Medicus Pharma (NASDAQ:MDCX) earlier this week announced that the US Food and Drug Administration (FDA) has cleared the company's Phase 2b registration-enabling clinical trial for SkinJect in patients with Gorlin syndrome, representing an important regulatory milestone for its lead investigational asset.
Speaking with Proactive, CEO Raza Bokhari said the FDA had approved the company's proposed study following discussions and feedback, allowing Medicus Pharma (NASDAQ:MDCX) to move ahead with a crossover registration study involving up to 50 patients.
Bokhari said SkinJect is the company's investigational minimally invasive microneedle array technology, licensed from Carnegie Mellon University and the University of Pittsburgh, for the treatment of basal cell carcinoma.
He noted that the company previously completed a Phase 2 study that generated decision-grade data and demonstrated a clean dose-response curve, providing support for advancing into the next stage of development.
He described the FDA clearance as an important development, stating that the company believes it is "a step closer in the probability of our SkinJect product becoming a commercial reality."
According to Bokhari, the study targets patients with Gorlin syndrome, a rare inherited disorder that can result in the development of numerous basal cell lesions over a patient's lifetime.
He noted there are approximately 10,000 affected patients in the United States and around 130,000 worldwide, adding that there is currently no FDA-approved noninvasive treatment option available for this population.
The CEO explained that Medicus Pharma has incorporated FDA recommendations into its revised study protocol and expects to begin patient recruitment before the end of the year, with the first patient potentially enrolled by early next year if not sooner.
If the registration study achieves its objectives, Bokhari said the company could submit a New Drug Application for SkinJect in Gorlin syndrome while continuing clinical development for the broader nodular basal cell carcinoma indication.
He also highlighted the significant burden faced by Gorlin syndrome patients, explaining that repeated surgical procedures can lead to permanent scarring and considerable psychological and physical impacts over a lifetime.
Looking ahead, potential catalysts for Medicus Pharma include initiation of patient enrollment, progress through the Phase 2b registration study, possible NDA submission for the Gorlin syndrome indication if successful, continued advancement of the broader SkinJect development program, and updates regarding the company's Rare Pediatric Disease Voucher application currently under FDA review.
