Praxis announces
Praxis announces accelerated development path for relutrigine in SCN2A and SCN8A DEE patients following positive FDA feedback
BOSTON -- Praxis Precision Medicines, Inc., 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 that, following a comprehensive Type B meeting and recent meeting minutes and written advice on the protocol and SAP, it has reached alignment with the FDA on several aspects of the relutrigine program in SCN2A and SCN8A developmental and epileptic encephalopathies (DEEs), including the use of the ongoing EMBOLD study to serve as the basis of substantial evidence of effectiveness for the NDA submission.
The FDA agreed that Praxis’ proposed interim analysis, if positive, may serve as the basis of the NDA submission in early 2026. The interim analysis is planned to be conducted in the fourth quarter of 2025.
"We are appreciative of the recent collaborative discussion with the FDA about opportunities to bring relutrigine to SCN2A and 8A patients sooner, which came as a result of our breakthrough therapy designation (BTD),” commented Steven Petrou, chief scientific officer. "We reached alignment on key elements of the NDA with FDA, and upon a successful result from the upcoming interim analysis, we would quickly submit the NDA for relutrigine. This could be our second NDA under review, pending acceptance of the ulixacaltamide NDA.”
SCN2A- and SCN8A-DEEs are ultra-rare, life-threatening pediatric epilepsies characterized by early-onset, pharmacoresistant seizures, rapid neurodevelopmental decline, and high premature mortality. There are no FDA approved therapies. Off-label polytherapy with ASMs remains the only therapeutic option but is largely ineffective, carries substantial toxicity risks, and imposes a significant burden for the approximately 5,000 patients living with the condition in the US.
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 been generally well-tolerated in three Phase 1 studies and has demonstrated biomarker changes indicative of NaV channel modulation.
Data from cohort 1 of the Phase 2 EMBOLD study demonstrated a well-tolerated, robust, short- and long-term improvement in motor seizures in a heavily pre-treated population, alongside maintained seizure freedom in some patients with SCN2A- and SCN8A-DEE. Relutrigine has received Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation from the FDA for the treatment of SCN2A-DEE, SCN8A-DEE and Dravet syndrome; as well as Breakthrough Therapy Designation (BTD), and ODD from the European Medicines Agency for the treatment of SCN2A-DEE and SCN8A-DEE.
