IONIS HTTRx - Ionis Pharmaceuticals

Drug Profile

IONIS HTTRx - Ionis Pharmaceuticals

Alternative Names: HTT ASO; IONIS-HTTRx; ISIS-443139; ISIS-HTT Rx; RG 6042

Latest Information Update: 09 Mar 2018

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At a glance

  • Originator Isis Pharmaceuticals; Roche
  • Developer Ionis Pharmaceuticals; Roche
  • Class Antisense oligonucleotides
  • Mechanism of Action HD protein inhibitors
  • Orphan Drug Status

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

    Yes - Huntington's disease
  • New Molecular Entity Yes

Highest Development Phases

  • Phase II Huntington's disease

Most Recent Events

  • 01 Mar 2018 Efficacy, safety and pharmacodynamics data from a phase I/II trial in Huntington's disease released by Ionis Pharmaceuticals
  • 01 Mar 2018 Roche plans a pivotal trial of IONIS-HTTRx for Huntington's disease
  • 14 Dec 2017 Efficacy data from a phase I/II trial in Huntington's disease released by Ionis Pharmaceuticals
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