On September 21, 2026 Medicus Pharma Ltd. (NASDAQ: MDCX) ("Medicus" or the "Company"), a precision guided, biotech/life sciences company focused on advancing novel and potentially disruptive therapeutic assets, reported that it will focus future internally sponsored clinical development of SkinJect exclusively on Gorlin syndrome patients with multiple basal cell carcinoma lesions and conclude further development in sporadic/nodular basal cell carcinoma ("nBCC").

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The decision represents a further implementation of the Company’s capital-efficient portfolio strategy announced on September 8, 2026, under which Medicus is concentrating capital and organizational resources on programs where focused clinical development may offer the most compelling risk-adjusted opportunity. In that strategic update, the Company identified Gorlin syndrome, an autosomal dominant rare disease, presenting with multiple basal cell carcinoma lesions, as the strongest development opportunity for SkinJect.

The Company has now completed a comprehensive assessment incorporating findings from an independent third-party commercial assessment commissioned by the Company, clinical input from Babar K. Rao, MD, FAAD, Clinical Associate Professor of Dermatology at Weill Cornell Medical College and Principal Investigator of the SkinJect Phase 2 program (SKNJCT-003), findings from the final Phase 2 SkinJect Clinical Study Report (CSR) and feedback from the U.S. Food and Drug Administration’s Office of Orphan Products Development ("OOPD") regarding the Company’s requests for Orphan Drug Designation ("ODD") and Rare Pediatric Disease Designation ("RPDD").

Taken together, these inputs support Medicus’s decision to concentrate SkinJect clinical development and capital allocation exclusively on Gorlin syndrome opportunity.

Management Commentary

"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. Today’s decision applies that discipline directly to SkinJect," stated Dr. Raza Bokhari, Executive Chairman and CEO of Medicus. "Patients with Gorlin syndrome may face a lifetime of recurrent basal cell carcinomas and repeated procedures. We believe the FDA-authorized, NDA-enabling, Registrational SKNJCT-005 study provides us with a focused path to evaluate whether SkinJect can offer a meaningful lesion-directed treatment option for these patients, while we continue working with the FDA to address the issues identified in our ODD and RPDD designation requests."

FDA Feedback and Rare-Disease Strategy

In its feedback on the Company’s ODD and RPDD designation requests, the OOPD stated that it considers BCC occurring in Gorlin syndrome to be the same disease as sporadic BCC and noted that the overall prevalence of BCC exceeds the statutory prevalence threshold applicable to the designation requests. The OOPD further identified SkinJect’s prior clinical development in BCC outside of Gorlin syndrome as relevant to its assessment of whether the program could satisfy applicable orphan-subset requirements.

The FDA has not closed either regulatory pathway. The OOPD held further review of both requests in abeyance pending receipt of additional information from the Company and provided the Company an opportunity to respond.

Medicus believes that focusing future SkinJect clinical development exclusively on Gorlin syndrome is responsive to the issues identified by the OOPD regarding the Company’s prior development of D-MNA in BCC outside of Gorlin syndrome and provides a more coherent basis for the Company’s continued pursuit of ODD and RPDD designation. The Company intends to continue to engage with the FDA and submit additional information to the OOPD.

The OOPD also advised the Company that, irrespective of the status of the RPDD designation request, if Medicus believes SkinJect ultimately satisfies the statutory requirements for a Rare Pediatric Disease Priority Review Voucher ("PRV"), a voucher request may be included in the original marketing application together with the relevant FDA correspondence and supporting information.

If ODD is ultimately granted and applicable requirements are satisfied, potential benefits may include certain clinical-development tax incentives, exemption from certain FDA application user fees and up to seven years of U.S. orphan-drug exclusivity following approval. If SkinJect ultimately receives a qualifying approval and satisfies all statutory requirements under the RPDD program, the sponsor could potentially receive a transferable PRV.

There can be no assurance that the FDA will ultimately grant either of these designations.

FDA-Authorized, NDA-Enabling, SkinJect Gorlin Development Program

As previously disclosed on July 27, 2026, the FDA issued a "Study May Proceed" letter to the Company authorizing the initiation of its SKNJCT-005, NDA-enabling Phase 2b study of SkinJect in patients with Gorlin syndrome, an autosomal dominant rare disease, presenting with multiple BCC lesions.

The Gorlin program builds upon the Company’s completed SKNJCT-003 Phase 2 study, which identified the 200-µg doxorubicin-loaded dissolvable microneedle array ("D-MNA") patch as the proposed pivotal development dose.

Gorlin syndrome is characterized by the development of multiple and recurrent BCCs, which can result in substantial lifetime treatment burden from repeated surgery and other lesion-directed interventions. The Company believes SkinJect’s localized microneedle-array delivery approach has the potential, if approved, to provide a localized lesion-directed treatment option for this population. Approximately 11,000 patients in the United States with Gorlin syndrome are estimated to have active BCC, representing approximately 50,000 Gorlin-associated BCC procedures annually.

The Gorlin-focused strategy may also potentially support a differentiated reimbursement profile. A Company-commissioned 2026 independent commercial assessment incorporating interviews with physicians and payers modeled rare-disease-anchored net pricing of approximately $25,800 per treatment, an implied U.S. total addressable market of approximately $1.1 billion, and potential peak U.S. revenue of approximately $383 million. These estimates are non-risk-adjusted and dependent upon successful clinical development, regulatory approval, reimbursement and commercial adoption.

(Press release, Medicus Pharma, SEP 21, 2026, View Source [SID1234670972])