Abstract
What is this summary about?
This summary describes the results from a phase 2 study called FOENIXCCA2. The study evaluated treatment with futibatinib in people with a rare form of advanced bile duct cancer called intrahepatic cholangiocarcinoma (or iCCA), where the tumors have changes in the structure of a gene called FGFR2. These changes include FGFR2 gene fusions. Bile duct cancer often returns after surgery or cannot be treated by surgery because the tumor has spread, so it requires treatment with chemotherapy. People live for a median of 1 year after their first chemotherapy treatment and 6 months after their second treatment. This study included people whose cancer had grown/spread after one or more chemotherapy treatments. The aims of the study were to see if futibatinib could shrink the size of tumors and stop the cancer from growing/spreading and to see how long people lived when treated with futibatinib. Clinicians also looked at side effects from taking futibatinib and at how it affected people's quality of life.
What were the results?
Futibatinib treatment shrank tumors in over 80% of people who received treatment. Tumors shrank by at least 30% in 42% of people. Futibatinib stopped tumors from growing/spreading for a median of 9.7 months. People who took the medicine lived for a median of 21.7 months, and 72% of people were still alive after 1 year. Side effects from taking futibatinib were like those reported for similar medicines, and clinicians considered the side effects to be manageable by adjusting the dose of futibatinib or treating the side effects. Most people reported that their quality of life stayed the same or improved during the first 9 months of taking futibatinib.
What do the results mean?
The results support the use of futibatinib for treating people with advanced bile duct cancer. Based on the results of this study, futibatinib is now approved in the US, Europe, and Japan. Futibatinib is approved for treating adults with advanced bile duct cancer who have received previous treatment for their cancer, and whose tumors have a gene fusion or other change in the FGFR2 gene.
Clinical Trial Registration: NCT02052778 (FOENIX-CCA2)
Keywords: :
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Financial disclosure
This study was sponsored by Taiho Oncology, Inc., and Taiho Pharmaceutical Co., Ltd. LG has received consulting fees from Alentis, Basilea, Black Diamond, Blueprint Medicines Corporation, Eisai, Exelixis, Genentech USA, Inc., H3Biomedicine, Incyte Corporation, Kinnate, Merck, QED Therapeutics, Servier, Sirtex Medical Inc., Synthekine, Taiho Pharmaceutical Co., Transthera Biosciences, and Tyra Therapeutics. FM-B has received consulting fees from Alkermes, AstraZeneca, Daiichi Sankyo Company, DebioPharm, Genentech, Inc., Harbinger Health, Jackson Laboratory, Lengo Therapeutics, OrigiMed, Protai Bio, Samsung Bioepis, Tallac Therapeutics, Tyra Biosciences, and Xencor; has received grant or research support from Aileron Therapeutics, AstraZeneca, Bayer, Calithera Biosciences, Curis Inc., CytomX Therapeutics, Daiichi Sankyo Company, DebioPharm, eFFECTOR Therapeutics, Genentech, Inc., Guardant Health, Novartis, Puma Biotechnology, and Taiho Pharmaceutical Co.; has participated in an advisory committee or advisory board meeting for AbbVie, Aduro BioTech, Biovica, Black Diamond, eFFECTOR Therapeutics, Eisai, F. Hoffman-La Roche, FogPharma, IBM Watson, Immunomedics, Infinity Pharmaceutical, Inflection Biosciences, Karyopharm Therapeutics, Kolon Life Science, LOXO Oncology, Mersana Therapeutics, OnCusp Therapeutics, PACT Pharma, Parexel International, Pfizer, Puma Biotechnology, Sanofi US Services Inc., Seattle Genetics, Silverback