NDV-01 Confers Durable Responses in High-Risk NMIBC

Treatment with NDV-01, an intravesical formulation of gemcitabine and docetaxel, conferred durable responses among patients with high-risk non–muscle invasive bladder cancer (NMIBC), according to a news release from the developer, Relmada Therapeutics.¹

Specifically, among 38 patients treated with the investigational agent in a phase 2 trial (NCT06663137), the 12-month complete response (CR) rate was 76%. Moreover, CRs were observed in 95% of patients, with respective 3-, 6-, and 9-month rates of 87%, 86%, and 85%. Moreover, among 17 patients with Bacillus Calmette-Guérin (BCG)-unresponsive disease, the 12-month CR rate was 80%. The overall CR rate was 94%, with respective 3-, 6-, and 9-month rates of 82%, 86%, and 91%.

Additionally, no patients on the trial progressed to muscle-invasive disease or underwent a radical cystectomy.

According to the developer, the findings support a best-in-class profile of NDV-01, supporting an advancement into a phase 3 RESCUE registrational program. This program would evaluate NDV-01 in the BCG-unresponsive and adjuvant intermediate-risk NMIBC groups.

“These 12-month data show the potential durability of NDV-01’s clinical response profile while continuing to demonstrate a clean safety profile,” Raj S. Pruthi, MD, chief medical officer-Oncology of Relmada Therapeutics, stated in the news release.¹ “Importantly, we continue to observe strong responses in patients with BCG-unresponsive disease, with no progression to muscle-invasive disease and no patients requiring radical cystectomy. We believe these interim results provide meaningful clinical validation of the program and support advancing NDV-01 into the registrational phase 3 RESCUE program with 2 separate registrational pathways: 2L BCG-unresponsive and adjuvant intermediate-risk, which we expect to initiate in mid-2026.”

Safety data revealed no patients experienced a grade 3 or higher treatment-related adverse effect (TRAE), and none discontinued treatment due to AEs. Among 48 patients in the safety population, 63% experienced any-grade TRAEs, the most common of which included transient uncomfortable urination lasting less than 24 hours (54%), asymptomatic positive urine culture (8%), and hematuria (8%).

Patients enrolled on the study were treated with 6 biweekly instillations of NDV-01 on days 1, 14, 28, 42, 56, and 70 of study treatment.² A maintenance phase comprised of monthly dosing occurring on days 107, 140, 187, 220, 250, 287, 310, and 340. Moreover, patients who did not meet a CR at permitted daily exposure underwent reintroduction of biweekly dosing followed by maintenance per protocol.

The primary end points of the study were CR rate in the high-grade NMIBC population and frequency and severity of AEs. Secondary end points included CR durability, CR rate in the carcinoma in situ population, and event-free survival.

“I am highly encouraged by NDV-01’s high response rates, 12-month durability and favorable tolerability profile. Building on the clinical community’s familiarity with conventional [gemcitabine/docetaxel], these phase 2 results provide robust validation of NDV-01’s novel sustained release formulation. In addition, NDV-01’s less than 5-minute administration simplifies dosing for clinical staff, supporting broad adoption in community urology practices where [approximately] 80% of [patients with] NMIBC are treated – and potentially offering a significantly more streamlined user experience than currently approved therapies,” said Max Kates, MD, director of Urologic Oncology at Johns Hopkins and Relmada Clinical Advisor, said in the news release.¹

The phase 3 RESCUE Registrational study will be an open-label randomized controlled trial among patients with intermediate-risk disease following treatment with transurethral resection of bladder tumor (TURBT). The primary end point will be disease-free survival, with secondary end points being high-grade recurrence free survival, progression-free survival, and quality of life metrics.

References

1. Relmada Therapeutics reports 12-Month phase 2 interim data for NDV-01 in non-muscle invasive bladder cancer. News release. Relmada Therapeutics. March 9, 2026. Accessed March 9, 2026. https://tinyurl.com/4jujdpdc
2. Open label study to evaluate the safety and efficacy of NDV01 KIT in high grade NMIBC (HGNMIBC). ClinicalTrials.gov. Updated June 13, 2025. Accessed March 9, 2026. https://tinyurl.com/4kj4c9n5