8-K: Relmada Reports Strong NDV-01 Data, Q2 Loss Narrows

Relmada Therapeutics announced positive 6-month follow-up data for its NDV-01 bladder cancer therapy, alongside narrowed Q2 2025 net loss and plans for Phase 3 initiation.

Summary

• NDV-01 6-month follow-up data in non-muscle invasive bladder cancer (NMIBC) showed a 91% overall response rate (ORR) at any time, with good overall safety.
• Enrollment in the Phase 2 study for NDV-01 continues, with updates expected at 9 and 12-month data follow-up.
• Preparations are underway to start a Phase 3 registration trial for NDV-01 in the first half of 2026.
• A Phase 2 study for sepranolone in Prader-Willi syndrome is expected to initiate in the first half of 2026.
• Net loss for the second fiscal quarter ended June 30, 2025, was $9.9 million, or $0.30 per share, compared to $17.8 million, or $0.59 per share, for the same period in 2024.
• Research and Development (R&D) expense decreased to $2.8 million in Q2 2025 from $10.7 million in Q2 2024.
• General and Administrative (G&A) expense decreased to $7.4 million in Q2 2025 from $8.1 million in Q2 2024.
• Cash, cash equivalents, and short-term investments were $20.6 million as of June 30, 2025, down from $44.9 million at year-end 2024.
• Shares outstanding were 33,191,622 as of August 4, 2025.

Sentiment

Score: 8

Explanation: The strong clinical data for NDV-01, particularly the high response rate and good safety profile, coupled with clear plans for Phase 3 initiation, are highly positive. The significant reduction in net loss and operating expenses also indicates improved financial management. While cash burn remains a concern, the clinical progress in a high-value indication outweighs the current financial position for a clinical-stage biotech.

Positives

• NDV-01 6-month follow-up data showed an impressive 91% overall response rate at any time in non-muscle invasive bladder cancer.
• No patient treated with NDV-01 had progression to muscle invasive disease.
• No patient treated with NDV-01 underwent a radical cystectomy.
• No patient treated with NDV-01 experienced a Grade 3 or higher Treatment Related Adverse Event (TRAE), and no patients discontinued treatment due to AEs.
• Net loss significantly narrowed to $9.9 million in Q2 2025 from $17.8 million in Q2 2024, indicating improved financial performance.
• Research and Development (R&D) expense decreased by 73.8% to $2.8 million in Q2 2025 from $10.7 million in Q2 2024, primarily due to the wind-down of REL-1017 trial costs.
• General and Administrative (G&A) expense decreased by 8.6% to $7.4 million in Q2 2025 from $8.1 million in Q2 2024.
• Net cash used in operations decreased to $24.5 million for the first half of 2025 from $26.3 million for the first half of 2024.
• Sepranolone has shown positive Phase 2 Tourette data with a 28% drop in tic severity and consistent positive impact on secondary Quality of Life measures.
• Sepranolone has an excellent safety profile, with over 335 patients treated in clinical trials to date.

Negatives

• Cash, cash equivalents, and short-term investments decreased significantly to $20.6 million as of June 30, 2025, from $44.9 million at year-end 2024, indicating substantial cash burn.
• The accumulated deficit increased to $(668,307,942) as of June 30, 2025, from $(640,882,035) at year-end 2024, reflecting ongoing losses.
• The company continues to operate at a net loss.

Risks

• Potential for product candidates to not progress as expected.
• Clinical trials may fail to deliver statistically and/or clinically significant evidence of efficacy and/or safety.
• Top-line results may not accurately reflect the complete results of the trial.
• Planned or ongoing preclinical and clinical studies may fail to demonstrate expected results.
• Potential failure to secure FDA agreement on the regulatory path for NDV-01 and sepranolone.
• Future NDV-01 or sepranolone clinical results may not be acceptable to the FDA.
• Failure to secure adequate NDV-01 or sepranolone drug supply.
• It is not possible to predict or identify all risks, uncertainties, and other factors that may affect future results.

Future Outlook

Relmada Therapeutics plans FDA interactions and product supply scale-up for NDV-01 in the second half of 2025, aiming to initiate a Phase 3 registration-track study in the first half of 2026. For sepranolone, the company expects FDA engagement and manufacturing activities in the second half of 2025, with a planned Phase 2 study launch in Prader-Willi syndrome in the first half of 2026. Further updates for NDV-01 Phase 2 study are expected at 9 and 12-month data follow-up.

