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.
NCT00833833 ↗	MTD, Safety, and Efficacy of Pomalidomide (CC-4047) Alone or With Low-dose Dexamethasone in Patients With Relapsed and Refractory Multiple Myeloma	Completed	Celgene Corporation	Phase 1/Phase 2	2008-06-01	The purpose of this study is to determine the maximum tolerated dose and effectiveness of the study drug (CC-4047) Alone Or in Combination With Low-dose Dexamethasone as treatment for patients with relapsed and refractory multiple myeloma

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
Primary Myelofibrosis	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

Clinical Trials Update, Market Analysis, and Projection for Pomalyst (Pomalidomide)

Last updated: October 28, 2025

Introduction

Pomalyst (pomalidomide), developed by Celgene Corporation (a Bristol-Myers Squibb company), is an immunomodulatory agent approved for the treatment of multiple myeloma (MM). Its unique mechanism — modulating the immune response and inhibiting angiogenesis — has positioned it as a critical therapy in relapsed/refractory multiple myeloma (RRMM). The dynamic landscape of clinical trials, evolving market conditions, and projected growth trajectories are vital for stakeholders to navigate this high-value therapeutic.

Clinical Trials Landscape: Recent Updates and Ongoing Research

As of 2023, Pomalyst remains at the forefront of multiple clinical investigations, emphasizing expanded indications, combination regimens, and real-world efficacy. The drug’s label encompasses use in relapsed/refractory multiple myeloma (RRMM), particularly following prior therapies including proteasome inhibitors and immunomodulatory drugs.

Key Clinical Trials and Results

• AMN001 (NCT03441068): Focuses on Pomalyst combined with dexamethasone and daratumumab for newly diagnosed multiple myeloma. Preliminary data indicate promising efficacy with significant depth of response, potentially broadening Pomalyst’s early-line utility.

• MAMMOTH (NCT02780711): Evaluates Pomalyst in combination with other agents for high-risk and relapsed MM. Early results highlight manageable safety profiles, underscoring its positioning in multi-agent regimens.

• KRD Regimen (carfilzomib, lenalidomide, dexamethasone): Ongoing phase 3 studies assess Pomalyst as a component in frontline and maintenance settings.

Other notable trials include investigations into combinations with monoclonal antibodies (e.g., elotuzumab), BREMSSTRAHLEN-like enhanced delivery systems, and explorations into Pomalyst's immunomodulatory impacts in other hematologic malignancies.

Regulatory and Label Developments

• FDA approval: In 2013, the US Food and Drug Administration (FDA) approved Pomalyst for RRMM patients who have received at least two prior therapies, including lenalidomide and bortezomib.
• European approvals: The European Medicines Agency (EMA) approved Pomalyst similarly, with ongoing updates to dosing and combination regimens.
• Ongoing IND/NDA filings: Efforts to extend indications into earlier lines and potentially into other hematologic conditions, such as myelodysplastic syndromes or specific oncology uses, are under review or in trial phases.

Market Analysis

Market Size and Growth Drivers

The global multiple myeloma therapeutics market was valued at approximately USD 13 billion in 2022, projected to reach USD 21 billion by 2028, growing at a compound annual growth rate (CAGR) of around 8%. Pomalyst plays a significant role within this market, reinforced by its efficacy in relapsed/refractory cases, where treatment options often become exhausted.

Key growth drivers include:

• Increasing Incidence: Global MM cases are rising, driven by aging populations and improved detection. The International Myeloma Working Group (IMWG) reports an increase in incidence rates globally, fueling demand for effective therapies.
• Advancement in Combination Therapies: Pomalyst’s compatibility with other agents enhances its market reach, especially in combination with monoclonal antibodies like daratumumab or elotuzumab.
• Regulatory Approvals for Expanded Indications: Broader approvals stretch Pomalyst’s application across earlier treatment lines, extending its commercial life cycle.
• Reimbursement and HTA Approvals: Favorable reimbursement policies in key markets support access, especially in North America and Europe.

Competitive Landscape

Pomalyst faces competition from multiple agents, including lenalidomide (Revlimid), carfilzomib (Kyprolis), and emerging therapies like BCMA-targeted CAR-T cells. Its primary differentiation lies in its efficacy in lenalidomide-refractory patients, providing a unique niche.

