Expanded access program for ALS drug MN-166 at 50% enrollment
It allows patients to receive experimental therapies outside of clinical trials
- MN-166, an experimental drug, aims to slow ALS progression.
- An expanded access program for MN-166 in ALS has reached 50% enrollment.
- It holds Orphan Drug and Fast Track designations, with clinical trial results expected soon.
Patient enrollment has reached the halfway mark in an ongoing expanded access program (EAP) for MN‑166 (ibudilast), Medicinova’s oral anti-inflammatory drug for the treatment of amyotrophic lateral sclerosis (ALS).
EAPs, also known as compassionate use programs, are designed to help people with serious or life-threatening diseases receive experimental therapies outside of traditional clinical trials when no other options are available. They also allow researchers to collect additional clinical data.
The ongoing program, called SEANOBI-ALS (NCT06743776), is designed to include about 200 ALS patients across the U.S. who are not eligible for the Phase 2b/3 COMBAT-ALS trial (NCT04057898). Thus far, 12 clinical sites have been activated, and 100 participants have been enrolled.
“Achieving 100 enrolled patients … marks substantial progress in the clinical development of MN‑166,” Yuichi Iwaki, MD, PhD, president and CEO of Medicinova, said in a company press release. “We are deeply grateful to the patients and families who chose to participate in SEANOBI, as their commitment makes this important program possible.”
SEANOBI is being funded through a $22 million grant from the National Institute of Neurological Disorders and Stroke (NINDS), part of the National Institutes of Health, under the Accelerating Access to Critical Therapies for ALS Act (ACT for ALS). Signed into law in 2021, the ACT for ALS provides $100 million annually through fiscal 2026 for efforts to improve ALS prevention, diagnosis, and treatment.
“We also sincerely appreciate the continued support from NINDS under the ACT for ALS initiative, which has enabled [expanded] access to MN‑166 while gathering meaningful clinical and biomarker insights,” Iwaki said.
MN-166 aims to reduce activity of immune cells in the brain
MN-166 is an experimental therapy that aims to reduce the activity of immune cells in the brain, potentially helping limit inflammation and nerve cell damage in ALS. This is expected to slow disease progression.
The therapy is currently being investigated in the COMBAT-ALS trial, which completed enrollment last year and is expected to support regulatory applications seeking the approval of MN-166 for ALS.
The trial is testing whether MN-166 can safely slow ALS progression in 234 adults with ALS, ages 18 to 80, who began experiencing ALS symptoms 18 months before entering the study. Participants are receiving either MN-166 or a placebo twice daily for 12 months, after which all are offered the option to enter a six-month open-label extension.
An interim analysis suggests that MN-166 may slow ALS progression, extend survival, and reduce decline in key physical functions, though more data are needed to confirm these effects. Top‑line results are expected by the end of the year, the company stated.
SEANOBI is designed to add real-world evidence to the controlled clinical trial results from COMBAT-ALS by providing ALS patients access to the medication outside of clinical trials. Those who are ineligible for the COMBAT-ALS trial or who have completed COMBAT-ALS and its open-label extension are eligible to join the EAP.
We believe these combined data along with having Orphan Drug Designation from FDA and EMA and Fast Track Designation from FDA, will help us advance MN‑166 one step closer to becoming an approved treatment option for people living with ALS, who urgently need more choices.
The study’s main goal is to understand how MN-166 influences disease progression and neurofilament light chain levels, a biomarker of nerve cell damage, in a real-world setting.
“Together with our COMBAT‑ALS study, SEANOBI brings forward both clinical and real‑world evidence that will support discussions with regulators,” Iwaki added.
MN-166 has received orphan drug designation from both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for potential treatment of ALS. This status is granted to therapies intended to treat rare diseases and provides incentives such as market exclusivity and regulatory support.
The therapy also holds fast track designation from the FDA, a status intended to speed the development and review of medicines for serious conditions with unmet medical needs.
“We believe these combined data, along with having Orphan Drug Designation from FDA and EMA and Fast Track Designation from FDA, will help us advance MN‑166 one step closer to becoming an approved treatment option for people living with ALS, who urgently need more choices,” Iwaki said.
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