Announcement • May 08
Medicus Pharma Ltd Announces Results From Pre-Specified Expanded Phase 2 SKNJCT-003 Data Analysis Demonstrating Positive Dose-Response
Medicus Pharma Ltd. announced results from a pre-specified expanded dataset analysis demonstrating positive dose response from its Phase 2 SKNJCT-003 study evaluating safety and efficacy of Doxorubicin Microneedle Array (D-MNA) to treat nodular basal cell carcinoma (BCC) of the skin, the most common type of skin cancer. These additional pre-specified analysis, build upon the previously reported positive topline results, provide expanded biological, histologic, and safety insights that further strengthen SkinJect’s therapeutic profile and future registrational discussions with the U.S. Food and Drug Administration (FDA). These additional findings are also consistent with prior Phase 1 clinical observations in the SKNJCT-001 study in March 2021, and interim analysis of SKNJCT-003 in March 2025, reinforcing reproducibility across studies. The SKNJCT-003 (NCT06608238) clinical study was designed as a randomized, double-blind, three-arm Phase 2 study evaluating two dose levels of microneedle-mediated delivery of doxorubicin compared with a device-only control in patients with nodular basal cell carcinoma. It was a multi-center study designed to enroll 90 participants presenting with nodular type basal cell carcinoma. The participants were randomized 1:1:1 into three groups: Device-controlled group receiving P-MNA, Low-dose group receiving 100µg of D-MNA, High-dose group receiving 200µg of D-MNA. Expanded central pathology reconciliation demonstrated that 69 participants met intended nodular BCC inclusion criteria, while 21 participants were identified as superficial or mixed subtype lesions. The expanded analysis demonstrates a progressive and dose-dependent improvement, with the strongest separation emerging at Day 57. The Company previously reported topline results from the population of 90 randomized patients, which demonstrated a positive topline and decision-grade dataset. The 69-patient refined efficacy analysis further strengthens the regulatory alignment of the study as the Company advances toward End-of-Phase 2 (EOP2) discussions with the FDA. The topline dataset showed that: The 200µg cohort achieved the highest observed activity at Day 57. Clearance rates improved from Day 29 to Day 57, consistent with continued biological activity over time. The pre-specified expanded dataset analysis builds on this foundation by: Revealing a clear and consistent dose-response relationship across endpoints. Demonstrating stronger separation between the 200µg cohort and control, particularly at Day 57. Strengthens differentiation between drug-driven efficacy and device-only biological activity. Importantly, this expanded central pathology verified dataset provides a more precise and clinically interpretable view of treatment effect, with the 200µg cohort at Day 57 emerging as the leading dose with the most robust and consistent efficacy signal. These findings suggest that many treated lesions may in the future be able to avoid immediate surgical intervention, representing a potentially meaningful shift in the treatment paradigm for BCC. Given the short treatment and excision timeline evaluated, these results are particularly encouraging and may suggest clinically meaningful anti-tumor activity within a highly practical therapeutic window. Collectively, this dataset may support future registration-intent or NDA-enabling development discussions, including optimized patient population, lesion subtype, dose regimen, and treatment-to-excision interval. D-MNA continued to demonstrate a highly favorable safety and tolerability profile, was generally well tolerated with no drug-related serious adverse events, no evidence of systemic doxorubicin toxicity, and predominantly mild localized treatment-site reactions, supporting repeatable lesion-directed administration consistent with prior Phase 1 observations. The device-only arm also demonstrated early biological activity consistent with microneedle-induced local immune response, but it did not show sustained or deepening efficacy over time. In contrast, the 200µg D-MNA cohort demonstrated progressive improvement from Day 29 to Day 57, resulting in clear separation across both clinical and histological endpoints. This pattern is consistent with drug-driven therapeutic effect, rather than a device-only response. These findings are further supported by independent investigator validation from a leading academic dermatologist and clinical investigator. Dr. Rao has evaluated the dataset and noted that he believes it demonstrates a clinically meaningful rapid onset efficacy, clear differentiation between drug and device effect and a profile that supports continued development and regulatory progress. Dr. Rao noted the consistency between visual, histologic, and central pathology findings is highly encouraging and provides growing confidence that SkinJect® is producing meaningful biologic anti-tumor effects. Notably, clinically meaningful anti-tumor responses were observed within weeks, potentially differentiating D-MNA from many existing non-surgical therapies that often require substantially longer treatment durations. This analysis also improves the understanding of the patient populations and treatment parameters most likely to optimize future clinical outcomes. The Company believes these findings further de-risk advancement of the 200µg regimen and informs the design of subsequent development studies, including assessment timing, lesion selection, and endpoint strategy.
