Basmisanil
Basmisanil (also known as RG-1662 and RO5186582) is an experimental drug candidate for the treatment of cognitive impairment associated with Down syndrome. It is being developed by Roche and is currently in Phase II clinical trials.
Mechanism of Action
Basmisanil is a negative allosteric modulator of the alpha-5 subunit of the GABA-A receptor. It is hypothesized to work by reducing the excessive inhibition in the brains of individuals with Down syndrome, thereby improving cognitive function.
Clinical Trials
Basmisanil has undergone Phase I and II clinical trials. The Phase II trial, conducted by Roche, aimed to assess the safety, tolerability, and efficacy of the drug in individuals with Down syndrome aged between 12 and 30 years. However, the results of the trial, published in 2020, showed that Basmisanil did not meet its primary endpoint of improving cognitive function.
Future Development
Despite the disappointing results of the Phase II trial, research into the potential benefits of Basmisanil continues. Roche has stated that they remain committed to exploring new treatment options for individuals with Down syndrome.
See Also
References
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Contributors: Prab R. Tumpati, MD
