
Epilepsy
Our Roots Started
With Pediatric
Epilepsy
Genomics will transform the care of epilepsy. With the largest industry pipeline in epilepsy, Praxis is leading the way.
An Unmet Need
Even with treatment, patients continue to live with seizures that impact all aspects of life, family, and work. In the past 20 years, many new drugs have emerged, but the rate of uncontrolled seizures hasn’t changed.
It is time for a different approach — a precision approach.
We are looking at both
common and rare genetic epilepsies
Focal Epilepsy
Focal epilepsy is the most common epilepsy, with about 50% heritability. One-third of patients have uncontrollable seizures, and patients often have multiple comorbidities such as depression, anxiety, migraines, and heart disease.
Rare Genetic Development and Epileptic Epilepsies (DEE)
These rare genetic pediatric disorders have very diverse phenotypes that usually include severe, early-onset seizures, developmental disabilities, and increased risk of death. They are treatment resistant.

Epilepsy is more
than seizures
“Our life is complicated. We have to see so many specialists, it drains us. Our son deals with so much every day. The seizures are just one part”
Current Epilepsy Pipeline
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Current clinical stage programs
Inspired and informed by breakthroughs in epilepsy genomics, our epilepsy pipeline is the largest in the industry.
PRAX-562 is an experimental small molecule in development for SCN2A, SCN8A and additional DEEs that precisely targets the persistent sodium current. It has the potential to provide best-in-class seizure control with reduced treatment burden.
Underscoring the high unmet medical need for people living with rare genetic DEEs, PRAX-562 received Orphan Drug Designations for severe pediatric epilepsy indications from the FDA and EMA, and Rare Pediatric Disease designation from the FDA.
A Phase 2 trial is expected to start in 2022 in patients with SCN2A, SCN8A, or other DEEs or rare genetic epilepsies.
PRAX-222 is an Antisense Oligonucleotide (ASO) that targets the SNC2A gene
PRAX-222 directly targets the underlying genetic cause of sodium channel dysfunction. Designed to address both seizure reduction and improvement in the quality of life of patients.
Current Clinical Trials
Please click below for more information on our epilepsy program clinical trials
Medical Papers + Presentations
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Science Paper
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Science Paper
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Science Paper
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Our Other
CNS Franchises
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