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SAGE-319 is an oral, small molecule drug that acts as an extrasynaptic-preferring positive allosteric modulator (PAM) of GABAA receptors. It is designed to enhance GABAergic neurotransmission by selectively modulating extrasynaptic GABAA receptors, which are implicated in disorders associated with GABA hypofunction. The drug is being developed primarily for the treatment of behavioral symptoms in neurodevelopmental disorders and social behavior disorders such as autism spectrum disorder and fragile X syndrome. Developed by Sage Therapeutics, SAGE-319 represents a novel pharmacological approach targeting the central nervous system to improve social interaction deficits and other behavioral symptoms linked to impaired inhibitory signaling.
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