Stoke Therapeutics, Inc. (STOK)

Key figures

The last twelve months reported, with the latest price and balance sheet.

Revenue, last twelve months

$27.6M

Revenue growth

-86.2%

Net income, last twelve months

-$207.9M

Earnings per share

-$3.34

Operating margin

-809.7%

Net margin

-753.6%

Free cash flow

-$180.7M

Return on equity

-60.2%

Market value

$1.6B

Cash

$110.0M

Debt

$0

Debt to equity

0.00

Shares outstanding

62.4M

Financial statements

Revenue, profit and cash flow by fiscal year, each year linked to its annual report.

By quarter

What the company said with its results

From the second quarter 2026 results, filed 3 August 2026.

• Net loss widened to $61.6M. Net loss for the quarter was $61.6 million, or $0.93 per share, against $23.5 million, or $0.40 per share, a year earlier. Stock-based compensation was $10.8 million.
• Revenue fell on Acadia and Biogen. Revenue was $9.3 million, down from $13.8 million a year earlier. The company said it comes from satisfying obligations under its Acadia and Biogen collaboration and licensing agreements.
• Six-month revenue fell on 2025 license. Six-month revenue was $15.6 million against $172.4 million, mainly because $150.8 million of Biogen IP license revenue was recognized in the first half of 2025.
• Six-month net loss versus prior-year income. Six-month net loss was $111.6 million, or $1.73 per share, compared with net income of $89.4 million, or $1.50 per diluted share, a year earlier.
• EMPEROR enrollment completed at 162. Enrollment of 162 patients in the U.S., UK and Japan was completed in June. The company said this cohort is the planned primary analysis population for the U.S. NDA.
• No EMPEROR treatment discontinuations. As of July 31, 2026, no patients had discontinued treatment in the study. About 145 patients were through Week 8 and about 60 were through Week 28, when the primary endpoint is measured.
• Phase 3 readout expected Q3 2027. Data from the 162-patient cohort are anticipated in the third quarter of 2027 and are expected to be the final clinical data for the planned rolling NDA submission.
• Patients approaching Week 52. About 80 patients were through Week 24 with one dose remaining. The first patients are expected to reach Week 52 in August 2026.
• Europe and China cohorts ongoing. About 30 more patients are being enrolled in Germany, France, Spain and Italy with a needle-prick sham. China site activation and screening are underway, with enrollment completing in the second half of 2026.
• Pre-NDA meeting set for second half 2026. The company will hold a pre-NDA meeting with the FDA in the second half of 2026 to align on data package content, timing and the statistical analysis plan.
• Rolling NDA start planned Q1 2027. The company plans to start the rolling NDA in the first quarter of 2027. The prior release used the same timing for the rolling U.S. NDA submission.
• China Breakthrough designation granted. China's Center for Drug Evaluation granted zorevunersen Breakthrough Therapy Designation. Site activation and screening in China are underway.
• OSPREY first cohort dosed, no serious events. The first cohort of three patients in the Phase 1 OSPREY study of STK-002 is fully dosed. No serious or severe safety events have been observed to date, and all eight planned sites are active.
• OSPREY data expected first half 2027. Dosing of the second cohort begins in August 2026. The third and fourth cohorts follow, subject to safety assessments, with safety and efficacy data expected in the first half of 2027.

Company events

The newest 8-K filings of 85 held.

Every filing

The newest filings with the SEC of 198 held.

Insider trades

Form 4 trades by officers, directors and 10% owners, last 12 months.

Planned insider sales

Form 144 notices, filed when the sell order goes to the broker.

Large holders

Schedule 13D and 13G filings by holders of more than 5% of the voting shares.

Short interest

Shares sold short and not yet bought back, on each FINRA settlement date.

Funds that hold it

From each fund's latest Form 13F.

226 funds held it at 30 Jun 2026: 101 added and 57 trimmed in the quarter. The largest:

Peers

Companies in the same industry.

• Eli Lilly & Co. (LLY): revenue $79.7B, market value $1.21T
• Johnson & Johnson (JNJ): revenue $99.3B, market value $652.9B
• AbbVie Inc. (ABBV): revenue $64.4B, market value $506.1B
• Merck & Co., Inc. (MRK): revenue $66.6B, market value $348.1B
• Novartis AG (NVS): revenue $54.5B, market value $297.8B
• AstraZeneca PLC (AZN): revenue $58.7B, market value $263.1B
• Abbott Laboratories (ABT): revenue $46.6B, market value $177.7B
• Novo Nordisk A/S (NVO): revenue $46.8B, market value $171.0B
• Pfizer Inc. (PFE): revenue $63.7B, market value $168.0B
• Vertex Pharmaceuticals Inc. (VRTX): revenue $12.6B, market value $134.2B

What the company does

Stoke Therapeutics describes itself as a late-stage clinical company that develops RNA-based medicines to raise the production of proteins that are missing in severe genetic diseases. It uses a proprietary approach called TANGO (Targeted Augmentation of Nuclear Gene Output) to design antisense oligonucleotides, known as ASOs. Its lead medicine, zorevunersen (STK-001), is in a global Phase 3 study for Dravet syndrome, a severe genetic epilepsy. A second candidate, STK-002, is in a Phase 1 study for autosomal dominant optic atrophy (ADOA). The company was founded in June 2014, is based in Bedford, Massachusetts, and had 170 employees as of 31 December 2025.

