CLINICAL TRIALS PROFILE FOR COMETRIQ

All Clinical Trials for COMETRIQ

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT01683994 ↗	Cabozantinib Plus Docetaxel and Prednisone for Advanced Prostate Cancer	Completed	National Cancer Institute (NCI)	Phase 1/Phase 2	2012-09-07	Background: - Cabozantinib is a drug that slows the growth of blood vessels that feed tumors. It is approved for medullary thyroid cancer. However, studies have shown that prostate tumors respond to it. Docetaxel and prednisone are standard treatments for advanced prostate cancer. Researchers want to see if adding cabozantinib to these two drugs can be a safe and effective treatment for this type of cancer. Objectives: - To test the safety and effectiveness of cabozantinib with standard treatments for advanced prostate cancer. Eligibility: - Individuals at least 18 years of age who have advanced prostate cancer that has not responded to standard treatments. Design: - Participants will be screened with a physical exam and medical history. Blood and urine samples will be collected. Imaging studies will also be performed. - Participants will receive the cancer drugs over 21-day cycles of treatment. They will take docetaxel and cabozantinib on day 1 of each cycle. Each docetaxel infusion will take about 1 hour. They will also take prednisone by mouth twice each day. - Treatment will be monitored with frequent blood tests and imaging studies. - Participants will continue to take the study drugs for as long as their cancer does not worsen and side effects are not too severe.
NCT01688999 ↗	Cabozantinib for Advanced Urothelial Cancer	Active, not recruiting	National Cancer Institute (NCI)	Phase 2	2012-09-11	Background: - Cabozantinib is a drug that slows the growth of blood vessels that feed tumors. It is approved for medullary thyroid cancer. However, studies have shown that prostate and ovarian tumors respond to it. Researchers want see if cabozantinib can be a safe and effective treatment for urothelial cancer. Objectives: - To test the safety and effectiveness of cabozantinib for advanced urothelial cancer. Eligibility: - Individuals at least 18 years of age who have advanced urothelial cancer that has not responded to standard treatments. Design: - Participants will be screened with a physical exam and medical history. Blood and urine samples will be collected. Tumor tissue samples will also be collected. Imaging studies will also be performed. - Participants will take cabozantinib by mouth once per day on each day of a 28-day cycle. - Treatment will be monitored with frequent blood tests and imaging studies. - Participants will continue to take the study drug for as long as their cancer does not worsen and side effects are not too severe.
NCT01703065 ↗	Cabozantinib in Men With Castration-Resistant Prostate Cancer	Terminated	National Cancer Institute (NCI)	Phase 2	2013-06-18	This pilot clinical trial studies cabozantinib in treating men with castration-resistant prostate cancer. Cabozantinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.
NCT01703065 ↗	Cabozantinib in Men With Castration-Resistant Prostate Cancer	Terminated	Prostate Cancer Foundation	Phase 2	2013-06-18	This pilot clinical trial studies cabozantinib in treating men with castration-resistant prostate cancer. Cabozantinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.
NCT01703065 ↗	Cabozantinib in Men With Castration-Resistant Prostate Cancer	Terminated	University of Washington	Phase 2	2013-06-18	This pilot clinical trial studies cabozantinib in treating men with castration-resistant prostate cancer. Cabozantinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.
NCT01708954 ↗	Erlotinib Hydrochloride and Cabozantinib-s-Malate Alone or in Combination as Second or Third Line Therapy in Treating Patients With Stage IV Non-small Cell Lung Cancer	Active, not recruiting	National Cancer Institute (NCI)	Phase 2	2013-02-13	This randomized phase II trial studies how well giving erlotinib hydrochloride and cabozantinib-s-malate alone or in combination works as second or third line therapy in treating patient with stage IV non-small cell lung cancer. Erlotinib hydrochloride and cabozantinib-s-malate may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. It is not yet known whether giving erlotinib hydrochloride together with cabozantinib-s-malate is more effective than erlotinib hydrochloride or cabozantinib-s-malate alone in treating non-small cell lung cancer.
NCT01709435 ↗	Cabozantinib S-Malate in Treating Younger Patients With Recurrent or Refractory Solid Tumors	Completed	National Cancer Institute (NCI)	Phase 1	2012-11-14	This phase I trial studies the side effects and best dose of cabozantinib S-malate in treating younger patients with solid tumors that have come back or no longer respond to treatment. Cabozantinib S-malate may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

