Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT05032196 | Study of WVE-003 in Patients With Huntington's Disease | PHASE1 | COMPLETED | 47 | — | — | Sep 6, 2021 | May 24, 2024 | Aug 12, 2025 | 23 | Australia, Canada +8 |
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 | 2 | PHASE3 | Valbenazine |
| Alnylam Pharmaceuticals, Inc | ALNY | 1 | PHASE1 | ALN-HTT02 |
| uniQure N.V. | QURE | 2 | PHASE1 | intra-striatal rAAV5-miHTT |
| Sarepta Therapeutics, Inc. | SRPT | 1 | PHASE1 | SRP-1005 |