Ivosidenib 是一种口服具有活力的异柠檬酸脱氢酶 1 的突变体酶 (mIDH1 enzyme) 抑制剂，能够使 d-2- hydroxyglutatrate (2-HG) 在体内降低。它具有良好的的安全性和临床活性，具有研究 AML 的潜力。

Ivosidenib is an orally available inhibitor of isocitrate dehydrogenase type 1 (IDH1), with potential antineoplastic activity. Ivosidenib specifically inhibits a mutated form of IDH1 in the cytoplasm, which inhibits the formation of the oncometabolite, 2-hydroxyglutarate (2 hG). This may lead to both an induction of cellular differentiation and an inhibition of cellular proliferation in IDH1-expressing tumor cells.

TF-1 cells or primary human AML patient samples expressing mutant IDH1 are treated with AG-120.Treatment with AG-120 decreases intracellular 2-HG levels, inhibites growth factor independent proliferation and restores erythropoietin (EPO)-induced differentiation in TF-1 IDH1-R132H cells. Similarly, pharmacological inhibition of mutant IDH1 enzyme with AG-120 in primary human blast cells cultured ex vivo provides an effective way to lower intracellular 2-HG levels and induces myeloid differentiation[1].

TF-1 cells or primary human AML patient samples expressing mutant IDH1 are treated with AG-120. (Only for Reference)

1448347-49-6

C28H22ClF3N6O3

582.97

AG-120;艾伏尼布

H2O:<1 mgml
DMSO:39 mg/mL
Ethanol:100 mg/mL (171.54 mM)
Powder: -20°C for 3 years
In solvent: -80°C for 2 years