Drug Profile


Alternative Names: AKCEA-APOCIII-LRx; GalNAc3 conjugated antisense oligonucleotide - Ionis Pharmaceuticals; IONIS-APOCIII-LRx; ISIS 678354; ISIS-APOCIII-LRx

Latest Information Update: 26 Mar 2018

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At a glance

  • Originator Ionis Pharmaceuticals
  • Developer Akcea Therapeutics; Ionis Pharmaceuticals
  • Class Antihyperlipidaemics; Antisense oligonucleotides
  • Mechanism of Action Apolipoprotein C-III inhibitors; RNA modulators
  • Orphan Drug Status

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

  • New Molecular Entity Yes

Highest Development Phases

  • Phase II Hypertriglyceridaemia

Most Recent Events

  • 12 Mar 2018 Updated safety and efficacy data from a phase I/II trial in Hypertriglyceridaemia presented at the 67th Annual Scientific Session of the American College of Cardiology (ACC-2018)
  • 05 Jan 2018 Phase-II clinical trials in Hypertriglyceridaemia in Canada (SC) (NCT03385239)
  • 06 Nov 2017 Akcea Therapeutics and Ionis Pharmaceuticals plan a phase IIb dose-ranging study for Hypertriglyceridemia in patients with established cardiovascular disease in January 2018 (NCT03385239)
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