Drugs / zorevunersen
Regulatory milestones approvals, filings & regulatory actions · 1 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed | US (FDA) | — | Dravet syndrome | — | “Submitted an Investigational New Drug (IND) application for STK-001 to the FDA in late 2019.” stoketherapeutics.com ↗ |
Trials 3
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/21 trial · 1 met primary | ||||||
| Phase 1/2 | NCT04442295 | Jun 2020 → Nov 2023 | Dravet syndrome | Stoke Therapeutics, Inc | Completed | Met primary |
| Phase 21 trial · 1 met primary | ||||||
| Phase 2 | NCT04740476 | Jan 2021 → Mar 2029 expected | Dravet syndrome | Stoke Therapeutics, Inc | Active not recruiting | Met primary |
| Phase 31 trial | ||||||
| Phase 3 | NCT06872125 | Jun 2025 → Mar 2027 expected | Dravet syndrome | Stoke Therapeutics, Inc | Recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 40
| Date | Issuer | Release |
|---|---|---|
| 2026-09-03 | Stoke Therapeutics, Inc | Results Stoke Therapeutics and Biogen Present Long-Term Clinical Data that Support the Disease-Modifying Potential of Zorevunersen, an Investigational Medicine for the Treatment of Dravet Syndrome, at the 16th European Epilepsy Congress (EEC) stoketherapeutics.com ↗
Four-year OLE results showed substantial and durable reductions in seizures and continuing improvements in cognition and behavior. |
| 2026-09-01 | Stoke Therapeutics, Inc | Results Stoke Therapeutics and Biogen Announce Presentations of Clinical Data from Studies of Zorevunersen, an Investigational Medicine for the Treatment of Dravet Syndrome, at the 16th European Epilepsy Congress (EEC) stoketherapeutics.com ↗
Improvements in cognition and behavior were sustained through 4 years, in addition to durable seizure reductions and a generally well-tolerated safety profile. |
| 2026-05-07 | Stoke Therapeutics, Inc | Results Stoke Therapeutics Announces First Quarter 2026 Financial Results and Provides Business Updates stoketherapeutics.com ↗
New 4-year longitudinal data from the Phase 1/2a open-label extension (OLE) studies provide additional support for the disease-modifying potential of zorevunersen, an investigational medicine for the treatment of Dravet syndrome |
| 2026-03-04 | Stoke Therapeutics, Inc | Results The New England Journal of Medicine Publishes First Data to Demonstrate the Potential for Disease Modification in Dravet Syndrome stoketherapeutics.com ↗
The publication includes results from two completed Phase 1/2a and ongoing open-label extension (OLE) studies that demonstrate, for the first time, the potential for disease modification in people living with Dravet syndrome. |
| 2025-12-05 | Biogen | Results Biogen and Stoke Therapeutics Present Data that Further Support the Disease-Modifying Potential of Zorevunersen, an Investigational Medicine for the Treatment of Dravet Syndrome, at the 2025 American Epilepsy Society (AES) Annual Meeting biogen.com ↗
Long-term Phase 1/2a and open label extension (OLE) data for zorevunersen on top of standard of care anti-seizure medicines (ASMs) demonstrate durable seizure reductions, including increases in seizure-free days, in addition to improvements in cognition, behavior and quality of life |
| 2025-12-05 | Stoke Therapeutics, Inc | Results Stoke Therapeutics and Biogen Present Data that Further Support the Disease-Modifying Potential of Zorevunersen, an Investigational Medicine for the Treatment of Dravet Syndrome, at the 2025 American Epilepsy Society (AES) Annual Meeting stoketherapeutics.com ↗ |
| 2025-12-01 | Biogen | Results Biogen and Stoke Therapeutics Announce Presentations at the 2025 American Epilepsy Society Annual Meeting biogen.com ↗
New analyses from the ongoing open label extension (OLE) studies and findings from electroencephalogram (EEG) assessments in patients with Dravet syndrome treated with zorevunersen support the potential for disease modification |
| 2025-12-01 | Stoke Therapeutics, Inc | Results Stoke Therapeutics and Biogen Announce Presentations at the 2025 American Epilepsy Society Annual Meeting stoketherapeutics.com ↗
New analyses from the ongoing open label extension (OLE) studies and findings from electroencephalogram (EEG) assessments in patients with Dravet syndrome treated with zorevunersen support the potential for disease modification |
| 2025-10-09 | Stoke Therapeutics, Inc | Results Stoke Therapeutics and Biogen Present New Data at the 54th Child Neurology Society (CNS) Annual Meeting that Support the Potential of Zorevunersen as a Disease-Modifying Medicine for Dravet Syndrome stoketherapeutics.com ↗
