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Biohaven Receives FDA May Proceed Letter For Phase 3 Clinical Trial Of BHV-3241 For Multiple System Atrophy

NEW HAVEN, Conn., Jan. 23, 2019 /PRNewswire/ -- Biohaven Pharmaceutical Holding Company Ltd. (NYSE: BHVN) ("Biohaven") announced today the U.S. Food and Drug Administration (FDA) has notified the Company that it may proceed with its clinical investigation of BHV-3241, a novel myeloperoxidase (MPO) inhibitor, which Biohaven is developing as a potential treatment for multiple system atrophy (MSA). The FDA May Proceed Letter was received following Biohaven's reactivation of the investigational new drug (IND) application initially filed by AstraZeneca prior to licensing the compound to Biohaven.

BHV-3241 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. MPO inhibition is a promising therapeutic strategy for treating a number of neurodegenerative diseases, including MSA.

Vlad Coric, M.D., CEO of Biohaven, said, "We are very pleased to report the FDA's acceptance of the IND for BHV-3241 for the treatment of MSA. We believe BHV-3241 has the potential to be the first effective treatment for people living with MSA and expect to start our Phase 3 trial in the third quarter of 2019."

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.

Irfan Qureshi, M.D., Executive Director of Neurology at Biohaven, commented, "People living with MSA urgently need new therapies. BHV-3241 offers the potential to treat MSA by inhibiting myeloperoxidase enzyme activity, thereby alleviating oxidative stress and neuroinflammation." He added, "AstraZeneca's Phase 2 clinical trial suggested that BHV-3241 may have reduced MSA disease progression in a dose-dependent manner, measured by the Unified MSA Rating Scale. We are excited to advance into a Phase 3 trial to assess the efficacy of BHV-3241 for the treatment of MSA."

About BHV-3241

BHV-3241 is a potential first-in-class, oral, brain-penetrant, irreversible inhibitor of MPO, which Biohaven is developing as a treatment for MSA. BHV-3241 also has the potential to be developed in a number of other disease indications associated with oxidative stress, inflammation, and neurodegeneration. Approximately 250 healthy volunteers and patients have been treated with BHV-3241 in Phase 1 and Phase 2 clinical trials performed by AstraZeneca. Results from a Phase 2 trial in MSA showed evidence of target engagement and favorable trends over 12 weeks on the Unified MSA Rating Scale, an exploratory clinical outcome measure. Biohaven licensed BHV-3241 from AstraZeneca in September 2018, where it was known as AZD3241.

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 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, including the Company's plans for the clinical development and potential regulatory approval and commercialization of BHV-3241 and other product candidates for various neurological conditions, the potential for BHV-3241 to be an effective treatment for neurodegenerative disorders including multiple system atrophy and the potential for BHV-3241 to be a first-in-class treatment for multiple system atrophy, 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 BHV-3241, and whether the results observed in AstraZeneca's prior studies and clinical trials of BHV-3241 will be observed in Biohaven's future clinical trials of BHV-3241 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.

SOURCE Biohaven Pharmaceutical Holding Company Ltd.

Posted: January 2019

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