PHILADELPHIA, PA — Medicus Pharma is ending further development of SkinJect for sporadic nodular basal cell carcinoma and concentrating its investment on an FDA-authorized Phase 2b study in Gorlin syndrome, narrowing the drug program as the company seeks a more focused clinical and regulatory path.
The decision directs internally sponsored SkinJect development exclusively toward patients with Gorlin syndrome who have multiple basal cell carcinoma lesions. Medicus characterized the move as part of the capital-allocation strategy it outlined Sept. 8, which prioritizes programs management believes offer stronger risk-adjusted development opportunities.
The shift follows a commercial assessment commissioned by Medicus, results from its completed Phase 2 SkinJect study, clinical input from the study’s principal investigator and feedback from the Food and Drug Administration’s Office of Orphan Products Development.
The FDA feedback presents a regulatory complication for Medicus as it pursues Orphan Drug Designation and Rare Pediatric Disease Designation for SkinJect.
The agency’s orphan-products office informed Medicus that it considers basal cell carcinoma occurring in Gorlin syndrome to be the same disease as sporadic basal cell carcinoma and noted that overall BCC prevalence exceeds the statutory threshold relevant to the designation requests.
The office also identified SkinJect’s previous clinical development in BCC outside Gorlin syndrome as relevant to determining whether the program could meet orphan-subset requirements.
Neither designation request has been closed. The FDA has held further review in abeyance while Medicus provides additional information, and the company intends to continue discussions with the agency.
“There can be no assurance that the FDA will ultimately grant either of these designations,” Medicus cautioned.
Medicus believes ending further SkinJect development in sporadic nodular BCC addresses issues raised by the FDA and provides a more coherent basis for pursuing the two rare-disease designations.
The FDA previously authorized Medicus to proceed with SKNJCT-005, an NDA-enabling Phase 2b study in Gorlin syndrome. The program builds on the completed SKNJCT-003 Phase 2 study, which identified a 200-microgram doxorubicin-loaded dissolvable microneedle-array patch as the proposed pivotal dose.
“Our September 8 strategic update established a clear principle for Medicus: we intend to concentrate capital where focused clinical development can create the greatest risk-adjusted value,” Executive Chairman and Chief Executive Officer Raza Bokhari stated.
SkinJect is designed to deliver doxorubicin locally through a dissolvable microneedle-array patch. Medicus is evaluating whether that approach can provide a lesion-directed treatment for Gorlin syndrome patients who can develop recurrent basal cell carcinomas and undergo repeated procedures over their lifetimes.
The company estimates about 11,000 U.S. patients with Gorlin syndrome have active basal cell carcinoma, accounting for approximately 50,000 procedures annually.
Medicus is also tying the narrower development strategy to a potential rare-disease pricing model. A company-commissioned 2026 commercial assessment involving physician and payer interviews modeled net pricing of approximately $25,800 per treatment, an implied U.S. total addressable market of about $1.1 billion and potential peak U.S. revenue of roughly $383 million.
Those projections are not risk-adjusted and depend on successful clinical development, regulatory approval, reimbursement and commercial adoption.
If Orphan Drug Designation is ultimately granted and applicable requirements are met, SkinJect could qualify for benefits including certain clinical-development tax incentives, exemption from some FDA application user fees and as much as seven years of U.S. orphan-drug exclusivity following approval.
Medicus is also continuing to pursue Rare Pediatric Disease Designation. The FDA advised the company that regardless of the pending designation request, Medicus could include a request for a Rare Pediatric Disease Priority Review Voucher with an eventual marketing application if it believes SkinJect satisfies the statutory requirements.
A qualifying approval would still have to meet all applicable requirements for Medicus to receive a transferable priority review voucher.
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