Biological Activity
Soticlestat is a cholesterol 24-hydroxylase inhibitor used in cholesterol turnover and neuronal-excitability research. The authors estimated 40–53 mg/kg/day from food intake and body weight; actual exposure depends on intake.
In Vitro
No separate compound-specific cell or biochemical result is available from the sources summarized here.
In Vivo
| Endpoint | Model / conditions / source | Reported result |
|---|---|---|
| Exposure and model | Scn1a-haploinsufficient Dravet mouse model; 0.02% soticlestat chow. [1], Methods | The authors estimated 40–53 mg/kg/day from food intake and body weight; actual exposure depends on intake. |
| Brain target response | Brain 24S-hydroxycholesterol measurements. [1] | Treatment lowered brain 24HC by approximately 50%, supporting cholesterol-24-hydroxylase inhibition. |
| Seizures and survival | Spontaneous generalized tonic–clonic seizures and survival in Scn1a+/− mice. [1] | Soticlestat reduced spontaneous seizure burden and prevented premature lethality in the studied cohort. |
| Evoked seizure response | Hyperthermia-induced seizure tests; 7-day chow-treatment experiment. [1] | Treatment increased resistance to hyperthermia-provoked seizures. The findings are model-specific and do not establish effects in every epilepsy syndrome. |
Interpretation
Complete prevention of this mouse endpoint does not establish prevention of sudden death in patients.
