Relmada Therapeutics (NASDAQ:RLMD) outlined progress on its lead urology oncology program and reviewed fourth-quarter results during its fourth quarter and full year 2025 earnings call, highlighting durable Phase 2 data for NDV-01 in non-muscle invasive bladder cancer (NMIBC), plans to begin a Phase 3 registrational program in mid-2026, and upcoming clinical work for sepranolone.
NDV-01 Phase 2 results and clinical profile
Chief Executive Officer Dr. Sergio Traversa said 2025 was a “transformational year” for the company, pointing to “compelling responses and durable 12-month efficacy data” from the ongoing Phase 2 study of NDV-01. NDV-01 is a sustained-release intravesical formulation of gemcitabine and docetaxel being developed for NMIBC.
Pruthi reported the following efficacy and safety outcomes discussed on the call:
- 12-month complete response (CR) rate: 76% overall.
- 12-month CR rate in BCG-unresponsive patients: 80%.
- CR at any time: 95% overall (based on 38 patients) and 94% among BCG-unresponsive patients.
- Safety: No progression to muscle-invasive disease, no radical cystectomies, no grade 3 or higher treatment-related adverse events, and no interruptions or discontinuations due to adverse events; most treatment-related adverse events were grade 1.
Pruthi also emphasized administration and design elements he believes could help NDV-01 fit in routine practice, saying the product is designed to form a “soft matrix” in the bladder to enhance local exposure while minimizing systemic toxicity, and that it can be delivered in an office setting in “less than 5 minutes.”
Phase 3 RESCUE program design and timelines
Management said it reached alignment with the FDA on a Phase 3 registration strategy built around two independent pathways within the planned RESCUE program. Traversa said the company plans to initiate the Phase 3 program in the middle of 2026, while Pruthi added the company expects to secure U.S. IND clearance and begin RESCUE in mid-2026 at an estimated 80 sites in North America.
The two registrational studies described were:
- Pathway 1 (intermediate-risk adjuvant post-TURBT): An open-label randomized controlled trial evaluating NDV-01 versus observation after TURBT. The primary endpoint is disease-free survival, with secondary endpoints including high-grade recurrence-free survival, progression-free survival, and quality-of-life metrics. Pruthi estimated about 70,000 to 75,000 U.S. patients per year in this setting, and Traversa cited approximately 75,000.
- Pathway 2 (BCG-unresponsive carcinoma in situ, second-line): A single-arm open-label trial in patients with BCG-unresponsive CIS who are refractory to first-line therapies. The primary endpoint is CR at any time, with secondary endpoints including duration of response, progression-free survival, and recurrence-free survival among responders. Pruthi estimated about 5,000 U.S. patients per year in this setting.
Pruthi said the company expects to report initial 3-month response data from the second-line BCG-unresponsive study by the end of 2026. In the Q&A, he added that once enrollment begins mid-year, the company expects it will be able to share 3-month data externally by year-end and then provide additional follow-up data roughly every three months as 6-, 9-, and 12-month data mature.
Conference Q&A: disclosures on presentations, prior therapy limits, and FDA feedback
During questions, Pruthi said the company’s 12-month Phase 2 NDV-01 data have been accepted for presentation at the American Urological Association (AUA) meeting, and that the company also expects to present a “trial in progress” as RESCUE gets underway.
In response to investor questions about whether the second-line study could include more heavily pretreated patients, Pruthi said the protocol limits prior therapies to a maximum of two lines, offering an example of allowing prior Adstiladrin followed by Anktiva. He also said the company plans to look at outcomes at three months among the first 15 patients stratified by whether they received one versus two prior therapies, and noted this approach reflects conversations with the FDA.
Asked whether the FDA stipulated a minimum follow-up duration for all patients prior to a potential NDA submission in the second-line setting, Pruthi said the agency had not required a specific minimum follow-up time, but indicated it wants to see a response along with some level of durability, describing the agency’s interest as the “totality of the data.”
On enrollment, Pruthi acknowledged the BCG-unresponsive space has been crowded historically, but argued that RESCUE’s second-line positioning offers a competitive advantage, adding that he was not aware of other pivotal studies in that specific second-line setting. For intermediate-risk disease, he cited investigator interest in the category and said he expects enrollment could be “pretty rapidly ahead of schedule,” referencing another company’s experience in intermediate-risk trials.
On how the company is thinking about benchmarks, Pruthi discussed historical complete response data in first-line BCG-unresponsive CIS therapies, citing Valrubicin, Keytruda, and Adstiladrin, and said he believes second-line expectations should be in line with or lower than first-line levels as a precedent. In response to questions about CIS versus papillary disease comparisons, he referenced a 2020 Journal of Urology paper by Steinberg describing similar outcomes for gemcitabine/docetaxel in CIS and papillary populations, and noted Relmada’s own CIS subset numbers were small.
Sepranolone: planned proof-of-concept study in Prader-Willi syndrome
Chief Financial Officer Maged Shenouda reviewed the company’s plans for sepranolone, describing it as a “GABA Modulating Steroid Antagonist” (GAMSA). He said sepranolone’s mechanism could have potential across compulsive disorders, including obsessive-compulsive disorder, Tourette syndrome, and Prader-Willi syndrome. Shenouda said the company is preparing to initiate a proof-of-concept study in Prader-Willi syndrome in mid-2026, with immediate efforts focused on study preparations, FDA engagement on trial design, and establishing a supply chain. Traversa also noted sepranolone previously demonstrated proof-of-concept in Tourette syndrome.
Financial update: cash position and fourth-quarter results
Shenouda said Relmada closed 2025 with $93 million in cash, compared with approximately $45 million in cash equivalents and short-term investments at December 31, 2024. He stated the year-end cash balance included net proceeds of approximately $94 million from an underwritten stock offering announced November 5, 2025.
He also discussed a $160 million private financing announced March 9, 2025, with net proceeds of approximately $160 million. Management said the financing, together with year-end cash, is expected to fund operations through 2029, including completion of the Phase 3 RESCUE program.
For the fourth quarter, Shenouda reported:
- R&D expense: $8.1 million for Q4 2025 versus $11.0 million for Q4 2024, with the decrease primarily tied to reduced study costs following completion of two Phase 3 trials for REL-1017, partially offset by costs related to NDV-01 Phase 3 startup, a sepranolone Phase 2b study, and additional R&D personnel.
- G&A expense: $12.3 million for Q4 2025 versus $8.1 million for Q4 2024, driven primarily by higher compensation costs, partially offset by lower stock compensation costs.
- Net cash used in operating activities: $14.6 million for Q4 2025 versus $8.8 million for Q4 2024.
- Net loss: $19.9 million, or $0.27 per basic and diluted share, for Q4 2025, compared with a net loss of $18.7 million, or $0.62 per basic and diluted share, for Q4 2024.
In closing remarks, Traversa said the company is focused on execution as it prepares to initiate the RESCUE registrational program and plans to update investors in coming quarters.
About Relmada Therapeutics (NASDAQ:RLMD)
Relmada Therapeutics, Inc is a clinical-stage biopharmaceutical company focused on the development of novel therapies for pain and other central nervous system (CNS) disorders. The company applies a proprietary stereochemical approach to optimized drug candidates, aiming to improve safety, tolerability and efficacy profiles compared with existing treatments. Relmada’s research efforts center on modulation of NMDA receptors to address unmet needs in depression, neuropathic pain and related indications.
Relmada’s lead product candidate, REL-1017 (d-methadone), is being evaluated as a potential rapid-acting and maintenance treatment for major depressive disorder, with clinical studies underway to assess its utility in both acute and long-term settings.
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