Biohaven Provides Update From Pivotal Phase 2/3 Trial with Verdiperstat in Amyotrophic Lateral Sclerosis (Healy ALS Platform Trial)


NEW HAVEN, Conn., Sept. 29, 2022 /PRNewswire/ -- Biohaven Pharma 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

Forward-Looking Statements
This news release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. The use of certain words, including "believe", "continue", "may", "will", "anticipate", "expect" and similar expressions, are intended to identify forward-looking statements. These forward-looking statements involve substantial risks and uncertainties, including statements that are based on the current expectations and assumptions of Biohaven's management about verdiperstat as a treatment for patients with ALS. Factors that could affect these forward-looking statements include those related to Biohaven's ability to effectively develop verdiperstat. Various important factors could cause actual results or events to differ materially from those that may be expressed or implied by forward-looking statements. Additional important factors to be considered in connection with forward-looking statements are described in the "Risk Factors" section of the Company's Annual Report on Form 10-K filed with the Securities and Exchange Commission on February 25, 2022, and in Biohaven's subsequent filings with the Securities and Exchange Commission. The forward-looking statements are made as of the date of this new release, and Biohaven does not undertake any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.

NURTEC and NURTEC ODT are registered trademarks of Biohaven Pharmaceutical Ireland DAC. VYDURA is a trademark of Biohaven Pharmaceutical Ireland DAC. Neuroinnovation is a trademark of Biohaven Pharmaceutical Holding Company Ltd.

Biohaven Contact:
Jennifer Porcelli
VP, Investor Relations

Media Contact:
Mike Beyer
Sam Brown Inc.


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SOURCE Biohaven Pharmaceutical Holding Company Ltd.

Company Codes: NYSE:BHVN

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