Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
SAD: WVE-003 · 1 trial · 1 indication
The primary outcome for this study was safety and is reported as the proportion of patients with TEAEs related to study drug.
| Arm | Type | Description |
|---|---|---|
| SAD: Pooled Placebo | PLACEBO_COMPARATOR | Placebo |
| SAD: 30mg WVE-003 | EXPERIMENTAL | Single Ascending Dose - 30mg WVE-003 |
| SAD: 60mg WVE-003 | EXPERIMENTAL | Single Ascending Dose - 60mg WVE-003 |
| SAD: 90mg WVE-003 | EXPERIMENTAL | Single Ascending Dose - 90mg WVE-003 |
| MD: Placebo | PLACEBO_COMPARATOR | Placebo |
| MD: 30mg WVE-003 | EXPERIMENTAL | Multiple Dose - 30mg WVE-003 |
| Name | Type | Description |
|---|---|---|
| SAD: 30mg WVE-003 | DRUG | Single ascending dose of 30mg WVE-003, an allele-selective stereopure antisense oligonucleotide (ASO) |
| SAD: 60mg WVE-003 | DRUG | Single ascending dose of 60mg WVE-003, an allele-selective stereopure antisense oligonucleotide (ASO) |
| SAD: 90mg WVE-003 | DRUG | Single ascending dose of 90mg WVE-003, an allele-selective stereopure antisense oligonucleotide (ASO) |
| SAD: Pooled Placebo | DRUG | Single dose of placebo |
| MD: 30mg WVE-003 | DRUG | Three doses of 30mg WVE-003 Q8WK an allele-selective stereopure, antisense oligonucleotide (ASO) |
| MD: Placebo | DRUG | Three doses of placebo Q8WK |
Inclusion Criteria: 1. Presence of the A variant of SNP3 on the same allele as the pathogenic CAG triplet expansion 2. Ambulatory, male or female patients aged ≥25 to ≤60 years 3. Clinical diagnostic motor features of HD, defined as Unified Huntington's Disease Rating Scale (UHDRS) Diagnostic Confi...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Novartis AG Sponsored ADR | NVS | 2 | PHASE3 | Votoplam |
| Neurocrine Biosciences, Inc. | NBIX | 1 | PHASE3 | Valbenazine |
| Alnylam Pharmaceuticals, Inc | ALNY | 1 | PHASE1 | ALN-HTT02 |
| uniQure N.V. | QURE | 2 | PHASE1 | AMT-130 |
| Sarepta Therapeutics, Inc. | SRPT | 1 | PHASE1 | SRP-1005 |
SAD: WVE-003 is an investigational small molecule developed by Wave Life Sciences Ltd. (ticker: WVE) for the treatment of Huntington's disease. It is a single-ascending-dose (SAD) formulation, specifically the 30 mg dose, and has been studied in a Phase 1 clinical trial.
SAD: WVE-003 is being developed for the treatment of Huntington's disease, a progressive neurological disorder. It is an investigational therapy and has not been approved for any indication. The Phase 1 study evaluated the 30 mg single-ascending dose in patients with Huntington's disease.
SAD: WVE-003 is being developed by Wave Life Sciences Ltd., a biotechnology company whose ticker symbol is WVE. Wave Life Sciences is responsible for the clinical development of this investigational small molecule for Huntington's disease.
SAD: WVE-003 is in Phase 1 clinical development. The single Phase 1 trial, NCT05032196, has been completed. The drug is investigational and has not been approved by the FDA or any regulatory agency for Huntington's disease.
SAD: WVE-003 has been studied in one completed Phase 1 clinical trial, NCT05032196, titled "Study of WVE-003 in Patients With Huntington's Disease." The trial enrolled 47 participants across Australia, Canada, Denmark, France, Germany, Italy, Netherlands, Poland, Spain, and the United Kingdom.