Enpatoran (M5049) 是一种口服有效的TLR7/8选择性抑制剂，在HEK293 细胞中，IC50s 分别为 11.1 nM 和 24.1 nM。Enpatoran 可以阻断分子合成配体和天然内源性 RNA 配体。Enpatoran 在体内具有良好的药代动力学特性。Enpatoran 在先天性和适应性自身免疫阻断方面有研究的价值。

Enpatoran is an inhibitor of TLR7(IC50 = 11.1 nM) and TLR8(IC50 = 24.1 nM). Enpatoran was found to be inactive against TLR3, TLR4, and TLR9 in vitro and in vivo.

Enpatoran inhibited activity against different TLRs in a variety of cellular systems. Enpatoran potently inhibited TLR7 and TLR8 in HEK NF-κB-luciferase reporter cells with IC50 values in the low nanomolar range. Additionally, Enpatoran was found to be potent against TLR7 and TLR8 in PBMCs as well as whole blood. There appears to be an increased selectivity for TLR7 inhibition relative to TLR8 for Enpatoran in whole blood compared with PBMCs and transfected HEK cells[1].

In the BXSB-Yaa model, there is a robust increase in multiple autoantibodies, and Enpatoran significantly reduced both DNA-containing reactivities such as anti-dsDNA and anti-histones, as well as RNA-binding protein reactivities such as anti-RiboP and anti-SmRNP. At high dosages of Enpatoran, the effect on autoantibodies is so profound that titers are reduced below the baseline levels[1].

2101938-42-3

C16H15F3N4

320.31

DMSO:24.44 mg/mL (76.30 mM)
Powder: -20°C for 3 years
In solvent: -80°C for 2 years