First Patient Enrolled in Hereditary Spinocerebellar Ataxia Pivotal Trial

First Patient Enrolled in Hereditary Spinocerebellar Ataxia Pivotal Trial
BioHaven Pharmaceuticals has enrolled the first patient in their Phase 2b/3 clinical trial assessing their orphan drug, BHV-4157 for the treatment of hereditary spinocerebellar ataxia (SCA).

The Phase 2b/3 trial is a multicenter randomized, double-blind, placebo-controlled trial with an estimation enrollment of 120 SCA patients. The study will look to see if BHV-4157 will change the total score on the Scale for Assessment and Rating of Ataxia (SARA) from baseline in the patients administered.

If the trial shows positive results, BioHaven will submit a New Drug Application (NDA) for BHV-4157.

For more information on this clinical trial, visit
About BHV-4157
BHV-4157 modulates the body’s glutamate which is the major mediator of excitatory signals in the CNS and is involved in many aspects of normal brain function including cognition, memory and learning.

Drugs such as BHV-4157 that modulate glutamate neurotransmission can have therapeutic application towards numerous diseases, including amyotrophic lateral sclerosis (ALS), spinocerebellar ataxia, Alzheimer’s disease, neurodevelopmental disorders, cancer, anxiety disorders and treatment resistant-depression.
About Spinocerebellar Ataxia (SCA)
SCA is a form of genetically inherited disorder that is characterized by abnormalities in the person’s brain functioning. The cerebellum is a key area that is often atrophied in these patients, resulting in progressive loss of coordination.

There are a number of gene mutations that cause SCA. It does not differentiate between genders, both females and males are equally likely to inherit a defective gene. To date, over 60 different types of SCA have been identified.


More interesting news Alan that gives me hope. I hope there will be more interest in this as it may benefit other disorders. Thanks for posting

Thank you

“We are all in this together”

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