Praxis announces accelerated development path for relutrigine in SCN2A and SCN8A DEE patients following positive FDA feedback

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]