Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
WVE-210201 · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| WVE-210201 (Dose A) or placebo | EXPERIMENTAL | - |
| WVE-210201 (Dose B) or placebo | EXPERIMENTAL | - |
| WVE-210201 (Dose C) or placebo | EXPERIMENTAL | - |
| WVE-210201 (Dose D) or placebo | EXPERIMENTAL | - |
| WVE-210201 (Dose E) or placebo | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| WVE-210201 | DRUG | WVE-210201 is a stereopure antisense oligonucleotide (ASO) |
| Placebo | DRUG | Sodium Chloride |
Inclusion Criteria: * Diagnosis of Duchenne muscular dystrophy (DMD) based on clinical phenotype with increased serum creatine kinase * Documented mutation in the Dystrophin gene associated with DMD that is amenable to exon 51 skipping * Ambulatory or non-ambulatory male patients aged ≥5 - ≤18 year...
WVE-210201 is an investigational small molecule developed by Wave Life Sciences Ltd. for Duchenne Muscular Dystrophy, a genetic muscle-wasting disorder. It is designed to target exon 51, a specific segment of the dystrophin gene, and was studied in a Phase 1 clinical trial. It is not an approved therapy.
WVE-210201 is being developed for Duchenne Muscular Dystrophy, a neuromuscular condition that causes progressive muscle weakness. The program targets patients whose disease involves exon 51, making it a biomarker-selected approach. The drug has completed a Phase 1 trial and remains investigational rather than approved for patient use.
WVE-210201 targets exon 51, a defined segment of the dystrophin gene, placing it in the exon-targeting class of therapies. By addressing exon 51, the molecule is intended for Duchenne Muscular Dystrophy patients with that specific genetic profile. The Phase 1 study enrolled only biomarker-selected participants.
WVE-210201 is developed by Wave Life Sciences Ltd., a biotechnology company whose shares trade under the ticker WVE. Wave Life Sciences sponsored the Phase 1 trial of the drug in Duchenne Muscular Dystrophy. The company is the sole developer named for this program.
WVE-210201 is in Phase 1 development. Its single clinical trial, NCT03508947, has been completed, and no trials are currently active. The study was randomized, double-blind, and controlled, and it enrolled 36 male participants aged 5 years and older with Duchenne Muscular Dystrophy.
WVE-210201 was evaluated in one clinical trial, NCT03508947, titled Safety and Tolerability of WVE-210201 in Patients With Duchenne Muscular Dystrophy. The Phase 1 study is completed and enrolled 36 participants across the United States, Belgium, Canada, France, Italy, the Netherlands, and the United Kingdom. It did not include healthy volunteers.