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SAGE-324 (also known as BIIB124) is an investigational oral neuroactive steroid and small molecule that acts as a positive allosteric modulator of the GABA A receptor. By binding to both synaptic and extrasynaptic GABA A receptors, it enhances inhibitory activity in the GABAergic system—the primary inhibitory neurotransmission system in the brain. This mechanism is relevant because dysregulation of GABA signaling has been implicated in essential tremor and other neurological disorders. SAGE-324 was developed by Sage Therapeutics, with Biogen as a collaborator for some indications. The drug reached Phase 2 clinical trials for essential tremor but was discontinued for this indication in 2024 due to lack of efficacy. It remains under investigation at earlier stages for epilepsy[1][3][4][5][6].
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