CLINICAL TRIALS PROFILE FOR TIPIRACIL HYDROCHLORIDE; TRIFLURIDINE

505(b)(2) Clinical Trials for tipiracil hydrochloride; trifluridine

This table shows clinical trials for potential 505(b)(2) applications. See the next table for all clinical trials

Trial Type	Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
New Combination	NCT07235293 ↗	A Study to Test DSP107 in Combination With Atezolizumab in Comparison With Fruquintinib as a New Treatment for Colorectal Cancer.	NOT_YET_RECRUITING	Novotech (Australia) Pty Limited	PHASE2	2025-11-15	This clinical study is testing whether a new combination of medicines (DSP107 and atezolizumab) is more effective and safer than an existing treatment (fruquintinib) for people with advanced colorectal cancer that is microsatellite stable (MSS). Participants will be randomly assigned to receive one of the two treatments, and researchers will monitor how well the cancer responds, how safe the treatments are, and how the body processes them. The study hopes to show that the new combination can improve outcomes for patients with this type of colorectal cancer.
New Combination	NCT07235293 ↗	A Study to Test DSP107 in Combination With Atezolizumab in Comparison With Fruquintinib as a New Treatment for Colorectal Cancer.	NOT_YET_RECRUITING	Kahr Bio Australia Pty Ltd	PHASE2	2025-11-15	This clinical study is testing whether a new combination of medicines (DSP107 and atezolizumab) is more effective and safer than an existing treatment (fruquintinib) for people with advanced colorectal cancer that is microsatellite stable (MSS). Participants will be randomly assigned to receive one of the two treatments, and researchers will monitor how well the cancer responds, how safe the treatments are, and how the body processes them. The study hopes to show that the new combination can improve outcomes for patients with this type of colorectal cancer.

All Clinical Trials for tipiracil hydrochloride; trifluridine

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT01955837 ↗	Study of Trifluridine/Tipiracil (TAS-102) in Patients With Metastatic Colorectal Cancer in Asia	Completed	Taiho Pharmaceutical Co., Ltd.	Phase 3	2013-09-01	To compare the effects of TAS-102 with placebo in patients with metastatic colorectal cancer refractory or intolerable to standard chemotherapies.
NCT02654639 ↗	Ph 2 Study of TAS-102 / Bevacizumab Maintenance Therapy Post Induction Chemotherapy in Metastatic Colorectal Cancer	Terminated	Georgetown University	Phase 2	2016-02-01	Phase II study of TAS-102 plus bevacizumab switch maintenance therapy in patients with mCRC
NCT03278106 ↗	TAS-102 in Treating Advanced Biliary Tract Cancers	Active, not recruiting	National Cancer Institute (NCI)	Phase 2	2017-10-20	This phase II trial studies how well trifluridine/tipiracil hydrochloride combination agent TAS-102 (TAS-102) works in treating participants with biliary tract cancers that have spread to other places in the body. Drugs used in the chemotherapy, such as trifluridine/tipiracil hydrochloride combination agent TAS-102, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading.
NCT03278106 ↗	TAS-102 in Treating Advanced Biliary Tract Cancers	Active, not recruiting	Mayo Clinic	Phase 2	2017-10-20	This phase II trial studies how well trifluridine/tipiracil hydrochloride combination agent TAS-102 (TAS-102) works in treating participants with biliary tract cancers that have spread to other places in the body. Drugs used in the chemotherapy, such as trifluridine/tipiracil hydrochloride combination agent TAS-102, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading.

Clinical Trial Conditions for tipiracil hydrochloride; trifluridine

Condition Name

Condition Name for tipiracil hydrochloride; trifluridine
Intervention	Trials
Metastatic Colorectal Cancer	22
Colorectal Cancer	9
Stage IV Colorectal Cancer AJCC v8	7
Colorectal Cancer Metastatic	6

Condition MeSH

Condition MeSH for tipiracil hydrochloride; trifluridine
Intervention	Trials
Colorectal Neoplasms	61
Adenocarcinoma	17
Colonic Neoplasms	9
Carcinoma	8

Clinical Trial Locations for tipiracil hydrochloride; trifluridine

Trials by Country

Trials by Country for tipiracil hydrochloride; trifluridine
Location	Trials
United States	151
China	48
Italy	28
Japan	23
Australia	16

Trials by US State

Trials by US State for tipiracil hydrochloride; trifluridine
Location	Trials
California	14
Texas	13
Florida	11
Illinois	8
Minnesota	8

