8-K: Relmada Reports Strong NDV-01 Data, Secures $160M PIPE

Relmada Therapeutics announced positive 12-month Phase 2 data for NDV-01 in bladder cancer, a $160 million PIPE financing, and plans to initiate Phase 3 trials in mid-2026.

Summary

• Positive 12-month Phase 2 data for NDV-01 in non-muscle invasive bladder cancer (NMIBC) demonstrated a 95% complete response (CR) rate at any time and a durable 76% CR rate at 12 months, with a favorable safety profile.
• Completed an oversubscribed $160 million PIPE financing in March 2026, strengthening the balance sheet to support NDV-01 Phase 3 development.
• On track to initiate Phase 3 RESCUE registrational program for NDV-01 in second-line (2L) BCG-unresponsive and adjuvant intermediate-risk NMIBC in mid-2026.
• Cash balance of $93.0 million as of December 31, 2025, combined with the $160 million PIPE proceeds, is expected to fund operations through 2029, including completion of the NDV-01 RESCUE program.
• Net loss for the full year ended December 31, 2025, was $57.4 million, or $1.45 per basic and diluted share, an improvement from $80.0 million, or $2.65 per share, in 2024.
• Research and development (R&D) expense decreased to $26.9 million in 2025 from $46.2 million in 2024.
• General and administrative (G&A) expense decreased to $32.2 million in 2025 from $37.7 million in 2024.

Sentiment

Score: 8

Explanation: StockSavvy.ai views this as a highly positive update, driven by strong clinical data for NDV-01, successful financing that extends the cash runway, and clear regulatory pathways for its lead programs.

Positives

• NDV-01 Phase 2 data showed a durable 76% complete response rate at 12 months and 95% at any time in high-risk NMIBC, with an 80% CR rate at 12 months in BCG-unresponsive patients.
• No patient in the Phase 2 study had progression to muscle-invasive disease or underwent a radical cystectomy.
• NDV-01 demonstrated a favorable tolerability profile with no Grade 3 treatment-related adverse events and no treatment-related discontinuations.
• Successful oversubscribed $160 million PIPE financing completed in March 2026.
• Cash runway extended through 2029, covering the completion of the NDV-01 RESCUE program.
• Received written feedback from the U.S. Food and Drug Administration (FDA) confirming alignment on two registrational development pathways for NDV-01.
• Full-year 2025 net loss decreased to $57.4 million from $80.0 million in 2024.
• Full-year 2025 Research and development (R&D) and General and administrative (G&A) expenses decreased compared to 2024.

Negatives

• Net loss for the three months ended December 31, 2025, increased to $19.9 million from $18.6 million for the same period in 2024.
• Net cash used in operating activities for the three months ended December 31, 2025, increased to $14.6 million from $8.8 million for the same period in 2024.
• General and administrative expense for the three months ended December 31, 2025, increased to $12.3 million from $8.1 million for the same period in 2024.

Risks

• Potential for Relmada's product candidates to not progress as expected.
• Clinical trials may fail to deliver statistically and/or clinically significant evidence of efficacy and/or safety.
• Failure of interim or top-line results to accurately reflect the complete results of the trial.
• Failure of planned or ongoing preclinical and clinical studies to demonstrate expected results.
• Potential failure to continue to secure FDA agreement on the regulatory path for NDV-01 and/or sepranolone, or that future clinical results will be acceptable to the FDA.
• Failure to secure adequate NDV-01 and/or sepranolone drug supply.
• Uncertainties inherent in estimating the Company's cash runway, future expenses, and other financial results, including its ability to fund future operations and clinical trials.

Future Outlook

Relmada expects to initiate the Phase 3 RESCUE registrational program for NDV-01 in mid-2026, targeting two independent pathways for NMIBC. Initial 3-month data from the Phase 3 2L BCG-unresponsive study is anticipated by year-end 2026. The company also plans to initiate a Phase 2 study for Sepranolone in Prader-Willi syndrome in mid-2026. The current cash position is projected to fund operations through 2029, covering the completion of the NDV-01 RESCUE program.

