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.

Clinical Trial Conditions for Ipilimumab

Condition Name

Condition Name for Ipilimumab
Intervention	Trials
Melanoma	120
Metastatic Melanoma	50
Renal Cell Carcinoma	26

Condition MeSH

Condition MeSH for Ipilimumab
Intervention	Trials
Melanoma	238
Carcinoma	98
Carcinoma, Renal Cell	70

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

Ipilimumab (anti-CTLA-4): Clinical Trials Update, Market Analysis, and Projection (Global)

Last updated: April 25, 2026

What is the current clinical-trials footprint for ipilimumab?

Ipilimumab is an approved anti-CTLA-4 monoclonal antibody used across advanced melanoma and other oncology settings, typically in combination regimens. Clinical development remains active in both front-line and post-progression strategies, including combinations with PD-1/PD-L1 inhibitors and other agents.

How the modern program is structured (trial types and endpoints)

• Combination expansion: Trials emphasize ipilimumab plus PD-1/PD-L1 antibodies across disease settings, with primary endpoints typically OS and PFS, plus safety and response durability.
• Earlier-line positioning: Ongoing studies assess whether adding CTLA-4 blockade to PD-1-based regimens improves long-term survival when used earlier.
• Biomarker-driven stratification: Studies incorporate correlative biomarker efforts tied to response and immune-related adverse event (irAE) risk.
• Safety management protocols: More recent programs emphasize irAE mitigation strategies, including steroid tapering algorithms and immune monitoring.

Active clinical-trials signal (high level) Public registries show continued enrollment activity across:

• Melanoma (advanced): ipilimumab combinations remain a key platform in both treatment-naïve and previously treated populations.
• Other solid tumors: combinations are tested in settings beyond melanoma, consistent with broader checkpoint pairing strategies.

Which ipilimumab clinical outcomes currently shape the market narrative?

Market perception for ipilimumab is anchored by durability and survival in advanced melanoma, particularly in multi-agent checkpoint combinations where it contributes to depth of response and longer tail outcomes.

Key clinical outcome themes used by developers and payers:

• Durable OS benefit in subsets of melanoma patients treated with ipilimumab-based combinations.
• Trade-off profile: improved efficacy versus higher incidence of irAEs compared with PD-1 monotherapy, driving more stringent patient selection and monitoring.
• Combination regimen adoption: where ipilimumab is used, it is often as part of a fixed combination backbone or as a standard add-on to PD-1 therapy in specific disease stages and risk groups.

Supporting trial-era evidence basis Ipilimumab’s clinical position in melanoma is rooted in long-term OS data and the immunologic rationale validated across combination regimens; these data are reflected in labeling and professional guidance. (See sources [1]–[3].)

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How big is the ipilimumab market today?

Ipilimumab market size is best analyzed through a combination of (1) observed prescription dynamics and (2) mature checkpoint competition intensity, then translated into a forward revenue model based on share, penetration, and price erosion.

Market demand drivers

• Advanced melanoma and adjacent indications sustain the base addressable population.
• Treatment strategy shift toward PD-1-based regimens constrains standalone CTLA-4 monotherapy volume, but combinations still carry meaningful share.
• Payer preference for evidence-backed combinations supports consistent uptake in regimens with strong survival signals and acceptable safety management.

Market constraints

• Higher irAE burden can limit use in frail populations and regions with strict infusion-center and monitoring capacity.
• Checkpoint category competition is intense in melanoma, with PD-1/PD-L1 combinations and newer CTLA-4 strategies competing for same-line and next-line slots.

Reference point: product maturity

Ipilimumab is a long-established oncology asset with:

• entrenched clinician awareness,
• mature manufacturing and supply,
• generic and biosimilar pressure depending on jurisdiction and exclusivity status (variable by market and product form).

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What is the competitive landscape for ipilimumab?

Ipilimumab competes primarily within the checkpoint class, competing for:

• front-line and second-line places with PD-1/PD-L1 combinations,
• durability-seeking strategies in patients who respond to combination checkpoint regimens.

Primary competitor set

• PD-1 inhibitors (and PD-L1 inhibitors): pembrolizumab, nivolumab and related agents anchor the dominant regimen patterns.
• Other CTLA-4 approaches: activity exists for next-gen CTLA-4 strategies and alternate immune combinations, though ipilimumab remains the clinical standard reference.

Implication for share Even where ipilimumab continues to be used, growth is often driven by combination penetration rather than monotherapy expansion, so revenue trajectories track combination uptake and tolerability/management protocols rather than broad therapy switching.

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What market projection should be used for ipilimumab?

A forward projection should be modeled along three layers: (1) indication population evolution, (2) regimen mix and share, (3) net price erosion.

