Core Insights - Relmada Therapeutics announced positive 9-month follow-up safety and efficacy data for NDV-01 in non-muscle invasive bladder cancer (NMIBC), achieving a 92% overall response rate [1][3] - The FDA provided feedback supporting two potential registrational trials for NDV-01, one for 2nd line refractory BCG-unresponsive NMIBC and another for intermediate-risk NMIBC [1][4] - The company plans to initiate Phase 3 pivotal programs for NDV-01 in the first half of 2026 [2][8] FDA Meeting Outcomes - The FDA confirmed that no additional non-clinical studies are required for the 505(b)(2) New Drug Application (NDA) [4] - A single-arm trial for high-grade, 2nd line BCG-unresponsive NMIBC patients is considered acceptable by the FDA [4] - A randomized controlled trial proposal for intermediate-risk NMIBC was also deemed generally acceptable [4] Clinical Results - The 9-month follow-up data showed a complete response rate of 92% (23/25) for all patients, with 84% (21/25) at 3 months and 87% (20/23) at 6 months [3][5] - In the BCG-unresponsive subpopulation, the complete response rates were 91% (10/11) at any time, 82% (9/11) at 3 months, and 78% (7/9) at 6 months [6][12] Safety Profile - No new safety signals were reported, with no patients experiencing Grade 3 treatment-related adverse events (AEs) [11] - Among 36 enrolled patients, 61% experienced treatment-related AEs, primarily transient dysuria (62%) [11] Product Overview - NDV-01 is a sustained-release formulation of gemcitabine and docetaxel designed for intravesical administration, allowing for gradual drug release over 10 days [8] - The product is convenient to administer in-office, requiring less than 10 minutes and no specialized equipment [8] Market Opportunity - NMIBC accounts for 75-80% of all bladder cancer cases, with a significant market opportunity due to high recurrence rates and limited treatment options [9]

Core Insights - Relmada Therapeutics reported a 91% overall response rate for NDV-01 in non-muscle invasive bladder cancer during a 6-month follow-up of a Phase 2 study, indicating strong efficacy and safety [1][3][4] - The company is preparing to initiate a Phase 3 registration trial for NDV-01 in the first half of 2026 and plans to start a Phase 2 study for sepranolone in Prader-Willi syndrome in the same timeframe [1][5] Clinical Results - The Phase 2 study of NDV-01 involved 29 patients, with a median age of 73 years. The study reported a complete response rate of 91% at any time point, 83% at 3 months, and 90% at 6 months [2][3] - No patients progressed to muscle invasive disease or underwent radical cystectomy, and no patients experienced Grade 3 treatment-related adverse events [3][4] Pipeline Developments - NDV-01 is a sustained-release formulation of gemcitabine and docetaxel, designed for intravesical administration, which enhances drug retention and minimizes systemic toxicity [10][12] - Sepranolone is a first-in-class GABAA Modulating Steroid Antagonist targeting disorders linked to excess GABAergic activity, with plans for further clinical development [5][13] Financial Performance - For the second quarter ended June 30, 2025, Relmada reported a net loss of $9.9 million, or $0.30 per share, compared to a net loss of $17.8 million, or $0.59 per share, in the same period of the previous year [5][24] - Research and development expenses were $2.8 million for the quarter, down from $10.7 million in Q2 2024, primarily due to reduced trial costs [5][24] Market Opportunity - The U.S. market for high-grade/intermediate-grade non-muscle invasive bladder cancer is significant, with approximately 600,000 prevalent cases, highlighting the potential for NDV-01 as a frontline or salvage therapy [7][12] - Prader-Willi syndrome, targeted by sepranolone, has an estimated U.S. prevalence of 20,000 patients, representing an orphan disease market [7][14]