Rational Design, Synthesis, and Structure-Activity Relationship of a Novel Isoquinolinone-Based Series of HBV Capsid Assembly Modulators Leading to the Identification of Clinical Candidate AB-836

J Med Chem. 2024 Sep 26;67(18):16773-16795. doi: 10.1021/acs.jmedchem.4c01568. Epub 2024 Sep 4.

Abstract

Inhibition of Hepatitis B Virus (HBV) replication by small molecules that modulate capsid assembly and the encapsidation of pgRNA and viral polymerase by HBV core protein is a clinically validated approach toward the development of new antivirals. Through definition of a minimal pharmacophore, a series of isoquinolinone-based capsid assembly modulators (CAMs) was identified. Structural biology analysis revealed that lead molecules possess a unique binding mode, exploiting electrostatic interactions with accessible phenylalanine and tyrosine residues. Key analogs demonstrated excellent primary potency, absorption, distribution, metabolism, and excretion (ADME) and pharmacokinetic properties, and efficacy in a mouse model of HBV. The optimized lead also displayed potent inhibition of capsid uncoating in HBV-infected HepG2 cells expressing the sodium-taurocholate cotransporting polypeptide (NTCP) receptor, affecting the generation of HBsAg and cccDNA establishment. Based on these results, isoquinolinone derivative AB-836 was advanced into clinical development. In Phase 1b trials, AB-836 demonstrated >3 log10 reduction in serum HBV DNA, however, further development was discontinued due to the observation of incidental alanine aminotransferase (ALT) elevations.

MeSH terms

  • Animals
  • Antiviral Agents* / chemical synthesis
  • Antiviral Agents* / chemistry
  • Antiviral Agents* / pharmacokinetics
  • Antiviral Agents* / pharmacology
  • Capsid / drug effects
  • Capsid / metabolism
  • Capsid Proteins / antagonists & inhibitors
  • Capsid Proteins / metabolism
  • Drug Design*
  • Hep G2 Cells
  • Hepatitis B virus* / drug effects
  • Humans
  • Isoquinolines / chemical synthesis
  • Isoquinolines / chemistry
  • Isoquinolines / pharmacology
  • Mice
  • Quinolones / chemical synthesis
  • Quinolones / chemistry
  • Quinolones / pharmacology
  • Structure-Activity Relationship
  • Virus Assembly / drug effects

Substances

  • Antiviral Agents
  • Capsid Proteins
  • Isoquinolines
  • Quinolones