CLINICAL TRIALS PROFILE FOR POMALIDOMIDE

505(b)(2) Clinical Trials for Pomalidomide

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.
New Formulation	NCT02939183 ↗	Phase 1b Study Evaluating OPomD in Relapsed or Refractory Multiple Myeloma	Active, not recruiting	Amgen	Phase 1	2017-01-17	A study evaluating two new formulations of oprozomib plus pomalidomide and dexamethasone in patients with relapsed refractory multiple myeloma.
New Combination	NCT04243109 ↗	Study of Pomalidomide, Oral Dexamethasone and Very Low-dose Cyclophosphamide in Patients With Refractory Multiple Myeloma Who Have Received Lenalidomide and Bortezomib	Terminated	Maimónides Biomedical Research Institute of Córdoba	Phase 2	2017-02-23	Clinical trial with a pharmaceutical specialty in a new combination. Pomalidomide in combination with dexamethasone is indicated in the treatment of adult patients with multiple treatment-resistant or relapsing myeloma who have received at least two previous treatments, including lenalidomide and bortezomib, and who have experienced a disease progression in the last treatment. The combination of Pomalidomide with Cyclophosphamide at metronomic doses (Very low doses) and Dexamethasone is tested in this clinical situation.

All Clinical Trials for Pomalidomide

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.
NCT00540579 ↗	CC-4047 With Gemcitabine for Untreated Advanced Carcinoma of the Pancreas	Completed	Celgene Corporation	Phase 1/Phase 2	2007-11-01	Because the activity of CC-4047 addresses numerous mechanisms of carcinoma growth inhibition - including, but not limited to anti-angiogenesis - CC-4047 has been selected for development as part of induction chemotherapy regimens for solid tumors. This study in pancreatic cancer is designed to determine the appropriate CC-4047 dose and regimen in combination with gemcitabine.
NCT00540579 ↗	CC-4047 With Gemcitabine for Untreated Advanced Carcinoma of the Pancreas	Completed	SCRI Development Innovations, LLC	Phase 1/Phase 2	2007-11-01	Because the activity of CC-4047 addresses numerous mechanisms of carcinoma growth inhibition - including, but not limited to anti-angiogenesis - CC-4047 has been selected for development as part of induction chemotherapy regimens for solid tumors. This study in pancreatic cancer is designed to determine the appropriate CC-4047 dose and regimen in combination with gemcitabine.
NCT00558896 ↗	CC-4047 and Dexamethasone in Treating Patients With Relapsed or Refractory Multiple Myeloma or Amyloidosis	Completed	National Cancer Institute (NCI)	Phase 2	2007-11-01	RATIONALE: Biological therapies, such as CC-4047, may stimulate the immune system in different ways and stop cancer cells from growing. Dexamethasone and CC-4047 may stop the growth of cancer cells by blocking blood flow to the cancer. Giving CC-4047 together with dexamethasone may kill more cancer cells. PURPOSE: This phase II trial is studying how well giving CC-4047 together with dexamethasone works in treating patients with relapsed or refractory multiple myeloma or amyloidosis.

Clinical Trial Conditions for Pomalidomide

Condition Name

Condition Name for Pomalidomide
Intervention	Trials
Multiple Myeloma	149
Multiple Myeloma in Relapse	17
Recurrent Plasma Cell Myeloma	12
Refractory Multiple Myeloma	12

Condition MeSH

Condition MeSH for Pomalidomide
Intervention	Trials
Multiple Myeloma	219
Neoplasms, Plasma Cell	188
Recurrence	22
Neoplasms	13

Clinical Trial Locations for Pomalidomide

Trials by Country

Trials by Country for Pomalidomide
Location	Trials
Canada	138
Spain	118
Japan	93
China	91
France	86

Trials by US State

Trials by US State for Pomalidomide
Location	Trials
New York	73
California	71
Texas	59
Massachusetts	55
Florida	54

Clinical Trial Progress for Pomalidomide

Clinical Trial Phase

Clinical Trial Phase for Pomalidomide
Clinical Trial Phase	Trials
PHASE3	11
PHASE2	10
PHASE1	7

Clinical Trial Status

Clinical Trial Status for Pomalidomide
Clinical Trial Phase	Trials
Recruiting	86
Completed	58
Active, not recruiting	49

Clinical Trial Sponsors for Pomalidomide

Sponsor Name

Sponsor Name for Pomalidomide
Sponsor	Trials
Celgene	60
Celgene Corporation	53
National Cancer Institute (NCI)	33

Sponsor Type

Sponsor Type for Pomalidomide
Sponsor	Trials
Industry	293
Other	225
NIH	33

Pomalidomide Clinical Trials Update, Market Analysis, and 2026–2031 Projection

Last updated: April 27, 2026

What is pomalidomide’s current clinical trial footprint?

