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Also known as ION582
obudanersen · 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...
Obudanersen is an investigational RNA therapy being developed by Ionis Pharmaceuticals, Inc. for Angelman Syndrome, a rare neurogenetic disorder. It is an antisense oligonucleotide designed to target UBE3A-ATS and is currently in Phase 3 clinical development. It has not been approved by any regulatory agency.
Obudanersen is being studied as a potential treatment for Angelman Syndrome, a rare disease caused by loss of function of the maternal UBE3A gene. The clinical program enrolls children and adults aged 2 years and older. It remains investigational and is not approved for any indication.
Obudanersen targets UBE3A-ATS, a long non-coding antisense transcript that silences the paternal copy of the UBE3A gene. By targeting UBE3A-ATS, the drug is designed to unsilence paternal UBE3A expression. Obudanersen is an antisense oligonucleotide, part of the -sen chemical class.
Obudanersen is being developed by Ionis Pharmaceuticals, Inc., which trades on the Nasdaq under the ticker IONS. Ionis is the sponsor of the Phase 3 clinical program evaluating the drug in Angelman Syndrome.
Obudanersen is in Phase 3 clinical development for Angelman Syndrome. It is an investigational therapy and has not been approved by the FDA or any other regulatory authority. Two Phase 3 trials are ongoing, and no trials have been completed to date.
Obudanersen is being evaluated in two Phase 3 trials in Angelman Syndrome. The CHAMPION study, NCT07782827, is recruiting in the United States with an enrollment of 30. The REVEAL study, NCT06914609, is active but not recruiting across the United States, Australia, Canada, Germany, Israel, Italy, Japan, Poland, Singapore, South Korea, Spain, and the United Kingdom, with 156 participants.
Yes, obudanersen is also known as ION582. Both names refer to the same investigational antisense oligonucleotide developed by Ionis Pharmaceuticals for Angelman Syndrome. The Phase 3 trials list the drug under the ION582 designation in their study titles.