AG-10 (0.1-10 μM for TTR ∼5 µM) stabilizes WT- and V122I-TTR equally well and also exceeds their efficacy to stabilize WT and mutant TTR in whole serum. AG-10 exhibits minimal inhibition of two common off-targets in drug discovery, the potassium ion channel hERG (IC50 > 100 µM) and a number of cytochrome P450 isozymes (IC50 > 50 µM) (low toxicity)[1]. AG-10 (10-100 μM) stimulates the mitochondrial QO2 in a concentration-dependent manner[2].

AG-10 (Oral gavage; 50 mg/kg/d; daily for 28 d) exhibited the plasma Cmax of ∼40 µM and histopathological evaluation of liver, thymus, kidney, spleen, heart, and lung showed no signs of pathologic processes in the AG10-treated Wistar rats[1].