Discovery of Quinazoline-2,4(1H,3H)-dione Derivatives Containing 3-Substituted Piperizines as Potent PARP-1/2 Inhibitors─Design, Synthesis, In Vivo Antitumor Activity, and X-ray Crystal Structure Analysis

Journal of Medicinal Chemistry
2021.0

Abstract

Inhibiting PARP-1/2 offered an important arsenal for cancer treatments via interfering with DNA repair of cancer cells. Novel PARP-1/2 inhibitors were designed by capitalizing on methyl- or ethyl-substituted piperizine ring to capture the characteristics of adenine-ribose binding site (AD site), and their unique binding features were revealed by the cocrystal structures of compounds <b>4</b> and <b>6</b> in PARP-1. The investigation on structure-activity relationship resulted in compounds <b>24</b> and <b>32</b> with high enzymatic potency, binding selectivity, and significantly longer residence time for PARP-1 over PARP-2 (compound <b>24</b>, PARP-1: IC<sub>50</sub> = 0.51 nM, PARP-2: IC<sub>50</sub> = 23.11 nM; compound <b>32</b>, PARP-1: IC<sub>50</sub> = 1.31 nM, PARP-2: IC<sub>50</sub> = 15.63 nM). Furthermore, compound <b>24</b> was determined to be an attractive candidate molecule, which possessed an acceptable pharmacokinetic profile and produced remarkable antitumor activity in both breast cancer xenograft model and glioblastoma orthotopic model in mice, either alone or in combination treatment.

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