Characteristics of the tumor microenvironment in IDH1-mutated cholangiocarcinoma patients from ClarIDHy trial

• Somatic isocitrate dehydrogenase 1 mutations
(IDH1m) convert α-ketoglutarate to the oncogenic
metabolite R-2-hydroxyglutarate (2-HG).IDH1m
are detected in approximately 13% of intrahepatic
cholangiocarcinomas (CCAs).

• Ivosidenib (IVO), an oral inhibitor of the IDH1m
protein inhibits 2-HG, and data suggests restores
immune response in CCA.

• In the global, phase 3 ClarIDHy study evaluating
IVO vs placebo (PBO) in patients with
nonresectable or metastatic IDH1m CCA
(ClarIDHy, ClinicalTrials.gov NCT02989857) :
• IVO demonstrated a favorable safety profile
• IVO significantly improved
progression-free survival vs PBO
(HR = 0.37, p < 0.0001)