CLINICAL TRIALS PROFILE FOR KYPROLIS

505(b)(2) Clinical Trials for kyprolis

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 kyprolis

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.
NCT00531284 ↗	Phase 1b/2 Study of Carfilzomib in Relapsed Solid Tumors, Multiple Myeloma, or Lymphoma	Completed	Amgen	Phase 1/Phase 2	2007-09-01	The primary objectives of this Phase 1b/2 study were as follows: - Phase 1b (Bolus and Infusion): To evaluate the safety and tolerability of carfilzomib in patients with relapsed solid tumors and in patients with relapsed and/or refractory multiple myeloma and in patients with refractory lymphoma. - Phase 2 (Bolus): To evaluate the overall response rate (ORR) after 4 cycles of carfilzomib in patients with relapsed solid tumors.

Clinical Trial Conditions for kyprolis

Condition Name

Condition Name for kyprolis
Intervention	Trials
Multiple Myeloma	42
Recurrent Plasma Cell Myeloma	14
Refractory Plasma Cell Myeloma	13
Plasma Cell Myeloma	5

Condition MeSH

Condition MeSH for kyprolis
Intervention	Trials
Multiple Myeloma	74
Neoplasms, Plasma Cell	73
Lymphoma	12
Lymphoma, Non-Hodgkin	7

Clinical Trial Locations for kyprolis

Trials by Country

Trials by Country for kyprolis
Location	Trials
United States	340
China	29
India	16
United Kingdom	15
Spain	9

Trials by US State

Trials by US State for kyprolis
Location	Trials
Illinois	22
California	21
New York	18
Texas	17
Ohio	15

Clinical Trial Progress for kyprolis

Clinical Trial Phase

Clinical Trial Phase for kyprolis
Clinical Trial Phase	Trials
Phase 4	1
Phase 3	7
Phase 2	43

Clinical Trial Status

Clinical Trial Status for kyprolis
Clinical Trial Phase	Trials
Recruiting	31
Completed	25
Active, not recruiting	15

Clinical Trial Sponsors for kyprolis

Sponsor Name

Sponsor Name for kyprolis
Sponsor	Trials
Amgen	31
National Cancer Institute (NCI)	26
Onyx Therapeutics, Inc.	11

Sponsor Type

Sponsor Type for kyprolis
Sponsor	Trials
Industry	102
Other	95
NIH	26

Clinical Trials Update, Market Analysis, and Projection for KYPROLIS (Carfilzomib)

Last updated: October 27, 2025

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Introduction

KYPROLIS (carfilzomib), developed by Amgen, is an innovative proteasome inhibitor indicated primarily for multiple myeloma, particularly in relapsed or refractory cases. Since its FDA approval in 2012, KYPROLIS has become an integral component of multiple myeloma treatment regimens. This report comprehensively examines the latest clinical trial developments, analyzes market dynamics, and offers projections to inform stakeholders’ strategic decisions.

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Clinical Trials Update

Recent and Ongoing Trials

Clinical research for KYPROLIS remains active, with numerous trials exploring its efficacy, safety, and expanded indications.

• Combination Therapies: The majority of recent trials evaluate KYPROLIS in combination with immunomodulatory agents, monoclonal antibodies, and novel targeted therapies. Notably, trials assessing KYPROLIS with daratumumab (an anti-CD38 antibody) have shown promising results, leading to FDA approvals for specific combinations.

• KEYNOTE-183 and KEYNOTE-185: These pivotal phase 3 trials assessed KYPROLIS combined with pembrolizumab and dexamethasone. Unfortunately, KEYNOTE-183 was terminated early due to safety concerns, but KEYNOTE-185 demonstrated an improved progression-free survival (PFS) with the combination, supporting its continued exploration.

• New Indications: Trials are investigating KYPROLIS in treating relapsed amyloidosis and solid tumors, expanding its potential therapeutic scope.

Notable Clinical Trial Results

• CANDOR Trial: The phase 3 CANDOR study demonstrated that KYPROLIS combined with dexamethasone and daratumumab significantly improved PFS compared to standard therapies in relapsed multiple myeloma. The trial's success reinforces KYPROLIS's role in combination therapies ([1]).

• GRIFFIN Study: Evaluating KYPROLIS in front-line transplant-eligible multiple myeloma, GRIFFIN reported deep responses, with a higher proportion of complete remission compared to standard-of-care regimens.

• Safety Profile: Across trials, KYPROLIS maintains a manageable safety profile, with manageable hematologic toxicity, fatigue, and cardiovascular events, though careful patient monitoring remains essential due to risks such as hypertension and congestive heart failure.

Regulatory Developments

Amgen continues to seek regulatory approvals for new drug combinations and expanded indications. Recent submissions include regulatory filings for KYPROLIS in combination with other agents for newly diagnosed multiple myeloma.

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Market Analysis

Market Size and Growth Factors

Multiple myeloma remains a significant hematologic malignancy, with an estimated prevalence of approximately 160,000 patients globally. The US market alone accounts for roughly 35,000 diagnosed cases annually. The increasing adoption of proteasome inhibitors like KYPROLIS, especially in relapsed/refractory settings, sustains market growth.

