Praxis Precision Medicines has stopped its phase 2 Embold epilepsy study for relutrigine due to a successful interim analysis for efficacy of the oral drug.
The study was stopped early based on a recommendation from the Data Monitoring Committee. The FDA will be reviewing the data and discussing the next steps in a meeting with Praxis within the coming weeks, after which the company will decide when to file an NDA.
Relutrigine (PRAX-652) is a first-in-class small molecule that treats SCN2A and SCN8A developmental and epileptic encephalopathies (DEEs). It is a preferential inhibitor of persistent sodium current that uses precision sodium channel modulation. The high seizure burden of DEEs results in an extremely high mortality rate, and there are currently no approved treatments. In mouse models, relutrigine has shown a dose-dependent inhibition to even complete control of seizures.
Praxis is a clinical-stage biopharmaceutical company that uses genetic insights to create therapies for central nervous system disorders caused by neuronal excitation–inhibition imbalance. It uses its proprietary small molecule platform and antisense oligonucleotide platform along with knowledge of biological targets and circuits in the brain to develop therapies.