A highly potent long-acting small-molecule HIV-1 capsid inhibitor with efficacy in a humanized mouse model

Nat Med. 2019 Sep;25(9):1377-1384. doi: 10.1038/s41591-019-0560-x. Epub 2019 Sep 9.

Abstract

People living with HIV (PLWH) have expressed concern about the life-long burden and stigma associated with taking pills daily and can experience medication fatigue that might lead to suboptimal treatment adherence and the emergence of drug-resistant viral variants, thereby limiting future treatment options1-3. As such, there is strong interest in long-acting antiretroviral (ARV) agents that can be administered less frequently4. Herein, we report GS-CA1, a new archetypal small-molecule HIV capsid inhibitor with exceptional potency against HIV-2 and all major HIV-1 types, including viral variants resistant to the ARVs currently in clinical use. Mechanism-of-action studies indicate that GS-CA1 binds directly to the HIV-1 capsid and interferes with capsid-mediated nuclear import of viral DNA, HIV particle production and ordered capsid assembly. GS-CA1 selects in vitro for unfit GS-CA1-resistant capsid variants that remain fully susceptible to other classes of ARVs. Its high metabolic stability and low solubility enabled sustained drug release in mice following a single subcutaneous dosing. GS-CA1 showed high antiviral efficacy as a long-acting injectable monotherapy in a humanized mouse model of HIV-1 infection, outperforming long-acting rilpivirine. Collectively, these results demonstrate the potential of ultrapotent capsid inhibitors as new long-acting agents for the treatment of HIV-1 infection.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Anti-HIV Agents / pharmacology*
  • Anti-HIV Agents / therapeutic use
  • Capsid / drug effects
  • Capsid / metabolism
  • Capsid Proteins / antagonists & inhibitors*
  • Capsid Proteins / genetics
  • DNA, Viral / drug effects
  • Delayed-Action Preparations
  • Drug Resistance, Viral / drug effects
  • HIV Infections / drug therapy*
  • HIV Infections / genetics
  • HIV Infections / virology
  • HIV-1 / drug effects*
  • HIV-1 / genetics
  • HIV-1 / pathogenicity
  • HIV-2 / drug effects
  • HIV-2 / pathogenicity
  • Humans
  • Indazoles / pharmacology*
  • Indazoles / therapeutic use
  • Medication Adherence
  • Mice
  • Pyridines / pharmacology*
  • Pyridines / therapeutic use
  • Small Molecule Libraries / pharmacology*

Substances

  • Anti-HIV Agents
  • Capsid Proteins
  • DNA, Viral
  • Delayed-Action Preparations
  • GS-CA1
  • Indazoles
  • Pyridines
  • Small Molecule Libraries