Management Comments

• "The six-month follow-up from the Phase 2 study of NDV-01, a gemcitabine/docetaxel (Gem/Doce) sustained release formulation, produced impressive results, with a 91% CR rate at any time point following NDV-01 treatment." Raj S. Pruthi, MD, CMO of Relmada.
• "The data reported today, in combination with the previously reported 3-month results, raise our confidence in NDV-01 as a potential durable treatment for bladder cancer." Raj S. Pruthi, MD, CMO of Relmada.
• "The sustained release formulation of NDV-01, which is designed to increase exposure to drug and simplify delivery and accessibility, has the potential to significantly change how we manage patients with NMIBC in routine practice." Raj S. Pruthi, MD, CMO of Relmada.
• "NDV-01’s simple, ready-to-use sustained release formulation overcomes those burdens and could enable wider patient adoption transforming the care of NMIBC." Yair Lotan, MD, Chair of Relmada’s Clinical Advisory Board.

Industry Context

The announcement positions Relmada Therapeutics as a key player in the non-muscle invasive bladder cancer (NMIBC) and rare neurological disorder markets. NDV-01 addresses a significant unmet need in NMIBC, a condition with high recurrence rates and limited treatment options, especially given existing supply issues for standard therapies like BCG. The sustained-release formulation aims to improve upon the complexity of administration of current Gem/Doce treatments, potentially expanding patient access. Sepranolone targets Prader-Willi Syndrome, an orphan disease with an estimated 20,000 US prevalence, where current treatments only address symptoms, not underlying pathology, indicating a high-value opportunity for a first-in-class candidate.

Comparison to Industry Standards

• NDV-01's 91% overall response rate at any time in NMIBC compares favorably to existing intravesical Gem/Doce treatments, which have shown 2-year Relapse Free Survival (RFS) of ~50% as salvage therapy and 82% as first-line in high-risk BCG naive patients.
• The filing highlights other emerging NMIBC treatments such as ZUSDURI (Mitomycin C by UroGen, FDA approved for low-grade, intermediate risk), TAR-200 (Gemcitabine by Johnson & Johnson, Phase 3), and Nadofaragene Firadenovec (Adstiladrin, approved for BCG-unresponsive NMIBC). NDV-01 differentiates itself by being a sustained-release Gem/Doce combination, ready-for-use, and not requiring device removal, unlike TAR-200.
• The company emphasizes that the Gem/Doce combination is embraced by the urologic oncology community, with a Urology Times survey indicating Gemcitabine plus docetaxel as a commonly preferred treatment for BCG-unresponsive NMIBC, suggesting strong market acceptance for the active agents in NDV-01.
• Sepranolone's mechanism of action (GABAA Modulating Steroid Antagonist) and its potential in Prader-Willi Syndrome (PWS) address an orphan disease with an estimated 20,000 US prevalence, where current treatments are symptomatic, indicating a significant unmet need compared to standard care.

Stakeholder Impact

• Shareholders: Positive clinical data for NDV-01 and sepranolone could increase long-term value, while reduced net loss indicates improved financial efficiency. However, continued cash burn and the need for future funding could dilute existing shares.
• Patients (NMIBC): NDV-01 offers a promising, potentially more convenient and effective treatment option for non-muscle invasive bladder cancer, especially given its high response rate and favorable safety profile.
• Patients (Prader-Willi Syndrome): Sepranolone represents a potential first-in-class therapy addressing the underlying neurobehavioral pathology, offering hope beyond symptomatic treatments.
• Healthcare Providers: NDV-01's sustained-release, in-office administration could simplify treatment protocols for NMIBC, making it easier to manage patients.

Next Steps

• Continue enrollment in the Phase 2 study for NDV-01.
• Provide updates on NDV-01 Phase 2 data at 9 and 12-month follow-up (Q4 2025 / Q1 2026).
• Engage with FDA for NDV-01 and scale up product supply (2H 2025).
• Initiate Phase 3 registration-track study for NDV-01 (1H 2026).
• Engage with FDA for sepranolone and conduct manufacturing activities (2H 2025).
• Launch Phase 2 study for sepranolone in Prader-Willi syndrome (1H 2026).

Key Dates

Recommendation

The strong 6-month clinical data for NDV-01, showing a 91% overall response rate with a favorable safety profile in NMIBC, is a significant de-risking event for a clinical-stage biotechnology company. This positive data, combined with the clear pathway to a Phase 3 trial in 1H 2026, indicates strong progress towards commercialization in a large market with unmet needs. While the company continues to incur losses and burn cash, the substantial reduction in net loss and operating expenses demonstrates improved financial discipline. The pipeline asset sepranolone also shows promise for an orphan disease. For a seasoned investor, the compelling clinical efficacy and clear development path for NDV-01, despite the current cash position, present a strong 'buy' opportunity based on the potential for significant future value creation.

Keywords

Relmada Therapeutics, RLMD, Biotechnology, Clinical-stage, Oncology, Central Nervous System, NDV-01, Non-Muscle Invasive Bladder Cancer, NMIBC, Sepranolone, Prader-Willi Syndrome, PWS, Clinical Trials, Phase 2, Phase 3, Financial Results, Drug Development, Biopharma