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Dravet Syndrome Trial Exceeds Enrollment, Shows Promise
18 Aug
Summary
- Dravet syndrome trial enrollment surpassed target, with no patient discontinuations.
- Zorevunersen study evaluates drug efficacy with stable background anti-seizure medications.
- The trial assesses seizure reduction, cognition, and behavior over 52 weeks.
Stoke Therapeutics announced that its Phase III EMPEROR study, investigating zorevunersen for Dravet syndrome, has successfully enrolled 162 patients, surpassing its goal of 150. Notably, no participants have discontinued the trial to date. This sham-controlled study is evaluating zorevunersen in patients who are already receiving consistent anti-seizure medication. The primary objective is to measure seizure reduction at week 28.
Secondary assessments will focus on cognitive and behavioral outcomes at week 52. The trial's design incorporated a 15% discontinuation assumption, partly due to the sham lumbar puncture procedure in the control group. The high patient retention is attributed to effective trial execution, the drug's apparent tolerability, and the availability of an open-label extension. Stoke remains blinded to the study data and anticipates providing another update by the end of the third quarter.
The U.S. Food and Drug Administration has granted breakthrough therapy designation for zorevunersen, based on earlier data showing seizure reductions and improvements in adaptive behavior. The EMPEROR trial is powered by the receptive communication endpoint, with researchers seeking a modest treatment benefit over natural history, as Dravet syndrome patients typically do not show functional gains over time.
