ESMO Asia 2020: Futibatinib for Patients With Refractory Intrahepatic Cholangiocarcinoma and FGFR2 Fusions/Rearrangements

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Clinical benefit was provided by the FGFR1–4 inhibitor futibatinib in patients with refractory intrahepatic cholangiocarcinoma harboring FGFR2 gene fusions or other rearrangements, including those enrolled in Asian countries. These findings were presented by Furuse et al at the ESMO Asia Virtual Congress 2020 (Abstract 116M0).

Data was presented from a preplanned interim analysis of the global phase II FOENIX-CCA2 study, which evaluated the efficacy, safety, and quality of life of futibatinib, a highly selective irreversible FGFR1–4 inhibitor, in patients with intrahepatic cholangiocarcinoma and FGFR2 fusions and/or rearrangements.


FOENIX-CCA2 enrolled 103 patients with unresectable/metastatic intrahepatic cholangiocarcinoma and FGFR2 fusion/rearrangements who experienced disease progression after one or more lines of systemic therapy that included gemcitabine/cisplatin but not prior FGFR inhibitors.

All patients received oral futibatinib at 20 mg once daily until disease progression or intolerability.

The primary endpoint of the study was objective response rate per independent central radiology review and Response Evaluation Criteria in Solid Tumors version 1.1; secondary endpoints included disease control rate, duration of response, progression-free survival, safety, and patient-reported outcomes. Subgroup analysis of objective response rate (per baseline demographic, fusion partner, and the presence of other molecular alterations, such as TP53) was also performed.

This planned interim analysis reported data from 67 patients who had a minimum follow-up period of 6 months; of these, 54% of patients were White and 24% were Asian; 55% of patients had received two or more prior therapy lines. Eighty-two percent of patients had tumors with an FGFR2 fusion, and BICC1 was the fusion partner in 15 patients.

Activity of Futibatinib

An objective response rate of 37.3% was reported in this interim analysis. The disease control rate was 82.1%, and median duration of response was 8.3 months.

Objective responses occurred in all subgroups of patients evaluated and were consistent across baseline characteristics that included age, FGFR2 fusion partner (BICC1; 33.3%), or the presence of another genetic mutation (TP53; 16.7%). Notably, an objective response rate of 57.1% was observed in the subgroup of patients aged ≥ 65 years.

The median progression-free survival was 7.2 months. Patient-reported outcomes remained stable through 273 days (13 cycles) of treatment.

The most common any-grade treatment-related adverse events included hyperphosphatemia (in 81% of patients), diarrhea in 37%, and dry mouth in 33% of patients. Grade 3 hyperphosphatemia occurred in 27% of patients. No grade 4 or 5 treatment-related adverse events were reported. Treatment-related adverse events were managed with dosing interruption in 55% of patients and with dose reduction in 51%; one patient discontinued treatment due to a treatment-related adverse event.

According to the authors, futibatinib provided durable objective responses in patients with refractory intrahepatic cholangiocarcinoma and FGFR2 fusions/rearrangements, including within patient subgroups. In addition, adverse events were manageable, and quality of life as assessed by patient-reported outcomes was maintained throughout treatment.

Disclosure: The study was funded by Taiho Oncology, Inc, and Taiho Pharmaceutical Co, Ltd. For full disclosures of the study authors, visit

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