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
Colorectal Neoplasms	23
Hepatocellular Carcinoma	23

Condition MeSH

Condition MeSH for Regorafenib
Intervention	Trials
Colorectal Neoplasms	130
Carcinoma, Hepatocellular	44
Carcinoma	38
Gastrointestinal Stromal Tumors	26

Clinical Trial Locations for Regorafenib

Trials by Country

Trials by Country for Regorafenib
Location	Trials
United States	659
China	114
Italy	114
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	41
PHASE1	9

Clinical Trial Status

Clinical Trial Status for Regorafenib
Clinical Trial Phase	Trials
Recruiting	114
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	190
NIH	18

Regorafenib: Clinical Trials Update, Market Analysis, and 2030 Projection

Last updated: April 28, 2026

What is regorafenib’s current clinical trial position?

Regorafenib (Stivarga) is an oral multikinase inhibitor used in oncology, with ongoing clinical work spanning combinations, new lines, and biomarker-driven strategies. As of the latest publicly available trial landscape, the most active development themes are:

• Combination regimens with immune checkpoint inhibitors and cytotoxic backbones
• Earlier lines and broader histology coverage beyond current approvals
• Biomarker selection using molecular signatures and imaging endpoints

Snapshot of active development directions (high level)

Because regorafenib is already integrated into standard care in multiple settings, most late-stage efforts focus on whether it can improve outcomes when paired with:

• PD-1/PD-L1 inhibitors
• VEGFR-targeting agents in different schedules/dosing
• Radiation and systemic chemotherapy sequencing

Regulatory and evidence base context: regorafenib’s clinical footprint rests on pivotal outcomes across metastatic colorectal cancer (mCRC), gastrointestinal stromal tumor (GIST), and hepatocellular carcinoma (HCC), with subsequent expansions and label refinements. The current clinical cycle is predominantly incremental in signal-seeking, with trial designs built around progression-free survival (PFS), overall survival (OS), and response durability.

What endpoints and trial designs dominate?

Across ongoing regorafenib studies, the dominant endpoints remain:

• PFS as the primary readout in combination-phase trials
• OS in phase 3 confirmatory studies, particularly where standards of care change over time
• Safety and dose intensity as a key secondary, given established class toxicities (hand-foot skin reaction, hypertension, liver enzyme elevations, diarrhea, fatigue)

Actionable implication for investors/R&D: late-stage probability of success increases when a trial:

• uses a clear control arm aligned to the latest standard of care
• includes dose-optimization plans to reduce discontinuation
• stratifies by baseline disease biology (CT/biomarkers) when feasible

How is regorafenib positioned in the global market?

Regorafenib is an established oncology product with a mature commercial footprint. Market performance is driven by:

• Line-of-therapy dynamics in mCRC, GIST, and HCC
• Availability of subsequent therapies that can blunt incremental OS impact
• Competition from other targeted agents and immunotherapy combinations
• Geographic and reimbursement coverage, which can vary significantly by indication

Market drivers by indication (commercial logic)

Metastatic colorectal cancer (mCRC)

• Regorafenib’s role is anchored in later-line use where options are sequential and heterogeneous.
• Commercial velocity depends on clinician adoption and time-to-treatment after prior lines.

Gastrointestinal stromal tumor (GIST)

• Regorafenib benefits from a niche in patients who progress after multiple prior TKI lines.
• Uptake tracks with guideline inclusion and formulary access.

Hepatocellular carcinoma (HCC)

• Regorafenib’s market is linked to the fast-changing HCC landscape (first-line immunotherapy and combination standards), which shifts the proportion of patients arriving in later-line settings.

Pricing and competitive context (what matters for projections)

At a high level, regorafenib market growth (or decline) typically hinges on:

• Net price erosion from competition and tendering
• Conversion rates from trial evidence into guideline and payer coverage
• Speed of displacement as new standards move into earlier lines

What is the market projection through 2030?

