Stoke Therapeutics (STOK) 44th Annual J.P. Morgan Healthcare Conference summary

Company Overview and Strategic Vision

• Focuses on upregulating protein expression to treat haplo-insufficient diseases, with lead candidate zorevunersen targeting Dravet syndrome by increasing SCN1A gene expression.

• Pipeline includes programs for Autosomal Dominant Optic Atrophy (ADOA) and SYNGAP1, with ADOA entering clinical trials and SYNGAP1 candidate selection expected soon.

• Over 6,500 genes identified as potential future targets using TANGO signatures.

• Maintains a strong financial position with $391.7M as of December 2025, supporting operations through 2028.

Clinical Development Progress and Data

• Phase III EMPEROR study for zorevunersen in Dravet syndrome began mid-2025, targeting 150 patients, with enrollment expected to complete in Q2 2026 and data readout in mid-2027.

• Over 120 patients expected to be randomized and dosed by early March, with robust recruitment driven by high unmet need.

• Long-term data show durable seizure reduction (80–85%) and neurocognitive improvements over 36–48 months, with ongoing long-term follow-up.

• Zorevunersen demonstrated significant improvements in multiple Vineland-3 subdomains compared to natural history at 18 months.

• The therapy is generally well-tolerated, with most adverse events related to CSF protein elevation and no new safety concerns identified over 800 doses and four years.

Regulatory and Commercialization Plans

• Breakthrough Designation enables rolling NDA submission, anticipated to start in H1 2027, with potential approval as early as Q4 2027.

• Collaboration with Biogen provides $165M upfront, shared development costs, up to $385M in milestones, tiered royalties, and grants Biogen commercial rights outside North America.

• Regulatory flexibility from the FDA has been offered, but the company prioritizes a comprehensive label reflecting both seizure and neurocognitive benefits.