Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
WVE-006 · 2 trials · 2 indications
| Arm | Type | Description |
|---|---|---|
| WVE-006 (Dose 1) | EXPERIMENTAL | - |
| WVE-006 (Dose 2) | EXPERIMENTAL | - |
| WVE-006 (Dose 3) | EXPERIMENTAL | - |
| WVE-006 (Dose 4) | EXPERIMENTAL | - |
| WVE-006 (Dose 5) | EXPERIMENTAL | - |
| Single Ascending Dose (SAD): WVE-006 30 milligram (mg) or placebo | EXPERIMENTAL | - |
| SAD: WVE-006 100 mg or placebo | EXPERIMENTAL | - |
| SAD WVE-006 200 mg or placebo | EXPERIMENTAL | - |
| SAD: WVE-006 400 mg or placebo | EXPERIMENTAL | - |
| SAD: WVE-006 600 mg or placebo | EXPERIMENTAL | - |
| Multiple Dose: WVE-006 600 mg Every 2 weeks (Q2W) or placebo | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| WVE-006 | DRUG | RNA editing oligonucleotide |
Inclusion Criteria: * Healthy as determined by the Investigator, based on a medical evaluation, or has mild to moderate AATD-induced lung disease (FEV1 ≥50%) and/or stable mild AATD-induced liver disease (≤F2 (≤10 kPa) on FibroScan. * Genetic testing confirming Pi\*ZZ. * Participant has been a non-...
WVE-006 is an investigational small molecule being developed by Wave Life Sciences for alpha-1 antitrypsin deficiency (AATD), a rare genetic disease. It is designed to target the AAT protein and is currently in Phase 1 clinical development. It has received an Accelerated Approval designation from the FDA.
WVE-006 is being developed as a treatment for alpha-1 antitrypsin deficiency, a rare inherited condition. Development is focused on participants with the Pi*ZZ genotype, the form of AATD studied in the ongoing Phase 1b/2a trial. It remains investigational and has not been approved for any use.
WVE-006 targets AAT, the alpha-1 antitrypsin protein. AAT is a protein target, and the drug is a small molecule. In AATD, the goal of targeting AAT is to address the deficient or abnormal alpha-1 antitrypsin protein that defines the condition.
WVE-006 is being developed by Wave Life Sciences Ltd., which trades on the Nasdaq under the ticker WVE. The company is running the clinical program for the asset in alpha-1 antitrypsin deficiency.
WVE-006 is in Phase 1 clinical development. It is investigational and has not been approved by the FDA. Two trials are recorded for the program: one completed Phase 1 study and one active Phase 1b/2a study that is no longer recruiting participants.
WVE-006 has been studied in two trials. NCT06186492, also called RestorAATion-1, was a completed Phase 1 study of 47 healthy participants with wild-type AAT expression in the United Kingdom. NCT06405633 is an active Phase 1b/2a study of 24 participants with Pi*ZZ AATD across Australia, Canada, Finland, Germany, New Zealand, and the United Kingdom.