CLINICAL TRIALS PROFILE FOR CARFILZOMIB

505(b)(2) Clinical Trials for CARFILZOMIB

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	NCT02188368 ↗	Pomalidomide for Lenalidomide for Relapsed or Refractory Multiple Myeloma Patients	Active, not recruiting	Celgene Corporation	Phase 2	2014-08-01	The purpose of this clinical research study is to evaluate the safety and effectiveness (good and bad effects) of pomalidomide given as part of a combination therapy that include more than just steroids to treat subjects with relapsed (subjects whose disease came back) or refractory (subjects whose disease did not respond to past treatment) multiple myeloma (MM). Pomalidomide (alone or in combination with dexamethasone) has been approved by the United States Food and Drug Administration (FDA) for the treatment of MM patients who have received at least two prior therapies, including lenalidomide and bortezomib, and have demonstrated disease progression on or within 60 days of completion of their last therapy. However, the use of pomalidomide in combination with other drugs used to treat MM, such as chemotherapeutic agents and proteasome inhibitors, is currently being tested and is not approved. Pomalidomide is in the same drug class as thalidomide and lenalidomide. Like lenalidomide, pomalidomide is a drug that alters the immune system and it may also interfere with the development of small blood vessels that help support tumor growth. Therefore, in theory, it may reduce or prevent the growth of cancer cells. The testing done with pomalidomide thus far has shown that it is well-tolerated and effective for subjects with MM both on its own and in combination with dexamethasone. Using another drug class, namely proteasome inhibitors, we have demonstrated that simply replacing a proteasome inhibitor with another in an established anti-myeloma treatment regimen can frequently overcome resistance regardless of the other agents that are part of the anti-myeloma regimen. Importantly, the toxicity profile of the new combinations closely resembled that of the proteasome inhibitor administered as a single agent. Based on this experience, we hypothesize that the replacement of lenalidomide with pomalidomide will yield similar results in a similar relapsed/refractory MM patient population.
New Combination	NCT02188368 ↗	Pomalidomide for Lenalidomide for Relapsed or Refractory Multiple Myeloma Patients	Active, not recruiting	Oncotherapeutics	Phase 2	2014-08-01	The purpose of this clinical research study is to evaluate the safety and effectiveness (good and bad effects) of pomalidomide given as part of a combination therapy that include more than just steroids to treat subjects with relapsed (subjects whose disease came back) or refractory (subjects whose disease did not respond to past treatment) multiple myeloma (MM). Pomalidomide (alone or in combination with dexamethasone) has been approved by the United States Food and Drug Administration (FDA) for the treatment of MM patients who have received at least two prior therapies, including lenalidomide and bortezomib, and have demonstrated disease progression on or within 60 days of completion of their last therapy. However, the use of pomalidomide in combination with other drugs used to treat MM, such as chemotherapeutic agents and proteasome inhibitors, is currently being tested and is not approved. Pomalidomide is in the same drug class as thalidomide and lenalidomide. Like lenalidomide, pomalidomide is a drug that alters the immune system and it may also interfere with the development of small blood vessels that help support tumor growth. Therefore, in theory, it may reduce or prevent the growth of cancer cells. The testing done with pomalidomide thus far has shown that it is well-tolerated and effective for subjects with MM both on its own and in combination with dexamethasone. Using another drug class, namely proteasome inhibitors, we have demonstrated that simply replacing a proteasome inhibitor with another in an established anti-myeloma treatment regimen can frequently overcome resistance regardless of the other agents that are part of the anti-myeloma regimen. Importantly, the toxicity profile of the new combinations closely resembled that of the proteasome inhibitor administered as a single agent. Based on this experience, we hypothesize that the replacement of lenalidomide with pomalidomide will yield similar results in a similar relapsed/refractory MM patient population.

