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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 and has not 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 a Phase 1 clinical trial to evaluate the drug's safety, tolerability, and pharmacokinetics in adults with Multiple System Atrophy.
ION464 is in Phase 1 clinical development. It is an investigational drug and has not received regulatory approval. The ongoing Phase 1 trial is recruiting participants with Multiple System Atrophy to assess the drug's safety, tolerability, and how it is processed by the body.
ION464 is being studied in a Phase 1 clinical trial with the identifier NCT04165486, titled 'Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ION464 Administered to Adults With Multiple System Atrophy (HORIZON)'. The trial is recruiting 40 participants across Austria, France, Germany, Portugal, and the United Kingdom.
ION464 is a distinct investigational small molecule targeting SNCA, the gene for alpha-synuclein. It is not known to be the same as any other approved or investigational drug. Its development is specific to Multiple System Atrophy, and it is currently in Phase 1 trials to evaluate its safety and tolerability.