PHILADELPHIA, PA — 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 for SkinJect® (D-MNA) for the treatment of basal cell carcinoma (BCC) in patients with Gorlin 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 a high unmet need orphan indication, where current treatment options are limited and often involve repeated surgical procedures associated with cumulative morbidity and disfigurement.
Addressing a High-Burden Rare Disease
Gorlin Syndrome, also known as nevoid basal cell carcinoma syndrome, is a rare, inherited condition driven primarily by mutations in the PTCH1 gene, leading to dysregulation of the Hedgehog signaling pathway and early, recurrent tumor formation.
Patients with this condition may develop dozens 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 are no FDA-approved therapies specifically for BCC in Gorlin Syndrome
- Current treatments rely heavily on surgical excision or Mohs surgery
- Systemic therapies, such as Hedgehog inhibitors, are limited by tolerability and recurrence upon discontinuation
Medicus believes SkinJect® has the potential to address this gap through a localized, repeatable, non-surgical treatment approach.
SkinJect®: A Targeted, Localized Therapeutic Approach
SkinJect® (D-MNA) is a doxorubicin-containing dissolvable microneedle array designed for direct intradermal delivery into BCC lesions.
Key attributes:
- Localized drug delivery directly into tumor tissue
- High local concentration with minimal systemic exposure
- Minimally invasive, lesion-directed treatment
- Designed for repeat 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
This mechanism is particularly well-suited for Gorlin Syndrome, where patients require ongoing management of multiple lesions.
Clinical Foundation and Development Progress
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 and histological complete responses in treated lesions
While these studies were conducted in the broader BCC population, the Company believes the shared tumor biology between sporadic and Gorlin-associated BCC provides a strong rationale for expansion into this orphan indication.
Orphan Drug Designation Strategy
Based on available epidemiological data, Gorlin Syndrome affects an estimated:
- ~11,000 patients in the United States (conservative estimate)
- Well below the 200,000 threshold required for orphan designation
If granted, Orphan Drug Designation may provide:
- 7 years of U.S. market exclusivity upon approval
- Potential eligibility for tax credits and fee waivers
- Enhanced regulatory interaction with the FDA
Strategic Implications
The ODD submission strengthens Medicus’ broader SkinJect strategy by:
- Expanding into a regulatory-favorable orphan indication
- Positioning SkinJect as a repeatable, non-surgical platform therapy
- Supporting a differentiated clinical and regulatory pathway
- Enhancing the program’s partnering and out-licensing attractiveness
About Medicus Pharma Ltd. (https://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 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.
The Company is actively engaged in following collaborations:
Skinject™ Platform Expansion
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.
Patient Access and Advocacy
In October 2025, the Company announced a 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.
AI Enabled Clinical Development
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.
For further information, contact:
Carolyn Bonner, President and Chief Financial Officer
(610) 636-0184
[email protected]
Anna Baran-Djokovic, SVP Investor Relations
(305) 615-9162
[email protected]