Intravesical gemcitabine/docetaxel formulation shows encouraging efficacy in NMIBC

NDV-01, an intravesical formulation of gemcitabine/docetaxel, demonstrated encouraging safety and initial efficacy in patients with high-grade non–muscle invasive bladder cancer (NMIBC), according to 6-month follow-up data from a phase 2 trial (NCT06663137) of the agent.¹

According to the Relmada Therapeutics, NDV-01 is “designed to enable gem/doce bladder retention and gradual drug release over 10 days.” Treatment with NDV-01 does not require anesthesia or specialized equipment.

“As clinicians, we have embraced the combination of gem/doce as a highly effective therapy for many years,” said Yair Lotan, MD, chair of Relmada’s Clinical Advisory Board, in a news release from the company.¹ “However, it is difficult to formulate outside of the hospital setting, often creating a significant burden for patient and provider. NDV-01’s simple, ready-to-use sustained release formulation overcomes those burdens and could enable wider patient adoption, transforming the care of NMIBC.”

The current 6-month data analysis included 29 patients, of whom 24 (83%) were male and 5 (17%) were female. The median age of participants was 73 years (range, 54 to 93), and the median number of BCG doses at baseline was 6 (range, 0 to 18). Among all patients, 3 (10%) presented with pure carcinoma in situ (CIS), 4 (14%) presented with Ta/T1 disease plus CIS, 18 (62%) presented with Ta disease, and 4 (14%) presented with T1 disease.

Overall, data showed a complete response (CR) rate at any time point of 91% (21 of 23). At 3- and 6-months, the CR rates were 83% (19 of 23) and 90% (19 of 21), respectively. Further, 1 patient had reached 9-month follow-up and had a CR.

Regarding safety, no grade 3 treatment-related adverse events (AEs) were reported, and no patients discontinued treatment due to AEs. Further, no patients had progressed to muscle-invasive disease, and no patient underwent a radical cystectomy.

“We are pleased to report that the 6-month follow-up from the phase 2 study of NDV-01, a gemcitabine/docetaxel (Gem/Doce) sustained release formulation, produced impressive results, with a 91% CR rate at any time point following NDV-01 treatment,” said Raj S. Pruthi, MD, CMO of Relmada, in a news release from the company.¹ “The data reported today, in combination with the previously reported 3-month results, raise our confidence in NDV-01 as a potential durable treatment for bladder cancer.”

In total, the prospective, open-label phase 2 trial plans to enroll 70 participants with high-grade NMIBC.² To be eligible for enrollment, patients needed to have an ECOG status of 2 or less and a life expectancy of over 2 years. Additionally, patients must be ineligible for or have elected not to undergo radical cystectomy.

Those enrolled in the study receive NDV-01 in a biweekly induction phase followed by monthly maintenance for up to 1 year. Patients will also undergo regular assessments via cystoscopy, cytology, and biopsy, as indicated.

The primary end points for the trial are safety and the 12-month CR rate. Secondary end points include duration of response and event-free survival.

Final completion of the trial is expected in August 2026. According to Remalda, the company plans to initiate a phase 3 registrational trial of NDV-01 in the first half of 2026.

“Gem/doce have long shown clinical utility in NMIBC, but the complexity of administration has limited their broader use,” Pruthi added in the news release.¹ “The sustained release formulation of NDV-01, which is designed to increase exposure to drug and simplify delivery and accessibility, has the potential to significantly change how we manage patients with NMIBC in routine practice.”

REFERENCES

1. Relmada Therapeutics reports second quarter 2025 financial results and announces NDV-01 6-month follow-up safety and efficacy data in NMIBC. News release. Relmada Therapeutics. August 7, 2025. Accessed August 8, 2025. https://www.globenewswire.com/news-release/2025/08/07/3129672/0/en/Relmada-Therapeutics-Reports-Second-Quarter-2025-Financial-Results-and-Announces-NDV-01-6-Month-Follow-up-Safety-and-Efficacy-Data-in-NMIBC.html

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 August 8, 2025. https://clinicaltrials.gov/study/NCT06663137