Medicus Pharma Ltd. (NASDAQ: MDCX) has announced a strategic collaboration with the Gorlin Syndrome Alliance (GSA) to advance compassionate access to SKINJECT™, the company's investigational doxorubicin-containing microneedle arrays (D-MNA) for patients suffering from Gorlin Syndrome, also known as nevoid basal cell carcinoma syndrome.
Under the collaboration, Medicus and the GSA will jointly pursue the Expanded Access IND Program with the Food and Drug Administration (FDA) to allow patients with multiple, recurrent, or inoperable basal cell carcinomas (BCCs) to access SKINJECT™ under physician-supervised treatment protocols. The initiative aims to establish a framework for expanded access while collecting valuable real-world safety and tolerability data to inform future regulatory filings.
Addressing Critical Unmet Medical Need
Gorlin Syndrome is an inherited condition affecting approximately 1 in 31,000 people worldwide. Patients can develop hundreds to over one thousand basal cell carcinomas (BCCs) over their lifetime, often beginning in early childhood. The disease imposes not only a medical but also a physical, emotional and economic toll, requiring frequent scar-producing surgeries, topical treatments and lifelong dermatologic care.
"Patients with Gorlin Syndrome endure a lifelong burden of recurring skin cancers that often require repeated surgeries and disfiguring treatments," said Dr. Raza Bokhari, Executive Chairman & CEO of Medicus. "By working together with the Gorlin Syndrome Alliance, we hope to unite clinical science, regulatory leadership and advocacy to deliver hope for individuals facing lifelong burdens of Gorlin Syndrome, reinforcing our mission to deliver targeted innovation where medical need is greatest."
Meredith Weiss, GSA Executive Director, emphasized the patient perspective: "Our families live with a relentless disease, never free, in an endless cycle with cancer. This collaboration may help bridge the gap between breakthroughs in skin cancer therapy and real-world needs faced by Gorlin Syndrome patients. It gives our community a voice in designing pathways to access novel therapies responsibly and ethically."
SKINJECT™ Clinical Development Progress
SKINJECT™ is a novel localized immunogenic investigational precision therapy focused on non-melanoma skin diseases, especially basal cell carcinoma (BCC) of the skin. The therapy utilizes patent-protected, dissolvable doxorubicin-containing microneedle arrays to non-invasively treat BCC.
Medicus is currently conducting a Phase 2 clinical study (SKNJCT-003) in nine clinical sites across the United States, which commenced randomizing patients in August 2024. SKNJCT-003 is a double-blinded, placebo-controlled triple arm proof of concept Phase 2 clinical study designed to non-invasively treat basal cell carcinoma using the novel D-MNA technology.
In March 2025, the company announced a positively trending interim analysis for SKNJCT-003 demonstrating more than 60% clinical clearance. The interim analysis was conducted after more than 50% of the then-targeted 60 patients in the study were randomized. In April 2025, the investigational review board approved increasing the number of participants in SKNJCT-003 to ninety subjects.
The company has expanded its trial sites to Europe and has randomized more than 75% of the ninety participants expected to be enrolled in the study. In September 2025, Medicus received positive feedback from the FDA regarding its Type C meeting supporting the development of SKINJECT™, indicating that the company may follow the 505(b)(2) regulatory pathway to non-invasively treat BCC using dissolvable D-MNA.
International Clinical Expansion
Medicus also has a clinical study (SKNJCT-004) currently underway in the United Arab Emirates (UAE). The study is expected to randomize thirty-six patients across six sites in the UAE. Cleveland Clinic Abu Dhabi (CCAD) serves as the principal investigator, along with Sheikh Shakbout Medical City (SSMC), Burjeel Medical City (BMC), Rashid Hospital (RH), Clemenceau Medical Center (CMC) and American Hospital of Dubai (AHD).
The company completed a Phase 1 safety and tolerability study (SKNJCT-001) in March 2021, which met its primary objective of safety and tolerability. The study also demonstrated efficacy of the investigational product D-MNA, with six participants experiencing complete response on histological examination of the resected lesion.
Patient Registry and Community Support
The Gorlin Syndrome Alliance serves as a U.S.-based 501(c)(3) nonprofit organization with a mission to "thoughtfully support, comprehensively educate, and aggressively seek the best treatments and a cure" for individuals affected by Gorlin syndrome. The GSA operates as a bridge between patients, clinicians, researchers, and industry, offering education, support networks, advocacy, and research infrastructure.
A major component of GSA's research efforts is the Gorlin Syndrome Alliance Patient Registry (GSAPR), designed to collect standardized, longitudinal data including demographics, genotype, clinical features, treatments, outcomes, and quality-of-life measures from people with Gorlin syndrome or their caregivers. The registry is hosted on a secure platform in collaboration with the National Organization for Rare Disorders (NORD), ensuring data privacy, encryption, and controlled access.
The collaboration will more tightly integrate patient community-led insights and data into the design, monitoring, and long-term development of SKINJECT™ in this rare disease population, representing a patient-centric approach to drug development for rare diseases.
