Basilea provides more evidence of derazantinib's potential in iCCA

Swiss biotech Basilea Pharmaceutica (SIX: BSLN) has announced positive results of a pre-planned interim analysis in cohort 2 of the Phase II study FIDES-01 (Fibroblast growth factor Inhibition with DErazantinib in Solid tumors), which is assessing the anti-tumor efficacy of the orally-administered fibroblast growth factor receptor (FGFR) inhibitor, derazantinib, in patients with inoperable or advanced intrahepatic cholangiocarcinoma (iCCA), a form of bile duct cancer.

After cohort 1 of the study provided the clinical proof of concept for derazantinib monotherapy in the treatment of iCCA patients with FGFR2 gene fusions, cohort 2 is enrolling iCCA patients with FGFR2 gene mutations or amplifications. The efficacy data obtained in the interim analysis met the pre-specified threshold so that the study will proceed to the next stage as planned.

The interim analysis of cohort 2 is based on 14 evaluable patients who had at least one post-baseline tumor assessment. The pre-specified criterion that at least eight patients met the primary endpoint of obtaining progression-free survival (PFS) of at least three months was successfully achieved. The positive interim analysis allows the study to advance to its next stage and enrol a total of 43 patients. As a number of patients are still ongoing with treatment the median PFS was not yet mature at the time of the interim analysis and will be defined at a later time point.