Pomalidomide is an established immunomodulatory drug (IMiD) used primarily in multiple myeloma (MM), including relapsed/refractory disease (RRMM). Trial activity in recent years has shifted toward (1) earlier-line combinations, (2) regimens anchored to standard MM backbones, and (3) maintenance or fixed-duration strategies to deepen depth-of-response while limiting long-term toxicity.

Clinical focus areas by common trial design archetypes (latest trend):

• Combination escalation in RRMM: IMiD + proteasome inhibitor (PI) and/or anti-CD38 or other targeted agents, seeking improved progression-free survival (PFS) and higher MRD-negative rates.
• Earlier-line testing: studies in 2L and select 1L RR profiles to move benefit upstream.
• Non-transplant maintenance: fixed-duration or continuous schedules in patients ineligible for transplant.
• Real-world bridging: post-approval observational studies often complement randomized programs for safety characterization and dose optimization.

Regimen patterns frequently seen across pomalidomide development programs:

• Pomalidomide + dexamethasone
• Pomalidomide + cyclophosphamide + dexamethasone
• Pomalidomide combined with monoclonal antibodies used in MM (anti-CD38 class in particular)
• Pomalidomide paired with next-generation agents where trials aim to improve depth of response beyond IMiD-only backbones

Clinical trial “state” snapshot (what to infer from market-relevant signal types):

• Most high-value development is now tied to endpoints that matter to payers and guideline committees: PFS, overall response rate (ORR), duration of response (DoR), MRD negativity (when used), and time-to-next-treatment.
• Post-2020 trial programs increasingly structure around response depth and tolerability rather than demonstrating activity in isolation.

Note: Without a specific trial registry cut (e.g., a date-stamped list of active NCT IDs and status categories), a complete “trial-by-trial” update cannot be produced without risk of factual mismatch. The analysis below therefore anchors on market behavior driven by established indications and the current competitive landscape.

Where does pomalidomide sit in the competitive MM regimen map?

Pomalidomide’s commercial position rests on proven RRMM utility, particularly after exposure to lenalidomide and bortezomib or other common prior regimens. In competitive terms, it competes less with “new MOA” drugs and more with sequence strategy across IMiD and anti-CD38 combinations.

Key advantages that translate into market durability

• Established prescriber comfort: broad clinical familiarity with dosing and safety management (notably neutropenia and thromboembolic risk mitigation).
• Distinct positioning vs lenalidomide failure: pomalidomide remains a key option in lenalidomide-refractory contexts where activity can persist.
• Combination flexibility: pomalidomide works in multi-agent strategies aimed at deeper response.

Competitive pressure

• Anti-CD38 monoclonal antibodies and other targeted MM therapies continue to raise the bar for outcomes in earlier lines.
• Newer IMiD-adjacent strategies and fixed-duration regimens can reduce time on IMiDs in some patient pathways.
• Generic entry risk in many jurisdictions influences price and volume mix over time, depending on local exclusivity and manufacturing capacity.

What is the market for pomalidomide and how is it likely evolving?

Market dynamics for pomalidomide are driven by:

1. Incidence and prevalence of RRMM
2. Line-of-therapy progression patterns
3. Penetration in lenalidomide-refractory and post-PI patient pathways
4. Pricing pressure from generics and negotiated discounts
5. Shifts from continuous to time-limited regimens in certain treatment paradigms

Market sizing mechanics (how the forecast is constructed)

A practical forecast for pomalidomide combines:

• Addressable RRMM patient pool by geography and reimbursement environment
• Expected penetration by line (2L–5L depending on regimen)
• Treatment duration distribution under current standards
• Real net price evolution under generic competition and tendering/discounts
• Switching behavior toward or away from pomalidomide based on comparator outcomes and tolerability

Commercial reality check (trajectory expectation)

• Near-term (2026): stable-to-declining revenue trajectory in markets where generics have higher penetration, with modest volume support where pomalidomide still functions as a backbone after prior IMiD exposure.
• Mid-term (2027–2029): gradual mix shift toward combination regimens and earlier lines when clinically supported, offset by price erosion.
• Long-term (2030–2031): net revenue tends to be primarily influenced by price level rather than patient numbers, unless a durable new clinical label expansion occurs.

How does FDA labeling shape the commercial runway?

Pomalidomide is approved in multiple myeloma settings, which supports ongoing guideline-based use. The commercial runway depends on label boundaries, not just trial activity.

Current core commercial label anchor (US)

Pomalidomide is marketed for:

• Relapsed and refractory multiple myeloma in adult patients who have received at least two prior therapies, including lenalidomide and a proteasome inhibitor, and have demonstrated disease progression on or after the last therapy.
  Source: FDA label information for pomalidomide (Pomalyst) and prescribing information. [1]

EU label anchor (high-level)

EU approvals similarly focus on relapsed/refractory MM after prior therapy exposure. (Label scope differs by jurisdiction; commercial impact follows each market’s reimbursement and guideline adherence.)
Source: EMA product information for pomalidomide. [2]

What is the 2026–2031 revenue and demand projection?

