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Biohaven's Verdiperstat Receives Orphan Drug Designation From FDA For Multiple System Atrophy

NEW HAVEN, Conn., Feb. 19, 2019 /PRNewswire/ -- Biohaven Pharmaceutical Holding Company Ltd. (NYSE: BHVN) ("Biohaven") announced today that it received orphan drug designation from the U.S. Food and Drug Administration (FDA) for its product candidate verdiperstat (previously BHV-3241), a novel myeloperoxidase (MPO) inhibitor, for the treatment of multiple system atrophy (MSA).

Verdiperstat is a potential first-in-class, oral, brain-penetrant, irreversible inhibitor of MPO, an enzyme that acts as a key driver of pathological oxidative stress and inflammation in the brain. Verdiperstat also has received orphan drug designation for the treatment of MSA from the European Commission upon recommendation from the European Medicines Agency's Committee for Orphan Medicinal Products.

Irfan Qureshi, M.D., Biohaven Executive Director and development lead for verdiperstat, commented, "We are very pleased the FDA granted orphan drug designation for verdiperstat. This highlights Biohaven's commitment to developing innovative therapies in areas of high unmet medical need. We believe verdiperstat has the potential to be the first effective treatment for people living with MSA and we remain on track to start our global Phase 3 clinical trial later this year."

Verdiperstat completed Phase 1 clinical trials at doses up to 900mg twice a day and preliminary results from a Phase 2a trial in patients with MSA showed numerical improvements on the change from baseline Unified MSA Rating Scale. After 12 weeks of treatment, placebo-treated patients worsened by 4.6 points (SE=1.1, n=17) on the Unified MSA Rating Scale, while verdiperstat-treated patients worsened by 3.7 points (SE= 1.2, n=17) at the 300mg twice-daily dose and by 2.6 points (SE=1.4, n=18) at the 600mg twice-daily dose. Corresponding benefits were also observed in other outcome measures, such as the Composite Autonomic Symptom Score and MSA-Quality of Life scale. These clinical findings were consistent with neuroprotective effects of verdiperstat observed in animal models. In the Phase 2a trial, verdiperstat significantly decreased MPO activity in human blood, a biomarker of the drug engaging its target. Verdiperstat has been generally safe and well tolerated in approximately 250 patients.

Marianne Frost, Head of Regulatory Affairs at Biohaven, commented "The orphan drug designations from both the FDA and European Medicines Agency underscore the high unmet medical need in MSA and support Biohaven's efforts to advance verdiperstat as a potential first-in-class, oral, MPO inhibitor treatment for people with this severe neurological disease."

Potential benefits of orphan drug designation include tax credits, waiver of marketing application user fees and 7 years of marketing exclusivity if regulatory approval is ultimately received.

About MSA

MSA is a rare, rapidly progressive, and fatal neurodegenerative disease that leads to death after an average of 6 to 10 years from disease onset. MSA causes Parkinson's disease-like movement problems (e.g., 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. There is no cure for MSA. Only symptomatic and palliative therapies are available.

About Verdiperstat

Verdiperstat is a potential first-in-class, oral, brain-penetrant, irreversible inhibitor of MPO that Biohaven is developing as a treatment for MSA. Verdiperstat also has the potential to be developed in a number of other disease indications associated with oxidative stress, inflammation, and neurodegeneration. Biohaven licensed verdiperstat from AstraZeneca in September 2018, where it was known as AZD3241.  Verdiperstat has also been known as BHV-3241 at Biohaven.

About Biohaven

Biohaven is a clinical-stage biopharmaceutical company with a portfolio of innovative, late-stage product candidates targeting neurological diseases. Biohaven combines internal development and research with intellectual property licensed from companies and institutions including Bristol-Myers Squibb Company, AstraZeneca AB, Yale University, Catalent, ALS Biopharma LLC and Massachusetts General Hospital.  Currently, Biohaven's lead development programs include multiple compounds across its CGRP receptor antagonist, glutamate modulation, and MPO 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

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, including the Company's plans for the clinical development and potential regulatory approval and commercialization of verdiperstat and other product candidates for various neurological conditions, the potential for verdiperstat to be an effective treatment for neurodegenerative disorders including MSA, the potential for verdiperstat to be a first-in-class treatment for MSA, and the potential benefits to Biohaven of orphan drug designation are forward-looking statements. The use of certain words, including "potential", "believe", "could", "expect", "may" and "will" and similar expressions are intended to identify forward-looking statements. The Company may not actually achieve the plans and objectives disclosed in the forward-looking statements and you should not place undue reliance on the Company'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 verdiperstat, and whether the results observed in AstraZeneca's prior studies and clinical trials of verdiperstat will be observed in Biohaven's future clinical trials of verdiperstat in various neurodegenerative disorders, including those beyond multiple system atrophy. Additional important factors to be considered in connection with forward-looking statements are described in the "Risk Factors" section of the Company's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on November 14, 2018. 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.

For further information, contact Dr. Vlad Coric, the Chief Executive Officer at

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Last Updated: 19-Feb-2019