CLINICAL TRIALS PROFILE FOR REGORAFENIB

505(b)(2) Clinical Trials for Regorafenib

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 Regorafenib

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT01002378 ↗	The Effect of Food (High Fat and Low Fat Breakfast) on the Pharmacokinetics of Regorafenib	Completed	Bayer	Phase 1	2009-10-01	An open label, three way crossover study to determine the effect of a high fat breakfast or a low fat breakfast on the pharmacokinetics of a single dose of regorafenib.
NCT01068769 ↗	Regorafenib in Patients With Metastatic and/or Unresectable Gastrointestinal Stromal Tumor	Completed	Bayer	Phase 2	2010-02-01	The purpose of this research study is to determine the safety and activity of regorafenib in participants with advanced gastrointestinal stromal tumor (GIST) if the standard approved therapies, imatinib and sunitinib, have failed to control the disease. Regorafenib is a drug that blocks abnormally active signaling enzymes called "tyrosine kinases" which are important to the growth of GIST. This "tyrosine kinase inhibition" is similar to the way that both imatinib and sunitinib work; however, regorafenib blocks certain additional signaling pathways that are not blocked by imatinib or sunitinib. Regorafenib has been not been tested in GIST participants before this research study.
NCT01068769 ↗	Regorafenib in Patients With Metastatic and/or Unresectable Gastrointestinal Stromal Tumor	Completed	Brigham and Women's Hospital	Phase 2	2010-02-01	The purpose of this research study is to determine the safety and activity of regorafenib in participants with advanced gastrointestinal stromal tumor (GIST) if the standard approved therapies, imatinib and sunitinib, have failed to control the disease. Regorafenib is a drug that blocks abnormally active signaling enzymes called "tyrosine kinases" which are important to the growth of GIST. This "tyrosine kinase inhibition" is similar to the way that both imatinib and sunitinib work; however, regorafenib blocks certain additional signaling pathways that are not blocked by imatinib or sunitinib. Regorafenib has been not been tested in GIST participants before this research study.
NCT01068769 ↗	Regorafenib in Patients With Metastatic and/or Unresectable Gastrointestinal Stromal Tumor	Completed	Fox Chase Cancer Center	Phase 2	2010-02-01	The purpose of this research study is to determine the safety and activity of regorafenib in participants with advanced gastrointestinal stromal tumor (GIST) if the standard approved therapies, imatinib and sunitinib, have failed to control the disease. Regorafenib is a drug that blocks abnormally active signaling enzymes called "tyrosine kinases" which are important to the growth of GIST. This "tyrosine kinase inhibition" is similar to the way that both imatinib and sunitinib work; however, regorafenib blocks certain additional signaling pathways that are not blocked by imatinib or sunitinib. Regorafenib has been not been tested in GIST participants before this research study.
NCT01068769 ↗	Regorafenib in Patients With Metastatic and/or Unresectable Gastrointestinal Stromal Tumor	Completed	Massachusetts General Hospital	Phase 2	2010-02-01	The purpose of this research study is to determine the safety and activity of regorafenib in participants with advanced gastrointestinal stromal tumor (GIST) if the standard approved therapies, imatinib and sunitinib, have failed to control the disease. Regorafenib is a drug that blocks abnormally active signaling enzymes called "tyrosine kinases" which are important to the growth of GIST. This "tyrosine kinase inhibition" is similar to the way that both imatinib and sunitinib work; however, regorafenib blocks certain additional signaling pathways that are not blocked by imatinib or sunitinib. Regorafenib has been not been tested in GIST participants before this research study.
NCT01068769 ↗	Regorafenib in Patients With Metastatic and/or Unresectable Gastrointestinal Stromal Tumor	Completed	Oregon Health and Science University	Phase 2	2010-02-01	The purpose of this research study is to determine the safety and activity of regorafenib in participants with advanced gastrointestinal stromal tumor (GIST) if the standard approved therapies, imatinib and sunitinib, have failed to control the disease. Regorafenib is a drug that blocks abnormally active signaling enzymes called "tyrosine kinases" which are important to the growth of GIST. This "tyrosine kinase inhibition" is similar to the way that both imatinib and sunitinib work; however, regorafenib blocks certain additional signaling pathways that are not blocked by imatinib or sunitinib. Regorafenib has been not been tested in GIST participants before this research study.
NCT01068769 ↗	Regorafenib in Patients With Metastatic and/or Unresectable Gastrointestinal Stromal Tumor	Completed	Suzanne George, MD	Phase 2	2010-02-01	The purpose of this research study is to determine the safety and activity of regorafenib in participants with advanced gastrointestinal stromal tumor (GIST) if the standard approved therapies, imatinib and sunitinib, have failed to control the disease. Regorafenib is a drug that blocks abnormally active signaling enzymes called "tyrosine kinases" which are important to the growth of GIST. This "tyrosine kinase inhibition" is similar to the way that both imatinib and sunitinib work; however, regorafenib blocks certain additional signaling pathways that are not blocked by imatinib or sunitinib. Regorafenib has been not been tested in GIST participants before this research study.

