Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
SKY-0515 · 1 trial · 1 indication
cUHDRS includes the Total Functional Capacity (range, 0-13; higher score means better functioning), Total Motor Score (range, 0-124; higher score means worse motor severity), Symbol Digit Modality Test (range, 0-110, correctly paired numbers-symbols in 90 seconds; higher score means better cognitive performance), and Stroop Color and Word Test (range, 0-no max value, correctly read color words in 45 seconds; higher score means better cognitive performance) scores. A z-score for each test is calculated, which alone can be used to describe relationship between an individual's test score and the mean score of a target population. A z-score of 0 is the mean, and ±1 is 1 standard deviation from the mean. For cUHDRS, z-scores of each test are summed, whereby a higher cUHDRS score is better (score of -3.06-no max value) and a change of ≥1.2 is a meaningful worsening, shown to track functional decline.
| Arm | Type | Description |
|---|---|---|
| Active Comparator (1) | ACTIVE_COMPARATOR | Dosage Level(s): Low Dose |
| Active Comparator (2) | ACTIVE_COMPARATOR | Dosage Level(s): Mid Dose |
| Active Comparator (3) | ACTIVE_COMPARATOR | Dosage Level(s): High Dose |
| Placebo Comparator (4) | PLACEBO_COMPARATOR | Matching placebo once daily oral |
| Name | Type | Description |
|---|---|---|
| SKY-0515 | DRUG | Type: Small molecule Dosage Level(s): Low, mid, high Route of Administration: Oral Dosage Frequency: Once daily Blinded Treatment Duration: 18 months Use: Experimental |
| SKY-0515 Placebo | DRUG | Route of Administration: Oral Dosage Frequency: Once daily Blinded Treatment Duration: 18 months Use: Experimental |
Inclusion Criteria: * 25 years or older. * Huntington's Disease confirmed through genetic testing, with a specific change in exon 1 of the HTT gene (CAG repeat of 40 or more). * Total Functional Capacity (TFC) score of 10 or more). * Total Motor Score (TMS) of 6 or more). * Independence Score (IS) ...
| 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 | intra-striatal rAAV5-miHTT |
| Sarepta Therapeutics, Inc. | SRPT | 1 | PHASE1 | SRP-1005 |