BMN 255

BMN 255 is a potent, orally bioavailable small molecule inhibitor of glycolate oxidase (GO), developed by BioMarin Pharmaceutical. Its primary mechanism of action is the inhibition of glycolate oxidase, which reduces the production of oxalate—a compound that can contribute to kidney stone formation and hyperoxaluria. The drug was investigated for the treatment of hyperoxaluria, particularly in patients with non-alcoholic fatty liver disease (NAFLD) and metabolic dysfunction-associated steatohepatitis (MASH). Clinical trials aimed to assess its safety and efficacy in lowering urinary oxalate levels to prevent kidney stones and related complications. As of April 2024, development for these indications was discontinued due to portfolio prioritization rather than safety concerns[1][2][3][4][5].