Acoramidis hydrochloride (Alxn2060 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 effectively, outperforming their efficacy in whole serum [1]. Acoramidis (AG10) stimulates mitochondrial QO2 concentration-dependently between 10 and 100 μM [3]. It exhibits minimal inhibition of the potassium ion channel hERG (IC50 > 100 μM) and several cytochrome P450 isozymes (IC50 > 50 μM) [low toxicity] [1]. Western Blot Analysis [1], using human serum (TTR ～5 μM), at concentrations of 0.1 and 10 μM, with a 72-hour incubation, shows AG10 is significantly more effective than tafamidis in stabilizing TTR, with 10 μM stabilizing almost all 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 Acoramidis-treated animals