Stoke Therapeutics (STOK) Q1 2026 earnings summary

Executive summary

• Four-year longitudinal data from open-label extension (OLE) studies show durable seizure reduction and cognitive/behavioral improvements with zorevunersen in Dravet syndrome patients, supporting its disease-modifying potential; data published in The New England Journal of Medicine and presented at major conferences.

• Phase III EMPEROR study enrollment is expected to complete in June 2026, with rapid recruitment in the U.S., U.K., and Japan, and data readout anticipated in mid-2027 to support a rolling NDA submission in early 2027.

• Zorevunersen continues to be generally well tolerated, with some patients treated for over 5 years and no serious adverse events linked to CSF protein elevations.

• U.S. launch preparations are underway, targeting a concentrated market of Dravet syndrome patients, with expansion of medical affairs and commercial planning.

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

Financial highlights

• Ended Q1 2026 with $411 million in cash, cash equivalents, and marketable securities, including $80.7 million raised through ATM equity 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 potential U.S. approval in late 2027 or early 2028.

• Expects to include five-year OLE data in regulatory filings and product labeling.

• Lean commercial infrastructure planned due to concentrated patient and provider base.

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