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.

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

Pomalyst (pomalidomide) clinical trials update, market analysis, and exclusivity-led launch projections

Last updated: May 22, 2026

Executive summary

Pomalyst (pomalidomide) is a late-line multiple myeloma (MM) agent whose near-term clinical pipeline is concentrated in combination regimens across relapsed/refractory MM, with regulatory activity focused on expanding labeled use rather than replacing the core treatment setting. Commercially, Pomalyst remains a niche but high-value immunomodulatory (IMiD) franchise compared with broader lenalidomide and thalidomide adoption, supported by continued demand in patients who progress after lenalidomide and bortezomib. Patent and exclusivity dynamics are central to generic or biosimilar risk: pomalidomide is a small molecule with small-molecule generic pathways, so the primary launch timing constraints are patent expiries and Orange Book-listed listed protections, plus any settlement outcomes tied to Paragraph IV certifications.

What is Pomalyst (pomalidomide) and how is it used in relapsed/refractory multiple myeloma?

Pomalyst is pomalidomide, a thalidomide analog IMiD used in relapsed/refractory MM. The key business driver is where it fits after failure of earlier IMiDs and proteasome inhibitors. In practice, this is a “difficult-to-treat” segment with fewer effective alternatives, which limits price erosion even when generic risk increases.

Standard-of-care positioning

• Common positioning is relapsed/refractory MM after prior lenalidomide and bortezomib exposure (exact labeling depends on the approved label language and regimen context).
• The market’s clinical value proposition is response depth in heavily pretreated patients where other agents face reduced effectiveness.

What clinical trials are active for Pomalyst and what do recent updates show?

Clinical trial activity for Pomalyst is dominated by combination studies in MM, including regimens with:

• Proteasome inhibitors (class-level competition and sequencing effect)
• Monoclonal antibodies (post-2015 standard backbone)
• Other IMiDs or novel agents designed to reduce resistance

Which trial categories drive the next labeling cycle?

1. Relapsed/refractory MM combination expansions
   • Goal is improved response rate and progression-free survival in earlier relapsed settings.
2. High-risk cytogenetics and refractory subgroups
   • Goal is to demonstrate benefit in patients with poor prognostic markers.
3. Sequential therapy strategy
   • Goal is to improve outcomes by positioning Pomalyst after prior IMiD/proteasome exposure.

What endpoints matter for market adoption?

• Confirmed overall response rate (ORR)
• Duration of response (DoR)
• Progression-free survival (PFS)
• Overall survival (OS) when statistically powered
• MRD (minimal residual disease) when used for claims and adoption

How to read pipeline signal for commercial projection

• Positive ORR/PFS with manageable safety tends to increase early-line penetration through combination incorporation.
• Safety constraints in frail populations (hematologic toxicity, infection risk) limit share gains even with efficacy.

What is the current market size and demand profile for Pomalyst?

Pomalyst is a specialty oncology product with demand anchored in heavily pretreated MM. The demand profile typically tracks:

• MM incidence and the proportion progressing to lenalidomide-refractory and proteasome-inhibitor-refractory disease
• Treatment duration (how quickly regimens switch after progression)
• Share of wallet in physician prescribing across multiple competing MM backbone regimens

Market characteristics

• Prescription concentration in large oncology practices and academic centers that run clinical protocols.
• Switching behavior influenced by newly approved MM combinations, especially those that deliver fixed-duration regimens or superior survival.

How does Pomalyst revenue projection change if generics enter?

Generic entry for pomalidomide would pressure net pricing quickly, but the pace and magnitude depend on:

• Patent expiration and whether Orange Book protections block entry
• Potential “authorized generic” dynamics
• Payer formulary behavior and patient assistance programs
• Competition from newer MM combinations that change the treat-or-switch landscape

Launch-impact framework for projection

• Year 0-1 post-entry: price compression and share shift to the lowest-cost therapeutically equivalent option
• Year 2-3: stabilization if competitors have sufficient supply and access
• Ongoing: offset from uptake in new labeled combinations or trial-driven evidence expansion

Which patents protect Pomalyst and how do they affect generic entry risk?

The generic-entry calendar is governed by:

• Orange Book-listed patents covering active ingredient, formulation, and method-of-use
• Expiration dates for each listed patent
• Any pediatric exclusivity extensions
• Any regulatory exclusivities tied to approval history

What Orange Book protections typically block generic substitution?

For small molecules like pomalidomide, Orange Book protections often include:

• Composition of matter (active ingredient and key chemical variants)
• Polymorph/crystal form (if applicable)
• Formulation and manufacturing process patents
• Method-of-use patents tied to dosing schedules or specific combination regimens

Paragraph IV challenge dynamics

• The most relevant generic risk is to identify whether any ANDA has been filed with Paragraph IV certifications against Orange Book patents and whether the brand faced litigation and settlements.
• Where settlements include “time-to-market” limitations, the practical launch date can shift by years.

When does Pomalyst lose exclusivity and what does that mean for market timing?

Exclusivity timelines are driven by the latest patent expiration plus any regulatory exclusivity (if present). The business question is whether the latest listed protection expires early enough to enable first generic launch before major next-wave clinical adoption and contracting.

Exclusivity-driven projection logic

• If Orange Book has multiple later-expiring protections, generic entry can be delayed even if earlier patents expire.
• If a method-of-use patent controls the labeled patient population used in practice, biosimilar-like “clinical non-interchangeability” constraints can arise through labeling design, even for small molecules.

What is the competitive landscape for Pomalyst in multiple myeloma?

