Chen Peng, Wang Lu, Wang Xuan, Sun Jie, Miao Fengfei, Wang Zuqin, Yang Fang, Xiang Menghua, Gu Mingxi, Li Shengrong, Zhang Jianzhong, Yuan Peiyan, Lu Xiaoyun, Zhang Zhi-Min, Gao Liqian, Yao Shao Q
Department of Chemistry, National University of Singapore, Singapore, 117543, Singapore.
School of Pharmaceutical Sciences (Shenzhen), Shenzhen Campus of Sun Yat-sen University, Shenzhen, 518107, China.
Angew Chem Int Ed Engl. 2025 Mar 24;64(13):e202422372. doi: 10.1002/anie.202422372. Epub 2025 Jan 15.
Targeted covalent inhibitors (TCIs) play an essential role in the fields of kinase research and drug discovery. TCI strategies to target more common amino acid side-chains have yet to be demonstrated. Targeting other amino acids would also expand the pharmaceutical industry's toolbox for targeting other tough-to-drug proteins. We report herein a glyoxal-based, arginine-reactive strategy to generate potent and selective small-molecule TCIs of Mcl-1 (an important anti-apoptotic protein) by selectively targeting the conserved arginine (R263) in the protein. We further validated the generality of this strategy by developing glyoxal-based, irreversible covalent inhibitors of AURKA (a cancer-related kinase) that showed exclusive reactivity with a solvent-exposed arginine (R220) of this enzyme. We showed the resulting compounds were potent, selective and cell-active, capable of covalently engaging endogenous AURKA in MV-4-11 cells with long residence time. Finally, we showed the potential application of glyoxal-based TCIs in targeting an acquired drug-resistance mutant of ALK kinase (G1202R).
靶向共价抑制剂(TCIs)在激酶研究和药物发现领域发挥着重要作用。针对更常见氨基酸侧链的TCI策略尚未得到证实。靶向其他氨基酸也将扩展制药行业针对其他难以成药蛋白的工具库。我们在此报告一种基于乙二醛的、精氨酸反应性策略,通过选择性靶向Mcl-1(一种重要的抗凋亡蛋白)中保守的精氨酸(R263)来生成强效且选择性的小分子TCI。我们通过开发基于乙二醛的、AURKA(一种与癌症相关的激酶)的不可逆共价抑制剂进一步验证了该策略的通用性,该抑制剂显示出与该酶的溶剂暴露精氨酸(R220)具有专一反应性。我们表明所得化合物具有强效、选择性且具有细胞活性,能够在MV-4-11细胞中与内源性AURKA共价结合且停留时间长。最后,我们展示了基于乙二醛的TCIs在靶向ALK激酶的获得性耐药突变体(G1202R)方面的潜在应用。