AH001

AH001 is an orally active compound that binds to a concealed pocket near GDP within RhoA, exhibiting a binding affinity of 73.16 nM. By interacting with GDP, AH001 stabilizes the interaction between RhoA and its endogenous inhibitor, RhoGDIα. This compound reduces nuclear translocation of downstream MRTFA and downregulates fibrosis/hypertrophy-related proteins. AH001 alleviates myocardial remodeling in various heart failure animal models and 3D cardiac tissue models. Its cardioprotective effects are mediated through the RhoA-RhoGDIα axis, effectively inhibiting downstream RhoA activation signals.