New two-year data from an analysis that was initially performed to understand the potential effects of the Phase 3 dosing regimen on cognition and behavior showed continuing improvements at two years. |
| 2025-09-02 | Biogen | Results Biogen and Stoke Therapeutics Present Data at the 36th International Epilepsy Congress that Support the Potential for Zorevunersen to be the First Disease-Modifying Medicine for Dravet Syndrome biogen.com ↗ |
| 2025-09-02 | Stoke Therapeutics, Inc | Results Stoke Therapeutics and Biogen Present Data at the 36th International Epilepsy Congress that Support the Potential for Zorevunersen to be the First Disease-Modifying Medicine for Dravet Syndrome stoketherapeutics.com ↗
Stoke Therapeutics , Inc. (Nasdaq: STOK), a biotechnology company dedicated to restoring protein expression by harnessing the body’s potential with RNA medicine, and Biogen Inc. (Nasdaq: BIIB), today announced data from Phase 1/2a and open-label extension (OLE) studies of zorevunersen that support the potential for zorevunersen to be the first disease-modifying medicine for Dravet syndrome. |
| 2025-08-25 | Biogen | Results Biogen and Stoke Therapeutics Announce Presentations of Clinical Data from Studies of Zorevunersen for the Potential Treatment of Dravet Syndrome at the 36th International Epilepsy Congress biogen.com ↗
3-year data from open-label extension (OLE) studies demonstrate the potential for disease modification with durable seizure reductions and improvements in cognition and behavior on top of standard anti-seizure medicines |
| 2025-08-25 | Stoke Therapeutics, Inc | Results Stoke Therapeutics and Biogen Announce Presentations of Clinical Data from Studies of Zorevunersen for the Potential Treatment of Dravet Syndrome at the 36th International Epilepsy Congress stoketherapeutics.com ↗
3-year data from open-label extension (OLE) studies demonstrate the potential for disease modification with durable seizure reductions and improvements in cognition and behavior on top of standard anti-seizure medicines |
| 2025-08-12 | Stoke Therapeutics, Inc | Results Stoke Therapeutics Reports Second Quarter 2025 Financial Results and Provides Business Updates stoketherapeutics.com ↗
New 3-year zorevunersen OLE data provide additional support for disease modification: continuing and durable reductions in seizures and improvements in cognition and behavior and generally well tolerated |
| 2025-07-10 | Stoke Therapeutics, Inc | Results Stoke Therapeutics and Biogen Announce Presentation of Data from Studies of Zorevunersen, an Investigational Medicine for Dravet syndrome, at the 16th European Paediatric Neurology Society (EPNS) Congress stoketherapeutics.com ↗
The new analysis is best aligned with the timing and dosing regimen that will be evaluated in the pivotal Phase 3 EMPEROR study and showed improvements in multiple measures of cognition and behavior at Week 68. |
| 2024-12-06 | Stoke Therapeutics, Inc | Results Stoke Therapeutics Presents New Open-Label Extension (OLE) Study Data That Further Support the Potential for Zorevunersen as a Disease-Modifying Medicine for the Treatment of Dravet Syndrome stoketherapeutics.com ↗
Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to restoring protein expression by harnessing the body’s potential with RNA medicine, today announced new data from an analysis of nine patients treated with an initial 2 or 3 doses of 70mg, followed by 45mg maintenance dosing in the Phase 1/2a and open-label extension (OLE) studies of zorevunersen. |
| 2024-12-04 | Stoke Therapeutics, Inc | Regulatory Stoke Therapeutics Receives FDA Breakthrough Therapy Designation for Zorevunersen for the Treatment of Dravet Syndrome stoketherapeutics.com ↗ |
| 2024-12-02 | Stoke Therapeutics, Inc | Results Stoke Therapeutics Announces Plans to Present New Data that Support Zorevunersen as Potentially the First Disease-Modifying Medicine for Dravet Syndrome at the American Epilepsy Society 2024 Annual Meeting stoketherapeutics.com ↗ |
| 2024-09-10 | Stoke Therapeutics, Inc | Results Stoke Therapeutics Presents Zorevunersen Data Showing Substantial Reductions in Seizures and Improvements in Multiple Measures of Cognition and Behavior That Support the Potential for Disease Modification in Dravet Syndrome stoketherapeutics.com ↗
Zorevunersen (STK-001) data showing substantial and sustained reductions in seizures and meaningful improvements in multiple measures of cognition and behavior were presented for the first time in a scientific forum. |
| 2024-09-03 | Stoke Therapeutics, Inc | Results Stoke Therapeutics to Present Encore Data at the 15th European Epilepsy Congress that Demonstrate the Potential for Zorevunersen to be the First Disease-Modifying Medicine for the Treatment of Dravet Syndrome stoketherapeutics.com ↗
