NDV‑01 Phase 2: 12‑Month CRs and Planned RESCUE Phase 3 Program

Relmada Therapeutics reported 12-month Phase 2 interim efficacy and tolerability findings for its sustained-release intravesical gemcitabine/docetaxel formulation NDV-01 in high-risk non-muscle invasive bladder cancer (NMIBC), alongside plans for a two-pathway Phase 3 RESCUE registrational program, in its update on NDV-01 Phase 2 interim data. The release presents the interim dataset and the proposed registrational studies as sequential steps informed by these outcomes and the company’s stated development timeline.

The company described the results as 12-month interim outcomes from an ongoing Phase 2 program in high-risk NMIBC, reporting that 48 patients had been dosed overall and that 20 patients had been dosed in a BCG-unresponsive subpopulation. In the overall cohort, Relmada reported a 12-month complete response (CR) rate of 76% and a 12-month Kaplan–Meier CR estimate of 83%. In the BCG-unresponsive subgroup, the company reported a 12-month CR rate of 80% and a 12-month Kaplan–Meier CR estimate of 84%. The press release notes that patients were assessed with cystoscopy, cytology, and biopsy as indicated. The company reported both point estimates and Kaplan–Meier estimates to describe response durability through 12 months.

Through 12 months, Relmada reported that among patients who received at least one dose, 63% experienced a treatment-related adverse event (TRAE), with no patients experiencing a ≥Grade 3 TRAE and no discontinuations due to adverse events. The company listed the most common TRAEs as transient dysuria described as uncomfortable urination lasting <24 hours (Grade 1), as well as asymptomatic positive urine culture and hematuria. The release lists selected urinary treatment-related adverse events but does not discuss immune-related events or systemic toxicities in detail. Overall, the most commonly cited treatment-related events were urinary and low grade, and the company reported no grade 3 or higher treatment-related adverse events.

Relmada described NDV-01 as a sustained-release intravesical formulation of gemcitabine and docetaxel intended to enable bladder retention with gradual drug release over 10 days, with a ready-to-use presentation described as convenient for in-office administration in approximately 5 minutes without anesthesia or specialized equipment. The company also outlined a Phase 3 RESCUE program with two registrational pathways differentiated by setting and design. One pathway is described as an open-label randomized controlled trial of adjuvant therapy following TURBT in intermediate-risk NMIBC (NDV-01 versus observation) with disease-free survival as the primary endpoint. The other pathway is described as a single-arm trial in second-line BCG-unresponsive NMIBC with carcinoma in situ, with complete response rate at any time as the primary endpoint. The company stated that these interim findings support advancement into the Phase 3 RESCUE registrational program.

For the adjuvant intermediate-risk NMIBC pathway, Relmada listed key secondary endpoints of high-grade recurrence-free survival, progression-free survival, and quality-of-life metrics. For the second-line BCG-unresponsive CIS pathway, the company listed duration of response as a key secondary endpoint and also cited progression-free survival and recurrence-free survival among responders. The release also listed forward-looking milestones, including expected U.S. IND clearance in mid-2026, expected initiation of the Phase 3 RESCUE program in mid-2026, and an expected initial 3-month readout from the Phase 3 second-line BCG-unresponsive study by year-end 2026. Enrollment targets and key inclusion criteria for the Phase 3 program are not specified in the announcement.

Key Takeaways:

• Relmada reported 12-month complete responses in the overall Phase 2 cohort and in the BCG-unresponsive subgroup, alongside Kaplan–Meier estimates intended to describe durability at that timepoint.
• The press release described treatment-related events as generally low grade, with no ≥Grade 3 TRAEs and no treatment-related discontinuations; commonly cited events were transient urinary symptoms.
• The company outlined two RESCUE Phase 3 registrational pathways (randomized adjuvant intermediate-risk NMIBC with DFS primary endpoint; single-arm second-line BCG-unresponsive CIS with CR-at-any-time primary endpoint) and stated mid-2026 to year-end 2026 timing expectations for IND clearance, trial start, and an early readout.