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Also known as obudanersen
ION582 · 3 trials · 1 indication
The Bayley-4 is a performance-based assessment of developmental functioning across communication, cognition, and motor skills. The expressive communication subdomain of communication measures preverbal and verbal communication. The total raw score reflects the sum of all the item scores within the expressive communication subdomain, with higher scores reflecting greater expressive communication ability.
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 |
|---|---|---|
| obudanersen 80 mg | EXPERIMENTAL | Participants in Cohorts 1 and 2 will be administered obudanersen via intrathecal (IT) bolus injection every 12 weeks during the Treatment Evaluation and LTE periods. |
| Cohort 1 obudanersen 80 mg | EXPERIMENTAL | Participants (aged 2 to \<18 years old) will be administered obudanersen 80 mg via intrathecal (IT) injection every 12 weeks during the double blind and LTE treatment periods. |
| Cohort 1 Placebo | PLACEBO_COMPARATOR | Participants (aged 2 to \<18 years old) will be administered obudanersen matching placebo via IT injection every 12 weeks during the double-blind treatment period and then administered obudanersen 40 mg or 80 mg every 12 weeks during the LTE treatment period. |
| Cohort 2 obudanersen 80 mg | EXPERIMENTAL | Participants (aged 18 to ≤50 years old) will be administered obudanersen 80 mg via IT injection every 12 weeks during the double blind and LTE treatment periods. |
| Cohort 2 Placebo | PLACEBO_COMPARATOR | Participants (aged 18 to ≤50 years old) will be administered obudanersen matching placebo via IT injection every 12 weeks during the double-blind treatment period and then randomized to obudanersen 40 mg or 80 mg every 12 weeks during the LTE treatment period. |
| 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 |
|---|---|---|
| obudanersen | DRUG | ION582 will be administered by IT injection. |
| Placebo | DRUG | obudanersen matching placebo will be administered by IT injection. |
| ION582 | DRUG | ION582 will be administered by IT injection. |
Key Inclusion Criteria: 1. Clinical diagnosis of AS with molecular result indicating either paternal UPD of 15q11.2-q13 or ID of the maternal 15q11.2-q13 region, provided by the Investigator and confirmed by either a qualified central vendor or a qualified local geneticist at the site. 2. The parti...
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.
ION582 targets UBE3A-ATS, a long non-coding RNA that regulates the expression of the UBE3A gene. By targeting UBE3A-ATS, ION582 aims to address the underlying genetic cause of 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 safety and efficacy of ION582.
ION582 is in Phase 1 clinical development. It is an investigational drug and has not yet been approved by the FDA or other regulatory agencies. The ongoing Phase 1 trial is evaluating its safety, tolerability, pharmacokinetics, and pharmacodynamics.
ION582 is being studied in the HALOS trial (NCT05127226), a Phase 1 study of multiple ascending doses 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.