Junji Furuse1, Lipika Goyal2, Rastsilav Bahleda3, Juan Valle4, Markus Moehler5, Do-Youn Oh6, Heung-Moon Chang7, Robin Kate Kelley8, Milind Javle9, Mitesh Borad10, Li-Tzong Chen11, Nataliya Uboha12, Heinz-Josef Klümpen13, Peter J. O’Dwyer14, Daneng Li15, Chigusa Morizane16, Jerry Huang17, John A. Bridgewater18
1Department of Medical Oncology, Kyorin University, Tokyo, Japan;2Department of Hematology/Oncology, Massachusetts General Hospital, Boston, MA, USA;3Department of Drug Development, Gustave Roussy Cancer Campus, Villejuif, France;4Department of Medical Oncology, The Christie NHS Foundation Trust, Manchester, UK;5Division of Internal Medicine, Johannes Gutenberg-University of Mainz, Mainz, Germany;6Department of Internal Medicine, Seoul National University Hospital, Seoul, Republic of Korea;7Department of Oncology, Asan Medical Center, Seoul, Republic of Korea;8Department of Medicine, University of California San Francisco, Helen Diller Family Comprehensive Cancer Center, San Francisco, CA, USA;9Division of Gastrointestinal Medical Oncology, The University of Texas, MD Anderson Cancer Center, Houston, TX, USA;10Department of Internal Medicine, Mayo Clinic Cancer Center, Rochester, MN, USA;11National Institute of Cancer Research, National Health Research Institutes, Tainan, Taiwan;12Department of Medicine, University of Wisconsin Carbone Comprehensive Cancer Center, Madison, WI, USA;13Department of Medical Oncology, Academic Medical Center, Amsterdam, The Netherlands;14Department of Medicine, University of Pennsylvania Abramson Cancer Center, Philadelphia, PA, USA;15Department of Medical Oncology and Therapeutics Research, City of Hope National Medical Center, Duarte, CA, USA;16Department of Hepatobiliary and Pancreatic Oncology, National Cancer Center Hospital, Tokyo, Japan;17Department of Clinical Development, Taiho Oncology, Inc., Princeton, New Jersey, NJ, USA;18Research Department of Oncology, UCL Cancer Institute, London, UK
Correspondence to: Junji Furuse. Department of Medical Oncology, Kyorin University, Tokyo, Japan. Email: jfuruse@ks.kyorin-u.ac.jp.
Abstract: TAS-120 is a highly selective, irreversible fibroblast growth factor receptor (FGFR) 1–4 inhibitor in development as a once-daily oral treatment for intrahepatic cholangiocarcinoma (iCCA). An ongoing phase 1/2 study of TAS-120 in CCA showed tolerability and preliminary efficacy, particularly in patients with FGFR2 rearrangements. The purpose of FOENIX-CCA2 (NCT02052778) is to evaluate the efficacy and safety of TAS-120 in patients with iCCA with FGFR2 rearrangements. FOENIX-CCA2 is a global, single-arm study of TAS-120 in patients with iCCA bearing FGFR2 gene fusions and other rearrangements including deletion, duplication, truncation, or rearrangement of unknown significance. The study will enroll approximately 100 patients with locally advanced or metastatic iCCA that progressed after ≥1 systemic therapies and with an ECOG PS of 0 or 1. Prior systemic therapy must include gemcitabine plus platinum-based chemotherapy and no prior FGFR inhibitor. Screening for FGFR2 gene rearrangements will be performed at a central laboratory or locally and confirmed by a central laboratory. The primary endpoint is objective response rate based on RECIST v1.1. Secondary endpoints include duration of response, disease control rate, overall survival, progression-free survival, safety, and health-related quality of life.
Keywords: Intrahepatic cholangiocarcinoma (iCCA); FGFR2 gene rearrangements; TAS-120
