safusidenib

Safusidenib is an orally available small molecule inhibitor that selectively targets mutant forms of isocitrate dehydrogenase type 1 (IDH1), particularly those with substitution mutations at arginine position 132 (IDH1 R132). By binding to and inhibiting these mutant IDH1 enzymes, safusidenib blocks the production of the oncometabolite 2-hydroxyglutarate from alpha-ketoglutarate. This inhibition prevents abnormal signaling pathways that drive tumor growth and block cellular differentiation in cancers such as glioma. Safusidenib has demonstrated brain penetration and antitumor activity in preclinical models and early clinical trials for patients with IDH1-mutated gliomas. It minimally affects wild-type IDH1 found in normal cells[5][7][3][6].

Brand names

safusidenib

Other names

safusidenib(2E)-3-[1-[[5-(1-Fluoro-1-methylethyl)-3-(2,4,6-trichlorophenyl)-4-isoxazolyl]carbonyl]-3-methyl-1H-indol-4-yl]-2-propenoic acid2-Propenoic acid, 3-[1-[[5-(1-fluoro-1-methylethyl)-3-(2,4,6-trichlorophenyl)-4-isoxazolyl]carbonyl]-3-methyl-1H-indol–4–yl]-, (2E)-