Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
ION582 · 1 trial · 1 indication
The safety and tolerability of ION582 will be assessed by determining the incidence, severity, and dose relationship of adverse effects and changes in the laboratory parameters by dose.
| Arm | Type | Description |
|---|---|---|
| Part 1 MAD: Cohort A | EXPERIMENTAL | ION582 will be administered as IT injection over a period of 13 weeks, with a minimum of approximately 4 weeks between each dose administration. |
| Part 1 MAD: Cohort B | EXPERIMENTAL | ION582 will be administered as IT injection over a period of 13 weeks, with a minimum of approximately 4 weeks between each dose administration. |
| Part 1 MAD: Cohort C | EXPERIMENTAL | ION582 will be administered as IT injection over a period of 13 weeks, with a minimum of approximately 4 weeks between each dose administration. |
| Part 1 MAD: Cohort D | EXPERIMENTAL | ION582 will be administered as IT injection over a period of 13 weeks, with a minimum of approximately 4 weeks between each dose administration. |
| Part 1 MAD: Cohort E | EXPERIMENTAL | ION582 will be administered as IT injection of over a period of 13 weeks, with a minimum of approximately 4 weeks between each dose administration. |
| Part 1 MAD: Cohort F | EXPERIMENTAL | ION582 will be administered as IT injection over a period of 13 weeks, with a minimum of approximately 12 weeks between each dose administration. |
| Part 2 Group 1 | EXPERIMENTAL | ION582 will be administered as IT injection of over a period of 49 weeks, with additional dosing intervals. |
| Part 2 Group 2 | EXPERIMENTAL | ION582 will be administered as IT injection of over a period of 49 weeks, with additional dosing intervals. |
| Part 3 Group 1 | EXPERIMENTAL | ION582 will be administered as IT injection of over a period of 145 weeks, with additional dosing intervals. |
| Part 3 Group 2 | EXPERIMENTAL | ION582 will be administered as IT injection of over a period of 145 weeks, with additional dosing intervals. |
| Name | Type | Description |
|---|---|---|
| ION582 | DRUG | ION582 will be administered by IT injection. |
Key Inclusion Criteria: 1. Participant has a documented and certified diagnosis of Angelman syndrome (AS) (ubiquitin-protein ligase E3A \[UBE3A\] deletion or UBE3A mutation) 2. Male or female between the ages of 0-50 years of age, with signed informed consent from parent(s) or legal guardian(s) 3. ...
ION582 is an investigational small molecule being developed for the treatment of Angelman Syndrome, a rare genetic disorder. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities. The drug is being studied in the HALOS trial, which is evaluating its safety, tolerability, pharmacokinetics, and pharmacodynamics in participants with Angelman Syndrome.
ION582 is being developed by Ionis Pharmaceuticals, Inc., a biopharmaceutical company traded on NASDAQ under the ticker symbol IONS. The company is conducting clinical trials to evaluate the drug's potential as a treatment for Angelman Syndrome, a rare disease. Ionis is responsible for the research, development, and regulatory activities associated with ION582.
ION582 is currently in Phase 1 clinical development. The drug is being studied in the HALOS trial, a Phase 1 study that is active but not recruiting participants. As an investigational drug, ION582 has not yet received regulatory approval and is still undergoing clinical evaluation for safety and efficacy in treating Angelman Syndrome.
ION582 is being evaluated in the HALOS trial, registered as NCT05127226. This Phase 1 study is a safety, tolerability, pharmacokinetics, and pharmacodynamics study of multiple ascending doses of ION582 in participants with Angelman Syndrome. The trial is active but not recruiting, with an enrollment of 71 participants across the United States, Australia, France, Israel, Italy, and the United Kingdom.
No, ION582 is not the same as HALOS. HALOS is the name of the clinical trial, registered as NCT05127226, that is studying ION582. The trial is a Phase 1 study evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of multiple ascending doses of ION582 in participants with Angelman Syndrome. ION582 is the investigational drug being tested in this trial.