Biohaven completes enrollment in drug trial for rare brain disorder

Biohaven Pharmaceuticals said Tuesday that a clinical trial for its experimental drug to treat a rare, fatal brain disease has reached full enrollment.

The New Haven company said it completed the enrollment ahead of schedule despite challenges from COVID-19. Results are expected by the end of 2021.

The trial, called M-STAR, is testing the safety and efficacy of Biohaven’s drug verdiperstat for patients with Multiple System Atrophy (MSA), a progressive and fatal neurodegenerative disease. 

The disease afflicts around 50,000 people in the United States and Europe. Patients usually die within six to 10 years of diagnosis, according to Biohaven. 

Around 300 MSA patients in roughly 50 sites in the United States and Europe are taking part in the 48-week study, Biohaven said. 

Biohaven licensed verdiperstat, an MPO inhibitor, from drug giant AstraZeneca in 2018. It works by targeting sources of brain inflammation that can contribute to brain cell death in neurodegenerative diseases.

Biohaven is also testing the drug for the treatment of ALS (amyotrophic lateral sclerosis) as part of a unique clinical trial being led by Massachusetts General Hospital.

Contact Natalie Missakian at news@newhavenbiz.com