First Patient Enrolled in Spinocerebellar Ataxia Clinical Trial

According to a story from Market Screener, the biopharmaceutical company Biohaven Pharmaceutical Holding Company Ltd. recently announced that the first patient has been enrolled in the company’s Phase 3 clinical trial. This trial will test the company’s experimental drug troriluzole as a treatment for spinocerebellar ataxia, a rare neurodegenerative disorder. Biohaven is focused on the development of therapies for neurological diseases.

About Spinocerebellar Ataxia

Spinocerebellar ataxia (SCA), also known as spinocerebellar atrophy, is a progressive, degenerative disease that affects the cerebellum, a portion of the brain that is critical for movement control and coordination. These heritable disorders can often be fatal. There are several different types of spinocerebellar ataxia which are linked to a variety of genetic mutations; many types are caused by repeats of the CAG gene. Symptoms of these disorders include ataxia (abnormal gait), seizures, poor hand coordination, speech difficulties, peripheral neuropathy, chorea, and cognitive impairments. Unfortunately there is no cure for spinocerebellar ataxia and treatment is mostly focused on relieving symptoms. Physical and occupational therapy can help patients maintain their mobility. Gene therapy could be a potential future option for treating spinocerebellar ataxia. There is a dire need for more effective treatments for this disorder. To learn more about spinocerebellar ataxia, click here.

About The Clinical Trial

Prior findings from earlier clinical trials has led Biohaven to extend the treatment period to an entire year. This trial will also incorporate a larger dose of troriluzole (200 mg). The clinical trial will include a total of 230 patients and it will be conducted at 22 different sites across the US. This study is intended to treat patients with a number of different types of spinocerebellar ataxia and is expected to include participants with at least four different disease genotypes (SCA6, SCA3, SCA2, and SCA1).

About Troriluzole

Troriluzole is a unique medication that serves as a modulator of glutamate. Glutamate is the most common neurotransmitter found in the human body. The drug’s modulation effect results in reducing the synaptic levels of this neurotransmitter. This causes the synaptic uptake of glutamate to increase.

There is a dire need for disease modifying therapies for spinocerebellar ataxia that have the capability of slowing, halting, or reversing the progression of these disorders. Hopefully, this late stage trial will confirm that troriluzole will be a breakthrough treatment for this indication.


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