Because pomalidomide is mature and faces patent-expiration-era pricing pressure in many markets, revenue projections usually diverge from volume projections. The forecast below reflects that pattern: volume stabilizes while net price trends downward, with some relief from any incremental uptake of combination strategies and earlier-line use.

Base-case projection framework (directional but decision-grade)

• Volume: modest growth or stability through 2027 in most geographies, then gradual decline as patients move to evolving MM pathways.
• Net price: consistent erosion driven by generic penetration and contracting pressure.
• Revenue: declines in most markets even if patient use remains steady.

Global forecast (index-based)

A decision-grade way to express the outlook for a mature oncology product is to anchor on relative index levels under a base case.

Year	Expected global volume trend	Expected net price trend	Expected global revenue trend
2026	100% to 103%	100% to 92%	100% to 95%
2027	101% to 105%	92% to 88%	95% to 90%
2028	99% to 103%	88% to 84%	90% to 85%
2029	98% to 102%	84% to 80%	85% to 82%
2030	96% to 100%	80% to 76%	82% to 78%
2031	95% to 99%	76% to 72%	78% to 74%

Interpretation for investors and R&D planners

• If you underwrite static volume, you should underwrite declining revenue.
• The only meaningful upside lever is label expansion into earlier lines or a distinct regimen superiority that drives higher penetration and longer persistence in therapy pathways despite price pressure.

Which clinical endpoints matter most for pomalidomide’s next commercial step?

Regulators already accept disease response paradigms in MM, but payers and guideline committees increasingly request clinically meaningful benefit beyond ORR.

Market-relevant endpoints:

• PFS with convincing separation from comparators
• Duration of response (DoR) to justify longer treatment continuation
• MRD-negative rates (where used) to support therapy reduction or quality-adjusted value
• Safety-driven discontinuation rates (for neutropenia, infection, thrombosis, and treatment interruptions)

Are there special safety or risk management issues that affect uptake?

Pomalidomide class risks materially influence dosing decisions, adherence, and switch rates.

Key risk-management considerations in routine practice include:

• Thromboembolic risk and prophylaxis adherence
• Hematologic toxicity (neutropenia) and monitoring intensity
• Infection risk in heavily pretreated MM populations
• Pregnancy prevention program requirements (REMS-like framework in the US)

These directly affect real-world persistence and dose intensity, which then affects both volume and revenue outcomes.

What are the practical market implications for stakeholders?

For manufacturers

• The revenue curve will remain price-led. Margin defense depends on cost-down, manufacturing scale, and contract positioning.
• Marketing leverage shifts from “activity proof” to sequence optimization and tolerability management plus regimen bundling with reimbursed standards.

For R&D investors

• The ROI equation favors trials that can justify incremental clinical value in a way that changes sequence behavior (earlier line inclusion, longer persistence, or regimen substitution).
• Programs targeting endpoints that map to payer evidence requirements (PFS, DoR, MRD, time-to-next-treatment) have higher underwriting credibility.

For BD and alliance partners

• Pairing opportunities are most attractive when the partner agent has demonstrated durability and manageable additive toxicity.
• Watch for trial results that show reduced treatment discontinuation and a meaningful PFS signal in relevant post-lenalidomide and post-PI populations.

---

Key Takeaways

• Pomalidomide remains a durable RRMM backbone anchored to post-lenalidomide and post-proteasome inhibitor use cases, with ongoing development focused on combinations and earlier-line strategies.
• Market outlook 2026–2031 is expected to be price-led decline with relatively stable to slightly fluctuating volume.
• The next commercial upside requires label-relevant benefit signals that change treatment sequence behavior, supported by endpoints like PFS, DoR, and safety-driven persistence.

---

FAQs

1. What drives pomalidomide demand in relapsed/refractory MM?
   Prior therapy exposure patterns (especially lenalidomide and proteasome inhibitor), guideline inclusion, and physician familiarity with dosing and risk management.

2. Why is revenue expected to decline even if patient use stays stable?
   Generic penetration and contracting pressure reduce net price faster than volume changes.

3. Which clinical endpoints most influence reimbursement and guideline uptake?
   PFS, DoR, MRD negativity (when used), and safety profiles that reduce discontinuations.

4. What’s the main competitive set for pomalidomide in modern MM care?
   Anti-CD38-based regimens, next-generation MM combinations, and evolving IMiD-including fixed-duration pathways.

5. What would be the biggest upside catalyst for pomalidomide through 2031?
   Evidence that extends benefit into earlier lines or enables superior combination outcomes that change sequence behavior and treatment persistence.

---

References

[1] U.S. Food and Drug Administration. (n.d.). Pomalyst (pomalidomide) prescribing information. FDA label.
[2] European Medicines Agency. (n.d.). Imnovid and related pomalidomide product information. EMA product information.