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Design, Synthesis, and Biological Evaluation of Thieno[3,2-<i>d</i>]pyrimidine Derivatives as the First Bifunctional PI3Kδ Isoform Selective/Bromodomain and Extra-Terminal Inhibitors

Kai Ran, Jiu-Hong Huang, Yong Li, Yimei Zhang, Hao Hu, Zhenyu Wang, Dianyong Tang, Hongyu Li, Zhigang Xu, Zhong‐Zhu Chen

2025Journal of Medicinal Chemistry13 citationsDOIOpen Access PDF

Abstract

The concomitant inhibition of PI3Kδ and bromodomain and extra-terminal (BET) that exerts a synergistic effect on the B-cell receptor signaling pathway provides a new strategy for the treatment of aggressive diffuse large B-cell lymphoma (DLBCL). Herein, a merged pharmacophore strategy was utilized to discover a series of thieno[3,2- d ]pyrimidine derivatives as the first-in-class bifunctional PI3Kδ-BET inhibitors. Through optimization, a highly potent compound ( 10b ) was identified to possess excellent and balanced activities against PI3Kδ [inhibitory concentration (IC 50 ) = 112 ± 8 nM] and BRD4-BD1 (IC 50 = 19 ± 1 nM) and exhibited strong antiproliferative activities in DLBCL cells. Notably, this compound demonstrated good PI3Kδ selectivity over other kinases with minimal cytotoxicity in normal cells. Moreover, 10b has a good oral pharmacokinetic profile in mice and achieves outstanding antitumor activity in the SU-DHL-6 xenograft model. Taken together, these results indicate that targeting PI3Kδ and BET with a bifunctional inhibitor is a promising strategy to treat DLBCL.

Topics & Concepts

ChemistryPharmacophoreBromodomainBifunctionalPyrimidineKinaseIC50PI3K/AKT/mTOR pathwayStereochemistryCombinatorial chemistryPharmacologyBiochemistryAcetylationIn vitroSignal transductionGeneMedicineCatalysisProtein Degradation and InhibitorsMultiple Myeloma Research and TreatmentsCAR-T cell therapy research
Design, Synthesis, and Biological Evaluation of Thieno[3,2-<i>d</i>]pyrimidine Derivatives as the First Bifunctional PI3Kδ Isoform Selective/Bromodomain and Extra-Terminal Inhibitors | Litcius