About tuberous sclerosis complex (TSC)-related epilepsy

TSC is a rare genetic disorder of the mTOR pathway caused by mutations in TSC1 or TSC2, with multi-organ involvement and high rates of epilepsy. An estimated ~50,000 people in the U.S. live with TSC, and 80–90% experience seizures—many of which remain refractory to current therapies.  

Why new options are needed  

Despite available antiseizure medications, mTOR inhibitors, and surgery, many individuals with TSC continue to have seizures or face significant treatment burdens. There is a clear need for novel, non-invasive, mechanism-based approaches.  

Our approach  

Simufilam targets a protein call filamin A (“FLNA”) in the brain of patients with central nervous system disorders, such as TSC.  Our and our collaborators’ published studies have demonstrated that changes in the expression or conformation of FLNA are linked to seizures, neuronal abnormalities, and neuroinflammation in certain circumstances and disorders. Our goal is to reduce seizure frequency and improve quality of life in patients with TSC.