First Patient in Pivotal Trial of BHV-4157 in Patients With Hereditary Spinocerebellar Ataxia

Biohaven Pharmaceutical Holding Company Ltd. (“Biohaven” or the “Company”) announced today that it has enrolled the first patient in its potentially pivotal Phase 2b/3 clinical trial assessing the efficacy and safety of its drug candidate BHV-4157 in patients with hereditary spinocerebellar ataxia (SCA). SCA is a rare and debilitating neurodegenerative disorder with no currently approved therapies. In May 2016, the U.S. Food and Drug Administration (FDA) granted the company’s request for orphan drug designation for BHV-4157, a new chemical entity (NCE) that modulates brain glutamate, for the treatment of SCA. SCA is estimated to affect approximately 10,000 to 20,000 people in the United States. The standard of care treatment is supportive treatment, and no medications are currently approved for this debilitating condition.
Glutamate is one of the most important neurotransmitters in the central nervous system that is present in more than 90% of all brain synapses. Agents that modulate glutamate neurotransmission may have therapeutic potential in multiple disease states involving glutamate dysfunction, including SCA, amyotrophic lateral sclerosis (ALS), Alzheimer’s disease, Rett syndrome, dementia, dystonia, tinnitus, anxiety disorders, and affective disorders like major depressive disorder.
Vlad Coric M.D., Chief Executive Officer at Biohaven, commented, “Enrolling the first patient in our SCA trial is an important milestone and demonstrates Biohaven’s commitment to exploring novel treatments for individuals suffering from severe neurologic conditions.” Dr. Coric added, “Treatment with BHV-4157 represents a promising therapeutic option for patients, and this trial advances our glutamate modulating platform into the clinic.”
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