Biohaven's Verdiperstat, An Oral Myeloperoxidase Inhibitor, Selected For Platform Trial Collaboration At Massachusetts General Hospital's Healey & AMG Center For Amyotrophic Lateral Sclerosis (ALS)

NEW HAVEN, Conn., Sept. 18, 2019 /PRNewswire/ -- Biohaven Pharmaceutical Holding Company Ltd. (NYSE: BHVN) ("Biohaven" or the "Company"), a clinical stage biopharmaceutical company with a portfolio of late-stage product candidates for neurological and neuropsychiatric diseases, announced today that the Company's myeloperoxidase (MPO) inhibitor, verdiperstat, has been selected as an investigational therapy in the first Amyotrophic Lateral Sclerosis (ALS) platform trial, an innovative program designed by the Sean M. Healey & AMG Center for ALS at Mass General to help accelerate a path to new and effective ALS treatments.

Verdiperstat is a potential first-in-class, oral, brain-penetrant, irreversible inhibitor of the MPO enzyme. MPO is believed to play an important role in multiple neurodegenerative diseases, because it increases oxidative stress and inflammation levels in the brain. Inhibiting MPO is anticipated to ameliorate these pathological mechanisms, which are strongly implicated in the onset and progression of ALS, a progressive and fatal neurodegenerative disease with limited treatment options.

The HEALEY ALS Platform Trial is being conducted by leading ALS experts from the Healey Center in collaboration with the Northeast ALS Consortium (NEALS) clinical trial network and TackleALS. Promising investigational drugs for the HEALEY ALS Platform Trial were chosen through a competitive process, with the Healey Center providing partial financial support to successful applicants. An independent advisory council selected verdiperstat for inclusion in the platform based on a thorough scientific and clinical review.

Merit Cudkowicz, MD, MSc, Director of the Healey Center, Chief of Neurology at MGH, and co-founder of NEALS commented, "We are excited to accelerate how therapies are developed for people with ALS. We are in a new era for ALS therapy development and we must think differently and boldly about how to find effective treatments for our patients."

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. In contrast with 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 using specialized statistical methods. This approach helps to maximize resources and efficiency. The Food and Drug Administration is now strongly encouraging the use of innovative clinical trial designs, such as platform trials, which can be used to provide substantial evidence of clinical efficacy to support a drug approval. More information about the HEALEY ALS Platform Trial can be found at www.massgeneral.org/als.

"We are extremely pleased to have the opportunity to study myeloperoxidase inhibition with verdiperstat as a novel therapeutic approach for people with ALS," said Irfan Qureshi, MD, Biohaven Vice President and Development Lead for verdiperstat. "This study would not be possible on these timelines without the support and collaboration of the HEALEY ALS Platform Trial."

About Verdiperstat
Biohaven licensed verdiperstat (BHV-3241) from AstraZeneca in September 2018, where it was known as AZD3241. Approximately 250 healthy volunteers and patients have been treated with verdiperstat in Phase 1 and Phase 2 clinical trials. A Phase 3 clinical trial is currently ongoing to evaluate the efficacy of verdiperstat in Multiple System Atrophy (MSA), a rare, progressive and fatal neurodegenerative disease. Additional details about the trial (NCT03952806) can be found at www.clinicaltrials.gov.

About Biohaven
Biohaven is a clinical-stage biopharmaceutical company with a portfolio of innovative, late-stage product candidates targeting neurological diseases, including rare disorders. Biohaven has combined internal development and research with intellectual property licensed from companies and institutions including Bristol-Myers Squibb Company, AstraZeneca, Yale University, Catalent, Rutgers, and ALS Biopharma LLC. Currently, Biohaven's lead development programs include multiple compounds across its CGRP receptor antagonist, glutamate modulation, and myeloperoxidase inhibitor platforms. Biohaven's common shares are listed on the New York Stock Exchange and traded under the ticker symbol BHVN. More information about Biohaven is available at 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 the Company's management. All statements, other than statements of historical facts, included in this press release regarding the Company's business and product candidate plans and objectives are forward-looking statements. Forward-looking statements include those related to: the effectiveness and safety of verdiperstat, the expected timing, commencement and outcomes of the Company'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 the Company's product candidates and the potential for the Company's product candidates to be first in class or best in class therapies. The use of certain words, including "believe", "continue", "may", "on track", "expects" and "will" and similar expressions, are intended to identify 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. 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 28, 2019 and the Company's Quarterly Report on Form 10-Q for the quarter ended June 30, 2019, filed with the Securities and Exchange Commission on August 9, 2019. The forward-looking statements are made as of this date and the Company 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
[email protected]

SOURCE Biohaven Pharmaceutical Holding Company Ltd.

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