Acoramidis hydrochloride 是具有口服活性的、选择性的甲状腺素转运蛋白 (TTR (transthyretin)) 的稳定剂，对野生型和 V1221 突变型均有效。Acoramidis hydrochloride 可用于转体甲状腺素淀粉样变性的研究。

Acoramidis hydrochloride is an orally active and selective kinetic stabilizer of WT and V122I- TTR (transthyretin) . Acoramidis hydrochloride is used in the study for transthyretin amyloidosis.

Acoramidis (AG10, 0.1-10 μM for TTR ～5 μM) stabilizes V122I- and WT-TTR equally well and also exceeds their efficacy to stabilize WT and mutant TTR in whole serum [1] . Acoramidis (AG10) stimulates the mitochondrial QO2 in a concentration-dependent manner between 10 and 100 μM [3] . Acoramidis (AG10) has very minimal inhibition of two common off-targets in drug discovery, the potassium ion channel hERG (IC 50 >100 μM) and a number of cytochrome P450 isozymes (IC 50 >50 μM) (low toxicity) [1] . Western Blot Analysis [1] . Cell Line: Human serum (TTR ～5 μM). Concentration: 0.1 and 10 μM. Incubation Time: 72 h. Result: Was significantly more effective than tafamidis in stabilizing TTR. The concentration of AG10 to 10 μM resulted in stabilization of almost all of TTR in serum.

Animal Model: Wistar rats [1] . Dosage: 50 mg/kg/d (Toxicity Analysis). Administration: Oral gavage, daily for 28 d. Result: Showed the plasma C max of ～40 μM and histopathological evaluation of liver, kidney, heart, spleen, thymus, and lung showed no signs of pathologic processes in the AG10-treated animals

2242751-53-5

C15H18ClFN2O3

328.77

Alxn2060 hydrochloride;AG10 hydrochloride;Acoramidis hydrochloride

DMSO:58.5mg/mL (177.9mM),Need ultrasonic
Powder: -20°C for 3 years
In solvent: -80°C for 2 years