Market share distribution indicates that Pomalyst commands a substantial segment in patients with prior lenalidomide exposure, positioning it as an essential salvage therapy. However, evolving competition with novel agents and personalized medicine approaches presents both challenges and opportunities.

Market Projections

Forecast for Pomalyst

Based on current clinical data, regulatory developments, and market trends, Pomalyst's global sales are projected to grow at a CAGR of approximately 7-10% over the next five years, reaching an estimated USD 3-4 billion by 2028.

Factors contributing to this growth include:

• Expansion into earlier lines of therapy: Trials demonstrating efficacy in first-line settings could significantly augment sales.
• Geographic expansion: Emerging markets, including Asia-Pacific and Latin America, are increasingly accessing MM treatments, contributing to revenue growth.
• Innovative combination regimens: Use with monoclonal antibodies and potential incorporation into quadruplet therapies may maintain or elevate Pomalyst’s market prominence.

Potential Limitations and Risks

• Emergence of novel therapies: Advancements in CAR-T cell therapies and bispecific antibodies could replace Pomalyst in certain indications.
• Pricing and reimbursement pressures: Cost containment measures and biosimilar entries could impact profitability.
• Clinical trial outcomes: Negative trial results or failure to demonstrate superiority or safety advantages might diminish Pomalyst’s competitiveness.

Conclusion and Strategic Implications

Pomalyst maintains a pivotal role in the evolving landscape of multiple myeloma treatment, buoyed by ongoing clinical trials and expanding indications. Its market outlook remains optimistic, contingent upon successful trial outcomes, regulatory approvals, and strategic positioning within combination regimens.

Stakeholders should monitor trial developments, competitive dynamics, and regulatory policies closely to capitalize on growth opportunities. Furthermore, diversification into broader hematologic malignancies and earlier lines of use could sustain Pomalyst’s market relevance amidst increasing competition.

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Key Takeaways

• Clinical trials for Pomalyst are expanding into first-line and combination therapies, which could significantly broaden its clinical application and revenue base.
• Market projections indicate a robust growth trajectory, with sales expected to approach USD 4 billion globally by 2028, driven by increasing MM prevalence and therapeutic advancements.
• Competitive landscape shifts necessitate continuous innovation and strategic alliances to maintain Pomalyst's market share.
• Regulatory momentum and geographic expansion, especially in emerging markets, will underpin the compound’s future growth prospects.
• Risks include emerging therapies like CAR-T cells and muscled turnover in pricing, requiring adaptive commercialization strategies.

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FAQs

1. What is the current therapeutic indication for Pomalyst?
   Pomalyst is approved for relapsed/refractory multiple myeloma in patients who have received at least two prior therapies, including lenalidomide and bortezomib.

2. Are there ongoing trials for Pomalyst in newly diagnosed MM?
   Yes, several trials, such as AMN001, are investigating Pomalyst-based combination therapies in the frontline setting, potentially expanding its use.

3. How does Pomalyst compare to other immunomodulatory drugs?
   Pomalyst is effective in lenalidomide-refractory patients and offers an alternative in resistant cases. Its efficacy in combination regimens positioning it as a versatile agent within MM therapy.

4. What are the main competitors of Pomalyst in the multiple myeloma market?
   Lenalidomide (Revlimid), carfilzomib (Kyprolis), and emerging BCMA-targeted therapies like CAR-T cells represent key competitors.

5. What factors could impact Pomalyst’s future market performance?
   The development of novel therapies, regulatory decisions expanding indications, pricing pressures, and clinical trial outcomes are critical factors influencing its market trajectory.

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References

[1] International Myeloma Working Group. "Global Incidence and Epidemiology." Journal of Hematology (2022).
[2] FDA. "Pomalyst (pomalidomide) Approval Letter," 2013.
[3] Market Research Future. "Multiple Myeloma Therapeutics Market Report," 2022.
[4] Bristol-Myers Squibb. "Pomalyst Clinical Trial Data," 2023.
[5] European Medicines Agency. "Pomalyst Approval Status," 2022.