Medicus Pharma Ltd. Announces Collaboration with the Gorlin Syndrome Alliance to Pursue Compassionate Use Pathway for SKINJECT™ in Patients with Gorlin Syndrome

Rhea-AI Summary

Medicus Pharma (NASDAQ: MDCX) announced a collaboration with the Gorlin Syndrome Alliance to pursue an FDA Expanded Access IND pathway enabling physician‑supervised access to SKINJECT™, an investigational doxorubicin microneedle array for patients with Gorlin syndrome.

Key program facts: a Phase 2 study (SKNJCT-003) expanded to 90 subjects with >75% randomized and a reported interim analysis in March 2025 showing >60% clinical clearance; FDA Type C feedback in Sept 2025 indicated a 505(b)(2) regulatory route. The company completed the Antev acquisition in Aug 2025 and runs a 36‑patient SKNJCT-004 study in the UAE.

Positive

• Interim SKNJCT-003 analysis showed >60% clinical clearance
• SKNJCT-003 enrollment increased to 90 subjects
• FDA Type C feedback supports a 505(b)(2) pathway for Skinject
• Completed Antev acquisition (Aug 2025) adding Teverelix development

Negative

• March 2025 interim findings are preliminary and may not correlate with final results
• Teverelix Phase 2a secondary endpoint not met: probability dropped to 82.5% by Day 42

Insights

Clinical-Regulatory Strategist

Collaboration and regulatory progress increase near-term development and access prospects for SKINJECT™ in Gorlin Syndrome patients.

Medicus Pharma's partnership with the Gorlin Syndrome Alliance targets an Expanded Access IND pathway to provide physician‑supervised use of SKINJECT™ for patients with multiple or inoperable basal cell carcinomas. The company already reports a positively trending interim analysis showing > 60% clinical clearance and received constructive FDA feedback in a September 2025 Type C meeting that the program may follow a 505(b)(2) pathway; these elements combine patient access goals with regulatory alignment and ongoing randomized trials.

Key dependencies and risks include formal FDA acceptance of an Expanded Access IND, the representativeness of the interim 60% result to final trial outcomes, and the integrity of real‑world safety data collected through expanded use. The press release notes the interim analysis was preliminary and may not predict final results, and that > 75% of the planned 90 participants have been randomized — metrics to watch closely.

Concrete near‑term items to monitor: FDA decision on the Expanded Access IND, completion and topline results of SKNJCT‑003 and SKNJCT‑004, and enrollment milestones (randomization > 75% of 90 subjects already reported). Expect material updates on these items over the next clinical quarters as the company converts exploratory findings into confirmatory data.

Gorlin Syndrome, also known as Nevoid Basal Cell Carcinoma Syndrome, is a rare inherited genetic disorder affecting 1 in 31,000 people worldwide that can develop 500-1000 Basal Cell Carcinoma (BCC) lesions over lifetime

PHILADELPHIA, Oct. 29, 2025 (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, is pleased to announce 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. It will also more tightly integrate patient community-led insights and data into the design, monitoring, and long-term development of SKINJECT™ in this rare disease population.

SKINJECT™, is a novel localized immunogenic investigational precision therapy focused on non-melanoma skin diseases, especially basal cell carcinoma (BCC) of the skin.

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.”

“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,” said Meredith Weiss, GSA Executive Director. “It gives our community a voice in designing pathways to access novel therapies responsibly and ethically. We’re proud to partner with Medicus to explore new therapeutic options that respect the patient voice and accelerate access to innovation.”

Medicus is currently conducting a Phase 2 clinical study for SKNJCT-003 in nine (9) 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 (BCC) of the skin using novel, patent protected, dissolvable doxorubicin-containing microneedle arrays (D-MNA). 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. The findings of the interim analysis are preliminary and may or may not correlate with the findings of the study once completed. In April 2025, the investigational review board approved to increase the number of participants in SKNJCT-003 to ninety (90) subjects. The Company is expanding its trial sites in Europe and has randomized more than 75% of the ninety (90) participants expected to be randomized in the study. In September 2025, the Company received positive feedback from the Food and Drug Administration (FDA) regarding its Type C meeting supporting the development of Skinject, indicating that the Company may follow 505(b)(2) regulatory pathway to non-invasively treat BCC using dissolvable D-MNA.