Management Comments

• Sergio Traversa, CEO: "This has truly been a transformational year for Relmada, marked by significant progress with our lead program NDV-01. Our recently reported 12-month data for NDV-01 demonstrated durable complete responses with a favorable safety profile, reinforcing the program's potential to become a best-in-class therapy for patients with non-muscle invasive bladder cancer. With a successful $160M PIPE financing and regulatory alignment with the FDA on two registrational pathways, we believe that we are well positioned to advance NDV-01 into the Phase 3 RESCUE program in mid-2026. Our team is now focused on executing this plan and initiating the RESCUE registrational program as we work to bring NDV-01 to patients as efficiently as possible."
• Raj S. Pruthi, MD, Chief Medical Officer-Oncology: "NDV-01's compelling efficacy, durability, and favorable safety profile, combined with operational ease-of-use are the cornerstone of its differentiated product profile and best-in-class potential. We continue to be encouraged by the high response rates and durable clinical benefit observed through 12 months, including in the BCG-unresponsive population, alongside a favorable safety profile with no Grade 3 treatment-related adverse events and no treatment-related discontinuations. Our clinical program builds on the urologic oncology community's comfort with conventional Gem/Doce's efficacy and safety profile with a sustained release product that could provide physicians and patients with a streamlined, less than 5-minute in-office procedure. These results reinforce our confidence as we advance NDV-01 into the Phase 3 RESCUE registrational program in mid-2026."

Industry Context

StockSavvy.ai notes that Relmada's NDV-01 addresses a significant unmet need in non-muscle invasive bladder cancer (NMIBC), particularly in BCG-unresponsive and intermediate-risk settings where treatment options are limited or non-existent. The sustained-release intravesical formulation of gemcitabine/docetaxel (Gem/Doce) aims to improve upon conventional Gem/Doce, which is already a preferred treatment in academic settings, by offering ease of administration and prolonged tumor exposure. This positions NDV-01 to potentially become a next-generation standard of care, expanding utilization into community urology practices. The development of Sepranolone for Prader-Willi syndrome also targets a rare genetic disorder with high unmet needs, aligning with a broader industry trend of focusing on orphan drug indications.

Comparison to Industry Standards

• NDV-01's 76% complete response rate at 12 months in high-risk NMIBC and 80% in BCG-unresponsive patients compares favorably to existing therapies, where 12-month CR rates for first-line BCG-unresponsive therapies are typically 19%-46%.
• The absence of progression to muscle-invasive disease or radical cystectomy in the Phase 2 study highlights a significant advantage, as radical cystectomy is often the only alternative for BCG-unresponsive NMIBC patients.
• NDV-01's "less than 5-minute in-office procedure" and "ready to use" formulation offer a significant operational advantage over conventional Gem/Doce, which requires specialized pharmacy preparation and 4-hour total procedure times, making it more accessible for community urologists.
• The favorable safety profile with no Grade 3 treatment-related adverse events or discontinuations is a strong indicator compared to other cancer therapies that often have more severe side effects.

Stakeholder Impact

• Shareholders: Positive impact due to strong clinical data, successful financing, extended cash runway, and clear path to Phase 3, potentially increasing shareholder value.
• Patients (NMIBC): Potential for a new, effective, and well-tolerated treatment option, especially for those with BCG-unresponsive disease who have limited alternatives.
• Patients (Prader-Willi Syndrome): Potential for a novel therapy addressing underlying neurobehavioral pathology, offering hope for a high-unmet-need rare disease.
• Healthcare Providers (Urologists): NDV-01's ease of administration (less than 5-minute in-office procedure) could streamline treatment and expand access beyond academic centers.
• Employees: Continued employment and potential growth opportunities as programs advance.

Next Steps

• Present NDV-01 Phase 2 data at upcoming medical meetings.
• Continue enrollment in NDV-01 High-Risk NMIBC (Study TRCG-001).
• Initiate NDV-01 United States IND clearance in mid-2026.
• Initiate NDV-01 Phase 3 RESCUE Program (Cohort 1 for Intermediate-Risk NMIBC and Cohort 2A for 2L BCG-Unresponsive NMIBC) in mid-2026.
• Initiate NDV-01 Phase 2 exploratory Cohort 2B (2L BCG-Unresponsive Ta/T1 disease) in mid-2026.
• Initiate Sepranolone Phase 2b study in Prader-Willi syndrome in mid-2026.
• Identify next indication for Sepranolone in 2026/2027.
• Expect initial 3-month NDV-01 data from Phase 3 2L BCG-unresponsive study by year-end 2026.

Key Dates

Recommendation

The filing presents exceptionally strong 12-month Phase 2 data for NDV-01, demonstrating high and durable complete response rates in a challenging bladder cancer population with a favorable safety profile. This, coupled with successful and oversubscribed financing that extends the cash runway through 2029 and clear FDA-aligned registrational pathways, significantly de-risks the company's lead asset. The planned initiation of Phase 3 trials in mid-2026 and the promising Sepranolone program further enhance the long-term growth potential, making Relmada Therapeutics a compelling investment opportunity.

Keywords

biotechnology, oncology, NMIBC, bladder cancer, NDV-01, clinical trial, Phase 3, PIPE financing, drug development, central nervous system, Prader-Willi syndrome, sepranolone, rare disease, FDA, NASDAQ, RLMD