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Cholangiocarcinoma News

FOENIX-CCA2: Phase 2, Open-Label, Multicenter Study of Futibatinib in Patients with iCCA Harboring FGFR2 Gene Fusions

Web Exclusives — May 28, 2020

Futibatinib is a highly selective irreversible FGFR1-4 inhibitor given as a continuous once-daily oral regimen. This phase 2 registrational trial was initiated following results from a phase 1 dose-escalation/expansion study showing tolerability and preliminary efficacy of futibatinib in patients with intrahepatic cholangiocarcinoma (iCCA) with FGFR2 fusions.1

This was a single-arm, multicenter phase 2 study enrolling patients with locally advanced/metastatic unresectable iCCA harboring FGFR2 gene fusions or other rearrangements, disease progression after ≥1 lines of systemic therapy (including gemcitabine plus platinum-based chemotherapy), no prior FGFR inhibitor treatment, and an ECOG performance status of 0 or 1.2 Patients received futibatinib 20 mg once daily until disease progression/unacceptable toxicity. The primary end point was objective response rate (ORR) based on independent central radiology review. Secondary end points include disease control rate (DCR), duration of response (DOR), and safety.

A total of 103 patients were enrolled in this study. For this interim analysis, data were reported for the 67 (65%) patients with ≥6 months of follow-up. Of these, 82.1% of patients had tumors harboring an FGFR2 fusion. One, 2, or ≥3 prior therapies were received by 44.8%, 28.4%, and 26.9% of patients, respectively. ORR was 34.3% (all partial response; n = 23), and DCR was 76.1%; assessment was pending for 8 patients. Median time to response was 1.6 months (range, 1.0-4.9 months), and median DOR was 6.2 months (range, 2.1-14.2 months). The most common treatment-related adverse events (all grade, grade ≥3) were hyperphosphatemia (79.1%, 25.4%), diarrhea (37.3%, 0%), and dry mouth (32.8%, 0%). Any-cause grade ≥3 adverse events were reported in 73.1% of patients. Dose delay or dose reduction was required in 65.7% and 53.7% of patients, respectively; 6.0% of patients discontinued treatment because of adverse events.

The authors concluded that preliminary data from this phase 2 study demonstrate that futibatinib may be efficacious in patients with locally advanced/metastatic unresectable iCCA harboring FGFR2 gene fusions who progress after ≥1 lines of chemotherapy. number NCT02052778.


  1. Rovithi M, et al. J Clin Oncol. 2019;37(5):411-418.
  2. Goyal L, et al. ASCO 2020. Abstract 108.

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