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

Core Insights - The FDA has approved changes to the EMBRAVE3 trial design, converting it to a single-arm, baseline-controlled study for the treatment of SCN2A developmental and epileptic encephalopathy (DEE) with elsunersen [1][2] - Enrollment in the EMBRAVE3 trial is accelerating, with topline results expected in 2026 [1][2] - Elsunersen is an antisense oligonucleotide targeting SCN2A gene expression, showing promise in reducing seizures and improving symptoms in preclinical models [3][5] Group 1: EMBRAVE3 Trial Updates - The EMBRAVE3 study will now enroll 30 patients instead of the previously planned 40, with all patients receiving elsunersen from the start [5] - The primary analysis will focus on the change from baseline in countable motor seizures [5] - The ongoing EMBRAVE study (Part A) has enrolled 9 patients, with topline results expected in the first half of 2026 [1][5] Group 2: Elsunersen Overview - Elsunersen is designed to selectively decrease SCN2A gene expression, addressing the underlying cause of early-onset SCN2A DEE [3] - In vitro studies have shown a reduction in SCN2A gene expression and protein levels, while in vivo studies demonstrated significant reductions in seizures and improved survival in SCN2A mouse models [3] - Elsunersen has received Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA, as well as similar designations from the European Medicines Agency [3] Group 3: Company Background - Praxis Precision Medicines is focused on developing therapies for CNS disorders characterized by neuronal excitation-inhibition imbalance [4] - The company utilizes genetic insights to create therapies for both rare and prevalent neurological disorders through its proprietary platforms [4]