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ION 260

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Drug Profile

ION 260

Alternative Names: BIIB-132; ION-260

Latest Information Update: 21 Feb 2024

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At a glance

  • Originator Ionis Pharmaceuticals
  • Class Antisense oligonucleotides
  • Mechanism of Action Ataxin 3 expression inhibitors; RNA interference
  • Orphan Drug Status

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

    No
  • New Molecular Entity Yes
  • Available For Licensing Yes

Highest Development Phases

  • Discontinued Machado-Joseph Disease

Most Recent Events

  • 21 Feb 2024 Discontinued - Phase-I for Machado-Joseph Disease (In the elderly, In adults) in Israel, Netherlands, Portugal, United Kingdom, Germany, USA (Intrathecal) (Ionis Pharmaceuticals pipeline, February 2024)
  • 25 Jul 2023 Biogen terminates a phase-I trial in Machado-Joseph Disease (In the elderly, In adults) in USA, United Kingdom, Portugal, Netherlands, Israel, Germany (Intrathecal) (NCT05160558)
  • 02 Feb 2022 Phase-I clinical trials in Machado-Joseph Disease (In adults, In the elderly) in USA (Intrathecal) (NCT05160558) (EudraCT2021-002223-37)

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