All Clinical Trials for CARFILZOMIB

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00150462 ↗	Safety Study of the Proteasome Inhibitor PR-171 (Carfilzomib for Injection) in Patients With Hematological Malignancies	Completed	Amgen	Phase 1	2005-09-01	The purpose of this study is to test the safety and tolerability of carfilzomib at different dose levels on hematological cancers such as multiple myeloma, non-Hodgkin's lymphoma, Hodgkin's disease, or Waldenstrom's macroglobulinemia. Carfilzomib is a proteasome inhibitor, an enzyme responsible for degrading a wide variety of cellular proteins.
NCT00150462 ↗	Safety Study of the Proteasome Inhibitor PR-171 (Carfilzomib for Injection) in Patients With Hematological Malignancies	Completed	Onyx Pharmaceuticals	Phase 1	2005-09-01	The purpose of this study is to test the safety and tolerability of carfilzomib at different dose levels on hematological cancers such as multiple myeloma, non-Hodgkin's lymphoma, Hodgkin's disease, or Waldenstrom's macroglobulinemia. Carfilzomib is a proteasome inhibitor, an enzyme responsible for degrading a wide variety of cellular proteins.
NCT00461045 ↗	Phase 2 Clinical Trial of NPI-0052 in Patients With Relapsed or Relapsed/Refractory Multiple Myeloma	Completed	Celgene	Phase 2	2007-03-01	This is a Phase 2, open-label, multicenter study examining the safety, pharmacokinetics and pharmacodynamics, and best overall response to escalating doses of the proteasome inhibitor NPI-0052 (also known as marizomib) in patients with relapsed or relapsed/refractory multiple myeloma. NPI-0052 is a novel, second generation proteasome inhibitor that prevents the breakdown of proteins involved in signal transduction which blocks growth and survival in cancer cells. The study is a Phase 2 study and is a 2-stage efficacy design in a selected subgroup of patients (Arm C) treated with the recommended phase 2 dose of NPI-0052, as determined in a previously completed Phase 1 study. The study is to evaluate the safety and any preliminary evidence of efficacy of NPI-0052 in multiple myeloma patients who have previously received carfilzomib (PR-171, Kyprolis™) and subsequently had disease progression.
NCT00461045 ↗	Phase 2 Clinical Trial of NPI-0052 in Patients With Relapsed or Relapsed/Refractory Multiple Myeloma	Completed	Triphase Research and Development I Corporation	Phase 2	2007-03-01	This is a Phase 2, open-label, multicenter study examining the safety, pharmacokinetics and pharmacodynamics, and best overall response to escalating doses of the proteasome inhibitor NPI-0052 (also known as marizomib) in patients with relapsed or relapsed/refractory multiple myeloma. NPI-0052 is a novel, second generation proteasome inhibitor that prevents the breakdown of proteins involved in signal transduction which blocks growth and survival in cancer cells. The study is a Phase 2 study and is a 2-stage efficacy design in a selected subgroup of patients (Arm C) treated with the recommended phase 2 dose of NPI-0052, as determined in a previously completed Phase 1 study. The study is to evaluate the safety and any preliminary evidence of efficacy of NPI-0052 in multiple myeloma patients who have previously received carfilzomib (PR-171, Kyprolis™) and subsequently had disease progression.
NCT00511238 ↗	Phase 2 Study of Carfilzomib in Relapsed and Refractory Multiple Myeloma	Completed	Amgen	Phase 2	2007-08-01	To evaluate the overall response rate and safety and tolerability of carfilzomib in subjects with relapsed and refractory multiple myeloma. Patients must have received prior treatment with bortezomib and either thalidomide or lenalidomide and be refractory to their last treatment.
NCT00511238 ↗	Phase 2 Study of Carfilzomib in Relapsed and Refractory Multiple Myeloma	Completed	Onyx Therapeutics, Inc.	Phase 2	2007-08-01	To evaluate the overall response rate and safety and tolerability of carfilzomib in subjects with relapsed and refractory multiple myeloma. Patients must have received prior treatment with bortezomib and either thalidomide or lenalidomide and be refractory to their last treatment.

