Relmada Therapeutics Presents Positive Initial Phase 2 NDV-01 Data at AUA2025
Relmada Therapeutics announced positive initial Phase 2 data for NDV-01, their treatment for non-muscle invasive bladder cancer (NMIBC), at AUA2025. The study showed remarkable efficacy with 90% of patients achieving high-grade disease-free status.
Key results include:
- 85% overall response rate at 3 months (17/20 patients)
- 83.3% high-grade recurrence-free survival in papillary disease
- 100% complete response in CIS patients
- 100% disease-free status at 6 months for evaluable patients
The treatment showed strong effectiveness across all patient groups, including both BCG-naïve and BCG-unresponsive patients. NDV-01's safety profile was favorable, with only mild, temporary side effects reported. The treatment can be administered in under 10 minutes, potentially offering a significant advancement in outpatient bladder cancer care.
The ongoing study has enrolled 26 patients total, with 20 reaching the 3-month assessment mark. The company aims to position NDV-01 as a leading therapy for NMIBC treatment.
Relmada Therapeutics ha annunciato dati positivi iniziali di Fase 2 per NDV-01, il loro trattamento per il carcinoma della vescica non muscolo-invasivo (NMIBC), presentati all'AUA2025. Lo studio ha mostrato un'efficacia notevole con il 90% dei pazienti che ha raggiunto uno stato libero da malattia ad alto grado.
I risultati chiave includono:
- 85% di tasso di risposta complessiva a 3 mesi (17/20 pazienti)
- 83,3% di sopravvivenza senza recidiva ad alto grado nella malattia papillare
- 100% di risposta completa nei pazienti con CIS
- 100% di stato libero da malattia a 6 mesi per i pazienti valutabili
Il trattamento ha dimostrato una forte efficacia in tutti i gruppi di pazienti, compresi quelli naïve al BCG e quelli non responsivi al BCG. Il profilo di sicurezza di NDV-01 è risultato favorevole, con effetti collaterali lievi e temporanei. La somministrazione del trattamento richiede meno di 10 minuti, offrendo potenzialmente un significativo progresso nella cura ambulatoriale del cancro alla vescica.
Lo studio in corso ha arruolato in totale 26 pazienti, di cui 20 hanno raggiunto la valutazione a 3 mesi. L'azienda punta a posizionare NDV-01 come terapia di riferimento per il trattamento del NMIBC.
Relmada Therapeutics anunció datos iniciales positivos de la Fase 2 para NDV-01, su tratamiento para el cáncer de vejiga no músculo invasivo (NMIBC), en AUA2025. El estudio mostró una eficacia notable con el 90% de los pacientes alcanzando un estado libre de enfermedad de alto grado.
Los resultados clave incluyen:
- 85% de tasa de respuesta global a los 3 meses (17/20 pacientes)
- 83.3% de supervivencia libre de recurrencia de alto grado en enfermedad papilar
- 100% de respuesta completa en pacientes con CIS
- 100% de estado libre de enfermedad a los 6 meses para pacientes evaluables
El tratamiento mostró una fuerte efectividad en todos los grupos de pacientes, incluyendo tanto pacientes naïve a BCG como no respondedores a BCG. El perfil de seguridad de NDV-01 fue favorable, con solo efectos secundarios leves y temporales reportados. El tratamiento puede administrarse en menos de 10 minutos, lo que podría representar un avance significativo en la atención ambulatoria del cáncer de vejiga.
El estudio en curso ha reclutado un total de 26 pacientes, con 20 alcanzando la evaluación de los 3 meses. La compañía busca posicionar a NDV-01 como una terapia líder para el tratamiento del NMIBC.
Relmada Therapeutics는 AUA2025에서 비근육침윤성 방광암(NMIBC) 치료제인 NDV-01의 초기 2상 긍정적 데이터를 발표했습니다. 연구 결과, 90%의 환자가 고등급 무병 상태를 달성하는 뛰어난 효능을 보였습니다.
