Praxis Announces Positive EMBRAVE Trial Results Showing Significant Seizure Reduction With Elsunersen In Pediatric SCN2A Epilepsy

Praxis Precision Medicines, Inc. has reported positive topline results from the EMBRAVE Part A clinical trial evaluating elsunersen in pediatric patients with early-onset seizure SCN2A developmental and epileptic encephalopathy (DEE). The Phase 1/2 randomized, placebo-controlled study assessed both safety and efficacy in children aged 2 to 12 years, with participants receiving treatment every four weeks over a 24-week period, followed by an open-label extension.

The trial demonstrated strong efficacy outcomes, with elsunersen achieving a 77% placebo-adjusted reduction in seizure frequency. More than half of the treated patients experienced at least 28 consecutive days without seizures, and these benefits were sustained for up to one year in the extension phase. In addition to seizure control, all treated patients showed improvements in areas such as sleep, motor skills, muscle tone, attention, and overall neurodevelopment, while no such improvements were observed in the placebo group.

“We are thrilled to see the remarkable, consistent results from EMBRAVE Part A, showing 77% reduction in monthly seizures and disease modifying improvements in children with SCN2A early-seizure onset DEE. We are well underway with our pivotal EMBRAVE3 study and look forward to sharing this placebo-controlled data from the EMBRAVE Part A study with all key stakeholders,” said Marcio Souza, president and chief executive officer.

From a safety perspective, elsunersen was well tolerated across all tested doses, with no drug-related serious adverse events or treatment discontinuations reported. Most adverse events were mild to moderate in severity and aligned with previous study findings, with no evidence of neuroinflammation observed during the trial.

Overall, these findings highlight the potential of elsunersen as a promising therapeutic option for SCN2A-related epilepsy, a rare and severe neurological condition. Further data from the EMBRAVE study are expected to be presented at upcoming scientific conferences.