Abstract
Background Cholangiocarcinoma (CCA), an aggressive epithelial neoplasm of biliary tract, presents limited treatment options and poor overall survival. Aberrant fibroblast growth factor receptor (FGFR) signaling has been implicated in CCA carcinogenesis especially in intrahepatic cholangiocarcinoma (iCCA). Debio 1347 is an orally available selective FGFRi with potent antitumor effect in preclinical models of cancer bearing FGFR alterations. Debio 1347 showed encouraging preliminary clinical activity and manageable treatment-emergent adverse events (TEAE) in its first-in-human (FIH) Ph1 study (NCT1948297). Here we report results from the patients with CCA patients enrolled.
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