MediciNova, Inc. (NASDAQ:MNOV) presented encouraging interim analysis results from its Phase 2b/3 COMBAT-ALS clinical trial of MN-166 (ibudilast) for Amyotrophic Lateral Sclerosis (ALS) at the 35th International Symposium on ALS/MND. The interim data, which assessed the correlation between 6-month and 12-month outcomes, suggests potential benefits of ibudilast in slowing disease progression. The trial, which has enrolled 217 participants, is expected to complete patient assignments by June 2025, with results anticipated in 2026. These findings offer a beacon of hope for individuals affected by this devastating neurodegenerative disease.
Interim Analysis Details
The interim analysis focused on evaluating the correlation between data collected at 6 months and 12 months to assess the trial design's validity. Key findings include:
- CAFS Score: A correlation coefficient of 0.71 was observed.
- Modified CAFS Score: A correlation coefficient of 0.70 was noted.
- ALSFRS-R: A correlation coefficient of 0.69 was found.
- Bulbar Score: Showed a correlation of 0.74.
- Fine Motor Score: Showed a correlation of 0.71.
- Gross Motor Score: Showed a correlation of 0.67.
Notably, no correlation was observed for the Respiratory score. These results were reviewed and validated by an independent Data Safety Monitoring Board (DSMB), which recommended the trial continue without modifications.
MN-166 (Ibudilast) and ALS Treatment Landscape
ALS is a progressive neurodegenerative disease affecting motor neurons, leading to muscle weakness, paralysis, and eventually, respiratory failure. Current treatments offer limited efficacy, underscoring the urgent need for novel therapeutic options. Ibudilast, a small molecule compound, inhibits phosphodiesterase type-4 (PDE4) and inflammatory cytokines, including macrophage migration inhibitory factor (MIF). Its potential to address neuroinflammation makes it a promising candidate for ALS treatment.
Ongoing Trial and Expanded Access Program
As of November 15, 2024, the COMBAT-ALS trial has enrolled 217 participants, with 183 already assigned to either the MN-166 or placebo group. MediciNova anticipates completing patient assignments by June 2025, with trial results expected in 2026. In parallel, the company is supporting continued treatment for eligible patients through the FDA's Expanded Access Program (EAP), ensuring access to MN-166 beyond the clinical trial setting. Furthermore, a large-scale EAP trial funded by the NIH is slated to begin next year, potentially broadening access to ibudilast for more ALS patients.
Expert Commentary
"The interim analysis showed a positive correlation between the 6-month and 12-month data," commented MediciNova CMO and Director Kazuko Matsuda. "We considered a change in the treatment period but decided to continue the trial with the current treatment plan based on the DSMB's recommendation. We believe these results will be valuable in designing studies for rapidly progressing diseases like ALS."
