Stoke Therapeutics (STOK) investor relations material

Executive summary

• Four-year longitudinal data from open-label extension studies show durable seizure reduction and cognitive/behavioral improvements with zorevunersen in Dravet syndrome, supporting its disease-modifying potential; zorevunersen is generally well tolerated, with some patients treated over 5 years and no serious adverse events linked to CSF protein elevations.

• Phase III EMPEROR study enrollment is expected to complete in June 2026, with rapid recruitment in the U.S., U.K., and Japan; data readout anticipated mid-2027 and rolling NDA submission to begin Q1 2027, targeting potential U.S. approval in late 2027 or early 2028.

• U.S. launch preparations are underway, targeting a concentrated Dravet syndrome market; lean commercial infrastructure planned with less than 100 sales and marketing staff anticipated.

• Growing awareness and conviction among clinicians, payers, and investors, supported by New England Journal of Medicine publication and robust engagement with stakeholders.

• No product sales to date; revenue generated from collaboration and licensing agreements with Acadia and Biogen.

Financial highlights

• Ended Q1 2026 with $411.0 million in cash, cash equivalents, and marketable securities, including $80.7 million from ATM sales.

• Q1 2026 revenue was $6.2 million, down from $158.6 million in Q1 2025, reflecting lower recognition of upfront and milestone payments.

• Net loss for Q1 2026 was $50.0 million, or $0.79 per share, compared to net income of $112.9 million, or $1.90 per diluted share, in Q1 2025.

• Research and development expenses increased to $39.7 million in Q1 2026 from $32.7 million in Q1 2025, driven by higher personnel and program costs.

• Sales, general, and administrative expenses rose to $20.0 million in Q1 2026 from $14.7 million in Q1 2025, mainly due to commercial readiness and personnel costs.

Outlook and guidance

• Cash runway is expected to fund operations into 2028, supporting ongoing clinical development and commercial preparedness.

• Rolling NDA submission for zorevunersen planned to initiate in Q1 2027, with data readout in mid-2027 and potential U.S. launch in late 2027 or early 2028.

• Anticipates continued net losses and increased R&D and SG&A expenses as clinical programs advance and commercial preparations begin.

• No product revenue expected until regulatory approval and commercialization of product candidates.

• Enrollment of ~150 patients in the U.S., UK, and Japan expected to complete in June 2026, with European enrollment finishing in Q3 2026.