Synthesis and biological evaluation of a series of liver-selective phosphonic acid thyroid hormone receptor agonists and their prodrugs

J Med Chem. 2008 Nov 27;51(22):7075-93. doi: 10.1021/jm800824d.

Abstract

Phosphonic acid (PA) thyroid hormone receptor (TR) agonists were synthesized to exploit the poor distribution of PA-based drugs to extrahepatic tissues and thereby to improve the therapeutic index. Nine PAs showed excellent TR binding affinities (TRbeta(1), K(i) < 10 nM), and most of them demonstrated significant cholesterol lowering effects in a cholesterol-fed rat (CFR) model. Unlike the corresponding carboxylic acid analogue and T(3), PA 22c demonstrated liver-selective effects by inducing maximal mitochondrial glycerol-3-phosphate dehydrogenase activity in rat liver while having no effect in the heart. Because of the low oral bioavailability of PA 22c, a series of prodrugs was synthesized and screened for oral efficacy in the CFR assay. The liver-activated cyclic 1-(3-chlorophenyl)-1,3-propanyl prodrug (MB07811) showed potent lipid lowering activity in the CFR (ED(50) 0.4 mg/kg, po) and good oral bioavailability (40%, rat) and was selected for development for the treatment of hypercholesterolemia.

MeSH terms

  • Animals
  • Cholesterol / administration & dosage
  • Cholesterol / blood
  • Disease Models, Animal
  • Dose-Response Relationship, Drug
  • Drug Design
  • Drug Evaluation, Preclinical
  • Glycerolphosphate Dehydrogenase / metabolism
  • Hypercholesterolemia / drug therapy
  • Ligands
  • Liver / drug effects*
  • Liver / metabolism
  • Molecular Structure
  • Organophosphonates / chemical synthesis*
  • Organophosphonates / chemistry
  • Organophosphonates / pharmacology*
  • Prodrugs / chemical synthesis*
  • Prodrugs / chemistry
  • Prodrugs / pharmacology*
  • Rats
  • Rats, Sprague-Dawley
  • Receptors, Thyroid Hormone / agonists*
  • Stereoisomerism
  • Structure-Activity Relationship

Substances

  • 2-((3,5-dimethyl-4-(4'-hydroxy-3'-isopropylbenzyl)phenoxy)methyl)-4-(3-chlorophenyl)-2-oxido(1,3,2)dioxaphosphonane
  • Ligands
  • Organophosphonates
  • Prodrugs
  • Receptors, Thyroid Hormone
  • Cholesterol
  • Glycerolphosphate Dehydrogenase