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Praxis Precision Medicines Announces Positive Results from the EMBRAVE Part A Trial of Elsunersen in Patients with SCN2A Early-Onset Developmental and Epileptic Encephalopathy
Elsunersen demonstrated placebo-adjusted seizure reduction from baseline of 77% (p=0.015) 71% of elsunersen-treated patients achieved >50% seizure reduction
About this update from Praxis Precision Medicines, Inc.
[{"type":"text","content":"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 freedomEfficacy was sustained in the OLE for up to one year100% 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 mgMost tr...