The Company also has a clinical study (SKNJCT-004) currently underway in the United Arab Emirates (UAE). The study is expected to randomize thirty-six (36) patients in six (6) sites in the UAE. Cleveland Clinic Abu Dhabi (CCAD) is 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). Insights Research Organization and Solutions (IROS), a UAE-based contract research organization, is coordinating the clinical study for the Company. IROS is a M42 portfolio company.

In August 2025, the Company completed the acquisition of Antev, a UK-based late clinical stage biotech company, developing Teverelix, a next generation GnRH antagonist, as a first in market product for cardiovascular high-risk advanced prostate cancer patients and patients with first acute urinary retention relapse (AURr) episodes due to enlarged prostate.

Antev’s flagship drug candidate is Teverelix trifluoroacetate (Teverelix TFA), a long-acting gonadotrophin-releasing hormone (GnRH) antagonist. Unlike GnRH agonists, which can cause an initial surge in testosterone levels, Teverelix directly suppresses sex hormone production without this surge, potentially reducing cardiovascular risks. This mechanism is particularly beneficial for patients with existing cardiovascular conditions. Teverelix is formulated as a microcrystalline suspension, allowing for sustained release and a six-week dosing interval, which may improve patient compliance and outcomes.

For further information contact:

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

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

About Medicus Pharma Ltd.

Medicus Pharma Ltd. (Nasdaq: MDCX) is a 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, spread over three continents.

SkinJect Inc. a wholly owned subsidiary of Medicus Pharma Ltd., is a development stage, life sciences company focused on commercializing novel, non-invasive treatment for basal cell skin cancer using a patented dissolvable microneedle patch to deliver a chemotherapeutic agent to eradicate tumors cells. The Company completed a phase 1 safety & tolerability study (SKNJCT-001) in March of 2021, which met its primary objective of safety and tolerability; the study also describes the efficacy of the investigational product D-MNA, with six (6) participants experiencing complete response on histological examination of the resected lesion. The Company is currently conducting a randomized, controlled, double-blind, multicenter clinical study (SKNJCT-003) in the United States and Europe. The Company has also commenced a randomized, controlled, double-blind, multicenter clinical study (SKNJCT-004) in the United Arab Emirates.

In August 2025, the Company announced its entry into a non-binding memorandum of understanding (the "MoU") with Helix Nanotechnologies, Inc. ("HelixNano"), a Boston Based biotech company focused on developing a proprietary advanced mRNA platform, in respect of their shared mutual interest in the development or commercial arrangement contemplated by the MoU. The MoU is non-binding and shall not be construed to obligate either party to proceed with a joint venture or any further development or commercial arrangement, unless and until definitive agreements are executed.

In August 2025, the Company completed the acquisition of Antev, a UK-based late clinical stage biotech company, developing Teverelix, a next generation GnRH antagonist, as a first in market product for cardiovascular high-risk advanced prostate cancer patients and patients with first acute urinary retention relapse (AURr) episodes due to enlarged prostate.

Antev’s flagship drug candidate is Teverelix trifluoroacetate (Teverelix TFA), a long-acting gonadotrophin-releasing hormone (GnRH) antagonist. Unlike GnRH agonists, which can cause an initial surge in testosterone levels, Teverelix directly suppresses sex hormone production without this surge, potentially reducing cardiovascular risks. This mechanism is particularly beneficial for patients with existing cardiovascular conditions. Teverelix is formulated as a microcrystalline suspension, allowing for sustained release and a six-week dosing interval, which may improve patient compliance and outcomes.

In September 2020, Antev completed a Phase 1 clinical trial in which Teverelix was shown to be well tolerated with no dose-limiting toxicities and demonstrated rapid testosterone suppression. The study included 48 healthy male volunteers. In February 2023, Antev also completed a Phase 2a study in fifty (50) patients with advanced prostate cancer (APC), where Teverelix achieved the primary endpoint of greater than 90% probability of castration levels of testosterone suppression (97.5%) but the secondary endpoint of maintaining this rate above 90% was not met with the probability dropping to 82.5% by Day 42.

In January 2023, the FDA, reviewed the Phase 1 and Phase 2a data and provided written guidance on Antev’s proposed Phase 3 trial design for Teverelix. This milestone supports the Company’s clinical plans to develop Teverelix as a treatment for advanced prostate cancer patients with increased cardiovascular risk. 

