PHILADELPHIA, PA — Medicus Pharma Ltd. (NASDAQ: MDCX) will end internally funded development of SkinJect for sporadic nodular basal cell carcinoma and concentrate its clinical spending on Gorlin syndrome, narrowing the biotech company’s program as it pursues a rare-disease regulatory strategy with the U.S. Food and Drug Administration.
The decision extends a capital-allocation strategy Medicus outlined Sept. 8 to concentrate resources on programs it considers to have the strongest risk-adjusted development opportunities.
Medicus based the shift on its final Phase 2 SkinJect clinical study report, an independent commercial assessment, clinical input from Phase 2 principal investigator Dr. Babar K. Rao and FDA feedback on requests for Orphan Drug Designation and Rare Pediatric Disease Designation.
The regulatory feedback presents a hurdle for that strategy. The FDA’s Office of Orphan Products Development 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 applicable to the company’s designation requests.
The agency also identified SkinJect’s previous development in BCC outside Gorlin syndrome as relevant to determining whether the program could satisfy 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 plans to continue discussions with the agency.
“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. “Today’s decision applies that discipline directly to SkinJect.”
Medicus believes ending future internally sponsored SkinJect development outside Gorlin syndrome addresses issues raised by the FDA and provides a clearer basis for continuing to seek the two rare-disease designations. The company cautioned that there is no assurance either will ultimately be granted.
The FDA has separately authorized Medicus to begin SKNJCT-005, an NDA-enabling Phase 2b study of SkinJect in Gorlin syndrome patients with multiple basal cell carcinoma lesions. The agency issued a “Study May Proceed” letter in July.
That 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 development dose.
Gorlin syndrome is an autosomal dominant rare disease associated with multiple and recurrent basal cell carcinomas. Medicus estimates about 11,000 U.S. patients with Gorlin syndrome have active BCC, accounting for approximately 50,000 related procedures annually.
SkinJect uses a dissolvable microneedle array to deliver doxorubicin locally to individual lesions. Medicus is evaluating whether that approach could reduce the treatment burden associated with repeated surgeries and other lesion-directed interventions.
The company’s commercial assumptions indicate potentially significant economics if the program succeeds, though those projections remain contingent on clinical, regulatory and market outcomes.
A company-commissioned 2026 assessment modeled net pricing of about $25,800 per treatment, an implied U.S. total addressable market of approximately $1.1 billion and potential peak U.S. revenue of about $383 million. Medicus characterized those estimates as non-risk-adjusted and dependent on successful development, regulatory approval, reimbursement and commercial adoption.
Orphan Drug Designation, if granted and followed by an eligible approval, could provide 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.
The Rare Pediatric Disease program could potentially provide a transferable Priority Review Voucher if SkinJect ultimately receives a qualifying approval and meets statutory requirements. The FDA advised Medicus that regardless of the status of its current designation request, the company could seek a voucher with an eventual marketing application if it believes SkinJect satisfies those requirements.
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