Relmada Therapeutics Provides Regulatory Update Confirming FDA Alignment on Registrational Studies Design for NDV-01 for Two Separate Indications

Core Insights - The FDA has provided written feedback supporting the registrational development pathway for Relmada Therapeutics' NDV-01 in treating high-grade BCG-unresponsive non-muscle invasive bladder cancer (NMIBC) and intermediate-risk NMIBC in the adjuvant setting [1][2][3] Group 1: FDA Feedback and Clinical Trials - The FDA indicated that a single-arm, open-label clinical trial for NDV-01 in high-grade, BCG-unresponsive NMIBC is an appropriate registrational approach, facilitating a potential NDA submission [2] - The FDA also supported a planned single registrational study in intermediate-risk NMIBC, which will follow an open-label, randomized-to-observation design [3] - Both registrational trials are anticipated to initiate in the first half of 2026 [1][3] Group 2: Study Designs and Objectives - The pivotal Phase 3 study for high-grade NMIBC will evaluate the drug's efficacy based on FDA guidance regarding study population, endpoint selection, and evaluation methodology [5][8] - The primary endpoint for the intermediate-risk NMIBC study will be the complete response (CR) rate, with a key secondary endpoint of duration of response (DOR) [6][7] - Assessments for both studies will include cystoscopy, cytology, and biopsy as per protocol [8] Group 3: Product Overview - NDV-01 is a sustained-release, intravesical formulation of gemcitabine and docetaxel, designed for gradual drug release over ten days, enhancing local exposure while minimizing systemic toxicity [9] - The formulation is convenient for in-office administration, requiring less than five minutes and no anesthesia or specialized equipment [9] - NDV-01 is protected by patents until 2038, indicating a long-term competitive advantage [9] Group 4: Market Opportunity - NMIBC accounts for 75-80% of all bladder cancer cases, with a high recurrence rate of 50-80% over five years, highlighting a significant market opportunity [10] - High-grade BCG-unresponsive disease is particularly challenging to treat, with limited bladder-sparing options available [10] - Intermediate-risk NMIBC in the adjuvant setting currently has no approved therapies, positioning NDV-01 as a potential frontline or salvage therapy [10]