Biohaven Pharmaceutical Holding Company Ltd. (Biohaven), a privately-held biopharmaceutical company, 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 US 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."

Biohaven expects to enroll approximately 120 patients in this randomized, double-blind, placebo-controlled trial across approximately 15 sites in the United States. Researchers will evaluate acute symptomatic treatment with BHV-4157 in patients with SCA. The primary outcome measure is the  change from baseline in the total score on the Scale for Assessment and Rating of Ataxia (SARA). The trial will also assess the safety, tolerability and pharmacokinetics of BHV-4157. If the results of the pivotal trial are positive, Biohaven expects to be able to submit a New Drug Application (NDA) for BHV-4157 for the treatment of SCA.

"The prognosis for these patients is poor," said Theresa A. Zesiewicz, M.D., Professor of Neurology and Director of the University of South Florida Ataxia Research Center and an investigator in the trial. Dr. Zesiewicz added, "There is no FDA-approved treatment for SCA, which progresses relentlessly. BHV-4157 offers hope for much-needed new therapies. We are pleased to have enrolled the first patient in this clinical trial and look forward to further investigating BHV-4157 in patients living with SCA."

Robert Berman M.D., chief medical officer at Biohaven, stated, "Biohaven has worked with the leading ataxia experts to design this trial, and we are excited to see the culmination of our efforts with the initiation of patient enrollment. We will be working closely with investigators to complete this trial in an efficient manner with the highest quality data to assess whether BHV-4157 has efficacy in SCA."

Spinocerebellar Ataxias, or SCAs, are disorders of the cerebellum and its outflow tracts that are characterized clinically by progressive ataxia and are attributed to various autosomal dominant genetic mutations. While different mutations may lead to some variation in clinical presentation, the typical clinical course of SCAs may be described as starting with balance and coordination impairment. Incoordination of hands, arms, and legs, and slurring of speech are other common early symptoms. Walking becomes difficult and is characterized by a gait with feet placed further apart to compensate for poor balance. Impaired coordination of the arms and hands affects the ability to perform tasks requiring fine motor control such as writing and eating. As time goes on, ataxia can affect speech and swallowing and some patients require wheelchair assistance. There are no approved treatments for SCAs.

Biohaven is a privately-held biopharmaceutical company with particular expertise in neurologic and rare diseases, with a portfolio of multiple late-stage drug candidates.