Biohaven Announces Submission of IND for BHV-3500, Third-Generation, Small Molecule CGRP-Receptor Antagonist for the Treatment of Migraine

NEW HAVEN, Conn., Sept. 12, 2018 /PRNewswire/ -- Biohaven Pharmaeutical Holding Company Ltd. (NYSE: BHVN), a biotechnology company focused on advancing innovative therapies for neurological diseases, today announced that it has submitted an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) to evaluate the safety and tolerability of BHV-3500, the first small molecule calcitonin gene-related peptide (CGRP) receptor antagonist to be advanced into the clinic in an intranasal formulation. BHV-3500 is the second of Biohaven’s CGRP-targeting compounds to reach this developmental milestone. Biohaven’s multiple CGRP product candidates, including rimegepant, and formulations including oral tablet, Zydis^® (Catalent) rapid dissolving tablet and intranasal delivery are designed to meet patients needs for both acute treatment and prevention of migraine.

“BHV-3500 has novel properties including high water solubility and a high free fraction which allows for a low dose that we believe is well suited to intranasal delivery,” said Charlie Conway, Ph.D., Chief Scientific Officer of Biohaven. “BHV-3500 also exhibits high affinity binding to the human CGRP receptor with a preference to remain bound to the receptor much longer than unbound, and when paired with intranasal administration has the potential for both rapid onset of action and sustained activity.”

Biohaven’s 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 in a range of scenarios.

“People with migraine seek rapid, long-lasting, convenient, and non-invasive treatments. We are pleased to advance BHV-3500 toward first in human dosing and look forward to expanding our current CGRP platform. BHV-3500’s intranasal dosing has the potential to complement rimegepant, which has established efficacy and safety in Phase 3 clinical trials, and provides convenient dual options of a traditional tablet and a rapid dissolving oral formulation,” said Elyse Stock, M.D., Chief of Portfolio Strategy and Development. “We believe it is important for migraine sufferers to have a range of dosing options for the acute and preventive treatment of migraine and look forward to expanding our CGRP platform with the addition of intranasal delivery and the potential for rapid onset.”

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 chemical 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. Preliminary proof-of-concept of BHV-3500 has been observed in a preclinical marmoset assay with oral delivery, 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 and glutamate modulation 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 provde an improved, effective and safe treatment option for the acute treatment of migraine and prevention of migraine, the potential FDA approval of rimegepantand 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

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SOURCE Biohaven Pharmaceutical Holding Company Ltd.