Soticlestat, formerly known as TAK-935/OV935, is an investigational drug developed by Takeda Pharmaceuticals. It is a highly selective, potent inhibitor of the enzyme cholesterol 24-hydroxylase, which plays a crucial role in the regulation of neurosteroid synthesis. Neurosteroids are naturally occurring molecules in the brain that modulate neuronal activity, including in epilepsy.

Our recent study adds to a growing body of evidence to suggest that this compound may represent a novel therapeutic treatment option for people with epilepsy. In any case, this agent is targeting an entirely untested molecular target – cholesterol 24-hydroxylase. The preclinical studies conducted on soticlestat have yielded promising results that highlight its potential as a therapeutic option for epilepsy:

1. 1. Seizure Reduction: In rodents, soticlestat has demonstrated a significant reduction in the frequency and severity of seizures. Our findings with the TMEV model of infection-induced epilepsy demonstrate that soticlestat can significantly reduce seizure burden.
   2. Improved Cognitive Function: Epilepsy often leads to cognitive impairments, but soticlestat has shown the ability to improve cognitive function in preclinical models.
   3. Safety Profile: Preclinical studies have indicated minimal side effects in experimental animal. In our study with TMEV-induced acute seizures, animals did not exhibit any marked infection-related body weight loss during the acute infection period.
   4. Mechanism of Action: Soticlestat’s mechanism of action, specifically targeting a brain-specific mechanism of cholesterol catabolism catalyzed by cholesterol 24-hydroxylase, represents a novel approach to epilepsy treatment. Whether clinical trials will ultimately provide the efficacy demonstration to bring this agent to market remains to be established, but will provide clearer understanding of soticlestat’s potential as a groundbreaking therapeutic option for epilepsy.