Mitapivat

国际站：Mitapivat

CAS：1260075-17-9

品牌：MedChemExpress (MCE)

存储条件：Powder: -20°C, 3 years; 4°C, 2 years.In solvent: -80°C, 6 months; -20°C, 1 month.

生物活性：Mitapivat (AG-348) 是一种具有口服活性的丙酮酸激酶变构激活剂。 Mitapivat 在广泛的 PKLR 基因型范围内提高酶活性、蛋白质稳定性和 ATP 水平，显示出恢复PK（丙酮酸激酶） 缺陷糖酵解途径活性的潜力. Mitapivat 可用于PK缺陷的研究^[1][2][3]。 IC50 和目标：丙酮酸激酶^[1][2][3]。 

体外：Mitapivat (0.1 nM-100 µM; 16 h) 激活健康供体红细胞中的 WT PK-R^[1]。Mitapivat (0.01 nM-10 µM; 16 h) 促进产生红细胞中的 ATP 呈剂量依赖性^[1]。

体内：Mitapivat（50 mg/kg；口服；每天两次，持续 21 天）改善 β-地中海贫血小鼠模型的贫血^[2]。

参考文献：[1]. Kung C, et al. AG-348 enhances pyruvate kinase activity in red blood cells from patients with pyruvate kinase deficiency. Blood. 2017 Sep 14;130(11):1347-1356.[2]. Matte A, et al. The pyruvate kinase activator mitapivat reduces hemolysis and improves anemia in a β-thalassemia mouse model. J Clin Invest. 2021 May 17;131(10):e144206.[3]. Rab MAE, et al. AG-348 (Mitapivat), an allosteric activator of red blood cell pyruvate kinase, increases enzymatic activity, protein stability, and ATP levels over a broad range of PKLR genotypes. Haematologica. 2021 Jan 1;106(1):238-249.