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	238
Carcinoma	98
Carcinoma, Renal Cell	70
Carcinoma, Non-Small-Cell Lung	69

Clinical Trial Locations for ipilimumab

Trials by Country

Trials by Country for ipilimumab
Location	Trials
Japan	320
Australia	212
China	201
Argentina	82
Mexico	80

Trials by US State

Trials by US State for ipilimumab
Location	Trials
California	171
Texas	165
New York	150
Pennsylvania	128
Florida	121

Clinical Trial Progress for ipilimumab

Clinical Trial Phase

Clinical Trial Phase for ipilimumab
Clinical Trial Phase	Trials
PHASE4	3
PHASE3	8
PHASE2	33

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	267
National Cancer Institute (NCI)	72
M.D. Anderson Cancer Center	43

Sponsor Type

Sponsor Type for ipilimumab
Sponsor	Trials
Other	711
Industry	538
NIH	76

Last updated: July 25, 2026

Ipilimumab (Yervoy) clinical trials update, market analysis, and 2030 projection

Executive summary: Ipilimumab is a foundational immune checkpoint inhibitor in metastatic and adjuvant oncology, driven by combination regimens (notably with nivolumab) and expanding adjuvant/curative-intent use. Clinical development remains active across melanoma and multiple solid tumors, with ongoing trials aimed at earlier-stage disease, neoadjuvant settings, and new partner combinations. Commercially, the product mix continues to pivot toward combination use and higher-acuity settings where checkpoint blockade is standard. Market growth is constrained by patent/grant timelines and competitive dynamics in checkpoint inhibitors, but demand durability is supported by entrenched guideline positioning and a large installed base.

Market snapshot (directional): Ipilimumab revenue is dominated by melanoma (advanced and adjuvant) and increasingly benefits from oncology center-of-excellence prescribing patterns for combination therapy. The next 12 to 36 months hinge on (1) maturation of adjuvant/neoadjuvant outcomes, (2) label expansions tied to positive efficacy readouts, and (3) competitive penetration by other CTLA-4 approaches and checkpoint combinations.

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What clinical trials are currently testing ipilimumab, and what are the key readouts?

Ipilimumab’s current trial landscape is structured around three themes: (a) earlier-stage treatment (adjuvant and neoadjuvant), (b) combination intensification with PD-1 inhibitors and other immunomodulators, and (c) tumor-agnostic biomarker exploration to extend beyond melanoma.

Melanoma: what are the active development directions?

1) Adjuvant and curative-intent melanoma

• Trials continue to test sequencing and duration of ipilimumab-containing regimens after complete resection.
• Readouts typically focus on event-free survival (EFS), recurrence-free survival (RFS), and overall survival (OS), with stratification by stage (IIIB/C vs IV) and biomarkers where available.

2) Neoadjuvant melanoma

• Neoadjuvant studies focus on pathologic response, immune activation biomarkers, and long-term relapse outcomes.
• Key endpoints include major pathologic response (MPR) and immune correlates that can support label expansions or regimen optimization.

3) Combination with nivolumab and other partners

• The dominant clinical question is whether combination therapy can reduce time-to-progression and improve OS in metastatic settings while preserving tolerability.

Beyond melanoma: what is ipilimumab testing for solid tumors?

Ipilimumab trials outside melanoma generally fall into:

• Combination checkpoint regimens with PD-1 inhibitors or other agents (IL-2, anti-CTLA-4 variants, or pathway-targeted therapies).
• Tumor-specific cohorts (lung, genitourinary, GI) where historical response rates have supported continued exploration.
• Biomarker selection trials that attempt to enrich for responders using immune signatures, tumor mutational burden proxies, or HLA/antigen presentation features.

How do trial endpoints typically drive ipilimumab expansion?

• Regulatory expansion in oncology is most sensitive to OS and EFS/RFS in curative-intent contexts, and to confirmed response rate and PFS in metastatic settings.
• Safety remains a gating factor: CTLA-4-related immune-related adverse events (irAEs) directly affect dose, schedule, and patient selection.

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When do ipilimumab clinical trial results translate into label changes and approvals?

Timing depends on phase and endpoint maturity:

• Phase 3 readouts for adjuvant/neoadjuvant settings generally require longer follow-up to demonstrate durable EFS/RFS and, in some cases, OS.
• In metastatic settings, regulatory decisions can occur sooner if PFS/OS separation appears early and safety profiles remain consistent.

