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.
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.
NCT03306394 ↗	A Study of Trifluridine/Tipiracil (Also Known as S 95005 or TAS-102) in Patients With a Pretreated Colorectal Cancer That Has Spread (Metastatic).	Completed	ADIR, a Servier Group company	Phase 3	2016-10-18	The purpose of this study is to collect additional safety and efficacy data during treatment with trifluridine / tipiracil in patients with a pretreated metastatic colorectal cancer (mCRC). Eligible patients may receive an early access to trifluridine / tipiracil through this clinical study until progression of disease, unacceptable toxicity, investigator decision, patient refusal or until market authorization or reimbursement has been granted by the relevant Authority of the country where that patient is treated or until trifluridine / tipiracil is available by a doctor's prescription or can be accessed from another source or Sponsor decision.

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
Stage IVB Colorectal Cancer AJCC v8	6

Condition MeSH

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

Tipiracil hydrochloride; trifluridine Market Analysis and Financial Projection

Last updated: April 27, 2026

TIPIRACIL HYDROCHLORIDE + TRIFLURIDINE: Clinical-trials update, market analysis, and projections

What is the current clinical-trials signal for tipiracil hydrochloride + trifluoridine?

Tipiracil hydrochloride plus trifluridine (fixed-dose combination; marketed in multiple regions as trifluridine/tipiracil) is positioned for late-line colorectal cancer and other gastrointestinal oncology indications, with ongoing efforts focused on combination regimens and earlier-line placements.

Clinical development themes (observed across ongoing registries and sponsor programs):

• Combination regimens to expand response durability and broaden patient selection
• Earlier-line exploration to reduce reliance on later-line treatment windows
• Biomarker and stratification strategies to refine benefit-risk and target resistant disease biology

Practical read-through for R&D and investment

• Competitive pressure is concentrated in late-line GI oncology and in sequences around standard-of-care backbone therapy (fluoropyrimidine-, oxaliplatin-, irinotecan-based regimens), where incremental efficacy and tolerability drive uptake.
• Trials that pair the combination with agents that improve depth of response tend to show the highest commercial leverage if endpoints translate into meaningful OS or mOS gains with manageable toxicity.

Key watch items for trial monitoring

• Phase: prioritize registrational Phase 3 and high-enrollment Phase 2 programs with confirmatory endpoints
• Endpoint: OS and mOS trend lines matter more than ORR in heavily pretreated CRC
• Safety profile consistency: treatment-emergent hematologic toxicity and dose intensity are decisive for adoption and payor coverage pathways

Result: tipiracil/trifluridine remains a core late-line CRC option with an active development pipeline aimed at schedule, sequencing, and combination differentiation.

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How does the product fit the global market landscape?

Market structure

• Base therapy: late-line colorectal cancer where payers and clinicians expect clear survival benefit in pretreated populations
• Differentiation: oral administration with a defined toxicity profile, enabling outpatient delivery and sequence flexibility versus purely IV options

Commercial drivers

• CRC incidence and survival curve: creates a persistent addressable late-line pool
• Line-of-therapy depth: adoption correlates with how physicians perceive marginal OS benefit relative to available later-line alternatives
• Tolerability and dose management: hematologic toxicities influence adherence and physician preference, affecting real-world persistence

Competitive set (functional, not brand-level)

• Late-line CRC competitors with oral and IV options spanning fluoropyrimidine derivatives, targeted agents in molecularly defined subsets, and immune checkpoint strategies in MSI-H populations
• Local practice patterns shape which sequences preserve tipiracil/trifluridine as the “go-to” after standard regimens

Pricing and access mechanics that impact revenue

• Reimbursement depends on survival benefit evidence, prescribing restrictions by line, and health technology assessment outcomes
• Patient support programs can improve persistence, which is critical where dose interruptions affect net delivered therapy

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What is the market forecast logic for tipiracil/trifluridine?

Forecasting hinges on four interacting variables: eligible patient pool, share of treated patients, net price, and lifetime treatment duration (persistence).

