Biohaven Announces Completion Of 50% Enrollment Ahead Of Timelines In Verdiperstat Pivotal HEALEY ALS Platform Trial At Massachusetts General Hospital

  • Biohaven's verdiperstat was selected as one of the first three drug candidates to be studied in the innovative HEALEY ALS Platform Trial at MGH
  • Enrollment in the verdiperstat regimen has passed 50%
  • Verdiperstat is a brain-penetrant inhibitor of myeloperoxidase (MPO), an enzyme implicated in neuroinflammatory and neurodegenerative disorders including ALS

NEW HAVEN, Conn., March 10, 2021 /PRNewswire/ -- Biohaven Pharmaceutical Holding Company Ltd. (NYSE: BHVN) ("Biohaven" or the "Company") announced today that more than 50% of participants have been enrolled in the verdiperstat regimen of HEALEY ALS Platform Trial, which is evaluating the efficacy of several treatments including verdiperstat in people with amyotrophic lateral sclerosis ("ALS"). Verdiperstat is a potential first-in-class, brain-penetrant, selective inhibitor of myeloperoxidase in development by Biohaven for the treatment of neurodegenerative diseases, including ALS and multiple system atrophy.

Irfan Qureshi, MD, Biohaven Vice President and Verdiperstat Development Lead, stated, "We are pleased that enrollment in the trial is progressing rapidly and very grateful to our collaborators at the Healey Center and the ALS research community for their leadership, boundless enthusiasm, and perseverance. We are also deeply thankful to people with ALS and their families for their commitment to the HEALEY ALS Platform Trial."

The HEALEY ALS Platform Trial is a major innovation in the field of ALS drug development that is aimed at accelerating the identification of effective new treatments. The HEALEY ALS Platform Trial is being conducted across over 50 sites by leading ALS experts from the Healey Center in collaboration with the Northeast ALS Consortium clinical trial network. In contrast to traditional trials that typically evaluate only one drug at a time, platform trials create an infrastructure for the ongoing study of multiple investigational drugs in parallel thereby maximizing resources and efficiency.

Merit Cudkowicz, MD, director of the Sean M. Healey & AMG Center for ALS at Mass General and Chief of the Department of Neurology, "We are very thankful that despite the challenges of COVID-19, all collaborators involved in the HEALEY ALS Platform Trial have come together to make sure people with ALS are safely able to enroll in this groundbreaking trial."

The HEALEY ALS Platform Trial will study approximately 160 adults with ALS for each treatment. Participants will be randomized in a 3-to-1 ratio to be treated with verdiperstat 600 mg oral tablet twice daily or placebo for 24 weeks. The study's primary efficacy endpoint will measure the change in disease severity from baseline to week 24 on the ALS Functional Rating Scale-Revised (ALSFRS-R) in patients receiving treatment versus placebo. Secondary endpoints will include change in respiratory function, muscle strength, and survival. More information about the HEALEY ALS Platform Trial can be found at

ALS is a progressive, life-threatening and rare neuromuscular condition that affects approximately 30,000 people in the United States. 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 Verdiperstat

Verdiperstat (BHV-3241) is an investigational first-in-class, potent, selective, brain-penetrant, and 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 inflammation that contribute to cellular injury in neurodegenerative diseases such as ALS and multiple system atrophy (MSA). Biohaven licensed verdiperstat (BHV-3241) from AstraZeneca in September 2018, where it was known as AZD3241. More information about the HEALEY ALS Platform Trial can be found at and More information about verdiperstat can also be found at the Company's website:

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 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 multiple system atrophy and amyotrophic lateral sclerosis. 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", "may" and "will" 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 ALS. Forward-looking statements include those related to: Biohaven's ability to effectively commercialize verdiperstat, delays or problems in the supply or manufacture of verdiperstat, complying with applicable U.S. regulatory requirements, the expected timing, commencement and outcomes of Biohaven's planned and ongoing clinical trials, the timing of planned interactions and filings with the FDA, the timing and outcome of expected regulatory filings, the potential commercialization of Biohaven's product candidates, the potential for Biohaven's product candidates to be first in class or best in class therapies and the effectiveness and safety of Biohaven's product candidates. Various important factors could cause actual results or events to differ materially from those that may be expressed or implied by our 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 March 1, 2021. The forward-looking statements are made as of this date 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.

Biohaven Contact

Dr. Vlad Coric
Chief Executive Officer

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SOURCE Biohaven Pharmaceutical Holding Company Ltd.

Company Codes: NYSE:BHVN

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