CLINICAL TRIALS PROFILE FOR FUTIBATINIB

All Clinical Trials for FUTIBATINIB

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT02052778 ↗	A Study of TAS-120 in Patients With Advanced Solid Tumors	Completed	Taiho Oncology, Inc.	Phase 1/Phase 2	2014-07-01	This is an open-label, nonrandomized, Phase 1/2 study for the fibroblast growth factor receptor (FGFR) inhibitor futibatinib (TAS-120). The purpose of the study is to evaluate the safety, tolerability, PK, pharmacodynamic, and anti-tumor activity of futibatinib in patients with advanced solid tumors with and without genomic FGF/FGFR abnormalities. The study will be conducted in 3 parts: 1. Dose escalation portion to determine the MTD and/ or RP2D of futibatinib. 2. Phase 1 expansion portion to further evaluate the safety and efficacy of futibatinib in patients with tumors harboring FGF/FGFR aberrations, including patients with cholangiocarcinoma (CCA), primary CNS tumors, urothelial carcinoma, breast cancer, gastric cancer and 3. Phase 2 study portion to confirm ORR of futibatinib in intrahepatic CCA patients with tumors harboring FGFR2 gene rearrangements (incl fusions).
NCT04024436 ↗	A Study of TAS-120 in Patients With Metastatic Breast Cancer	Recruiting	Taiho Oncology, Inc.	Phase 2	2019-08-30	The purpose of the trial is to evaluate a patient's response to a Fibroblast Growth Factor Receptor (FGFR) inhibitor, futibatinib (TAS-120), used either alone or in combination with the hormonal therapy, fulvestrant. This study will be conducted in patients with metastatic breast cancer who have specific Fibroblast Growth Factor Receptor gene abnormalities and who have previously received conventional therapies to treat their breast cancer, or who are not able to tolerate certain cancer therapies. This study will also evaluate the safety of taking futibatinib, or futibatinib and fulvestrant, by learning about the potential side effects.
NCT04093362 ↗	Futibatinib Versus Gemcitabine-Cisplatin Chemotherapy as First-Line Treatment of Patients With Advanced Cholangiocarcinoma Harboring FGFR2 Gene Rearrangements	Recruiting	Taiho Oncology, Inc.	Phase 3	2020-03-01	This is an open-label, multinational, parallel 2-arm, randomized Phase 3 study evaluating the efficacy and safety of futibatinib versus gemcitabine-cisplatin chemotherapy as first-line treatment of patients with advanced, metastatic, or recurrent unresectable iCCA harboring FGFR2 gene rearrangements
NCT04189445 ↗	Futibatinib in Patients With Specific FGFR Aberrations	Recruiting	Taiho Oncology, Inc.	Phase 2	2020-08-31	The purpose of this study is to evaluate the efficacy and safety of futibatinib in patients with FGFR aberrations in 3 distinct cohorts. Patients will be enrolled into one of 3 cohorts: patients with advanced, metastatic or locally-advanced solid tumors harboring FGFR1-4 rearrangements (excluding primary brain tumors and intrahepatic cholangiocarcinoma [iCCA]); patients with gastric or gastro-esophageal junction (GEJ) cancer harboring FGFR2 amplification; and patients with myeloid or lymphoid neoplasms with FGFR1 rearrangements.
NCT04601857 ↗	Futibatinib and Pembrolizumab Combination in the Treatment of Advanced or Metastatic Urothelial Carcinoma	Recruiting	Merck Sharp & Dohme Corp.	Phase 2	2021-01-21	The purpose of the trial is to evaluate the antitumor activity and confirm the safety for the combination of Fibroblast Growth Factor Receptor (FGFR) inhibitor futibatinib and anti-programmed cell death-1 (PD-1) antibody pembrolizumab in patients with advanced or metastatic urothelial cancer who are not candidates to receive a platinum-based treatment regimens.
NCT04601857 ↗	Futibatinib and Pembrolizumab Combination in the Treatment of Advanced or Metastatic Urothelial Carcinoma	Recruiting	Taiho Oncology, Inc.	Phase 2	2021-01-21	The purpose of the trial is to evaluate the antitumor activity and confirm the safety for the combination of Fibroblast Growth Factor Receptor (FGFR) inhibitor futibatinib and anti-programmed cell death-1 (PD-1) antibody pembrolizumab in patients with advanced or metastatic urothelial cancer who are not candidates to receive a platinum-based treatment regimens.
NCT04828486 ↗	Futibatinib and Pembrolizumab for the Treatment of Advanced or Metastatic FGF19 Positive BCLC Stage A, B, or C Liver Cancer	Recruiting	National Cancer Institute (NCI)	Phase 2	2021-05-07	This phase II trial studies the effect of futibatinib and pembrolizumab in treating patients with FGF19 positive BCLC stage A, B, or C liver cancer that has spread to other parts of the body (advanced or metastatic). Futibatinib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Immunotherapy with monoclonal antibodies, such as pembrolizumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Giving futibatinib and pembrolizumab may help treat patients with FGF19 positive liver cancer.

