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Praxis Precision Medicines Announces Alignment with FDA on Simplified and Accelerated Registrational Pathway for Elsunersen in Early Onset SCN2A Developmental and Epileptic Encephalopathy

PRAX
  • Praxis Precision Medicines completed a Type C meeting with the FDA regarding the EMBRAVE3 trial for elsunersen in SCN2A-DEE patients.
  • The FDA agreed to convert EMBRAVE3 from a double-blind, sham-controlled study to a single-arm, baseline-controlled design.
  • Under the new design, all 30 enrolled patients will receive elsunersen for 24 weeks followed by an open-label extension, reducing the original target from 40 patients.
  • The primary analysis will measure the change from baseline in countable motor seizures.
  • Enrollment in EMBRAVE3 is accelerating, with top-line results expected in 2026.
  • Top-line results from the ongoing EMBRAVE study (Part A, n=9) are also expected in the first half of 2026.
  • Elsuneersen is an antisense oligonucleotide designed to reduce SCN2A gene expression to treat gain-of-function SCN2A mutations.
  • Elsuneersen has received Orphan Drug Designation, Rare Pediatric Disease Designation, \& PRIME designation from regulatory bodies.