American Association for Cancer Research
cd-21-0697_supplementary_methods_tables_and_figure_suppsm1.pdf (211.81 kB)

Supplementary Methods, Tables, and Figure from Futibatinib, an Irreversible FGFR1–4 Inhibitor, in Patients with Advanced Solid Tumors Harboring FGF/FGFR Aberrations: A Phase I Dose-Expansion Study

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journal contribution
posted on 2023-04-03, 23:40 authored by Funda Meric-Bernstam, Rastislav Bahleda, Cinta Hierro, Marc Sanson, John Bridgewater, Hendrik-Tobias Arkenau, Ben Tran, Robin Kate Kelley, Joon Oh Park, Milind Javle, Yaohua He, Karim A. Benhadji, Lipika Goyal

Supplementary Appendix including all supplementary tables and figures


Taiho Pharmaceutical (Taiho Pharma)

Taiho Oncology



Futibatinib, a highly selective, irreversible FGFR1–4 inhibitor, was evaluated in a large multihistology phase I dose-expansion trial that enrolled 197 patients with advanced solid tumors. Futibatinib demonstrated an objective response rate (ORR) of 13.7%, with responses in a broad spectrum of tumors (cholangiocarcinoma and gastric, urothelial, central nervous system, head and neck, and breast cancer) bearing both known and previously uncharacterized FGFR1–3 aberrations. The greatest activity was observed in FGFR2 fusion/rearrangement–positive intrahepatic cholangiocarcinoma (ORR, 25.4%). Some patients with acquired resistance to a prior FGFR inhibitor also experienced responses with futibatinib. Futibatinib demonstrated a manageable safety profile. The most common treatment-emergent adverse events were hyperphosphatemia (81.2%), diarrhea (33.5%), and nausea (30.4%). These results formed the basis for ongoing futibatinib phase II/III trials and demonstrate the potential of genomically selected early-phase trials to help identify molecular subsets likely to benefit from targeted therapy. This phase I dose-expansion trial demonstrated clinical activity and tolerability of the irreversible FGFR1–4 inhibitor futibatinib across a broad spectrum of FGFR-aberrant tumors. These results formed the rationale for ongoing phase II/III futibatinib trials in cholangiocarcinoma, breast cancer, gastroesophageal cancer, and a genomically selected disease-agnostic population.This article is highlighted in the In This Issue feature, p. 275

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