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
Circulating Tumor Cell	1
Gastric Cancer	1
Advanced or Metastatic Solid Tumor	1

Condition MeSH

Condition MeSH for FUTIBATINIB
Intervention	Trials
Cholangiocarcinoma	4
Neoplasms	2
Carcinoma, Non-Small-Cell Lung	2
Carcinoma	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
Texas	6
California	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
UNICANCER	1
M.D. Anderson Cancer Center	1

Sponsor Type

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

Futibatinib: Clinical Trials Update, Market Analysis, and Future Projections

Last updated: January 25, 2026

Executive Summary

Futibatinib, an investigational tyrosine kinase inhibitor targeting FGFR (Fibroblast Growth Factor Receptor) alterations, has gained significant attention in oncology therapeutics. Currently progressing through critical clinical development phases, it demonstrates promising efficacy in FGFR-driven cancers, notably cholangiocarcinoma. This report synthesizes the latest clinical data, analyzes current market dynamics, and offers future market projections integral for stakeholders and investors.

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Clinical Trials Update: What Is the Status of Futibatinib?

Overview of Clinical Development Phases

Phase	Trials Active/Completed	Key Objectives	Sample Size	Status	Date of Last Data Update
Phase 1	Ongoing	Safety, Dose-escalation, Pharmacokinetics	50+	Preliminary	December 2022
Phase 2	Ongoing	Efficacy in FGFR-altered biliary tract cancers	100+	Recruitment	June 2023
Phase 3	Planned	Confirm efficacy and safety	N/A	Pending	N/A

Regulatory Submissions and Approvals

• FDA: Futibatinib has obtained Breakthrough Therapy Designation for intrahepatic cholangiocarcinoma associated with FGFR2 fusions or rearrangements (October 2021). A New Drug Application (NDA) submission is anticipated pending Phase 2/3 results.
• EMA: Not yet filed; regulatory review awaited.

Recent Published Data Highlights

• Efficacy:

Study	Population	ORR (Objective Response Rate)	PFS (Progression-Free Survival)	Status	Reference
FOENIX-CCA2 (Phase 2)	FGFR2-rearranged intrahepatic cholangiocarcinoma	25% (Complete + Partial responses)	Median 7.2 months	Published (May 2023)	[1]

• Safety Profile:

Common Adverse Events	Incidence	Severity	Comments
Hyperphosphatemia	50%	Grade 1-2	Manageable with phosphate binders
Diarrhea	30%	Grade 1-2	Often reversible
Stomatitis	20%	Mild	No dose adjustments needed in most cases

Latest News & Clinical Trial Registrations

• Completion of recruiting in Phase 2 (NCT04603423) for biliary tract cancers.
• Initiation of Phase 3 (NCT05171377): Comparing futibatinib versus standard of care in advanced cholangiocarcinoma (expected in Q3 2023).

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Market Analysis: The Current Landscape for FGFR Inhibitors

Market Drivers

Driver	Description	Implication
Unmet Need	Limited options for FGFR-altered cholangiocarcinoma	High potential for targeted therapies
Biomarker-Driven Therapies	Increasing adoption of genomic testing	Facilitates precision medicine
Regulatory Incentives	Breakthrough designations	Accelerates market entry
Competitive Landscape	Few FGFR inhibitors approved	Less market saturation

Competitive Landscape

Drug	Company	Indications	Approvals	Key Data	Market Position
Pemigatinib	Incyte	Cholangiocarcinoma	FDA Approved (April 2020)	ORR 36% (Phase 2)	Market leader among FGFR inhibitors
Infigratinib	QED	Cholangiocarcinoma	Limited approvals	ORR 23%	Competitor
Debio 1347	Debiopharm	Multiple FGFR cancers	Under clinical evaluation	Ongoing trials	Emerging contender
Futibatinib	Taiho, Janssen	Cholangiocarcinoma	Pending approval	ORR 25% in Phase 2	Near-term potential

Market Size & Forecast

Market Segment	2022 Revenue	Projected 2027 Revenue	CAGR	Sources
FGFR Inhibitors	$350 million	$1.2 billion	28%	[2]
Cholangiocarcinoma (total)	$600 million	$1.8 billion	23%	[3]
Futibatinib-specific	Part of above	Dominant in FGFR2 patients	—	—

Note: The global oncology targeted therapy market is expected to reach $216 billion by 2027 (CAGR 8%) [4].

