New Haven, Connecticut July 6, 2017 -Biohaven Pharmaceutical Holding Company Ltd. (NYSE: BHVN) announced today that BHV-5000, an investigational compound in the company’s glutamate modulation platform, has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA) for the treatment for patients with Rett syndrome. This is the company’s fourth product candidate to receive orphan drug designation from the FDA.
Rett syndrome is a rare and severe genetic neurodevelopmental disorder affecting females almost exclusively. Symptoms emerge from six to 18 months of age after apparently normal post-natal development. Patients with Rett syndrome develop global deceleration of psychomotor function, loss of acquired cognitive skills (such as language and purposeful hand movement) as well as brain-mediated episodes of transient respiratory suppression. Patients may survive into adulthood, yet they are severely physically and cognitively impaired. Rett syndrome affects approximately 15,000 individuals in the United States. No approved drug therapies for Rett syndrome are currently available and care is supportive.
“BHV-5000 is a novel glutamate antagonist that Biohaven plans to evaluate for efficacy across several therapeutic indications,” said Vlad Coric, M.D., CEO of Biohaven. “Our clinical development program in Rett syndrome is particularly important because patients and their families currently have no approved drugs for this severe neurodevelopmental disorder. The orphan drug designation for BHV-5000 in Rett syndrome supports Biohaven’s global development strategy of providing improved therapies for patients suffering from some of the most disabling neurologic disorders.”
Potential benefits of orphan drug designation include tax credits for 50% of clinical trial costs, waiver of marketing application user fees (estimated at over $2 million in savings) and 7 years of marketing exclusivity if regulatory approval is ultimately received.
“Patients with Rett syndrome develop debilitating symptoms,” said Robert Berman, M.D., Chief Medical Officer of Biohaven. “Preclinical studies on BHV-5000 have demonstrated benefits in transgenic mouse models of Rett syndrome, particularly on symptoms of abnormal breathing. We are eager to test BHV-5000 in patients with Rett syndrome to understand if these preclinical effects translate into therapeutic benefits for patients.”
Biohaven expects to initiate a Phase 1 trial of BHV-5000 in healthy volunteers with a commercially-ready formulation in 2017, and then proceed to a study in patients with Rett syndrome in 2018.
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. The company’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.
BHV-5000 is a low-trapping, potent N-methyl-D-aspartate (NMDA) receptor antagonist licensed to Biohaven from AstraZeneca in October 2016. BHV-5000 has previously advanced into Phase 1 study and its active metabolite, lanicemine, was previously advanced through Phase 2 studies. Unlike other agents in this class, BHV-5000 is a low-trapping NMDA antagonist that has not been associated with the prominent psychotomimetic effects which have plagued other NMDA targeting compounds. In addition, BHV-5000 is orally bioavailable.
About Orphan Drug Designation
The FDA, through its Office of Orphan Products Development (OOPD), grants orphan status to drugs and biologic products that are intended for the safe and effective treatment, diagnosis, or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S. Orphan drug designation provides a drug developer with certain benefits and incentives, including a period of marketing exclusivity if regulatory approval is ultimately received for the designated indication.
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 future development of BHV-5000, the potential benefits of orphan drug status and the Company’s business and product candidate plans and objectives are forward-looking statements. The use of certain words, including the “believe” 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-5000, and whether the preclinical effects of BHV-5000 observed in preclinical models of Rett syndrome will be observed in human patients. 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 June 16, 2017. 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. Vladimir Coric, the Chief Executive Officer at Vlad.Coric@biohavenpharma.com