An interim analysis from Medicus Pharma’s randomized, double-blind, placebo-controlled Phase 2 study (SKNJCT-003) reported more than 60% clinical clearance with SKINJECT, a dissolvable doxorubicin microneedle patch for basal cell carcinoma. Following the readout, the study expanded from 60 to 90 subjects across U.S. and European sites, with more than 75% of participants now randomized. FDA feedback from a recent Type C interaction indicated the program may proceed via the 505(b)(2) pathway, aligning with a strategy that leverages established doxorubicin safety while generating new data on localized delivery.
Against that backdrop, Medicus has formed a collaboration with the Gorlin Syndrome Alliance to pursue an Expanded Access IND, enabling physician-supervised access to SKINJECT for patients with Gorlin syndrome facing multiple, recurrent, or inoperable BCCs. The effort is designed to formalize compassionate-use protocols while capturing real-world safety and tolerability, potentially complemented by patient community data infrastructure. In parallel, Medicus continues its controlled trials in the U.S., EU, and the UAE (SKNJCT-004), positioning the access program as an adjunct rather than a substitute for registrational evidence generation.
The move signals a pragmatic development tack: pair a relatively efficient regulatory route with advocacy-driven access to address a concentrated unmet need. Gorlin patients, often contending with hundreds of lesions over a lifetime, are poorly served by repetitive surgery and the toxicity or logistics of systemic options. A lesion-directed patch that is office-based could fit clinical workflows if the efficacy signal proves durable and the local safety profile remains manageable. The alliance also offers operational advantages—faster patient identification, richer phenotyping, and a path to embed standardized imaging and outcome measures—while reinforcing the narrative that SKINJECT’s clinical utility may be greatest in populations with extreme lesion burden where procedural fatigue and scarring are significant.
For sites and CROs, an Expanded Access IND introduces both opportunity and complexity. Dermatology practices that can operationalize protocolized lesion selection, serial photography, and dermatopathology confirmation may see increased patient flow from advocacy channels, but they will also need to stand up IND-compliant oversight, IRB approvals, and training for consistent patch application and documentation. Data quality will matter; FDA’s tolerance for incorporating real-world evidence hinges on standardized assessments that can be mapped to trial endpoints. Safety monitoring must address cumulative local exposure to doxorubicin across multiple lesions, pediatric considerations common in Gorlin syndrome, and the logistics of tracking adverse events across potentially numerous treated sites. Technology vendors with eSource, imaging, and registry integrations could find near-term demand if Medicus and GSA harmonize data capture with the natural history registry.
The next inflection point is the full Phase 2 readout, including histologic confirmation, durability of clearance, cosmetic outcomes, and a clearer view of local tissue effects such as ulceration or infection. If Medicus attempts to convert SKNJCT-003 into a pivotal package, alignment on patient-level versus lesion-level endpoints and consistency of assessment across regions will be decisive. Expanded access data could help characterize use patterns and safety in high-lesion-load patients, but its evidentiary weight will depend on protocol rigor and endpoint concordance with the controlled trials. Manufacturing reliability and scale for microneedle arrays, site training reproducibility, and clarity on reimbursement pathways for an in-office procedure remain unresolved. Finally, the company’s broadened scope following the Antev acquisition introduces a resource allocation question; sustained execution on SKINJECT’s clinical and access programs will be the signal to watch over the next two to three quarters.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
