NDV-01 Shows Durable Responses in Bladder Cancer Study

A new investigational therapy may offer hope for patients with non–muscle-invasive bladder cancer (NMIBC), particularly those who have limited treatment options or whose disease returns after standard therapies, according to a news release from Relmada Therapeutics, Inc. Early results from an ongoing phase 2 clinical trial suggest that the novel sustained-release chemotherapy combination NDV-01 may produce durable responses with a favorable safety profile.

At 12 months of follow-up, interim data from the study showed strong response rates among patients treated with NDV-01. The therapy previously demonstrated a complete response rate of approximately 92% at some point during treatment, with durable responses continuing over time. Importantly, researchers reported that no patients in the study progressed to muscle-invasive disease or required removal of the bladder, outcomes that are particularly meaningful for patients seeking bladder-preserving treatment options.

“These 12-month data show the potential durability of NDV-01’s clinical response profile while continuing to demonstrate a clean safety profile,” Dr. Raj S. Pruthi, chief medical officer, Oncology, Relmada Therapeutics, said in a news release. “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.”

NDV-01 shows notable response rates in NMIBC

NDV-01 is an investigational treatment designed for delivery directly into the bladder, a method known as intravesical therapy. The drug combines two established chemotherapy agents in a sustained-release formulation that gradually delivers medication over approximately 10 days. This approach is designed to maximize exposure of cancer cells to the drugs while limiting systemic side effects.

In the phase 2 study evaluating NDV-01 in patients with high-grade NMIBC, the therapy demonstrated strong clinical activity. Earlier analyses showed a complete response in 92% of treated patients at some point during therapy. Response rates remained high over time, with 84% of patients responding at three months, 87% at six months, and 85% at nine months.

The treatment also appeared safe and well tolerated. Researchers reported no treatment-related side effects classified as grade 3 or higher, and no participants stopped treatment because of adverse events. In addition, none of the patients progressed to muscle-invasive disease or required radical cystectomy, a major surgery that removes the bladder.

These results suggest that NDV-01 may provide durable disease control for patients with NMIBC, particularly those whose disease is considered high risk.

Understanding NMIBC

Bladder cancer is among the most common cancers worldwide, and NMIBC accounts for approximately 75% to 80% of newly diagnosed cases. Although this form of the disease has not invaded the bladder muscle, it often returns after treatment and requires ongoing monitoring and repeated therapies.

Standard treatment typically begins with surgery to remove tumors through a procedure called transurethral resection of bladder tumor (TURBT). Patients may then receive intravesical therapy, such as Bacillus Calmette-Guérin (BCG), to reduce the risk of recurrence. However, some patients do not respond to BCG or experience disease recurrence, leaving them with limited options.

Because of this unmet need, researchers are actively studying new therapies that can control the disease while allowing patients to keep their bladder.

Phase 2 Trial Design and Patient Population

The ongoing phase 2 study evaluating NDV-01 is a single-arm, open-label clinical trial designed to assess both safety and efficacy in patients with high-grade NMIBC. The trial enrolled up to 70 participants with localized, non-metastatic disease and an Eastern Cooperative Oncology Group performance status of 2 or lower.

Participants in the study represent a range of clinical scenarios, including patients who were BCG-naïve, those previously exposed to BCG, and patients whose disease did not respond to BCG treatment. In earlier analyses, the median patient age was 73 years, reflecting the older population commonly affected by bladder cancer.

The primary goals of the trial include evaluating safety and measuring the complete response rate at 12 months. Researchers are also examining secondary outcomes such as duration of response and event-free survival.

Additional findings and next steps for NDV-01

Beyond encouraging response rates, researchers noted several additional findings that highlight NDV-01’s potential advantages. The sustained-release formulation allows the chemotherapy drugs to remain in the bladder longer than traditional intravesical therapies, potentially improving effectiveness while simplifying administration.

Investigators also observed that some patients who required re-induction therapy still achieved responses, with a reported 60% complete response rate after re-treatment in earlier analyses.

“I am highly encouraged by NDV-01’s high response rates, 12-month durability and favorable tolerability profile... 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 [about] 80% of NMIBC patients are treated, and potentially offering a significantly more streamlined user experience than currently approved therapies,” concluded Dr. Max Kates, director, Urologic Oncology, Johns Hopkins and Relmada Clinical Advisor.

Based on the positive phase 2 findings, Relmada Therapeutics plans to move the therapy into phase 3 clinical trials. The next stage of research will evaluate NDV-01 in two settings: high-risk NMIBC that does not respond to BCG and intermediate-risk NMIBC following surgery.

Editor's note: This article is for informational purposes only and is not a substitute for professional medical advice, as your own experience will be unique. Use this article to guide discussions with your oncologist. Content was generated with AI, reviewed by a human editor, but not independently verified by a medical professional.

References

1. “Relmada Therapeutics Reports 12-Month Phase 2 Interim Data for NDV-01 in Non-Muscle Invasive Bladder Cancer,” by Relmada Therapeutics, Inc. News release; March 9, 2026.

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