Stoke Therapeutics (STOK) investor relations material

Key milestones and clinical progress

• Four-year data show up to 80% seizure reduction in Dravet syndrome patients, with durable effects and additional cognitive and behavioral improvements measured by Vineland-3 scores.

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

• The pivotal EMPEROR study uses a 52-week design, with seizure reduction as the primary endpoint at 28 weeks and cognition/behavior as secondary endpoints at 52 weeks.

• No differential response observed across ages 2–18; approximately 80–90 patients have been followed in open-label extension studies.

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

Regulatory and label strategy

• Recent FDA meeting acknowledged strong data but requested more direct, non-cross-study comparisons for accelerated approval consideration.

• Company plans to present patient baseline comparisons to address FDA concerns and keep the accelerated pathway open.

• Emphasis on achieving an optimal label that includes primary, secondary, and observed long-term data to support payer discussions and market positioning.

• Observed data from four-year studies are considered critical for demonstrating true value and supporting label claims.

Commercial outlook 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.

• Pricing strategy aligns with other disease-modifying genetic medicines, with Spinraza cited as a benchmark.

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

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