MediciNova Announces Completion of Patient Enrollment in COMBAT-ALS Phase 2b/3 Clinical Trial of MN-166 (ibudilast)
MediciNova (NASDAQ:MNOV) has completed patient enrollment in its Phase 2b/3 COMBAT-ALS clinical trial evaluating MN-166 (ibudilast) for Amyotrophic Lateral Sclerosis (ALS) treatment. The study has successfully randomized 234 patients across sites in the United States and Canada.
The trial is designed as a randomized, double-blind, placebo-controlled study with a 12-month treatment period followed by a 6-month open-label extension. The primary endpoint focuses on the Combined Assessment of Function and Survival (CAFS), with top-line data expected by end of 2026.
MN-166 has received Orphan Drug and Fast Track Designations from the FDA and Orphan Designation from the EMA, having shown promising results in earlier studies.
MediciNova (NASDAQ:MNOV) ha completato l'arruolamento dei pazienti nello studio di fase 2b/3 COMBAT-ALS che valuta MN-166 (ibudilast) per il trattamento della sclerosi laterale amiotrofica (ALS). Lo studio ha randomizzato con successo 234 pazienti presso siti negli Stati Uniti e in Canada. Il trial è progettato come uno studio randomizzato, in doppio cieco, controllato con placebo, con un periodo di trattamento di 12 mesi seguito da una estensione aperta di 6 mesi. L'obiettivo primario è la valutazione combinata della funzione e sopravvivenza (CAFS), con i dati principali previsti entro la end of 2026.
MN-166 ha ricevuto designazioni Orphan Drug e Fast Track dalla FDA e la designazione Orfana dall'EMA, avendo mostrato risultati incoraggianti in studi precedenti.
MediciNova (NASDAQ:MNOV) ha completato el reclutamiento de pacientes en su ensayo clínico de fase 2b/3 COMBAT-ALS que evalúa MN-166 (ibudilast) para el tratamiento de la esclerosis lateral amiotrófica (ELA). El estudio ha randomizado con éxito 234 pacientes en sitios de Estados Unidos y Canadá.
El ensayo está diseñado como un estudio aleatorizado, doble ciego, controlado con placebo, con un periodo de tratamiento de 12 meses seguido de una extensión abierta de 6 meses. El criterio principal se centra en la Evaluación Combinada de Función y Supervivencia (CAF S), con datos principales esperados para finales de 2026.
MN-166 ha recibido designaciones de Orphan Drug y Fast Track por la FDA y la designación de Orphan por la EMA, tras haber mostrado resultados prometedores en estudios anteriores.
MediciNova (NASDAQ:MNOV)가 MN-166(ibudilast)을 위한 ALS 치료제(COMBAT-ALS) 2b/3상 임상에서 환자 모집을 완료했습니다. 연구는 미국과 캐나다의 센터에서 234명의 환자를 성공적으로 무작위 배정했습니다.
이 시험은 무작위 이중맹검, 위약 대조 연구로 설계되었으며, 12개월의 치료 기간과 이어지는 6개월의 공개 라벨 확장이 있습니다. 주요 평가 지표는 기능 및 생존의 종합 평가(CAFS)에 초점을 두고 있으며, 최종 데이터는 2026년 말까지 나올 예정입니다.
MN-166은 FDA로부터 고아 의약품(Orphan Drug) 및 Fast Track 지정을 받았고, EMA로부터 고아 지정을 받았으며, 이전 연구에서 유망한 결과를 보였습니다.
MediciNova (NASDAQ: MNOV) a terminé l'enrôlement des patients dans son essai clinique de phase 2b/3 COMBAT-ALS évaluant MN-166 (ibudilast) pour le traitement de la sclérose latérale amyotrophique (SLA). L'étude a randomisé avec succès 234 patients dans des sites situés aux États-Unis et au Canada.
