Futibatinib in Patients with Intrahepatic CCA Harboring FGFR2 Gene Fusions: A Phase 2 Open-Label Study (FOENIX-CCA2)

2020 Year in Review - Cholangiocarcinoma —December 20, 2020

Preliminary results of the FOENIX-CCA2 study show that futibatinib may be effective and well-tolerated in patients with locally advanced or metastatic unresectable intrahepatic CCA harboring FGFR2 gene fusions following failure of prior lines of chemotherapy.

Futibatinib (TAS-120), administered as a continuous once-daily oral regimen, is a highly selective, irreversible fibroblast growth factor receptor (FGFR)1-4 inhibitor. The FOENIX-CCA2 phase 2 clinical trial was initiated after phase 1 dose-escalation/expansion study results showed the preliminary efficacy and tolerability of futibatinib in patients with intrahepatic CCA and FGFR2 fusions.1

At this year’s American Society of Clinical Oncology (ASCO) annual meeting, Lipika Goyal, MD, MPhil, Medical Oncologist, Tucker Gosnell Center for Gastrointestinal Cancers, Massachusetts General Hospital, Boston, presented the phase 2 study results. “The interim analysis demonstrated that treatment with the covalently binding FGFR inhibitor futibatinib may lead to meaningful clinical benefit in patients with refractory [intrahepatic] CCA with FGFR2 gene fusions or other rearrangements,” said Dr Goyal. “In a disease with limited treatments, this drug could be an effective and well-tolerated option for patients and the oncologists that care for them.”

Patients enrolled in this single-arm, multicenter phase 2 clinical trial had locally advanced or metastatic unresectable intrahepatic CCA that harbors FGFR2 gene fusions or other rearrangements, disease progression after ≥1 lines of systemic therapy (including gemcitabine plus platinum-based chemotherapy), no previous FGFR inhibitor treatment, and an Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1.2 Patients received futibatinib 20 mg once daily until disease progression or unacceptable toxicity. The primary end point was objective response rate (ORR) based on independent central radiology review. The secondary end points were disease control rate, duration of response, and safety.

A total of 103 patients were enrolled in the study. For this interim analysis, data were reported for the 67 (65%) patients with ≥6 months of follow-up, 67 (82.1%) of whom had tumors harboring an FGFR2 fusion; 44.8%, 28.4%, and 26.9% of patients, respectively, received a total of 1, 2, or ≥3 previous therapy regimens.

The ORR was 34.3% (N = 23), all partial responses, and the disease control rate was 76.1%; assessment was pending for 8 patients. Median time to response to therapy was 1.6 months (range, 1.0-4.9 months), and median duration of response was 6.2 months (range, 2.1-14.2 months). The most common all-grade and grade ≥3 treatment-related adverse events were hyperphosphatemia (79.1%, 25.4%, respectively), diarrhea (37.3%, 0%), and dry mouth (32.8%, 0%).

All-cause grade ≥3 adverse events were reported in 73.1% of patients. A dosing delay was required in 65.7% of patients, and dose reduction was required in 53.7% of patients; adverse events caused 6% of the patients to discontinue treatment.

The investigators concluded that the preliminary data from this study show that futibatinib may be efficacious in patients with locally advanced or metastatic unresectable intrahepatic CCA harboring FGFR2 gene fusions that progress after ≥1 lines of chemotherapy.

Source: Goyal L, et al. J Clin Oncol. 2020;38(suppl_15). Abstract 108.


  1. Meric-Bernstam F, Arkenau H, Tran B, et al. Efficacy of TAS-120, an irreversible fibroblast growth factor receptor (FGFR) inhibitor, in cholangiocarcinoma patients with FGFR pathway alterations who were previously treated with chemotherapy and other FGFR inhibitors. Ann Oncol. 2018;29(suppl 5):Abstract O-001.
  2. Goyal L, Meric-Bernstam F, Hollebecque A, et al. FOENIX-CCA2: a phase II, open-label, multicenter study of futibatinib in patients (pts) with intrahepatic cholangiocarcinoma (iCCA) harboring FGFR2 gene fusions or other rearrangements. J Clin Oncol. 2020;38(15_suppl). Abstract 108.
Last modified: March 11, 2021

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