Basilea Pharmaceutica Ltd. announced results from the interim analysis of the registrational phase 2 study with the orally administered pan-fibroblast growth factor receptor (FGFR) kinase inhibitor derazantinib (BAL087). The analysis showed promising efficacy in patients with FGFR2 gene fusion-expressing intrahepatic cholangiocarcinoma (iCCA) and also confirmed the safety profile and tolerability of the drug candidate observed in previous clinical studies. The interim analysis in the ongoing registrational phase 2 study was conducted after 42 patients have been enrolled in the study, with a subset of 29 evaluable patients who had at least one post-baseline imaging assessment. The objective response rate (ORR) in these 29 patients was 21%. The disease control rate (DCR), reflecting the proportion of patients with a partial response or with stable disease, was 83%. The safety data obtained from all 42 patients enrolled to date was consistent with the results from previous clinical studies with derazantinib. The ongoing registrational open-label phase 2 study1 is expected to enroll up to 100 patients with inoperable or advanced iCCA expressing FGFR2 gene fusions. The patients receive once-daily oral derazantinib, to evaluate its anti-cancer activity with respect to objective response rate, progression free survival, overall survival and duration of response, and to further explore the safety and tolerability of the drug candidate. The additional cohort of iCCA patients whose tumors express FGFR gene mutations is expected to enroll approximately 50 patients.