Stoke Therapeutics Reports Third Quarter 2025 Financial Results and Provides Business Updates
Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company pioneering RNA-based medicines to restore protein expression, today reported financial results for the third quarter ended September 30, 2025, and provided business updates on its lead program, zorevunersen, and expanding pipeline.
CEO Commentary
Stoke’s momentum in 2025 has set the stage for significant growth,” said Ian F. Smith, Chief Executive Officer and Director of Stoke Therapeutics. “We are building awareness of the profound impact of Dravet syndrome and demonstrating how zorevunersen may transform outcomes through durable seizure reduction and cognitive improvement.”
In just three months since launching our global Phase 3 EMPEROR study, we have randomized more than 20 patients, with additional participants entering screening,” Smith continued. “We look forward to engaging with the FDA under our Breakthrough Therapy Designation before year-end to discuss how we can expedite access to zorevunersen for patients in need.”
Smith added, “Our strong financial foundation enables us to invest strategically in commercialization readiness and pipeline expansion in ADOA and SYNGAP1.
Recent Business Highlights
Zorevunersen (Dravet Syndrome)
- The global Phase 3 EMPEROR study is enrolling patients in the U.S., UK, and Japan; European sites are expected to open in 1H 2026.
- More than 20 patients have been randomized, and an additional 35 are in the 8-week screening phase.
- New two-year data presented at the 2025 Child Neurology Society Annual Meeting showed sustained cognitive and behavioral improvements in zorevunersen-treated patients, contrasting with minimal changes in natural history controls.
- Three-year safety and efficacy data presented at the 36th International Epilepsy Congress demonstrated durable seizure reduction and continued cognitive benefits, with no new safety concerns.
- New data will be presented at the American Epilepsy Society (AES) 2025 Annual Meeting in December.
- A Type B meeting with the FDA is planned before year-end 2025 to review four years of safety and efficacy data and discuss expedited regulatory pathways under the Breakthrough Therapy Designation.
Pipeline and Corporate Updates
- STK-002 (Autosomal Dominant Optic Atrophy):
The Phase 1 OSPREY study has begun patient enrollment in the UK. EMA authorization has been received, with European sites expected to initiate in early 2026.
At the 2025 American Academy of Ophthalmology Meeting, the Company presented 24-month FALCON natural history study data providing critical insights into ADOA progression. - SYNGAP1 Program:
Lead optimization is underway to select a clinical candidate in 2026 for this rare neurodevelopmental disorder. - Leadership Update:
In October, the Board appointed Ian F. Smith as permanent Chief Executive Officer. Dr. Arthur Tzianabos resumed his position as Chairman after serving as Executive Chairman during the CEO search.
Third Quarter 2025 Financial Results
- Cash Position: $328.6 million as of September 30, 2025, expected to fund operations into mid-2028.
- Subsequent Event: Since quarter-end, the Company raised $48.7 million through the sale of ~1.8 million shares under its Controlled Equity Offering.
- Revenue: $10.6 million, up from $4.9 million in Q3 2024, driven by collaboration revenue from Acadia and Biogen.
- Net Loss: $38.3 million, or $0.65 per share, compared to $26.4 million, or $0.47 per share, in Q3 2024.
- R&D Expenses: $37.7 million (vs. $22.2 million in Q3 2024), reflecting increased program activity and personnel costs.
- SG&A Expenses: $16.0 million (vs. $12.7 million in Q3 2024), driven by higher personnel and launch readiness costs.
Year-to-Date 2025 Financial Results
- Revenue: $183.0 million (vs. $13.9 million YTD 2024), primarily reflecting IP license and collaboration revenue from Biogen and Acadia.
- Net Income: $51.0 million, or $0.85 per diluted share, compared to a net loss of $78.5 million, or $1.48 per share, for the same period in 2024.
- R&D Expenses: $96.2 million (vs. $65.7 million YTD 2024).
- SG&A Expenses: $45.9 million (vs. $36.0 million YTD 2024).
