Trial testing experimental ALS treatment completes first part
Medicinova expects top-line data for MN-166 by year's end
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- Medicinova completed the last patient visit in the placebo-controlled portion of the COMBAT-ALS clinical trial of MN-166.
- Top-line data are expected by year's end, with final visits in the six-month extension planned for March 2027.
- MN-166 aims to calm brain inflammation and slow ALS progression.
Medicinova has completed the last patient visit in the placebo-controlled portion of the COMBAT-ALS clinical trial, which is evaluating its investigational oral therapy MN-166 (ibudilast) in people with amyotrophic lateral sclerosis (ALS).
The Phase 2b/3 trial (NCT04057898) is testing MN-166’s safety and efficacy at slowing disease progression in 234 adults with ALS. After an initial one-year part in which patients received either MN-166 or a placebo, patients were given the option to continue into a six-month extension.
Top-line data from the initial portion are expected by the end of the year, and the final visits in the extension are planned for March 2027.
“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,” Yuichi Iwaki, MD, PhD, president and CEO of Medicinova, said in a company press release. “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.”
MN-166 aimed at slowing disease progression
Excess inflammation in the brain and spinal cord is thought to contribute to the development and progression of ALS, a disease marked by the progressive loss of motor neurons, the nerve cells that control muscle movement.
The active agent in MN-166, ibudilast, is a small molecule designed to block the activity of proteins that trigger immune responses and inflammation. This is expected to reduce immune cell activity in the brain and boost the survival and growth of nerve cells, potentially slowing disease progression.
The treatment was tested in a previous Phase 2 trial (NCT02238626) as an add-on to riluzole oral tablets (formerly sold as Rilutek, now available as generics only). This study evaluated the feasibility, safety, tolerability, and clinical outcomes associated with MN-166, taken as 30 mg twice daily for six months, versus a placebo.
While no differences were observed in disease progression, muscle strength, or quality of life between patients on MN-166 or a placebo over the initial six months, analyses conducted after the study ended suggested than more patients on MN-166 had stable disease. Longer-term data involving a six-month extension portion also suggested a potential survival benefit from MN-166.
These findings supported the launch of the ongoing COMBAT-ALS study, which is being run at 16 sites in the U.S. and Canada. Participants were allowed to continue receiving their standard ALS medications during the study.
The trial’s main goal is to track disease progression using the ALS Functional Rating Scale Revised (ALSFRS-R) after one year, as well as survival time. Secondary outcomes include changes in muscle strength and quality of life, the proportion of patients whose ALSFRS-R scores were stable or improved, and time to death or permanent ventilatory support.
An interim analysis has suggested that MN-166 may slow disease progression and extend survival, but more data will be needed to know for sure if the therapy can significantly impact those measures.
MN-166 has received fast track and orphan drug designations in the U.S, as well as orphan drug status in Europe, for the treatment of ALS.
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