Therapeutics, Spectrum Pharmaceuticals, Zentalis, and Zymeworks; and is the chair of Investigational Cancer Therapeutics for MD Anderson Cancer Center. AH has received consulting fees from Amgen, Basilea Pharmaceutica, Bristol Myers Squibb, Incyte Corporation, Relay Therapeutics, and Servier; and has received grant or research support from Incyte Corporation. JWV has received consulting fees from AstraZeneca, Cantargia, Debiopharm, Delcath, Genoscience Pharma, Incyte Corporation, Merck, Mundipharma EDO, NuCana, QED Therapeutics, Servier, Sirtex Medical Inc., Taiho Oncology, Inc., and Zymeworks; and travel support from NuCana. CM has received grant or research support from AstraZeneca, Daiichi Sankyo Company, Eisai, Hitachi, J-Pharma, Merck, Ono Pharmaceutical, Taiho Pharmaceutical Co., and Yakult Honsha; has participated in advisory boards for AstraZeneca, Merck, MSD, Servier, Taiho Pharmaceutical Co., and Yakult Honsha; and has received honoraria from Eisai, MSD, Novartis, Teijin Pharma, and Yakult Honsha. TBK has received consulting fees from AstraZeneca, Incyte Corporation, Ipsen Biopharmaceuticals Inc., Pfizer, and Taiho Oncology, Inc.; and has received grant or research support from Bristol Myers Squibb, Eli Lilly and Company, and Xencor. TAA has received consulting fees from AstraZeneca, Eisai, Eli Lilly and Company, Exelixis Inc., Ipsen Biopharmaceuticals Inc., and Merck. JF has received consulting fees from Astellas Pharma, AstraZeneca, Fujifilm, Incyte Corporation, Merck Bio, MSD, Onco Therapy Science, Ono Pharmaceutical, and Taiho Pharmaceutical Co.; has received grant or research support from Astellas Pharma, AstraZeneca, Eisai, J-Pharma, Merck Bio, MSD, Ono Pharmaceutical, and Taiho Pharmaceutical Co.; and has received honoraria from Bayer, Chugai, Eisai, Eli Lilly Japan, MSD, Novartis, Ono Pharmaceutical, and Yankult Honsha. RKK has received consulting fees from Agios Pharmaceuticals Inc., Exact Sciences, Exelixis Inc., Gilead Sciences, Kinnate, and Merck; and has received grant or research support from Agios Pharmaceuticals Inc., AstraZeneca, Bayer, Bristol Myers Squibb, Eli Lilly and Company, EMD Serono, Exelixis Inc., Genentech Inc., Merck, Novartis, Partner Therapeutics Inc., Roche, Surface Oncology, and Taiho Pharmaceutical Co. PAC has received grant or research support from AbbVie, Amgen, AstraZeneca, Bayer, Blueprint Medicines Corporation, Bristol Myers Squibb, Eli Lilly and Company, EMD Serono, Exelixis, F. Hoffmann-La Roche, GlaxoSmithKline, Janssen Biotech, Merck Sharp and Dohme, Novartis Pharma, Taiho Oncology, Inc., and Toray Industries; and has participated in advisory boards for EMD Serono and Janssen Biotech. H-JK has participated in advisory boards for AstraZeneca, Janssen Global Services, and Merck. H-MC is a principal investigator in clinical trials for AbbVie, Astellas, BeiGene USA Inc., Celgene Corporation, Golden Biotechnology Corporation, Senhwa Biosciences Inc., and Taiho Oncology, Inc. L-TC has received grant or research support from ACT Genomics Taiwan and Taiho Pharmaceutical Co.; and has received honoraria from ACT Genomics Taiwan. JT has received consulting fees from Array BioPharma, AstraZeneca, Bayer, Boehringer Ingelheim, Chugai, Daiichi Sankyo Company, Eli Lilly and Company, F. Hoffmann-La Roche, Genentech, Inc., HalioDX SAS, Hutchinson MediPharma International, Ikena Oncology, Inspirna Inc., IQVIA, Menarini Ricerche S.p.A, Merck Serono, Merus N.V., Mirati, MSD, Neophore, Novartis, Ona Therapeutics, Orion Biotechnology, Peptomyc, Pfizer, Pierre Fabre, Samsung Bioepis, Sanofi, Scandion Oncology, Seattle Genetics, Servier, Sotio Biotech, Taiho Pharmaceutical Co., Tessa Therapeutics, and Theramyc. D-YO has received