Biohaven Enrolls First Patient In Phase 3 Clinical Trial Of Verdiperstat, Oral Myeloperoxidase Inhibitor, For The Treatment Of Multiple System Atrophy

7/31/2019 7:45:20 AM

(MENAFN- PR Newswire) NEW HAVEN, Conn., July 31, 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 it enrolled the first patient in a Phase 3 clinical trial to evaluate the efficacy and safety of verdiperstat in subjects with Multiple System Atrophy ("MSA").

Jeremy D. Schmahmann, MD, Professor of Neurology at Harvard Medical School and Founding Director of the Massachusetts General Hospital Ataxia Unit

Verdiperstat is a potential first-in-class, oral, brain-penetrant, irreversible inhibitor of myeloperoxidase, an enzyme that acts as a key driver of pathological oxidative stress and inflammation in the brain. Results from a previous Phase 2 trial in MSA completed by AstraZeneca AB showed evidence of target engagement and favorable trends over 12 weeks on the Unified MSA Rating Scale, an exploratory clinical outcome measure. Verdiperstat has received orphan drug designation for the treatment of MSA from the FDA Office of Orphan Products Development as well as the European Commission upon recommendation from the European Medicines Agency's Committee for Orphan Medicinal Products.

"We are so pleased to partner with Biohaven in building hope for the patients and families affected by this devastating disease. This clinical trial presents an exciting opportunity to empower people living with MSA by playing an active role in research," commented Pamela Bower of the MSA Coalition. "We are grateful for Biohaven's commitment to joining our community in standing up against MSA!" added Philip Fortier of Defeat MSA. More information about the MSA Coalition and Defeat MSA is available at www.multiplesystematrophy.org andwww.defeatmsa.org .

MSA is a rare, rapidly progressive, and fatal neurodegenerative disease. Only symptomatic and palliative therapies are currently available. MSA causes Parkinson's disease-like movement problems (slow movement, rigid muscles, tremor, and poor balance), cerebellar ataxia, as well as problems with involuntary (autonomic) functions, including blood pressure control, bladder function, and digestion. MSA usually leads to death after an average of 6 to 10 years from the onset of symptoms.

Irfan Qureshi, MD, Biohaven Vice President and Development Lead for Verdiperstat, commented, "Verdiperstat is the first product candidate from Biohaven's neuroinflammation platform and highlights our commitment to developing innovative medicines for neurological diseases with high unmet need."

Biohaven expects to recruit approximately 250 patients in the study, across approximately 50 sites in the United States and Europe. The study will enroll subjects with MSA diagnosed using consensus clinical criteria and include both subtypes of MSA, MSA-Parkinsonism (MSA-P) and MSA-Cerebellar (MSA-C). Researchers will evaluate the efficacy of verdiperstat, compared to placebo, as measured by a change from baseline in a modified version of the Unified MSA Rating Scale (UMSARS) at Week 48. Additional details about the trial (NCT03952806) can be found atwww.clinicaltrials.gov .

Jeremy D. Schmahmann, MD, Professor of Neurology at Harvard Medical School and Founding Director of the Massachusetts General Hospital Ataxia Unit, stated, "We are excited to have enrolled the first patient in this study. Working together with our patients and the Biohaven team, we are optimistic that this is the beginning of a new era of treatment and eventually cure for this challenging neurodegenerative disorder."

About Verdiperstat Biohaven licensed verdiperstat (BHV-3241) from AstraZeneca AB in September 2018, where it was known as AZD3241. Approximately 250 healthy volunteers and patients were treated with verdiperstat in Phase 1 and Phase 2 clinical trials. Results from a Phase 2 trial in MSA showed evidence of target engagement and favorable trends over 12 weeks on the UMSARS, an exploratory clinical outcome measure. Verdiperstat received Orphan Drug designation from the U.S Food and Drug Association as well as the European Commission due to the unmet medical need in MSA. Verdiperstat also has the potential to be developed in a number of other diseases associated with oxidative stress, inflammation, and neurodegeneration.

About BiohavenBiohaven 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 AB, 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 StatementsThis 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 March 31, 2019, filed with the Securities and Exchange Commission on May 8, 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.

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Dr. Vlad CoricChief Executive Officer

SOURCE Biohaven Pharmaceutical Holding Company Ltd.