Razumkov Hlib, Jiang Zixuan, Baek Kheewoong, You Inchul, Geng Qixiang, Donovan Katherine A, Tang Michelle T, Metivier Rebecca J, Mageed Nada, Seo Pooreum, Li Zhengnian, Byun Woong Sub, Hinshaw Stephen M, Sarott Roman C, Fischer Eric S, Gray Nathanael S
bioRxiv. 2024 May 5:2024.05.04.592550. doi: 10.1101/2024.05.04.592550.
Small molecules promoting protein-protein interactions produce a range of therapeutic outcomes. Molecular glue degraders exemplify this concept due to their compact drug-like structures and ability to engage targets without reliance on existing cognate ligands. While Cereblon molecular glue degraders containing glutarimide scaffolds have been approved for treatment of multiple myeloma and acute myeloid leukemia, the design of new therapeutically relevant monovalent degraders remains challenging. We report here an approach to glutarimide-containing molecular glue synthesis using multicomponent reactions as a central modular core-forming step. Screening the resulting library identified HRZ-01 derivatives that target casein kinase 1 alpha (CK1α) and Wee-like protein kinase (WEE1). Further medicinal chemistry efforts led to identification of selective monovalent WEE1 degraders that provide a potential starting point for the eventual development of a selective chemical degrader probe. The structure of the hit WEE1 degrader complex with CRBN-DDB1 and WEE1 provides a model of the protein-protein interface and a rationale for the observed kinase selectivity. Our findings suggest that modular synthetic routes combined with in-depth structural characterization give access to selective molecular glue degraders and expansion of the CRBN-degradable proteome.
促进蛋白质-蛋白质相互作用的小分子可产生一系列治疗效果。分子胶降解剂因其紧凑的类药物结构以及无需依赖现有同源配体即可作用于靶点的能力,成为了这一概念的典型代表。虽然含有戊二酰亚胺支架的 Cereblon 分子胶降解剂已被批准用于治疗多发性骨髓瘤和急性髓系白血病,但设计新的具有治疗相关性的单价降解剂仍然具有挑战性。我们在此报告一种利用多组分反应作为核心模块化成核步骤来合成含戊二酰亚胺分子胶的方法。对所得文库进行筛选,鉴定出了靶向酪蛋白激酶 1α(CK1α)和类 Wee 蛋白激酶(WEE1)的 HRZ-01 衍生物。进一步的药物化学研究工作导致鉴定出了选择性单价 WEE1 降解剂,为最终开发选择性化学降解剂探针提供了一个潜在的起点。命中的 WEE1 降解剂与 CRBN-DDB1 和 WEE1 的复合物结构提供了蛋白质-蛋白质界面的模型以及观察到的激酶选择性的理论依据。我们的研究结果表明,模块化合成路线与深入的结构表征相结合能够获得选择性分子胶降解剂,并扩展 CRBN 可降解蛋白质组。