Positive initial Phase 2 proof-of-concept data for NDV-01 at AUA 2025 showing a 90% overall response rate at any time in non-muscle invasive bladder cancer (US prevalence 600K patients)
The Phase 2 study for NDV-01 continues with updates at 6, 9 and 12 month data follow-up over the course of 2025. Plans to start Phase III registration trial in H1 2026
Expecting to initiate a Phase 2 study for sepranolone in Prader-Willi syndrome in H1 2026 (US prevalence 20,000 patients)
Conference Call and Webcast Today at 4:30 PM ET
CORAL GABLES, Fla., May 12, 2025 (GLOBE NEWSWIRE) -- Relmada Therapeutics, Inc. (Nasdaq: RLMD, “Relmada” or the “Company”), a clinical-stage biotechnology company advancing innovative therapies for central nervous system and oncology indications, today reported financial results for the first quarter ended March 31, 2025, and provided a corporate and pipeline update.
“2025 is off to an outstanding start for Relmada,” said Sergio Traversa, CEO of Relmada. “With the addition of NDV-01 and sepranolone, two differentiated Phase 2 product candidates targeting large, underserved markets, we’ve significantly enhanced our pipeline and long-term value proposition. The recently announced proof-of-concept data for NDV-01 presented at AUA 2025 reinforce its potential to become a best-in-class, bladder-sparing treatment for non-muscle invasive bladder cancer (NMIBC).”
“Our strategic focus remains on developing a diverse pipeline with strong scientific rationale, intellectual property protection, and capital-efficient development paths,” added Mr. Traversa. “Looking ahead, we are prioritizing the advancement of NDV-01 and sepranolone into further clinical development.”
“In the second half of 2025, we plan to complete the ongoing NDV-01 Phase 2 study and prepare for the initiation of a Phase III registration trial in the first half of 2026,” said Maged Shenouda, CFO of Relmada. “We also expect to initiate a new Phase 2 study of sepranolone in Prader-Willi syndrome (PWS) in the first half of 2026.”
Pipeline Highlights
NDV-01 A sustained-release intravesical formulation of gemcitabine and docetaxel (GEM/DOCE)
Indication: High-Grade Non-Muscle Invasive Bladder Cancer (HG-NMIBC)
U.S. Market Opportunity: ~600,000 prevalent cases
Current Status: Phase 2 single-arm study actively enrolling
Recent Data (American Urological Association, AUA 2025):
90% Overall Response Rate (18/20 patients)
89% High-Grade Recurrence-Free Survival in papillary disease (16/18)
100% Complete Response in carcinoma in situ (2/2)
Next Steps:
FDA interactions and product supply scale-up in 2H 2025
Initiation of Phase 3 registration-track study in 1H 2026
Sepranolone A first-in-class GABAA Modulating Steroid Antagonist (GAMSA)
Indications: Prader-Willi Syndrome (PWS), Tourette Syndrome (TS), Essential Tremor, and other compulsivity-related disorders
US Prevalence: Estimated 20,000 for PWS, orphan disease
Current Status: Preparing for Phase 2 initiation in PWS
Supporting Data:
In Tourette syndrome, Phase 2 trial showed a 28% reduction in tic severity (p=0.051) per YGTSS (Yale Global Tic Severity Scale)
Improvements in quality of life and symptom scales
Favorable safety profile with no CNS or systemic side effects
Next Steps:
FDA engagement and manufacturing activities in 2025
Planned Phase 2 study launch in 1H 2026
First Quarter 2025 Financial Results
R&D Expense: $11.9 million (vs. $13.3 million in Q1 2024), reflecting reduced REL-1017 trial costs, partially offset by new asset integration
G&A Expense: $6.3 million (vs. $9.7 million), primarily due to lower stock-based compensation
Net Cash Used in Operations: $18.1 million (vs. $13.0 million)
Net Loss: $17.5 million or $0.58 per share (vs. $21.8 million or $0.72 per share)
Cash, Equivalents & Short-Term Investments: $27.1 million as of March 31, 2025 (vs. $44.9 million at year-end 2024)
Shares Outstanding: 33,191,622 as of May 7, 2025
Conference Call and Webcast Relmada will host a conference call today, May 12, 2025, at 4:30 PM ET to discuss its Q1 results and pipeline progress.
NDV-01 is a sustained-release, intravesical formulation of gemcitabine and docetaxel (GEM/DOCE), in development for the treatment of bladder cancer. It was designed to enable GEM/DOCE bladder retention and gradual drug release over 10 days. The formulation creates a soft matrix that enhances local exposure while minimizing systemic toxicity. NDV-01 is easy to administer in-office, in less than 10 minutes, and does not require anesthesia or specialized equipment. It is protected by patents through 2038.
About NMIBC
NMIBC represents ~75% of all bladder cancer cases and is associated with high recurrence (50–75% over 7 years). With over 600,000 prevalent cases in the U.S. and limited treatment options, the market opportunity is significant. NDV-01 has the potential to serve as a frontline or salvage therapy and could be applicable across multiple NMIBC subtypes.
About Sepranolone and GABA Modulation
Sepranolone, a synthetic isoallopregnanolone, selectively modulates GABAA receptors by antagonizing allopregnanolone (ALLO), without disrupting GABA signaling. It targets disorders linked to excess GABAergic activity such as Prader-Willi syndrome, Tourette syndrome, and Obsessive-Compulsive Disorder (OCD). More than 335 patients have been treated in trials to date, with an excellent safety profile.
About Prader-Willi Syndrome (PWS)
PWS is a rare genetic disorder caused by chromosomal deletions on chromosome 15, leading to neurodevelopmental and behavioral complications. US prevalence is estimated at 20,000 patients. Current treatments address symptoms but do not modify the underlying neurobehavioral pathology.
About Tourette Syndrome (TS)
TS affects more than 350,000 U.S. children and is marked by involuntary tics. Current therapies can have substantial side effects. Sepranolone is an investigational agent that offers a novel mechanism by modulating ALLO, potentially reducing compulsive symptoms more safely than existing dopaminergic therapies.
About Relmada Therapeutics, Inc.
Relmada Therapeutics is a clinical-stage biotechnology company focused on developing transformative therapies for central nervous system and oncology-related conditions. Its lead candidates, NDV-01 and sepranolone, are advancing through mid-stage clinical development with the potential to address significant unmet needs.
Forward-Looking Statements: The Private Securities Litigation Reform Act of 1995 provides a safe harbor for forward-looking statements made by us or on our behalf. This press release contains statements which constitute “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Any statement that is not historical in nature is a forward-looking statement and may be identified by the use of words and phrases such as “if”, “may”, “expects”, “anticipates”, “believes”, “will”, “will likely result”, “will continue”, “plans to”, “potential”, “promising”, and similar expressions. These statements are based on management’s current expectations and beliefs and are subject to a number of risks, uncertainties and assumptions that could cause actual results to differ materially from those described in the forward-looking statements, including potential for Phase 2 NDV-01 data to continue to deliver positive results supporting further development, potential for clinical trials 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, or that future sepranolone, or NDV-01 clinical results will be acceptable to the FDA, failure to secure adequate sepranolone, or NDV-01 drug supply, and the other risk factors described under the heading “Risk Factors” set forth in the Company’s reports filed with the SEC from time to time. No forward-looking statement can be guaranteed, and actual results may differ materially from those projected. Relmada undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events, or otherwise. Readers are cautioned that it is not possible to predict or identify all the risks, uncertainties and other factors that may affect future results and that the risks described herein should not be a complete list.