First presentation of landmark data from Phase 1/2a and OLE studies of zorevunersen that showed substantial reductions in seizures and continued improvements in multiple measures of cognition and behavior |
| 2024-08-07 | Stoke Therapeutics, Inc | Regulatory Stoke Therapeutics Reports Second Quarter Financial Results and Provides Business Updates stoketherapeutics.com ↗
FDA removes Partial Clinical Hold as company advances toward a Phase 3 registrational study of zorevunersen (STK-001) in children and adolescents with Dravet syndrome |
| 2024-03-25 | Stoke Therapeutics, Inc | Results Stoke Therapeutics Announces Landmark New Data That Support the Potential for STK-001 to be the First Disease-Modifying Medicine for the Treatment of Patients with Dravet Syndrome stoketherapeutics.com ↗
Data from these studies showed clinically meaningful effects, including substantial and durable reductions in convulsive seizure frequency and improvements in multiple measures of cognition and behavior that support the potential for disease modification. |
| 2023-12-01 | Stoke Therapeutics, Inc | Results Stoke Therapeutics Presents Data From Multiple Studies of Children and Adolescents With Dravet Syndrome at the American Epilepsy Society (AES) 2023 Annual Meeting stoketherapeutics.com ↗
The substantial and sustained reductions in seizure frequency and improvements in cognition and behavior observed in our STK-001 clinical studies give us confidence that we are addressing the root cause of Dravet syndrome. |
| 2023-11-21 | Stoke Therapeutics, Inc | Results Stoke Therapeutics to Present Data from the Company’s Dravet Syndrome Clinical Program at the American Epilepsy Society 2023 Annual Meeting stoketherapeutics.com ↗
Additional presentations include data that support advancement of STK-001 as potentially the first disease-modifying new medicine for Dravet syndrome, including data showing substantial reductions in seizures and improvements in cognition and behavior as well as pharmacokinetic modeling |
| 2023-09-05 | Stoke Therapeutics, Inc | Results Stoke Therapeutics Presents Data Related to the Ongoing Clinical Development of STK-001 for the Treatment of Dravet Syndrome at the 35th International Epilepsy Congress stoketherapeutics.com ↗
In addition, a new pharmacokinetic (PK) analysis of 61 patients treated in STK-001 clinical trials is being presented for the first time, and demonstrates a correlation between higher STK-001 drug exposure in brain and greater reductions in seizure frequency over time. |
| 2023-07-25 | Stoke Therapeutics, Inc | Results Stoke Therapeutics Announces Positive New Safety & Efficacy Data from Patients Treated with STK-001 in the Phase 1/2a Studies (MONARCH & ADMIRAL) and the SWALLOWTAIL Open-Label Extension (OLE) Study in Children and Adolescents with Dravet Syndrome stoketherapeutics.com ↗ |
| 2023-03-15 | Stoke Therapeutics, Inc | Regulatory Stoke Therapeutics Announces That FDA Will Allow the Administration of a Higher Single Dose of STK-001 (70mg) in the Ongoing Phase 1/2a MONARCH Study of Children and Adolescents with Dravet Syndrome stoketherapeutics.com ↗
today announced that the FDA will allow the administration of a higher single dose of STK-001 (70mg) in its ongoing Phase 1/2a MONARCH study of children and adolescents with Dravet syndrome. |
| 2022-12-02 | Stoke Therapeutics, Inc | Results Stoke Therapeutics Presents Data from a Combined Interim Analysis of the Phase 1/2a MONARCH and ADMIRAL Studies of STK-001 in Children and Adolescents with Dravet Syndrome at the American Epilepsy Society (AES) 2022 Annual Meeting stoketherapeutics.com ↗
55% median reduction from baseline in convulsive seizure frequency was observed among patients treated with three doses of 45mg (n=6) |
| 2022-11-29 | Stoke Therapeutics, Inc | Results Stoke Therapeutics to Present Data from the Company’s Dravet Syndrome Program at the American Epilepsy Society 2022 Annual Meeting stoketherapeutics.com ↗ |
| 2022-11-14 | Stoke Therapeutics, Inc | Results Stoke Therapeutics Reports Third Quarter Financial Results and Provides Business Updates stoketherapeutics.com ↗ |
| 2021-12-03 | Stoke Therapeutics, Inc | Results Stoke Therapeutics Presents Data from the Phase 1/2a MONARCH Study of STK-001 in Children and Adolescents with Dravet Syndrome at the American Epilepsy Society (AES) 2021 Annual Meeting stoketherapeutics.com ↗
70.6% (12/17) of patients treated with STK-001 experienced a reduction from baseline in convulsive seizure frequency measured from Day 29 to Day 84 |
