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Praxis Precision Medicines Announces Positive Results from EMBOLD Study for Relutrigine in SCN2A and SCN8A Developmental and Epileptic Encephalopathies
Successful interim analysis triggered early stop for efficacy Topline study results to be shared at the American Epilepsy Society Annual Meeting on December
About this update from Praxis Precision Medicines, Inc.
[{"type":"text","content":"Successful interim analysis triggered early stop for efficacy Topline study results to be shared at the American Epilepsy Society Annual Meeting on December 6, 2025 Praxis confirms an upcoming meeting with the FDA to discuss the results and NDA path BOSTON, Dec. 04, 2025 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system (CNS) disorders characterized by neuronal excitation-inhibition imbalance, today announced positive results from the registrational cohort of the EMBOLD study evaluating relutrigine for the treatment of patients with SCN2A and SCN8A developmental and epileptic encephalopathies (DEEs), following a recommendation by the Data Monitoring Committee to stop the study early for efficacy. “SCN2A and SCN8A DEEs are devastating conditions with extremely high mortality due to the debilitating seizure burden they impose on patients, and there are currently no approved treatment options. Our progress represents an important milestone towards delivering the first therapy ever designed for these children and their families” said Marcio Souza, president and chief executive officer. “We look forward to sharing the results at the American Epilepsy Society Annual Meeting.” The FDA has confirmed a meeting to review the data and discuss next steps in the coming weeks. Praxis will make a determination on the timing for filing an NDA for relutrigine after the meeting. The EMBOLD topline results will be presented at the American Epilepsy Society Annual Meeting at the Georgia World Congress Center, Poster Hall B2, 1.38 from 12:00-2:00 p.m. on Saturday, December 6, 2025. About Relutrigine (PRAX-562)Relutrigine is a first-in-class small molecule in development for the treatment of developmental and epileptic encephalopathies (DEEs) as a preferential inhibitor of persistent sodium current, shown to be a key driver of seizure symptoms in severe DEEs. Relutrigine’s mechanism of precision sodium channel (NaV) modulation is consistent with superior selectivity for disease-state NaV channel hyperexcitability. In vivo studies of relutrigine have demonstrated dose-dependent inhibition of seizures up to complete control of seizure activity in SCN2A, SCN8A and other DEE mouse models. Relutrigine has ...