1) Eligible patient pool

• Defined primarily by late-line CRC and any additional approved settings gained through trial readouts.
• Pool grows slower than incidence due to prior exposure to standards and death, but it remains stable because CRC survival permits multiple lines of therapy.

2) Share of treated patients

Determinants include:

• Physician familiarity and guideline inclusion
• Ability to manage toxicities (real-world dose intensity)
• Relative efficacy against late-line alternatives

3) Net price

• Influenced by discounting and payer negotiation
• Sensitive to new indication uptake and competitive entry

4) Treatment duration

• Oral dosing supports outpatient continuity but hematologic toxicity can reduce dose intensity.
• Programs and label-based dose modifications drive realized treatment duration and total drug usage per patient.

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What do projections imply for growth and downside scenarios?

A credible commercial projection for tipiracil/trifluridine should use three scenarios: base, upside, and downside. The scenario logic follows trial outcomes and sequence adoption.

Base case (steady late-line CRC footprint)

• Growth comes from gradual share gains, modest persistence improvements through dosing refinements, and geographic uptake where coverage expands.
• Revenue growth is supported by stable late-line volume and normalized utilization after initial adoption maturity.

Upside case (label expansion from successful trials)

• Additional approved indications or earlier-line adoption can pull patients from other regimens.
• Upside requires evidence strong enough to overcome payer and guideline inertia.

Downside case (competitive displacement or safety/persistence issues)

• New entrants or superior regimens in late-line sequences compress share.
• Safety-driven dose reductions reduce net drug consumption and can limit persistence, hurting revenue even if patient numbers remain stable.

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What is the investment and R&D take on the development pipeline?

Strategic priorities for the program

• Combination strategy selection: choose partners that improve OS/mOS and do not add prohibitive toxicity
• Sequence clarity: define where the regimen fits in real-world ordering after standard regimens
• Dose-intensity operationalization: show that clinicians can preserve benefit with manageable hematology events

Decision points for stakeholders

• Whether registrational endpoints meet pre-specified survival thresholds
• Whether adverse event rates translate into acceptable dose intensity in practice
• Whether guideline compendia adopt the regimen quickly after label expansion

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Key Takeaways

• Tipiracil hydrochloride + trifluridine remains a late-line colorectal cancer anchor with ongoing combination and sequence efforts.
• Commercial outcomes depend on four levers: eligible pool, share, net price, and persistence/dose intensity.
• Forecast upside requires clear label expansion supported by OS or mOS benefit with tolerability that sustains delivered dose.

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FAQs

1. What cancers does tipiracil/trifluridine primarily target?
   Late-line colorectal cancer is the core setting, with ongoing work aiming to expand across GI oncology segments.

2. Why is dose intensity a major driver of realized revenue for this regimen?
   Hematologic toxicity management affects how much drug patients actually receive, which directly changes total units consumed per treated patient.

3. What endpoints matter most for commercial adoption in late-line CRC?
   OS and mOS trends are the highest-impact measures for payers, guideline developers, and prescribing behavior.

4. How do combination trials change the competitive positioning?
   They can move tipiracil/trifluridine from a late-line default to a more central option if combinations show durable survival benefit without unacceptable additional toxicity.

5. What market variable typically creates the largest forecast dispersion?
   Share-of-treated-patients outcomes after new evidence, driven by guideline inclusion speed and real-world tolerability.

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References

[1] FDA. “TAS-102 (trifluridine and tipiracil hydrochloride) label.” U.S. Food and Drug Administration.
[2] EMA. “Lonsurf (trifluridine/tipiracil) summary of product characteristics.” European Medicines Agency.
[3] ClinicalTrials.gov. “Trifluridine and tipiracil hydrochloride (TAS-102) studies.” U.S. National Library of Medicine.
[4] NCCN Clinical Practice Guidelines in Oncology. “Colon Cancer.” National Comprehensive Cancer Network.
[5] ESMO Clinical Practice Guidelines. “Metastatic colorectal cancer.” European Society for Medical Oncology.