Sustained-release gemcitabine/docetaxel formulation shows durable responses in NMIBC

Interim results from a phase 2 study (NCT06663137) suggest that the investigational intravesical therapy NDV-01 may produce durable responses in patients with high-risk non–muscle invasive bladder cancer (NMIBC), particularly among those with BCG-unresponsive disease. At 12 months, the therapy achieved a complete response (CR) rate of 76% in the overall study population and 80% among patients with BCG-unresponsive disease, Relmada Therapeutics announced in a news release.¹

According to Relmada, NDV-01 is a sustained-release intravesical formulation of gemcitabine and docetaxel that is under investigation for the treatment of patients with NMIBC. The formulation is designed to create a soft intravesical matrix that retains the drugs in the bladder and releases them gradually over approximately 10 days. By prolonging bladder exposure, the therapy aims to enhance local drug activity while minimizing systemic toxicity.

“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 gem/doce, these phase 2 results provide robust validation of NDV-01’s novel sustained release formulation,” said Max Kates, MD, director of Urologic Oncology at Johns Hopkins Medicine, Baltimore, Maryland, and a Clinical Advisor for Relmada. “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 NMIBC patients are treated, potentially offering a significantly more streamlined user experience than currently approved therapies.”

In total, the open-label, single-arm, single-center, phase 2 trial included 48 patients who received at least 1 dose of NDV-01. Patients with NMIBC were eligible for the study if they were aged 18 to 80 years of age, had an ECOG status of 2 or less, and were ineligible for or elected not to undergo radical cystectomy.² The treatment regimen consisted of a biweekly induction phase followed by monthly maintenance therapy for up to 1 year, with disease monitoring through cystoscopy, cytology, and biopsy when clinically indicated. The primary end points are safety and CR rate at 12 months. Secondary end points included duration of response (DOR) and event-free survival.

Across the overall study population, response rates remained relatively stable over time. The CR rate at any time point was 95%, with rates of 87% at 3 months, 86% at 6 months, 85% at 9 months, and 76% at 12 months. Kaplan-Meier analysis estimated a 12-month CR probability of 83%.

Among the subset of patients with BCG-unresponsive disease (n = 20), investigators reported a CR rate of 94% at any time point, with rates of 82% at 3 months, 86% at 6 months, 91% at 9 months, and 80% at 12 months. In this subgroup, the Kaplan-Meier–estimated CR rate at 12 months was 84%.

No patients in the study experienced progression to muscle-invasive disease nor underwent a radical cystectomy.

Treatment-related adverse events (TRAEs) occurred in 63% of patients, most commonly transient dysuria lasting less than 24 hours (54%). Other reported adverse events included asymptomatic positive urine cultures (8%) and hematuria (8%). No TRAEs of grade 3 or higher were reported, and no patients discontinued therapy due to adverse events.

“These 12-month data show the potential durability of NDV-01’s clinical response profile while continuing to demonstrate a clean safety profile,” added Raj S. Pruthi, MD, Chief Medical Officer of Oncology at Relmada Therapeutics, 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.”

Registrational pathway 1 involves an open-label randomized controlled trial of NDV-01 vs observation as an adjuvant therapy following transurethral resection of bladder tumor (TURBT) in patients with intermediate-risk NMIBC. The primary end point is disease free survival, with key secondary end points including high-grade recurrence-free survival, progression-free survival (PFS), and quality of life metrics.

Registrational pathway 2 involves a single-arm trial of NDV-01 as a second line therapy for patients with BCG-unresponsive NMIBC with carcinoma in situ (CIS) who are currently refractory to approved or developmental therapies. The primary end point is CR rate at any time point, and key secondary end points include DOR, PFS, and recurrence-free survival among responders.

The planned phase 3 RESCUE program is expected to begin in mid-2026, with initial 3-month results from registrational pathway 2 expected by the end of 2026.

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://www.relmada.com/for-investors/news/detail/332/relmada-therapeutics-reports-12-month-phase-2-interim-data

2. Open Label Study to Evaluate the Safety and Efficacy of NDV01 KIT in High Grade NMIBC (HGNMIBC). ClinicalTrials.gov. Last updated June 13, 2025. Accessed March 9, 2026. https://clinicaltrials.gov/study/NCT06663137