CLINICAL TRIALS PROFILE FOR IPILIMUMAB

Biosimilar Clinical Trials for ipilimumab

This table shows clinical trials for biosimilars. See the next table for all clinical trials

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT06841185 ↗	A Study to Compare the Efficacy, Safety, Immunogenicity, and Pharmacokinetic Profile of HLX13 with YERVOY As a First-Line Treatment for Patients with Unresectable Hepatocellular Carcinoma	NOT_YET_RECRUITING	Shanghai Henlius Biotech	PHASE3	2025-04-30	This is a multicenter, randomized, double-blind, parallel-controlled integrated phase I/III clinical study to evaluate the efficacy, safety, PK, and immunogenicity of HLX13 and YERVOY in patients with unresectable hepatocellular carcinoma who have not received prior systemic therapy.
NCT07176650 ↗	Phase I Clinical Study To Evaluate Pharmacokinetic Profile, Safety, Efficacy and Immunogenicity Of Ipilimumab Biosimilar HLX13 Vs. YERVOY (US-Sourced YERVOY) As A First-Line Treatment For Patients With Unresectable Hepatocellular Carcinoma	NOT_YET_RECRUITING	Shanghai Henlius Biotech	PHASE1	2025-10-01	This is a multicenter, randomized, double-blind, parallel-controlled, phase I clinical study to evaluate the PK characteristics, safety, efficacy, and immunogenicity of HLX13 and US-sourced YERVOY in patients with unresectable hepatocellular carcinoma who have not received prior systemic therapy.

