ALS Drug Trial Fully Enrolled for 250 Participants
Researchers have reached full enrollment for a trial investigating a potential new treatment for ALS.
Updated on Oct. 13, 2026 in Alzheimer’s
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Neurizon Therapeutics has completed enrollment of 250 patients for a combined phase II/III trial of its ALS drug candidate, monepantel. The study is being conducted at 78 sites across the United States to evaluate the drug's potential for regulatory approval.
Why it matters
This milestone advances the testing of monepantel, a drug candidate that the company hopes will eventually provide a new treatment option for those living with ALS. Patients who do not qualify for the study may soon have access to the drug through an upcoming expanded access program.
This phase II/III trial includes 250 patients across 78 sites in the United States, building on a small proof-of-concept study of 12 individuals. The FDA has agreed to accept plasma neurofilament light chain measurements as supporting evidence alongside the trial data.
The players
Neurizon Therapeutics
A developer focused on therapeutics for neurodegenerative conditions including ALS.
Sean M. Healey & AMG Center for ALS
An academic medical research center that conducts clinical trials to advance ALS care.
The details
The drug candidate NUZ-001, also known as monepantel, is being tested within the Healey ALS platform trial. This study evaluates the drug's impact by measuring the decline rate of the ALSFRS-R score, which tracks 12 physical functions. The research team is also initiating an expanded access protocol for 200 additional patients, which will provide 96 weeks of treatment for those who do not meet the criteria for the primary trial.
Timeline
October 2024: Pharmaust Ltd. changed its name to Neurizon Therapeutics.
January 2026: The FDA declined a fast track designation request for the drug.
Q1 2027: An expanded access program is expected to begin.
Q2 2027: Top-line results from the trial are expected.
Health Landscape
This study is being conducted as part of the Healey ALS platform trial, which uses a unified protocol to efficiently test multiple experimental therapies. The research marks a shift toward utilizing specific biomarkers like plasma neurofilament light chain to support potential regulatory filings.
Those interested in how these clinical developments might affect future treatment options should discuss current research pipelines with their neurologist. Patients and caregivers can monitor future updates on the trial results to understand if this therapy becomes a viable candidate for standard care.
The takeaway
Clinical trials are the primary mechanism for determining the safety and efficacy of new drug candidates. Patients interested in experimental treatments can talk with their healthcare provider about whether an expanded access program or clinical trial registration is appropriate for their needs.
What happens next
The company expects to begin an expanded access program in the first quarter of 2027 and reach top-line trial results in the second quarter of 2027.
Further reading
For more on the current state of neurodegenerative disease research, visit the Alzheimer’s section.
Source note: This article includes information reported by Bio World.
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