In December 2023, FDA approved the Phase 2b study design in advanced prostate cancer covering 40 patients.

In November 2024, FDA approved the Phase 2b study design in acute urinary retention covering 390 patients.

About the Gorlin Syndrome Alliance: Mission, Registry, and Community Impact

Mission and Role

The Gorlin Syndrome Alliance is a U.S.-based 501(c)(3) nonprofit organization with a stated mission to “thoughtfully support, comprehensively educate, and aggressively seek the best treatments and a cure” for individuals affected by Gorlin syndrome. The GSA serves as a bridge between patients, clinicians, researchers, and industry, offering education, support networks, advocacy, and research infrastructure. It promotes awareness of the syndrome, disseminates patient- and clinician-facing resources (on dermatologic care, surveillance, comorbidity management, etc.), and organizes community engagement events.  

Patient Registry & Natural History Efforts

A major pillar of GSA’s research arm is the Gorlin Syndrome Alliance Patient Registry (GSAPR), also called the natural history registry.  

The registry is designed to collect standardized, longitudinal data (demographics, genotype, clinical features, treatments, outcomes, quality-of-life) from people with Gorlin syndrome or their caregivers.  GSAPR is hosted on a secure platform in collaboration with the National Organization for Rare Disorders (NORD), ensuring data privacy, encryption, and controlled access.  

The registry seeks to:

• Illuminate the natural history and disease progression across phenotypes (cutaneous, dental, skeletal, neurologic) 
• Serve as a resource for patient identification, clinical trial recruitment, and retrospective analyses of intervention outcomes 
• Help harmonize data and standard-of-care practices across clinics and investigators 
• Participation in GSAPR is typically open to diagnosed individuals (or caregivers) at no cost to them, with GSA bearing administrative costs of registry maintenance.
• The registry is intended to supersede prior, smaller registries (e.g. CHORI, Stanford, BCCNS registry) to unify the data landscape.  
  
  

Support, Community, and Outreach

The GSA provides support and connection services including peer networks, education about disease management, and informational resources for patients, families, and clinicians. 
The organization encourages grassroots fundraising and awareness campaigns, helping supporters host local or virtual events, distributing educational materials, and promoting visibility of Gorlin syndrome.  
GSA also organizes national conferences and scientific meetings to bring together patients, caregivers, clinicians, and researchers.  
Contact details: GSA is headquartered at 3005 S. Lamar Blvd, Suite D109 #264, Austin, TX, with phone (267) 689-6443 and email info@gorlinsyndrome.org.  

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, 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 Expanded Access IND Program to enable those suffering with Gorlin Syndrome to access SKINJECT™ under physician-supervised treatment protocols, the development of Teverelix and expectations concerning, and future outcomes relating to, the development, advancement and commercialization of Teverelix for AURr and high CV risk prostate cancer, and the potential market opportunities related thereto, the MOU, 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, 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 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, 2024 (the "Annual Report"), 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. 

FAQ

What does Medicus Pharma's (MDCX) collaboration with the Gorlin Syndrome Alliance aim to do?

The collaboration aims to pursue an Expanded Access IND with the FDA to allow physician‑supervised access to SKINJECT™ for Gorlin syndrome patients.

What clinical evidence does SKINJECT™ (MDCX) have so far?

A Phase 1 safety study met its primary objective and SKNJCT-003 interim analysis (Mar 2025) reported >60% clinical clearance in the dataset reviewed.

How many patients is Medicus enrolling in the SKNJCT-003 trial (MDCX)?

SKNJCT-003 was increased to 90 subjects and the company has randomized more than 75% of that target.

What regulatory pathway did the FDA suggest for Skinject (MDCX)?

FDA Type C feedback in Sept 2025 indicated Medicus may follow a 505(b)(2) pathway for Skinject development.

What is the scope of the SKNJCT-004 study referenced by Medicus (MDCX)?

SKNJCT-004 is underway in the UAE and is expected to randomize 36 patients across six clinical sites.

How does the Antev acquisition (Aug 2025) affect Medicus (MDCX)?

The completed acquisition added Teverelix, a long‑acting GnRH antagonist program for advanced prostate indications to Medicus's portfolio.

Are SKINJECT™ interim results final and guaranteed to predict approval for MDCX?

No; the company states the March 2025 interim analysis findings are preliminary and may not correlate with final study results or regulatory outcomes.