Base-case market model (directional projection structure)

A credible projection for a mature oncology drug uses:

1. Indication-level patient pool trajectories (incidence, diagnosis rates, treatment sequencing)
2. Penetration rates (share of eligible treated patients)
3. Price and reimbursement trajectory (gross-to-net dilution)
4. Competitive displacement from newer regimens
5. Patent and generic risk (where applicable by region)

Projection (global regorafenib sales)

A complete, defensible numeric projection requires up-to-date market consensus inputs (global sales history, regional reimbursement, competitive landscape, and patent status by country). Without that dataset in the current context, a precise 2030 dollar forecast cannot be produced to investment-grade standards.

What can be stated precisely from market mechanics:

• Regorafenib’s sales trajectory is most likely steady-to-slow growth if combinations/biomarker-selected trials expand label scope and payers keep coverage.
• Regorafenib’s trajectory can become declining if standards of care shift further forward, reducing the proportion of patients reaching later-line use where regorafenib is most valuable.

Which clinical programs are most likely to move revenue?

Programs that typically shift commercial outcomes

In mature oncology assets like regorafenib, revenue-impacting programs usually do at least one of:

• Expand line of therapy (earlier use increases eligible patient pool)
• Expand monotherapy or combo utility to additional molecularly defined groups
• Improve OS in phase 3 so the product retains central guideline relevance as standards evolve

What to watch in trial readouts (investment checklist)

• Magnitude of PFS and OS separation versus best available control
• Dose intensity and discontinuation rates in combo regimens
• Subgroup consistency (avoid signals that collapse outside prespecified strata)
• Safety management evidence that reduces real-world discontinuations

What are the key risk factors for clinical and commercial outcomes?

Clinical risks

• Heterogeneity of prior therapies in later-line oncology trials reduces effect sizes
• Cross-trial endpoint drift as standards change during the study window
• Toxicity stacking in combination regimens can increase dropouts

Commercial risks

• Displacement as newer first-line or second-line regimens move earlier
• Payer friction if incremental benefit declines relative to standard combinations
• Pricing pressure in multi-TKI competitive settings

What is the business outlook for regorafenib?

Regorafenib’s outlook is tied to whether new trials:

• convert into expanded labeling or
• establish combination regimens that maintain guideline relevance

If ongoing development demonstrates clear survival benefit with manageable safety, it can extend the commercial runway. If efficacy signals are modest or constrained to small subgroups, regorafenib’s market will likely remain anchored to current late-line niches and face steady competitive pressure.

---

Key Takeaways

• Regorafenib’s current clinical activity is dominated by combinations and earlier-line exploration, with endpoints focused on PFS and OS plus safety-driven dose management.
• Commercial performance is driven by therapy sequencing, payer access, and standard-of-care displacement in mCRC, GIST, and HCC.
• A precise 2030 dollar forecast requires contemporaneous market inputs; the strategic direction is clear: label expansion and combination success support growth, while earlier displacement and price erosion drive decline.

---

FAQs

1) Is regorafenib primarily a monotherapy or combination-drug asset in current development?
Combination strategies dominate new late-stage interest, while monotherapy remains important for later-line positioning where regorafenib is already established.

2) What endpoint best reflects regorafenib’s impact in most ongoing trials?
PFS is typically the primary readout in combination development, with OS used in confirmatory settings and where trial design supports long-term differentiation.

3) What safety issues most influence regorafenib uptake?
The class and drug-specific toxicities most associated with discontinuation risk include hand-foot skin reaction, hypertension, liver enzyme elevations, diarrhea, and fatigue, which can limit dose intensity.

4) How does the changing HCC standard of care affect regorafenib’s sales?
Earlier uptake of immunotherapy combinations can reduce the number of patients reaching later-line settings where regorafenib is used, impacting addressable demand.

5) What trial outcomes would most strengthen a 2030 revenue outlook?
Clear OS improvement with consistent subgroup benefit plus evidence of manageable combination toxicity and maintained dose intensity.

---

References

[1] FDA. Stivarga (regorafenib) Prescribing Information. U.S. Food and Drug Administration.
[2] EMA. Stivarga (regorafenib) Product Information. European Medicines Agency.
[3] ClinicalTrials.gov. Regorafenib trials (search results and listings). U.S. National Library of Medicine.
[4] NCCN Clinical Practice Guidelines in Oncology. Colorectal Cancer, Gastrointestinal Stromal Tumor, Hepatocellular Carcinoma (latest editions). National Comprehensive Cancer Network.