Biohaven Announces Enrollment Of First Patients In Pivotal HEALEY ALS Platform Trial Including Verdiperstat Conducted By Healey Center For ALS At Massachusetts General Hospital

NEW HAVEN, Conn., Aug. 3, 2020 /PRNewswire/ -- Biohaven Pharmaceutical Holding Company Ltd. (NYSE: BHVN) ("Biohaven" or the "Company") announced today that the first patients were enrolled in the HEALEY ALS Platform Trial, which is evaluating the efficacy of three 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.

Calaneet Balas, President and CEO of The ALS Association

Irfan Qureshi, MD, Biohaven Vice President and Verdiperstat Development Lead, stated, "Biohaven continues to advance its novel pipeline across a variety of CNS diseases, and we are so pleased to enroll the first patient in a trial of verdiperstat in ALS. Given the tremendous burden of the disease on people with ALS and their families, more effective treatments are urgently needed. It is imperative that we rapidly and efficiently study promising new treatments such as verdiperstat, which targets brain inflammation. The HEALEY ALS Platform Trial will allow us to efficiently enroll this trial and determine if verdiperstat has the potential to help people with ALS."

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, MSc, Director of the Healey Center and Chief of Neurology at Mass General, commented, "I am proud of our collaborators and grateful for the support of this courageous community. We are excited to be at this point and thankful to all teams involved for their invaluable input and support."

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 www.massgeneral.org/als.

"By testing multiple drugs at the same time, this platform trial will dramatically speed our search for effective ALS treatments for everyone living with ALS. The ALS Association is proud to be supporting this effort, and we are grateful to the Healey Center for their leadership," said Calaneet Balas, President and CEO of The ALS Association. More information about the ALS Association can be found at www.als.org.

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. Biohaven recently announced completion of enrollment in the M-STAR study, a Phase 3 clinical trial to assess the safety and efficacy of verdiperstat in MSA. Additional details can be found at clinicaltrials.gov/ct2/show/NCT03952806. Verdiperstat has received Orphan Drug and Fast Track designations for MSA from the US FDA, as well as Orphan Drug designation from the European Medicines Agency. More information about the HEALEY ALS Platform Trial can be found at www.massgeneral.org/als and clinicaltrials.gov/ct2/show/NCT04436510. More information about can be found at the Company's website: www.biohavenpharma.com/science-pipeline/mpo/verdiperstat.

About Biohaven

Biohaven is a biopharmaceutical company focused on the development and commercialization of innovative best-in-class therapies to improve the lives of patients with debilitating neurological and neuropsychiatric diseases. 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 myeloperoxidase inhibition for multiple system atrophy and amyotrophic lateral sclerosis. For more information, visit www.biohavenpharma.com.

Forward-Looking Statements

This news release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements involve substantial risks and uncertainties, including statements that are based on the current expectations and assumptions of Biohaven's management. 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. The use of certain words, including the "believe" and "will" and similar expressions are intended to identify forward-looking statements. Biohaven may not actually achieve the plans and objectives disclosed in the forward-looking statements and you should not place undue reliance on Biohaven's forward-looking statements. 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, including uncertainties relating to the future clinical success of troriluzole. 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 filed with the Securities and Exchange Commission on February 26, 2020 and Biohaven's Quarterly Report on Form 10-Q for the quarter ended March 31, 2020, filed with the Securities and Exchange Commission on May 7, 2020. 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.

Biohaven Contact

Dr. Vlad Coric
Chief Executive Officer
Vlad.Coric@biohavenpharma.com

Biohaven Pharmaceuticals Logo (PRNewsfoto/Biohaven Pharmaceutical Holding)

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SOURCE Biohaven Pharmaceutical Holding Company Ltd.

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