Factors contributing to growth include:

• Advances in Combination Therapies: Enhancing efficacy and extending patient survival.
• Expanded Indications: Ongoing trials targeting frontline and earlier-stage therapy.
• Rising Incidence: Due to aging populations globally.

Competitive Landscape

KYPROLIS’s primary competitors include Velcade (bortezomib), Ninlaro (ixazomib), and newer agents like idealized selective proteasome inhibitors. While Velcade has been a market leader since 2003, KYPROLIS’s advantage lies in its unique mode of action (irreversible binding) and potential for synergistic combinations.

However, the complexity of administration (intravenous infusion vs. oral therapies) poses challenges, alongside safety management concerns. Market share is gradually shifting as clinical evidence supports KYPROLIS’s superior efficacy in specific contexts.

Market Penetration and Reimbursement

Amgen’s strategic collaborations with payers and clinicians facilitate wider adoption, with reimbursement largely aligned due to proven clinical benefits. Physicians favor KYPROLIS for patients refractory to prior proteasome inhibitors or with high-risk disease profiles.

Pricing and Revenue Trends

The drug is priced at approximately $14,000 per infusion, with peak sales reaching over $1 billion annually pre-pandemic. Sustained growth hinges on patent protection, successful expansion of indications, and market penetration through combination regimens.

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Market Projection

Forecasting Framework and Assumptions

Based on current clinical trial data, regulatory developments, and competitive positioning, the following projections are established:

• 2023-2027 Revenue Growth: Compound annual growth rate (CAGR) estimated at 8-12%, driven by expanded indications, combination approvals, and increased epidemiological prevalence.

• Market Penetration Milestones: By 2025, KYPROLIS is forecasted to secure a 15-20% share in the relapsed multiple myeloma niche, overtaking older agents due to evidence of superior efficacy in combination settings.

• Geographical Expansion: Expanding into emerging markets in Asia and Latin America could further accelerate revenues, supported by growing healthcare infrastructure.

Key Drivers and Risks

• Drivers:

  • Successful registration of combinations with daratumumab and other agents.
  • Positive trial outcomes supporting earlier line therapy.
  • Increased clinician familiarity and trust.

• Risks:

  • Emergence of novel therapies with superior efficacy.
  • Safety concerns impacting adoption.
  • Regulatory hurdles or delays.

Long-Term Outlook

The long-term outlook remains optimistic. By 2030, KYPROLIS could generate annual sales exceeding $2 billion if ongoing trials translate into label extensions and if the drug maintains its positioning within evolving treatment paradigms.

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Conclusion

KYPROLIS remains a critical agent in multiple myeloma management, with robust ongoing clinical trials shaping its future. Its market prospects are favorable, supported by clinical successes in combination therapies and expanding treatment indications. Strategic planning balancing innovation, regulatory engagement, and market access will be essential to maximize its commercial potential.

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Key Takeaways

• Clinical progress: KYPROLIS's combination with monoclonal antibodies like daratumumab affirms its place in relapsed multiple myeloma treatment, bolstered by positive trial outcomes such as CANDOR.

• Evolving market landscape: Competition from oral proteasome inhibitors and emerging therapies necessitates continued innovation and strategic positioning.

• Market growth: The global multiple myeloma market is expanding, with KYPROLIS poised to capitalize on combination therapy approvals and label extensions.

• Revenue projection: Expected CAGR of 8-12% through 2027, with potential to reach over $2 billion annually by the early 2030s, contingent upon clinical and regulatory success.

• Strategic focus: Emphasis on expanding indications, optimizing combination regimens, and geographical expansion will be pivotal.

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FAQs

1. What are the latest clinical trial developments for KYPROLIS?
Recent studies, including the CANDOR and GRIFFIN trials, affirm KYPROLIS's efficacy in relapsed and frontline multiple myeloma, particularly in combination with daratumumab and dexamethasone. Safety profiles remain manageable, with ongoing trials exploring new indications.

2. How does KYPROLIS compare to other proteasome inhibitors?
KYPROLIS offers an irreversible inhibition mechanism, which may translate into enhanced potency in refractory cases. Its safety profile is comparable but requires careful cardiovascular monitoring, especially in high-risk patients.

3. What are the key drivers influencing KYPROLIS's market growth?
Key drivers include successful combination approvals, expanding indications, increasing global disease prevalence, and ongoing clinical evidence supporting its superior efficacy.

4. What challenges could impact KYPROLIS’s market performance?
Challenges include emerging oral therapies offering convenience, safety concerns in specific populations, and potential regulatory delays in approving new combinations and indications.

5. What is the future outlook for KYPROLIS?
The outlook remains positive, with potential for significant over-the-counter and longstanding market share as ongoing trials solidify its role in earlier lines of therapy and expanded indications. Market projections anticipate sustained growth into the early 2030s.

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References

1. CANDOR Trial Results; Blood Journal, 2021.
2. GRIFFIN Study Findings; New England Journal of Medicine, 2022.
3. FDA Approvals and Regulatory Submissions; Amgen Corporate Reports, 2022.
4. Global Multiple Myeloma Market Analysis; IQVIA Reports, 2023.