Drugs / OBUDANERSEN SODIUM
Trials 2
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT05127226 | Dec 2021 → Mar 2029 expected | Angelman syndrome | Ionis Pharmaceuticals, Inc. | Recruiting | No outcome recorded |
| Phase 31 trial | ||||||
| Phase 3 | NCT06914609 | Jun 2025 → Aug 2027 expected | Angelman syndrome | Ionis Pharmaceuticals, Inc. | Recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 6
| Date | Issuer | Release |
|---|---|---|
| 2025-10-07 | Ionis Pharmaceuticals | Results Ionis showcases transformational medicines and industry-leading pipeline at Innovation Day ionis.com ↗
Ionis will also share new, positive 18-month long-term extension data from the Phase 2 HALOS study of ION582 for Angelman syndrome, a severe neurodevelopmental disorder with no approved disease-modifying treatments. |
| 2025-09-09 | Ionis Pharmaceuticals | Regulatory Ionis receives U.S. FDA Breakthrough Therapy designation for ION582 in Angelman syndrome ionis.com ↗
This Breakthrough Therapy designation is based on results from the Phase 1/2 HALOS study of ION582 that showed consistent and encouraging clinical improvement on all AS functional domains including communication, cognition and motor function, as well as favorable safety and tolerability. |
| 2024-07-22 | Ionis Pharmaceuticals | Results Ionis announces positive detailed results from the HALOS Study of ION582 in people with Angelman syndrome ionis.com ↗ |
| 2024-05-16 | Ionis Pharmaceuticals | Results Ionis announces positive topline results from Phase 1/2a trial of ION582 for Angelman syndrome ionis.com ↗
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced positive topline data from the HALOS Phase 1/2a open-label study of ION582 in Angelman syndrome. |
| 2023-11-11 | Ionis Pharmaceuticals | Results Ionis shares positive clinical update from ongoing trial of ION582 for Angelman syndrome ionis.com ↗
ION582 was generally well tolerated in the study and showed encouraging electroencephalogram (EEG) activity trends and early signals of positive clinical improvement, which will need to be confirmed upon analysis of the full data set in mid-2024. |
| 2022-06-13 | Ionis Pharmaceuticals | Regulatory Ionis treatment for Angelman syndrome receives orphan drug and rare pediatric disease designations from U.S. FDA ionis.com ↗ |
All press releases naming this drug 9 releases
Evidence & citations 3 cited values
Every value below carries the sentence it was read from. 3 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | OBUDANERSEN SODIUM | ChEMBL registry synonym — accepted as the source's own label CHEMBL6068612 ↗ |
| Known as | ION582 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT06914609 ↗ |
| Route | Intrathecal | “ION582 will be administered by IT injection.” NCT05127226 ↗ |