8-K: Relmada Highlights Strong NDV-01 Data, Outlines 2026 Milestones

Relmada Therapeutics, Inc. updated its corporate presentation, showcasing positive Phase 2 data for NDV-01 in NMIBC and outlining key development milestones for both NDV-01 and sepranolone through H1 2026.

Summary

• Relmada Therapeutics, Inc. furnished an updated corporate presentation dated September 17, 2025, via a Form 8-K filing.
• The presentation highlights two key pipeline assets: NDV-01 for Non-Muscle Invasive Bladder Cancer (NMIBC) and sepranolone for Prader-Willi Syndrome (PWS) and other compulsivity disorders.
• NDV-01, a sustained-release intravesical formulation of gemcitabine/docetaxel (Gem/Doce), demonstrated a 91% anytime Complete Response Rate (CRR) and a 90% 6-month CRR in high-risk NMIBC patients in the TRCG-011 Phase 2 study.
• No patients in the NDV-01 study progressed to muscle-invasive disease or underwent radical cystectomy, and no Grade 3 or higher treatment-related adverse events were reported.
• Sepranolone, a novel candidate, has shown positive Phase 2 data in Prader-Willi Syndrome and is being explored for Tourette Syndrome, Essential Tremor, and OCD.
• The company reported cash, cash equivalents, and short-term investments of $20.6 million as of June 30, 2025, with no outstanding debt.
• Common shares outstanding are approximately 33.2 million (45.1 million as converted).

Sentiment

Score: 8

Explanation: The filing presents strong positive clinical data for NDV-01, outlines clear and actionable development pathways for both lead candidates, and highlights a healthy balance sheet with no debt. This indicates solid operational and financial standing, with significant progress in its pipeline.

Positives

• NDV-01 achieved a high anytime Complete Response Rate of 91% in high-risk NMIBC patients, indicating strong efficacy.
• The 6-month Complete Response Rate for NDV-01 was 90%, demonstrating durable response over time.
• No patients treated with NDV-01 experienced progression to muscle-invasive disease or required radical cystectomy, suggesting a favorable safety and efficacy profile.
• NDV-01 treatment was well-tolerated, with no Grade 3 or higher treatment-related adverse events and no patient discontinuations due to adverse events.
• NDV-01 offers competitive advantages including sustained drug release for 10 days, a ready-for-use pre-filled syringe format, and convenient in-office administration, significantly reducing procedure time compared to conventional therapies.
• Sepranolone has positive Phase 2 data in Prader-Willi Syndrome and a unique mechanism of action that could address compulsivity disorders.
• The company maintains an unlevered balance sheet with $20.6 million in cash, cash equivalents, and short-term investments as of June 30, 2025, and no outstanding debt.

Risks

• Potential for Phase 2 NDV-01 data to not continue to deliver positive results supporting further development.
• Potential for clinical trials to fail to deliver statistically and/or clinically significant evidence of efficacy and/or safety.
• Failure of 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 secure FDA agreement on the regulatory path for sepranolone and NDV-01.
• Future sepranolone or NDV-01 clinical results may not be acceptable to the FDA.
• Failure to secure adequate sepranolone or NDV-01 drug supply.
• General risks described under the heading 'Risk Factors' in the company's reports filed with the SEC from time to time.

Future Outlook

Relmada expects to advance NDV-01 towards registration-track studies in H1 2026, following planned FDA interactions and manufacturing scale-up. For sepranolone, the company plans to initiate a pilot Phase 2 study in Prader-Willi Syndrome in H1 2026, focusing on evaluating early proof-of-concept, after planned FDA interactions and further development of product supply. The company also aims to identify next opportunities for sepranolone in other compulsivity-related indications by year-end 2025.

Management Comments

• The company is focused on 'Unlocking Life Changing Therapies' through its innovative pipeline.
• Sergio Traversa, Chief Executive Officer, signed the Form 8-K.

Industry Context

The NMIBC market is experiencing innovation due to supply issues with prior BCG-standard treatments and existing gaps in care, positioning NDV-01 to address these unmet needs with a sustained-release Gem/Doce formulation, a combination already recognized by the urologic oncology community. Sepranolone's novel mechanism of action for compulsivity disorders, including Prader-Willi Syndrome, targets areas with significant unmet medical needs, offering a potential new therapeutic approach in neuropsychiatry.

Comparison to Industry Standards

• NDV-01 (Relmada) is positioned against FDA-approved NMIBC treatments like ZUSDURI (UroGen) and INLEXZO (Johnson & Johnson).
• Unlike ZUSDURI (Mitomycin C) and INLEXZO (Gemcitabine), NDV-01 utilizes a Gemcitabine/docetaxel (Gem/Doce) combination, which is an existing and effective treatment frequently used by urologists.
• NDV-01's sustained-release hydrogel delivery system aims to provide continuous drug exposure for 10 days, potentially enhancing anti-tumor activity and reducing administration frequency compared to conventional intravesical therapies.
• NDV-01 is supplied as a pre-filled syringe, ready for use and easily instilled manually in less than 5 minutes, contrasting with ZUSDURI's requirement for in-office reconstitution under chilled conditions and INLEXZO's catheter-based insertion and cystoscopic removal.
• NDV-01 does not require device removal, a feature that differentiates it from INLEXZO.
• Conventional Gem/Doce intravesical therapy typically involves a 4-hour total procedure time and specialized pharmacy preparation, whereas NDV-01 significantly streamlines this process to approximately 5 minutes of administration time.

Stakeholder Impact

• Shareholders: Positive clinical data and clear development timelines could increase investor confidence and potential future valuation.
• Patients (NMIBC): NDV-01 offers a potentially more convenient and effective treatment option with sustained drug release and reduced procedure time compared to conventional therapies.
• Patients (PWS/Compulsivity Disorders): Sepranolone offers a novel therapeutic approach for conditions with significant unmet needs.
• Healthcare Providers: NDV-01's ready-for-use format and in-office administration could simplify treatment protocols and reduce burden.

Next Steps

• Q4 2025: NDV-01 9-month data update.
• Q4 2025: Sepranolone Phase 2 Prader-Willi Syndrome (PWS) preparations, including planned FDA interactions and further product supply development.
• Q4 2025: Identify next opportunity for Sepranolone, including Tourette Syndrome (TS), Essential Tremor, Obsessive-Compulsive Disorder (OCD), and other compulsivity-related indications.
• Q1 2026: NDV-01 12-month data update.
• H1 2026: Initiate NDV-01 registration-track Phase 3 study (target population to be confirmed through FDA discussions).
• H1 2026: Initiate pilot Phase 2 study for Sepranolone in Prader-Willi Syndrome, focusing on evaluating early proof-of-concept.

Key Dates

Recommendation

While the clinical data for NDV-01 is promising and the company has a clear development roadmap, both programs are still in clinical stages (Phase 2/Phase 3 initiation). The company has a solid cash position and no debt, which is positive. However, the inherent risks of drug development, including potential FDA hurdles and trial outcomes, warrant a cautious 'hold' recommendation for a seasoned investor, awaiting further de-risking through advanced clinical trial results and regulatory progress.

Keywords

Relmada Therapeutics, NDV-01, Sepranolone, NMIBC, Non-Muscle Invasive Bladder Cancer, Prader-Willi Syndrome, PWS, Oncology, Urology, Compulsivity Disorders, Clinical Trials, Phase 2, Phase 3, FDA, Biotechnology, Pharmaceuticals, Drug Development