Elsunersen (PRAX-222)
for children with early-onset SCN2A Developmental and Epileptic Encephalopathies (DEEs)
SCN2A DISEASE BACKGROUND AND PROGNOSIS
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Early onset SCN2A developmental and epileptic encephalopathy (SCN2A-DEE) is a rare, severe, and fatal pediatric disorder caused by gain-of-function (GoF) variants in the SCN2A gene encoding the voltage-gated sodium channel NaV1. 2.. Children with SCN2A-DEE present with relentless seizures, severe movement disorders, profound global developmental delays and significant intellectual disability, ultimately leading to early mortality.  
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Currently, there are no available treatments specifically designed and approved for use in the treatment of SCN2A-DEE. 
CHALLENGES/LIMITATIONS OF CURRENT STANDARD-OF-CARE
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Current standard-of-care requires a trial-and-error approach using sodium channel blockers aimed at balancing an effective dose and management of significant tolerability challenges. Despite best efforts, seizure control remains a significant burden for patients.   
 
There is an urgent need for new and targeted therapies. 
WHAT IS ELSUNERSEN (PRAX-222)?
Elsunersen is an antisense oligonucleotide (ASO) designed to selectively decrease SCN2A gene expression, directly targeting the underlying cause of early onset SCN2A-DEE to treat seizures and other symptoms in patients with GoF SCN2A mutations. 
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Elsunersen has received Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPD) from the FDA, and ODD and PRIME designations from the European Medicines Agency (EMA) for the treatment of SCN2A-DEE.
WHAT IS AN ASO?
An ASO is a short, synthetic strand of DNA or RNA designed to bind specifically to a target RNA molecule, typically to alter its function or expression.
HOW DOES ELSUNERSEN WORK?
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Elsunersen is designed for SCN2A GoF mutations, with a modified core allowing it to bind to target RNA
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Induces RNA degradation via RNase H activation 
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Resulting in decreased number and hyperactivity of sodium channels 
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Monthly administration through intrathecal (IT) injections 
EMBRAVE STUDY (PRAX-222-111) PART 1 DATA
SAFETY
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Emerging data shows that elsunersen is safe and well-tolerated
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No AEs related to elsunersen
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No AEs leading to discontinuation
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UNPRECEDENTED EFFICACY AND EFFECTIVENESS
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Rapid onset and sustained treatment effect in children with early onset SCN2A-DEE
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Clinically meaningful seizure control when added to standard-of-care vs. standard-of-care alone 
To learn more about elsunersen, schedule a call with our team below
"When I started practicing, we didn't even have the genetic test to make a diagnosis for SCN2A epilepsies. Now we can tell you exactly what's causing it down to the gene with a tailored precise therapy to target it. It's amazing to think of that progress compared to where we were not long ago in how we cared for these children."
James Wheless, MD
Principal Investigator EMBRAVE (PRAX-222-111) Part 1
Professor and Chief of Pediatric Neurology,
University of Tennessee Health Science Center
HOW YOU CAN HELP
Connect patients with resources to stay informed about the next phase of the EMBRAVE study. SCN2A variants from prospective patients must be characterized to confirm future eligibility criteria. Praxis is utilizing a characterization method which can take up to 12 weeks. The best way for families to get involved is to visit www.embravestudy.com to schedule a call with our nurse navigator.
