Kelly Knupp, MD, Pediatric Epileptologist at the University of Colorado, discusses the phase 3 EMPEROR clinical trial for pediatric patients with Dravet syndrome.
Dravet syndrome is the most severe of a group of conditions known as SCN1A-related seizure disorders. Symptoms include seizures which first occur in infancy that are often triggered by high temperatures. In childhood, many types of seizures may occur and they may increase in frequency. Seizures may be difficult to treat. Other symptoms include loss of motor skills, intellectual disability, speech impairment, and difficulty with movement. Most cases of Dravet syndrome occur when the SCN1A gene is not working correctly.
The EMPEROR clinical trial will be a phase 3, global, randomized, double-blind, sham-controlled study evaluating zorevunersen in pediatric patients with Dravet syndrome. Protocols have recently been finalized, with plans to initialize the trial mid-2025.
Zorevunersen is an investigational proprietary antisense oligonucleotide designed to upregulate NaV1.1 protein expression by targeting the non-mutant copy of SCN1A gene. It was recently granted Breakthrough Therapy Designation by the U.S. Food and Drug Administration.
The study will evaluate two loading doses of 70mg followed by two maintenance doses of 45mg over 52 weeks compared to sham in patients ages 2-17 years with Dravet syndrome. The primary endpoint is reduction in major motor seizure frequency and key secondary endpoints include improvements in cognition and behavior.
The EMPEROR study follows a phase 1/2a study and open-label extension, where patients experienced an 87% median reduction in seizure frequency following treatment with zorevunersen. Improvements were also observed in cognition and behavior with a generally well-tolerated safety profile.
For more information, click here.
To learn more about Dravet syndrome and other rare neurological disorders, visit https://checkrare.com/diseases/neurology-nervous-system-diseases/