Investigation into the Mechanism(s) That Leads to Platelet Decreases in Cynomolgus Monkeys During Administration of ISIS 104838, a 2'-MOE-Modified Antisense Oligonucleotide

Toxicol Sci. 2018 Aug 1;164(2):613-626. doi: 10.1093/toxsci/kfy119.

Abstract

ISIS 104838, a 2'-O-methoxyethyl (2'-MOE)-modified antisense oligonucleotide (ASO), causes a moderate, reproducible, dose-dependent, but selflimiting decrease in platelet (PLT) counts in monkeys and humans. To determine the etiology of PLT decrease in cynomolgus monkeys, a 12-week repeat dose toxicology study in 5 cynomolgus monkeys given subcutaneous injections of ISIS 104838 (30-60 mg/kg/week). Monkeys were also injected intravenously with 111Indium(In)-oxine-labeled PLTs to investigate PLT sequestration. In response to continued dosing, PLT counts were decreased by 50%-90% by day 30 in all monkeys. PLT decreases were accompanied by 2- to 4.5-fold increases in immunoglobulin M(IgM), which were typified by a 2- to 5-fold increase in antiplatelet factor 4 (antiPF4) IgM and antiPLT IgM, respectively. Monocyte chemotactic protein 1 increased upon dosing of ISIS 104838, concomitant with a 2- to 6-fold increase in monocyte-derived extracellular vesicles (EVs), indicating monocyte activation but not PLT activation. Despite a 2- to 3-fold increase in von Willebrand factor antigen in all monkeys following ASO administration, only 2 monkeys showed a 2- to 4-fold increase in endothelial EVs. Additionally, a ∼60 - 80%% increase in PLT sequestration in liver and spleen was also observed. Collectively, these results suggest the overall increase in total IgM, antiPLT IgM and/or antiPF4 IgM, in concert with monocyte activation contributed to increased PLT sequestration in spleen and liver, leading to decreased PLTs in peripheral blood.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Blood Platelets / cytology
  • Blood Platelets / drug effects*
  • Chemokine CCL2 / metabolism
  • Extracellular Vesicles / metabolism
  • Female
  • Immunoglobulin G / blood
  • Immunoglobulin G / metabolism
  • Immunoglobulin M / blood
  • Immunoglobulin M / metabolism
  • Liver / drug effects
  • Liver / metabolism
  • Macaca fascicularis / blood*
  • Monocytes / drug effects
  • Monocytes / metabolism
  • Oligonucleotides, Antisense / pharmacology*
  • P-Selectin / metabolism
  • Phosphorothioate Oligonucleotides / metabolism
  • Phosphorothioate Oligonucleotides / pharmacokinetics
  • Phosphorothioate Oligonucleotides / pharmacology*
  • Platelet Count
  • Spleen / drug effects
  • Spleen / metabolism
  • von Willebrand Factor / metabolism

Substances

  • Chemokine CCL2
  • ISIS104838
  • Immunoglobulin G
  • Immunoglobulin M
  • Oligonucleotides, Antisense
  • P-Selectin
  • Phosphorothioate Oligonucleotides
  • Von Willebrand antigen
  • von Willebrand Factor