At Praxis, we are committed to changing the future for those diagnosed with Developmental and Epileptic Encephalopathy (DEE).
At Praxis, we are committed to changing the future for those diagnosed with Developmental and Epileptic Encephalopathy (DEE).
DEE refers to a group of epilepsies that are characterized by both seizures as well as encephalopathy, which is a term used to describe developmental delay or loss of developmental skills.
We are excited to be investigating 2 first-in-class potential DEE treatments, Relutrigine (PRAX-562) and Elsunersen (PRAX-222), both of which are purposefully developed to reduce seizures and improve quality of life for those living with a DEE.
Our Resilience program is comprised of 3 unique clinical trials, each intended to study the impact of our potential treatments in people diagnosed with DEEs. Click below to learn more about each trial.
EMBOLD Study
(Relutrigine)
for children diagnosed with early-onset SCN2A or SCN8A DEE
EMERALD Study
(Relutrigine)
for children and adults diagnosed with any DEE and related syndrome
Learn more about our 2 potential treatments for children and adults with DEEs
Elsunersen
Elsunersen is an investigational antisense oligonucleotide (ASO) administered monthly by intrathecal injection. Elsunersen has the potential to be the first disease-modifying treatment for early-onset gain-of-function SCN2A DEE, designed to selectively decrease SCN2A gene expression. By targeting the underlying genetic cause of disease, elsunersen has demonstrated potential to go beyond seizures to treat other symptoms of the disease.
What's next?
Promising Early Results
In EMBRAVE Part A, children receiving elsunersen experienced an average 77% reduction in seizures, and more than half had seizure-free periods lasting at least 28 days.
Broader Benefits Observed
100% of patients in EMBRAVE Part A receiving elsunersen showed improvements in sleep, motor function, muscle tone, attention, or neuropsychomotor development.
The EMBRAVE 3 Study Enrolling
This study is designed to further evaluate how elsunersen may reduce seizures and improve other symptoms by targeting the underlying cause of the condition.
Families who participate in EMBRAVE 3 have the opportunity to contribute directly to this research and help advance a potentially life-changing treatment for the SCN2A community.
Relutrigine
Relutrigine is an investigational medicine that can be taken orally or administered through a G/J tube. Relutrigine is designed to regulate sodium flow in brain cells by targeting overactive sodium channels that cause seizures; therefore, potentially offering better seizure control with fewer side effects. Relutrigine has been designed to maximize its effects against overactive sodium channels that are believed to cause seizure activity while minimizing the blocking of normal activity needed for healthy brain function.
What's next?
Recruitment Complete
- The EMBOLD phase 2/3 study for children diagnosed with early-onset SCN2A or SCN8A DEE
- The EMERALD phase 3 study for children and adults diagnosed with any DEE
Under FDA Review
Relutrigine is now under review by the US Food and Drug Administration (FDA) as a potential treatment for children with early-onset SCN2A and SCN8A-related epilepsies. The FDA has also granted Priority Review status, recognizing the urgent need for new treatment options and expediting the review process accordingly.
Important Milestone
If approved, relutrigine could become the first treatment specifically developed for these conditions, bringing new hope to families who currently have no targeted therapies available.
