CLINICAL TRIALS PROFILE FOR POMALYST

505(b)(2) Clinical Trials for Pomalyst

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	NCT02103335 ↗	Combination Study of Pomalidomide, Marizomib, and Low-Dose Dexamethasone in Relapsed and Refractory Multiple Myeloma	Completed	Celgene Corporation	Phase 1	2014-06-05	This is a Phase 1 clinical trial to evaluate a new combination of drugs for the treatment of relapsed or refractory (drug-resistant) multiple myeloma. The drugs being studied are: - Pomalidomide (POMALYST®) is a drug that affects the immune system (an immunomodulatory drug) that has been approved by the United States (US) Food and Drug Administration (FDA) for the treatment of multiple myeloma. - Marizomib is an investigational drug being developed by Triphase that is being studied for the treatment of multiple myeloma. Investigational drugs are drugs that have not yet been approved by health authorities, such as the FDA, for general use but have been approved for use in specific clinical studies. Marizomib inhibits a cellular machine called the proteasome, which destroys unnecessary or damaged proteins. Other proteasome inhibitors have been shown to be effective in the treatment of multiple myeloma. - Dexamethasone is a corticosteroid drug that affects the immune system (an immunomodulatory drug) that has been approved by the FDA for the treatment of multiple myeloma. This is the first study to evaluate the three-drug combination of pomalidomide (POM), marizomib (MRZ), and dexamethasone (LD-DEX) in humans. Pomalidomide, alone or in combination with dexamethasone, is approved by the FDA for the treatment of relapsed or refractory multiple myeloma. The primary objective of this study is to determine the best drug dosing levels for this three-drug combination, including the highest safe doses and/or the recommended doses for future clinical studies of this drug combination. The secondary purposes of this study are to determine the safety of this drug combination and its effectiveness in treating relapsed or refractory multiple myeloma. The study will include examination of levels of all three drugs in the blood during various time points during treatment.
New Combination	NCT02103335 ↗	Combination Study of Pomalidomide, Marizomib, and Low-Dose Dexamethasone in Relapsed and Refractory Multiple Myeloma	Completed	Celgene	Phase 1	2014-06-05	This is a Phase 1 clinical trial to evaluate a new combination of drugs for the treatment of relapsed or refractory (drug-resistant) multiple myeloma. The drugs being studied are: - Pomalidomide (POMALYST®) is a drug that affects the immune system (an immunomodulatory drug) that has been approved by the United States (US) Food and Drug Administration (FDA) for the treatment of multiple myeloma. - Marizomib is an investigational drug being developed by Triphase that is being studied for the treatment of multiple myeloma. Investigational drugs are drugs that have not yet been approved by health authorities, such as the FDA, for general use but have been approved for use in specific clinical studies. Marizomib inhibits a cellular machine called the proteasome, which destroys unnecessary or damaged proteins. Other proteasome inhibitors have been shown to be effective in the treatment of multiple myeloma. - Dexamethasone is a corticosteroid drug that affects the immune system (an immunomodulatory drug) that has been approved by the FDA for the treatment of multiple myeloma. This is the first study to evaluate the three-drug combination of pomalidomide (POM), marizomib (MRZ), and dexamethasone (LD-DEX) in humans. Pomalidomide, alone or in combination with dexamethasone, is approved by the FDA for the treatment of relapsed or refractory multiple myeloma. The primary objective of this study is to determine the best drug dosing levels for this three-drug combination, including the highest safe doses and/or the recommended doses for future clinical studies of this drug combination. The secondary purposes of this study are to determine the safety of this drug combination and its effectiveness in treating relapsed or refractory multiple myeloma. The study will include examination of levels of all three drugs in the blood during various time points during treatment.
New Combination	NCT02103335 ↗	Combination Study of Pomalidomide, Marizomib, and Low-Dose Dexamethasone in Relapsed and Refractory Multiple Myeloma	Completed	Triphase Research and Development I Corporation	Phase 1	2014-06-05	This is a Phase 1 clinical trial to evaluate a new combination of drugs for the treatment of relapsed or refractory (drug-resistant) multiple myeloma. The drugs being studied are: - Pomalidomide (POMALYST®) is a drug that affects the immune system (an immunomodulatory drug) that has been approved by the United States (US) Food and Drug Administration (FDA) for the treatment of multiple myeloma. - Marizomib is an investigational drug being developed by Triphase that is being studied for the treatment of multiple myeloma. Investigational drugs are drugs that have not yet been approved by health authorities, such as the FDA, for general use but have been approved for use in specific clinical studies. Marizomib inhibits a cellular machine called the proteasome, which destroys unnecessary or damaged proteins. Other proteasome inhibitors have been shown to be effective in the treatment of multiple myeloma. - Dexamethasone is a corticosteroid drug that affects the immune system (an immunomodulatory drug) that has been approved by the FDA for the treatment of multiple myeloma. This is the first study to evaluate the three-drug combination of pomalidomide (POM), marizomib (MRZ), and dexamethasone (LD-DEX) in humans. Pomalidomide, alone or in combination with dexamethasone, is approved by the FDA for the treatment of relapsed or refractory multiple myeloma. The primary objective of this study is to determine the best drug dosing levels for this three-drug combination, including the highest safe doses and/or the recommended doses for future clinical studies of this drug combination. The secondary purposes of this study are to determine the safety of this drug combination and its effectiveness in treating relapsed or refractory multiple myeloma. The study will include examination of levels of all three drugs in the blood during various time points during treatment.
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 Pomalyst

