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ION 541

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Drug Profile

ION 541

Alternative Names: BIIB-105; ION-541

Latest Information Update: 22 May 2024

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At a glance

  • Originator Ionis Pharmaceuticals
  • Developer Biogen; Ionis Pharmaceuticals
  • Class Antisense oligonucleotides; Biological factors; Small molecules
  • Mechanism of Action Ataxin-2 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

Highest Development Phases

  • Discontinued Amyotrophic lateral sclerosis

Most Recent Events

  • 16 May 2024 Discontinued - Phase-II for Amyotrophic lateral sclerosis in Italy, Netherlands, Canada, USA (Intrathecal) based upon the results from the phase I/II ALSpire trial
  • 16 May 2024 Adverse events and efficacy data from a phase I/II trial in Amyotrophic lateral sclerosis released by Biogen
  • 01 Oct 2022 Phase-II clinical trials in Amyotrophic lateral sclerosis in Italy (Intrathecal), before March 2023 (NCT04494256)

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