SkinJect Advances into Registrable Development as Potential First-in-Class Therapy for Gorlin Syndrome

Medicus Pharma has advanced its investigational SkinJect program into registrational development for Gorlin syndrome, also known as nevoid basal cell carcinoma syndrome (NBCCS), following submission of a phase 2b study protocol to the US Food and Drug Administration (FDA) under the company’s existing Investigational New Drug application.¹

The submission marks a significant step in the development of a potential lesion-directed therapy for patients with Gorlin syndrome, a rare inherited disorder characterized by lifelong development of multiple basal cell carcinomas (BCCs). There are currently no FDA-approved lesion-directed therapies specifically developed for this patient population.

“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,” Raza Bokhari, MD, MBA, executive chairman and CEO, said in a statement. “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.”¹

Upcoming Study

The newly submitted protocol, SKNJCT-005, outlines a phase 2b, open-label, multicenter registrational study designed to generate efficacy and safety data that could support a future New Drug Application (NDA) for SkinJect in Gorlin syndrome. The planned study will evaluate SkinJect 200 mcg in up to 50 patients with Gorlin syndrome who have multiple BCC lesions. The primary endpoint is the proportion of patients achieving complete clinical, or visual, clearance in at least 50% of prospectively selected target lesions by week 10.

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Patients enrolled in the study may receive treatment on 2 to 4 target lesions simultaneously, reflecting the real-world management challenges associated with Gorlin syndrome. Each lesion will receive 3 SkinJect applications on days 1, 8, and 15, with an optional fourth treatment on day 22 if visual clearance has not been achieved. Participants will be followed through week 24 to assess the durability of response. The protocol includes blinded independent central review of standardized clinical photography, lesion-level assessments, reflectance confocal microscopy (RCM), and line-field optical coherence tomography (LF-OCT).

Existing Research

The registrational program builds upon findings from the company’s completed SKNJCT-003 (NCT06608238) phase 2 study in nodular basal cell carcinoma.² That study evaluated doxorubicin-containing microneedle arrays (D-MNA), the technology underlying SkinJect. Results from the 200 mcg D-MNA cohort demonstrated 64% clinical clearance and 55% histological clearance at day 57. Investigators also observed dose-dependent therapeutic activity and reported no treatment-related serious adverse events.

Gorlin syndrome is a rare autosomal dominant disorder caused primarily by mutations affecting the Hedgehog signaling pathway. Patients can develop dozens, hundreds, or even more than 1,000 basal cell carcinomas throughout their lifetime. Lesions often begin appearing during childhood or adolescence and continue to develop over time, frequently requiring repeated surgical interventions. The condition is estimated to affect approximately 1 in 30,000 to 60,000 individuals worldwide, corresponding to an estimated 6,000 to 12,000 patients in the US.

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Next Steps

The company is also pursuing additional regulatory pathways to support development. In April 2026, Medicus applied for FDA Orphan Drug Designation for SkinJect in Gorlin syndrome and plans to seek a Rare Pediatric Disease Priority Review Voucher. Because Gorlin syndrome affects fewer than 200,000 Americans and often presents during childhood or adolescence, the company believes the program may qualify for both designations.

If granted, Orphan Drug Designation could provide development incentives, including fee exemptions and 7 years of market exclusivity following approval. Medicus also noted that a Rare Pediatric Disease Priority Review Voucher could potentially shorten FDA review timelines if the product ultimately receives approval.

References

1. Medicus Pharma Advances SkinJect® into Registrational Development for Gorlin Syndrome. News release. Globe Newswire. Published June 3, 2026. Accessed June 3, 2026. https://www.globenewswire.com/news-release/2026/06/03/3305990/0/en/medicus-pharma-advances-skinject-into-registrational-development-for-gorlin-syndrome.html?_gl=1*42bpxa*_up*MQ..*_ga*MTQxNTk5Mzk2NS4xNzgwNDkxNjUy*_ga_B6167QB2TF*czE3ODA0OTE2NTIkbzEkZzAkdDE3ODA0OTE2NTIkajYwJGwwJGgyMDgwMDU3NDc3*_ga_ERWPGTJ5X8*czE3ODA0OTE2NTIkbzEkZzAkdDE3ODA0OTE2NTIkajYwJGwwJGgw

2. Medicus Pharma reports positive phase 2 SKNJCT-003 topline data observing 73% clinical clearance and 40% histological clearance (CR) at day 57 in 200μg cohort. News release. Medicus Pharma. Published May 5, 2026. Accessed June 3, 2026. https://medicuspharma.com/medicus-pharma-reports-positive-phase-2-sknjct-003-topline-data-observing-73-clinical-clearance-and-40-histological-clearance-cr-at-day-57-in-200g-cohort/