Clinical Trial Conditions for FUTIBATINIB

Condition Name

Condition Name for FUTIBATINIB
Intervention	Trials
Advanced Cholangiocarcinoma	2
Endometrium Cancer	1
Metastatic Breast Cancer	1
BCLC Stage B Hepatocellular Carcinoma	1

Condition MeSH

Condition MeSH for FUTIBATINIB
Intervention	Trials
Cholangiocarcinoma	4
Carcinoma	2
Neoplasms	2
Carcinoma, Non-Small-Cell Lung	2

Clinical Trial Locations for FUTIBATINIB

Trials by Country

Trials by Country for FUTIBATINIB
Location	Trials
United States	63
France	14
Spain	7
Japan	7
Germany	6

Trials by US State

Trials by US State for FUTIBATINIB
Location	Trials
California	6
Texas	6
Michigan	5
Massachusetts	4
Arizona	4

Clinical Trial Progress for FUTIBATINIB

Clinical Trial Phase

Clinical Trial Phase for FUTIBATINIB
Clinical Trial Phase	Trials
PHASE2	4
Phase 3	2
Phase 2	6

Clinical Trial Status

Clinical Trial Status for FUTIBATINIB
Clinical Trial Phase	Trials
RECRUITING	7
Not yet recruiting	3
NOT_YET_RECRUITING	2

Clinical Trial Sponsors for FUTIBATINIB

Sponsor Name

Sponsor Name for FUTIBATINIB
Sponsor	Trials
Taiho Oncology, Inc.	10
Universittsmedizin Mainz, I. Medizinische Klinik, Forschungslabor Prof. Mhler	1
Cancer Research UK & UCL Cancer Trials Centre	1

Sponsor Type

Sponsor Type for FUTIBATINIB
Sponsor	Trials
Industry	15
Other	13
NIH	1

Clinical Trials Update, Market Analysis, and Projection for Futibatinib

Last updated: October 26, 2025

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Introduction

Futibatinib, a potent and selective irreversible inhibitor of fibroblast growth factor receptor (FGFR) kinases, continues to garner attention within oncology therapeutics. As an emerging treatment modality targeting FGFR-altered cancers, its developmental trajectory, clinical progress, and market potential warrant comprehensive analysis. This report provides an up-to-date review of futibatinib’s ongoing clinical trials, evaluates its current market positioning, and projects future growth prospects based on recent data.

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Clinical Trials Update

Current Clinical Development Stage

Futibatinib is predominantly investigated in Phase II and Phase III clinical trials, primarily targeting FGFR-driven malignancies such as cholangiocarcinoma, bladder cancer, and other solid tumors. The drug's pivotal studies focus on efficacy, safety, and combinatorial regimens, with several trials sponsored by Taiho Pharmaceutical and other collaborators.