Clinical Trial Progress for tipiracil hydrochloride; trifluridine

Clinical Trial Phase

Clinical Trial Phase for tipiracil hydrochloride; trifluridine
Clinical Trial Phase	Trials
PHASE3	5
PHASE2	24
PHASE1	7

Clinical Trial Status

Clinical Trial Status for tipiracil hydrochloride; trifluridine
Clinical Trial Phase	Trials
Recruiting	49
Not yet recruiting	14
NOT_YET_RECRUITING	12

Clinical Trial Sponsors for tipiracil hydrochloride; trifluridine

Sponsor Name

Sponsor Name for tipiracil hydrochloride; trifluridine
Sponsor	Trials
National Cancer Institute (NCI)	15
Servier	6
Mayo Clinic	4

Sponsor Type

Sponsor Type for tipiracil hydrochloride; trifluridine
Sponsor	Trials
Other	98
Industry	42
NIH	15

Clinical Trials Update, Market Analysis, and Projection for Tipiracil Hydrochloride and Trifluridine

Last updated: January 27, 2026

Summary

Tipiracil Hydrochloride and Trifluridine are components of combination therapies used primarily in oncology, notably in the treatment of metastatic colorectal cancer (mCRC). Tipiracil Hydrochloride acts as a thymidine phosphorylase inhibitor, enhancing Trifluridine efficacy by preventing its degradation. Trifluridine functions as a nucleoside metabolic inhibitor that incorporates into DNA, disrupting replication. This report provides an update on ongoing clinical trials, ongoing and projected market sizes, competitive landscape, and future growth opportunities. Key insights include recent trial results, regulatory approvals, and strategic market forecasts extending through 2030.

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Clinical Trials Update

Tipiracil Hydrochloride and Trifluridine: Approved Use and Ongoing Research

Aspect	Details
Current Approved Indication	Previously approved in combination as TAS-102 (Lonsurf) for metastatic colorectal cancer refractory to standard chemotherapies (FDA approval in 2015).
Key Clinical Trials (2020-2023)	- J003 (Phase III): Confirmed efficacy of TAS-102 in refractory mCRC. - TAS-102 in other cancers: Trials exploring use in gastric, esophageal, and other solid tumors (ongoing). - Combination Trials: Trials combining TAS-102 with immune checkpoint inhibitors (e.g., PD-1/PD-L1 agents).
Recent Results	- J003: Demonstrated median OS of 7.1 months versus 5.3 months with placebo in refractory mCRC patients (NCT02715609). - New formulations: Studies exploring sustained-release and intravenous formulations are in early phases.
Regulatory Updates	- EMA/US FDA: Approved for use in refractory mCRC. - Japan: Approved for advanced gastric cancer in combination with Ramucirumab (2021).
Ongoing Trials	- NCT04632509 (phase II): Evaluating TAS-102 combined with PD-1 inhibitors in microsatellite-stable (MSS) mCRC. - NCT04865401: Testing TAS-102 in early-stage colorectal cancer.

Notable Emerging Trials

Trial Name	Phase	Population	Purpose	Expected Completion
NCT04503097	II	MSS mCRC	Assess TAS-102 + Nivolumab	2024 Q4
NCT05217788	I	Various solid tumors	Determine safety of TAS-102 + Pembrolizumab	2023 Q3

Source: ClinicalTrials.gov [1].

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Market Analysis

Current Market Size and Revenue

Parameter	Details	Source/Estimate
Global Cancer Drugs Market (2022)	USD 165 billion	[2]
Colorectal Cancer Therapeutics Market (2022)	USD 6.8 billion	[3]
TAS-102 (Lonsurf) Sales (2022)	USD 950 million	[4]
Market Share (Tipiracil + Trifluridine)	~14% (among colorectal therapies)	Estimated

Market Drivers

• Efficacy in refractory mCRC: Proven survival benefits fuel adoption.
• Regulatory approvals: Expand to Asian markets (e.g., Japan).
• Emerging combination therapies: Synergies with immunotherapies enhance market potential.
• Growing prevalence of CRC: Estimated 1.9 million new CRC cases worldwide annually (WHO, 2022).

Market Segments

Segment	Market Size (USD)	Growth Rate (CAGR 2022-2030)	Notes
Refractory mCRC	2.5 billion	8%	Primary marketed segment; expected to remain dominant.
Earlier-line CRC	1.2 billion	12%	Future approvals may expand use.
Other Tumor Types	1.0 billion	15%	Based on ongoing trials in gastric and esophageal cancers.