Clinical Trial Conditions for CARFILZOMIB

Condition Name

Condition Name for CARFILZOMIB
Intervention	Trials
Multiple Myeloma	128
Recurrent Plasma Cell Myeloma	14
Refractory Plasma Cell Myeloma	13
Refractory Multiple Myeloma	12

Condition MeSH

Condition MeSH for CARFILZOMIB
Intervention	Trials
Multiple Myeloma	199
Neoplasms, Plasma Cell	180
Lymphoma	22
Neoplasms	8

Clinical Trial Locations for CARFILZOMIB

Trials by Country

Trials by Country for CARFILZOMIB
Location	Trials
United States	964
Japan	106
Canada	92
Spain	71
Germany	61

Trials by US State

Trials by US State for CARFILZOMIB
Location	Trials
New York	69
Texas	58
California	56
New Jersey	45
Florida	45

Clinical Trial Progress for CARFILZOMIB

Clinical Trial Phase

Clinical Trial Phase for CARFILZOMIB
Clinical Trial Phase	Trials
PHASE4	1
PHASE3	5
PHASE2	6

Clinical Trial Status

Clinical Trial Status for CARFILZOMIB
Clinical Trial Phase	Trials
Recruiting	77
Completed	63
Active, not recruiting	45

Clinical Trial Sponsors for CARFILZOMIB

Sponsor Name

Sponsor Name for CARFILZOMIB
Sponsor	Trials
Amgen	81
Onyx Therapeutics, Inc.	35
National Cancer Institute (NCI)	32

Sponsor Type

Sponsor Type for CARFILZOMIB
Sponsor	Trials
Industry	272
Other	255
NIH	33

Carfilzomib (Kyprolis): Clinical Trials Update, Market Analysis, and Revenue Projection

Last updated: April 27, 2026

What is carfilzomib’s clinical position right now?

Carfilzomib is an IV proteasome inhibitor used in multiple myeloma (MM), with established use across lines of therapy and continued development focused on earlier lines, combination regimens, and maintenance strategies.

Core regulatory and clinical anchor

• Indications (US, baseline)
  • Relapsed or refractory MM (RRMM), including in combination regimens and as monotherapy in specific post-therapy settings (US label context: Kyprolis).
• Clinical modality
  • Carfilzomib is a next-generation proteasome inhibitor delivered intravenously, typically in multi-day cycles depending on regimen.

Where trials concentrate

Trial activity clusters in four practical zones:

1. Earlier-line RRMM and transplant-ineligible settings using standard-of-care backbones.
2. Combination therapies to improve depth of response and durability (common partners include IMiDs and anti-CD38 antibodies).
3. Maintenance and extended treatment strategies aimed at prolonging progression-free survival (PFS).
4. Safety and dosing optimization to reduce cardiovascular, renal, and hematologic adverse events (AEs) while maintaining efficacy.

Expected near-term clinical readouts (what tends to move the market)

For carfilzomib, the market impact typically comes from:

• PFS and overall survival (OS) confirmation in phase 3 or late-phase phase 2 programs.
• Subset efficacy (high-risk cytogenetics, renal impairment, frailty/elderly).
• Safety deltas tied to dosing schedule refinement and combination adjustments.

Clinical development is ongoing, but the commercial impact hinges on whether late-phase programs show statistically significant improvements in PFS/OS versus current standards and whether toxicity is manageable in real-world profiles.

How big is the carfilzomib market today?

Carfilzomib is a high-value branded oncology drug with commercial sales tied to:

• RRMM incidence and treatment intensity
• Uptake into earlier lines
• Competition from other proteasome inhibitors (e.g., bortezomib, ixazomib where relevant), anti-CD38 therapies, IMiDs, and newer agents (including cellular and bispecific approaches in later years)

Commercial drivers

• Depth of response: stronger cytoreduction is commercially attractive in RRMM.
• Line-of-therapy expansion: performance in earlier lines increases eligible patient pools.
• Combination positioning: carfilzomib’s use with IMiDs and anti-CD38 antibodies supports durable regimen usage.

Commercial headwinds

• Shift to regimens with oral convenience where efficacy and tolerability compete favorably.
• BiTE/bispecific proliferation in RRMM and emerging use patterns.
• Pricing pressure from portfolio rationalization and payer controls.

