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September 29, 2021

Biohaven drug fails in trial for rare brain disease


Biohaven Pharmaceuticals gave an early report this week on its clinical trial of a drug to treat the rare brain disease Muscular System Atrophy, and the news wasn’t good.

The drug, verdiperstat, failed to slow progression of the disease when compared to a placebo, Biohaven said. It also fell short on its secondary goals, which included improvement on measures of quality of life.

The company, however, said it’s still hopeful the drug will be proven successful in treating other devastating neurological diseases, and that more analyses on the Phase 3 trial data are forthcoming.
Biohaven licensed the drug, a myeloperoxidase (MPO) inhibitor that targets brain inflammation,  from AstraZeneca in 2018, paying $3 million upfront and $4 million in shares, according to regulatory filings.

A unique clinical trial testing the drug against amyotrophic lateral sclerosis, or ALS, is ongoing at Massachusetts General Hospital.

Dr. Ifran Qureshi, vice president of neurology at Biohaven, said, “While we are disappointed that verdiperstat did not demonstrate efficacy for the treatment of MSA, Biohaven remains committed to fighting on behalf of people living with neurodegenerative diseases. There are currently no approved disease modifying therapies for MSA and we must continue to advance the science to improve treatment outcomes for patients suffering from this disease.”
Wall Street analysts were unfazed by the trial failure, Reuters reported, saying expectations for the trial were low and the focus remains on the company’s migraine drug Nurtec ODT, which brought in $93 million last quarter.

Nurtec was first approved for acute migraine attacks in February 2020 and won a second FDA approval earlier this year for migraine prevention in adults with episodic migraine.

Contact Natalie Missakian at

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