Praxis Reports Early-Stop Success In EMBOLD Trial, Advancing Relutrigine For SCN2A And SCN8A Epileptic Encephalopathies

Praxis Precision Medicines announced strong positive results from the registrational cohort of its EMBOLD study evaluating relutrigine in patients with SCN2A and SCN8A developmental and epileptic encephalopathies (DEEs). The Data Monitoring Committee recommended stopping the study early due to clear efficacy, marking an important milestone for a population with severe, treatment-resistant seizure disorders.

Following these findings, the FDA has scheduled a meeting in the coming weeks to review the data and discuss potential regulatory pathways. Praxis plans to determine the timing of its New Drug Application (NDA) submission for relutrigine after the agency meeting. This positions relutrigine as one of the most advanced precision-medicine candidates for DEEs.

“SCN2A and SCN8A DEEs are devastating conditions with extremely high mortality due to the debilitating seizure burden they impose on patients, and there are currently no approved treatment options. Our progress represents an important milestone towards delivering the first therapy ever designed for these children and their families” said Marcio Souza, president and chief executive officer. “We look forward to sharing the results at the American Epilepsy Society Annual Meeting.”

Relutrigine is a first-in-class small molecule that selectively inhibits persistent sodium current, a major driver of seizure activity in patients with severe DEEs. Its precision NaV channel modulation provides enhanced selectivity for hyperexcitable, disease-state sodium channels. Preclinical studies showed dose-dependent and near-complete seizure suppression in SCN2A and SCN8A mouse models.

The therapy has demonstrated a consistent and favorable safety profile across Phase 1 studies and in cohort 1 of the Phase 2 EMBOLD trial, showing both short- and long-term seizure reduction in heavily pre-treated patients, with some achieving complete seizure freedom. Relutrigine holds multiple regulatory designations, including Orphan Drug, Breakthrough Therapy, and Rare Pediatric Disease designations, underscoring its potential to address significant unmet needs in DEEs.