Stoke Therapeutics (STOK) Guggenheim Securities Emerging Outlook: Biotech Summit 2026 summary

Key milestones and clinical progress

• Four-year open-label extension data show up to 80% seizure reduction on top of standard care, with durable effects in Dravet syndrome patients.

• Cognition and behavioral improvements observed over three years, measured by Vineland-3, with patients showing developmental gains previously unseen in Dravet.

• Phase 3 program for Dravet is well underway, with enrollment expected to complete in Q2 2026 and data readout anticipated mid-2027.

• First patient dosed in ADOA (Autosomal Dominant Optic Atrophy) trial, with proof-of-concept data expected by late 2026 or early 2027.

• Additional pipeline candidates, including SYNGAP1, are advancing toward clinical development.

Study design and regulatory strategy

• Phase 3 EMPEROR study uses a 52-week design: primary endpoint (seizure reduction) at week 28, secondary (Vineland) at week 52.

• Study powered conservatively to detect receptive communication improvements (secondary endpoint) with high statistical rigor.

• FDA denied accelerated approval based on post hoc and cross-study analyses but left the door open for further discussions with new baseline-comparison data.

• Optimal label sought to include primary, secondary, and observed long-term data, emphasizing both seizure reduction and neurodevelopmental benefits.

Market opportunity and financials

• Dravet syndrome market estimated at 36,000–40,000 patients globally, with 16,000 in North America and 6,000–8,000 immediately addressable.

• ADOA market estimated at 16,000 patients in top geographies.

• Pricing strategy aligns with other disease-modifying genetic medicines, aiming for Spinraza-like positioning.

• Cash position at end of 2025 was ~$400 million, with runway extending into 2028; Biogen funds 30% of Dravet R&D.