NEW HAVEN, CT, USA I September 29, 2022 I Biohaven Pharmaceutical Holding Company Ltd. ( NYSE: BHVN) today announced results from a focused analysis of treatment with verdiperstat in the HEALEY ALS Platform Trial in amyotrophic lateral sclerosis (ALS). The HEALEY ALS Platform Trial, the first-ever platform trial in ALS, is designed as an adaptive trial to evaluate multiple investigational treatments simultaneously, thus accelerating the development of effective and breakthrough treatments for people living with ALS.

Verdiperstat did not statistically differentiate from placebo on the prespecified primary efficacy outcome, disease progression measured by the ALS Functional Rating Scale-Revised and survival, nor the key secondary efficacy measures during the 24-week study period. Initial analysis of safety data was consistent with the overall profile of verdiperstat from prior clinical trial experience. Additional analyses are ongoing, and complete study results will be presented at an upcoming scientific meeting.

Irfan Qureshi, M.D., Senior Vice President of Neurology at Biohaven, stated, “We are incredibly grateful to our collaborators at the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital, HEALEY ALS Platform Trial study investigators, and the ALS community – especially people with ALS and their families –– who made the trial possible. While we are disappointed that verdiperstat did not demonstrate efficacy for ALS, Biohaven remains committed to developing treatments for people who suffer from neurodegenerative diseases.”

About ALS
ALS is a progressive, life-threatening, and rare neuromuscular disease that affects approximately 30,000 people in the United States. The median age of onset is 55 years and average survival is 3-5 years after onset of first symptoms. ALS is characterized by the loss of motor neurons in the brain, brainstem, and spinal cord that leads to progressive muscle weakness and difficulties in speaking, swallowing, and breathing. There are currently limited treatment options and no cure for ALS. 

About Biohaven
Biohaven is a global 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 (EMA-approved as Vydura™ (rimegepant) for the acute treatment of migraine with or without aura, and prophylaxis of episodic migraine in adults who have at least four migraine attacks per month) and a broad pipeline of late-stage product candidates across five distinct mechanistic platforms: CGRP receptor antagonism for the acute and preventive treatment of migraine; glutamate modulation for obsessive-compulsive disorder and spinocerebellar ataxia; and MPO inhibition for amyotrophic lateral sclerosis; Kv7 Ion Channel Activators (Kv7) for focal epilepsy and neuronal hyperexcitability, and myostatin inhibition for neuromuscular diseases. More information about Biohaven is available at

SOURCE: Biohaven Pharmaceutical Holding Company