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Future Market Projection for Futibatinib

Key Factors Influencing Growth

• Regulatory Milestones: Approval could catalyze market penetration.
• Biomarker Testing Expansion: Increased genomic profiling will identify suitable patient subsets.
• Clinical Efficacy and Safety Profile: Strong data will underpin label expansion across additional FGFR-driven cancers, including bladder and lung cancers.

Forecasted Revenue & Market Share (2023-2027)

Year	Estimated Revenue	Market Share in FGFR Inhibitors	Drivers	Risks
2023	$50–$100 million	10–15%	Pending approvals	Denied or delayed FDA approval
2024	$150–$250 million	20–30%	Data readouts, approvals	Competition expansion
2025	$300–$500 million	25–35%	Label expansion, robust clinical data	Safety concerns or pricing pressures
2026	$800 million	35–40%	Market penetration, new indications	Regulatory hurdles
2027	$1.2 billion	40–45%	Established presence, expanded indications	Market saturation

Regional Market Opportunities

Region	Market Size (2022)	Growth Drivers	Challenges
North America	$200 million	High genomic testing rates	Reimbursement policies
Europe	$100 million	Approvals & clinical trials	Regulatory harmonization
Asia-Pacific	$50 million	Growing oncology market	Limited local approvals

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Comparative Analysis: Futibatinib vs Competitors

Parameter	Futibatinib	Pemigatinib	Infigratinib	Debio 1347
Approval Status	Pending / Breakthrough	Approved	Limited	Preclinical/Clinical
Target Points	FGFR1-4	FGFR1-3	FGFR1-3	FGFR1-3
Dosing	Once daily	Once daily	Once daily	Oral, experimental
Toxicity	Manageable	Manageable	Manageable	Under investigation
Clinical Response	25–30% (depending on trial)	36% (FDA approved)	23%	Early-stage

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Key Takeaways

• Futibatinib remains in late-stage clinical evaluation, with promising signs of efficacy in FGFR2-rearranged cholangiocarcinoma.
• Regulatory milestones, notably NDA submissions, are anticipated within 2023, contingent on ongoing trial outcomes.
• Market potential for Futibatinib is robust, driven by the rising prevalence of FGFR alterations and limited current approvals.
• Competitive landscape favors early approval and label expansion to sustain market share.
• Strategic focus on biomarker testing, regional regulatory pathways, and clinical trial outcomes will influence future market success.

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FAQs

Q1: When is Futibatinib expected to receive FDA approval?
A1: Likely in late 2023 or early 2024, contingent on positive Phase 3 trial data and NDA review outcomes.

Q2: What indications are most promising for Futibatinib's market application?
A2: Intrahepatic cholangiocarcinoma with FGFR2 fusions or rearrangements, with potential expansion into urothelial carcinomas and other FGFR-driven cancers.

Q3: How does Futibatinib compare to approved FGFR inhibitors?
A3: It offers once-daily dosing, a favorable safety profile, and the potential for broader FGFR targeting, which could translate into efficacy advantages once approved.

Q4: What are the main risks affecting Futibatinib’s market success?
A4: Delays or failure in clinical trial outcomes, regulatory rejection, strong competition, and adverse safety signals.

Q5: How will biomarker testing impact Futibatinib’s adoption?
A5: Widespread genomic profiling enhances patient selection, increasing treatment efficacy and market penetration.

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References

[1] FOENIX-CCA2 Trial Results, Journal of Clinical Oncology, May 2023.

[2] Market Research Future, “Global FGFR Inhibitors Market Analysis,” 2022.

[3] GlobalData, “Oncology Market Forecast,” 2023.

[4] Grand View Research, “Oncology Targeted Therapy Market Size & Trends,” 2022.

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Disclaimer: This analysis is for informational purposes only and does not constitute investment advice. Stakeholders should consult relevant sources and regulatory updates for decision-making.