NEW HAVEN, CT–(Marketwired – July 05, 2016) – Biohaven Pharmaceutical Holding Company Ltd. (“Biohaven” or the “Company”) announced today that the U.S. Food and Drug Administration (FDA) has completed its review of the company’s investigational new drug application (IND) for BHV-4157 filed on May 31, 2016 and informed Biohaven that clinical trials in humans may proceed. The IND for BHV-4157 includes plans for a pivotal trial in the indication of Spinocerebellar Ataxia (SCA), a rare and debilitating neurodegenerative disorder with no currently approved treatment. Biohaven plans to initiate a pivotal Phase III clinical trial in SCA before the end of the year.
BHV-4157 is a new chemical entity (NCE) that modulates glutamate, the most abundant excitatory neurotransmitter in the human body. Agents that modulate glutamate neurotransmission may have therapeutic potential in multiple disease states involving glutamate dysfunction, including amyotrophic lateral sclerosis (“ALS”), SCA, Alzheimer’s disease, Rett syndrome, dementia, dystonia, tinnitus, anxiety disorders, affective disorders like major depressive disorder and cancers.
Vlad Coric, M.D., CEO at Biohaven, commented, “The successful filing of our IND and approval by the FDA to proceed with clinical trials is another major milestone for Biohaven. Our team has demonstrated how efficiently and professionally it can advance drug candidates into the clinic.” Biohaven has now advanced two investigational drugs into clinical testing, BHV-0223 and BHV-4157. Dr. Coric added, “The active metabolite of BHV-4157 is known to modulate glutamate and confer neuroprotective effects. Given the fact that glutamate abnormalities have been implicated in a number of neurodegenerative disorders, BHV-4157 has the potential to become a pipeline within a single drug candidate.”
Declan Doogan, M.D., Chairman of Biohaven’s Board of Directors, added, “We are excited to project that Biohaven is poised for significant growth this next year with two internal glutamate programs progressing into the clinic and we are actively pursuing in-licensing opportunities to further expand the pipeline. The next 1-2 years will also be important for the Company as it transitions development products into the market.”
Biohaven fully acquired world-wide intellectual property rights to over 300 prodrugs of a glutamate modulating agent as well as other innovative technologies from ALS Biopharma LLC. Biohaven then entered into a strategic alliance with Fox Chase Chemical Diversity Center, Inc. (FCCDC) to optimize a lead prodrug candidate. FCCDC identified an optimal prodrug candidate in BHV-4157, which Biohaven subsequently advanced into the required IND-enabling studies as well as developed a commercial-ready formulation to support pivotal trials.
Kim Gentile, VP of Clinical Operations, stated, “Planning for success, our team anticipated potential clearance from the FDA to begin clinical testing of BHV-4157 and we have already prepared our clinical trial site for the first pharmacokinetic study to begin dosing subjects within the next few weeks. Results from this study will establish dose levels for our pivotal trial in SCA that is expected to initiate within approximately the next 6 months.” SCA is a rare, debilitating neurodegenerative disorder that is estimated to affect approximately 150,000 people in the United States. Standard of care treatment is supportive and no medications are approved for this debilitating condition.
Robert Berman, M.D., Chief Medical Officer at Biohaven, commented, “The team continues to execute on its strategy of advancing drug programs first for orphan indications and then expanding to other larger disease areas. BHV-0223 will be assessed in an upcoming bioequivalence study designed to support an NDA for ALS. BHV-4157 will enter a pivotal trial in SCA as soon as dosing exposures are confirmed in our initial pharmacokinetic study.” Biohaven has previously received orphan drug designation status from FDA for BHV-0223 and BHV-4157.
Biohaven is a privately-held biopharmaceutical company engaged in the identification and development of clinical stage compounds targeting the glutamatergic system and other neurological pathways. Biohaven has licensed intellectual property from Yale University, Catalent, ALS Biopharma LLC and Massachusetts General Hospital. Biohaven is owned by a group of investors including Portage Biotech Inc. (OTC PINK: PTGEF) (CNSX: PBT.U), Yale University, and other private investors. The Company’s first drug candidate, BHV-0223, is a novel formulation of a glutamate-modulating agent, being developed under FDA 505(b)(2) guidelines. The FDA cleared the Company’s IND for BHV-0223 in August 2015, and Biohaven has completed a pharmacokinetic study in humans and is planning to launch a pivotal bioequivalence study by 4Q2016. Biohaven’s second clinical compound, BHV-4157, is a New Molecular Entity (NCE) for neurodegenerative diseases, neuropsychiatric disorders and potentially cancer indications. The Company plans to advance other glutamatergic approaches and is actively exploring licenses for additional compounds. Further information regarding Biohaven can be found at: http://biohavenpharma.com
About Fox Chase Chemical Diversity Center, Inc. (FCCDC)
Biohaven entered into a strategic alliance with FCCDC in October 2015 to develop Biohaven’s family of over 300 prodrugs of glutamate modulating agents as well as support other innovative initiatives. Under this agreement, Biohaven has provided research funding and other support to advance its early drug discovery platform. FCCDC is a Pennsylvania-based drug discovery company with exceptional medicinal chemistry, target validation, pharmacology and chemical biology capabilities. FCCDC has approximately 22 staff members and over 300 years of cumulative drug discovery experience ranging from big pharma (Johnson & Johnson, Merck) to numerous biotech companies. FCCDC is led by CEO Dr. Allen Reitz with has extensive experience in project and portfolio management, target validation, hit triage, hit to lead and lead optimization medicinal chemistry, eADME profiling, and preclinical candidate selection. Through its strategic alliance with FCCDC, Biohaven has the capabilities for drug discovery from medicinal chemistry through pivotal clinical trials. Further information about FCCDC can be found at: http://www.fc-cdci.com
About Investigational New Drug Applications
The Investigational New Drug (IND) Application is the process by which the FDA provides a sponsor with permission or an exemption for a drug candidate to be transported or distributed across state lines. A sponsor must collect sufficient data and information necessary to establish that the candidate drug product will not expose humans to unreasonable risks when used in limited, early-stage clinical studies. Once an IND is submitted to the FDA, the Agency has the opportunity to review the IND Application to assure that research subjects will not be subjected to unreasonable risk. Further information regarding IND Applications can be found at:http://www.fda.gov/drugs/developmentapprovalprocess/howdrugsaredevelopedandapproved/approvalapplications/investigationalnewdrugindapplication/default.htm
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. Further information regarding Orphan Drug Designation can be found at: http://www.fda.gov/forindustry/developingproductsforrarediseasesconditions/howtoapplyfororphanproductdesignation/default.htm
This news release includes forward-looking statements within the meaning of the U.S. federal and Canadian securities laws. 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 regarding the Company’s plans and objectives, expectations and assumptions of management are forward-looking statements. The use of certain words, including the words “estimate,” “project,” “intend,” “expect,” “believe,” “anticipate,” “will, “plan,” “could,” “may” and similar expressions are intended to identify forward-looking statements. The Company may not actually achieve the plans, intentions or expectations 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 receipt of regulatory approvals and market conditions. 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.