BEDFORD, Mass.– Stoke Therapeutics Inc. (Nasdaq: STOK) reported a net loss of $73.6 million for the third quarter of 2026, nearly doubling its loss from a year earlier, as the biotechnology company increased spending on clinical development and preparations for a potential regulatory submission of its experimental Dravet syndrome treatment.
The Bedford, Massachusetts-based company reported a quarterly loss of $1.08 per share, compared with a loss of $38.3 million, or 65 cents per share, during the same period in 2025.
Revenue increased to $11.3 million from $10.6 million a year earlier, primarily reflecting income from collaboration and licensing agreements with Acadia Pharmaceuticals and Biogen.
Stoke ended September with $358.8 million in cash, cash equivalents and marketable securities, which the company expects will support operations into early 2028.
The company continues to advance zorevunersen, its lead investigational treatment for Dravet syndrome, a rare genetic form of epilepsy characterized by severe seizures and developmental challenges.
Stoke said its global Phase 3 EMPEROR clinical trial is progressing toward a planned data release in the third quarter of 2027, with a U.S. New Drug Application submission expected during the second half of that year.
The trial has completed enrollment of 162 patients in its primary analysis population across the United States, United Kingdom and Japan.
As of October 29, approximately 110 patients had completed their Week 28 assessments, when researchers measure changes in major motor seizure frequency, the study’s primary endpoint.
About 20 patients had completed the full 52-week treatment period, and all had entered the study’s open-label extension phase. No patients had discontinued treatment.
“The Phase 3 EMPEROR study of zorevunersen continues to progress well. Approximately 110 of 162 enrolled patients have completed their Week 28 visit when the primary endpoint of change in major motor seizure frequency is measured and approximately 20 are through Week 52, the end of the treatment period. All 20 of these patients are continuing into the open-label extension period of the study and to date, no patients have discontinued treatment,” said Ian F. Smith, chief executive officer and director of Stoke Therapeutics.
“Our NDA planning is on track following our recent discussion with the FDA. Importantly, the key secondary endpoint of changes in patients’ cognition and behavior will be measured using a multicomponent assessment of four Vineland-3 subdomains. This multicomponent measure reflects the diversity of effects that people with Dravet syndrome experience and how to best evaluate them in a clinical trial. We look forward to the Phase 3 data readout in the third quarter of 2027 and completing our NDA submission in the second half of 2027.”
Stoke also announced progress in discussions with the U.S. Food and Drug Administration regarding the planned regulatory submission for zorevunersen.
Following a recent meeting with the agency, the company plans to evaluate changes in cognition and behavior using a combined assessment of four areas: expressive communication, receptive communication, interpersonal relationships and personal skills.
The assessment will serve as a key secondary endpoint in the EMPEROR study, providing additional information about the treatment’s potential effects beyond seizure reduction.
Stoke expects to submit supporting clinical data to the FDA throughout the first half of 2027 as it prepares to complete its regulatory application later that year.
In addition to the primary trial population, Stoke has enrolled 34 patients in Germany, France, Spain and Italy, along with five patients in China.
The company is developing zorevunersen as a potential disease-modifying therapy designed to address an underlying genetic cause of Dravet syndrome.
Stoke and Biogen recently presented long-term clinical findings suggesting that the treatment may reduce certain severe seizure types. However, the drug remains investigational, and its safety and effectiveness have not yet been established through the ongoing Phase 3 trial.
If the clinical results support regulatory approval, Stoke is preparing for a potential U.S. commercial launch in early 2028.
Beyond zorevunersen, the company is advancing STK-002, an experimental treatment for autosomal dominant optic atrophy (ADOA), a rare inherited eye disease that can cause progressive vision loss and blindness.
The Phase 1 OSPREY study is continuing its dose-escalation phase, with dosing completed in the first two patient cohorts and underway in the third.
Stoke expects the third cohort to complete dosing by the end of 2026, with a fourth cohort scheduled to begin in early 2027.
Initial safety and efficacy data from the later cohorts are anticipated during the first half of 2027.
“Our second clinical program, STK-002, targets the protein haploinsufficiency that causes ADOA and therefore may have the potential to improve vision in people living with this severe genetic disease that often leads to blindness. Dosing in the first two cohorts is complete and dosing in the third cohort is underway with the fourth cohort expected to follow in early 2027. Safety and efficacy data are anticipated in the first half of 2027. With this progress across the business, we are building Stoke for the future with the team and capabilities to realize the full potential of zorevunersen, our pipeline and our unique scientific approach,” Smith said.
Stoke is also conducting early-stage research to identify a potential treatment candidate for SYNGAP1-related disorders, a group of rare neurodevelopmental conditions.
The company’s expanding clinical programs contributed to higher operating expenses during the third quarter.
Research and development spending rose to $59.8 million from $37.7 million a year earlier, driven primarily by increased clinical trial activity and personnel expenses associated with zorevunersen.
Selling, general and administrative expenses increased to $28.7 million from $16 million, reflecting additional staffing and preparations for a potential commercial launch.
For the first nine months of 2026, Stoke reported revenue of $26.8 million, down from $183 million during the comparable period in 2025.
The sharp decline primarily reflected the recognition of $150.8 million in revenue associated with a Biogen intellectual property licensing obligation during the previous year.
Stoke recorded a nine-month net loss of $185.3 million, or $2.81 per share, compared with net income of $51 million, or 85 cents per diluted share, during the same period in 2025.
Research and development expenses for the nine-month period increased to $149 million from $96.2 million, while selling, general and administrative costs climbed to $74 million from $45.9 million.
Despite the widening losses, Stoke maintained that its existing cash reserves should provide sufficient funding to continue its clinical programs and regulatory preparations into early 2028.