L'essai est conçu comme une étude randomisée en double aveugle, contrôlée par placebo, avec une période de traitement de 12 mois suivie d'une extension ouverte de 6 mois. Le critère principal porte sur l'évaluation combinée de la fonction et de la survie (CAF S), avec des données préliminaires attendues d'ici la fin 2026.
MN-166 a reçu des désignations Orphan Drug et Fast Track de la FDA et la désignation d'Orphan de l'EMA, après des résultats prometteurs dans des études antérieures.
MediciNova (NASDAQ:MNOV) hat die Einschreibung von Patienten in seine Phase-2b/3-Studie COMBAT-ALS abgeschlossen, in der MN-166 (Ibudilast) als Behandlung der amyotrophen Lateralsklerose (ALS) evaluiert wird. Die Studie hat erfolgreich 234 Patienten an Standorten in den USA und Kanada randomisiert.
Die Studie ist als randomisierte, doppelblinde, placebokontrollierte Studie konzipiert, mit einer 12-monatigen Behandlungsdauer gefolgt von einer 6-monatigen offenen Erweiterung. Der primäre Endpunkt fokussiert sich auf die kombinierte Funktions- und Überlebensbewertung (CAFS), mit ersten Daten voraussichtlich bis Ende 2026.
MN-166 hat von der FDA die Des- ignationen Orphan Drug und Fast Track erhalten und von der EMA die Orphan Designation, nachdem in früheren Studien vielversprechende Ergebnisse erzielt wurden.
MediciNova (بورصة ناسداك: MNOV) أكملت تسجيل المرضى في تجربة المرحلة 2b/3 COMBAT-ALS التي تقيم MN-166 (ibudilast) لعلاج التصلب الجانبي الضموري (ALS). أكملت الدراسة عشوائية 234 مريضاً عبر مواقع في الولايات المتحدة وكندا.
التجربة مصممة كدراسة عشوائية مزدوجة التعمية، مُراقبة بالدواء الوهمي، مع فترة علاج مدتها 12 شهراً تليها امتداد مفتوح لمدة 6 أشهر. الهدف الأساسي يركّز على التقييم المشترك للوظيفة والبقاء على قيد الحياة (CAFS)، مع توقع البيانات الأولية بحلول نهاية 2026.
قد حازت MN-166 على تخصيص دواء يتيم (Orphan Drug) وت designation Fast Track من FDA، وعلى designation يتيم من EMA، بعد أن أظهرت نتائج واعدة في دراسات سابقة.
MediciNova (NASDAQ:MNOV) 已完成其阶段2b/3 COMBAT-ALS 临床试验中对 MN-166（ibudilast）的患者招募，评估用于 ALS 的治疗。该研究在美国和加拿大的多家中心共随机分配了 234 名患者。
该试验设计为随机、双盲、安慰剂对照研究，治疗期为 12 个月，随后有 6 个月的开放标签扩展。主要终点关注功能与生存的综合评估（CAFS），预计 2026 年底前公布初步数据。MN-166 已获得美国 FDA 的 孤儿药和快速通道（Fast Track） designation，以及 EMA 的 孤儿药资格，此前的研究已显示出有希望的结果。
- Successfully completed enrollment of 234 patients in Phase 2b/3 trial
- MN-166 previously demonstrated promising results in earlier clinical phases
- Drug has secured both FDA Fast Track and Orphan Drug Designations
- Previous studies showed favorable safety profile and higher treatment responder rates
- Top-line data not expected until end of 2026
- Trial still needs to complete 12-month treatment period plus 6-month extension
Insights
MediciNova hits major development milestone with completed enrollment in pivotal ALS trial; potential regulatory submission timeline now clearer.
MediciNova has reached a significant clinical development milestone with the completion of patient enrollment in its Phase 2b/3 COMBAT-ALS trial evaluating MN-166 (ibudilast) for ALS treatment. The company has successfully randomized 234 patients across sites in the US and Canada, officially closing recruitment for this pivotal study.
