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Discovery and biological evaluation of N-(3-(7-((2-methoxy-4- (4-methylpiperazin-1-yl)phenyl)amino)-4-methyl-2-oxo-2H- pyrimido[4,5-d][1,3]oxazin-1(4H)-yl)phenyl)acrylamide as potent Bruton’s tyrosine kinase inhibitors

Meng-zhen Lai1,2, Pei-ran Song2,3,4, Dou Dou5, Yan-yan Diao5, Lin-jiang Tong2,4, Tao Zhang2,3,4, Hua Xie2,4, Hong-lin Li5, Jian Ding2,3,4
1 School of Pharmacy, Nanchang University, Nanchang 330006, China
2 Division of Anti-tumor Pharmacology, State Key Laboratory of Drug Research, Shanghai Institute of Materia Medica, Chinese Academy of Sciences, Shanghai 201203, China
3 School of Life Science and Technology, ShanghaiTech University, Shanghai 201210, China
4 University of Chinese Academy of Sciences, Beijing 100049, China
5 Shanghai Key Laboratory of New Drug Design, State Key Laboratory of Bioreactor Engineering, School of Pharmacy, East China University of Science and Technology, Shanghai 200237, China
Correspondence to: Hua Xie: hxie@simm.ac.cn, Hong-lin Li: hlli@ecust.edu.cn, Jian Ding: jding@simm.ac.cn,
DOI: 10.1038/s41401-019-0250-8
Received: 7 March 2019
Accepted: 12 May 2019
Advance online: 17 July 2019

Abstract

>Bruton’s tyrosine kinase (BTK) is a key component of the B cell receptor (BCR) signaling pathway and plays a crucial role in B cell malignancies and autoimmune disorders; thus, it is an attractive target for the treatment of B cell related diseases. Here, we evaluated the BTK inhibitory activity of a series of pyrimido[4,5-d][1,3]oxazin-2-one derivatives. Combining this evaluation with structure-activity relationship (SAR) analysis, we found that compound 2 exhibited potent BTK kinase inhibitory activity, with an IC50 of 7 nM. This derivative markedly inhibited BTK activation in TMD8 B cell lymphoma cells and thus inhibited the in vitro growth of the cells. Further studies revealed that compound 2 dose dependently arrested TMD8 cells at G1 phase, accompanied by decreased levels of Rb, phosphorylated Rb, and cyclin D1. Moreover, following treatment with compound 2, TMD8 cells underwent apoptosis associated with PARP and caspase 3 cleavage. Interestingly, the results of the kinase activity assay on a small panel of 35 kinases showed that the kinase selectivity of compound 2 was superior to that of the first-generation inhibitor ibrutinib, suggesting that compound 2 could be a second-generation inhibitor of BTK. In conclusion, we identified a potent and highly selective BTK inhibitor worthy of further development.
Keywords: B cell receptor; Bruton’s tyrosine kinase; ibrutinib; small-molecule inhibitor; B cell malignancies

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