주요 결과는 다음과 같습니다:
- 3개월 시점 전체 반응률 85% (20명 중 17명)
- 유두암 환자에서 고등급 재발 없는 생존율 83.3%
- CIS 환자 100% 완전 반응
- 평가 가능한 환자 대상 6개월 무병 상태 100%
이 치료법은 BCG 치료 경험이 없는 환자와 BCG에 반응하지 않는 환자 모두에서 강력한 효과를 보였습니다. NDV-01의 안전성 프로필은 경미하고 일시적인 부작용만 보고되어 우수했습니다. 치료는 10분 이내에 투여 가능해 외래 방광암 치료에 큰 진전을 가져올 수 있습니다.
현재 진행 중인 연구에는 총 26명의 환자가 등록되었으며, 이 중 20명이 3개월 평가 시점에 도달했습니다. 회사는 NDV-01을 NMIBC 치료의 선도적 치료제로 자리매김하는 것을 목표로 하고 있습니다.
Relmada Therapeutics a annoncé des données initiales positives de phase 2 pour NDV-01, leur traitement contre le cancer de la vessie non invasif à muscle (NMIBC), lors de l'AUA2025. L'étude a montré une efficacité remarquable avec 90 % des patients atteignant un statut sans maladie de haut grade.
Les résultats clés incluent :
- Taux de réponse global de 85 % à 3 mois (17/20 patients)
- Survie sans récidive de haut grade de 83,3 % dans la maladie papillomateuse
- Réponse complète à 100 % chez les patients atteints de CIS
- Statut sans maladie à 6 mois à 100 % pour les patients évaluables
Le traitement a montré une forte efficacité dans tous les groupes de patients, y compris chez les patients naïfs au BCG et ceux non réactifs au BCG. Le profil de sécurité de NDV-01 était favorable, avec uniquement des effets secondaires légers et temporaires rapportés. Le traitement peut être administré en moins de 10 minutes, offrant potentiellement une avancée significative dans les soins ambulatoires du cancer de la vessie.
L'étude en cours a recruté au total 26 patients, dont 20 ont atteint le point d'évaluation à 3 mois. L'entreprise vise à positionner NDV-01 comme une thérapie de premier plan pour le traitement du NMIBC.
Relmada Therapeutics gab positive erste Phase-2-Daten für NDV-01 bekannt, ihre Behandlung für nicht muskelinvasiven Blasenkrebs (NMIBC), auf der AUA2025. Die Studie zeigte bemerkenswerte Wirksamkeit mit 90 % der Patienten, die einen hochgradig tumorfrei Status erreichten.
Wichtige Ergebnisse umfassen:
- 85 % Gesamtansprechrate nach 3 Monaten (17 von 20 Patienten)
- 83,3 % hochgradiges rezidivfreies Überleben bei papillärer Erkrankung
- 100 % komplette Remission bei CIS-Patienten
- 100 % tumorfrei nach 6 Monaten bei auswertbaren Patienten
Die Behandlung zeigte starke Wirksamkeit in allen Patientengruppen, einschließlich BCG-naiver und BCG-resistenter Patienten. Das Sicherheitsprofil von NDV-01 war günstig, mit nur milden, vorübergehenden Nebenwirkungen. Die Behandlung kann in unter 10 Minuten verabreicht werden, was einen bedeutenden Fortschritt in der ambulanten Blasenkrebsbehandlung darstellen könnte.
Die laufende Studie hat insgesamt 26 Patienten eingeschlossen, von denen 20 die 3-Monats-Bewertung erreicht haben. Das Unternehmen strebt an, NDV-01 als führende Therapie für die Behandlung von NMIBC zu positionieren.
- 90% of patients achieved high-grade disease-free status with NDV-01 in Phase 2 trial
- 85% Overall Response Rate at 3-month assessment (17/20 patients)
- 100% Complete Response in CIS patients (2/2 patients)
- 100% disease-free status achieved in evaluable patients at 6 months (7/7 patients)
- Strong safety profile with no treatment-related adverse events above Grade 1
- Treatment administration time under 10 minutes, indicating operational efficiency
- Consistent treatment effect across all patient groups, including BCG-naïve and BCG-unresponsive patients
- Small patient sample size (only 20 patients in total)
- Limited long-term data - only 7 patients reached 6-month assessment
- Very small CIS patient subset (only 2 patients)
- Study is still ongoing and primary endpoint (12-month complete response rate) data not yet available
Insights
Relmada's NDV-01 shows impressive 90% disease-free rates in bladder cancer with excellent safety profile, demonstrating strong therapeutic potential.