The trial design features several strengths worth noting. It employs a rigorous randomized, double-blind, placebo-controlled methodology with a 12-month treatment period followed by a 6-month open-label extension. The primary endpoint is the Combined Assessment of Function and Survival (CAFS) – a composite endpoint specifically designed for ALS trials that balances functional decline with survival outcomes. Secondary endpoints comprehensively assess disease progression through ALSFRS-R scores, muscle strength measurements, and quality of life metrics.
MN-166's mechanism targeting neuroinflammation and oxidative stress represents a differentiated approach in the ALS treatment landscape. The drug has already demonstrated promising signals in earlier studies, with the company reporting a favorable safety profile and higher proportion of treatment responders in previous phases. Importantly, MN-166 carries both Orphan Drug and Fast Track designations from the FDA, potentially accelerating its regulatory pathway.
With enrollment now complete, MediciNova has established a clearer timeline for data readout, with top-line results expected by end-2026. This milestone effectively derisks one of the most challenging aspects of clinical development – patient recruitment – particularly noteworthy for a rare disease like ALS where enrollment challenges frequently delay trials. The completion of enrollment suggests the company's trial design and site selection strategy has executed effectively, and now positions MediciNova on a defined path toward potential regulatory submission if the data proves positive.
~Patient Recruitment Closed~
LA JOLLA, Calif., Sept. 22, 2025 (GLOBE NEWSWIRE) -- MediciNova, Inc., a biopharmaceutical company traded on the NASDAQ Global Market (NASDAQ:MNOV) and the Standard Market of the Tokyo Stock Exchange (Code Number: 4875), today announced the successful completion of patient enrollment in its Phase 2/3 clinical trial, COMBAT-ALS, evaluating MN-166 (ibudilast) for the treatment of Amyotrophic Lateral Sclerosis (ALS). A total of 234 patients have been randomized across two treatment arms at multiple clinical sites in the United States and Canada. Patient recruitment is now officially closed.
MN-166 (ibudilast) is an orally administered small molecules designed to modulate neuroinflammation and oxidative stress pathways implicated in ALS progression. The COMBAT-ALS study is a randomized, double-blind, placebo-controlled trial designed to assess the efficacy, safety, and tolerability of MN-166 over a 12-month treatment period followed by 6-month open-label treatment period.
The trial’s primary endpoint is the Combined Assessment of Function and Survival (CAFS). Secondary endpoints include ALSFRS-R score progression, muscle strength measured by hand-held dynamometry, and quality of life assessments. Top-line data are expected by the end of 2026.
MN-166 has previously demonstrated promising results in preclinical models and earlier Phase 1/2 and Phase 2 studies, showing a favorable safety profile and a higher proportion of treatment responders among MN-166-treated patients. The compound has received Orphan Drug Designation and Fast Track Designation from the U.S. Food and Drug Administration (FDA), as well as Orphan Designation from the European Medicines Agency (EMA).
“Completing enrollment in this Phase 2b/3 trial marks a major milestone for our MN-166 program and reflects the dedication of our clinical team and the ALS community,” said Dr. Kazuko Matsuda, Chief Medical Officer of MediciNova. “We are deeply grateful to the patients and families participating in this study and look forward to advancing MN-166 (ibudilast) toward potential regulatory submission.”
“ALS remains a devastating disease with limited treatment options,” said Dr. Yuichi Iwaki, President and Chief Executive Officer of MediciNova. “We are hopeful that MN-166 may offer a meaningful therapeutic advance for patients living with ALS.”
About MN-166 (ibudilast)
MN-166 (ibudilast) is a small molecule compound that inhibits phosphodiesterase type-4 (PDE4) and inflammatory cytokines, including macrophage migration inhibitory factor (MIF). It is in late-stage clinical development for the treatment of neurodegenerative diseases such as ALS (amyotrophic lateral sclerosis), progressive MS (multiple sclerosis), and DCM (degenerative cervical myelopathy); and is also in development for glioblastoma, Long COVID, CIPN (chemotherapy-induced peripheral neuropathy), and substance use disorder. In addition, MN-166 (ibudilast) was evaluated in patients that are at risk for developing acute respiratory distress syndrome (ARDS).
