Stoke Therapeutics, Inc.

What they do

Stoke Therapeutics uses a proprietary protein upregulation platform to develop antisense oligonucleotides (ASOs) that selectively restore protein levels for diseases caused by haploinsufficiency, where loss of roughly 50% of normal protein leads to disease. Its lead candidate, zorevunersen (STK-001), is in a global Phase 3 study called EMPEROR for Dravet syndrome, a developmental and epileptic encephalopathy. A second candidate, STK-002, is in a Phase 1 open-label study called OSPREY for autosomal dominant optic atrophy (ADOA). The company has collaboration agreements with Biogen and Acadia Pharmaceuticals.

Revenue drivers

• Collaboration revenue (Biogen and Acadia) — Revenue is generated primarily from collaboration agreements, including potential milestone or royalty payments and option exercises under the Biogen International GmbH agreement and the Acadia Pharmaceuticals collaboration. Annual revenue rose from $12.4M in 2022 to $184.4M in 2025, though quarterly revenue has been uneven ($10.6M in Q3 2025, $1.4M in Q4 2025, $6.2M in Q1 2026, $9.3M in Q2 2026).
• zorevunersen (STK-001) for Dravet syndrome — The lead investigational medicine, currently in the global Phase 3 EMPEROR study; no product revenue has been recorded as it is not approved. It is the primary focus of development spending and the planned rolling U.S. NDA submission.
• STK-002 for ADOA — A clinical candidate for autosomal dominant optic atrophy, in a Phase 1 open-label study (OSPREY). It is an earlier-stage program with no revenue to date.

Recent performance

For the second quarter ended June 30, 2026, Stoke reported revenue of $9.3M, up from $6.2M in Q1 2026 and $1.4M in Q4 2025. Full-year 2025 revenue was $184.4M, compared with $36.6M in 2024, while the net loss narrowed to $6.9M in 2025 from $89.0M in 2024. Operating cash flow turned positive at $45.6M in 2025 versus negative $86.9M in 2024. As of June 30, 2026, total assets were $394.0M, total liabilities $48.4M, shareholder equity $345.5M, and cash and equivalents $110.0M.

Strategy

Stoke is focused on completing the Phase 3 EMPEROR study of zorevunersen in Dravet syndrome and preparing for a pre-NDA meeting with the FDA in the second half of 2026, with a rolling U.S. NDA submission planned to begin in Q1 2027. The company is also advancing dose escalation in the Phase 1 OSPREY study of STK-002 in ADOA and expanding early research to identify new haploinsufficient disease targets. It has strengthened leadership with the addition of Tom McCauley as Chief Scientific Officer. The company states it is enhancing commercial capabilities and expects its approximately $420M in cash, cash equivalents, and marketable securities to fund operations through a potential U.S. commercialization of zorevunersen in early 2028.

Risks

• Clinical and regulatory risk — The company's ability to develop, obtain regulatory approval for, and commercialize zorevunersen, STK-002, and future product candidates is uncertain and depends on clinical trial success and regulatory review.
• Profitability and funding risk — Stoke has a limited operating history and has not been profitable, with annual net losses through 2024 and only a narrow net loss of $6.9M in 2025; it may need to procure sufficient additional funding.
• Collaboration dependence — The success of collaborations with Acadia Pharmaceuticals and Biogen, and the receipt of potential milestone or royalty payments or exercise of the Biogen option, are outside the company's full control.
• Early trial predictiveness and macroeconomic risk — Success in early preclinical studies or clinical trials may not be indicative of results in later studies, and inflation, interest rates, foreign currency exchange rates, tariffs, instability in the global banking system, geopolitical conflict, and macroeconomic conditions could affect operations.

Outlook

Management expects a Phase 3 EMPEROR data readout in Q3 2027, following completion of enrollment of 162 patients in June 2026. A pre-NDA meeting with the FDA is scheduled for the second half of 2026, with a rolling U.S. NDA submission planned to initiate in Q1 2027 and complete in the second half of 2027. Initial safety and efficacy data from the Phase 1 OSPREY study of STK-002 in ADOA are anticipated in the first half of 2027. The company states its approximately $420M in cash, cash equivalents, and marketable securities should fund operations through a potential U.S. commercialization of zorevunersen in early 2028.