Biogen And Stoke Therapeutics Present Promising Long-Term Data For Zorevunersen In Dravet Syndrome

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(RTTNews) - Biogen Inc. (BIIB) and Stoke Therapeutics Inc. (STOK) unveiled new long-term follow-up data from ongoing open-label extension (OLE) studies of zorevunersen, reinforcing its potential as a disease-modifying treatment for Dravet syndrome. The findings were presented at the 54th Annual Meeting of the Child Neurology Society (CNS).

Two-year data from a cognitive and behavioral analysis revealed sustained improvements in patients treated with zorevunersen, contrasting sharply with minimal changes observed in a natural history study of patients receiving standard care. Additionally, three-year results from Clinical and Caregiver Global Impression of Change (CGI-C and CaGI-C) scales showed that 95% of patients experienced improvements in overall clinical status, as reported independently by caregivers and clinicians. These findings complement previously presented caregiver-reported quality of life improvements using the EQ-VAS scale.

The data were part of a broader poster presentation covering Phase 1/2a and ongoing OLE studies. Safety and tolerability remained the primary endpoints, while secondary endpoints included major motor seizure frequency, cognition, and behavior. Through three years, patients demonstrated substantial and durable reductions in seizure frequency and marked improvements in cognitive and behavioral measures, even while continuing standard anti-seizure medications.

The most significant benefits were observed in patients who received a loading dose of 70mg followed by maintenance doses of 45mg in the Phase 1/2a study. This dosing regimen is currently being evaluated in the Phase 3 EMPEROR study, which aims to further validate zorevunersen's therapeutic potential in Dravet syndrome.

STOK closed Thursday regular trading at $29.90 down $0.60 or $1.97%.

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