Biohaven Announces FDA Acceptance of 505(b)(2) NDA Filing for BHV-0223, a Novel Sublingual Delivery Form of Riluzole for the Treatment of Amyotrophic Lateral Sclerosis (ALS)

NEW HAVEN, Conn., Nov. 26, 2018 /PRNewswire/ -- Biohaven Pharmaceutical Holding Company Ltd. (NYSE: BHVN) today announced that its 505(b)(2) new drug application (NDA) for BHV-0223, an investigational sublingual form of riluzole for the treatment of people living with amyotrophic lateral sclerosis (ALS), has been accepted for review by the U.S. Food and Drug Administration (FDA). Biohaven previously announced that the FDA granted orphan drug designation for BHV-0223 in ALS.  BHV-0223 is a novel, lower dose formulation of riluzole designed to be placed under the tongue where it dissolves in seconds and is absorbed sublingually. The active ingredient, riluzole, is the only FDA-approved treatment to extend tracheostomy-free survival in people living with ALS.

"We are extremely proud to announce the FDA acceptance for review of our 505(b)(2) NDA submission of BHV-0223, as it represents Biohaven's first NDA. If approved, we believe BHV-0223 would provide an important advancement in treatment for people living with ALS, including the large number of patients who have difficulty swallowing tablets or liquids," said Robert Berman, M.D., Chief Medical Officer of Biohaven. "We are committed to designing new treatment options for life-altering neurological diseases and believe that our sublingual, orally-dissolving tablet formulation of riluzole can provide a meaningful alternative for people living with ALS."

ALS is a progressive neurodegenerative motor neuron disease affecting nerve cells in the brain and the spinal cord in which patients experience muscle weakness, trouble swallowing and muscle atrophy that ultimately progresses to paralysis, impaired breathing and death. More than 80% of people living with ALS experience dysphagia, which is difficulty swallowing, during their disease course.

The 505(b)(2) NDA submission is supported by data from multiple studies and clinical trials evaluating the bioequivalence of BHV-0223 to riluzole oral tablets (Rilutek®), as well as the safety and tolerability of BHV-0223. Key findings from the clinical program showed that sublingual administration of BHV-0223 (40 mg) achieved similar blood exposures to orally ingested Rilutek (50 mg). In these studies and trials, BHV-0223 was generally well tolerated. Patients also reported the sublingual formulation easy to use in clinical trials in which BHV-0223 was administered to patients with ALS and dysphagia.

About BHV-0223

BHV-0223 is a sublingually administered, orally dissolving tablet (ODT) that makes use of the unique Zydis® ODT fast-dissolve technology. It is being developed under an exclusive worldwide agreement with Catalent. While riluzole is FDA-approved for ALS, existing formulations consist only of an oral tablet taken with water or an oral suspension (thickened liquid). BHV-0223 is designed to be placed under the tongue where it dissolves in seconds and is absorbed by the vasculature, thereby eliminating the need for swallowing.

About ALS and Dysphagia

ALS is a progressive, neurodegenerative motor neuron disease that affects nerve cells in the brain and the spinal cord. The disease belongs to a group of disorders known as motor neuron diseases, which are characterized by the gradual degeneration and death of motor neurons. ALS affects approximately 20,000 individuals in the United States. Since its FDA's approval in 1995, riluzole remains the only approved drug demonstrated to enhance survival and/or time to tracheostomy.

More than 80% of people living with ALS experience dysphagia at some point in their disease course. Even mild or moderate dysphagia can lead to difficulty managing small oral boluses such as tablets through the swallowing process. The impaired ability to swallow safely puts people with ALS at risk of aspiration and its potentially life-threatening consequences such as aspiration pneumonia.

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 AB, Yale University, Catalent, Rutgers, 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 may contain 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 belief of the clinical efficacy and safety of BHV-0223 and its ability to become the standard of care and improve upon existing treatment options, are forward-looking statements. 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. 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 [email protected]

SOURCE Biohaven Pharmaceutical Holding Company Ltd.

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