Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
ISIS 443139 · 1 trial · 1 indication
An adverse event (AE) was any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the study or use of investigational drug product, whether or not the AE was considered related to the investigational drug product. An AE was to be regarded as a TEAE if it was present prior to receiving the first dose of Study Drug and subsequently worsened or was not present prior to receiving the first dose of Study Drug but subsequently appeared.
| Arm | Type | Description |
|---|---|---|
| ISIS 443139 10 mg | EXPERIMENTAL | Participants received ISIS 443139, 10 milligrams (mg), by intrathecal injection, on Study Days 1, 29, 57, and 85. |
| ISIS 443139 30 mg | EXPERIMENTAL | Participants received ISIS 443139, 30 mg, by intrathecal injection, on Study Days 1, 29, 57, and 85. |
| ISIS 443139 60 mg | EXPERIMENTAL | Participants received ISIS 443139, 60 mg, by intrathecal injection, on Study Days 1, 29, 57, and 85. |
| ISIS 443139 90 mg | EXPERIMENTAL | Participants received ISIS 443139, 90 mg, by intrathecal injection, on Study Days 1, 29, 57, and 85. |
| ISIS 443139 120 mg | EXPERIMENTAL | Participants received ISIS 443139, 120 mg, by intrathecal injection, on Study Days 1, 29, 57, and 85. |
| Placebo | PLACEBO_COMPARATOR | Participants received placebo, by intrathecal injection, on Study Days 1, 29, 57, and 85. |
| Name | Type | Description |
|---|---|---|
| ISIS 443139 10 mg | DRUG | ISIS 443139, 10 mg, was administered by intrathecal injection, on Study Days 1, 29, 57, and 85. |
| ISIS 443139 30 mg | DRUG | ISIS 443139, 30 mg, was administered by intrathecal injection, on Study Days 1, 29, 57, and 85. |
| ISIS 443139 60 mg | DRUG | ISIS 443139, 60 mg, was administered by intrathecal injection, on Study Days 1, 29, 57, and 85. |
| ISIS 443139 90 mg | DRUG | ISIS 443139, 90 mg, was administered by intrathecal injection, on Study Days 1, 29, 57, and 85. |
| ISIS 443139 120 mg | DRUG | ISIS 443139, 120 mg, was administered by intrathecal injection, on Study Days 1, 29, 57, and 85. |
| Placebo | OTHER | Placebo was administered by intrathecal injection, on Study Days 1, 29, 57, and 85. |
Key Inclusion Criteria: * Diagnosed with early manifest Huntington's disease * Male or female, aged 25 to 65 years, inclusive, at the time of informed consent * Able and willing to meet all study requirements, including travel to Study Center and participation in all procedures and measurements at ...
| 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 |
ISIS 443139 is an investigational small molecule being developed for the treatment of Huntington's Disease. It is intended for participants with early manifest Huntington's Disease, as studied in a Phase 1 clinical trial. The drug is not yet approved and remains in clinical development.
ISIS 443139 is being developed by Ionis Pharmaceuticals, Inc., a biopharmaceutical company traded on NASDAQ under the ticker symbol IONS. The company conducted a Phase 1 clinical trial to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of the drug in participants with early manifest Huntington's Disease.
ISIS 443139 is in Phase 1 clinical development. A Phase 1 trial has been completed, evaluating the drug in 46 participants with early manifest Huntington's Disease. The drug is investigational and has not been approved by regulatory authorities.
ISIS 443139 has one completed clinical trial, registered as NCT02519036. This Phase 1 study assessed safety, tolerability, pharmacokinetics, and pharmacodynamics in 46 participants with early manifest Huntington's Disease. The trial was randomized, double-blind, and placebo-controlled, conducted in Canada, Germany, and the United Kingdom.
ISIS 443139 is the name used in clinical development for this investigational drug. No alternative names have been reported for this compound. It is being studied specifically for Huntington's Disease and is not known to be marketed under any other name.