Drug Profile


Alternative Names: BIIB 065; IONIS-DMPK-2.5Rx; ISIS 598769; ISIS DMPKRx; ISIS-DMPK-2.5Rx

Latest Information Update: 15 Nov 2016

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At a glance

  • Originator Isis Pharmaceuticals
  • Developer Ionis Pharmaceuticals
  • Class Antisense oligonucleotides
  • Mechanism of Action RNA inhibitors
  • Orphan Drug Status

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

    Yes - Myotonic dystrophy
  • New Molecular Entity Yes

Highest Development Phases

  • Phase II Myotonic dystrophy

Most Recent Events

  • 01 Aug 2016 Ionis Pharmaceuticals completes a phase-I/II clinical trial in Myotonic dystrophy in USA (SC) (NCT02312011)
  • 15 Jun 2016 Phase-II clinical trials in Myotonic dystrophy in USA (SC) (Ionis Pharmaceuticals pipeline, June 2016)
  • 18 Dec 2015 Isis Pharmaceuticals is now called Ionis Pharmaceuticals
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