Stoke Therapeutics to Report 2027 EMPEROR Study Results
The company expects to file a New Drug Application in late 2027 following FDA guidance on clinical trial endpoints.
Updated on Oct. 30, 2026 in Biotech
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Stoke Therapeutics plans to submit data in the first half of 2027 and report EMPEROR study results in the third quarter of 2027. Following recent guidance from the Food and Drug Administration, the company intends to file a New Drug Application in the second half of 2027.
Why it matters
The company’s roadmap for regulatory submission follows an agreement with the Food and Drug Administration to use the multicomponent Vineland-3 assessment as a secondary endpoint, establishing a clearer pathway for clinical validation.
Of the 162 total patients enrolled in the EMPEROR study, 110 have successfully completed the week 28 visit. The company reported $358.8 million in cash, cash equivalents, and marketable securities as of September 30, 2026.
The players
Stoke Therapeutics
A biotechnology company focused on developing RNA-based medicines to address the underlying causes of severe genetic diseases.
Food and Drug Administration
The federal agency responsible for regulating new drug approvals and establishing clinical trial endpoint requirements in the United States.
The details
The EMPEROR study employs the Vineland-3 assessment — a standardized scale used to measure adaptive behavior in individuals with intellectual and developmental disabilities — as a secondary endpoint. The Food and Drug Administration has confirmed it will accept this multicomponent metric for the study submission. The company is now preparing to finalize and submit its accumulated data throughout the first half of 2027.
Timeline
September 30, 2026: Stoke Therapeutics held $358.8 million in cash and securities.
First half of 2027: The company plans to submit study data for review.
Q3 2027: Stoke Therapeutics expects to report final EMPEROR study results.
Second half of 2027: The company intends to file a New Drug Application.
Early 2028: Existing funding is expected to sustain company operations.
The Tech Race
Stoke Therapeutics is aligning its clinical development roadmap with the FDA’s standard for adaptive behavior measurements in genetic disorder trials. This approach mirrors current industry efforts to validate molecular therapies through standardized functional outcomes rather than isolated biomarkers alone.
Patients and caregivers awaiting potential therapies should look for the EMPEROR study results in the third quarter of 2027. If the application proceeds as planned, current funding levels ensure the company remains operational into early 2028.
The takeaway
The trajectory of this therapeutic development depends on meeting the secondary endpoint metrics established by the FDA. Stakeholders should monitor the third-quarter 2027 results, which will serve as the primary indicator for the feasibility of the subsequent New Drug Application.
What happens next
Stoke Therapeutics will report the EMPEROR study results in the third quarter of 2027 and file a New Drug Application in the second half of 2027.
Further reading
Learn more about the latest developments in the Biotech sector.
Source note: This article includes information reported by TokenPost.
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