The Phase 2 results for NDV-01 in non-muscle invasive bladder cancer (NMIBC) demonstrate remarkably strong clinical efficacy with
Looking at the granular data, the 3-month assessment showed an Overall Response Rate of
The safety profile appears exceptionally favorable with only Grade 1 adverse events reported - mainly urinary urgency, flank pain, and dysuria - all resolving within 24-28 hours. No treatment discontinuations due to adverse events were reported, which is critical for therapy adherence in this disease setting.
What's particularly significant is NDV-01's demonstrated efficacy in both BCG-naïve and BCG-unresponsive patients. BCG-unresponsive disease represents a substantial clinical challenge with limited treatment options, so new effective therapies are urgently needed. The administration time of under 10 minutes positions this as a potentially practice-changing outpatient treatment option.
While these results are undoubtedly encouraging, it's important to recognize this is a single-center study with a defined sample size (20 patients with 3-month data). The primary endpoint includes 12-month assessment, making these interim results that require longer follow-up to confirm durability of response.
Strong Phase 2 results provide clear proof-of-concept for Relmada's bladder cancer treatment, potentially addressing significant unmet needs.
Relmada's Phase 2 data for NDV-01 represents a significant positive development for their oncology pipeline. The
The treatment's demonstrated efficacy in BCG-unresponsive patients is particularly noteworthy as this represents a substantial area of unmet medical need with limited therapeutic alternatives. Many NMIBC patients who fail BCG therapy currently face radical cystectomy (surgical bladder removal) as their next treatment option, highlighting the value of effective bladder-sparing alternatives.
From a clinical utility perspective, NDV-01's simplified administration protocol (under 10 minutes) offers considerable advantages over more complex instillation procedures, potentially enhancing patient compliance and clinical workflow efficiency. This practical advantage, combined with the strong efficacy and favorable safety profile, creates a compelling value proposition.
The consistent treatment effect observed across different patient subgroups (regardless of prior BCG exposure or disease pathology) suggests broad applicability. This could position NDV-01 as a versatile option across the NMIBC treatment spectrum rather than being limited to a narrow patient segment.
While these interim results from a single-center study require further validation in larger trials, they provide a solid foundation for subsequent development stages. The ongoing enrollment (26 patients total with 20 reaching 3-month assessment) demonstrates continued momentum in building a more robust dataset. The upcoming Investor Event may provide additional insights into Relmada's development and commercialization strategy for this promising asset.
NDV-01 showed promising clinical activity in BCG-naïve and BCG-unresponsive patients, with favorable overall tolerability
Data to be reviewed at Investor Event on April 28, 2025 at 4:30 PM ET
CORAL GABLES, Fla., April 28, 2025 (GLOBE NEWSWIRE) -- Relmada Therapeutics, Inc. (Nasdaq: RLMD, “Relmada”, “the Company”), a clinical-stage biotechnology company, today announced that positive initial data from the Phase 2 study of NDV-01 showed that
Highlights of Phase 2 data presented at AUA2025:
Efficacy Data:
3-month Assessment:
- Overall Response Rate (ORR):
85% (17/20 patients at 3 months) - High-Grade Recurrence-Free Survival (HGRFS) in papillary disease:
83.3% (15/18 patients) - Complete Response (CR) in CIS patients:
100% (2/2 patients)
At any time point:
- ORR:
90% (18/20 patients) - HGRFS in papillary disease:
88.8% (16/18 patients) - CR in CIS patients:
100% (2/2 patients)
Disease status at 6 months:
100% (n=7) of evaluable patients (1 CIS, 6 Ta/T1) achieved disease free status. One of these patients was re-treated at 3 months.
“I am impressed by these promising NDV-01 data reported at AUA2025. Gemcitabine and docetaxel (GEM/DOCE) have long shown clinical utility in NMIBC, but the complexity of administration has limited their broader use,” said Yair Lotan, MD, Professor of Urology and Chief of Urologic Oncology at UT Southwestern Medical Center. “A formulation like 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.”
