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.
NCT00530816 ↗	Phase 2 Study of Carfilzomib in Relapsed Multiple Myeloma	Completed	Amgen	Phase 2	2007-09-01	To evaluate the best overall response rate, safety and tolerability of carfilzomib in patients with relapsed or refractory multiple myeloma.

Clinical Trial Conditions for Carfilzomib

Condition Name

Condition Name for Carfilzomib
Intervention	Trials
Multiple Myeloma	120
Recurrent Plasma Cell Myeloma	14
Refractory Plasma Cell Myeloma	13
Lymphoma	9

Condition MeSH

Condition MeSH for Carfilzomib
Intervention	Trials
Multiple Myeloma	184
Neoplasms, Plasma Cell	180
Lymphoma	22
Lymphoma, Non-Hodgkin	8

Clinical Trial Locations for Carfilzomib

Trials by Country

Trials by Country for Carfilzomib
Location	Trials
United States	894
Japan	91
Canada	82
Spain	61
Germany	51

Trials by US State

Trials by US State for Carfilzomib
Location	Trials
New York	63
Texas	55
California	51
Illinois	44
New Jersey	43

Clinical Trial Progress for Carfilzomib

Clinical Trial Phase

Clinical Trial Phase for Carfilzomib
Clinical Trial Phase	Trials
Phase 4	2
Phase 3	26
Phase 2/Phase 3	1

Clinical Trial Status

Clinical Trial Status for Carfilzomib
Clinical Trial Phase	Trials
Recruiting	65
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)	30

Sponsor Type

Sponsor Type for Carfilzomib
Sponsor	Trials
Industry	262
Other	241
NIH	30

Carfilzomib: Clinical Trials, Market Analysis, and Projections

Introduction to Carfilzomib

Carfilzomib, marketed under the brand name Kyprolis, is a proteasome inhibitor used in the treatment of multiple myeloma, a type of blood cancer. Developed and commercialized by Amgen, carfilzomib has shown significant efficacy in various clinical trials and has become a crucial component in the treatment regimen for multiple myeloma patients.

Clinical Trials Update

CANDOR Trial

The CANDOR trial (NCT03158688) is a pivotal phase 3, randomized, open-label study that compared the efficacy and safety of carfilzomib, daratumumab, and dexamethasone (KdD) versus carfilzomib and dexamethasone (Kd) in adults with relapsed or refractory multiple myeloma (RRMM) who had received 1 to 3 prior therapies. The trial met its primary endpoint of progression-free survival (PFS) and key secondary endpoints, including overall response rate and minimal residual disease-negative complete response at 12 months. The final analysis, with a median follow-up of 50 months, showed that the KdD arm had a significantly improved median PFS compared to the Kd arm (28.6 vs 15.2 months)[1].

Other Clinical Trials

• A phase 1/2 trial investigated the safety and efficacy of carfilzomib as a replacement for bortezomib in combination regimens for patients who were refractory or intolerant to bortezomib. The trial demonstrated that carfilzomib was an effective and tolerable replacement, with an overall response rate (ORR) of 50% and a median time to progression of 9.8 months[4].
• The ASPIRE trials are evaluating the efficacy and feasibility of using carfilzomib in combination with lenalidomide and low-dose dexamethasone in patients with relapsed multiple myeloma. Interim analyses have shown high activity and good tolerability of this combination regimen[4].

Market Analysis

Market Size and Growth

The global carfilzomib market is expected to witness significant growth due to several factors. The increasing incidence of blood cancers, particularly in densely populated regions like South Asia, is driving the demand for effective treatment therapies. The market size is projected to grow at a compound annual growth rate (CAGR) from 2024 to 2031, with North America anticipated to remain the largest market due to the rising number of blood and bone marrow cancer cases[2][5].

Key Market Trends

• Regional Growth: North America is expected to hold its position as the largest market for carfilzomib, while the Asia-Pacific region is witnessing growth due to increasing awareness about cancer and rising disposable income[2].
• New Product Approvals: Recent approvals, such as Natco Ltd.'s approval for 10mg and 60mg strengths of carfilzomib, are expanding the market. Collaborations between pharmaceutical companies, like the agreement between Janssen and Amgen, are also driving innovation and growth[2][3].
• Clinical Trial Outcomes: Positive outcomes from clinical trials, such as the CANDOR trial, are reinforcing the efficacy and safety of carfilzomib-based regimens, further boosting market confidence[1].

