Soticlestat

Soticlestat (TAK-935, OV-935) is an experimental anticonvulsant and cholesterol 24-hydroxylase inhibitor being investigated as a treatment for Dravet syndrome, Lennox–Gastaut syndrome, tuberous sclerosis complex, dup15q syndrome, and CDKL5 deficiency disorder. The development rights to the drug were purchased by Takeda Pharmaceuticals from Ovid Therapeutics in 2021.

Soticlestat was designated as an orphan drug by the FDA in 2017 for the treatment for both Dravet syndrome and Lennox–Gastaut syndrome. In a phase 2 study called ELEKTRA, soticlestat was well tolerated and reduced seizure frequency in patients with Dravet syndrome and Lennox–Gastaut syndrome.

Mechanism of action
Soticlestat functions by blocking cholesterol 24-hydroxylase (CH24H, also known as CYP46A1), an enzyme in the brain that converts cholesterol to the oxysterol 24S-hydroxycholesterol (24S-HC). Reduction of 24S-HC has been shown to reduce glutamatergic signaling, which reduces seizures. Soticlestat may also have neuroprotective and anti-inflammatory properties via glial cell modulation.