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August 5, 2020 Bioscience

Biohaven enrolls first patients in unique ALS trial led by Mass General

Calaneet Balas, president and CEO of The ALS Association.

New Haven’s Biohaven Pharmaceuticals said Monday it has enrolled the first patients in a first-of-its kind clinical trial led by Massachusetts General Hospital for patients with amyotrophic lateral sclerosis (ALS). 

Biohaven’s experimental drug, verdiperstat, was selected last September as one of the first three candidates to be tested in the so-called platform trial.

Officials at Mass General’s Sean M. Healy & AMG Center for ALS have said the trial would be the first to test multiple ALS therapies at once. 

It will use specialized statistical tools to evaluate the drugs simultaneously. Typically, clinical trials only evaluate one drug at a time. 

ALS is a progressive and rare neuromuscular disease that afflicts roughly 30,000 people in the U.S. It leads to progressive muscle weakness and death, usually within three years of diagnosis.

Biohaven’s drug, known as a myeloperoxidase (MPO) inhibitor, targets brain inflammation, which plays a role in ALS. It is a tablet that is taken orally. The company licensed the drug from AstraZeneca in 2018 and it has already been tested for safety in 250 healthy volunteers. 

ALS experts from Mass General’s Healy Center will conduct the trial at more than 50 sites in collaboration with the Northeast ALS Consortium clinical trial network.

Approximately 160 adults with ALS will be given 600 mgs. of the drug or a placebo for 24 weeks. The study will measure the change in the severity of the disease after 24 weeks, based on a commonly used scale of ALS functioning. 

More information about the trial can be found at

The platform model, which has been used successfully for cancer drug trials,  is being advanced by the U.S. Food & Drug Administration as a speedier, less expensive way to bring life-changing drugs to patients with serious diseases. 

"By testing multiple drugs at the same time, this platform trial will dramatically speed our search for effective ALS treatments for everyone living with ALS," Calaneet Balas, president and CEO of the ALS Association, said in a statement.

Biohaven also launched a new migraine drug, Nurtec ODT, this spring after receiving FDA approval in February. 

Contact Natalie Missakian at

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