Key Trials and Outcomes

• FOENIX-CCA2 (NCT03773302): A Phase II trial assessing futibatinib as a monotherapy in patients with intrahepatic cholangiocarcinoma (ICC) harboring FGFR2 gene fusions or rearrangements. Preliminary data indicated a promising objective response rate (ORR) of approximately 50%, with manageable adverse events (AEs) such as hyperphosphatemia and fatigue, aligning with FGFR inhibitor profiles [1].

• Futibatinib Plus Chemotherapy in Advanced Solid Tumors (NCT04660092): A Phase Ib/II study exploring the combination of futibatinib with standard chemotherapeutic agents to evaluate synergistic effects across various tumor types. Early results suggest manageable tolerability and signs of enhanced antitumor activity, meriting further investigation.

• Futibatinib in Bladder Cancer (NCT04684050): Recently initiated Phase II trial targeting FGFR alterations in metastatic urothelial carcinoma. Its progress indicates active exploration into expanding therapeutic indications.

Regulatory Progress

In 2022, Taiho Pharmaceutical submitted an application for futibatinib’s approval in intrahepatic cholangiocarcinoma in Japan, based on data from FOENIX-CCA2. The drug received regulatory priority review status, reflecting its potential as a targeted therapy for FGFR-altered cancers. However, regulatory approval in other jurisdictions remains pending, contingent upon ongoing trial outcomes.

Pipeline Outlook

The clinical pipeline underscores a strategic focus on FGFR-driven malignancies, with multiple ongoing trials designed to validate efficacy, optimize dosing, and evaluate combination strategies. While toxicity management — particularly of hyperphosphatemia — remains a challenge, the overall therapeutic profile is competitive.

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Market Analysis

Current Market Landscape

The global FGFR inhibitor market is expanding rapidly, driven by advances in precision oncology and increasing identification of FGFR genetic alterations across multiple tumor types. Leading competitors include Erdafitinib (JNJ), Pemigatinib (Incyte), and Soledentinib, with each boasting FDA approvals for specific indications [2].

Futibatinib’s market entry aims to capitalize on its unique irreversible binding mechanism, which may offer advantages in potency and overcoming resistance seen with reversible inhibitors. The drug's potential approval for intrahepatic cholangiocarcinoma positions it in a lucrative niche, as this disease has limited targeted options to date.

Market Drivers

• Rising Prevalence of FGFR Alterations: FGFR gene fusions, mutations, and amplifications are implicated in approximately 15-20% of intrahepatic cholangiocarcinoma cases and other cancers, underpinning a substantial patient base.

• Unmet Medical Need: Despite existing therapies, FGFR-targeted treatments lack broad approval outside specific indications. Futibatinib's promise as an irreversible inhibitor offers a compelling alternative.

• Regulatory Approvals: Early regulatory signals in Japan set a precedent; breakthrough status can accelerate market penetration internationally.

Market Challenges

• Safety and Tolerability: Side effects such as hyperphosphatemia necessitate monitoring, potentially complicating the dosing regimen and affecting patient adherence.

• Competitive Landscape: Erdafitinib, Pemigatinib, and other FGFR inhibitors already possess commercial presence, with established data and market share, creating a high entry barrier.

• Biomarker Testing: Widespread adoption hinges on robust genomic screening infrastructure, which varies across regions.

Market Projections

Based on current trends, the FGFR inhibitor market is projected to surpass USD 2.3 billion by 2025, expanding at a CAGR of approximately 12% [3]. Futibatinib’s targeted segmentation, focusing initially on cholangiocarcinoma, could contribute a significant share within this niche.

Assuming successful clinical and regulatory milestones, futibatinib is poised to capture 15-20% of the FGFR inhibitor market segment for cholangiocarcinoma and potentially expand into other FGFR-driven cancers, achieving peak sales of USD 500-700 million globally by 2030.

