Medicus Pharma Announces U.S. Food and Drug Administration (FDA) Submission of Rare Pediatric Disease Designation Request for SkinJect® in Gorlin Syndrome

PHILADELPHIA, (ZEEST MEDIA) — Medicus Pharma Ltd. (NASDAQ: MDCX) (“Medicus” or the “Company”), a biotech/life sciences company focused on advancing the clinical development programs of novel and potentially disruptive therapeutic assets, today announced the submission of a Rare Pediatric Disease Designation (“RPDD”) request to the U.S. Food and Drug Administration (“FDA”) for SkinJect®, the Company’s investigational doxorubicin-containing microneedle array patch (D-MNA), for the treatment of basal cell carcinoma (“BCC”) in patients with Gorlin Syndrome, also known as Nevoid Basal Cell Carcinoma Syndrome.

The RPDD submission was made pursuant to Section 529 of the Federal Food, Drug, and Cosmetic Act and follows the Company’s previously announced Orphan Drug Designation (“ODD”) application (DRU-2026-11578), and Registrational study design (SKNJCT-005) which remains under FDA review. The design of the Company’s Gorlin Syndrome development program has been informed through engagement with clinical experts and the Gorlin Syndrome Alliance, a patient advocacy group focused on the needs and priorities of this unique patient population.

“Patients with Gorlin Syndrome often face a lifetime of recurring surgeries and progressive treatment burden beginning at a young age,” said Dr. Raza Bokhari, Medicus’ Executive Chairman and CEO, “SkinJect® is designed to provide localized treatment directly to the lesion and our recently concluded Phase 2 study has shown positive results, with 64% clinical clearance (CC) and 55% complete response (CR). We believe it has the potential to become an important non-surgical treatment option for these patients. The submission of our Rare Pediatric Disease Designation request represents another meaningful milestone in our efforts to advance SkinJect® through the regulatory process and address a significant unmet medical need.”

Gorlin Syndrome is a rare autosomal dominant genetic disorder caused primarily by mutations affecting the Hedgehog signaling pathway. Patients may develop dozens, hundreds, or even more than one thousand basal cell carcinomas during their lifetime. These lesions often appear during early childhood and adolescence and continue to develop throughout life. The condition is estimated to affect approximately 1 in 30,000 to 60,000 individuals worldwide, corresponding to an estimated U.S. patient population of approximately 6,000 to 12,000 individuals and a substantially larger addressable population across major global markets.

SkinJect® is a proprietary dissolvable microneedle array designed to deliver doxorubicin (D-MNA) directly into nodular basal cell carcinoma lesions through localized intradermal administration. The technology is intended to achieve high drug concentrations at the tumor site while minimizing systemic exposure and associated toxicities.

In the Company’s recently completed Phase 2 study, the 200ug D-MNA treatment arm demonstrated the strongest rates of clinical and histological clearance of 64% and 55% respectively, among evaluable nodular basal cell carcinoma patients, while maintaining a favorable safety and tolerability profile.

The Company believes the clinical profile observed to date supports the continued evaluation of SkinJect® as a repeatable, lesion-directed therapy for patients who may develop numerous BCC lesions over the course of their lifetime.

If granted, Rare Pediatric Disease Designation would further support the Company’s regulatory strategy for SkinJect® in Gorlin Syndrome. Subject to compliance with applicable statutory requirements and future FDA approval of a marketing application for the designated indication, the program may provide eligibility for a Rare Pediatric Disease Priority Review Voucher. Such a voucher may be used to obtain priority review of a future marketing application or transferred to another company.

A Priority Review Voucher, if awarded and utilized, may reduce FDA review time for a future New Drug Application from approximately ten months under standard review to approximately six months under priority review, potentially accelerating patient access to innovative therapies and shortening the time required to bring new treatments to market.

In addition, if Orphan Drug Designation is granted and a future marketing application is approved, SkinJect® may become eligible for seven years of U.S. market exclusivity for the designated indication, waiver of certain FDA application fees, and additional regulatory incentives intended to encourage development of therapies for rare diseases.

The Company believes that Gorlin Syndrome represents a compelling development opportunity for SkinJect® given the significant unmet medical need, the recurring nature of the disease, the lack of approved therapies for pediatric patients, and the potential for a lesion-directed, non-surgical treatment approach that may reduce dependence on repeated surgical procedures. The Company further believes SkinJect® may be uniquely positioned to address both pediatric and adult manifestations of Gorlin Syndrome through a repeatable, lesion-directed treatment approach designed to manage newly emerging lesions throughout the course of the disease.

Medicus intends to continue engaging with the FDA regarding the clinical development pathway for SkinJect® in Gorlin Syndrome while advancing broader development opportunities in basal cell carcinoma.

For further information contact:

Carolyn Bonner, President and Chief Financial Officer
(610) 636-0184
cbonner@medicuspharma.com

Anna Baran-Djokovic, SVP Investor Relations
(305) 615-9162
adjokovic@medicuspharma.com

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