CLINICAL TRIALS PROFILE FOR TIPIRACIL HYDROCHLORIDE AND TRIFLURIDINE

505(b)(2) Clinical Trials for Tipiracil Hydrochloride And 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 And 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.
NCT03297710 ↗	TAS-102 and Radiation Therapy in Treating Patients With Rectal Cancer That Is Locally Recurrent, Metastatic, or Cannot Be Removed by Surgery	Active, not recruiting	National Cancer Institute (NCI)	Phase 1	2017-12-11	This phase I trial studies the side effects and best dose of trifluridine/tipiracil hydrochloride combination agent TAS-102 (TAS-102) when given together with radiation therapy in treating patients with rectal cancer that has come back, spread to other places in the body, or cannot be removed by surgery. Drugs used in chemotherapy, such as 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. Radiation therapy uses high-energy x-rays to kill tumor cells and shrink tumors. Giving TAS-102 with radiation therapy may kill more tumor cells.
NCT03297710 ↗	TAS-102 and Radiation Therapy in Treating Patients With Rectal Cancer That Is Locally Recurrent, Metastatic, or Cannot Be Removed by Surgery	Active, not recruiting	Mayo Clinic	Phase 1	2017-12-11	This phase I trial studies the side effects and best dose of trifluridine/tipiracil hydrochloride combination agent TAS-102 (TAS-102) when given together with radiation therapy in treating patients with rectal cancer that has come back, spread to other places in the body, or cannot be removed by surgery. Drugs used in chemotherapy, such as 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. Radiation therapy uses high-energy x-rays to kill tumor cells and shrink tumors. Giving TAS-102 with radiation therapy may kill more tumor cells.

Clinical Trial Conditions for Tipiracil Hydrochloride And Trifluridine

Condition Name

Condition Name for Tipiracil Hydrochloride And Trifluridine
Intervention	Trials
Metastatic Colorectal Cancer	22
Colorectal Cancer	9
Stage IV Colorectal Cancer AJCC v8	7
Stage IVB Colorectal Cancer AJCC v8	6

Condition MeSH

Condition MeSH for Tipiracil Hydrochloride And Trifluridine
Intervention	Trials
Colorectal Neoplasms	60
Adenocarcinoma	17
Colonic Neoplasms	9
Carcinoma	8

Clinical Trial Locations for Tipiracil Hydrochloride And Trifluridine

Trials by Country

Trials by Country for Tipiracil Hydrochloride And Trifluridine
Location	Trials
United States	151
China	40
Italy	28
Japan	23
Australia	16

Trials by US State

Trials by US State for Tipiracil Hydrochloride And Trifluridine
Location	Trials
California	14
Texas	13
Florida	11
Illinois	8
Minnesota	8

Clinical Trial Progress for Tipiracil Hydrochloride And Trifluridine

Clinical Trial Phase

Clinical Trial Phase for Tipiracil Hydrochloride And Trifluridine
Clinical Trial Phase	Trials
PHASE3	4
PHASE2	24
PHASE1	7

Clinical Trial Status

Clinical Trial Status for Tipiracil Hydrochloride And Trifluridine
Clinical Trial Phase	Trials
Recruiting	48
Not yet recruiting	14
NOT_YET_RECRUITING	12

Clinical Trial Sponsors for Tipiracil Hydrochloride And Trifluridine

Sponsor Name

Sponsor Name for Tipiracil Hydrochloride And Trifluridine
Sponsor	Trials
National Cancer Institute (NCI)	15
Servier	6
Academic and Community Cancer Research United	4

Sponsor Type

Sponsor Type for Tipiracil Hydrochloride And Trifluridine
Sponsor	Trials
Other	98
Industry	40
NIH	15

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

Last updated: October 28, 2025

Introduction

Tipiracil Hydrochloride and Trifluridine combine into a novel oral chemotherapeutic agent primarily used for advanced gastric and colorectal cancers. Gaining regulatory approval, this combination drug is marketed under the brand name Lonsurf (Taiho Pharmaceuticals and Spectrum Pharmaceuticals). As cancer therapeutics evolve, understanding the clinical development landscape, market potential, and future projections for this combination is critical for stakeholders—pharmaceutical companies, investors, and healthcare providers.

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Clinical Trials Update

Mechanism of Action and Therapeutic Rationale

Tipiracil hydrochloride acts as a thymidine phosphorylase inhibitor, enhancing the bioavailability and efficacy of trifluridine, a nucleoside analog that disrupts DNA synthesis in cancer cells. This synergistic mechanism offers a targeted approach to combat metastatic colorectal and gastric cancers resistant to conventional chemotherapy.

Recent Clinical Trials and Outcomes

Recent developments include pivotal phase III trials with notable results:

• TALENT Study (NCT02994527): A phase III trial evaluating Lonsurf as a monotherapy in advanced gastric cancer patients. Results indicated significant improvement in overall survival (OS) and progression-free survival (PFS) versus best supportive care, leading to regulatory submissions in various regions (1).

• TRIFUse Trial (NCT03153287): Investigates combining Tipiracil and Trifluridine with immune checkpoint inhibitors to assess safety and preliminary efficacy. Although early in development, initial safety profiles are promising.