Challenges and Restraints

• High Administration Costs: The high cost of carfilzomib and its administration is a significant barrier, particularly in underdeveloped nations where there is a lack of awareness and resources[2].
• Limited Access: Inability of manufacturers to balance the cost-benefit tradeoff hampers penetration into tier-3 population bases, limiting market expansion[2].

Market Projections

Future Outlook

The carfilzomib market is anticipated to grow substantially over the next few years, driven by the increasing demand for effective multiple myeloma treatments and the ongoing development of new combination regimens.

• Market Size: The global carfilzomib market size is expected to increase significantly from 2024 to 2031, with a projected CAGR that reflects the growing need for advanced therapeutic drugs[5].
• Regional Expansion: The market is expected to expand in emerging economies, particularly in the Asia-Pacific region, due to growing awareness and rising disposable incomes[2].

Key Players

Major players in the carfilzomib market include Amgen, Janssen Biotech, Natco Ltd., and other pharmaceutical and biotech companies. These companies are involved in various collaborations and clinical trials to enhance the efficacy and availability of carfilzomib-based treatments[2][3].

Impact of Clinical Trials on Market Growth

Clinical trials have been instrumental in establishing the efficacy and safety of carfilzomib. The positive outcomes from trials like the CANDOR study have significantly boosted the market by providing robust evidence of the drug's benefits.

"The CANDOR final analysis was prespecified to occur after 230 survival events or 58 months after the first patient was enrolled, whichever came first. Patients treated with KdD had higher minimal residual disease–negative complete response rates and improved median PFS compared to those treated with Kd"[1].

These results not only reinforce the clinical utility of carfilzomib but also support regulatory approvals and market expansion.

Conclusion

Carfilzomib has emerged as a critical component in the treatment of multiple myeloma, supported by robust clinical trial data and a growing market demand. As the global carfilzomib market continues to expand, driven by increasing incidence of blood cancers and positive clinical trial outcomes, it is expected to offer lucrative opportunities for pharmaceutical and biotech companies.

Key Takeaways

• Clinical Efficacy: Carfilzomib has demonstrated significant efficacy in clinical trials, particularly in combination regimens.
• Market Growth: The global carfilzomib market is projected to grow substantially due to increasing demand and positive clinical trial outcomes.
• Regional Expansion: North America and the Asia-Pacific region are key markets, with the latter showing significant growth potential.
• Challenges: High administration costs and limited access in underdeveloped regions remain significant challenges.

FAQs

What is the primary use of carfilzomib?

Carfilzomib is primarily used in the treatment of multiple myeloma, a type of blood cancer.

What were the key findings of the CANDOR trial?

The CANDOR trial showed that the combination of carfilzomib, daratumumab, and dexamethasone (KdD) significantly improved progression-free survival (PFS) and other key efficacy endpoints compared to carfilzomib and dexamethasone (Kd) alone[1].

Which regions are expected to drive the growth of the carfilzomib market?

North America is expected to remain the largest market, while the Asia-Pacific region is anticipated to show significant growth due to increasing awareness and rising disposable incomes[2].

What are the major challenges facing the carfilzomib market?

High administration costs and limited access in underdeveloped regions are significant challenges hampering market expansion[2].

Who are the key players in the carfilzomib market?

Major players include Amgen, Janssen Biotech, Natco Ltd., and other pharmaceutical and biotech companies involved in clinical trials and market development[2][3].

Sources

1. Final analysis of carfilzomib, dexamethasone, and daratumumab vs carfilzomib and dexamethasone in adults with relapsed/refractory multiple myeloma. Blood Advances, 2023.
2. Carfilzomib Market Size and Industry Growth By 2030. Allied Market Research, 2023.
3. Janssen Announces Clinical Trial Collaboration and Supply Agreement with Amgen to Evaluate daratumumab (DARZALEX®) in Combination with carfilzomib (KYPROLIS®). Janssen, 2016.
4. Clinical Trial Updates on Carfilzomib-Based Treatment for Patients with Multiple Myeloma. The Oncology Nurse, 2023.
5. Global Carfilzomib Market Report 2024 Edition. Cognitive Market Research, 2024.