Projection framework (base case logic)

• Population growth: limited by incidence growth and staging stability in advanced melanoma; growth is more dependent on screening and earlier diagnosis than on dramatic expansion of advanced cohorts.
• Regimen mix: PD-1/PD-L1 dominates; ipilimumab growth is tied to combination persistence and any incremental evidence supporting CTLA-4 add-on earlier in the pathway.
• Price and access: continued discounts, tender pressures, and competitive contracting compress net revenue. Biosimilar/generic dynamics apply in certain markets depending on exclusivity and local availability.

Directional 3-year outlook (typical pattern for mature checkpoint assets)

• Near-term: modest revenue stability or low growth in combination-led usage.
• Mid-term: gradual decline risk if PD-1-dominant regimens further displace CTLA-4 add-ons in specific lines.
• Upside scenario: stabilization if new trial results extend survival tail and strengthen combination adoption or if new subgroups expand eligible use.

This projection logic is consistent with checkpoint class market behavior captured in oncology market monitoring and with ipilimumab’s established role in immuno-oncology pathways. (See sources [4]–[6].)

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How do clinical-trial updates translate into commercial upside or downside?

Upside levers

• Improved survival signal that supports guideline reinforcement or broader label usage.
• Better safety management that expands eligibility (fewer discontinuations and manageable irAEs).
• Earlier-line adoption based on new evidence showing durable benefit in treatment-naïve populations.

Downside levers

• Negative or neutral efficacy in head-to-head or add-on studies.
• Increased irAE-related discontinuation reducing real-world adherence versus trial expectations.
• Regimen displacement by stronger PD-1-centric combinations.

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What is the investment-relevant risk profile for ipilimumab?

From a patent and competitive standpoint, ipilimumab’s commercial risk profile is driven by:

• erosion of net prices via competition and contracting,
• indication-by-indication share shifts as PD-1 and later-generation combinations standardize care,
• biosimilar and local generic dynamics that reduce realized prices.

From a clinical development standpoint:

• trial economics slow for mature assets unless there is a label expansion with clear differentiation,
• any new program must deliver OS-relevant endpoints or durable response improvements to materially change adoption patterns.

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Regulatory and evidence backbone

Ipilimumab’s clinical and regulatory position is reflected in:

• US FDA labeling and associated documentation that specifies approved uses and safety considerations. (See sources [1]–[2].)
• NCCN-aligned clinical practice approach for melanoma immunotherapy sequencing that continues to incorporate CTLA-4 in select combination regimens. (See source [3].)

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Key Takeaways

• Clinical trials remain active with ipilimumab focused on combination strategies, earlier-line positioning, and safety/biomarker-enriched approaches.
• Market growth is combination-led, not monotherapy-led: ipilimumab’s value proposition remains tied to durable outcomes paired with PD-1/PD-L1 backbone regimens.
• Forward revenues likely track mature checkpoint dynamics: modest stability or low growth near term with gradual compression risk mid-term from competitive contracting and PD-1 displacement, offset only if new evidence strengthens CTLA-4 add-on adoption.
• Commercial risk is primarily price and mix, with clinical risk concentrated in irAE management and subgroup efficacy signals that determine real-world eligibility and persistence.

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FAQs

1) What is ipilimumab’s core mechanism and how does it drive combination use?

Ipilimumab is an anti-CTLA-4 monoclonal antibody that modulates T-cell activation to complement PD-1/PD-L1 inhibition, which supports combination regimen adoption in advanced melanoma pathways.

2) Is ipilimumab growth mainly coming from new indications?

Not primarily. The dominant commercial driver is regimen mix in established oncology pathways, especially where CTLA-4 is added to PD-1-based regimens for durable outcomes.

3) What limits ipilimumab market penetration in routine practice?

The limiting factor is the immune-related adverse event profile, which increases monitoring and requires careful patient selection to maintain treatment continuity.

4) How should a market model incorporate ipilimumab price erosion?

Use net price contraction assumptions reflecting mature checkpoint contracting, tenders, and biosimilar/generic dynamics where applicable, then layer a regimen-mix effect that reflects PD-1 dominance and CTLA-4 add-on persistence.

5) What clinical endpoints most influence commercial adoption for ipilimumab combinations?

Overall survival (OS) and progression-free survival (PFS), plus response durability and safety/tolerability that translate into persistence and reduced discontinuations.

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References

[1] U.S. Food and Drug Administration. (2024). Yervoy (ipilimumab) prescribing information. FDA.
[2] U.S. Food and Drug Administration. (n.d.). Yervoy label and review documents. FDA.
[3] National Comprehensive Cancer Network. (n.d.). Melanoma: NCCN Clinical Practice Guidelines in Oncology. NCCN.
[4] IQVIA. (n.d.). Oncology market reporting and checkpoint drug trends. IQVIA.
[5] EvaluatePharma. (n.d.). World Preview and oncology market projections. EvaluatePharma.
[6] Fierce Pharma / IQVIA / EvaluatePharma market coverage. (n.d.). Checkpoint competition and pricing trend analyses.