Daijiworld Media Network - New Delhi
New Delhi, Mar 6: An antisense oligonucleotide treatment being co-developed by Biogen and Stoke Therapeutics has shown promising results in treating Dravet syndrome, a rare and severe form of epilepsy, raising hopes for the first disease-modifying therapy for the condition.
New clinical data for the drug zorevunersen revealed that it significantly reduced seizures in paediatric patients during two early phase I/IIa trials and ongoing extension studies.
In the initial trial, 81 children were administered up to 70 mg of zorevunersen, either as a single dose or through multiple doses over a six-month period. The patients experienced a reduction in seizure frequency ranging between 59 percent and 91 percent during the first 20 months of the extension studies, with the benefits persisting for up to three years.
Researchers noted that the results are particularly significant as current treatments are often ineffective in controlling seizures in many patients with Dravet syndrome, and no therapy currently targets the underlying cause of the disease.
According to Dr Barry Ticho, Chief Medical Officer of Stoke Therapeutics, the drug works by helping restore proper nerve-cell function by targeting the genetic cause of the disorder.
“By targeting the underlying genetic cause of the disease, zorevunersen has the potential to become the first disease-modifying medicine for the treatment of Dravet syndrome,” he said.
Lead author of the study published in the New England Journal of Medicine, Professor Helen Cross, said the findings could represent a major breakthrough in the treatment of the condition.
“These data mark a potential turning point in the treatment of Dravet syndrome. While reducing seizures remains critical, the improvements in cognition, behaviour and quality of life observed in these studies suggest we may be changing the course of the disease and the lives of patients and their families,” she said.
The drug is currently being evaluated in the ongoing phase III EMPEROR study, with results expected to be released in mid-2027.