“We believe NDV-01 has the potential to become the class-leading therapy for NMIBC across a wide spectrum of patients,” said Sergio Traversa, CEO of Relmada. “The data presented at AUA2025 provide compelling proof-of-concept for NDV-01 as a bladder-sparing therapy. With its strong responses and tolerability combined with an administration time of less than 10 minutes, NDV-01 represents a potentially significant advance in outpatient bladder cancer treatment. We are enthusiastic about advancing this differentiated, ready-to-use GEM/DOCE formulation to improve patient outcomes and expand treatment options in NMIBC.”
Overall:
The treatment effect of NDV-01 was consistent across all patient groups, demonstrating robust activity, regardless of prior BCG exposure (BCG-naïve and BCG-unresponsive) or disease pathology (including CIS or Ta/T1 papillary).
Safety:
NDV-01 was well tolerated, with no treatment related adverse events greater than Grade 1. The most common treatment emergent adverse events (TEAEs) were urinary urgency, flank pain, and dysuria, all of which were mild and transient, resolving in 24-28 hours. No patients discontinued treatment due to adverse events.
Study Overview:
- Open-label, single-arm, single-center Phase 2 study evaluating NDV-01 in patients with HG-NMIBC (including BCG-naïve (n=8) and BCG-unresponsive (n=12))
- AUA 2025 data based on 20 enrolled patients with HG-NMIBC, including 2 patients with carcinoma in situ (CIS) and 18 patients with papillary disease (Ta/T1)
- The study continues to actively enroll patients. As of the latest cut-off, a total of 26 patients have been enrolled: 20 patients have reached the 3-month assessment. Seven patients have reached 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).
About NDV-01
NDV-01 is an innovative, investigational sustained-release formulation of two complementary chemotherapy agents, gemcitabine and docetaxel (GEM/DOCE). Designed for intravesical administration, NDV-01 forms a soft matrix within the bladder, gradually releasing drug over 10 days without requiring anesthesia or special equipment. NDV-01 is intended to simplify outpatient NMIBC treatment while maximizing local exposure, minimizing systemic toxicity, and improving patient convenience.
NDV-01 is protected by patents that extend to 2038.
About the Phase 2 Study
The Phase 2 study (NCT06663137) is an open-label, single-arm, single-center study evaluating the safety and efficacy of NDV-01 in patients with HG-NMIBC. Patients are treated with NDV-01 in a biweekly induction phase, follow by monthly maintenance for up to one year, with regular assessments via cystoscopy, cytology, and biopsy, as indicated. 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).
About NMIBC
More than
The U.S. NMIBC market is estimated to be a multi-billion-dollar opportunity. Global numbers are higher, in line with projections for significant growth due to the increasing incidence of bladder cancer and the demand for effective, minimally invasive potential therapies like NDV-01. Approved treatment options remain limited (mainly the immunotherapy, BCG, which has been supply constrained for some time), with high recurrence rates leading to frequent re-treatment and progression. Other emerging programs include immunotherapy combinations, single agent chemotherapy formulations and targeted therapies. NDV-01 stands out based on the large body of published data that support the efficacy of treatment with gemcitabine and docetaxel, its ease of administration and potential for durability of action. Expansion beyond first-line treatment into use as a salvage treatment or in other subgroups of NMIBC, including naïve patients, could further increase the opportunity for NDV-01.
About Relmada Therapeutics, Inc.
Relmada Therapeutics is a clinical-stage biotechnology company committed to advancing innovative breakthrough therapies that have the potential to bring meaningful clinical benefits to targeted patient populations.
Lead investigational program, NDV-01, for High-Grade Non-Muscle Invasive Bladder Cancer, is being evaluated in a Phase 2 study. In addition, preparations are underway to advance sepranolone, a Phase 2b-ready investigational program for compulsion-related disorders including Tourette’s Syndrome and Prader-Willi Syndrome, into further studies.
For more information, visit www.relmada.com.
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 a 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.
Investor Contact:
Brian Ritchie
LifeSci Advisors
britchie@lifesciadvisors.com
Media Inquiries:
Corporate Communications
media@relmada.com
FAQ
What are the key results of Relmada's (RLMD) Phase 2 NDV-01 trial presented at AUA2025?
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What are the safety results for Relmada's NDV-01 bladder cancer treatment?
How many patients were included in RLMD's Phase 2 NDV-01 trial as of April 2025?
What makes Relmada's NDV-01 treatment different from current bladder cancer therapies?
