SKNJCT-005 registrational clinical study protocol strengthened through planned implementation of FDA recommendations, including combined clinical and histological clearance endpoints and extended patient follow-up
PHILADELPHIA, July 27, 2026 (GLOBE NEWSWIRE) — 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 that the U.S. Food and Drug Administration (“FDA”) has completed its safety review of the Company’s Investigational New Drug (“IND”) application for SKNJCT-005 and issued a “Study May Proceed” letter authorizing the initiation of the Company’s NDA-enabling registrational Phase 2b clinical study evaluating SkinJect® (D-MNA) 200 mcg in patients with Gorlin Syndrome presenting with multiple basal cell carcinomas (“BCCs”).
The FDA authorization follows Medicus’ submission of the SKNJCT-005 protocol and the Company’s comprehensive response to the Agency’s June 18, 2026, information requests.
“Receiving FDA clearance to initiate our registrational Gorlin Syndrome study represents a significant regulatory milestone for the SkinJect® program,” stated Dr. Raza Bokhari, Medicus Executive Chairman and CEO. “Importantly, the FDA’s recommendations strengthen the registrational study by enhancing the scientific rigor of the efficacy assessments while further supporting the long-term evaluation of treatment durability. We believe these refinements position SkinJect® to generate an even more robust data package as we advance toward a potential NDA submission for patients with Gorlin Syndrome, a population with substantial unmet medical need, who frequently face repeated surgeries throughout their lifetimes. The ability to treat multiple lesions using a localized precision therapy represents an important advancement and what we believe could become a new standard of care for this patient population.”
FDA Authorization Advances Registrational Development
SKNJCT-005 is designed as a Phase 2b, open-label, multicenter, NDA-enabling registrational study evaluating SkinJect® 200 mcg in patients with Gorlin Syndrome (Nevoid Basal Cell Carcinoma Syndrome) who develop multiple basal cell carcinomas requiring repeated surgical treatment. The study is intended to generate robust efficacy and safety data to support registration of SkinJect® in this rare disease population and is expected to enroll up to 50 patients.
SkinJect® is a proprietary dissolvable microneedle-array patch that delivers doxorubicin directly into basal cell carcinoma lesions, enabling localized treatment while minimizing systemic drug exposure. Previous Phase 1 and Phase 2 studies have demonstrated a favorable safety profile together with encouraging clinical and histological clearance rates in patients with basal cell carcinoma. The submitted protocol cites data from the completed 90-patient SKNJCT-003 study demonstrating 64% clinical clearance and 55% histological clearance following three treatments with the 200-mcg dose at Day 57.
Protocol Strengthened Following FDA Review
As part of the FDA review process, the Agency provided constructive recommendations designed to enhance the scientific and regulatory utility of the study. Medicus agreed to incorporate these recommendations through a protocol amendment, including:
- requiring baseline biopsy confirmation of nodular basal cell carcinoma for all enrolled patients;
- revising the primary efficacy endpoint to require both clinical and histological clearance at a prespecified time point;
- requiring in-person investigator assessment of clinical clearance in conjunction with histological confirmation;
- aligning the responder definition with the revised composite endpoint; and
- extending patient follow-up from 24 weeks to at least two years to evaluate durability of response.
In its “Study May Proceed” letter of July 23, 2026, the FDA has also provided additional recommendations relating to pharmacokinetic assessments, evaluation of used microneedle arrays, genetic confirmation of Gorlin Syndrome, establishment of an independent Data Safety Monitoring Board, refinement of statistical methodology, development of a standalone Statistical Analysis Plan, and future incorporation of randomized, blinded, comparator-controlled study designs intended to support substantial evidence of effectiveness. These recommendations were specifically identified by the FDA as not constituting clinical hold issues. The company is evaluating these recommendations and intends to submit an amended SKNJCT-005 protocol incorporating the FDA’s recommendations.
The Company expects to provide additional updates regarding study initiation and enrollment timing as development progresses.
Addressing a Significant Unmet Medical Need in Gorlin Syndrome:
Gorlin Syndrome is a rare autosomal dominant genetic disorder caused primarily by mutations affecting the Hedgehog signaling pathway, characterized by the lifelong development of multiple basal cell carcinomas beginning during childhood or early adulthood. Many patients develop dozens-or even hundreds-of tumors throughout their lifetime, often requiring repeated surgical excisions that can result in cumulative scarring, functional impairment and significant psychosocial burden. The current protocol notes that affected individuals may develop dozens to more than 1,000 basal cell carcinomas during their lifetime. 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 being developed as a lesion-directed, minimally invasive treatment designed to reduce reliance on repeated surgical procedures while preserving surgery as an option for lesions that do not respond or require definitive excision. If approved, SkinJect® has the potential to become the first FDA-approved lesion-directed therapy specifically developed for patients with Gorlin Syndrome and the first microneedle-based chemotherapy platform designed to achieve clinically meaningful visual and histological clearance of basal cell carcinoma lesions.
