Product Description
STK-001 is an investigational new medicine for the treatment of Dravet syndrome currently being evaluated in ongoing clinical trials. Stoke believes that STK-001, a proprietary antisense oligonucleotide (ASO), has the potential to be the first disease-modifying therapy to address the genetic cause of Dravet syndrome. STK-001 is designed to upregulate NaV1.1 protein expression by leveraging the non-mutant (wild-type) copy of the SCN1A gene to restore physiological NaV1.1 levels, thereby reducing both occurrence of seizures and significant non-seizure comorbidities. STK-001 has been granted orphan drug designation by the FDA and the EMA, and rare pediatric disease designation by the FDA as a potential new treatment for Dravet syndrome. (Sourced from: https://investor.stoketherapeutics.com/news-releases/news-release-details/stoke-therapeutics-announces-positive-new-safety-efficacy-data)
Mechanisms of Action: Voltage-Gated Sodium Channel Modulator
Novel Mechanism: Yes
Modality: Nucleic Acid
Route of Administration: Injection
FDA Designation: Breakthrough Therapy - Dravet Syndrome *
Approval Status: Not Approved
Approved Countries: None
Approved Indications: None
Company: Stoke Therapeutics
Company Location:
Company Founding Year: 2014
Additional Commercial Interests: Biogen
Clinical Description
Countries in Clinic: France, Germany, Italy, Japan, Spain, United Kingdom, United States
Active Clinical Trial Count: 4
Recent & Upcoming Milestones
- Clinical Outcomes Expected - Biogen announced they will present P3 Dravet Syndrome results in 3Q27 for Zorevunersen
- Clinical Outcomes Reported - Biogen presented P1 Dravet Syndrome results on 2026-05-07 for Zorevunersen
- Clinical Outcomes Reported - Stoke Therapeutics presented P1 Dravet Syndrome results on 2026-03-04 for Zorevunersen
Highest Development Phases
Phase 3: Dravet Syndrome|Epilepsies, Myoclonic
Trial ID |
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
Latest Trial Update Date |
Data Updated |
|---|---|---|---|---|---|---|---|---|
NCT06872125 |
EMPEROR | P3 |
Recruiting |
Dravet Syndrome|Epilepsies, Myoclonic |
2027-03-01 |
4% |
2026-04-14 |
Primary Endpoints|Treatments |
NCT04740476 |
STK-001-DS-501 | P2 |
Active, not recruiting |
Epilepsies, Myoclonic|Dravet Syndrome |
2029-03-31 |
12% |
2026-02-05 |
Primary Endpoints|Treatments |
2024-519555-28-00 |
STK-001-DS-301 | P3 |
Not yet recruiting |
Dravet Syndrome |
2027-08-31 |
|||
jRCT2071250035 |
jRCT2071250035 | P3 |
Not yet recruiting |
Dravet Syndrome |
2027-08-07 |
Recent News Events
Date |
Type |
Title |
|---|---|---|
|
04/07/2026 |
News Article |
Stoke Therapeutics Appoints Clare Kahn, Ph.D., to its Board of Directors |
|
04/16/2026 |
News Article |
Stoke Therapeutics Announces Inducement Grants Under Nasdaq Listing Rule 5635(C)(4) |
|
04/08/2026 |
News Article |
The Credibility Filter Wall Street Uses to Sort Biotech Winners |
|
04/08/2026 |
News Article |
Stoke Therapeutics to Present at the 25th Annual Needham Virtual Healthcare Conference |