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00463385 ↗	A Phase II Study of Pomalidomide in Myelofibrosis With Myeloid Metaplasia	Completed	Celgene	Phase 2	2007-04-01	The purpose of this study is to determine the safety of and to select a treatment regimen of pomalidomide (CC-4047) either as single-agent or in combination with prednisone to study further in patients with myelofibrosis with myeloid metaplasia (MMM).
NCT00463385 ↗	A Phase II Study of Pomalidomide in Myelofibrosis With Myeloid Metaplasia	Completed	Celgene Corporation	Phase 2	2007-04-01	The purpose of this study is to determine the safety of and to select a treatment regimen of pomalidomide (CC-4047) either as single-agent or in combination with prednisone to study further in patients with myelofibrosis with myeloid metaplasia (MMM).
NCT00537511 ↗	A Phase I/II Study to Determine the Maximum Tolerated Dose (MTD) and Safety of CC-4047 (Pomalidomide) Administered in Conjunction With Cisplatin and Etoposide	Terminated	Celgene	Phase 1/Phase 2	2008-02-01	The purpose of this study is to determine the maximum tolerated dose and safety of CC-4047 (pomalidomide) given in combination with cisplatin and etoposide in patients with extensive disease small cell lung cancer.
NCT00537511 ↗	A Phase I/II Study to Determine the Maximum Tolerated Dose (MTD) and Safety of CC-4047 (Pomalidomide) Administered in Conjunction With Cisplatin and Etoposide	Terminated	Celgene Corporation	Phase 1/Phase 2	2008-02-01	The purpose of this study is to determine the maximum tolerated dose and safety of CC-4047 (pomalidomide) given in combination with cisplatin and etoposide in patients with extensive disease small cell lung cancer.
NCT00717522 ↗	Efficacy and Safety Study of CC-4047 (Pomalidomide) to Treat Advanced Soft Tissue Sarcoma	Terminated	Celgene	Phase 2	2008-08-01	The purpose of the study is to determine the safety and efficacy of single agent CC-4047 (pomalidomide) in patients with advanced soft tissue sarcomas who have relapsed or are refractory to prior anticancer therapy.
NCT00717522 ↗	Efficacy and Safety Study of CC-4047 (Pomalidomide) to Treat Advanced Soft Tissue Sarcoma	Terminated	Celgene Corporation	Phase 2	2008-08-01	The purpose of the study is to determine the safety and efficacy of single agent CC-4047 (pomalidomide) in patients with advanced soft tissue sarcomas who have relapsed or are refractory to prior anticancer therapy.

