Structure-guided discovery of a novel, potent, and orally bioavailable 3,5-dimethylisoxazole aryl-benzimidazole BET bromodomain inhibitor

  • Bioorg Med Chem. 2019 Feb 1;27(3):457-469. doi: 10.1016/j.bmc.2018.11.020.
David Sperandio  1 ,  Vangelis Aktoudianakis  2 ,  Kerim Babaoglu  3 ,  Xiaowu Chen  3 ,  Kristyna Elbel  2 ,  Gregory Chin  2 ,  Britton Corkey  2 ,  Jinfa Du  2 ,  Bob Jiang  2 ,  Tetsuya Kobayashi  2 ,  Richard Mackman  2 ,  Ruben Martinez  2 ,  Hai Yang  2 ,  Jeff Zablocki  2 ,  Saritha Kusam  4 ,  Kim Jordan  4 ,  Heather Webb  4 ,  Jamie G Bates  4 ,  Latesh Lad  4 ,  Michael Mish  2 ,  Anita Niedziela-Majka  4 ,  Sammy Metobo  2 ,  Annapurna Sapre  4 ,  Magdeleine Hung  4 ,  Debi Jin  4 ,  Wanchi Fung  4 ,  Elaine Kan  4 ,  Gene Eisenberg  5 ,  Nate Larson  4 ,  Zachary E R Newby  3 ,  Eric Lansdon  3 ,  Chin Tay  4 ,  Richard M Neve  4 ,  Sophia L Shevick  2 ,  David G Breckenridge  4
Affiliations
  • 1. Department of Medicinal Chemistry, Gilead Sciences, Inc., 333 Lakeside Drive, Foster City, CA 94404, USA. Electronic address: [email protected].
  • 2. Department of Medicinal Chemistry, Gilead Sciences, Inc., 333 Lakeside Drive, Foster City, CA 94404, USA.
  • 3. Department of Structural Chemistry, Gilead Sciences, Inc., 333 Lakeside Drive, Foster City, CA 94404, USA.
  • 4. Department of Biology, Gilead Sciences, Inc., 333 Lakeside Drive, Foster City, CA 94404, USA.
  • 5. Department of Drug Metabolism, Gilead Sciences, Inc., 333 Lakeside Drive, Foster City, CA 94404, USA.
Abstract

The bromodomain and extra-terminal (BET) family of proteins, consisting of the bromodomains containing protein 2 (BRD2), BRD3, BRD4, and the testis-specific BRDT, are key epigenetic regulators of gene transcription and has emerged as an attractive target for Anticancer therapy. Herein, we describe the discovery of a novel potent BET bromodomain inhibitor, using a systematic structure-based approach focused on improving potency, metabolic stability, and permeability. The optimized dimethylisoxazole aryl-benzimidazole inhibitor exhibited high potency towards BRD4 and related BET proteins in biochemical and cell-based assays and inhibited tumor growth in two proof-of-concept preclinical animal models.

Keywords
Antitumor; BET proteins; Bromodomain inhibitor; Epigenetic readers.
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