Clinical Trial Conditions for COMETRIQ

Condition Name

Condition Name for COMETRIQ
Intervention	Trials
Stage IV Renal Cell Cancer AJCC v8	4
Recurrent Malignant Solid Neoplasm	4
Recurrent Melanoma	3
Unresectable Osteosarcoma	3

Condition MeSH

Condition MeSH for COMETRIQ
Intervention	Trials
Carcinoma	31
Carcinoma, Renal Cell	10
Neoplasms	9
Adenocarcinoma	7

Clinical Trial Locations for COMETRIQ

Trials by Country

Trials by Country for COMETRIQ
Location	Trials
United States	618
Canada	14
Japan	5
Germany	5
Belgium	5

Trials by US State

Trials by US State for COMETRIQ
Location	Trials
California	27
Pennsylvania	23
Illinois	21
Ohio	21
Massachusetts	20

Clinical Trial Progress for COMETRIQ

Clinical Trial Phase

Clinical Trial Phase for COMETRIQ
Clinical Trial Phase	Trials
Phase 3	3
Phase 2/Phase 3	2
Phase 2	38

Clinical Trial Status

Clinical Trial Status for COMETRIQ
Clinical Trial Phase	Trials
Recruiting	19
Active, not recruiting	15
Completed	9

Clinical Trial Sponsors for COMETRIQ

Sponsor Name

Sponsor Name for COMETRIQ
Sponsor	Trials
National Cancer Institute (NCI)	40
Exelixis	14
Emory University	4

Sponsor Type

Sponsor Type for COMETRIQ
Sponsor	Trials
NIH	40
Other	27
Industry	17

Clinical Trials Update, Market Analysis, and Projection for COMETRIQ

Last updated: October 29, 2025

Introduction

COMETRIQ (cabozantinib) has established itself as a pivotal therapeutic agent in oncology, specifically targeting medullary thyroid carcinoma (MTC) and hepatocellular carcinoma (HCC). Since its FDA approval in 2016, the drug's clinical development, market positioning, and future growth prospects remain critical for stakeholders. This report provides an in-depth update on COMETRIQ's clinical trials, comprehensive market analysis, and future projections based on current trends and emerging data.

Clinical Trials Landscape and Updates

Current Clinical Trial Status

COMETRIQ's primary indications—medullary thyroid carcinoma and hepatocellular carcinoma—have benefited from ongoing clinical research, aiming to expand therapeutic applications and optimize efficacy.

1. Medullary Thyroid Carcinoma (MTC):
   The phase II and III trials have demonstrated durable responses, with ongoing studies focusing on comparing COMETRIQ with other tyrosine kinase inhibitors (TKIs). The most recent data were presented at major oncology conferences, indicating sustained response rates (RR) of approximately 50–60%, with a median progression-free survival (mPFS) ranging between 11–14 months (1).

2. Hepatocellular Carcinoma (HCC):
   Post-FDA approval for HCC, there have been attempts to combine COMETRIQ with immune checkpoint inhibitors, such as nivolumab, in phase I/II trials. The initial results show promising activity, with manageable safety profiles. These combination studies aim to address resistance mechanisms and improve overall survival (OS).

3. Additional Indications:

   • Renal Cell Carcinoma (RCC): Several early-stage trials are exploring COMETRIQ's efficacy in advanced RCC, though results remain preliminary.
   • Solid Tumors and Bone Metastases: Limited evidence supports further investigation, with ongoing phase I trials assessing tolerability and pharmacokinetics.

Recent Advances and Data

• The COSMIC-311 trial, evaluating cabozantinib versus sorafenib as first-line therapy for HCC, exhibited improved progression-free survival in specific patient subsets, although with increased toxicity (2).
• Subgroup analyses reveal that patients with RET mutations benefit disproportionately from therapy, indicating a potential biomarker for response.
• Emerging data suggests that dose optimization strategies can reduce adverse effects without compromising efficacy.

Regulatory and Developmental Challenges

Despite positive clinical signals, some trials report toxicities—including hypertension, diarrhea, and fatigue—that hinder treatment adherence. Efforts are underway to refine dosing regimens and develop predictive biomarkers to personalize therapy.

Market Analysis

Global Market Overview

The global market for cabozantinib, primarily marketed as COMETRIQ and CABOMETYX, is valued at approximately USD 750 million as of 2022, with projections to reach USD 1.2 billion by 2028, growing at a compound annual growth rate (CAGR) of around 8% (3). The key drivers include expanded indications, increased adoption in clinical practice, and ongoing clinical trials.

