VT-001

VT-001 is a potent and selective small-molecule inhibitor of the Ephrin type-B receptor 3 (EPHB3), currently in preclinical development for the treatment of Alzheimer's disease and other neurodegenerative conditions. Developed by Vincere Biosciences, the compound targets EPHB3 to modulate cellular interactions between astrocytes and microglia, which are key drivers of neuroinflammation in the brain. In preclinical studies using mouse models of amyloidosis (5xFAD), tauopathy (PS19), and multiple sclerosis (EAE), VT-001 demonstrated significant rescue of behavioral deficits and a robust attenuation of reactive astrocyte gene signatures and neuroinflammatory markers. The molecule is designed to be brain-penetrant and has shown excellent CNS exposure and tolerability in animal models.