for children with early-onset SCN2A and SCN8A Developmental and Epileptic Encephalopathies (DEEs)
DISEASE BACKGROUND AND PROGNOSIS
Developmental Epilepsies and Encephalopathies (DEEs) are fatal diseases. SCN2A and SCN8A are amongst the most severe, refractory forms of DEE. Affected children often present with relentless, refractory seizures, severe movement disorders, profound global developmental delays and significant intellectual disability, ultimately leading to early mortality. DEE families face a frightening reality resulting from the continuous threat of status epilepticus and SUDEP
Currently, there are no available treatments specifically approved for use in the treatment of SCN2A and SCN8A, as well as a majority of other DEEs.
For Praxis, SCN2A and SCN8A are only the tip of the iceberg when it comes to treating DEEs with relutrigine.
CHALLENGES & LIMITATIONS OF CURRENT STANDARD-OF-CARE
Current standard-of-care requires trial-and-error using high dose sodium channel blockers aimed at balancing an effective dose and managing significant tolerability issues. Despite best efforts, seizure control remains a difficult challenge for patients. Many times, these drugs are pushed to nearly toxic levels without ever achieving adequate seizure control.
There is an urgent need for new and targeted therapies
WHAT IS RELUTRIGINE?
Relutrigine is a first-in-class small molecule in development for the treatment of DEEs as a preferential inhibitor of persistent sodium current, shown to be a key driver of seizure symptoms in severe DEEs including SCN2A-DEE and SCN8A-DEE. Relutrigine’s mechanism of precision sodium channel (NaV) channel modulation is consistent with superior selectivity for disease-state NaV channel hyperexcitability. Thus, relutrigine may offer greater seizure control and be better tolerated compared with current standard-of-care anti-seizure medications (ASMs).
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Functional state modulator, formulated for pediatric use
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Superior selectivity for disease-state NaV channel hyperexcitability, addressing seizures where they start
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Unprecedented therapeutic window with potential for superior safety and efficacy
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Well-tolerated in pediatric patients with DEEs
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Convenient 1x daily dosing with auto titration regimen and stable PK
HOW DOES RELUTRIGINE WORK?
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Relutrigine is designed to maximize action against seizure-causing neuronal activity while sparing the normal activity needed for healthy brain functioning.
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Consistent with superior selectivity for disease-state sodium channel hyperexcitability, relutrigine preferentially inhibits persistent sodium current, with more favorable tolerability when compared with antiquated sodium channel blockers.
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Relutrigine’s unique mechanism of precision sodium modulation results in decreased channel hyperactivity while alleviating side effects seen with traditional sodium channel drugs.
EMBOLD STUDY (PRAX-562-221)
RELUTRIGINE IS SAFE AND WELL TOLERATED
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No SAEs related to relutrigine
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No AEs leading to discontinuation
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No reductions of relutrigine dosing during the trial
DEMONSTRATED EFFICACY AND EFFECTIVENESS
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Placebo-adjusted monthly motor seizure reduction of 46% during double-blind period
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Over 30% of patients achieved seizure freedom status while on relutrigine
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Meaningful gains observed in alertness, communication and seizure severity
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75% reduction in median seizure rate seen for patients in the long-term extension
To learn more about relutrigine, schedule a call with our team below
HOW YOU CAN HELP
Connect patients with resources to stay informed about the next phase of the EMBOLD study and to connect them to the resources they need. The best way for them to get involved is to have them visit www.emboldstudy.com to schedule a call with our nurse navigator.