Clinical Trial Conditions for Regorafenib

Condition Name

Condition Name for Regorafenib
Intervention	Trials
Colorectal Cancer	34
Metastatic Colorectal Cancer	30
Hepatocellular Carcinoma	23
Colorectal Neoplasms	23

Condition MeSH

Condition MeSH for Regorafenib
Intervention	Trials
Colorectal Neoplasms	130
Carcinoma, Hepatocellular	43
Carcinoma	38
Gastrointestinal Stromal Tumors	26

Clinical Trial Locations for Regorafenib

Trials by Country

Trials by Country for Regorafenib
Location	Trials
United States	659
Italy	114
China	112
Japan	88
Germany	76

Trials by US State

Trials by US State for Regorafenib
Location	Trials
California	50
Florida	39
Texas	38
New York	33
Pennsylvania	32

Clinical Trial Progress for Regorafenib

Clinical Trial Phase

Clinical Trial Phase for Regorafenib
Clinical Trial Phase	Trials
PHASE3	2
PHASE2	40
PHASE1	9

Clinical Trial Status

Clinical Trial Status for Regorafenib
Clinical Trial Phase	Trials
RECRUITING	113
Completed	64
Not yet recruiting	36

Clinical Trial Sponsors for Regorafenib

Sponsor Name

Sponsor Name for Regorafenib
Sponsor	Trials
Bayer	92
National Cancer Institute (NCI)	18
Asan Medical Center	12

Sponsor Type

Sponsor Type for Regorafenib
Sponsor	Trials
Other	325
Industry	189
NIH	18

Regorafenib: Clinical Trials Update, Market Analysis, and Future Outlook

Last updated: October 28, 2025

Introduction

Regorafenib (brand name: Stivarga) is an oral multikinase inhibitor developed by Bayer AG, approved for multiple oncological indications including metastatic colorectal cancer (mCRC), gastrointestinal stromal tumors (GIST), and hepatocellular carcinoma (HCC). As a targeted therapy, regorafenib's efficacy hinges on its ability to inhibit several key kinases involved in tumor angiogenesis, oncogenesis, and the tumor microenvironment. This article provides a comprehensive update on regorafenib’s clinical trials, market dynamics, and future projections critical for pharmaceutical stakeholders and investors.

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Clinical Trials Update

Recent Phase III and Regulatory Developments

Regorafenib's primary approvals stem from pivotal Phase III trials: CORRECT for mCRC, GRID for GIST, and RESORCE for HCC. Recent updates reflect ongoing efforts to expand its therapeutic scope.

• Hepatocellular Carcinoma: The REFLECT trial, a Phase III study comparing regorafenib to sorafenib in first-line HCC, demonstrated non-inferiority, positioning regorafenib as a potential first-line alternative. Bayer is currently exploring marketing approval extensions in Asia based on these findings.

• Colorectal Cancer: The CONCUR trial reinforced regorafenib's efficacy in Asian populations, showing improved overall survival (OS) and progression-free survival (PFS). Extensions include combination studies with immunotherapies, leveraging its anti-angiogenic profile to potentially enhance immune response.

• Gastrointestinal Stromal Tumors: The ongoing phase IV studies investigate regorafenib in GIST patients resistant to imatinib and sunitinib, with preliminary data indicating manageable safety profiles and sustained efficacy.

Investigational and Early-Phase Trials

Several emerging studies aim to broaden regorafenib's applications:

• Combination Therapies: Trials assessing regorafenib with immune checkpoint inhibitors (e.g., pembrolizumab, nivolumab) show promise, targeting synergistic anti-tumor effects in HCC and other solid tumors.

• Other Solid Tumors: Phase II trials are investigating efficacy in ovarian, gastric, and pancreatic cancers, reflecting recognition of its multi-kinase inhibitory mechanism's potential for broad-spectrum anti-cancer activity.

Safety and Adverse Events

Ongoing surveillance underscores predictable adverse events like hypertension, hand-foot skin reaction, diarrhea, and hepatotoxicity. Dose optimization studies aim to mitigate toxicity, improving patient tolerability for extended therapy durations.

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Market Analysis

Market Size and Segmentation

Regorafenib's global market in oncology is projected to reach approximately USD 3.5 billion by 2028, driven by increasing approval breadth and expanding indications.

• Primary Markets: The U.S., Europe, and Asia-Pacific dominate sales, driven by the prevalence of colorectal cancer and HCC. The U.S. accounts for roughly 40% of revenue, with Europe contributing around 25%.

