BPI-221351

BPI-221351 is a novel, brain-penetrant, highly potent, and selective small molecule dual inhibitor of mutant isocitrate dehydrogenase 1 (IDH1) and 2 (IDH2). Developed by Betta Pharmaceuticals, it is designed to treat solid tumors harboring IDH1 or IDH2 mutations, including glioblastoma and biliary tract cancers. The drug demonstrates high blood-brain barrier penetration, with brain concentrations exceeding plasma levels, making it particularly suitable for primary brain tumors. It works by suppressing the production of the oncometabolite 2-hydroxyglutarate (2-HG), which is elevated in mutant IDH cells and drives oncogenesis. BPI-221351 is currently in the preclinical and IND-enabling stage of development.