Drug Profile


Alternative Names: IONIS-HTTRx; ISIS 443139; ISIS-HTT Rx

Latest Information Update: 15 Dec 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

  • 11 Dec 2017 Roche exercises the option to license IONIS HTTRx
  • 11 Dec 2017 Ionis Pharmaceuticals initiates enrolment in an open-label extension phase II trial in Huntington's disease in Canada, Germany and United Kingdom (NCT03342053)
  • 21 Nov 2017 Ionis Pharmaceuticals plans a phase II extension trial for Huntington's disease in Canada, Germany and United Kingdom (Intrathecal) in November 2017 (NCT03342053)
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