Practical commercial timeline:

• Expect the largest label-driven commercial impact when trial results align with guideline updates and payer coverage.
• Combination regimens tend to convert more quickly into standard-of-care pathways once efficacy and tolerability benchmarks are met.

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What patents protect ipilimumab, and how strong is the patent estate for blocking generics?

Ipilimumab is biologic and is not exposed to the same “small-molecule” exclusivity framework as conventional pharmaceuticals. Patent coverage typically includes:

• Composition of matter for the antibody and variants.
• Formulation and stability-related patents.
• Methods of use for specific indications and combinations (for example, with nivolumab).
• Manufacturing and process patents.

Key patent estate characteristics for biologics

• Multiple layers of protection across molecule, manufacturing, and method-of-use.
• Jurisdictional fragmentation: US, EU, JP, and other geographies can differ in expiration, scope, and enforcement.

What risks exist for biosimilar competition?

• Biosimilar entry depends on regulatory pathway eligibility, data requirements, and market approvals rather than Paragraph IV.
• Patent defense typically targets infringement by linking biosimilar product attributes and/or labeling claims to protected method-of-use or formulation claims.

(Patent numbers, expiration dates, and case-specific enforcement cannot be listed here without a complete, jurisdiction-specific source set. This section summarizes the estate structure only.)

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What is the Orange Book status of ipilimumab, and does it affect generic entry risk?

Ipilimumab is a biologic and therefore is not listed in the FDA Orange Book in the same manner as small-molecule products. Generic “entry” risk is instead governed by biosimilar pathways and biologics licensing. Orange Book status is not the primary determinant for clinical or regulatory competition timing for biologics.

(A product-specific Orange Book citation requires the exact NDC listing and is not included here.)

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What biosimilar or interchangeability risks exist for ipilimumab?

Biosimilar risk is schedule- and label-driven:

• Launch viability is influenced by the breadth of patent protection and the labeling freedom for biosimilars versus reference products.
• Even after biosimilar approval, adoption can remain slow if oncologists prefer the reference product for dosing familiarity, supportive data, and management of irAEs.

Adoption drivers

• Real-world safety comparability.
• Availability, contracting, and volume-based purchasing.
• Institutional protocols for CTLA-4/PD-1 combination regimens.

(Biosimilar status and approved products depend on FDA BLA and biosimilar designations; this section provides structural risk only.)

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What market factors drive ipilimumab demand, and how durable is the revenue base?

Demand drivers

• Ipilimumab’s role in melanoma across metastatic and adjuvant settings keeps a baseline demand anchored by standard-of-care pathways.
• Combination therapy with PD-1 inhibitors increases patient eligibility relative to monotherapy.
• Specialty practice concentration sustains volume even when new CTLA-4 competitors emerge.

Constraints

• CTLA-4 tolerability limits broad uptake in low-risk populations.
• Other checkpoint combinations and alternative immunotherapies compete for share of eligible patients.
• Payer management can affect patient mix and dosing patterns.

Commercial mix shift

• A continued shift from monotherapy toward regimens with PD-1 agents typically improves response rates but changes tolerability management and may affect prescribing behavior.

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How does ipilimumab compare with competing CTLA-4 and PD-1 regimens in melanoma?

Clinical positioning comparison

• Ipilimumab competes within the checkpoint blockade ecosystem where CTLA-4 plus PD-1 combinations set efficacy benchmarks.
• The competitive comparison is usually less about mechanism and more about:
  • durability (EFS/OS),
  • toxicity profile,
  • feasibility of combination schedules,
  • and payer/institution adoption.

Competitive set

• PD-1-based regimens and other combination immunotherapies that may be preferred due to tolerability or convenience.
• Any alternate CTLA-4 strategy competing for adjuvant or combination slots.

(A fully itemized competitor-by-competitor efficacy and trial comparison requires a specific set of registrational studies and current label versions.)

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What generic entry risks exist for ipilimumab?

There is no “generic ipilimumab” path analogous to small-molecule generics.

• The entry risk is biosimilar-driven.
• Timing depends on biosimilar development, regulatory clearance, and patent litigation outcomes in key jurisdictions.

Commercial implications of biosimilar entry

• Downward pricing and increased contracting leverage typically occur after at least one biosimilar successfully enters and establishes pharmacy/infusion center adoption.
• Adoption lags can persist in high-complexity oncology settings, slowing price erosion.

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What formulations and dosing regimens are protected, and do they affect competitive launches?

Protection can cover:

• Drug product concentration and stability.
• Excipients and buffer systems affecting shelf life.
• Storage conditions and handling.
• Manufacturing attributes that affect comparability.

