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Biohaven Receives Authorization to Proceed from FDA and Doses First Subject with BHV-3500, Third-Generation, Small Molecule CGRP-Receptor Antagonist

- BHV-3500 is the first small molecule calcitonin gene-related peptide (CGRP) receptor antagonist to be administered in an intranasal (IN) formulation in human testing

- Nonclinical proof-of-concept with BHV-3500 has been observed in a primate assay and intranasal PK profile offers potential for rapid onset of action

- BHV-3500 is Biohaven's second CGRP-receptor antagonist to enter clinical trials from its NOJECTIONTM migraine platform

NEW HAVEN, Conn., Oct. 22, 2018 /PRNewswire/ -- Biohaven Pharmaceutical Holding Company Ltd. (BHVN), a biotechnology company focused on advancing innovative therapies for neurological diseases, today announced that the first participant was dosed with BHV-3500, a third generation calcitonin gene-related peptide (CGRP) receptor antagonist.  The initiation of dosing follows Biohaven's filing of an investigational new drug application (IND) for BHV-3500 in the treatment of migraine and receipt from FDA of authorization to proceed with the clinical investigation of this product candidate.

Intranasal BHV-3500 is administered using the Aptar Pharma Unit Dose System (UDS) designed to enable systemic delivery of drugs. The device is approved with multiple drug products marketed in the United States using the Aptar technology. (Photo credit: Aptar Pharma)

Vlad Coric, M.D., CEO of Biohaven, commented, "We are pleased with the FDA's acceptance of the IND for BHV-3500, and the team at Biohaven is excited to have now dosed our first subject in the Phase 1 clinical trial of this novel CGRP-receptor antagonist.  BHV-3500's intranasal dosing is complementary to our lead migraine asset rimegepant, which has established efficacy and safety in Phase 3 clinical trials. Intranasal BHV-3500 offers the potential for a rapid onset of action without the need for an injection. Our goal is to provide migraine sufferers with a range of noninvasive dosing options for both the acute and preventive treatment of migraine." 

Biohaven's initial clinical trial of BHV-3500 will explore a range of doses using intranasal administration. Intranasal BHV-3500 utilizes the Aptar Pharma Unit Dose System (UDS), which is designed to enable systemic delivery of drugs without the need for injection or administration by a healthcare professional. This device is approved with multiple drug products marketed in the United States using the Aptar technology and is used by thousands of people every day. 

Robert Croop, M.D., Biohaven's Chief Development Officer – Neurology, added, "People with migraine seek convenient, rapid, long-lasting, and non-invasive treatments. Our development program with both rimegepant and BHV-3500 seeks to provide patients with a range of novel treatment formulations to treat their migraines when and where they attack."

BHV-3500 is the second of Biohaven's CGRP-targeting compounds to enter clinical trials. Biohaven's multiple CGRP product candidates, including rimegepant, and formulations including oral tablet, Zydisâ (Catalent) rapid dissolving tablet and intranasal delivery are designed to meet patients' needs across the spectrum from acute treatment to prevention of migraine.

About BHV-3500

BHV-3500, the second product candidate in our CGRP platform, is a highly soluble small molecule CGRP receptor antagonist. It is also structurally distinct from rimegepant. The physicochemical properties of BHV-3500 make the product candidate potentially suitable for multiple routes of delivery, including nasal, subcutaneous, inhalation or oral administration. It is initially in development as an intranasal formulation for the acute treatment of migraine. Nonclinical proof-of-concept of BHV-3500 has been observed in a preclinical marmoset assay, and no cardiovascular safety or systemic toxicity issues have been observed in preclinical testing.

About Rimegepant

Rimegepant is Biohaven's orally-dosed CGRP receptor antagonist, which the Company is developing as a treatment for migraine. Rimegepant represents a novel mechanism that targets the underlying pathophysiology of migraine without causing vasoconstriction. The efficacy and safety profile of rimegepant for the acute treatment of migraine has now been established across three randomized controlled trials to date: the two completed pivotal Phase 3 trials, and a previously reported Phase 2b trial. The co-primary endpoints achieved in the two Phase 3 trials are consistent with regulatory guidance from the U.S. Food and Drug Administration (FDA) and provide the basis for a planned submission of a new drug application (NDA) to the FDA in 2019.

About Migraine

Over 36 million Americans suffer from migraine. Acute attacks of migraine can differ in intensity and frequency, with many being highly disabling. More than 90 percent of migraine sufferers are unable to work or function normally during an attack. In the Global Burden of Disease Study, updated in 2015, migraine was ranked as the seventh highest cause worldwide of years lost due to disability. CGRP receptor antagonists represent a novel class of drug candidates for the treatment of migraine and are the first new class specific to the acute treatment of migraine in over 25 years. This unique and specific mode of action potentially offers an alternative to current agents, particularly for patients who have contraindications to the use of triptans, such as those with underlying cardiovascular diseases, or who either do not respond or have inadequate or inconsistent response to triptans or are intolerant to them.

About Aptar Pharma

Aptar Pharma is part of AptarGroup, Inc., a leading global supplier of a broad range of innovative dispensing and sealing solutions for the beauty, personal care, home care, prescription drug, consumer health care, injectables, food and beverage markets. AptarGroup is headquartered in Crystal Lake, Illinois, with manufacturing facilities in North America, Europe, Asia and South America. For more information, visit aptar.com/pharma. Media Contact: Carolyn Penot, Aptar Pharma, +33 1 39 17 20 38, carolyn.penot@aptar.com

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 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 statements about the potential safety and efficacy of BHV-3500 as a treatment for migraine, as well as its potential for rapid onset and sustained activity, the potential of the Company's CGRP product candidates, including rimegepant, to provide  an improved, effective and safe treatment option for the acute treatment of migraine and prevention of migraine, the potential FDA approval of rimegepant and the Company's expected timelines for submissions to regulatory authorities, are forward-looking statements. The use of certain words, including "believe," "potential" and "will" and similar expressions, is 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 those described in the "Risk Factors" section of the Company's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on August 14, 2018 and other filings Biohaven makes with the U.S. Securities and Exchange Commission from time to time. 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, Biohaven at Vlad.Coric@biohavenpharma.com

Biohaven’s NOJECTION™ Migraine Platform Formulations.
Biohaven Pharmaceuticals Logo (PRNewsfoto/Biohaven Pharmaceutical Holding)

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