Praxis stops EMBOLD study early after relutrigine shows efficacy

Praxis Precision Medicines Inc. (NASDAQ: PRAX) announced that its EMBOLD study evaluating relutrigine for SCN2A and SCN8A developmental and epileptic encephalopathies was stopped early for efficacy following a recommendation by the Data Monitoring Committee.

The company said the interim analysis triggered the early stop for the registrational cohort of the study. SCN2A and SCN8A developmental and epileptic encephalopathies are conditions that currently have no approved treatment options.

Praxis plans to present topline results from the EMBOLD study at the American Epilepsy Society Annual Meeting on December 6, 2025. The company has confirmed a meeting with the FDA to review the data and discuss next steps in the coming weeks.

The company will make a determination on the timing for filing a New Drug Application for relutrigine after the FDA meeting, according to the press release.

Relutrigine is described as a first-in-class small molecule designed as a preferential inhibitor of persistent sodium current. The drug has received Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA for treating SCN2A-DEE, SCN8A-DEE and Dravet syndrome.

The compound has also received Breakthrough Therapy Designation and Orphan Drug Designation from the European Medicines Agency for treating SCN2A-DEE and SCN8A-DEE.

Previous data from the first cohort of the Phase 2 EMBOLD study showed the drug was well-tolerated with improvements in motor seizures in patients with SCN2A- and SCN8A-DEE, according to the company.

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