Stoke Therapeutics (STOK) investor relations material

Company overview and pipeline

• Advancing a phase III medicine for Dravet syndrome, with full enrollment and data readout expected within 12 months.

• Pipeline includes a medicine for ADOA, progressing through phase I/II with dose escalation and anticipated entry into higher-dose cohorts.

• Financial position is strong, with over $400 million in cash as of June 30, sufficient to fund operations through early 2028.

Clinical data and patient impact

• Zorevunersen targets the genetic root cause of Dravet syndrome, aiming to upregulate NaV1.1 protein.

• Open label extension (OLE) data show sustained 75%± seizure reduction and continuous functional gains over four years.

• Patients on standard anti-seizure meds still experience significant seizures, but zorevunersen further reduces both frequency and severity.

• Improvements in communication and motor skills are measured by Vineland-3, with gains increasing over time.

Phase III EMPEROR study design and endpoints

• EMPEROR study enrolled 150 patients, using a sham-controlled, 52-week design with four doses.

• Primary endpoint is seizure reduction at week 28, measured over weeks 25–28 and compared to baseline and sham.

• Secondary endpoints focus on Vineland-3 domains, with plans to shift from hierarchical to composite scoring for better clinical relevance.

• Study is powered to detect a 2–3 point difference in Vineland scores, with 128 evaluable patients needed and low dropout rates.