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Praxis Precision Medicines, Inc.

Pipeline4 assets

  • UlixacaltamidePRAX-944 / T-type calcium channel / Small molecule / Neurology
    Essential Tremor
  • RelutriginePRAX-562 / Persistent sodium current (NaV) / Small molecule / Neurology
    Developmental and Epileptic Encephalopathies (DEEs)SCN2A-DEESCN8A-DEE
  • VormatriginePRAX-628 / Sodium channels (NaV) / Small molecule / Neurology
    Focal Onset EpilepsyEpilepsy
  • ElsunersenSCN2A mRNA / Antisense oligonucleotide (ASO) / Neurology
    SCN2A Developmental and Epileptic Encephalopathy

Latest news19 items

All news

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

The Breakthrough Therapy Designation (BTD) was granted based on the positive results from the EMBRAVE Part A trial of elsunersen Elsunersen demonstrated a 77% sham-adjusted reduction in monthly seizures (p=0.015), with benefit sustained in the open-label extension for up to one year The pivotal

Praxis Precision Medicines Provides Vormatrigine Program Update

POWER1 Study in highly refractory patients with focal onset seizures did not meet its primary success measure Secondary measure, the 50% response rate, was met and seizure reduction during the second half of the study on higher dose (30 mg) was more pronounced Vormatrigine was generally

Trial Results • Phase 2/3POWER1 results Mon, Jun 1, 2026, 4:15 PM EDT

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 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

Trial Results • Phase 1/2