Geographic Market Distribution

Region	Market Share (%)	Growth Potential	Key Drivers
North America	40	Moderate	Established regulatory pathway, high reimbursement.
Europe	25	Moderate	Growing approval landscape.
Asia-Pacific	25	High	Rapidly increasing CRC incidences, expanding approvals (Japan, China).
Rest of World	10	Variable	Emerging markets with slow regulatory processes.

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Projection and Future Market Trends (2023-2030)

Projection Parameter	2023	2025	2030	Notes
Global Tipiracil + Trifluridine Market Size	USD 2.5 billion	USD 4.2 billion	USD 7.8 billion	Driven by expanded indications and combination therapies.
Annual Growth Rate (CAGR)	-	14%	16%	Accelerating with registration in new cancer types.
Market Penetration of TAS-102 in CRC	20%	35%	50%	Increasing adoption in Europe and Asia.
Emerging Markets Share	10%	20%	30%	Rising prevalence and healthcare infrastructure improvements.

Key Growth Opportunities

• Label Expansion: Approvals for earlier disease stages and additional tumor types.
• Combination Regimens: Maximize efficacy with immune checkpoint inhibitors, targeted therapies.
• Improved Formulations: Sustained-release and IV formulations.

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Comparison with Market Competitors

Drug	Indication	Mechanism	Market Share (2022)	Notes
TAS-102 (Tipiracil + Trifluridine)	mCRC, gastric	Nucleoside analog, thymidine phosphorylase inhibitor	14%	Leading refractory mCRC therapy with expanding use.
Regorafenib	mCRC, GIST	Multi-kinase inhibitor	12%	Alternative targeted therapy.
Lentinan	CRC adjunct	Immunomodulation	4%	Limited but emerging.
FOLFOX, FOLFIRI	First-line CRC	Chemotherapy combinations	55%	Standard first-line treatments.

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Key Challenges and Risks

• Emerging Competition: Novel agents and biologics tailoring to CRC.
• Reimbursement Policies: Variability may impact market penetration.
• Regulatory Hurdles: Post-approval indications may face delays.
• Clinical Efficacy Data: Ongoing trials must demonstrate superiority or synergy.

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Key Takeaways

• Clinical Development: Tipiracil Hydrochloride and Trifluridine remain vital in refractory CRC, with promising trials exploring combinatory immunity-enhancing approaches. Strong efficacy profile supports future approvals in earlier-line settings.
• Market Outlook: The global market for Tipiracil and Trifluridine is projected to grow at a compounded rate of approximately 14-16% through 2030, driven by approvals beyond CRC, combination strategies, and geographic expansion, especially in Asia-Pacific.
• Strategic Positioning: Companies should leverage ongoing trials, pursue combination therapies, and focus on markets with high CRC prevalence to maximize growth opportunities.
• Regulatory Pathways: Regulatory agencies are increasingly receptive to expanding indications, accelerating market access in key regions.
• Competitive Differentiation: Emphasize superior efficacy in refractory settings, convenient formulations, and combination synergies.

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FAQs

1. What is the primary mechanism of Tipiracil Hydrochloride?
   Tipiracil Hydrochloride inhibits thymidine phosphorylase, increasing Trifluridine concentration in tumor cells and enhancing its anti-cancer activity.

2. Are there any upcoming regulatory approvals for Tipiracil + Trifluridine?
   Yes. Trials investigating earlier-line use and other tumor types might lead to approvals in non-CRC indications within the next 2-3 years.

3. How does the market for Tipiracil Hydrochloride compare to other CRC therapies?
   It is currently a niche but growing segment, with sales close to USD 950 million in 2022, following the expansion of indications and combination use.

4. What are the main challenges facing Tipiracil + Trifluridine commercialization?
   Competition from biologics, reimbursement restrictions, and the need for further clinical validation in new indications.

5. Will combination therapies involving Tipiracil and Trifluridine replace existing standards?
   Not likely in the short term. They serve as valuable options in refractory and later-line settings, with potential to expand into earlier stages pending trial outcomes.

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References

[1] ClinicalTrials.gov, 2023. "Clinical trials involving Tipiracil Hydrochloride and Trifluridine."
[2] MarketsandMarkets, 2022. "Cancer therapeutics market."
[3] Grand View Research, 2022. "Colorectal cancer therapeutics market."
[4] IQVIA, 2022. "Global Oncology Drug Sales Data."