Stoke Therapeutics (STOK) investor relations material

Executive summary

• Completed enrollment of 162 patients in the Phase III EMPEROR study for zorevunersen in Dravet syndrome, with no treatment discontinuations and strong patient retention; data readout expected in Q3 2027.

• Rolling NDA submission planned to begin in Q1 2027 and complete in H2 2027, with a pre-NDA meeting scheduled for H2 2026.

• Commercial launch preparations underway, targeting a potential U.S. launch in early 2028, supported by a lean infrastructure and established relationships with key treatment centers.

• Four-year OLE data show durable seizure reduction and improvements in cognition and behavior for zorevunersen.

• Expanded leadership with appointment of new Chief Scientific Officer to drive platform and pipeline expansion.

Financial highlights

• Pro-forma cash, cash equivalents, and marketable securities totaled $420M as of August 2026, including $354.3M as of June 30, 2026, and $65.7M from a subsequent ATM sale.

• Q2 2026 revenue was $9.3M, down from $13.8M in Q2 2025, due to lower collaboration and licensing revenue.

• Net loss for Q2 2026 was $61.6M ($0.93/share), with six-month net loss of $111.6M, reflecting higher R&D and SG&A expenses and lower collaboration revenue.

• Operating cash outflow for the first half of 2026 was $117.2M.

• Cash runway expected to fund operations through potential U.S. launch in early 2028, bolstered by Biogen reimbursements and milestone payments.

Outlook and guidance

• EMPEROR Phase III data readout expected in Q3 2027, with NDA submission to follow and potential FDA approval by early 2028.

• Initial safety and efficacy data from Phase 1 OSPREY study of STK-002 in ADOA anticipated in H1 2027.

• Additional studies planned for infants/toddlers and adults to expand label and access.

• Anticipates continued increases in R&D and SG&A expenses as clinical programs advance and commercial preparations accelerate.

• Cash reserves projected to fund operations through early 2028, including commercialization activities.