How the TANGO approach works

The company focuses on haploinsufficiencies, diseases in which one copy of a gene is mutated and the body makes only about 50% of the normal amount of a protein. TANGO ASOs bind to pre-mRNA and change how it is spliced. The aim is to prevent non-productive splicing events that send messenger RNA to be degraded, so the healthy gene copy produces more protein. The filing says the ASOs do not alter DNA and work whatever the specific mutation is, so one drug can address the full range of loss-of-function mutations in a gene.

To find new targets, the company uses its own bioinformatics tools and whole-transcriptome RNA sequencing.

Zorevunersen and Dravet syndrome

Dravet syndrome is mostly caused by mutations in one copy of the SCN1A gene, which leaves too little Nav1.1 protein in certain brain neurons. The company estimates that one in 15,600 babies are born with it and that approximately 15,700 people live with it in the U.S. Approved anti-seizure medicines for Dravet syndrome include Epidiolex, Fintepla and Diacomit. The filing says none of them treat the underlying cause or the developmental and cognitive effects of the disease.

The EMPEROR study is a global, randomized, double-blind, sham-controlled Phase 3 trial at approximately 60 sites across the UK, US, EU and Japan. It enrols approximately 150 children and adolescents ages 2 to under 18. Patients receive two loading doses of 70mg and then two maintenance doses of 45mg over 52 weeks, compared with sham. All patients then move to a 48-week period in which they receive zorevunersen. The primary endpoint is percent change from baseline in major motor seizure frequency. Key secondary endpoints include cognition and behavior measured by Vineland-3. The company says it expects to complete enrollment in the second quarter of 2026 and to start a rolling NDA submission to the FDA in the first half of 2027. The FDA granted zorevunersen Breakthrough Therapy Designation in December 2024.

The second program and the wider pipeline

STK-002 targets the OPA1 gene in ADOA, which the filing describes as the most common inherited optic nerve disorder and one with no approved treatments. The Phase 1 OSPREY study is open-label and dose-escalating, and enrols children and adults ages 6 to 55. The first patient was dosed in February 2026. It is recruiting in the United Kingdom, Germany and Denmark. A separate natural history study, FALCON, reported two-year data in 47 participants. The company is also advancing preclinical programs in the central nervous system, the eye and the heart.

Partners and rights

Under a license and collaboration agreement with Biogen, signed on 14 February 2025, Biogen holds rights to develop and commercialize zorevunersen outside the United States, Canada and Mexico. The company leads global development. It bears 70% of shared development costs and Biogen bears 30%. The company is also eligible for development and commercial milestone payments and for tiered royalties on Biogen's net sales, ranging from low double-digit to high teen percentages. In its own territory of the United States, Canada and Mexico, it says it is building medical affairs and commercial teams.

A January 2022 agreement with Acadia Pharmaceuticals covered neurodevelopmental targets. Acadia terminated the MECP2 and undisclosed target programs effective 3 September 2025, and those rights returned to the company. The SYNGAP1 collaboration continues, with each side carrying 50% of development and commercialization costs and sharing 50% of profits.

TANGO technology is licensed exclusively from the University of Southampton under an agreement dated April 2016.

Manufacturing and competition

The company uses third-party contractors for cGMP manufacturing, including all future commercial manufacturing of zorevunersen. The drug products are expected to be injectable formulations in saline or similar diluents, given intrathecally, intravitreally, subcutaneously or intravenously. The filing names several other companies developing Dravet therapies, including Ionis Pharmaceuticals, Lundbeck, Xenon Pharmaceuticals, Harmony Biosciences and Encoded Therapeutics. For ADOA it names PYC Therapeutics as the only company with an ongoing clinical program.

Its people

Of the 170 employees, 49 hold an M.D. or Ph.D. None are represented by a labor union or covered by collective bargaining agreements.

*From Stoke Therapeutics's annual report on Form 10-K for the year ended 31 December 2025. [The filing](https://www.sec.gov/Archives/edgar/data/1623526/000119312526108362/stok-20251231.htm)*

From the company's 10-K.