Biohaven Completes Enrollment Of Verdiperstat Arm Of Pivotal Healey ALS Platform Trial Conducted By The Healey Center For ALS At Massachussetts General Hospital

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Nov 15, 2021
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- Approximately 160 people with Amyotrophic Latera Sclerosis (ALS) are now enrolled in the verdiperstat treatment arm of the pivotal HEALEY ALS Platform Trial

- Verdiperstat is an investigational, first-in-class, brain-penetrant, oral small molecule designed to inhibit the myeloperoxidase (MPO) enzyme, a driver of neuroinflammation in ALS and other neurodegenerative diseases

- Amyotrophic Lateral Sclerosis is a progressive, life-threatening, and rare neurodegenerative disease that affects approximately 30,000 people in the United States

PR Newswire

NEW HAVEN, Conn., Nov. 15, 2021 /PRNewswire/ -- Biohaven Pharmaceutical Holding Company Ltd. (NYSE: BHVN) ("Biohaven") announced today that enrollment is now complete in the verdiperstat arm of the HEALEY ALS Platform Trial. This first-ever platform trial in ALS, sponsored by the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital (MGH), is designed to evaluate the safety and efficacy of multiple drug candidates in people with Amyotrophic Lateral Sclerosis ("ALS"). Verdiperstat was selected as one of the inaugural investigational treatments for the HEALEY ALS Platform Trial which began enrolling patients in July 2020 across 52 sites from the Northeast ALS Consortium (NEALS). Topline results for verdiperstat in the treatment of ALS are anticipated in mid-2022.


"We are enthusiastic about achieving this enrollment goal as it translates to being one step closer to finding a life-changing treatment for ALS. Our team is grateful to the ALS community and people living with ALS who have trusted us in this first-of-its-kind trial", said Merit Cudkowicz, MD, MSc, Principal Investigator and Sponsor of the HEALEY ALS Platform Trial, Director of the Sean M. Healey & AMG Center for ALS and chief of the Department of Neurology at Mass General, and the Julieanne Dorn Professor of Neurology at Harvard Medical School.

Irfan Qureshi, MD, Biohaven Vice President and Verdiperstat Development Lead, stated, "People with ALS deserve novel and more effective therapeutic options. Biohaven is very pleased that the verdiperstat arm of the trial is fully enrolled. This important milestone brings us closer to knowing whether verdiperstat can benefit people with ALS. We are deeply grateful to the ALS community, especially the study participants and their families, for their unwavering commitment to the trial."

The HEALEY ALS Platform Trial, the first-ever platform trial in ALS, is designed to evaluate multiple investigational treatments simultaneously, thus accelerating the development of effective and breakthrough treatments for people living with ALS. The trial has enrolled approximately 160 adults with ALS into the verdiperstat regimen. Participants are 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 is measurement of 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 include change in respiratory function, muscle strength, and survival. The study is designed to support a potential regulatory filing and approval if the top line results are positive. More information about the HEALEY ALS Platform Trial can be found at

Calaneet Balas, President and CEO of The ALS Association, added, "We celebrate the successful enrollment of the HEALEY ALS Platform Trial as it moves us closer to potential new treatment options for ALS. The ALS Association is proud to support the trial and grateful to the HEALEY Center for championing this effort." More information about the ALS Association can be found at

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 Verdiperstat

Verdiperstat (BHV-3241) is an investigational first-in-class, potent, selective, brain-penetrant, and myeloperoxidase (MPO) enzyme inhibitor in development by Biohaven 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 amyotrophic lateral sclerosis. Biohaven licensed verdiperstat (BHV-3241) from AstraZeneca in September 2018, where it was known as AZD3241. 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 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 and CGRP-mediated neuroimmune/neuroinflammatory diseases; glutamate modulation for obsessive-compulsive disorder, Alzheimer's disease, and spinocerebellar ataxia; and myeloperoxidase (MPO) inhibition for 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", "continue", "may", "will" and similar expressions, are intended to identify forward-looking statements. All statements, other than statements of historical facts, included in this press release, including the expected enrollment for Biohaven's pivotal trial of verdiperstat, the potential results of Biohaven's pivotal trial of verdiperstat in ALS, the potential role of verdiperstat in ALS, the possible benefits of verdiperstat as a disease-modifying therapy for ALS patients, as well as the timetable for the topline data and completion of trials, are forward-looking statements. 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 Biohaven's Annual Report on Form 10-K for the year ended December 31, 2020, filed with the Securities and Exchange Commission on March 1, 2021, and in Biohaven's subsequent filings with the Securities and Exchange Commission. 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
[email protected]


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SOURCE Biohaven Pharmaceutical Holding Company Ltd.

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