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Research programme: RNAi therapeutics - Arrowhead Pharmaceuticals

Drug Profile

Research programme: RNAi therapeutics - Arrowhead Pharmaceuticals

Alternative Names: ARC AMG1; ARC HIF2; ARC-F12; ARO APOC3; ARo Lung1; ARO-AMG1; ARO-ENaC; ARO-F12; ARO-Lung1

Latest Information Update: 10 Jan 2019

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At a glance

  • Originator Mirus Bio Corporation
  • Developer Arrowhead Pharmaceuticals; Roche Madison
  • Class Antifibrotics; Antihyperlipidaemics; Antineoplastics; Antithrombotics; Cardiovascular therapies; Small interfering RNA
  • Mechanism of Action ANGPTL3 protein inhibitors; Apolipoprotein C-III inhibitors; Endothelial PAS domain-containing protein 1 inhibitors; RNA interference
  • 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

  • Preclinical Cardiovascular disorders; Cystic fibrosis; Hereditary angioedema; Hypertriglyceridaemia; Liver cancer; Lung cancer; Renal cell carcinoma; Solid tumours; Thromboembolism
  • No development reported Hyperlipidaemia; Respiratory tract disorders

Most Recent Events

  • 07 Jan 2019 Arrowhead Pharmaceuticals files a clinical trial application (CTA) for ARO-APOC3 with the local Ethics Committee and to the New Zealand Medicines and Medical Devices Safety Authority for Hypertriglyceridemia
  • 03 Dec 2018 Arrowhead Pharmaceuticals plans a phase I AROANG1001 trial for ARO ANG3 in Hypertriglyceridaemia, Dyslipidaemias and Hypercholesterolaemia (In volunteers) (SC) in December 2018 (NCT03747224)
  • 16 Oct 2018 Arrowhead plans the phase I single and multiple ascending dose AROANG1001 trial for ARO APOC3 for Hypertriglyceridaemia in February 2019 (SC) (NCT03783377)
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