Relmada Therapeutics has secured key regulatory clarity from the US Food and Drug Administration, clearing a faster potential path to approval for its bladder cancer therapy NDV-01 in two high-need patient populations.

 

The clinical-stage biotechnology company said the FDA provided written feedback supporting a single-arm, open-label registrational trial for NDV-01 in patients with second-line, refractory, high-grade BCG-unresponsive non-muscle invasive bladder cancer with carcinoma in situ — one of the most aggressive and treatment-resistant forms of the disease.

 

In written responses to Relmada’s Type B pre-IND submission, the FDA indicated that this trial design would be appropriate for a potential new drug application, providing what the company called a clear and efficient development pathway for NDV-01 as a bladder-sparing therapy in a population with limited treatment options.

 

The agency also issued supportive written feedback for a separate registrational study of NDV-01 in intermediate-risk NMIBC in the adjuvant setting — an area where no approved therapies currently exist. That trial is expected to use an open-label, randomized-to-observation design.

 

Relmada plans to initiate both registrational trials in the first half of 2026.

 

“We are very pleased with the FDA’s alignment on the registrational design for NDV-01 in high-grade BCG-unresponsive NMIBC,” said Raj S. Pruthi, Chief Medical Officer – Oncology at Relmada Therapeutics. 

 

“A single-arm pivotal study in this setting represents a meaningful opportunity to advance an in-office, bladder-sparing therapy for patients who have few if any effective alternatives. This study represents the fastest path to approval for NDV-01.”

 

Dr Pruthi added, “We are also encouraged by the FDA’s feedback on our intermediate-risk registration plans, where we believe NDV-01 could potentially provide meaningful clinical benefit to patients where no approved treatments currently exist.”

 

The planned high-grade registrational study will be a pivotal Phase 3, open-label, single-arm trial evaluating NDV-01 in second-line, refractory, high-grade BCG-unresponsive NMIBC patients with carcinoma in situ. 

 

The primary endpoint will be complete response rate at any time, with duration of response as a key secondary endpoint. Patients will be assessed through cystoscopy, cytology and biopsy, in line with FDA guidance and prior regulatory precedents for NMIBC approvals.

 

The planned intermediate-risk registrational study will also be a pivotal Phase 3 trial, conducted in the adjuvant setting using an open-label, randomized-to-observation design. The primary endpoint will be disease-free survival, with duration of response as a key secondary endpoint, and similar assessment methods.

 

Relmada said both study designs closely follow the FDA’s written guidance on patient populations, endpoint selection and evaluation methodology.