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obudanersen · 2 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.
| 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. |
| Name | Type | Description |
|---|---|---|
| obudanersen | DRUG | ION582 will be administered by IT injection. |
| Placebo | DRUG | obudanersen matching placebo 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 small molecule being developed for Angelman Syndrome, a rare genetic disorder. It is currently in Phase 3 clinical trials. The drug is designed to address the underlying cause of the condition, though the specific mechanism is not detailed here.
Obudanersen is being developed by Ionis Pharmaceuticals, Inc., a biopharmaceutical company traded on NASDAQ under the ticker symbol IONS. The company is conducting Phase 3 clinical trials to evaluate the drug's efficacy and safety in patients with Angelman Syndrome.
Obudanersen is in Phase 3 clinical development. It is not yet approved by regulatory authorities and remains investigational. Two Phase 3 trials are ongoing or planned, with a combined enrollment of 188 participants, to assess its effects in Angelman Syndrome.
Obudanersen is being studied in two Phase 3 trials. The REVEAL study (NCT06914609) is recruiting 158 participants across multiple countries, while the CHAMPION study (NCT07782827) is not yet recruiting and plans to enroll 30 participants. Both trials are placebo-controlled and double-blind.
Obudanersen is also known as ION582, as indicated in the clinical trial titles. Both names refer to the same investigational drug being developed by Ionis Pharmaceuticals for Angelman Syndrome. The trials use the ION582 designation in their official titles.