LA JOLLA, Calif. — 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.
Investor Contact
David H. Crean, Ph.D.
Chief Business Officer
MediciNova, Inc
[email protected]