Pharmacokinetics-Driven Optimization of 4(3 H)-Pyrimidinones as Phosphodiesterase Type 5 Inhibitors Leading to TPN171, a Clinical Candidate for the Treatment of Pulmonary Arterial Hypertension

J Med Chem. 2019 May 23;62(10):4979-4990. doi: 10.1021/acs.jmedchem.9b00123. Epub 2019 May 8.

Abstract

Phosphodiesterase type 5 (PDE5) inhibitors are first-line therapy for pulmonary arterial hypertension (PAH) and erectile dysfunction. As a continuing work to improve the terminal half-lives and oral bioavailabilities of our previously reported 4(3 H)-pyrimidones, a pharmacokinetics-driven optimization focusing on the terminal substituent is described. Two major congeneric series of 4(3 H)-pyrimidones, the aminosulfonylphenylpyrimidones and acylaminophenylpyrimidones, were designed, synthesized, and pharmacologically assessed in vitro and in vivo. Among them, compound 15 (TPN171) with subnanomolar potency for PDE5 and good selectivity over PDE6 was finally recognized as a potential drug candidate, and its pharmacokinetic profiles in rats and dogs are significantly improved compared to the starting compound (3). Moreover, TPN171 was proven to exert a longer lasting effect than sildenafil in animal models, providing a foundation for a once-daily oral administration for its clinical use. TPN171 is currently being investigated in a phase II clinical trial for the treatment of PAH.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Dogs
  • Drug Design
  • Female
  • Half-Life
  • Hypertension, Pulmonary / drug therapy*
  • Male
  • Phosphodiesterase 5 Inhibitors / chemical synthesis
  • Phosphodiesterase 5 Inhibitors / pharmacokinetics*
  • Phosphodiesterase 5 Inhibitors / pharmacology*
  • Pyrimidines / chemical synthesis
  • Pyrimidines / pharmacokinetics*
  • Pyrimidines / pharmacology*
  • Rats
  • Rats, Inbred SHR
  • Rats, Sprague-Dawley
  • Sildenafil Citrate / pharmacology
  • Structure-Activity Relationship
  • Substrate Specificity

Substances

  • Phosphodiesterase 5 Inhibitors
  • Pyrimidines
  • Sildenafil Citrate