Clinical Trial Conditions for Pomalyst

Condition Name

Condition Name for Pomalyst
Intervention	Trials
Multiple Myeloma	31
Recurrent Plasma Cell Myeloma	12
Refractory Plasma Cell Myeloma	10
Multiple Myeloma in Relapse	6

Condition MeSH

Condition MeSH for Pomalyst
Intervention	Trials
Multiple Myeloma	61
Neoplasms, Plasma Cell	59
Recurrence	4
Leukemia	3

Clinical Trial Locations for Pomalyst

Trials by Country

Trials by Country for Pomalyst
Location	Trials
United States	278
Canada	35
Japan	24
China	20
Spain	17

Trials by US State

Trials by US State for Pomalyst
Location	Trials
Massachusetts	20
New York	18
Minnesota	18
California	18
Ohio	17

Clinical Trial Progress for Pomalyst

Clinical Trial Phase

Clinical Trial Phase for Pomalyst
Clinical Trial Phase	Trials
Phase 4	1
Phase 3	7
Phase 2	41

Clinical Trial Status

Clinical Trial Status for Pomalyst
Clinical Trial Phase	Trials
Active, not recruiting	19
Completed	18
Recruiting	17

Clinical Trial Sponsors for Pomalyst

Sponsor Name

Sponsor Name for Pomalyst
Sponsor	Trials
National Cancer Institute (NCI)	20
Celgene	19
Celgene Corporation	14

Sponsor Type

Sponsor Type for Pomalyst
Sponsor	Trials
Industry	85
Other	75
NIH	20

Pomalyst: A Comprehensive Overview of Clinical Trials, Market Analysis, and Projections

Introduction to Pomalyst

Pomalyst, also known as pomalidomide, is a significant advancement in the treatment of various cancers, notably multiple myeloma and Kaposi sarcoma. Developed by Bristol Myers Squibb, this immunomodulatory drug has garnered attention for its efficacy and safety profile.

Clinical Trials for Pomalyst

Kaposi Sarcoma Trials

Pomalyst was granted accelerated approval by the US FDA for the treatment of AIDS-related and HIV-negative Kaposi sarcoma (KS) based on a Phase 1/2 open-label, single-arm clinical trial. This trial, conducted under a Cooperative Research and Development Agreement (CRADA) with the National Cancer Institute (NCI), involved 28 patients (18 HIV-positive and 10 HIV-negative) with symptomatic KS. Patients received 5 mg of Pomalyst once daily for 21 of 28-day cycles until disease progression or unacceptable toxicity. All HIV-positive patients continued concomitant highly active antiretroviral therapy (HAART), and thromboprophylaxis with aspirin 81 mg once daily was administered throughout therapy[1][4].

The primary endpoint of the study was the overall response rate (ORR), which included complete response (CR), clinical complete response (cCR), and partial response (PR). The ORR was 71% (95% CI: 51, 87), with 14% of patients achieving CR and 57% achieving PR. The median time to first response was 1.8 months, and the median duration of response was 12.1 months (95% CI: 7.6, 16.8)[1][4].

Multiple Myeloma Trials

While the specific trial details for Pomalyst in multiple myeloma are not outlined in the provided sources, it is well-known that pomalidomide is a key treatment option for relapsed and refractory multiple myeloma. Clinical trials have demonstrated its efficacy in improving progression-free survival and overall survival in these patients. For instance, lenalidomide, another immunomodulatory drug in the same class, has shown significant improvements in survival outcomes, which can be extrapolated to some extent to pomalidomide due to their similar mechanisms of action[3].

Safety and Adverse Reactions

Common Adverse Reactions in Kaposi Sarcoma Trials

In the KS trial, common adverse reactions included maculopapular rash (71%), constipation (71%), fatigue (68%), nausea (36%), diarrhea (32%), and cough (29%). Grade 3 or 4 adverse reactions included maculopapular rash (3.6%), diarrhea (3.6%), and peripheral edema (3.6%). Laboratory abnormalities such as decreased absolute neutrophil count, elevated glucose, decreased phosphate, and elevated creatine kinase were also observed[1][4].

Common Adverse Reactions in Multiple Myeloma Trials

In multiple myeloma trials, neuropathy is a notable adverse reaction, with 18% of patients experiencing neuropathy and 12% experiencing peripheral neuropathy. Other common adverse reactions include decreased absolute neutrophil count, elevated creatinine or glucose, rash, constipation, fatigue, and decreased hemoglobin, platelets, phosphate, albumin, or calcium[4].

