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Also known as Votoplam (blinded)
Votoplam · 2 trials · 1 indication
The Composite Unified Huntingtons Disease Ratings Scale (cUHDRS) is an improved composite measure to assess multi-domain clinical progression in HD. The cUHDRS is a combined weighted score of measures of motor function (TMS), cognition (SDMT and SWRT) and overall functional capacity (TFC): cUHDRS = \[(TFC-10.4)/1.9 - (TMS-29.7)/14.9 + (SDMT-28.4)/11.3 + (SWRT - 66.1)/20.1\] + 10. In conjunction with one another, these measures provide a comprehensive, sensitive and specific tool for monitoring disease progression, with lower scores indicating more severe disease. cUHDRS score range from -8 to 25.
| Arm | Type | Description |
|---|---|---|
| Votoplam | EXPERIMENTAL | Votoplam (blinded) taken orally, randomized in a 3:2 ratio (Votoplam: Placebo) |
| Placebo | PLACEBO_COMPARATOR | Placebo (blinded) taken orally, randomized in a 3:2 ratio (Votoplam: Placebo) |
| Votoplam 5 mg | EXPERIMENTAL | Participants will receive Votoplam 5 mg tablets once daily orally for 48 months. |
| Votoplam 10 mg | EXPERIMENTAL | Participants will receive votoplam 10 mg tablets once daily orally for 48 months. |
| Name | Type | Description |
|---|---|---|
| Votoplam (blinded) | DRUG | Votoplam (blinded) active treatment |
| Placebo | DRUG | Placebo |
| Votoplam | DRUG | Votoplam will be administered per dose and schedule specified in the arm. |
Inclusion Criteria: * Signed informed consents must be obtained prior to participation in the study * Ambulatory male or female participants between 21 to 70 years of age, inclusive, on the day of Informed Consent signature * Genetically confirmed HD diagnosis with a cytosine-adenine-guanine (CAG) ...
| 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 |
Votoplam is an investigational small molecule being developed for Huntington's disease. It is a blinded study drug in clinical trials evaluating its efficacy, safety, and tolerability in participants with this condition. The drug is not yet approved and remains in clinical development.
Votoplam is being developed by Novartis AG, a global pharmaceutical company listed on the stock exchange under the ticker NVS. Novartis is conducting clinical trials to evaluate the drug's potential as a treatment for Huntington's disease.
Votoplam is currently in Phase 3 clinical development for Huntington's disease. A Phase 3 study is actively recruiting participants, and a separate extension study is ongoing to evaluate long-term safety and efficacy. The drug is investigational and has not been approved.
Votoplam is being studied in two clinical trials. NCT07326709 is a Phase 3 study investigating efficacy, safety, and tolerability in Huntington's disease, currently recruiting 770 participants. NCT06254482 is a Phase 2 extension study evaluating long-term safety and efficacy in 144 participants.
Yes, Votoplam is also known as Votoplam (blinded). The blinded designation indicates that in clinical trials, neither the participants nor the investigators know whether they are receiving the active drug or a placebo, which helps ensure unbiased results.
Votoplam is a small molecule being studied for Huntington's disease. Its specific molecular target has not been disclosed in the available clinical trial information. The ongoing studies are designed to determine its efficacy, safety, and tolerability in affected individuals.