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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

PRAX
  • Praxis Precision Medicines 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).
  • The Phase 1/2 randomized, placebo-controlled study enrolled 9 patients aged 2-12 years who received elsunersen or a sham procedure every 4 weeks for 24 weeks.
  • Elsunsersen treatment demonstrated a 77% placebo-adjusted seizure reduction from baseline (p=0.015) with a 95% confidence interval of [33,92].
  • 71% of elsunersen-treated patients achieved greater than 50% seizure reduction by period 6, with 57% experiencing at least one 28-day period of seizure freedom.
  • Sustained efficacy was observed in the open-label extension for up to one year.
  • 100% of elsunersen patients showed additional improvements in sleep, motor function, muscle tone, attention, or neuropsychomotor development, whereas the placebo group showed no improvements.
  • Elsunsersen was well-tolerated with no treatment-emergent serious adverse events (SAEs), no discontinuations, and no signals of neuroinflammation at doses up to 8 mg.
  • Most treatment-emergent adverse events were mild to moderate \& consistent with previous study data.
  • Elsunsersen is an antisense oligonucleotide designed to decrease SCN2A gene expression in patients with gain-of-function SCN2A mutations.
  • The drug has received Orphan Drug Designation (ODD) \& Rare Pediatric Disease Designation (RPDD) from the FDA, along with ODD \& PRIME designation from the EMA.
  • The elsunsersen program is ongoing under a collaboration between Praxis, Ionis Pharmaceuticals, \& RogCon.
  • A pivotal EMBRAVE3 study is currently underway to further evaluate the drug's safety \& efficacy.