All Clinical Trials for ipilimumab

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00060372 ↗	Ipilimumab After Allogeneic Stem Cell Transplant in Treating Patients With Persistent or Progressive Cancer	Completed	National Cancer Institute (NCI)	Phase 1	2003-04-01	This phase I trial is studying how well ipilimumab works after allogeneic stem cell transplant in treating patients with persistent or progressive cancer. Monoclonal antibodies can locate cancer cells and either kill them or deliver cancer-killing substances to them without harming normal cells.
NCT00094653 ↗	MDX-010 Antibody, MDX-1379 Melanoma Vaccine, or MDX-010/MDX-1379 Combination Treatment for Patients With Unresectable or Metastatic Melanoma	Completed	Bristol-Myers Squibb	Phase 3	2004-09-01	The purpose of this study is to determine the safety and efficacy of MDX-010 (ipilimumab, BMS-734016) (anti-CTLA4) in combination with MDX-1379 (gp100, BMS-734019) in patients with previously treated, unresectable Stage III or IV melanoma. Survival time will be evaluated, as well as patient responses and time to disease progression. Eligible patients are those who in response to a single regimen containing interleukin-2 (IL-2), dacarbazine, and/or temozolomide, have 1) relapsed following an objective response (partial response/complete response [PR/CR]); 2) failed to demonstrate an objective response (PR/CR); or 3) could not tolerate such a regimen due to unacceptable toxicity. Patients will be randomized into one of three groups, and will receive one of the following treatments: MDX-010 alone, MDX-1379 alone, or MDX-010 in combination with MDX-1379.
NCT00113984 ↗	Vaccine and Antibody Treatment of Prostate Cancer	Completed	National Cancer Institute (NCI)	Phase 1	2005-06-08	This study will evaluate the side effects of a fixed dose of vaccine and GM-CSF with increasing doses of anti-CTLA-4 antibody in patients with advanced prostate cancer. The vaccine consists of a "priming vaccine" called PROSTVAC/TRICOM, made from vaccinia virus, and a "boosting vaccine" called PROSTVAC-F/TRICOM, made from fowlpox virus. GM-CSF is a chemical that boosts the immune system, and anti-CTLA-4 antibody is a protein that may improve anti-tumor activity and the response to the vaccines. DNA is inserted into the priming and boosting vaccine viruses to cause production of proteins that enhance immune activity and also to produce prostate specific antigen (PSA)-a protein that is normally produced by the patient's tumor cells. Patients 18 years of age and older with androgen-insensitive prostate cancer that has spread beyond the original site may be eligible for this 7-month study. Candidates must have disease that has worsened despite treatments with hormones and up to one chemotherapy regimen. Their tumor must produce PSA, and they must have no history of allergy to eggs or egg products Candidates are screened with a medical history and physical examination, blood and urine tests, electrocardiogram, pathological confirmation of the diagnosis and presence of the PSA marker, chest x-rays, imaging studies to assess the extent of tumor, and, if clinically indicated, a cardiologic evaluation. Participants receive the priming vaccination on study day 1. After 2 weeks and then again every 4 weeks while on the study, they receive a boosting vaccine. All vaccines are injected under the skin. On the day of each vaccination and daily for the next 3 days, patients receive an injection of GM-CSF to increase the number of immune cells at the vaccination site. On the day of the first six boosting vaccinations, they receive anti-CTLA-4 antibody as an infusion through a vein over 90 minutes. Patients are monitored for safety and treatment response with the following tests and procedures: - Blood and urine tests monthly, or more often if needed, to monitor liver, kidney, and other organ function. - Imaging studies to assess the tumor before starting treatment, again around study days 99 and 183, and then every 3 months after that while on study. - Apheresis (a procedure for collecting immune cells called lymphocytes) to measure the immune response to treatment. Apheresis is done three times: before starting the study and again around study days 99 and 183. For this procedure, blood is collected through a needle in an arm vein. The blood circulates through a machine that separates it into its components by spinning, and the lymphocytes are extracted. The rest of the blood is returned to the patient through the same needle. This will only be done in participants who have the tissue marker HLA-A2 (about 50% of patients). Patients whose disease responds to treatment and who do not develop severe side effects may continue treatment beyond the initial 7-month study period on vaccine alone (without the antibody). After treatment is completed, patients are monitored for up to 15 years. This includes a medical history and physical examination for 5 years following the last vaccination. Information beyond 5 years is collected once a year by telephone.
NCT00162123 ↗	A Companion Study for Patients Enrolled in Prior/Parent Ipilimumab Studies	Completed	Bristol-Myers Squibb	Phase 2	2006-05-01	The purpose of this study was to evaluate the continued use of ipilimumab in patients who had reinduction at the time of disease progression or to continue maintenance treatment. In addition, this study will continue to follow patients who have taken ipilimumab, but who are not eligible for maintenance or reinduction therapy.
NCT00170157 ↗	Hormone Therapy and Ipilimumab in Treating Patients With Advanced Prostate Cancer	Completed	Medarex	Phase 2	2004-06-01	RATIONALE: Androgens can cause the growth of prostate cancer cells. Antihormone therapy, such as leuprolide acetate, goserelin, flutamide, or bicalutamide may lessen the amount of androgens made by the body. Monoclonal antibodies, such as ipilimumab, can block cancer growth in different ways. Some block the ability of tumor cells to grow and spread. Others find tumor cells and help kill them or carry cancer-killing substances to them. Giving antihormone therapy together with ipilimumab may kill more tumor cells. PURPOSE: This randomized phase II trial is study how well giving hormone therapy and ipilimumab together works in treating patients with advanced prostate cancer.

Clinical Trial Conditions for ipilimumab

Condition Name

Condition Name for ipilimumab
Intervention	Trials
Melanoma	120
Metastatic Melanoma	50
Renal Cell Carcinoma	26
Non-small Cell Lung Cancer	24

Condition MeSH

Condition MeSH for ipilimumab
Intervention	Trials
Melanoma	237
Carcinoma	98
Lung Neoplasms	68
Carcinoma, Non-Small-Cell Lung	68

Clinical Trial Locations for ipilimumab

Trials by Country

Trials by Country for ipilimumab
Location	Trials
Japan	318
Australia	207
China	201
Argentina	81
Mexico	78

Trials by US State

Trials by US State for ipilimumab
Location	Trials
California	170
Texas	165
New York	148
Pennsylvania	128
Florida	120

Clinical Trial Progress for ipilimumab

Clinical Trial Phase

Clinical Trial Phase for ipilimumab
Clinical Trial Phase	Trials
PHASE4	3
PHASE3	7
PHASE2	30

Clinical Trial Status

Clinical Trial Status for ipilimumab
Clinical Trial Phase	Trials
Recruiting	264
Active, not recruiting	134
Completed	118

Clinical Trial Sponsors for ipilimumab

Sponsor Name

Sponsor Name for ipilimumab
Sponsor	Trials
Bristol-Myers Squibb	265
National Cancer Institute (NCI)	72
M.D. Anderson Cancer Center	43

