Medicus Pharma Advances SkinJect® into Registrational Development for Gorlin Syndrome
New Drug Application (NDA) enabling Phase 2b Protocol submission to the U.S. FDA Targets Rare Disease Population with Significant Unmet Need and Lifetime Burden of Repeated Skin Cancer Surgeries
PHILADELPHIA, June 03, 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 therapeutics assets, today announced the submission of Protocol SKNJCT-005 to the U.S. Food and Drug Administration (FDA) under the Company’s existing Investigational New Drug (IND) application, for SkinJect® into registrational development for patients with Gorlin Syndrome, also called Nevoid Basal Cell Carcinoma Syndrome (NBCCS), a rare inherited genetic disorder associated with lifelong development of multiple basal cell carcinomas and recurrent skin cancers, often resulting in repeated surgical procedures.
The submitted protocol outlines a Phase 2b, open-label study designed to generate efficacy and safety data intended to support a future New Drug Application (NDA) for SkinJect® in Gorlin Syndrome. This submission is the latest step in the Company’s strategic expansion of the SkinJect® clinical program into a high unmet need orphan indication, where treatment options are limited and currently there are no FDA-approved lesion-directed therapies specifically developed for patients with Gorlin Syndrome.
“This FDA submission marks an important milestone in our SkinJect® strategy to develop what we believe could become the first FDA-approved lesion-directed therapy for patients with Gorlin Syndrome,” said Dr. Raza Bokhari, Medicus Executive Chairman and CEO, “Patients with Gorlin Syndrome frequently face repeated surgeries throughout their lifetimes. The ability to treat multiple lesions simultaneously using a localized precision therapy, while potentially preserving cosmetic outcomes represents an important advancement and what we believe could become a new standard of care for this patient population.”
Significant Unmet Need in Gorlin Syndrome
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.
The Company believes the Gorlin Syndrome program represents an attractive development opportunity. The successful development of SkinJect® for Gorlin Syndrome could provide an initial orphan drug indication and potential commercial entry point for broader SkinJect® expansion across non-melanoma skin cancer indications. 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 clearance of basal cell carcinoma lesions.
As part of its broader regulatory strategy for SkinJect® in Gorlin Syndrome, the Company previously applied to the FDA for Orphan Drug Designation in April 2026 and intends to apply for Rare Pediatric Disease Priority Review Voucher before the end of the second quarter of 2026. These submissions stem from the Company’s evaluation that SkinJect® qualifies for both of the FDA’s Orphan Drug Designation as well as Rare Pediatric Disease Priority Review Voucher, as Gorlin Syndrome is a rare autosomal dominant genetic mutation afflicting less than 200,000 Americans and frequently manifests during early childhood and adolescence.
If granted, Orphan Drug Designation may provide important development incentives, including eligibility for exemption from FDA new drug application (NDA) fees that currently exceed $5 million and seven (7) years of U.S. market exclusivity following approval. Rare Pediatric Disease Priority Review Voucher following approval, if granted, could potentially can shorten the FDA NDA review period by several months, compressing the time to market entry and commercialization, which could potentially provide meaningful early access to Gorlin Syndrome patients living with this rare disease while enhancing the commercial value and capital efficiency of the SkinJect® program.
Registrational Study Designed Around Clinically Meaningful Visual Clearance:
SKNJCT-005 is a Phase 2b, open-label, multi-center registrational study evaluating SkinJect® 200 mcg in up to 50 patients with Gorlin Syndrome and multiple basal cell carcinomas.
The study is specifically designed to evaluate clinical clearance, or visual clearance, of treated lesions using blinded independent central review of standardized clinical photography. The primary endpoint measures the proportion of patients achieving complete clinical clearance in at least 50% of prospectively selected target lesions at Week 10, representing a clinically meaningful patient-level outcome in a multi-lesion disease.
Importantly, the study focuses on visual clearance, because complete elimination of visible lesions represents a clinically meaningful outcome for Gorlin Syndrome patients, many of whom undergo repeated surgical interventions throughout their lives. The Company believes successful visual clearance may offer patients the opportunity to reduce procedural burden while preserving future treatment options.
Patients may receive treatment across two to four target lesions simultaneously, reflecting the real-world management of Gorlin Syndrome. Each target lesion will receive three applications of SkinJect® administered on Days 1, 8 and 15, with an optional fourth treatment on Day 22 if visual clearance has not yet been achieved. Patients will be followed through Week 24 to evaluate durability of response.
The protocol incorporates blinded central review, standardized photography, lesion-level assessments, Reflectance Confocal Microscopy (RCM), Line-Field Optical Coherence Tomography (LF-OCT), and durability follow-up designed to generate robust efficacy and safety data supporting potential registration.
The study was developed with input from leading dermatologic oncology experts, including Principal Investigator Dr. Babar Rao, MD, FAAD, a globally recognized Mohs Surgeon, and incorporates feedback from patient advocacy representatives of the Gorlin Syndrome Alliance.
