Stoke Therapeutics' Zorevunersen Shows Promise in Restoring Development in Dravet Syndrome Beyond Seizure Reduction
Stoke Therapeutics and Biogen are developing zorevunersen, an antisense oligonucleotide designed to boost functional NaV1.1 protein production from the non-mutated SCN1A gene copy, targeting the underlying genetic cause of Dravet syndrome1
Clinical data from phase 1/2 studies involving 81 patients aged 2-18 demonstrated seizure reductions up to 85% on top of standard care medicines, with improvements in cognition and behavior measured by Vineland-3 assessments13
In the three-year open-label extension data, patients receiving a 70mg dose saw seizure reductions between 59-91% over 20 months, with robust improvements in adaptive behavior scores indicating increased independence and safety13
The drug aims not only to reduce seizures but to restore developmental function in children who experience developmental plateauing after age 2, potentially placing them on a more neurotypical developmental pathway1
Stoke has initiated the phase 3 EMPEROR study, a sham-controlled global trial recruiting 150 patients across the U.S., U.K., and Japan, with full recruitment anticipated by Q2 2026 and data expected to support a rolling new drug application by mid-202712
Zorevunersen has received FDA breakthrough, orphan drug, and rare pediatric disease designations, with adverse events in trials being mostly mild to moderate1
The treatment represents the first potentially disease-modifying therapy targeting the genetic cause of Dravet syndrome, addressing a condition where one in five children do not reach age 18, largely due to sudden unexpected death in epilepsy (SUDEP)1
Sources:
1. https://www.fiercebiotech.com/biotech/beyond-seizures-stokes-experimental-drug-aims-restore-developmental-loss-dravet-syndrome
2. https://dravetfoundation.org/zorevunersen-stk-001-faq-spotlight-on-the-upcoming-phase-3-studies/
3. https://www.dravet.org.uk/news/life-changing-drug-to-treat-dravet-syndrome-shows-promising-results-in-trials/