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Praxis Precision Medicines Receives FDA Breakthrough Therapy Designation for Elsunersen for the Treatment of Seizures Associated with SCN2A Developmental and Epileptic Encephalopathy Caused by Gain of Function Variants

PRAX
  • The FDA granted Breakthrough Therapy Designation for elsunersen to treat seizures in early-seizure-onset SCN2A-DEE caused by Gain of Function variants.
  • EMBRAVE Part A trial results showed a 77% sham-adjusted reduction in monthly seizures (p=0.015) sustained for up to one year.
  • The pivotal EMBRAVE3 study is enrolling approximately 30 patients under a streamlined, single-arm registrational pathway aligned with the FDA.
  • Elsunesen now holds Breakthrough Therapy, Orphan Drug, & Rare Pediatric Disease Designations from the FDA, plus Orphan Drug & PRIME designations from the EMA.
  • In the EMBRAVE Part A trial, 71% of treated patients achieved >50% seizure reduction, while 57% achieved 28-day seizure freedom.
  • 100% of elsunersen-treated patients showed improvements in sleep, motor function, muscle tone, attention, or neuropsychomotor development, with no sham-treated patients showing similar gains.
  • The drug was well-tolerated with no drug-related serious adverse events, discontinuations, or neuroinflammation signals at doses up to 8 mg.
  • EMBRAVE3 uses a baseline-controlled design where all enrolled patients receive elsunersen for 24 weeks followed by a 24-week extension.
  • Elsunesen is an antisense oligonucleotide designed to decrease SCN2A gene expression to address the underlying genetic cause of the disease.
  • The program is ongoing under a collaboration between Praxis Precision Medicines, Ionis Pharmaceuticals, Inc., & RogCon, Inc.