GM-91466

GM-91466 is a preclinical small molecule drug candidate being developed by JD Bioscience for the treatment of Dravet syndrome (severe myoclonic epilepsy of infancy, SMEI). Discovered through joint research by the Gwangju Institute of Science and Technology (GIST) and the Korea Research Institute of Chemical Technology (KRICT), GM-91466 features a novel 1,3,4-oxadiazolone scaffold. Unlike traditional Dravet syndrome therapies that directly stimulate serotonin receptors, GM-91466 acts by upregulating the expression of tryptophan 5-hydroxylase 2 (TPH2), the rate-limiting enzyme in brain serotonin synthesis. This increases endogenous serotonin levels and restores the excitation-inhibition balance in neural circuits. In preclinical zebrafish and mouse models of SCN1A dysfunction, oral administration of GM-91466 significantly reduced seizure frequency and intensity, delayed seizure onset, and demonstrated a favorable safety and pharmacokinetic profile.

Other names

3-(2-chloro-4-fluorobenzyl)-5-(2-(trifluoromethyl)pyridin-4-yl)-1,3,4-oxadiazol-2(3H)-one