Date: 2016-12-22
Type of information: Recruitment of the first patient
phase: 2-3
Announcement: recruitment of the first patient
Company: Biohaven Pharmaceuticals (USA - CT)
Product: BHV-4157
Action
mechanism: BHV-4157 is a new chemical entity (NCE) that modulates brain glutamate, for the treatment of spinocerebellar ataxias. 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. In May 2016, the FDA granted orphan drug designation for BHV-4157 for the treatment of spinocerebellar ataxias.
Disease: spinocerebellar ataxias
Therapeutic area: Rare diseases - Neurodegenerative diseases - Neurological diseases
Country: USA
Trial
details: The purpose of this study is to compare the efficacy of BHV-4157 versus placebo on ataxia symptoms in subjects with spinocerebellar ataxia (SCA). (NCT02960893)
Latest
news: * On December 22, 2016, Biohaven Pharmaceutical announced 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. 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.
