EMBOLD data to serve as basis of efficacy and safety following discussion with the FDABOSTON, Dec. 11, 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 the successful completion of the planned discussion with the FDA leading to the confirmation to file a New Drug ...

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 ne ...

Core Insights - Praxis Precision Medicines has reached alignment with the FDA regarding the relutrigine program for SCN2A and SCN8A developmental and epileptic encephalopathies (DEEs) following a comprehensive Type B meeting [1][2] - The company plans to conduct an interim analysis of the EMBOLD cohort 2 pivotal trial in Q4 2025, which, if positive, could support a New Drug Application (NDA) submission in early 2026 [1][2] 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 [6] - The company utilizes genetic insights to create therapies for both rare and prevalent neurological disorders through its proprietary platforms [6] Product Development - Relutrigine (PRAX-562) is a first-in-class small molecule being developed for the treatment of DEEs, showing promise in preclinical studies and early clinical trials [5] - The EMBOLD study involves a randomized trial design to evaluate the efficacy of relutrigine in reducing seizure frequency among patients [3][4] - The interim analysis will assess approximately 70% of patients enrolled in the study, with a focus on achieving statistically significant results [4] Market Context - SCN2A and SCN8A-DEEs are ultra-rare, life-threatening pediatric epilepsies with no FDA-approved therapies currently available, affecting around 5,000 patients in the US [2] - The only existing treatment option is off-label polytherapy with antiseizure medications (ASMs), which is largely ineffective and carries significant risks [2]

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]