Market definition for projection

A practical projection scope is:

• US + major EU markets plus a residual ROW share
• Lines: RRMM and earlier-line settings where carfilzomib holds labeled or evidence-supported positioning
• Use in combination regimens and monotherapy post-exposure patterns

How will competition reshape share?

Carfilzomib’s share trajectory depends on how quickly alternative mechanisms and route-of-administration options absorb patients in:

• early RRMM (rapid switchers to other combinations)
• post-anti-CD38 exposure sequencing
• elderly/frail cohorts where dosing schedule tolerability affects payer and provider adoption

Key competitive dynamics

• Proteasome inhibitor alternatives
  • Bortezomib remains entrenched in many regimens and earlier use patterns.
  • Ixazomib offers oral access in selected strategies.
• Anti-CD38 backbone saturation
  • Anti-CD38 class penetration supports continued proteasome inhibitor combinations, but sequence decisions influence carfilzomib uptake.
• Newer RRMM mechanisms
  • Bispecifics and other later-line approaches can reduce the time patients spend in proteasome-based regimens.

What is the revenue projection for carfilzomib?

Because revenue forecasting requires consistent, time-specific sales baselines and currency assumptions, a clean projection model must start from a fixed “last reported” global sales number and then apply a scenario framework for:

• volume (patient starts and persistence)
• net price (discounting, rebates, payer behavior)
• mix (line of therapy and regimen composition)

In the absence of a single auditable last-reported sales baseline in the provided materials, a numeric projection cannot be produced with the precision your use case requires.

What outcomes in trials would most affect valuation?

If a late-phase program reads out positively, the specific endpoints that typically translate into commercial confidence are:

• PFS hazard ratio vs comparator backbone
• OS trend with statistical significance where available
• Overall response rate (ORR) and rate of deep response
• Cardiovascular and renal safety under real-world compatible dosing

A negative readout that:

• increases grade 3/4 AEs without durability gains, or
• fails to demonstrate superiority in clinically meaningful subgroups, usually reduces expected line-expansion and dampens valuation.

Which pipeline themes are most likely to extend the lifecycle?

Carfilzomib’s lifecycle extension tends to be driven by:

• New combinations that improve PFS without new tolerability burdens
• Maintenance or treatment duration strategies that reduce total exposure while preserving efficacy
• Dosing schedule optimization that improves adherence and reduces discontinuations
• Subgroup efficacy evidence that expands the addressable patient pool

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Key Takeaways

• Carfilzomib remains positioned in RRMM and continues development focused on combinations, earlier-line use, and durability.
• Market outcomes hinge on late-phase readouts tied to PFS/OS and manageable cardiovascular and renal safety.
• Competitive pressure from oral options and later-line bispecific approaches can reduce time on proteasome-based regimens.
• A numeric revenue projection cannot be produced without an auditable, time-specific sales baseline.

FAQs

1. What is carfilzomib’s main clinical role in multiple myeloma?
   It is a proteasome inhibitor used in relapsed or refractory multiple myeloma in combination regimens and as monotherapy in specific post-treatment settings.

2. Which trial endpoints most influence carfilzomib adoption?
   PFS, OS trend, ORR/deep response rates, and the incidence of grade 3/4 cardiovascular and renal AEs.

3. What are the biggest commercial headwinds?
   Regimen shifts toward oral convenience options and increasing use of bispecifics in later lines can shorten time patients spend on carfilzomib-based pathways.

4. What trial results would likely expand the addressable market?
   Demonstrated superiority or strong non-inferiority in earlier lines with durable response and acceptable safety.

5. How does dosing affect market outcomes?
   Dosing schedule and toxicity profile influence discontinuation rates, clinician adoption, and payer willingness to cover more lines of therapy.

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References

[1] FDA. Kyprolis (carfilzomib) Prescribing Information. U.S. Food and Drug Administration.
[2] EMA. Kyprolis (carfilzomib) European public assessment reports. European Medicines Agency.
[3] ClinicalTrials.gov. Carfilzomib interventional studies (results and ongoing trials). U.S. National Library of Medicine.
[4] NCCN. NCCN Clinical Practice Guidelines in Oncology: Multiple Myeloma. National Comprehensive Cancer Network.