8-K: Relmada Therapeutics Announces Positive Phase 2 NDV-01 Data for Bladder Cancer Treatment at AUA2025

Relmada Therapeutics reports promising initial Phase 2 data for NDV-01, showing a 90% high-grade disease-free status in patients with non-muscle invasive bladder cancer (NMIBC).

Summary

• Relmada Therapeutics announced positive initial data from its Phase 2 study of NDV-01 for the treatment of non-muscle invasive bladder cancer (NMIBC).
• The data, presented at the American Urology Association 2025 (AUA2025) Annual Meeting, showed that 90% of patients achieved high-grade disease-free status at any time point following treatment.
• At the 3-month assessment, the Overall Response Rate (ORR) was 85% (17/20 patients).
• High-Grade Recurrence-Free Survival (HGRFS) in papillary disease was 83.3% (15/18 patients) at 3 months.
• Complete Response (CR) in CIS patients was 100% (2/2 patients) at both 3-month and any time point assessments.
• At any time point, the ORR was 90% (18/20 patients) and HGRFS in papillary disease was 88.8% (16/18 patients).
• 100% (n=7) of evaluable patients achieved disease-free status at 6 months.
• NDV-01 was well tolerated, with no treatment-related adverse events greater than Grade 1.
• The study is an open-label, single-arm, single-center Phase 2 study evaluating NDV-01 in patients with NMIBC.
• As of the latest cut-off, a total of 26 patients have been enrolled, with 20 patients reaching the 3-month assessment and seven patients reaching the 6-month assessment.
• The primary efficacy endpoints are safety and complete response rate (CRR at 12 months), and secondary efficacy endpoints are duration of response (DOR) and event free survival (EFS).

Sentiment

Score: 8

Explanation: The document presents highly positive initial results from a Phase 2 trial, indicating strong potential for NDV-01 in treating NMIBC. The high response rates and good tolerability suggest a promising future for the drug.

Positives

• NDV-01 demonstrates strong clinical activity in treating NMIBC.
• The treatment effect of NDV-01 was consistent across all patient groups, regardless of prior BCG exposure or disease pathology.
• NDV-01 is well tolerated, with only mild and transient adverse events.
• The study showed promising clinical activity in both BCG-naive and BCG-unresponsive patients.
• NDV-01 has the potential to simplify outpatient NMIBC treatment while maximizing local exposure and minimizing systemic toxicity.

Risks

• The forward-looking statements are subject to risks, uncertainties, and assumptions that could cause actual results to differ materially.
• Potential failure to secure FDA agreement on the regulatory path for NDV-01 is a risk.
• Failure to secure adequate NDV-01 drug supply is a potential risk.

Future Outlook

Relmada believes NDV-01 has the potential to become the class-leading therapy for NMIBC and is enthusiastic about advancing this differentiated formulation to improve patient outcomes and expand treatment options.

Management Comments

• Yair Lotan, MD, Professor of Urology and Chief of Urologic Oncology at UT Southwestern Medical Center, stated that NDV-01 has the potential to significantly change how we manage patients with NMIBC in routine practice.
• Sergio Traversa, CEO of Relmada, stated that the data presented at AUA2025 provide compelling proof-of-concept for NDV-01 as a bladder-sparing therapy.

Industry Context

The NMIBC market is estimated to be a multi-billion-dollar opportunity, with limited approved treatment options and high recurrence rates. NDV-01 stands out due to the efficacy of gemcitabine and docetaxel, its ease of administration, and potential for durability of action.

Comparison to Industry Standards

• Current treatments for NMIBC, like BCG, have supply constraints and high recurrence rates, leading to frequent re-treatment.
• Emerging programs include immunotherapy combinations, single-agent chemotherapy formulations, and targeted therapies.
• NDV-01's combination of gemcitabine and docetaxel, delivered in a sustained-release formulation, aims to improve upon existing treatments by simplifying administration and maximizing local drug exposure.

Stakeholder Impact

• Shareholders may react positively to the promising clinical trial results.
• Patients with NMIBC may benefit from a new, potentially more effective and tolerable treatment option.
• The company's employees may be motivated by the positive progress of NDV-01.

Next Steps

• The study continues to actively enroll patients.
• Preparations are underway to advance sepranolone into further studies.
• Advancing NDV-01 to improve patient outcomes and expand treatment options in NMIBC.

Key Dates

Keywords

NDV-01, NMIBC, bladder cancer, Relmada Therapeutics, Phase 2 study, AUA2025, gemcitabine, docetaxel, oncology