NEW HAVEN, CT, USA I September 27, 2021 I Biohaven Pharmaceutical Holding Company Ltd. (NYSE: BHVN) today announced results from a focused analysis of a clinical trial of verdiperstat in multiple system atrophy (MSA).  Verdiperstat did not statistically differentiate from placebo on the prespecified primary efficacy measure, nor on the key secondary efficacy measures. Initial analysis of safety data was consistent with the overall profile of verdiperstat from prior clinical trial experience. Additional analyses are still pending, and full study results will be presented at an upcoming scientific meeting.

Irfan Qureshi, M.D., Vice President of Neurology at Biohaven commented, “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. We are extremely grateful to the international MSA community – especially the patients and their families, investigators and their teams, and patient advocacy groups – who made the trial possible.”

Verdiperstat is an investigational first-in-class, potent, selective, brain-penetrant, and irreversible myeloperoxidase (MPO) enzyme inhibitor that Biohaven is developing for the treatment of neurodegenerative diseases. Verdiperstat may help preserve neurons through inhibition of MPO-induced pathological oxidative stress and further inflammation that contribute to cellular injury in neurodegenerative disease.

Although the mechanism of action for verdiperstat, myeloperoxidase inhibition, was shown not to be effective for MSA, the rationale of targeting brain inflammation remains strong in other disease states.  An ongoing clinical trial evaluating the efficacy of verdiperstat in amyotrophic lateral sclerosis (ALS) is being conducted in collaboration with the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital and is expected to complete enrollment in the fourth quarter of 2021.

About MSA
MSA is a rare, rapidly progressive, severely debilitating, and fatal neurodegenerative disease that leads to death within a median of 6-10 years after the onset of symptoms. Manifestations of MSA can include urinary and sexual dysfunction, dizziness and fainting due to low blood pressure (orthostatic hypotension), and motor impairments such as tremor, rigidity, unsteady gait, and difficulty speaking and swallowing. The most common causes of death in MSA are infection and cardiopulmonary complications. Currently, patients receive only symptomatic and palliative therapies as there are no disease-modifying treatments and no cure for MSA. 

About Biohaven
Biohaven is a commercial-stage biopharmaceutical company with a portfolio of innovative, best-in-class therapies to improve the lives of patients with debilitating neurological and neuropsychiatric diseases, including rare disorders. Biohaven’s neuroinnovation portfolio includes FDA-approved NURTEC ODT (rimegepant) for the acute and preventive treatment of migraine and a broad pipeline of late-stage product candidates across three distinct mechanistic platforms: CGRP receptor antagonism for the acute and preventive treatment of migraine; glutamate modulation for obsessive-compulsive disorder, Alzheimer’s disease, and spinocerebellar ataxia; and MPO inhibition for neurodegenerative disease, including ALS. More information about Biohaven is available at

SOURCE: Biohaven Pharmaceutical Holding Company