Relmada Therapeutics reported that the U.S. Food and Drug Administration has issued written feedback confirming the company’s proposed registrational study designs for its lead candidate NDV‑01. The FDA approved a single‑arm, open‑label trial for patients with high‑grade, BCG‑unresponsive non‑muscle invasive bladder cancer (NMIBC) with carcinoma in situ, and a randomized‑to‑observation design for intermediate‑risk NMIBC in the adjuvant setting.
The approval of these study designs provides a clear and efficient development path toward a potential New Drug Application for NDV‑01. The single‑arm trial will evaluate the efficacy and safety of the sustained‑release gemcitabine/docetaxel formulation in a high‑risk population that currently has no approved bladder‑sparing options, while the randomized study will assess benefit in a broader intermediate‑risk cohort. Relmada’s CEO, Sergio Traversa, said the FDA’s alignment removes a major regulatory hurdle and accelerates the company’s timeline to initiate both registrational trials in the first half of 2026.
Relmada’s financial position supports this aggressive schedule. The company raised $100 million in November 2025, extending its cash runway through 2028 and providing sufficient liquidity to fund the two Phase 3 programs. The company’s management emphasized that the funding strategy and current cash balance give it the flexibility to execute the trials without additional capital raises in the near term.
The market opportunity for NDV‑01 is substantial. Non‑muscle invasive bladder cancer accounts for 75‑80% of all bladder cancer cases, with over 744,000 prevalent cases in the U.S. The high‑grade, BCG‑unresponsive population has no approved bladder‑sparing therapies, while the intermediate‑risk adjuvant setting also lacks approved options. NDV‑01’s sustained‑release intravesical formulation delivers gemcitabine and docetaxel over ten days, can be administered in‑office in less than five minutes, and offers a convenient, bladder‑sparing alternative to radical cystectomy.
Management highlighted the clinical promise of NDV‑01. Chief Medical Officer Raj S. Pruthi noted that the single‑arm pivotal study represents the fastest path to approval for a therapy that could transform care for patients with few alternatives. He added that the FDA’s feedback on the intermediate‑risk registration plans signals confidence that NDV‑01 could provide meaningful benefit where no approved treatments exist.
The FDA’s guidance on single‑arm trials for BCG‑unresponsive NMIBC, issued in August 2024, supports Relmada’s approach. The company’s Phase 2 data, including a 91% complete response rate at six months and a 100% 3‑month complete response rate in BCG‑naïve high‑risk patients, further strengthen the case for the upcoming Phase 3 studies.
Overall, the FDA’s confirmation of the registrational pathway marks a pivotal milestone for Relmada, de‑risking the development of NDV‑01 and positioning the company to address a large, unmet market with a convenient, bladder‑sparing therapy.
The company expects to begin enrollment in the high‑grade trial shortly after the FDA’s clearance, with the intermediate‑risk study to follow in the same period, setting the stage for a potential NDA submission in the late 2020s.
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