentially the first FDA-approved lesion-directed therapy specifically for this population.

The two-year follow-up requirement added by the FDA signals that the agency wants meaningful durability data before it accepts the package as NDA-enabling, which extends the timeline to a potential filing. For a 50-patient open-label study in a rare indication, investor attention will focus on whether the composite clearance endpoint generates a response rate persuasive enough to support approval on uncontrolled data alone, or whether the FDA's note on future randomised designs foreshadows a request for a confirmatory controlled trial.

Medicus said it will provide further updates on study initiation and enrolment timing as development progresses.