Stoke Therapeutics Advances Phase 3 Dravet Syndrome Trial with Strong Financial Position Through 2028
核心洞察
Stoke Therapeutics reports strong progress in its Phase 3 EMPEROR study for zorevunersen in Dravet syndrome (搜索), with more than 20 patients randomized and approximately 35 additional patients in screening as of October 2025.
The company maintains a robust financial position with $328.6 million in cash and securities as of September 30, 2025, providing funding runway through mid-2028 to support operations and commercialization preparations.
Recent clinical data presentations demonstrate zorevunersen's potential for disease modification, showing continuing improvements in cognition and behavior at two years, contrasting with minimal changes in standard-of-care treated patients.
Stoke Therapeutics reported significant progress in its Phase 3 EMPEROR study for zorevunersen, an investigational antisense oligonucleotide for Dravet syndrome (搜索), while maintaining a strong financial position to fund operations through mid-2028. The company announced third quarter 2025 financial results showing $328.6 million in cash, cash equivalents, and marketable securities as of September 30, 2025.
Phase 3 EMPEROR Study Shows Strong Enrollment Progress
The global Phase 3 EMPEROR study is actively recruiting patients across the U.S., UK, and Japan, with European sites expected to initiate in the first half of 2026. As of the end of October 2025, more than 20 patients were randomized to receive either zorevunersen or sham treatment, with approximately 35 additional patients entering the formal 8-week screening period. The company previously disclosed that more than 150 patients had been identified by investigators as potential study candidates, and the study remains on track to complete enrollment in the second half of 2026.
"In just three months since the initiation of our Phase 3 EMPEROR study, we have made significant progress with more than 20 patients randomized into dosing and a steady progression of more patients entering the screening period," said Ian F. Smith, Chief Executive Officer and Director of Stoke Therapeutics.
Clinical Data Demonstrates Disease-Modifying Potential
Recent clinical presentations have provided additional evidence of zorevunersen's disease-modifying potential. In October 2025, the company presented new two-year data at the 54th Child Neurology Society Annual Meeting showing continuing improvements in cognition and behavior that contrast with findings from a two-year natural history study where patients with Dravet syndrome (搜索) treated with standard of care showed minimal changes.
The data revealed that 95% of patients treated with zorevunersen in ongoing open-label extension studies (n=19) showed improvements in overall clinical status as reported separately by clinicians and caregivers. In September, three-year safety and efficacy data presented at the 36th International Epilepsy Congress demonstrated that zorevunersen was generally well tolerated with no new safety findings, while showing durable reductions in major motor seizure frequency and continuing improvements in cognition and behavior.
Regulatory Pathway and FDA Meeting
Stoke Therapeutics is scheduled to meet with the FDA before year-end 2025 to review four years of safety and efficacy data from clinical studies of zorevunersen in patients with Dravet syndrome (搜索). The meeting will focus on discussing how the company and FDA can work together under the Breakthrough Therapy Designation to deliver zorevunersen to patients through potential expedited regulatory pathways.
Zorevunersen has been granted orphan drug designation by both the FDA and EMA, along with rare pediatric disease designation and Breakthrough Therapy Designation from the FDA for treating Dravet syndrome (搜索) with confirmed SCN1A (搜索) gene mutations not associated with gain-of-function.
Pipeline Expansion and Corporate Updates
The company announced that patient recruitment into the Phase 1 OSPREY study of STK-002 is underway in the UK. STK-002 is being developed as a potential disease-modifying medicine for Autosomal Dominant Optic Atrophy (搜索) (ADOA), the most common inherited optic nerve disorder. The OSPREY study has been authorized by the European Medicines Authority, with European sites expected to activate in early 2026.
Lead optimization is underway to identify a clinical candidate for the treatment of SYNGAP1 (搜索), a severe and rare genetic neurodevelopmental disease, with plans to advance to clinical development in 2026. In October, the company's Board of Directors appointed Ian F. Smith as Chief Executive Officer after serving as Interim CEO since March 2025.
Financial Performance and Outlook
For the third quarter 2025, Stoke reported revenue of $10.6 million, an increase from $4.9 million in Q3 2024, driven by contractual obligations under agreements with Acadia (搜索) and Biogen. The net loss for Q3 2025 was $38.3 million, or $0.65 per share, compared to a net loss of $26.4 million, or $0.47 per share, for the same period in 2024.
Research and development expenses increased to $37.7 million from $22.2 million in Q3 2024, reflecting increased activities and personnel expenses to support zorevunersen advancement. Sales, general and administrative expenses rose to $16.0 million from $12.7 million, driven by personnel and launch readiness expenses.
For the nine months ended September 30, 2025, revenue reached $183.0 million, a significant increase from $13.9 million for the same period in 2024, primarily driven by $150.8 million related to IP license performance obligations under the Biogen Agreement.
About Dravet Syndrome and Zorevunersen
Dravet syndrome (搜索) is a severe developmental and epileptic encephalopathy characterized by severe, recurrent seizures and significant cognitive and behavioral impairments. Most cases are caused by mutations in the SCN1A (搜索) gene, leading to insufficient levels of NaV1.1 (搜索) protein in brain neuronal cells. More than 90% of patients continue to experience seizures despite treatment with the best available anti-seizure medicines.
Zorevunersen is designed to treat the underlying cause of Dravet syndrome (搜索) by increasing functional NaV1.1 (搜索) protein production from the non-mutated copy of the SCN1A (搜索) gene. This mechanism aims to reduce seizure frequency beyond what has been achieved with anti-seizure medicines while improving neurodevelopment, cognition, and behavior. It is estimated that up to 38,000 people are living with Dravet syndrome in the U.S., UK, EU-4, and Japan.