About Dravet Syndrome
Dravet syndrome is a severe developmental and epileptic encephalopathy (DEE) characterized by severe, recurrent seizures as well as significant cognitive and behavioral impairments. Most cases of Dravet are caused by mutations in one copy of the SCN1A gene, leading to insufficient levels of NaV1.1 protein in neuronal cells in the brain. More than 90 percent of patients continue to experience seizures despite treatment with the best available anti-seizure medicines. Complications of the disease often contribute to a poor quality of life for patients and their caregivers. Developmental and cognitive impairments often include intellectual disability, developmental delays, movement and balance issues, language and speech disturbances, growth defects, sleep abnormalities, disruptions of the autonomic nervous system and mood disorders. Compared with the general epilepsy population, people living with Dravet syndrome have a higher risk of sudden unexpected death in epilepsy, or SUDEP. Dravet syndrome occurs globally and is not concentrated in a particular geographic area or ethnic group. Currently, it is estimated that up to 38,000 people are living with Dravet syndrome in the U.S. (~16,000), UK, EU-4 and Japan.1
About Zorevunersen
Zorevunersen is an investigational antisense oligonucleotide that is designed to treat the underlying cause of Dravet syndrome by increasing functional NaV1.1 protein production in brain cells from the non-mutated (wild-type) copy of the SCN1A gene. This highly differentiated mechanism of action aims to reduce seizure frequency beyond what has been achieved with anti-seizure medicines and to improve neurodevelopment, cognition, and behavior. Zorevunersen has demonstrated the potential for disease modification and has been granted orphan drug designation by the FDA and the EMA. The FDA has also granted zorevunersen rare pediatric disease designation and Breakthrough Therapy Designation for the treatment of Dravet syndrome with a confirmed mutation not associated with gain-of-function, in the SCN1A gene. Stoke has a strategic collaboration with Biogen to develop and commercialize zorevunersen for Dravet syndrome. Under the collaboration, Stoke retains exclusive rights for zorevunersen in the United States, Canada, and Mexico; Biogen receives exclusive rest of world commercialization rights.
About the EMPEROR Study
The EMPEROR Phase 3 Study (NCT06872125) is a global, double-blind, sham-controlled study evaluating the efficacy, safety and tolerability of zorevunersen in children ages 2 to <18 with Dravet syndrome with a confirmed variant in the SCN1A gene not associated with gain-of-function. Study participants are randomized 1:1 to receive either zorevunersen via intrathecal administration or a sham comparator for a 52-week treatment period following an 8-week baseline period. An open-label extension treatment period will allow all patients the opportunity to receive treatment with zorevunersen following the 52-week treatment period. The primary endpoint of the study is percent change from baseline in major motor seizure frequency at week 28 in patients receiving zorevunersen as compared to sham. The key secondary endpoints are the durability of effect on major motor seizure frequency and improvements in behavior and cognition as measured by Vineland-3 subdomains, including expressive communication, receptive communication, interpersonal relationships, coping skills and personal skills. Additional endpoints include safety, Clinician Global Impression of Change (CGI-C), Caregiver Global Impression of Change (CaGI-C), EuroQol Visual Analog Scale (EQ-VAS) and the Bayley Scales of Infant Development (BSID-IV). EMPEROR has initiated in the United States, United Kingdom, Japan and is planned for Europe. For more information on the EMPEROR study,
About Autosomal Dominant Optic Atrophy (ADOA)
ADOA is the most common inherited optic nerve disorder. It is a rare disease that causes progressive and irreversible vision loss in both eyes starting in the first decade of life. Severity can vary and the rate of vision loss can be difficult to predict. Roughly half of people with ADOA fail driving standards and up to 46% are registered as legally blind. More than 400 different OPA1 variants have been reported in people diagnosed with ADOA. ADOA affects approximately one in 30,000 people globally with a higher incidence in Denmark of one in 10,000 due to a founder effect. Currently there is no approved treatment for people living with ADOA.
About STK-002
STK-002 is a proprietary antisense oligonucleotide (ASO) in clinical development for the treatment of ADOA. Stoke believes that STK-002 has the potential to be the first disease-modifying therapy for people living with ADOA. An estimated 65% to 90% of cases are caused by variants in the OPA1 gene, most of which lead to a haploinsufficiency resulting in 50% OPA1 protein expression and disease manifestation. STK-002 is designed to upregulate OPA1 protein expression by leveraging the non-mutant (wild-type) copy of the OPA1 gene to restore OPA1 protein expression with the aim to maintain or improve vision in patients with ADOA. Stoke has generated preclinical data demonstrating proof-of-mechanism and proof-of-concept for STK-002. STK-002 has been granted orphan drug designation by the FDA as a potential new treatment for ADOA. A Phase 1 study (OSPREY) of STK-002 in people with ADOA is now underway.
About Stoke Therapeutics
Stoke Therapeutics (Nasdaq: STOK), is a biotechnology company dedicated to restoring protein expression by harnessing the body’s potential with RNA medicine. Using Stoke’s proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach, Stoke is developing antisense oligonucleotides (ASOs) to selectively restore naturally-occurring protein levels. Stoke’s first medicine in development, zorevunersen, has demonstrated the potential for disease modification in patients with Dravet syndrome and is currently being evaluated in a Phase 3 study. Stoke’s initial focus are diseases of the central nervous system and the eye that are caused by a loss of ~50% of normal protein levels (haploinsufficiency). Proof of concept has been demonstrated in other organs, tissues, and systems, supporting broad potential for Stoke’s proprietary approach. Stoke is headquartered in Bedford, Massachusetts.
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