Elsunersen demonstrated placebo-adjusted seizure reduction from baseline of 77% (p=0.015)

71% of elsunersen-treated patients achieved >50% seizure reduction by period 6, with sustained benefit observed in the open-label extension for up to one year

100% of elsunersen patients - and none on placebo - had additional improvements, including sleep, motor function, muscle tone and attention

No treatment-emergent or serious adverse events related to study drug reported

BOSTON, April 06, 2026 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQ:PRAX), a fully integrated, leading central nervous system (CNS) precision neuroscience biopharmaceutical company, today announced positive topline results from the EMBRAVE Part A trial of elsunersen in pediatric patients with early-seizure onset SCN2A developmental and epileptic encephalopathy (DEE). 

 

"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.

EMBRAVE Part A is a randomized, placebo-controlled Phase 1/2 trial evaluating the safety and efficacy of ascending doses of elsunersen in patients with SCN2A DEE. Nine patients, aged 2-12 years old, were randomized 3:1 to receive either elsunersen or sham procedure every 4 weeks for 24 weeks, followed by an open-label extension (OLE); all 9 patients continued to the OLE. Patients received a starting dose of 1 mg with optional dose escalation based on observed seizure reduction and individual tolerability.

Key results from EMBRAVE Part A

Efficacy

  • Elsunersen treatment led to a 77% placebo-adjusted seizure reduction from baseline (p=0.015, 95 CI [33,92]
  • 57% of patients had at least a 28-day period of seizure freedom
  • Efficacy was sustained in the OLE for up to one year
  • 100% of elsunersen patients improved across sleep, motor function, muscle tone, attention or neuropsychomotor development compared to no improvements in placebo group

Safety

  • Elsunersen was well-tolerated, with no drug-related SAEs, no discontinuations and no neuroinflammation signals at doses up to 8 mg
  • Most treatment-emergent adverse events (TEAEs) were mild to moderate. TEAEs were consistent with the EMBRAVE Part 1 study

Additional results will be presented at upcoming scientific meetings.