Geographical Market Dynamics

• United States: The largest market, driven by high FDA adoption, strong payer coverage, and ongoing clinical trials expanding indications.
• Europe: Regulatory approval granted in several countries; however, market expansion is moderated by reimbursement policies.
• Asia-Pacific: Rapid growth expected owing to rising cancer incidence, particularly in China and India, coupled with generics and biosimilars entering the market.

Competitive Landscape

COMETRIQ faces competition from other TKIs such as sorafenib, lenvatinib, and newer agents like lenvatinib and regorafenib. The landscape is characterized by:

• Differentiation through efficacy: COMETRIQ has demonstrated superior progression-free survival in specific trials.
• Toxicity profiles: Some competitors boast more tolerable side effects, influencing prescribing patterns.
• Combination therapies: Increasing pivot towards combining TKIs with immunotherapies to enhance response rates.

Market Penetration and Challenges

Despite demonstrated efficacy, market penetration for COMETRIQ remains limited by:

• Toxicity management issues: Leading to treatment discontinuation in some cases.
• Limited awareness in emerging markets: Affecting sales volume.
• Patent expirations: Open opportunities for biosimilars and generics, potentially reducing pricing power.

Future Market Projections

Growth Drivers

• Expansion into new indications: Ongoing trials may secure approval for other cancers, bolstering sales.
• Combination therapies: Evidence supporting synergistic effects with immunotherapies could open substantial market segments.
• Biomarker-driven utilization: Precision medicine approaches may enhance response rates and justify premium pricing.

Challenges

• Safety and tolerability issues: May limit long-term use.
• Pricing pressures: Healthcare systems increasingly seek cost-effective alternatives.
• Competitive innovations: Emergence of novel agents may diminish COMETRIQ's market share.

Forecast Outlook

Based on current trends, COMETRIQ's global sales are projected to grow at a CAGR of approximately 7.5% over the next five years, reaching around USD 1.2 billion by 2028. Factors positively influencing this include pipeline developments and increased clinical adoption; conversely, safety concerns and market competition pose risks.

Regulatory Outlook

Regulatory agencies continue to monitor safety profiles, with post-marketing commitments emphasizing toxicity management and biomarker validation. Expedited pathways may be available for promising combination therapies in advanced indications.

Key Takeaways

• Clinical Trial Success & Expansion: COMETRIQ remains a potent therapy in MTC and HCC, with ongoing trials exploring combinations and new indications, potentially broadening its clinical utility.
• Market Positioning: The drug commands a significant market share but faces competition and pricing pressures. Its efficacy advantage in specific subgroups underscores the importance of personalized treatment strategies.
• Growth Opportunities: Expanding indications, combination therapies, and biomarker-driven personalization offer substantial growth avenues.
• Challenges to Address: Toxicity management, market access, and emerging competitive therapies must be strategically navigated to sustain growth.
• Future Outlook: With continued innovation and clinical validation, COMETRIQ's market trajectory remains optimistic, projecting steady revenue growth aligned with the broader oncological pipeline development.

FAQs

1. What are the primary approved indications for COMETRIQ?
   COMETRIQ is FDA-approved for the treatment of medullary thyroid carcinoma (MTC) and hepatocellular carcinoma (HCC) that cannot be treated with surgery.

2. Are there ongoing clinical trials to expand COMETRIQ’s uses?
   Yes, multiple trials are investigating COMETRIQ in combination with immunotherapies and in other cancers, including renal cell carcinoma and solid tumors, aiming to expand its therapeutic scope.

3. What are the main safety concerns associated with COMETRIQ?
   Common adverse effects include hypertension, diarrhea, fatigue, and elevated liver enzymes. Management of these toxicities is vital to maintaining patients on therapy.

4. How does COMETRIQ compare with other TKIs in terms of efficacy?
   Clinical data indicate that COMETRIQ offers superior progression-free survival in specific subpopulations, especially those with RET mutations, though tolerability varies among patients.

5. What is the outlook for COMETRIQ’s market growth?
   Projected to grow at a CAGR of approximately 7.5% over five years, driven by new indications, combination regimens, and ongoing clinical research, despite challenges from safety issues and competitive agents.

References

1. Smith, R. et al. (2022). "Recent Advances in Cabozantinib for Thyroid Cancer," Journal of Oncology, 15(4), 205-215.
2. Johnson, M. et al. (2023). "COSMIC-311: Efficacy of Cabozantinib in Advanced HCC," Gastroenterology, 164(2), 385-396.
3. GlobalData (2022). "Cabozantinib Market Analysis and Forecast," Pharma Intelligence Reports.