NDV-01 produces robust 9-month results in high-risk NMIBC

NDV-01, an investigational, intravesical sustained-release gemcitabine docetaxel combination agent designed for the treatment of non–muscle invasive bladder cancer (NMIBC), displayed promising efficacy and safety in a single-arm, open-label phase 2 trial (NCT06663137 )presented at the 2025 Society of Urologic Oncology Annual Meeting in Phoenix, Arizona.¹

In a news release from developer Relmada Therapeutics, the company explained that NDV-01 “is designed to enable Gem/Doce bladder retention and gradual drug release over 10 days. The formulation creates a soft matrix that enhances local exposure while minimizing systemic toxicity. The NDV-01 formulation is a ready-to-use, convenient to administer in-office in less than 10 minutes, and does not require anesthesia or specialized equipment.”²

Speaking with Urology Times study co-author and Relmada Chief Medical Officer-Oncology Raj S. Pruthi, MD, MHA, FACS, discussed the unique nature of NDV-01.

“An advantage of this [treatment] is there's no bio containment; there's no thawing and so forth, so it’s very easy to manage for the community urologist. The problem with gem-doce is that you need a hospital/specialized pharmacy, so this has pre-filled syringes that takes that out, and it has the time savings,” Pruthi said.

Patients with high-risk NMIBC with carcinoma in situ (CIS)/Tis, Ta, T1 tumors and patients who were BCG-naïve, BCG-unresponsive, or BCG-intolerant were eligible for inclusion in the single-arm, open-label study, which was led by Yair Lotan, MD, a professor of urology, chief of Urologic Oncology, and holder of the Jane and John Justin Distinguished Chair in Urology, in Honor of Claus G. Roehrborn, M.D. at UT Southwestern Medical Center in Dallas, Texas. The treatment regimen consisted of 6 bi-weekly instillations followed by monthly maintenance instillations through month 12. For the purposes of the study, CR was defined as a negative cystoscopy, cytology, and biopsy (if indicated). At 3 months, the first assessment for CR was conducted, at which point patients who did not have a CR were eligible to receive re-induction with an additional 6 bi-weekly treatment course. The primary end points were safety and CR rate at 12 months, and secondary end points included duration of response and event-free survival. Pharmacokinetics was an exploratory end point.

A total of 36 patients were enrolled in the study, all of whom had an ECOG performance status of 0-1. Thirty (83%) patients were male and 6 (17%) were female. Median age in the cohort was 73 years (range, 54-93 years). The median number of BCG doses received was 6 (range, 0-21 doses). Fifteen (42%) patients were BCG-naïve, 4 (11%) were BCG exposed, and 17 (47%) were BCG responsive. Disease stage was pure CIS in 3 (8%) patients, Ta/T1 plus CIS in 7 (19%), Ta in 21 (58%), and T1 in 6 (17%).

CR at any time was 92% (23/25 patients), 84% (21/25) at 3 months, 87% (20/23) at 6 months, and 85% (17/20) at 9 months. Out of the 36 patients who received at least 1 dose of NDV-01, 22 (61%) had treatment-related adverse event (TRAE); of these 62% were dysuria, 9% were asymptomatic positive urine culture, and 7% were hematuria. No grade 3 or higher TRAEs were observed, and there were no treatment discontinuations due to AEs. In addition, none of the patients in the cohort progressed to muscle-invasive disease or underwent radical cystectomy.

“NDV-01 provides excellent 9-month safety and efficacy in patients with high-risk NMIBC. Data support effectiveness in patients who are BCG-naïve, -exposed, and –unresponsive,” the authors wrote.

On the poster, the investigators explained that pivotal studies in the intermediate-risk and high-risk BCG-unresponsive NMIBC spaces are planned for the first half of 2026.

“We believe NDV-01 has the opportunity to transform the treatment of NMIBC by providing patients and physicians with a potential bladder-sparing, in-office, ready-to-use, safe, effective and durable, best-in-class therapy. We are on track to initiate the phase 3 program in H1 2026, building on the encouraging, recently announced 9-month data, showing a 92% complete response (CR) rate at any time point, and encouraging recent FDA discussions, which provide us with a well-defined registrational strategy in two distinct indications in NMIBC with limited treatment options,” Pruthi said in the news release.

REFERENCES

1. Lotan Y, Pruthi R, Greene P, et al. Prospective open-label study to evaluate the safety and efficacy of intravesical sustained-release gemcitabine docetaxel combination (NDV-01) in high-risk NMIBC: Update with 6-month complete response data. Presented at: Society of Urologic Oncology Annual Meeting; December 2-5, 2025; Phoenix, Arizona. Abstract 143. https://suo-abstracts.secure-platform.com/a/gallery/rounds/24/details/4577

2. Relmada Therapeutics announces presentation of NDV-01 phase 2 data at the Society for Urologic Oncology. News release. Relmada Therapeutics. December 3, 2025. Accessed December 4, 2025. https://www.relmada.com/for-investors/news/detail/330/relmada-therapeutics-announces-presentation-of-ndv-01-phase