Praxis、FDA就脑病资产elsunersen的关键试验变更达成一致

2025-12-10 10:51

Praxis Precision Medicines (PRAX) will adjust its EMBRAVE3 registrational trial of elsunersen in early-onset developmental and epileptic encephalopathy into a single-arm study following discussions with the US FDA.
All patients in the study will receive elsunersen for 24 weeks, followed by an open-label extension.
Previously, the trial was structured as double-blind and placebo-controlled enrolling 40 patients. That number has also been reduced to 30.
The primary endpoint is change from baseline in countable motor seizures.
Elsunersen is known as an antisense oligonucleotide designed selectively to decrease SCN2A gene expression to target the underlying cause of early-seizure-onset SCN2A developmental and epileptic encephalopathy.

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