Medicus Pharma Submits Orphan Drug Designation Application to U.S. FDA for SkinJect® in Gorlin Syndrome

PHILADELPHIA, April 17, 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 an Orphan Drug Designation (ODD) application to the U.S. Food and Drug Administration forSkinJect®(D-MNA)for the treatment of basal cell carcinoma (BCC) in patients withGorlin Syndrome, a rare genetic disorder characterized by the development of multiple, recurrent skin cancers.

The submission represents a strategic expansion of the SkinJect program into ahigh unmet need orphan indication, where current treatment options are limited and often involve repeated surgical procedures associated with cumulative morbidity and disfigurement.

Gorlin Syndrome, also known as nevoid basal cell carcinoma syndrome, is a rare, inherited condition driven primarily by mutations in thePTCH1 gene, leading to dysregulation of the Hedgehog signaling pathway and early, recurrent tumor formation.

Patients with this condition may developdozens to over 1,000 basal cell carcinomas over their lifetime, often beginning in childhood, requiring repeated surgical interventions that can result in significant physical and psychological burden.

Despite this high disease burden:

There areno FDA-approved therapies specifically for BCC in Gorlin Syndrome

Current treatments rely heavily onsurgical excision or Mohs surgery

Systemic therapies, such as Hedgehog inhibitors, arelimited by tolerability and recurrence upon discontinuation

Key attributes:

Localized drug deliverydirectly into tumor tissue

High local concentration with minimal systemic exposure

Minimally invasive, lesion-directed treatment

Designed forrepeat use across multiple lesions over time

The microneedle array penetrates the skin and dissolves in situ, releasing doxorubicin locally, enabling both:

Direct cytotoxic tumor cell killing

Induction of immunogenic cell death and local immune activation

SkinJect®has been evaluated in Phase 1 and Phase 2 clinical studies in patients with basal cell carcinoma, demonstrating:

Favorable safety profile, with adverse events primarily limited to treatment site

No reported serious adverse events or dose limiting toxicities in early studies

Evidence of clinical clearance andhistological complete responses in treated lesions

While these studies were conducted in the broader BCC population, the Company believes theshared tumor biology between sporadic and Gorlin-associated BCCprovides a strong rationale for expansion into this orphan indication.

Based on available epidemiological data, Gorlin Syndrome affects an estimated:

~11,000 patients in the United States (conservative estimate)

Well below the200,000 threshold required for orphan designation

If granted, Orphan Drug Designation may provide:

7 years of U.S. market exclusivity upon approval

Potential eligibility fortax credits and fee waivers

Enhanced regulatory interaction with the FDA

The ODD submission strengthens Medicus’ broader SkinJect strategy by:

Expanding into aregulatory-favorable orphan indication

Positioning SkinJect as arepeatable, non-surgical platform therapy

Supporting adifferentiated clinical and regulatory pathway

Enhancing the program’spartnering and out-licensing attractiveness

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

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

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 key therapeutics assets are:

SkinJect™, a novel localized immuno-oncology precision productfocused on non-melanoma skin diseases, especially basal cell carcinoma (BCC) andGorlin Syndrome, a rare autosomal dominant disease also called nevoid BCC syndrome, collectively representing a ~$2 billion market opportunity.

Teverelix®, a next generation GnRH antagonistis 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.

The Company is actively engaged in following collaborations:

In August 2025, the Company announced its entry into a non-binding memorandum of understanding (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, and there can be no assurance that such definitive agreements will be executed.

The Company is exploring co-development of thermostable infectious disease vaccines combining HelixNano’s proprietary mRNA technology with the Medicus microneedle array delivery platform.

In October 2025, the Company announceda strategic collaboration with the Gorlin Syndrome Alliance (GSA) to advance compassionate access to SkinJect for patients suffering from Gorlin Syndrome, also known as nevoid basal cell carcinoma syndrome.

In collaboration with the Gorlin Syndrome Alliance, Medicus is pursuing an Expanded Access IND program to provide Gorlin Syndrome patients with multiple or inoperable BCCs access to SkinJect™, the Company’s investigational D-MNAs, under physician supervision.

In December 2025, the Company signed a non-binding letter of intent to collaborate with Reliant AI Inc., a decision-intelligence company specializing in generative AI for the life sciences, to develop an AI-driven clinical data analytics platform to support capital-efficient and time-efficient clinical development through data-driven dynamic clinical-site selection, pharmacodynamic (PD) informed patient stratification, and enrollment forecasting. The initial phase of the collaboration is expected to support the upcoming Teverelix clinical study planned for 2026. There can be no assurance that a definitive agreement will be executed or that the proposed collaboration will proceed as contemplated.

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 Company’s ability to continue as a going concern, statements regarding the Company’s leadership and prospects, 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, 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 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, including with respect to the Company’s submission for approval in the FDACommissioner'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.