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ION 582

Drug Profile

ION 582

Alternative Names: BIIB-121; ION-582

Latest Information Update: 23 May 2025

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At a glance

  • Originator Ionis Pharmaceuticals
  • Class Antisense oligonucleotides
  • Mechanism of Action RNA interference; UBE3A protein expression stimulants
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Angelman syndrome
  • 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

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