Pomalyst competes within a crowded MM treatment ecosystem where efficacy, safety, and line-of-therapy fit drive share.

Direct and indirect competitors

• IMiD-linked therapies (lenalidomide-based and other thalidomide analogs)
• Proteasome inhibitors (including next-generation compounds)
• Anti-CD38 monoclonals (where the standard-of-care has moved toward combination backbones)
• BCMA-targeting therapies and bispecific antibodies in later lines (sequencing impact)

Competitive implications for clinical uptake

• When a new agent delivers a strong survival advantage in a similar line-of-therapy, physicians may shift away from pomalidomide, reducing duration and penetration.
• When Pomalyst shows retained activity in refractory disease, it can hold share even as combination choices expand.

What patent litigation affects Pomalyst and how does it shape licensing and settlements?

For branded small molecules, litigation impacts:

• The timing of ANDA approvals
• The settlement pay terms or “carve-out” agreements
• The scope of authorized generic or co-marketing arrangements

How to map litigation into a launch forecast

• Identify the asserted Orange Book patents and their expiration dates
• Track injunction outcomes or settlement triggers tied to specific patents
• Convert settlement terms into a practical launch timeline for generic entrants

What formulations and dosing regimens are protected, and can that delay generic substitution?

Formulation patents can delay generic interchange if they protect:

• Specific capsule formulations
• Dose strength-specific compositions
• Manufacturing processes that affect bioavailability or stability

Dosing schedule IP and method-of-use patents

Method-of-use patents can restrict clinical switching if they cover:

• Particular patient subsets (for example, lenalidomide-refractory)
• Specific combinations with named dosing schedules
• Use after exposure to defined prior therapies

What FDA status and regulatory pathway considerations matter for Pomalyst?

Pomalyst’s regulatory profile is tied to:

• Label indications and updates
• REMS requirements for IMiDs (affects distribution and patient access, not generic entry)
• The ANDA pathway for generics (bioequivalence and labeling alignment)

What does REMS mean for market access and substitution?

• REMS structures can complicate rapid post-entry ramp in some settings, but they generally do not prevent substitution if labeling is compatible.
• Operational delays can smooth early-week adoption curves, which matters for short-term revenue.

How does Pomalyst compare with lenalidomide and other IMiDs on lifecycle risk?

Pomalyst is often treated as the “next IMiD” after lenalidomide failure. Lifecycle risk depends on:

• Relative efficacy in lenalidomide-refractory populations
• How quickly competitors shift into similar niches
• Generic timing relative to new clinical expansions

Projection implication

• If Pomalyst retains a defensible niche (high refractoriness), generics may erode price but not fully extinguish demand for some period.
• If standards shift further toward bispecifics and cellular therapies, volume pressure increases regardless of generic entry.

How many patents cover Pomalyst and what is the strength of the patent estate?

The strength of the patent estate depends on:

• Number of Orange Book-listed patents
• Concentration of late-expiring patents
• Remaining term versus ANDA filing and litigation milestones
• Whether patents are broad composition claims or narrow formulation/method claims

Patent estate scoring for market impact

• High protection density: more barriers and higher risk of delayed generic entry
• Late-expiring core claims: most determinative of launch timing
• Multiple method-of-use barriers: can limit clinical substitution even post-expiry

Key market projection scenarios for Pomalyst (exclusivity-led)

Below are scenario templates that businesses typically use for forecast ranges driven by launch timing and utilization.

Scenario A: No meaningful generic erosion in the near term

• Drivers: late-expiring Orange Book patents; litigation delay; continued strong combination adoption
• Outcome: Pomalyst maintains specialty volume with slower net price compression

Scenario B: Generic entry after the last-late patent protection expires

• Drivers: successful end of litigation, ANDA final approval aligned to patent expiry
• Outcome: price compression with partial volume retention based on clinical inertia and refractoriness niche

Scenario C: Earlier-than-expected generic launch due to settlement terms

• Drivers: settlement triggers earlier than expected; patent carve-outs or consent judgments
• Outcome: faster revenue ramp-down; accelerated formulary substitution in community settings

Key takeaways

• Pomalyst’s commercial position remains anchored in relapsed/refractory MM after failure of key prior therapies, which supports demand even in a crowded MM market.
• Clinical pipeline value is concentrated in combination and earlier relapsed expansions that can broaden prescribing beyond the most refractory niche.
• Generic launch risk is a function of Orange Book patent expiries, Paragraph IV litigation outcomes, and any settlement terms that shift practical entry timing.
• Forecasting requires an exclusivity-led timeline view rather than a single “active ingredient” expiration date; formulation and method-of-use patents can materially delay interchange.

FAQs

1) What is the Orange Book status of Pomalyst and which patents are listed as “active”?

2) Have any generic ANDAs for pomalidomide filed Paragraph IV certifications against Pomalyst?

3) What exclusivity extensions could delay generic entry for pomalidomide products?

4) Do method-of-use patents limit substitution of Pomalyst in clinical practice even after patent expiry?

5) How do newer MM therapies (anti-CD38, bispecifics, CAR T) affect Pomalyst utilization and duration of therapy?

References

1. FDA. “Drugs@FDA: Pomalyst (pomalidomide)”. U.S. Food and Drug Administration.
2. FDA. “Orange Book: Approved Drug Products with Therapeutic Equivalence Evaluations”. U.S. Food and Drug Administration.
3. FDA. “ANDA Submissions: Paragraph IV Certifications and Hatch-Waxman Act”. U.S. Food and Drug Administration.
4. ClinicalTrials.gov. “Pomalidomide”. U.S. National Library of Medicine.