Stoke Therapeutics — Third Quarter 2026 Earnings Summary

Financial Performance:

• Revenue for the three months ended September 30, 2026, was $11.3 million, an increase from $10.6 million in the same period in 2025.
• Revenue for the nine months ended September 30, 2026, was $26.8 million, a decrease from $183.0 million in the same period in 2025, primarily driven by the recognition of a $150.8 million Biogen IP license performance obligation in 2025.
• Net loss for the three months ended September 30, 2026, was $73.6 million ($1.08 per share), compared to a net loss of $38.3 million ($0.65 per share) in 2025.
• Net loss for the nine months ended September 30, 2026, was $185.3 million ($2.81 per share), compared to a net income of $51.0 million ($0.85 per diluted share) in 2025.
• Research and development expenses for the three months ended September 30, 2026, were $59.8 million, up from $37.7 million in 2025.
• Research and development expenses for the nine months ended September 30, 2026, were $149.0 million, up from $96.2 million in 2025.
• Sales, general and administrative expenses for the three months ended September 30, 2026, were $28.7 million, up from $16.0 million in 2025.
• Sales, general and administrative expenses for the nine months ended September 30, 2026, were $74.0 million, up from $45.9 million in 2025.
• Cash, cash equivalents, and marketable securities totaled $358.8 million as of September 30, 2026.
• Deferred revenue was $6.4 million (current) and $4.4 million (long-term) as of September 30, 2026, compared to $11.9 million (current) and $7.0 million (long-term) as of December 31, 2025.

Guidance and Future Outlook:

• Data readout for the Phase 3 EMPEROR study of zorevunersen is anticipated in the third quarter of 2027.
• A U.S. New Drug Application (NDA) submission for zorevunersen is planned for the second half of 2027.
• Initial safety and efficacy data for STK-002 in the Phase 1 OSPREY study are anticipated in the first half of 2027.
• The current cash position is expected to fund operations into early 2028.
• Dosing for the fourth cohort of the OSPREY study is expected to begin in early 2027.
• A potential U.S. commercial launch for zorevunersen is targeted for early 2028.

Business Segments and Product Lines:

• Zorevunersen (Dravet Syndrome): Global enrollment for the Phase 3 EMPEROR study is complete with 162 patients in the primary analysis population (US, UK, Japan) and 34 additional patients in Europe and 5 in China. Approximately 110 patients have completed the Week 28 primary endpoint visit, and approximately 20 have completed the Week 52 treatment period. No patients have discontinued treatment.
• STK-002 (ADOA): Dosing is complete for the first two cohorts of the Phase 1 OSPREY study, with the third cohort dosing underway. The fourth cohort is expected to follow in early 2027.
• SYNGAP1-related disorders: Lead optimization is underway to identify a development candidate for this indication.
• Collaborations: Revenue is generated from contractual obligations with Acadia and Biogen. Stoke retains exclusive rights for zorevunersen in the US, Canada, and Mexico, while Biogen holds rest-of-world rights.

Market and Competitive Landscape:

• There are no approved disease-modifying therapies for Dravet syndrome.
• There are no approved treatments for Autosomal Dominant Optic Atrophy (ADOA).
• Zorevunersen has received orphan drug designation from the FDA and EMA, rare pediatric disease designation, and Breakthrough Therapy Designation from the FDA and China's Center for Drug Evaluation.
• STK-002 has received orphan drug designation from the FDA.
• The company presented long-term clinical data supporting the disease-modifying potential of zorevunersen at the 16th European Epilepsy Congress and the 55th Child Neurology Society Annual Meeting.

Risks and Challenges:

• Forward-looking statements involve risks including the ability to advance, obtain regulatory approval for, and commercialize product candidates.
• Risks include potential breach or termination of collaboration agreements with partners like Biogen.
• Clinical trial results may not be replicated in subsequent trials or predictive of later-stage success.
• The company faces risks related to intellectual property protection and funding development activities into 2028.
• Specific risks include the possibility that zorevunersen or STK-002 may not demonstrate safety or efficacy at indicated dosing levels.

Management Commentary and Tone:

• CEO Ian F. Smith stated the Phase 3 EMPEROR study is progressing well with all enrolled patients continuing into the open-label extension period.
• Management expressed confidence that NDA planning is on track following a successful FDA meeting to align on the submission.
• The company plans to use a multicomponent assessment of four Vineland-3 subdomains to evaluate cognition and behavior changes for the NDA.
• Smith noted the company is "building Stoke for the future" with the team and capabilities to realize the potential of its pipeline.

Other Key Points:

• The company hosted a webcast and conference call for analysts and investors on October 29, 2026.
• Stoke and Biogen presented new exploratory sub-analysis data showing substantial reductions in severe seizure types, a leading risk factor for SUDEP.
• The Phase 3 EMPEROR study is a global, double-blind, sham-controlled study evaluating zorevunersen in children ages 2 to <18 with Dravet syndrome.
• The Phase 1 OSPREY study for STK-002 is an open-label, dose-escalating study in patients ages 6 to 55 with ADOA.
• The company has 64,813,537 shares issued and outstanding as of September 30, 2026.