ION 582
Alternative Names: BIIB-121; ION-582Latest Information Update: 23 May 2025
At a glance
- Originator Ionis Pharmaceuticals
- Class Antisense oligonucleotides
- Mechanism of Action RNA interference; UBE3A protein expression stimulants
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Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity Yes
Highest Development Phases
- Phase III Angelman syndrome
Most Recent Events
- 01 May 2025 Phase-III clinical trials in Angelman syndrome (In adolescents, In children, In adults) in USA (Intrathecal) (NCT06914609)
- 06 Apr 2025 Ionis Pharmaceuticals plans a phase III trial for Angelman syndrome in unknown location (Intrathecal, Injection) in May 2025 (NCT06914609)
- 07 Nov 2024 Ionis Pharmaceuticals announced positive End-of-Phase II discussion with FDA for ION 582 for Angelman syndrome in USA