Pivotal study evaluating the efficacy, safety, tolerability, and pharmacokinetics of relutrigine, a precision sodium channel modulator, in pediatric participants with early onset SCN2A- and SCN8A-related developmental and epileptic encephalopathies (DEEs).

DEEs are devastating pediatric epilepsies, characterized by frequent, refractory seizures, global developmental impairment and increased early mortality. Relutrigine is a differentiated functional state sodium channel modulator with demonstrated superior selectivity for disease-state hyperexcitability, a known cause of seizure manifestation in all DEEs. Building on the promising, well-tolerated efficacy observed in Cohort 1 through open label extension (OLE), Cohort 2 of the ongoing EMBOLD study (NCT05818553) is designed to confirm efficacy and further define the therapeutic profile of relutrigine in a larger pediatric population.

The primary endpoint is the change from baseline in motor seizure frequency. Secondary endpoints will include incidence and severity of TEAEs, and further efficacy assessments based on seizure freedom and clinical (CGI) and caregiver (CgGI) global impression of improvement and severity. 

Cohort 2 represents the pivotal, registration-enabling phase of the EMBOLD study in patients with SCN2A- and SCN8A-DEE. Efficacy and safety data are expected to further define relutrigine’s therapeutic potential and support its advancement as a precision sodium channel modulator for broad DEEs.