VX-01

VX-01 is an orally administered small molecule therapy developed by Vantage Biosciences for the treatment of diabetic eye diseases, primarily non-proliferative diabetic retinopathy (NPDR) and metabolic dysfunction-associated steatohepatitis (MASH). It acts as a first-in-class inhibitor of amine oxidase copper-containing 3 (AOC-3), targeting neurovascular inflammation in the retina. By inhibiting AOC-3, VX-01 reduces leukocyte trafficking and pro-inflammatory products of local amine oxidation, aiming to slow or prevent disease progression before irreversible vision loss occurs. The drug is suitable for daily oral chronic dosing to achieve persistent inhibition within the neurovascular unit[1][3][4][5]. Clinical trials are ongoing to evaluate its efficacy in NPDR without center-involved diabetic macular edema[1][7].