Praxis Precision Medicines Announces Positive Results from EMBOLD Study for Relutrigine in SCN2A and SCN8A Developmental and Epileptic Encephalopathies

Core Insights - Praxis Precision Medicines announced positive results from the EMBOLD study for relutrigine, leading to an early stop for efficacy as recommended by the Data Monitoring Committee [1][2] - The results will be presented at the American Epilepsy Society Annual Meeting on December 6, 2025, and the FDA has confirmed a meeting to discuss the data and NDA path [1][3] Company Overview - Praxis Precision Medicines is a clinical-stage biopharmaceutical company focused on developing therapies for central nervous system disorders, particularly those related to neuronal excitation-inhibition imbalance [5] - The company utilizes genetic insights to create therapies for both rare and prevalent neurological disorders through its proprietary platforms, Cerebrum™ and Solidus™ [5] Product Information - Relutrigine is a first-in-class small molecule targeting developmental and epileptic encephalopathies (DEEs) by inhibiting persistent sodium current, which is a key driver of seizure symptoms [4] - In vivo studies have shown that relutrigine can achieve dose-dependent inhibition of seizures, with some patients experiencing maintained seizure freedom [4] - The drug has received Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA, as well as Breakthrough Therapy Designation from the European Medicines Agency for SCN2A-DEE and SCN8A-DEE [4]