Market Analysis

Current Market Status

Pomalyst has been a significant player in the multiple myeloma and Kaposi sarcoma treatment markets. However, recent developments have impacted its market position. In the EU, generic pomalidomide products entered the market in August 2024, which is expected to affect the sales of the branded version[2].

Market Projections

The global multiple myeloma drugs market, which includes Pomalyst, is projected to grow at a CAGR of 6% by 2028. This growth is driven by the increasing burden of multiple myeloma and the ongoing research and development of novel therapies. Despite the entry of generic versions, the demand for immunomodulatory drugs like pomalidomide is expected to remain strong due to their efficacy in improving survival outcomes[3][5].

Competitive Landscape

The market for multiple myeloma treatments is highly competitive, with several drugs available, including lenalidomide and other newer therapies. However, Pomalyst's unique position as a treatment for Kaposi sarcoma and its established efficacy in multiple myeloma continue to make it a valuable option for clinicians. The entry of generic versions will likely increase competition but also make the drug more accessible to a broader patient population[2][5].

Regional Market Insights

North America and Europe

In these regions, Pomalyst has been well-established, and its approval for Kaposi sarcoma has expanded its market reach. The entry of generics in the EU is expected to impact sales, but the drug's strong brand presence and clinical efficacy are likely to maintain its market share to some extent[2].

Asia Pacific and Latin America

In these regions, the growth of the multiple myeloma market is driven by increasing awareness and improving healthcare infrastructure. Pomalyst, along with other immunomodulatory drugs, is expected to see increased adoption as these markets grow[5].

Future Outlook

Research and Development

Continued research and development in the field of multiple myeloma and Kaposi sarcoma are crucial for the future of Pomalyst. New clinical trials and combinations with other therapies could further enhance its efficacy and expand its indications[3].

Generic Competition

The entry of generic pomalidomide products will be a significant factor in the future market dynamics. While this will increase competition, it also presents an opportunity for broader patient access and potentially lower costs, which could drive market growth in different segments[2].

Key Takeaways

• Clinical Efficacy: Pomalyst has demonstrated strong efficacy in treating Kaposi sarcoma and multiple myeloma, with a 71% overall response rate in KS trials.
• Safety Profile: Common adverse reactions include maculopapular rash, constipation, fatigue, and laboratory abnormalities such as decreased absolute neutrophil count.
• Market Impact: The entry of generic versions in the EU and upcoming in the US will impact sales but is expected to increase patient access.
• Market Growth: The multiple myeloma drugs market is projected to grow at a CAGR of 6% by 2028, driven by increasing disease burden and research in novel therapies.
• Regional Insights: Strong brand presence in North America and Europe, with growing potential in Asia Pacific and Latin America.

FAQs

What is Pomalyst used for?

Pomalyst (pomalidomide) is used for the treatment of Kaposi sarcoma and multiple myeloma.

What were the key findings of the Phase 1/2 trial for Kaposi sarcoma?

The trial showed a 71% overall response rate, with a median time to first response of 1.8 months and a median duration of response of 12.1 months.

What are the common adverse reactions associated with Pomalyst?

Common adverse reactions include maculopapular rash, constipation, fatigue, nausea, diarrhea, and laboratory abnormalities such as decreased absolute neutrophil count.

How will the entry of generic pomalidomide products affect the market?

The entry of generics will increase competition but is also expected to make the drug more accessible and potentially lower costs, driving market growth in different segments.

What is the projected growth rate of the multiple myeloma drugs market?

The global multiple myeloma drugs market is projected to grow at a CAGR of 6% by 2028.

Sources

1. Bristol Myers Squibb: "US Food and Drug Administration Approves Bristol Myers Squibb's Pomalyst (pomalidomide) for AIDS-Related and HIV-Negative Kaposi Sarcoma" - Press Release, May 15, 2020.
2. Bristol Myers Squibb: "Q3 2024 Results Presentation" - Investor Presentation, October 31, 2024.
3. Mordor Intelligence: "Multiple Myeloma Market - Trends, Forecast & Share" - Industry Report.
4. ACCC-Cancer.org: "POMALYST (pomalidomide) capsules approved to treat Kaposi sarcoma" - PDF Document.
5. iHealthcareAnalyst: "Multiple Myeloma Drugs Market and Forecast 2024-2031" - Market Report.