Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as zorevunersen (STK-001)
zorevunersen · 2 trials · 1 indication
Measured by daily seizure diary
Safety variables for analysis include the incidence, type, severity, and seriousness of AEs, and changes in vital signs, ECG, laboratory, immunogenicity, physical examination, and outcomes on the cerebellar function clinical screening battery.
| Arm | Type | Description |
|---|---|---|
| Zorevunersen | EXPERIMENTAL | Eligible patients will be randomly assigned in a 1:1 ratio to zorevunersen:sham in Treatment Period 1 (approximately 52 weeks). Upon the completion of Treatment Period 1 all eligible patients, will enter Treatment Period 2 and receive zorevunersen, regardless of initial treatment assignment. |
| Sham Comparator | SHAM_COMPARATOR | Eligible patients will be randomly assigned in a 1:1 ratio to zorevunersen:sham |
| zorevunersen (STK-001) multiple dose levels | EXPERIMENTAL | Enrollment of patients after completion of Study STK-001-DS-101 or Study STK-001-DS-102 if eligible. Patients will receive IT administration of study drug zorevunersen at the dose level they received while participating in Study STK-001-DS-101 or STK-001-DS-102, or at a dose level recommended by the Safety Monitoring Committee (SMC).The highest dose administered in this study may not exceed that which has already been evaluated in an zorevunersen Phase 1/2 study, and doses above 45 mg/dose in this study require approval from the Food and Drug Administration (FDA). Patients will initially receive 3 doses, one every approximately 4 months (16 weeks). Patients who are tolerating treatment may continue treatment with doses approximately every 4 months, with an End of Study/Follow-up Visit 24 weeks after the last dose of study drug. Patients who do not continue treatment after the third dose will have a Follow-up Visit (V5) at Week 48 and an End of Study Visit at Week 56. |
| Name | Type | Description |
|---|---|---|
| zorevunersen | DRUG | Treatment Period 1: Zorevunersen group will receive study drug by intrathecal (IT) administration on Day 1 (after the 8-week Baseline Period), Day 57 (Week 8), Day 169 (Week 24), and Day 281 (Week 40) at a dose level of 70 mg on Day 1 and Day 57, and 45 mg on Day 169 and Day 281. Treatment Period 2: Group assigned to zorevunersen in Treatment Period 1 will receive 45 mg of zorevunersen on Day 393 (Week 56), Day 477 (Week 68), and Day 589 (Week 84). |
| Sham Comparator | OTHER | Treatment Period 1: Sham group will not have drug administered. Sham group will have a procedure intended to mimic the drug administration. Treatment Period 2: Group assigned to sham in Treatment Period 1 will receive 70 mg of zorevunersen on Day 393 (Week 56) and on Day 477 (Week 68), and 45 mg of zorevunersen Day 589 (Week 84). |
| zorevunersen (STK-001) | DRUG | zorevunersen drug product is an antisense oligonucleotide administered as an intrathecal injection. |
Key Inclusion Criteria: 1. Patients must be ≥2 and \<18 years of age. 2. Patients must have a clinical diagnosis of DS confirmed by the Epilepsy Study Consortium, Inc. (ESCI) and as defined by: Onset, prior to 12 months (inclusive, \<13 months), of age, of recurrent focal with motor signs, hemi...
Zorevunersen is an investigational RNA therapy being developed for the treatment of Dravet syndrome, a severe form of epilepsy that begins in infancy. It is not yet approved for any indication and remains in clinical development.
Zorevunersen targets the SCN1A gene. It is an antisense oligonucleotide designed to increase expression of the SCN1A gene, which encodes a sodium channel protein. In Dravet syndrome, one copy of SCN1A is typically non-functional, and the therapy aims to boost output from the remaining functional copy.
Zorevunersen is being developed by Stoke Therapeutics, Inc., which trades under the ticker STOK. The company is the sponsor of the clinical program evaluating the drug in Dravet syndrome.
Zorevunersen is in Phase 3 clinical development. The Phase 3 trial is currently recruiting participants, and the program also includes an ongoing Phase 2 open-label extension study. The drug has not been approved by the FDA.
Zorevunersen is being evaluated in two clinical trials. The Phase 3 trial, NCT06872125, is a double-blind, sham-controlled study with an estimated enrollment of 170 patients across the United States, China, France, Germany, Italy, Japan, Spain, and the United Kingdom. The Phase 2 open-label extension study, NCT04740476, has an estimated enrollment of 60 patients in the United States.
Yes, zorevunersen is also known as STK-001. STK-001 was the earlier code name used for the same investigational antisense oligonucleotide developed by Stoke Therapeutics for Dravet syndrome.