Stoke reports full EMPEROR enrollment, sets pre-NDA meeting and Q1 2027 filing start
Stoke Therapeutics said its Phase 3 EMPEROR trial in Dravet syndrome finished enrolling 162 patients, with a data readout expected in the third quarter of 2027 and a rolling NDA submission targeted to begin in early 2027
Stoke Therapeutics reported second quarter 2026 results on August 3, 2026, alongside updates on its lead program zorevunersen for Dravet syndrome and its second clinical candidate, STK-002, for autosomal dominant optic atrophy (ADOA).
Enrollment of 162 patients into the Phase 3 EMPEROR study was completed in June 2026, with a Phase 3 readout anticipated in the third quarter of 2027.1 As of July 31, 2026, roughly 145 patients had passed Week 8 with two loading doses or sham, about 80 had passed Week 24, about 60 had passed Week 28 (the point of primary endpoint measurement), the first patients were expected to reach Week 52 in August 2026, and no patients had discontinued treatment.1 Separately, an additional cohort of about 30 patients is being enrolled in Europe using a needle-prick sham control, with screening closed and the last patient expected to enroll in August 2026.1 China site activation and screening are underway, with enrollment expected to complete in the second half of 2026, though European and Chinese patient data are not planned for the U.S. NDA submission.1
On regulatory timing, Stoke will hold a pre-NDA meeting with the FDA in the second half of 2026 as it prepares to start the rolling NDA submission for zorevunersen in the first quarter of 2027.1 The Phase 3 data are expected to be the final piece needed to complete that rolling submission, with completion expected in the second half of 2027.1
In the ADOA program, Stoke said all eight planned OSPREY trial sites are active, dosing of the first three-patient cohort is complete with no serious or severe safety events observed, dosing of the second cohort is expected to begin in August 2026, and a safety and efficacy readout is anticipated in the first half of 2027.1
On finances, the company held $420.0 million in cash, cash equivalents and marketable securities, comprising $354.3 million as of June 30, 2026 plus $65.7 million in net proceeds from a post-quarter ATM sale to a single investor.1 Stoke said these funds are expected to support operations through potential U.S. commercialization of zorevunersen in early 2028.1
Written by readthrough’s AI from the linked primary sources and fact-checked against them automatically before publishing. Not investment advice.