Relmada Therapeutics Secures FDA Alignment for NDV-01 Registrational Studies
On January 12, 2026, Relmada Therapeutics, Inc. (Nasdaq: RLMD) announced significant progress in its drug development strategy with regulatory feedback from the U.S. Food and Drug Administration (FDA). The FDA has provided essential guidance supporting the design of registrational studies for NDV-01, intended for treating high-grade non-muscle invasive bladder cancer (NMIBC) and intermediate-risk NMIBC.
FDA Guidance on Registrational Pathway
In its written response to Relmada’s Type B pre-Investigational New Drug (IND) submission, the FDA has approved a single-arm, open-label registrational trial for NDV-01 in patients suffering from 2nd-line refractory high-grade BCG-unresponsive NMIBC with carcinoma in situ (CIS). This population represents one of the highest-risk groups for NMIBC and has few effective treatment options.
The FDA has also indicated support for a separate registrational study in intermediate-risk NMIBC in the adjuvant setting, which is set to follow an open-label, randomized-to-observation design. Relmada plans to initiate both trials in the first half of 2026.
Statements from Leadership
“We are very pleased with the FDA’s alignment on the registrational design for NDV-01 in high-grade BCG-unresponsive NMIBC,” commented Dr. Raj S. Pruthi, Chief Medical Officer – Oncology at Relmada Therapeutics. “This pivotal study represents a critical opportunity to develop a bladder-sparing therapy in a population that currently has limited options.”
Dr. Pruthi added, “We are encouraged by the FDA’s feedback regarding our plans for the intermediate-risk registration study, where NDV-01 could bring meaningful benefits to patients lacking approved therapies.”
Details on the High-Grade Registrational Study
The pivotal Phase 3 study in high-grade BCG-unresponsive NMIBC will evaluate:
- Primary Endpoint: Complete response (CR) rate at any time
- Key Secondary Endpoint: Duration of response (DOR)
- Assessments: Cystoscopy, cytology, and biopsy per protocol
This trial design aligns with the FDA's previous guidance regarding endpoint selection and evaluation methodologies.
Overview of the Intermediate-Risk Registrational Study
The planned pivotal Phase 3 study for intermediate-risk NMIBC will be structured as an open-label randomized-to-observation trial, focusing on:
- Primary Endpoint: Disease Free Survival (DFS)
- Key Secondary Endpoint: Duration of response (DOR)
- Assessments: Cystoscopy, cytology, and biopsy per protocol
This study's design also adheres to the FDA's recommendations regarding endpoint selection and methodologies.
About NDV-01
NDV-01 is an innovative sustained-release intravesical formulation of gemcitabine and docetaxel (Gem/Doce) under development for NMIBC. The formulation is designed to facilitate bladder retention of the drug and ensure gradual release over ten days, enhancing local exposure while limiting systemic toxicity. Notably, NDV-01 can be administered in-office within five minutes and does not require anesthesia or special equipment. The product is under patent protection until 2038.
Understanding NMIBC
NMIBC constitutes approximately 75-80% of all bladder cancer cases and is known for high recurrence rates (50-80% over five years). With over 744,000 cases prevalent in the U.S. and scarce treatment options, there is a significant market potential. High-grade BCG-unresponsive NMIBC is particularly challenging to treat, emphasizing the need for effective therapies like NDV-01.
About Relmada Therapeutics
Relmada Therapeutics is committed to developing groundbreaking therapies for oncology and central nervous system disorders. Its lead candidates, including NDV-01 and sepranolone, are progressing through mid-stage clinical development to address critical unmet medical needs.
For more information, visit Relmada Therapeutics.
Forward-Looking Statements
This release contains forward-looking statements as defined by the Private Securities Litigation Reform Act of 1995. These statements are subject to various risks and uncertainties that may cause actual results to differ materially from those anticipated. Readers are encouraged to review the full risk factors documented in Relmada's reports filed with the SEC.