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 |
| Hoffmann-La Roche | RHHBY | 3 | PHASE2 | Tominersen, RG6496, SPK-10001 |
| Alnylam Pharmaceuticals, Inc | ALNY | 1 | PHASE1 | ALN-HTT02 |
| uniQure N.V. | QURE | 2 | PHASE1 | AMT-130 |
| Sarepta Therapeutics, Inc. | SRPT | 1 | PHASE1 | SRP-1005 |
ISIS 443139 is an investigational small molecule developed by Ionis Pharmaceuticals, Inc. for Huntington's disease. It was studied in a Phase 1 clinical trial in participants with early manifest Huntington's disease. The drug is also referred to as ISIS 443139 10 mg.
ISIS 443139 was developed for the treatment of Huntington's disease, a neurological disorder. Its clinical testing focused on participants with early manifest Huntington's disease. The program is investigational and has not been approved for any indication.
ISIS 443139 is developed by Ionis Pharmaceuticals, Inc., which trades on the Nasdaq under the ticker IONS. The company sponsored the Phase 1 study of the drug in Huntington's disease.
ISIS 443139 is in Phase 1 development. One Phase 1 trial has been completed, and no active trials are ongoing. The drug remains investigational and has not been approved by the FDA for Huntington's disease or any other indication.
ISIS 443139 was evaluated in one completed Phase 1 trial, NCT02519036, titled Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 443139 in Participants With Early Manifest Huntington's Disease. The randomized, double-blind, placebo-controlled study enrolled 46 participants across Canada, Germany, and the United Kingdom.