Medicus Pharma (NASDAQ:MDCX) earlier this week announced the filing of a registration study with the US Food and Drug Administration (FDA) for its SkinJect program targeting patients with Gorlin syndrome, marking a potentially important step toward commercialization of the technology.
Speaking with Proactive, chief executive Raza Bokhari said the company has continued to advance SkinJect, a non-invasive treatment platform designed for non-melanoma skin diseases, particularly basal cell carcinoma.
Bokhari noted that Medicus Pharma recently reported top-line Phase 2 results that he described as decision-grade, with a clear dose response. He said those results have helped accelerate discussions with potential partners as the company evaluates opportunities for later-stage development and commercialization.
The latest catalyst centres on Gorlin syndrome, a rare inherited disorder associated with multiple basal cell lesions and significant patient morbidity. Bokhari said Medicus Pharma has filed a registration study with the FDA and hopes to receive authorization to conduct an open-label study involving approximately 50 patients.
According to Bokhari, positive data from the study could support a New Drug Application, potentially allowing SkinJect to become commercially available for Gorlin syndrome patients.
The executive also highlighted the regulatory advantages associated with rare disease development. He said the company has already applied for orphan drug designation, which could provide seven years of market exclusivity and waive approximately US$5 million in FDA filing fees.
In addition, Bokhari said Medicus Pharma is preparing to seek pediatric rare disease designation because Gorlin syndrome often presents in childhood. If granted, the designation could make the company eligible for participation in the FDA's rare disease voucher program.
Bokhari described the registration study as "a substantial step forward" and said it "could be a paradigm shift for our SkinJect program."
Beyond regulatory progress, Medicus Pharma also strengthened its financial position. Bokhari said an institutional investor recently invested more than US$22 million, increasing the company's cash position to approximately US$30 million.
He stated that the funding is expected to provide more than 24 months of operating runway under current development plans and should help alleviate investor concerns regarding potential near-term dilutive financings.