Medicus Pharma (NASDAQ:MDCX) earlier this week outlined progress in advancing its Skinject program, highlighting a newly submitted orphan drug designation application with the US Food and Drug Administration targeting Gorlin syndrome, a rare genetic condition with no approved treatments.
In an interview with Proactive, CEO Dr Raza Bokhari said the company is positioning 2026 as a “catalyst rich year,” driven by developments across its pipeline, led by Skinject.
Proactive: You've got some interesting news out regarding an orphan drug application using Skinject for Gorlin syndrome, which currently has no treatment. Can you explain how Skinject is being used here?
Dr Raza Bokhari: We continue to advance forward, and 2026 is a catalyst-rich year for us. Skinject is a novel localized immunotherapy for non-melanoma skin diseases, especially basal cell carcinoma. We formed an alliance with the Gorlin Syndrome Alliance, representing a rare genetic disease affecting around 11,000 Americans. Patients can develop hundreds of lesions, and to date, there is no known treatment.
We’ve seen promising Phase 2 results, where three out of four patients are visually cleared. Based on this, we’ve submitted for orphan drug designation with the FDA and are exploring a registration-grade study.
This seems aligned with the purpose of orphan drug designation—addressing unmet needs with more patient-friendly approaches.
Absolutely. Surgical approaches are disfiguring and not truly viable treatments. This condition qualifies due to its rarity, and it may also be eligible for pediatric rare disease programs, allowing for faster regulatory pathways. This is where science, advocacy, and regulatory support align.
You’ve got multiple programs underway. Are you confident financially to support them?
Yes, for an emerging biotech company, we have reasonably adequate cash on hand to see us through the remainder of the year. This supports the Gorlin syndrome program, Teverelix studies, and a genomic-guided women’s health study in the Middle East. We are capital efficient and confident in our position.
Quotes have been lightly edited for style and clarity