Acoramidis (AG10) 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 (AG10) hydrochloride 可用于转体甲状腺素淀粉样变性的研究。

Acoramidis (AG10) hydrochloride is an orally active and selective kinetic stabilizer of WT and V122I-TTR (transthyretin). Acoramidis (AG10) hydrochloride is used in the study for transthyretin amyloidosis.

Solid

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.
Acoramidis (AG10) stimulates the mitochondrial QO2 in a concentration-dependent manner between 10 and 100 μM.
Acoramidis (AG10) has very 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).
has not independently confirmed the accuracy of these methods. They are for reference only.

Animal Model: Wistar rats. Dosage: 50 mg/kg/d (Toxicity Analysis).

Room temperature in continental US; may vary elsewhere.

-20°C, sealed storage, away from moistur In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)

[1]. Sravan C Penchala, et al. AG10 inhibits amyloidogenesis and cellular toxicity of the familial amyloid cardiomyopathy-associated V122I transthyretin. Proc Natl Acad Sci U S A. 2013 Jun 11;110(24):9992-7.
[2]. Jonathan C Fox, et al. First-in-Human Study of AG10, a Novel, Oral, Specific, Selective, and Potent Transthyretin Stabilizer for the Treatment of Transthyretin Amyloidosis: A Phase 1 Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Study in Healthy Adult Volunteers. Clin Pharmacol Drug Dev. 2020 Jan;9(1):115-129.

In Vitro: DMSO : 62.5 mg/mL (190.10 mM; Need ultrasonic)H₂O : < 0.1 mg/mL (ultrasonic;warming;heat to 60°C) (insoluble)配制储备液