Andrew Tsai

Edward Marks

Jeannie Kim

Jessica Fye

Laura Chico

Marc Goodman

Pete Stavropoulos

Rudy Li

Yaron Werber

- Biogen and Stoke Therapeutics presented promising long-term follow-up data for **zorevunersen** in Dravet syndrome, demonstrating **sustained cognitive and behavioral improvements over two years** and **95% of patients showing overall clinical status improvements at three years**.  **[1623526_5d929ddd3f1740d5aa9fb89c6a2d6c38]** 
- The data also highlighted **substantial reductions in seizure frequency** and a manageable safety profile, with related adverse events occurring in **30% of Phase 1/2a participants** and **53% of open-label extension participants**.  **[1623526_5d929ddd3f1740d5aa9fb89c6a2d6c38]** 
- The most effective regimen, a **70mg loading dose followed by 45mg maintenance doses**, is currently being evaluated in the Phase 3 EMPEROR study.  **[1623526_5d929ddd3f1740d5aa9fb89c6a2d6c38]** 
- Dravet syndrome affects up to **38,000 people** across the US, Europe, and Japan, indicating a significant market need for effective treatments.  **[1623526_5d929ddd3f1740d5aa9fb89c6a2d6c38]**

Stoke Therapeutics and Biogen Present Positive Zorevunersen Data for Dravet Syndrome

- Stoke Therapeutics and Biogen announced new longer-term follow-up analyses from open-label extension studies of **zorevunersen** for Dravet syndrome, supporting its potential as a disease-modifying medicine.  **[1623526_d213a7d6f9514ee5be16da7adafd60cd_0]** 
- The data showed **continuing improvements in cognition and behavior at 2 years** and **improvements in overall clinical status at 3 years in 95% of patients** treated with zorevunersen, which contrasts with minimal change in natural history.  **[1623526_d213a7d6f9514ee5be16da7adafd60cd_0]** 
- Zorevunersen also demonstrated **substantial and durable reductions in major motor seizure frequency** and improvements in cognition and behavior through three years.  **[1623526_d213a7d6f9514ee5be16da7adafd60cd_1]**  The Phase 3 EMPEROR study is evaluating a dosing regimen of 70mg loading doses followed by 45mg maintenance doses.  **[1623526_d213a7d6f9514ee5be16da7adafd60cd_1]** 
- The drug was **generally well tolerated**, with CSF protein elevations being the most common study drug related adverse event.  **[1623526_d213a7d6f9514ee5be16da7adafd60cd_1]**  Zorevunersen has received **Orphan Drug Designation, Rare Pediatric Disease Designation, and Breakthrough Therapy Designation** for Dravet syndrome.  **[1623526_d213a7d6f9514ee5be16da7adafd60cd_2]**

- Biogen and Stoke Therapeutics announced longer-term follow-up analyses for **zorevunersen** at the 54th Child Neurology Society (CNS) Annual Meeting, supporting its potential as a disease-modifying medicine for Dravet syndrome.  **[1623526_991966a499374786acc64009699591eb_0]** 
- The analyses showed **continuing improvements in cognition and behavior at two years** and improvements in overall clinical status in **95% of patients** at three years, as reported by clinicians and caregivers.  **[1623526_991966a499374786acc64009699591eb_0]** 
- Zorevunersen demonstrated **substantial and durable reductions in major motor seizure frequency** and improvements in cognition and behavior through three years in open-label extension studies.  **[1623526_991966a499374786acc64009699591eb_1]** 
- The drug was generally well tolerated, with the most common study drug related adverse event being **CSF protein elevations**, observed in **14%** of patients in Phase 1/2a and **44%** in OLE studies.  **[1623526_991966a499374786acc64009699591eb_1]** 
- Zorevunersen has received **Orphan Drug Designation** from the FDA and EMA, and **Rare Pediatric Disease** and **Breakthrough Therapy Designation** from the FDA for Dravet syndrome.  **[1623526_991966a499374786acc64009699591eb_2]**

Stoke Therapeutics (STOK)