grant or research support from Array BioPharma, AstraZeneca, BeiGene, Eli Lilly and Company, Handok Pharmaceuticals, Merck, Novartis, and Servier; and has participated in advisory boards for AstraZeneca, Bayer, BeiGene, Genentech USA Inc., Halozyme Inc., Merck, and Taiho Oncology, Inc. AM has participated in an advisory board for AstraZeneca. MM has received consulting fees from Amgen, Astellas, BeiGene, Bristol Myers Squibb, Falk Foundation, Lilly Deutschland, Merck, Pierre Fabre, Servier, and Taiho Pharmaceutical Co.; and has received travel support from Bristol Myers Squibb and Merck. YK has received consulting fees from Asahi Kasei Pharma Corporation, Bayer, Bristol Myers Squibb, Chugai, Daiichi Sankyo Company, Eli Lilly Japan, Nippon Kayaku Co., Nipro Corporation, Ono Pharmaceutical Co., Pfizer Japan, Sanofi, Taiho Pharmaceutical Co., Takeda Pharmaceutical Company, and Yakult Honsha. DHA has received consulting fees from Advanced Accelerator Applications, Daiichi Sankyo Company, Eisai, Exelixis, Genentech USA Inc., Ipsen Biopharmaceuticals Inc., and Novartis. RSE has received consulting fees from G1 Therapeutics Inc., Merck, and Taiho Oncology, Inc. KAB holds stock options with Eli Lilly and Company and is named on the following patents: PCT/US2022/030500 (pending), PCT/US2022/014125 (pending), PCT/US2021/048206 (pending), PCT/US2017/048308 and WO2018044662A1 (issued), 63/255577 (filed), PCT/US2017/052864 and WO2018063927A1 (issued), PCT/US2016/026119 and WO2016168014A1 (issued), PCT/US2017/055650 and WO2018071307A1 (issued). JAB was funded, in part, by the National Institute for Health and Care Research (NIHR), University College London Hospitals NHS Foundation Trust (UCLH)–University College London (UCL) Biomedical Research Centre; has received consulting fees from Bayer, Bristol Myers Squibb, F. Hoffmann-La Roche, Incyte Corporation, National Institute for Health Research, Servier Affaires Medicales, and Taiho Pharmaceutical Co.; and is an employee of National Institute of Health Research. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.
Writing disclosure
Medical writing assistance was provided by Farhana Burnett, PhD, CMPP, of Envision Pharma Group, funded by Taiho Oncology, Inc.
Acknowledgments
In memoriam of Edith P Mitchell, who sadly passed away on 21 January 2024, and who was an author of the original article on which this summary is based. The authors thank the patients in this study and their families and the worldwide team of investigators and study site personnel for their contributions to this study. The authors also thank Jennifer L Silhavy, MSc, of Illumina, for her contribution to the analyses of circulating tumor DNA. The authors thank the Envision the Patient team, of Envision Pharma Group, for reviewing the use of patient-friendly language in this PLSP, and for organizing and providing feedback from a patient reviewer.
Competing interests disclosure
LG has participated in a data safety monitoring board for AstraZeneca. RKK has served on a clinical trial steering committee for AstraZeneca; and has participated in an investigator meeting and satellite symposium for Ipsen Biopharmaceuticals Inc. JT has provided educational collaboration for Imedex, Medscape Education, MJH Life Sciences, and PeerView Institute for Medical Education and Physicians Education Resource. RSE is a member of the board of directors for Fate Therapeutics, Illumina Inc., and Veracyte. A-BH, VW, YH, ML, and KAB are current or former employees of Taiho Oncology, Inc. The authors have no other competing interests or relevant affiliations with any organization or entity with the subject matter or materials discussed in the manuscript apart from those disclosed.