Patient randomization nearly complete in MN-166 trial for ALS
Study seeks to assess whether therapy can slow disease progression
Full randomization is nearly complete in the Phase 2/3 COMBAT-ALS trial testing MN-166 (ibudilast) for the treatment of amyotrophic lateral sclerosis (ALS), its developer, Medicinova, reported. Fewer than 10 participants remain to be assigned to a treatment group.
The ongoing COMBAT-ALS trial (NCT04057898) aims to evaluate the safety and efficacy of MN-166 in up to 230 adults with ALS, ages 18 to 80. Participants are randomly assigned to receive MN-166 capsules twice daily or a placebo for 12 months. After that, all participants may choose to start or continue treatment with MN-166 during a six-month open-label extension.
The trial’s main goal is to assess whether the therapy can slow disease progression, as measured by changes in the ALS Functional Rating Scale-Revised, a clinical scale used to track disease progression by measuring certain aspects of physical function.
“We are pleased to report continued strong progress in our core COMBAT-ALS program. Following the release of interim results last year, we are now approaching the completion of patient randomization — a key milestone in the study,” Yuichi Iwaki, MD, PhD, president and CEO at Medicinova, said in a company press release. “Our MN-166 ALS program continues to generate significant interest and anticipation within the ALS community.”
Launched in 2019, the trial is being conducted across multiple sites in the U.S. and Canada and is sponsored by Medicinova. Study completion is expected by the end of 2026.
In parallel, the company made MN-166 available through an Expanded Access Program, which allows patients who are ineligible for the clinical trial to access the therapy. The program is funded by a $22 million grant from the National Institutes of Health, and enrollment is ongoing, according to the company.
Interim analysis suggests MN-166 may improve survival
In ALS, motor neurons, the nerve cells that control voluntary movement, become progressively damaged and die. Although the exact causes of ALS remain unclear, excess inflammation in the brain is believed to contribute to disease progression.
MN-166 is an orally administered small molecule designed to reduce inflammation, support the survival of motor neurons, and potentially slow disease progression. It works by inhibiting the activity of multiple pro-inflammatory cytokines, or signaling molecules, potentially upregulating an anti-inflammatory cytokine called IL-10 and blocking the TLR4 protein to help ease inflammation in the brain.
An interim analysis, released last year, of an initial subset of COMBAT-ALS participants with available data suggested MN-166 may help slow disease progression, improve survival, and preserve physical function over time. In particular, results suggested that benefits seen in the first six months of treatment were sustained for up to one year.
MN-166 holds orphan drug designation for ALS in both the U.S. and the European Union, providing potential market exclusivity and regulatory incentives. The therapy has also received fast track designation from the U.S. Food and Drug Administration, which is intended to accelerate the regulatory review process for treatments targeting serious conditions with unmet medical needs.
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