Biohaven Pharmaceutical Holding Company Ltd. (Biohaven), a privately-held biopharmaceutical company, announced that the US 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 programmes 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.