Launch impact

• If competitive entrants must maintain strict similarity to the reference product, any formulation or process differences can drive:
  • additional regulatory scrutiny,
  • or stronger patent defenses.

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What patent litigation affects ipilimumab commercialization?

For biologics, litigation typically centers on:

• patent scope for composition/manufacturing,
• and method-of-use infringement tied to labeling claims.

Business impact areas

• Settlement can delay biosimilar launch.
• Adjudication outcomes can shape labeling design (indication carve-outs) and determine substitution eligibility.

(Case listings require docket-specific sources and are not provided here.)

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How do licensing and settlement agreements shape ipilimumab availability?

Licensing and settlements in biologics are often used to:

• resolve patent disputes,
• set launch dates,
• define permissible labeling,
• and control distribution channels.

Commercial outcomes

• Settlements generally trade litigation uncertainty for scheduled market entry.
• The magnitude of price erosion depends on whether the entrant gains broad label coverage and formulary access.

(No settlement-specific terms are included here.)

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What is the FDA regulatory pathway for ipilimumab, and what approvals are most commercially material?

Ipilimumab’s regulatory history is grounded in standard oncology pathways:

• supplemental approvals driven by new indications or combinations,
• label expansions based on Phase 3 evidence,
• and oncology safety updates related to irAE management.

Commercially material approvals

• Adjuvant and neoadjuvant expansions.
• Combination regimens that become guideline standards.
• Any tumor expansion that changes the eligible population size.

(Specific FDA letter dates and approval codes are not listed due to missing citation-grade inputs.)

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Ipilimumab 2030 market projection: scenario framework and baseline assumptions

Projection structure used for high-integrity budgeting:

1. Indication-driven TAM: patient count and eligible proportion within melanoma and expanded use cases.
2. Share-of-regimen: monotherapy vs combination with PD-1.
3. Adoption curve: infusion center and community oncology uptake.
4. Price dynamics: reference pricing, contracting, and biosimilar discounting if/when applicable.
5. Competitive intensity: rival checkpoint combinations, tolerance-driven switching, and sequencing patterns.

Baseline view

• Up to 2026-2027: incremental growth supported by maturation of adjuvant and combination usage and any label expansions.
• 2027-2030: growth stability or modest decline if competitive alternatives compress combination share or if biosimilar pressure increases pricing.

Downside and upside cases

• Downside: tighter payer restrictions, slower adjuvant adoption due to tolerability or competing standards, and earlier biosimilar discounting.
• Upside: successful new trial readouts expand earlier-stage use or enable lower-toxicity dosing strategies that broaden eligibility, improving persistence.

(Numerical revenue forecasts require a source basis for current revenue, biosimilar timeline, and patent expiration schedule. Those data are not included.)

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Key Takeaways

• Ipilimumab development is anchored in melanoma, with ongoing trial readouts centered on earlier-stage care and PD-1-based combinations.
• Commercial durability is supported by guideline placement and combination regimen use, with demand tied to adjuvant/neoadjuvant momentum.
• Competitive pressure is mainly from other checkpoint combinations rather than true “generic” entry; biosimilar risk depends on regulatory and patent outcomes.
• The 2030 outlook is best modeled through indication-driven TAM plus pricing and adoption curves rather than mechanism-only assumptions.

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FAQs

1) What indications are driving ipilimumab use most strongly today?
Melanoma across metastatic and adjuvant settings, with combination regimens increasing patient eligibility.

2) Does ipilimumab monotherapy have an ongoing role versus PD-1 combinations?
Yes, but combination therapy generally drives a larger share of clinically favored regimens where efficacy targets justify tolerability.

3) Which adverse events most influence ipilimumab dosing and patient selection?
Immune-related adverse events, particularly colitis, hepatitis, dermatitis, endocrinopathies, and pneumonitis.

4) How does combination therapy change ipilimumab clinical adoption?
It increases response depth but increases the need for standardized irAE monitoring and management pathways.

5) What timing matters most for ipilimumab commercial projections?
Readout and label-expansion timing for adjuvant/neoadjuvant studies, plus any biosimilar launch scheduling driven by patent outcomes.

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References (APA)

1. FDA. (n.d.). Drug Trials Snapshots for ipilimumab. U.S. Food and Drug Administration.
2. FDA. (n.d.). Drugs@FDA: Ipilimumab. U.S. Food and Drug Administration.
3. ClinicalTrials.gov. (n.d.). Ipilimumab studies. U.S. National Library of Medicine.