Discovery and Optimization of Novel Pyrazolopyrimidines as Potent and Orally Bioavailable Allosteric HIV-1 Integrase Inhibitors

J Med Chem. 2020 Mar 12;63(5):2620-2637. doi: 10.1021/acs.jmedchem.9b01681. Epub 2020 Feb 21.

Abstract

The standard of care for HIV-1 infection, highly active antiretroviral therapy (HAART), combines two or more drugs from at least two classes. Even with the success of HAART, new drugs with novel mechanisms are needed to combat viral resistance, improve adherence, and mitigate toxicities. Active site inhibitors of HIV-1 integrase are clinically validated for the treatment of HIV-1 infection. Here we describe allosteric inhibitors of HIV-1 integrase that bind to the LEDGF/p75 interaction site and disrupt the structure of the integrase multimer that is required for the HIV-1 maturation. A series of pyrazolopyrimidine-based inhibitors was developed with a vector in the 2-position that was optimized by structure-guided compound design. This resulted in the discovery of pyrazolopyrimidine 3, which was optimized at the 2- and 7-positions to afford 26 and 29 as potent allosteric inhibitors of HIV-1 integrase that exhibited low nanomolar antiviral potency in cell culture and encouraging PK properties.

MeSH terms

  • Administration, Oral
  • Allosteric Regulation / drug effects*
  • Animals
  • Drug Discovery
  • HIV Infections / drug therapy
  • HIV Infections / virology
  • HIV Integrase / metabolism
  • HIV Integrase Inhibitors / administration & dosage
  • HIV Integrase Inhibitors / chemistry*
  • HIV Integrase Inhibitors / pharmacokinetics
  • HIV Integrase Inhibitors / pharmacology*
  • HIV-1 / drug effects*
  • Humans
  • Male
  • Molecular Docking Simulation
  • Pyrazoles / administration & dosage
  • Pyrazoles / chemistry*
  • Pyrazoles / pharmacokinetics
  • Pyrazoles / pharmacology*
  • Pyridines / administration & dosage
  • Pyridines / chemistry*
  • Pyridines / pharmacokinetics
  • Pyridines / pharmacology*
  • Rats, Sprague-Dawley

Substances

  • HIV Integrase Inhibitors
  • Pyrazoles
  • Pyridines
  • pyrazolo(3,4-b)pyridine
  • HIV Integrase
  • p31 integrase protein, Human immunodeficiency virus 1