soticlestat

Soticlestat is a first-in-class, potent and selective small molecule inhibitor of cholesterol 24-hydroxylase (CH24H or CYP46A1), an enzyme in the brain responsible for converting cholesterol to 24S-hydroxycholesterol (24HC). By inhibiting CH24H, soticlestat reduces levels of 24HC, which in turn decreases glutamatergic signaling and neuronal hyperexcitability—mechanisms implicated in seizure disorders. Soticlestat is being developed primarily as an adjunctive therapy for treatment-resistant epilepsies such as Dravet syndrome and Lennox-Gastaut syndrome. It has also been investigated for other rare epilepsies including tuberous sclerosis complex, dup15q syndrome, CDKL5 deficiency disorder, and complex regional pain syndrome. The drug was originally developed by Ovid Therapeutics; Takeda acquired development rights in 2021[3][5][7].

Other names

soticlestatTAK-935TAK935TAK 935OV-935OV935OV 935