Stoke Therapeutics announced that the New England Journal of Medicine has published the first data demonstrating disease‑modifying effects of its lead candidate zorevunersen in patients with Dravet syndrome.
The NEJM paper reports that 81 patients treated with zorevunersen on top of standard anti‑seizure medicines experienced substantial and durable reductions in seizure frequency, along with improvements in cognition and behavior. The benefits began during the Phase 1/2a treatment period and persisted through a three‑year open‑label extension, indicating a lasting therapeutic impact.
Zorevunersen is an antisense oligonucleotide that upregulates NaV1.1 protein production from the unaffected SCN1A allele, directly addressing the haploinsufficiency that drives Dravet syndrome. By restoring the missing protein, the drug targets the underlying genetic cause rather than merely controlling seizures.
The publication strengthens the case for the ongoing global Phase 3 EMPEROR trial and supports a potential New Drug Application submission to the FDA in mid‑2027. Stoke has received Breakthrough Therapy Designation from the FDA and Orphan Drug Designation from the EMA, and it is collaborating with Biogen on development and commercialization.
"These data mark a potential turning point in the treatment of Dravet syndrome," said Helen Cross, corresponding author of the NEJM paper. "This publication in NEJM demonstrates an appreciation of the severity of Dravet syndrome and the potential of this novel approach to transform the way the disease is treated," added Katherine Dawson. "By targeting the underlying genetic cause of the disease, zorevunersen has the potential to be the first disease‑modifying medicine for the treatment of Dravet syndrome. We look forward to the results of our Phase 3 EMPEROR study expected in mid‑2027," said Barry Ticho, Chief Medical Officer. "The rate of enrollment in the Phase 3 EMPEROR study is highly encouraging and supports the significant need for a disease‑modifying treatment for Dravet syndrome. The accelerated timing for completion of enrollment of 150 patients, in addition to Breakthrough Therapy Designation, positions us to initiate a rolling NDA submission in the first half of 2027, resulting in the potential to deliver zorevunersen to patients sooner than originally expected," said Ian F. Smith, CEO.
The announcement was well received by investors, reflecting confidence in the disease‑modifying potential of zorevunersen and the strategic trajectory of Stoke Therapeutics.
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