Drug Profile


Alternative Names: IONIS-HTTRx; ISIS-HTT Rx

Latest Information Update: 27 Jun 2017

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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

  • 22 Jun 2017 Ionis Pharmaceuticals completes enrolment in a Phase I/IIa trial for Huntington's disease in the United Kingdom, Canada and Germany
  • 22 Jun 2017 Ionis Pharmaceuticals plans an open label extension (OLE) trial of the phase I/IIa trial for Huntington's disease
  • 15 Jun 2016 Phase-II clinical trials in Huntington's disease in United Kingdom, Germany, Canada (Intrathecal) (Ionis Pharmaceuticals pipeline, June 2016)
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