Discovery of 2-[3,5-dichloro-4-(5-isopropyl-6-oxo-1,6-dihydropyridazin-3-yloxy)phenyl]-3,5-dioxo-2,3,4,5-tetrahydro[1,2,4]triazine-6-carbonitrile (MGL-3196), a Highly Selective Thyroid Hormone Receptor β agonist in clinical trials for the treatment of dyslipidemia

J Med Chem. 2014 May 22;57(10):3912-23. doi: 10.1021/jm4019299. Epub 2014 Apr 8.

Abstract

The beneficial effects of thyroid hormone (TH) on lipid levels are primarily due to its action at the thyroid hormone receptor β (THR-β) in the liver, while adverse effects, including cardiac effects, are mediated by thyroid hormone receptor α (THR-α). A pyridazinone series has been identified that is significantly more THR-β selective than earlier analogues. Optimization of this series by the addition of a cyanoazauracil substituent improved both the potency and selectivity and led to MGL-3196 (53), which is 28-fold selective for THR-β over THR-α in a functional assay. Compound 53 showed outstanding safety in a rat heart model and was efficacious in a preclinical model at doses that showed no impact on the central thyroid axis. In reported studies in healthy volunteers, 53 exhibited an excellent safety profile and decreased LDL cholesterol (LDL-C) and triglycerides (TG) at once daily oral doses of 50 mg or higher given for 2 weeks.

MeSH terms

  • Animals
  • Bone Density / drug effects
  • Clinical Trials as Topic
  • Drug Discovery*
  • Dyslipidemias / drug therapy*
  • Humans
  • Male
  • Mice
  • Mice, Inbred C57BL
  • Pyridazines / chemical synthesis*
  • Pyridazines / metabolism
  • Pyridazines / pharmacology
  • Pyridazines / therapeutic use
  • Rats
  • Rats, Sprague-Dawley
  • Structure-Activity Relationship
  • Thyroid Hormone Receptors beta / agonists*
  • Uracil / analogs & derivatives*
  • Uracil / chemical synthesis
  • Uracil / metabolism
  • Uracil / pharmacology
  • Uracil / therapeutic use

Substances

  • Pyridazines
  • Thyroid Hormone Receptors beta
  • Uracil
  • resmetirom