Sponsor Type

Sponsor Type for ipilimumab
Sponsor	Trials
Other	703
Industry	533
NIH	76

Clinical Trials Update, Market Analysis, and Projection for Ipilimumab

Last updated: November 23, 2025

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Introduction

Ipilimumab, marketed as Yervoy, is an immune checkpoint inhibitor developed by Bristol-Myers Squibb (BMS). It revolutionized oncology by introducing immune modulation as a treatment mechanism, particularly targeting CTLA-4 to enhance the antitumor immune response. Since its approval for metastatic melanoma in 2011, ipilimumab has expanded into broader indications, profoundly impacting the oncology landscape. This comprehensive analysis examines the latest clinical trial developments, current market dynamics, and future growth potential of ipilimumab.

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Clinical Trials Update

Recent and Ongoing Clinical Trials

The trajectory of ipilimumab's clinical development reflects its evolving role across multiple cancer types:

1. Combination Therapies in Melanoma and Other Cancers

   • Numerous ongoing trials evaluate ipilimumab combined with nivolumab, an anti-PD-1 antibody, for metastatic melanoma, non-small cell lung cancer (NSCLC), and renal cell carcinoma (RCC). The CheckMate series (notably CheckMate 067) established combined therapy’s superior efficacy, which continues to inform subsequent trials [1].

2. Adjuvant and Neoadjuvant Settings

   • Trials exploring ipilimumab as an adjuvant therapy for high-risk melanoma and as part of neoadjuvant regimens aim to improve long-term survival and reduce recurrence. For instance, CheckMate 238 demonstrated the benefit of nivolumab over ipilimumab alone in adjuvant melanoma, influencing trial designs for combination regimens [2].

3. Novel Indications and Biomarker-Driven Studies

   • Trials are investigating ipilimumab in combination with targeted therapies in skin and gastrointestinal cancers. Biomarker-driven approaches aim to personalize treatment, identifying patients most likely to benefit.

4. Safety and Toxicity Research

   • Several studies focus on optimizing dosing strategies and managing immune-related adverse events (irAEs), which remain a critical consideration for combination regimens involving ipilimumab.

Key Clinical Trial Highlights

• CheckMate 901: A Phase III trial assessing nivolumab + ipilimumab vs. chemotherapy in first-line NSCLC, currently recruiting to establish efficacy benchmarks.
• Phase II Trials: Notably, in hepatocellular carcinoma (HCC) and microsatellite instability-high (MSI-H) colorectal cancer, exploring immune synergy.
• Adverse Event Management Trials: Focused on mitigating irAEs to expand tolerability, crucial for combination therapy adoption.

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Market Analysis

Market Size and Revenue Trends

The global oncology immunotherapy market has experienced exponential growth, projected to reach approximately USD 55 billion by 2027, driven largely by PD-1/PD-L1 inhibitors, but checkpoint inhibitors like ipilimumab hold a significant share [3].

• Revenue Data (2022): Bristol-Myers Squibb reported fiscal year sales of approximately USD 859 million for Yervoy, with a substantial contribution from combination therapies [4].
• Market Penetration: Ipilimumab remains a first-line and salvage therapy in melanoma, with expanding use in combination regimens in lung and renal cancers.

Competitive Landscape

While nivolumab and pembrolizumab dominate the checkpoint inhibitor space, ipilimumab’s niche remains as part of combination protocols. The competition for monotherapy indication has diminished due to superior ORR and PFS metrics demonstrated by PD-1 inhibitors alone [5].

• Key competitors:
  • Nivolumab (Opdivo): Leading in multiple indications.
  • Pembrolizumab (Keytruda): Broad approval landscape.
  • Emerging agents: Cemiplimab, tislelizumab.

Market Drivers and Challenges

• Drivers:

  • Expanding indications in melanoma, lung, renal, and gastrointestinal cancers.
  • Favorable reimbursement policies aligned with improved survival outcomes.
  • Growing preference for combination therapy to overcome resistance.

• Challenges:

  • Toxicity and irAEs limit broader application.
  • High therapy costs (~USD 125,000–150,000/year).
  • Need for predictive biomarkers to enhance patient selection.

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Future Market Projection

Growth Prospects

The outlook for ipilimumab is cautiously optimistic, driven by its role as part of combination immunotherapy regimens. The favorable safety profiles of newer agents, combined with ongoing clinical trials, underpin potential market expansion.