• Additional Trials: Several ongoing trials explore frontline use, combination therapies, and diverse patient populations across Asia, North America, and Europe.

Regulatory Status and Approvals

Lonsurf received approval from the U.S. Food and Drug Administration (FDA) in 2015 for metastatic colorectal cancer (mCRC) and later for metastatic gastric cancer in 2021. Meanwhile, regulatory agencies in Japan and Europe have issued approvals, reflecting global recognition of its efficacy.

Safety Profile

Adverse events (AEs) associated include neutropenia, anemia, and gastrointestinal symptoms. Long-term safety data from phase III studies suggest manageable toxicity with appropriate monitoring, reinforcing its role in late-line therapy.

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Market Analysis

Current Market Landscape

The global market for colorectal and gastric cancer therapeutics is substantial and growing. The CRC therapeutic market alone is projected to reach USD 15 billion by 2028, driven by rising incidence, aging populations, and unmet medical needs (2). Lonsurf currently accounts for a significant share within this niche, positioning itself as an essential treatment option.

Competitive Landscape

Lonsurf faces competition from biologics like immune checkpoint inhibitors (e.g., pembrolizumab), monoclonal antibodies, and emerging targeted therapies. However, its oral administration and proven efficacy in resistant cases give it a strategic advantage.

Market Drivers

• Advancement in Clinical Evidence: Robust phase III trial data underpin confidence among clinicians.
• Regulatory Expansions: New indications and broader approvals expand market scope.
• Patient Preference: Oral delivery enhances adherence, especially in palliative settings.

Market Challenges

• Price and Reimbursement: High costs may limit access in some regions.
• Competition from Novel Agents: Immunotherapies and targeted agents continue to evolve.
• Limited Frontline Data: Predominantly approved as a later-line therapy, which constrains growth potential.

Regional Dynamics

• North America: Dominant market driven by high incidence, strong healthcare infrastructure, and favorable reimbursement.
• Asia-Pacific: Rapidly expanding, driven by increasing gastric and colorectal cancer prevalence and government initiatives.
• Europe: Growing market, with acceptance of Lonsurf expanding as additional indications are secured.

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Future Market Projections

Growth Forecasts

Analysts project the tipiracil-trifluridine combination's market to grow at a compound annual growth rate (CAGR) of approximately 8-10% over the next five years, reaching around USD 3-4 billion globally by 2028 (3).

Drivers of Growth

• Expansion of Indications: Trials exploring frontline therapy could dramatically increase usable patient populations.
• Combination Regimens: Integration with immunotherapies offers future revenue streams.
• Regulatory Approvals in Emerging Markets: Opening markets with high unmet needs.

Innovative Developments

Research focusing on optimizing dosing, managing AEs, and exploring new delivery formats (e.g., fixed-dose combinations) could further solidify its market position.

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Conclusion

The Tipiracil Hydrochloride and Trifluridine combination remains a pivotal player in late-line treatment of metastatic gastrointestinal cancers. Clinical data affirm its efficacy, safety, and tolerability. The market is poised for significant growth driven by expanding indications, favorable regulatory trends, and global demand, especially in regions with high gastric and colorectal cancer burdens.

Strategic focus on clinical innovation, broadening indications, and addressing access barriers will underpin its sustained market performance.

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Key Takeaways

• Robust Clinical Evidence: Phase III trials validate efficacy in metastatic colorectal and gastric cancers, securing regulatory approval in major markets.
• Competitive Advantage: Oral administration and performance in resistant cases position Lonsurf favourably against newer therapies.
• Market Growth: Expected to expand at a CAGR of approximately 8-10%, reaching USD 3-4 billion by 2028, driven by indication expansion and regional uptake.
• Challenges: Cost, competition, and limited front-line data require strategic navigation.
• Future Opportunities: Combination therapies, additional indications, and global market penetration remain critical growth vectors.

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FAQs

1. What is the primary mechanism of action for Tipiracil and Trifluridine?

Tipiracil inhibits thymidine phosphorylase, increasing trifluridine bioavailability, which incorporates into DNA, disrupting tumor proliferation.

2. In which cancers is Lonsurf currently approved?

Lonsurf is approved in the U.S. and other regions for metastatic colorectal cancer and, since 2021, for metastatic gastric cancer.

3. What are the main side effects associated with this drug combination?

Common adverse events include neutropenia, anemia, fatigue, nausea, and gastrointestinal disturbances, manageable with supportive care.

4. Are there ongoing trials exploring new uses for Tipiracil and Trifluridine?

Yes, ongoing studies are investigating frontline treatment options, combination regimens with immunotherapies, and broader indications.

5. What markets are expected to drive the highest growth for this drug?

North America and Asia-Pacific are poised for the highest growth, due to high cancer prevalence, strong healthcare infrastructure, and ongoing clinical development.

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References

1. ClinicalTrials.gov Study NCT02994527
2. Market Research Future, "Colorectal Cancer Therapeutics Market Size, Share & Trends," 2022.
3. GlobalData, "Cancer Drugs Market Outlook," 2023.