• Key Segments: The dominant segments include metastatic colorectal cancer, gastrointestinal stromal tumors, and hepatocellular carcinoma. The rising incidence of liver cancer in Asia and aging populations in Western countries bolster demand.

Competitive Landscape

Regorafenib faces competition from other multi-kinase inhibitors such as sorafenib, cabozantinib, and lenvatinib. Its unique pharmacodynamics and broad kinase inhibition profile give it a competitive edge, particularly in indications with unmet needs like advanced HCC and refractory mCRC.

• Pricing and Reimbursement: As an oncology agent, regorafenib commands premium pricing, with cost-effectiveness varying across markets. Payers in high-income countries increasingly require value-based assessments, influencing formulary positioning.

Market Penetration and Challenges

Despite established efficacy, market penetration faces obstacles:

• Side Effect Profile: Tolerability issues limit treatment continuity, especially in elderly or frail patients.

• Competition and Off-Label Use: Introduction of newer agents with improved safety profiles or combination regimens may erode regorafenib's market share.

• Access and Adoption: Regulatory delays in expanding indications or regional approvals slow adoption rates in emerging markets.

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Market Projections and Future Outlook

Growth Drivers

• Expanding Indications: Regulatory submissions for HCC as first-line therapy and exploration in other tumors could significantly augment revenues.

• Combination Therapies: Positive trial outcomes for regorafenib combined with immunotherapies may redefine its positioning, leading to combination product approvals.

• Biomarker Development: Advances in predictive biomarkers will enable personalized therapy, improving response rates and patient outcomes, further incentivizing use.

Potential Challenges

• Regulatory Hurdles: Delays or refusals for new indications could hamper growth trajectories.

• Pricing Pressures: Increased scrutiny over drug pricing and healthcare budgets could limit profitability.

• Safety Concerns: Manageable but persistent adverse effects necessitate careful patient selection and supportive care.

Long-Term Projections

Based on current trajectories, regorafenib's global market is expected to grow at a CAGR of 7%–9% over the next five years, driven by new indications, combination strategies, and heightened awareness among prescribers. The continued research into its immunomodulatory effects positions regorafenib as a cornerstone in future combination regimens.

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Key Takeaways

• Clinical advances position regorafenib as a versatile agent, with ongoing trials exploring expanded indications, particularly in hepatocellular carcinoma and combination therapies.
• Market dynamics predict steady growth, supported by the rising global burden of relevant cancers and evolving treatment paradigms.
• Challenges remain around toxicity management, regulatory approvals, and competitive pressures, emphasizing the need for strategic positioning and ongoing clinical innovation.
• Future opportunities hinge on biomarker-driven personalized medicine approaches and integration into combination regimens with immunotherapies.
• Stakeholders should monitor regulatory updates, clinical trial results, and evolving pricing landscapes to optimize investment and commercialization strategies.

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FAQs

1. What are the main indications for regorafenib currently approved worldwide?

Regorafenib is approved for metastatic colorectal cancer, gastrointestinal stromal tumors, and hepatocellular carcinoma, primarily based on pivotal Phase III trials demonstrating survival benefits in these indications.

2. Are there ongoing trials exploring regorafenib in other cancer types?

Yes, Phase II and III trials are investigating regorafenib in ovarian, gastric, pancreatic, and other solid tumors, often in combination with immunotherapies or targeted agents.

3. How does regorafenib compare to other multikinase inhibitors in efficacy and safety?

Regorafenib shows comparable efficacy to agents like sorafenib in HCC and sunitinib in GIST, with a similar adverse event profile. Its broader kinase inhibition may offer a therapeutic advantage in certain contexts, but tolerability remains a challenge.

4. What are the prospects for regorafenib in the realm of combination therapy?

Preliminary trials demonstrate potential synergistic effects when combined with immune checkpoint inhibitors, which could pioneer regorafenib's role in immune-oncology strategies.

5. What are the key regulatory considerations for expanding regorafenib's indications?

Regulatory approval hinges on demonstrating clinical benefit, manageable safety profiles, and biomarker validation where applicable. Expanding indications will require robust trial data and alignment with unmet medical needs.

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References

[1] Bayer AG. (2022). Stivarga (Regorafenib) Prescribing Information.
[2] Lim, H. Y., et al. (2021). Recent Advances in the Clinical Development of Regorafenib. Frontiers in Oncology.
[3] GlobalData. (2022). Regorafenib Market Analysis and Forecasts.
[4] Cheng, A., et al. (2020). Clinical Trials in Hepatocellular Carcinoma: Focus on Targeted Therapies. Liver Cancer.
[5] European Medicines Agency. (2022). Regorafenib Summary of Product Characteristics.