Stoke Therapeutics (STOK) Goldman Sachs 47th Annual Global Healthcare Conference 2026 summary

Leadership priorities and organizational progress

• Focused on advocacy for Dravet syndrome and education about the broader benefits of their medicine beyond seizure control.

• Accelerated development, regulatory, medical affairs, manufacturing, and commercial functions to support growth.

• Significant progress in phase III clinical development and increased awareness of the medicine's potential.

Clinical data and therapeutic impact

• Zorevunersen targets the genetic root of Dravet, boosting SCN1A expression and NaV1.1, leading to seizure reduction and cognitive/behavioral improvements.

• Four-year open-label extension (OLE) data show a durable 75% reduction in seizures and statistically significant cognitive and behavioral gains across all Vineland domains.

• Phase III EMPEROR study is a 52-week trial with 150-160 patients, primary endpoint at week 28 (seizure reduction), secondary endpoints at week 52 (Vineland-3 cognition/behavior).

• Study powered on secondary endpoints with conservative assumptions; zero dropouts reported so far.

Regulatory and commercial strategy

• Rolling NDA submission planned to start Q1 2027, leveraging breakthrough designation for accelerated review.

• Anticipated launch in the U.S. as early as Q4 2027 or Q1 2028, with CMC and clinical sections on track.

• OLE data expected to be included in the label, following analogs like SPINRAZA and SKYCLARYS.

• Pricing strategy aligned with disease-modifying rare disease therapies, not limited to symptomatic Dravet treatments.