Praxis Precision Medicines to Showcase Epilepsy Pipeline at AES 2025 with Full RADIANT Study Results
核心洞察
Praxis Precision Medicines will present comprehensive preclinical and clinical data across its precision epilepsy (搜索) pipeline at the American Epilepsy Society (搜索) Annual Meeting from December 5-9, 2025.
The company will unveil full results from the RADIANT study showing vormatrigine rapidly reduces seizures in adults with treatment-resistant epilepsy (搜索).
Multiple presentations will highlight sustained seizure reduction data from relutrigine's EMBOLD open-label extension study and preclinical findings demonstrating greater potency than fenfluramine in Dravet syndrome (搜索).
Praxis Precision Medicines will present comprehensive data across its precision epilepsy (搜索) pipeline at the American Epilepsy Society (搜索) Annual Meeting, scheduled for December 5-9, 2025, in Atlanta, Georgia. The clinical-stage biopharmaceutical company will showcase full results from its RADIANT study demonstrating that vormatrigine rapidly reduces seizures in adults with treatment-resistant epilepsy (搜索).
"The AES meeting is a cornerstone for the epilepsy (搜索) community, bringing together science, clinical innovation, and patient advocacy," said Steven Petrou, chief scientific officer and co-founder of Praxis. "We're excited to continue showcasing the strength of our precision epilepsy pipeline and the potential to meaningfully impact the lives of people living with epilepsy."
Conference Presentations Highlight Pipeline Progress
The company will deliver multiple poster and late-breaking presentations, including a dedicated late-breaker session on Saturday, December 6, featuring full results from the RADIANT study of vormatrigine. Additional presentations will showcase sustained seizure reduction data from relutrigine's EMBOLD open-label extension study and preclinical findings demonstrating the drug's anticonvulsant potential in Dravet syndrome (搜索).
Key presentations include preclinical data showing relutrigine exhibits greater potency than fenfluramine in Dravet syndrome (搜索) models, and findings on vormatrigine's favorable drug-drug interaction profile supporting broad combination use with antiseizure medications. The company will also present data on complementary antisense oligonucleotide treatment for early-onset SCN2A (搜索) developmental and epileptic encephalopathy, including emergency use cases in a preterm infant with refractory status epilepticus (搜索).
Vormatrigine Shows Promise in Treatment-Resistant Epilepsy
Vormatrigine represents a next-generation, functionally selective small molecule targeting hyperexcitable sodium channels (搜索) in the brain, developed as a once-daily oral treatment for adult focal onset seizures (搜索) and generalized epilepsy (搜索). Preclinical data demonstrates the compound's differentiation from standard of care, with potential to be best-in-class for focal epilepsy (搜索).
In vitro studies show vormatrigine demonstrates superior selectivity for disease-state sodium channel hyperexcitability. In vivo studies have demonstrated unprecedented potency in the maximal electroshock seizure model, a highly predictive translational model for efficacy in focal epilepsy (搜索). Data from the first cohort of patients in the RADIANT study demonstrated robust seizure reduction with a generally safe and well-tolerated profile.
Relutrigine Advances in Rare Epilepsy Syndromes
Relutrigine, a first-in-class small molecule in development for developmental and epileptic encephalopathies (搜索), functions as a preferential inhibitor of persistent sodium current, identified as a key driver of seizure symptoms in severe developmental and epileptic encephalopathies. The drug's precision sodium channel modulation mechanism demonstrates superior selectivity for disease-state sodium channel hyperexcitability.
In vivo studies of relutrigine have shown dose-dependent inhibition of seizures up to complete control of seizure activity in SCN2A (搜索), SCN8A (搜索) and other developmental and epileptic encephalopathy mouse models. The compound has been generally well-tolerated in three Phase 1 studies and has demonstrated biomarker changes indicative of sodium channel modulation.
Data from cohort 1 of the Phase 2 EMBOLD study demonstrated 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 (搜索)- and SCN8A (搜索)-developmental and epileptic encephalopathy. Relutrigine has received Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA for treatment of SCN2A-developmental and epileptic encephalopathy, SCN8A-developmental and epileptic encephalopathy and Dravet syndrome (搜索), as well as Breakthrough Therapy Designation and Orphan Drug Designation from the European Medicines Agency (搜索).
Antisense Approach Targets Genetic Root Cause
Elsunersen, an antisense oligonucleotide designed to selectively decrease SCN2A (搜索) gene expression, directly targets the underlying cause of early-seizure-onset SCN2A-developmental and epileptic encephalopathy in patients with gain-of-function SCN2A mutations. In vitro studies demonstrate reduction in both SCN2A gene expression and protein levels.
In vivo studies show elsunersen produces significant, dose-dependent reduction in seizures, improvement in behavioral and locomotor activity, and increased survival in SCN2A (搜索) mouse models, with potential to become the first disease-modifying treatment for SCN2A-developmental and epileptic encephalopathy. The program has received Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA, and Orphan Drug Designation and PRIME designations from the European Medicines Agency (搜索).
Precision Medicine Approach to CNS Disorders
Praxis Precision Medicines translates insights from genetic epilepsies into therapies for central nervous system disorders characterized by neuronal excitation-inhibition imbalance. The company applies genetic insights through its proprietary small molecule platform, Cerebrum, and antisense oligonucleotide platform, Solidus, leveraging understanding of shared biological targets and circuits in the brain.
The company has established a diversified, multimodal central nervous system portfolio including multiple programs across movement disorders and epilepsy (搜索), with four clinical-stage product candidates. The presentations at AES 2025 reflect the continued translation of the company's science into clinical progress and reinforce its commitment to advancing transformative therapies for people living with epilepsy.