Vorasidenib is a highly potent (IC50s = 7 nM IDH2-R140Q, 0.6 nM IDH1-R132H/WT, and 0.25 nM Neurosphere TS603 IDH1-R132H), selective, and brain-penetrant inhibitor of mutant isocitrate dehydrogenase 1 and 2 (IDH1/2).1 It led to >97% inhibition of 2-hydroxyglutarate production in mIDH1 glioma tissue. Vorasidenib significantly improved progression-free survival and delayed time to next intervention in a phase 3 glioma trial.2
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