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Market Opportunities & Strategic Outlook

Advantages

• Irreversible Binding: Differentiates futibatinib from competitors, potentially offering sustained inhibition and overcoming resistance pathways.

• Indication Expansion: Potential to treat FGFR-altered additional cancers, such as breast, lung, and urothelial carcinomas.

• Regulatory Probability: The priority review status in Japan and ongoing clinical success could expedite approvals elsewhere.

Strategic Recommendations

• Biomarker Development: Invest in genomic diagnostics to identify suitable patient populations efficiently.

• Combination Regimens: Explore synergistic combinations with immunotherapies or chemotherapy to expand indications and improve efficacy.

• Global Regulatory Strategy: Engage early with agencies to streamline approval processes, considering regional differences in molecular testing infrastructure.

• Post-Market Surveillance: Prioritize safety monitorization to manage side effects and optimize dosing strategies.

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Future Projections & Industry Outlook

Futibatinib’s trajectory appears optimistic, predicated on ongoing positive clinical outcomes and regulatory approval pathways. The drug's successful commercialization could reshape FGFR-targeted therapy, offering advantages such as durable responses and overcoming resistance associated with reversible inhibitors. The broader market is expected to grow as more FGFR-altered cancers are diagnosed early and targeted therapies become standard care.

Advancements in companion diagnostics and genomic testing will be pivotal for futibatinib’s optimal deployment, enabling precision medicine approaches. Competitive differentiation through safety profile management and combination strategies will determine its market penetration.

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Key Takeaways

• Clinical Progress: Futibatinib is in advanced trial phases, with promising efficacy signals in FGFR fusion-positive cholangiocarcinoma; regulatory applications are underway, especially in Japan.

• Market Positioning: The drug is positioned to leverage its irreversible mechanism to address unmet needs in FGFR-driven cancers, with significant potential in intrahepatic cholangiocarcinoma.

• Competitive Landscape: Despite competition from established FGFR inhibitors, futibatinib's unique features and evolving clinical data may confer a competitive edge, especially if safety and efficacy are validated.

• Market Potential: Projected to reach peak sales of USD 500-700 million by 2030, contingent on successful approvals and indications expansion.

• Strategic Priorities: Emphasize biomarker-driven patient selection, combination therapies, and global regulatory engagement to maximize revenue opportunities.

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FAQs

1. What cancer types is futibatinib primarily targeting?
Futibatinib is primarily aimed at FGFR-altered malignancies, with current focus on intrahepatic cholangiocarcinoma, bladder cancer, and ongoing exploration in other solid tumors.

2. How does futibatinib differ from existing FGFR inhibitors?
Futibatinib irreversibly binds to FGFR kinases, potentially offering sustained inhibition and overcoming resistance mechanisms associated with reversible inhibitors.

3. What are the main safety concerns associated with futibatinib?
Hyperphosphatemia is a common adverse event due to FGFR pathway modulation. Other side effects include fatigue, diarrhea, and dry mouth. Monitoring and management strategies are integral to therapy.

4. When is futibatinib expected to receive regulatory approval outside Japan?
While Japanese approval is imminent, regulatory decisions in the U.S. and Europe depend on comprehensive clinical data, expected to be available in the next 1-2 years.

5. What is the outlook for futibatinib’s market growth?
With positive trial results and regulatory progress, futibatinib's market potential is significant, likely reaching peak global sales of USD 500-700 million by 2030, contingent on approval and indication expansion.

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References

1. Clinical Data from FOENIX-CCA2 Study: Preliminary results indicating favorable efficacy in cholangiocarcinoma.
2. Incyte and Pfizer Market Reports, 2022.
3. Global Oncology Therapeutics Market Analysis, MarketsandMarkets, 2022.

This document provides a comprehensive perspective on futibatinib, equipping healthcare stakeholders and industry professionals to make informed strategic decisions.