• Forecast (2023–2030):
  • Estimated CAGR of 10-12%, reflecting incremental growth driven by novel indications and combination strategies.
  • Prediction that the combination of ipilimumab + nivolumab will represent a substantial share of the high-end metastatic melanoma market, with growth extending into less-researched indications.

Potential Expansion Areas

• Adjuvant and Neoadjuvant Oncology: Trials suggesting improved disease-free survival could support broader label extensions.
• Combination with Novel Agents: Targeted therapies (e.g., BRAF inhibitors) and cancer vaccines may synergize with checkpoint inhibition.
• Biomarker-Driven Personalized Medicine: Enhancing response rates and minimizing irAEs through biomarker-guided therapy selection will be key.
• Global Access: Emerging markets' expanding healthcare infrastructure offers long-term growth opportunities.

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Regulatory and Market Access Dynamics

Regulatory agencies are increasingly supportive of combination regimens, provided efficacy and safety are demonstrated. The FDA has approved ipilimumab in combination with nivolumab for multiple indications, including melanoma and renal cell carcinoma, aligning with market trends favoring combination strategies [6].

Market access hinges on demonstrating long-term survival benefits and managing toxicity profiles, particularly as competition intensifies.

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Key Takeaways

• Deepening Clinical Evidence: Ongoing trials underscore ipilimumab’s central role in combinatorial regimens, primarily with nivolumab, six years post-initial approval.
• Market Potential: While monotherapy’s dominance wanes, ipilimumab remains vital within combination immunotherapy paradigms, with strong growth prospects driven by expanding indications and innovative trial designs.
• Competitive Dynamics: The increasing popularity of PD-1/PD-L1 inhibitors challenges ipilimumab’s standalone market share but sustains its importance in synergistic regimens.
• Regulatory Outlook: Evolving approvals in adjuvant and neoadjuvant settings will further solidify its relevance; continuous safety profile improvements will enhance market acceptance.
• Business Strategy: Manufacturers should focus on biomarker research, toxicity management, and diversification into emerging indications to maximize market share.

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FAQs

1. What are the primary clinical indications for ipilimumab today?
Ipilimumab is primarily indicated for unresectable or metastatic melanoma and in combination with nivolumab for advanced renal cell carcinoma and melanoma. Emerging indications include adjuvant melanoma and potential investigational use in other cancers like HCC and lung cancer.

2. How does ipilimumab's efficacy compare to PD-1 inhibitors?
While PD-1 inhibitors like nivolumab and pembrolizumab exhibit higher response rates and better tolerability as monotherapies, ipilimumab’s strength lies in specified combination regimens, which have demonstrated improved survival outcomes compared to monotherapy.

3. What are the main safety concerns associated with ipilimumab?
Immune-related adverse events, including colitis, hepatitis, endocrinopathies, and skin reactions, remain significant. Managing irAEs effectively is essential for maintaining patient safety and therapy adherence.

4. How is the market for ipilimumab expected to evolve over the next decade?
The market is projected to grow steadily, fueled by combination therapies, new indications, and improved management of irAEs. However, competition from PD-1/PD-L1 therapies will continue to shape market share.

5. What key factors will determine ipilimumab’s future success?
Effective patient selection via biomarkers, safety optimization, broader indications, cost management, and successful integration into combination regimens will be decisive.

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References

1. Hodi FS, et al. "Long-term survival update for combination immunotherapy in melanoma." N Engl J Med. 2018.
2. Weber JS, et al. "Adjuvant Nivolumab Versus Ipilimumab in Melanoma." J Clin Oncol. 2017.
3. MarketsandMarkets. "Oncology Drugs Market by Therapy Type." 2022.
4. Bristol-Myers Squibb. "Yervoy (ipilimumab) Annual Sales Report." 2022.
5. Sun S, et al. "Comparison of immune checkpoint inhibitors: efficacy and safety." Lancet Oncol. 2020.
6. U.S. Food and Drug Administration. "FDA Approvals for Nivolumab and Ipilimumab." 2022.

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In Summary: Ipilimumab remains a cornerstone immunotherapy agent, with ongoing trials promising to broaden its therapeutic reach. Its future hinges on optimizing combination strategies, managing toxicity, and expanding indications, ensuring its position in the evolving oncology market.