SKNJCT-005 Study Design Built on Strengthened Phase 2 SkinJect® Dataset
The SKNJCT-005 registrational program builds on the Company’s completed SKNJCT-003 Phase 2 study in nodular basal cell carcinoma (nBCC), evaluating safety and efficacy of doxorubicin containing microneedle arrays (D-MNA). Previously reported results from the 200 mcg D-MNA cohort evaluation at Day 57 demonstrated 64% clinical clearance, 55% histological clearance, clear dose-dependent therapeutic activity, and favorable safety profile with no treatment-related serious adverse events reported.
The Company believes these findings support advancement of the 200 mcg D-MNA regimen into registrational development program for nBCC of the skin and provide a compelling foundation for evaluation in the Gorlin Syndrome population.
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
About Medicus Pharma Ltd.
Medicus Pharma Ltd. (Nasdaq: MDCX) is a precision-guided biotech/life sciences company focused on accelerating the clinical development programs of novel and potentially disruptive therapeutics assets. The Company is actively engaged in multiple countries across three continents.
Company’s current key therapeutics assets are:
SkinJect®, a novel localized immuno-oncology precision product focused on non-melanoma skin diseases, especially basal cell carcinoma (BCC) and Gorlin Syndrome, a rare autosomal dominant disease also called nevoid BCC syndrome, collectively representing a ~$2 billion market opportunity.
Teverelix®, a next generation GnRH antagonist is a first-in-market product for cardiovascular high-risk advanced prostate cancer patients and patients with acute urinary retention relapse (AURr) episodes due to enlarged prostate, collectively representing a ~$6 billion market opportunity.
Cautionary Notice on Forward-Looking Statements
Certain information in this news release constitutes "forward-looking information" under applicable securities laws. "Forward-looking information" is defined as disclosure regarding possible events, conditions or financial performance that is based on assumptions about future economic conditions and courses of action and includes, without limitation, statements regarding the collaboration with GSA including the potential benefits thereof for GSA, those suffering with Gorlin Syndrome and Medicus (including as it relates to the development of SkinJect®), ability to be approved for the Registrational IND Program to enable those suffering with Gorlin Syndrome to access SkinJect® under physician-supervised treatment protocols, the submission of Protocol SKNJCT-005 and the design, timing, conduct and results of the SKNJCT-005 Phase 2b registrational study, including whether the resulting data will be sufficient to support a future New Drug Application for SkinJect® in Gorlin Syndrome, the potential for SkinJect® to become the first FDA-approved lesion-directed therapy for patients with Gorlin Syndrome and a new standard of care for this patient population; and the Company's intention to apply for a Rare Pediatric Disease Priority Review Voucher and the potential issuance and benefits of such voucher, Orphan drug designation for SkinJect® the development of Teverelix and expectations concerning, and future outcomes relating to, the development, advancement and commercialization of Teverelix for AURr, high CV risk prostate cancer, women’s health indications like endometriosis, and the potential market opportunities related thereto, the memorandum of understanding with HelixNano, including the potential signing of definitive agreements between Medicus and HelixNano and the development of thermostable infectious diseases vaccines by combining HelixNano’s proprietary mRNA vaccine platform with Medicus’s proprietary microneedle array (MNA) delivery platform, the Company’s aim to fast-track the clinical development program and convert the SKNJCT-003 exploratory clinical trial into a pivotal clinical trial, and approval from the FDA and the timing thereof, including with respect to the Company’s submission for approval in the FDA Commissioner's National Priority Voucher program plans and expectations concerning, and future outcomes relating to, the development, advancement and commercialization of SkinJect® through SKNJCT-003 and SKNJCT-004, and the potential market opportunities related thereto, the Company’s expectations regarding reported efficacy findings, the overall response rate and potential changes thereto, and whether there will be material changes to its reported SKNJCT-003 topline results and to secure an EOP2 meeting with the FDA in the first half of 2026, entry into definitive documents with Reliant and the expected terms thereof, engaging in proposed Medicus-sponsored studies currently contemplated in the Reliant non-binding letter of intent and the expected benefits thereof, the expansion of SKNJCT-003 into the United Kingdom and the potential benefits therefrom, the advancement of the SKNJCT-004 study and the potential results of and benefits of such study. Forward-looking statements are often but not always, identified by the use of such terms as "may", “on track”, “aim”, "might", "will", "will likely result", “could,” “designed,” "would", "should", "estimate", "plan", "project", "forecast", "intend", "expect", "anticipate", "believe", "seek", "continue", "target", “potential” or the negative and/or inverse of such terms or other similar expressions. These statements involve known and unknown risks, uncertainties and other factors, which may cause actual results, performance or achievements to differ materially from those expressed or implied by such statements, including those risk factors described in the Company's annual report on form 10-K for the year ended December 31, 2025, and in the Company's other public filings on EDGAR and SEDAR+, which may impact, among other things, the trading price and liquidity of the Company's common shares. Forward-looking statements contained in this news release are expressly qualified by this cautionary statement and reflect our expectations as of the date hereof and thus are subject to change thereafter. The Company disclaims any intention or obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law. Readers are further cautioned not to place undue reliance on forward-looking statements as there can be no assurance that the plans, intentions or expectations upon which they are placed will occur. Such information, although considered reasonable by management at the time of preparation, may prove to be incorrect and actual results may differ materially from those anticipated.
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