A total of 234 participants were randomized to two treatment arms at clinical sites in the United States and Canada. Participants who remain active in the study are continuing treatment with MN-166 in the Open-Label Extension phase. The LPLV for the entire study is expected in March 2027. MediciNova expects to report topline results from the double-blind portion of the study by the end of 2026.

MN-166 (ibudilast) is an orally administered small molecule designed to modulate neuroinflammation and oxidative stress pathways implicated in ALS progression. COMBAT-ALS is a randomized, double-blind, placebo-controlled trial designed to evaluate the efficacy, safety, and tolerability of MN-166 during a 12-month double-blind treatment period, followed by a 6-month open-label extension.

The trial’s primary endpoint is the Combined Assessment of Function and Survival (CAFS). Secondary endpoints include change in the ALS Functional Rating Scale–Revised (ALSFRS-R), muscle strength measured by hand-held dynamometry, and quality-of-life assessments.

MN-166 has demonstrated a favorable safety profile in prior Phase 1/2 and Phase 2 clinical studies, with earlier studies also showing a higher proportion of treatment responders among participants who received MN-166. MN-166 has received Fast Track designation and Orphan Drug designation from the U.S. Food and Drug Administration (FDA), as well as orphan designation from the European Medicines Agency (EMA).

Yuichi Iwaki, M.D., Ph.D., President and Chief Executive Officer of MediciNova, commented: "Completion of the last patient’s final visit in the double-blind portion of COMBAT-ALS marks a significant milestone for our lead clinical program and keeps us on track to report topline results by the end of 2026. With all patient visits now complete in the double-blind portion of the study, our focus is on database lock and rigorous analysis of the study data. We believe COMBAT-ALS has the potential to provide important insight into MN-166 as a treatment option for people living with ALS, a disease with substantial unmet medical need. We are deeply grateful to the participants and their families, caregivers, and care partners, as well as the investigators and clinical site teams whose commitment made this milestone possible."