| 2021-09-21 | Stoke Therapeutics, Inc | Results Stoke Therapeutics Announces Positive Interim Safety, PK and CSF Exposure Data from the Phase 1/2a MONARCH Study of STK-001 in Children and Adolescents with Dravet Syndrome stoketherapeutics.com ↗
today announced positive safety, pharmacokinetic (PK) and cerebrospinal fluid (CSF) exposure data from a planned interim analysis of the multi-center, open-label Phase 1/2a MONARCH study of STK-001 in children and adolescents with Dravet syndrome. |
| 2021-03-30 | Stoke Therapeutics, Inc | Regulatory Stoke Therapeutics Announces MHRA Authorization to Initiate Phase 1/2a Clinical Trial of STK-001 for Dravet Syndrome in the United Kingdom stoketherapeutics.com ↗ |
| 2020-12-04 | Stoke Therapeutics, Inc | Results Stoke Therapeutics Announces Presentations Related to the Company’s Work to Advance STK-001, the First Potential New Medicine to Target the Underlying Cause of Dravet Syndrome at the American Epilepsy Society (AES) 2020 Annual Meeting stoketherapeutics.com ↗
New data in a mouse model of Dravet syndrome demonstrate that TANGO ASOs can help restore normal function of nerve cells which correlate to reductions in seizure frequency and extended survival |
| 2020-10-07 | Stoke Therapeutics, Inc | Regulatory Stoke Therapeutics Announces Plans to Move Forward With Dosing of STK-001 in Children and Adolescents in its Ongoing Phase 1/2a MONARCH Study for Dravet Syndrome stoketherapeutics.com ↗
Dosing above 30 mg in this study remains on FDA partial clinical hold. |
| 2020-08-26 | Stoke Therapeutics, Inc | Results Stoke Therapeutics Announces Publication of Preclinical Data on STK-001 in the Journal Science Translational Medicine that Demonstrate Significant Improvements in Survival and Reductions in Seizure Frequency in a Dravet Syndrome Mouse Model stoketherapeutics.com ↗
Data published today in the journal Science Translational Medicine also showed that STK-001 achieved target engagement, pharmacologic activity and efficacy by selectively increasing Scn1a gene and Na v 1.1 protein expression. |
| 2020-03-23 | Stoke Therapeutics, Inc | Regulatory Stoke Therapeutics Reports Full Year 2019 Financial Results and Provides Business Updates stoketherapeutics.com ↗
Company receives FDA clearance to begin dosing STK-001 in Part A of Phase 1/2a “Monarch” clinical trial in Dravet syndrome; enrollment and dosing expected to start in 2H 2020 |
| 2019-12-08 | Stoke Therapeutics, Inc | Results Stoke Therapeutics Presents Preclinical Data on the Biodistribution, Target Engagement and Safety of STK-001 in Non-Human Primates That Support the Planned Clinical Development of STK-001 for the Treatment of Dravet Syndrome stoketherapeutics.com ↗
Data from studies in non-human primates (NHP) showed STK-001 distributed throughout the brain and achieved target engagement and increased Na v 1.1 protein expression throughout the cortex after a single intrathecal injection. |
| 2019-12-07 | Stoke Therapeutics, Inc | Results Stoke Therapeutics Presents Preclinical Data From Studies of STK-001 That Showed Improvements in Survival and Reductions in Seizure Frequency in a Mouse Model of Dravet Syndrome stoketherapeutics.com ↗
New data from electroencephalography (EEG) recordings showed 76% (16/21) of DS mice treated with STK-001 were seizure free compared to 48% (10/21) that were treated with a placebo. |
| 2019-08-06 | Stoke Therapeutics, Inc | Regulatory Stoke Therapeutics Granted FDA Orphan Drug Designation for STK-001, an Investigational New Treatment for Dravet Syndrome stoketherapeutics.com ↗
today announced that the U.S. Food and Drug Administration ( FDA ) has granted orphan drug designation to its lead product candidate, STK-001, an investigational new treatment for Dravet syndrome. |
All press releases naming this drug 50 releases
Evidence & citations 6 cited values
Every value below carries the sentence it was read from. 3 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | zorevunersen | ClinicalTrials.gov intervention name — accepted as the source's own label NCT04740476 ↗ |
| Known as | STK-001 | “Evaluating the efficacy, safety, and tolerability of Zorevunersen (STK-001) in Patients With Dravet Syndrome” NCT06872125 ↗ |
| Action | Restore | “STK-001 is designed to upregulate Nav1.1 protein expression from the nonmutant (wild-type) copy of the SCN1A gene to restore physiological Nav1.1 levels.” NCT04442295 ↗ |
| Modality | Antisense oligonucleotide | “STK-001 is an antisense oligonucleotide (ASO)” NCT04442295 ↗ |
| Route | Intrathecal | “administered as an intrathecal injection” NCT04442295 ↗ |
| Target | SCN1A | “STK-001 is designed to upregulate Nav1.1 protein expression from the nonmutant (wild-type) copy of the SCN1A gene” NCT04442295 ↗ |