Identification of NVP-CLR457 as an Orally Bioavailable Non-CNS-Penetrant pan-Class IA Phosphoinositol-3-Kinase Inhibitor

J Med Chem. 2022 Jun 23;65(12):8345-8379. doi: 10.1021/acs.jmedchem.2c00267. Epub 2022 May 2.

Abstract

Balanced pan-class I phosphoinositide 3-kinase inhibition as an approach to cancer treatment offers the prospect of treating a broad range of tumor types and/or a way to achieve greater efficacy with a single inhibitor. Taking buparlisib as the starting point, the balanced pan-class I PI3K inhibitor 40 (NVP-CLR457) was identified with what was considered to be a best-in-class profile. Key to the optimization to achieve this profile was eliminating a microtubule stabilizing off-target activity, balancing the pan-class I PI3K inhibition profile, minimizing CNS penetration, and developing an amorphous solid dispersion formulation. A rationale for the poor tolerability profile of 40 in a clinical study is discussed.

MeSH terms

  • Aminopyridines / pharmacology
  • Antineoplastic Agents* / pharmacology
  • Cell Line, Tumor
  • Organic Chemicals
  • Phosphatidylinositol 3-Kinases*
  • Phosphoinositide-3 Kinase Inhibitors
  • Protein Kinase Inhibitors / pharmacology
  • Protein Kinase Inhibitors / therapeutic use

Substances

  • Aminopyridines
  • Antineoplastic Agents
  • CLR457
  • Organic Chemicals
  • Phosphoinositide-3 Kinase Inhibitors
  • Protein Kinase Inhibitors