GO inhibitor for primary hyperoxaluria type 1 (PH1) and recurrent kidney stone formers
5,000 / 1.5M
PH1 and FSF
BBP-711 is an orally-administered small molecule inhibitor of glycolate oxidase (GO) that is being developed to treat conditions of excess oxalate, including primary hyperoxaluria type 1 (PH1) and frequent kidney stone formation. In PH1, loss of function mutation in the AGXT gene results in accumulation of glyoxylate, which is converted into oxalate and leads to kidney stones and organ damage. Targeting GO is a clinically validated approach to reduce urinary oxalate by lowering the concentration of glyoxylate. The first-in-human Phase 1 trial of BBP-711 for hyperoxaluria was initiated in May 2021.