Medicus Pharma Targets Gorlin Syndrome and BCC Backlog with Novel Immuno-Oncology Patch

Basal cell carcinoma (BCC) is the most common form of skin cancer, and although it is typically slow growing and highly treatable when detected early, the current standard of care remains largely dependent on surgical intervention. According to Raza Bokhari, MD, executive chair and chief executive officer of Medicus Pharma, the demand for new treatment approaches continues to grow as surgical capacity remains limited, even in rare diseases like Gorlin Syndrome.

“Mohs surgery [for BCC], by virtue of its capacity constraints, has a backlog,” Bokhari said, noting that there are an estimated 7 million patients awaiting treatment opportunities and that many BCC lesions are of the nodular subtype. He explained that the cost and availability challenges associated with surgical management highlight the need for alternative approaches.

Medicus’ lead asset, SkinJect, is designed as a localized precision immuno-oncology therapy using dissolvable microneedle arrays. The patch contains approximately 400 microneedles measuring about 750 microns in size, each tip-loaded with microdoses of doxorubicin. When applied directly to a lesion, the microneedles penetrate into the dermis and release the drug payload into the tumor microenvironment, triggering an immunogenic response and apoptosis.

Unlike traditional needles, the SkinJect patch is designed to dissolve after application and is not intended to produce a painful injection sensation. Bokhari emphasized that the device’s “needle” terminology may be misleading for pediatric patients and caregivers, as the microneedles are small and designed for localized delivery rather than conventional injection.

Following a phase 2 proof-of-concept study evaluating SkinJect in BCC, Medicus Pharma reported a dose-response relationship, with the 200-microgram cohort demonstrating the strongest outcomes at day 57. In that group, the company reported clinical clearance in 64% of patients and histologic clearance in 55% of evaluated patients. The company plans to use the 200-microgram dose in future pivotal studies.

Beyond conventional BCC treatment, Medicus Pharma is also exploring SkinJect for patients with Gorlin Syndrome, also known as nevoid basal cell carcinoma syndrome. This rare autosomal dominant disorder is characterized by the development of multiple BCC lesions throughout a patient’s lifetime. Bokhari noted that affected patients may develop hundreds of lesions, often requiring repeated surgeries that can be physically and psychologically burdensome.

The company has submitted a cross-reference investigational new drug application to the FDA for an open-label study evaluating SkinJect in 50 patients with Gorlin syndrome. The proposed study would focus on visual clearance rather than requiring extensive biopsies, with the goal of providing a potential treatment option for a patient population with no currently approved first-line therapy.

Bokhari said the program has received support from patient advocacy groups and rare disease organizations, and Medicus Pharma is pursuing potential regulatory designations that could support development. He emphasized that the goal is to combine scientific innovation, patient advocacy, and regulatory collaboration to address unmet needs in both BCC and rare disease care.