Stoke Completes FDA Pre-NDA Meeting on Zorevunersen

The Tip Desk · November 1, 2026 · 3 min read

Cash of $358.8 million as of September 30, 2026 is expected to fund operations into early 2028 as EMPEROR patients advance toward the Week 28 primary endpoint.

Stoke Therapeutics (STOK) said it completed a pre-NDA meeting with the U.S. Food and Drug Administration to align on the planned New Drug Application for zorevunersen, its investigational antisense oligonucleotide for Dravet syndrome, and now plans to submit data throughout the first half of 2027 to support NDA completion in the second half of 2027.

The company had previously said it would initiate a rolling U.S. NDA submission in the first quarter of 2027 after a pre-NDA meeting scheduled for the second half of 2026. The October update replaced that rolling-start framing with a data-submission plan across the first half of 2027 and NDA completion in the second half of 2027.

A new element of the NDA plan is the statistical treatment of a key secondary endpoint. Stoke said changes in cognition and behavior in the Phase 3 EMPEROR study will be analyzed using a multicomponent assessment of four prespecified Vineland-3 subdomains — expressive communication, receptive communication, interpersonal relationships and personal skills — with treatment effects calculated using a multivariate mixed model for repeated measures and then averaged into a single score.

Enrollment of 162 patients in the primary analysis population (United States, United Kingdom and Japan) was completed in June 2026, matching the second-quarter update, and a data readout remains anticipated in the third quarter of 2027. As of October 29, 2026, all 162 patients were through Week 8, approximately 130 through Week 24, approximately 110 through the Week 28 primary-endpoint visit, approximately 65 through Week 40, and approximately 20 through Week 52. All 20 patients through Week 52 were continuing into the open-label extension, and no patients had discontinued treatment.

Additional enrollment beyond the primary analysis population included 34 patients in Germany, France, Spain and Italy, completed in August 2026, and 5 patients in China, completed in October 2026.

Chief Executive Officer Ian F. Smith said NDA planning is on track following the FDA discussion and that the company looks forward to the Phase 3 data readout in the third quarter of 2027 and completing the NDA submission in the second half of 2027.

In the Phase 1 OSPREY study of STK-002 for Autosomal Dominant Optic Atrophy, dosing is complete in the first two cohorts, dosing of the third cohort is underway and expected to complete by the end of 2026, and the fourth cohort is expected in early 2027. Initial safety and efficacy data from the later cohorts are anticipated in the first half of 2027. The second-quarter update had said dosing was complete only in the sentinel cohort. Lead optimization is underway to identify a development candidate for SYNGAP1-related disorders.

Revenue recognized for the three months ended September 30, 2026, was $11.3 million, up from $10.6 million a year earlier, from collaboration and licensing agreements with Acadia and Biogen.

Net loss for the three months ended September 30, 2026, was $73.6 million, or $1.08 a share, compared with $38.3 million, or $0.65 a share, a year earlier, including $11.0 million of non-cash stock-based compensation versus $8.0 million. Research and development expenses were $59.8 million, which the company attributed to increased activities and personnel expenses to support zorevunersen. Sales, general and administrative expenses were $28.7 million, which the company said was driven by growth in personnel and launch readiness expenses.

Cash, cash equivalents and marketable securities were $358.8 million as of September 30, 2026, down from $420 million after a subsequent $65.7 million ATM sale, based on $354.3 million as of June 30. The company said the cash position is expected to fund operations into early 2028.

Stoke said it is continuing educational efforts at neurology and epilepsy congresses through the remainder of 2026 and into 2027 while it prepares for a potential U.S. approval and launch in early 2028.