Identification of the Clinical Candidate PF-07284890 (ARRY-461), a Highly Potent and Brain Penetrant BRAF Inhibitor for the Treatment of Cancer

J Med Chem. 2024 Aug 8;67(15):13019-13032. doi: 10.1021/acs.jmedchem.4c00998. Epub 2024 Jul 30.

Abstract

Mutant BRAFV600E is one of the most common oncogenic drivers in metastatic melanoma. While first generation BRAFV600E inhibitors are capable of controlling tumors systemically, they are unable to adequately treat tumors that have metastasized to the brain due to insufficient penetration across the blood-brain barrier (BBB). Through a combination of structure-based drug design (SBDD) and the optimization of physiochemical properties to enhance BBB penetration, we herein report the discovery of the brain-penetrant BRAFV600E inhibitor PF-07284890 (ARRY-461). In mice studies, ARRY-461 proved to be highly brain-penetrant and was able to drive regressions of A375 BRAFV600E tumors implanted both subcutaneously and intracranially. Based on compelling preclinical safety and efficacy studies, ARRY-461 was progressed into a Phase 1 A/B clinical trial (NCT04543188).

Publication types

  • Clinical Trial, Phase I

MeSH terms

  • Animals
  • Antineoplastic Agents* / chemistry
  • Antineoplastic Agents* / pharmacokinetics
  • Antineoplastic Agents* / pharmacology
  • Antineoplastic Agents* / therapeutic use
  • Blood-Brain Barrier / metabolism
  • Brain / metabolism
  • Cell Line, Tumor
  • Female
  • Humans
  • Male
  • Melanoma / drug therapy
  • Melanoma / pathology
  • Mice
  • Mice, Nude
  • Protein Kinase Inhibitors* / chemistry
  • Protein Kinase Inhibitors* / pharmacokinetics
  • Protein Kinase Inhibitors* / pharmacology
  • Protein Kinase Inhibitors* / therapeutic use
  • Proto-Oncogene Proteins B-raf* / antagonists & inhibitors
  • Proto-Oncogene Proteins B-raf* / metabolism
  • Rats
  • Structure-Activity Relationship
  • Xenograft Model Antitumor Assays

Substances

  • Proto-Oncogene Proteins B-raf
  • Protein Kinase Inhibitors
  • Antineoplastic Agents
  • BRAF protein, human

Associated data

  • ClinicalTrials.gov/NCT04543188