November 4, 2025 — BEDFORD, Mass. — Stoke Therapeutics, Inc. (Nasdaq: STOK) reported its third-quarter financial results and business updates. The biotechnology company is focused on RNA medicine and is developing zorevunersen for Dravet syndrome.
Stoke’s CEO, Ian F. Smith, highlighted the company’s growth and understanding of Dravet syndrome’s impact, as well as zorevunersen’s potential. The Phase 3 EMPEROR study has randomized over 20 patients and is screening more. New clinical data presented at medical congresses has increased the understanding of zorevunersen’s potential, which is differentiated from other available treatments.
The company plans to meet with the FDA before year-end to review four years of safety and efficacy data and discuss expedited regulatory pathways. Stoke is also investing in scaling for potential commercialization and expanding its pipeline in ADOA and SYNGAP1.
Third-quarter highlights included active patient recruitment for the Phase 3 EMPEROR study in the U.S., UK, and Japan, with European sites expected to initiate in the first half of 2026. New two-year data presented at the CNS Annual Meeting showed continuing improvements in cognition and behavior. Data from ongoing OLE studies presented at the IEC showed durable reductions in major motor seizure frequency and improvements in cognition and behavior.
The company plans to present new data at the AES 2025 Annual Meeting. Patient recruitment for the Phase 1 OSPREY study of STK-002 for ADOA is underway in the UK.
Third-quarter financial results included $328.6 million in cash, cash equivalents, and marketable securities as of September 30, 2025, which are anticipated to fund operations to mid-2028. Revenue for Q3 2025 increased to $10.6 million, and net loss was $38.3 million, or $0.65 per share.
Ian F. Smith, Chief Executive Officer and Director of Stoke Therapeutics
Source: Stoke Therapeutics