About MediciNova
MediciNova, Inc. is a clinical-stage biopharmaceutical company developing a broad late-stage pipeline of novel small molecule therapies for inflammatory, fibrotic, and neurodegenerative diseases. Based on two compounds, MN-166 (ibudilast) and MN-001 (tipelukast), with multiple mechanisms of action and strong safety profiles, MediciNova has 11 programs in clinical development. MediciNova’s lead asset, MN-166 (ibudilast), is currently in Phase 3 for amyotrophic lateral sclerosis (ALS) and degenerative cervical myelopathy (DCM) and is Phase 3-ready for progressive multiple sclerosis (MS). MN-166 (ibudilast) is also being evaluated in Phase 2 trials in Long COVID and substance dependence. MN-001 (tipelukast) was evaluated in a Phase 2 trial in idiopathic pulmonary fibrosis (IPF) and a second Phase 2 trial in non-alcoholic fatty liver disease (NAFLD) is ongoing. MediciNova has a strong track record of securing investigator-sponsored clinical trials funded through government grants.
Statements in this press release that are not historical in nature constitute forward-looking statements within the meaning of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, without limitation, statements regarding the future development and efficacy of MN-166 and MN-001. These forward-looking statements may be preceded by, followed by, or otherwise include the words "believes," "expects," "anticipates," "intends," "estimates," "projects," "can," "could," "may," "will," "would," “considering,” “planning” or similar expressions. These forward-looking statements involve a number of risks and uncertainties that may cause actual results or events to differ materially from those expressed or implied by such forward-looking statements. Factors that may cause actual results or events to differ materially from those expressed or implied by these forward-looking statements include, but are not limited to, risks of obtaining future partner or grant funding for development of MN-166 and MN-001, and risks of raising sufficient capital when needed to fund MediciNova's operations and contribution to clinical development, risks and uncertainties inherent in clinical trials, including the potential cost, expected timing and risks associated with clinical trials designed to meet FDA guidance and the viability of further development considering these factors, product development and commercialization risks, the uncertainty of whether the results of clinical trials will be predictive of results in later stages of product development, the risk of delays or failure to obtain or maintain regulatory approval, risks associated with the reliance on third parties to sponsor and fund clinical trials, risks regarding intellectual property rights in product candidates and the ability to defend and enforce such intellectual property rights, the risk of failure of the third parties upon whom MediciNova relies to conduct its clinical trials and manufacture its product candidates to perform as expected, the risk of increased cost and delays due to delays in the commencement, enrollment, completion or analysis of clinical trials or significant issues regarding the adequacy of clinical trial designs or the execution of clinical trials, and the timing of expected filings with the regulatory authorities, MediciNova's collaborations with third parties, the availability of funds to complete product development plans and MediciNova's ability to obtain third party funding for programs and raise sufficient capital when needed, and the other risks and uncertainties described in MediciNova's filings with the Securities and Exchange Commission, including its annual report on Form 10-K for the year ended December 31, 2024 and its subsequent periodic reports on Form 10-Q and current reports on Form 8-K. Undue reliance should not be placed on these forward-looking statements, which speak only as of the date hereof. MediciNova disclaims any intent or obligation to revise or update these forward-looking statements.
INVESTOR CONTACT:
David H. Crean, Ph.D.
Chief Business Officer
MediciNova, Inc
info@medicinova.com
FAQ
What is the status of MediciNova's (MNOV) COMBAT-ALS Phase 2b/3 trial?
When will MediciNova (MNOV) release results from the COMBAT-ALS trial?
What regulatory designations has MediciNova's MN-166 received for ALS treatment?
What is the primary endpoint for MediciNova's COMBAT-ALS trial?
How many patients are enrolled in MediciNova's Phase 2b/3 COMBAT-ALS trial?
