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ION464 · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| Part 1: ION464 | EXPERIMENTAL | ION464 will be administered at multiple-ascending doses by IT injection at regular intervals over 12 weeks. |
| Part 1: Placebo | PLACEBO_COMPARATOR | ION464-matching placebo will be administered by IT injection at regular intervals over 12 weeks. |
| Part 2: ION464 | EXPERIMENTAL | ION464 will be administered at the same doses as Part 1 by IT injection, at regular intervals, for 72 weeks. |
| Part 2: Placebo | PLACEBO_COMPARATOR | ION464-matching placebo will be administered by IT injection, at regular intervals, for 72 weeks. |
| Name | Type | Description |
|---|---|---|
| ION464 | DRUG | ION464 will be administered by IT injection. |
| Placebo | DRUG | ION464-matching placebo will be administered by IT injection. |
Key Inclusion Criteria: * Screening single-photon emission computed tomography (SPECT) with DaTscan™ (ioflupane I123 injection) results demonstrating loss (whether symmetric or asymmetric) of dopamine nerve terminals in the striatum consistent with neurodegenerative parkinsonism, as assessed with q...
ION464 is an investigational small molecule being developed for the treatment of Multiple System Atrophy (MSA), a rare neurodegenerative disorder. It is currently in Phase 1 clinical development, meaning it has not yet been approved by regulatory authorities. The drug is being studied to evaluate its safety, tolerability, and pharmacokinetics in adults with MSA.
ION464 targets SNCA, the gene that encodes the alpha-synuclein protein. Abnormal accumulation of alpha-synuclein is a hallmark of Multiple System Atrophy. By targeting SNCA, ION464 aims to address the underlying pathology of the disease, though its exact mechanism of action is still under investigation in clinical trials.
ION464 is being developed by Ionis Pharmaceuticals, Inc., a biopharmaceutical company traded on NASDAQ under the ticker symbol IONS. Ionis is conducting the clinical development program for ION464, which is currently in Phase 1 trials for Multiple System Atrophy.
ION464 is in Phase 1 clinical development. It is an investigational drug and has not been approved by the FDA or any other regulatory agency. The ongoing Phase 1 study is designed to assess the safety, tolerability, and pharmacokinetics of ION464 in adults with Multiple System Atrophy.
ION464 is being evaluated in a Phase 1 clinical trial with the identifier NCT04165486, known as the HORIZON study. This randomized, double-blind, placebo-controlled trial is recruiting 40 adults with Multiple System Atrophy across Austria, France, Germany, Portugal, and the United Kingdom. The study is currently active and recruiting participants.