Core Insights - The U.S. FDA has granted Breakthrough Therapy Designation (BTD) for relutrigine, a sodium channel functional state modulator, aimed at treating pediatric patients with SCN2A and SCN8A developmental and epileptic encephalopathies (DEEs) [1][2] - The EMBOLD cohort 2 pivotal trial is expected to deliver topline results in the first half of 2026, with a New Drug Application (NDA) filing to follow [1][3] - Praxis has initiated the EMERALD study to evaluate relutrigine in a broader DEE patient population [1][3] Clinical Trial Results - The EMBOLD cohort 1 study included a severely affected DEE population, with patients averaging three failed treatments prior to enrollment [2] - Results from the open-label extension of the study showed approximately 90% reduction in seizures and an average of 67 days without seizures compared to 3 days in the baseline period [2][4] - During the double-blind period, a placebo-adjusted monthly motor seizure reduction of 46% was observed, with over 30% of patients achieving seizure freedom while on relutrigine [2][4] Drug Mechanism and Designation - Relutrigine is a first-in-class small molecule that preferentially inhibits persistent sodium current, a key driver of seizure symptoms in severe DEEs [4] - The drug has received Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation from the FDA for SCN2A-DEE and SCN8A-DEE, in addition to the BTD [4][5] Company Overview - Praxis Precision Medicines is focused on developing therapies for CNS disorders characterized by neuronal excitation-inhibition imbalance, utilizing genetic insights [6] - The company has a diversified CNS portfolio, including multiple programs across epilepsy and movement disorders, with four clinical-stage product candidates [6]

Company Overview - Praxis Precision Medicines, Inc. is a clinical-stage biopharmaceutical company focused on developing therapies for central nervous system (CNS) disorders characterized by neuronal excitation-inhibition imbalance [3] - The company utilizes genetic insights to create therapies for both rare and more prevalent neurological disorders through its proprietary platforms, Cerebrum™ and Solidus™ [3] - Praxis has a diversified CNS portfolio that includes multiple programs across epilepsy and movement disorders, featuring four clinical-stage product candidates [3] Upcoming Event - Praxis will host a virtual investor event on May 2, 2025, from 10:00 a.m. to 11:30 a.m. EDT, focusing on its clinical programs in developmental and epileptic encephalopathies (DEEs) [1][2] - The event will be led by the Praxis management team, and a replay will be available for 90 days on the company's website [2] Clinical Studies - The company is conducting several studies, including: - The EMBOLD study evaluating relutrigine for SCN2A and SCN8A DEEs [4] - The EMERALD study evaluating relutrigine for broader DEEs [4] - The EMBRAVE3 study evaluating elsunersen for SCN2A gain-of-function DEE [4]