CLINICAL TRIALS PROFILE FOR TARGRETIN

505(b)(2) Clinical Trials for TARGRETIN

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	NCT01504490 ↗	Phase I Study of CS-7017 and Bexarotene	Terminated	Daiichi Sankyo Inc.	Phase 1	2011-12-01	This study is for patients with advanced solid tumors. The purpose of this study is to test the safety and effectiveness of a new combination of drugs, CS-7017 and Bexarotene in patients with advanced cancer. CS-7017 and Bexarotene both have many effects on cancer cells, including stopping cancer cells from growing and dividing, and causing the cancer cells to die. CS-7017 and Bexarotene work on cancer cells in a similar manner and both drugs together may have an even greater effect against cancer cells, hopefully, increasing the killing of cancer cells. CS-7017 is an investigational or experimental anti-cancer agent that has not yet been approved by the Food and Drug Administration (FDA) for use in any type of cancer. Bexarotene is an anti-cancer agent that has been approved by the FDA for patients with a specific type of cancer, cutaneous T-cell lymphoma. This study will help find out what effects the combination of drugs, CS-7017 and Bexarotene, has on cancer. This research is being done because it is not known if CS-7017 is safe to be given with Bexarotene.
New Combination	NCT01504490 ↗	Phase I Study of CS-7017 and Bexarotene	Terminated	Daiichi Sankyo, Inc.	Phase 1	2011-12-01	This study is for patients with advanced solid tumors. The purpose of this study is to test the safety and effectiveness of a new combination of drugs, CS-7017 and Bexarotene in patients with advanced cancer. CS-7017 and Bexarotene both have many effects on cancer cells, including stopping cancer cells from growing and dividing, and causing the cancer cells to die. CS-7017 and Bexarotene work on cancer cells in a similar manner and both drugs together may have an even greater effect against cancer cells, hopefully, increasing the killing of cancer cells. CS-7017 is an investigational or experimental anti-cancer agent that has not yet been approved by the Food and Drug Administration (FDA) for use in any type of cancer. Bexarotene is an anti-cancer agent that has been approved by the FDA for patients with a specific type of cancer, cutaneous T-cell lymphoma. This study will help find out what effects the combination of drugs, CS-7017 and Bexarotene, has on cancer. This research is being done because it is not known if CS-7017 is safe to be given with Bexarotene.
New Combination	NCT01504490 ↗	Phase I Study of CS-7017 and Bexarotene	Terminated	Georgetown University	Phase 1	2011-12-01	This study is for patients with advanced solid tumors. The purpose of this study is to test the safety and effectiveness of a new combination of drugs, CS-7017 and Bexarotene in patients with advanced cancer. CS-7017 and Bexarotene both have many effects on cancer cells, including stopping cancer cells from growing and dividing, and causing the cancer cells to die. CS-7017 and Bexarotene work on cancer cells in a similar manner and both drugs together may have an even greater effect against cancer cells, hopefully, increasing the killing of cancer cells. CS-7017 is an investigational or experimental anti-cancer agent that has not yet been approved by the Food and Drug Administration (FDA) for use in any type of cancer. Bexarotene is an anti-cancer agent that has been approved by the FDA for patients with a specific type of cancer, cutaneous T-cell lymphoma. This study will help find out what effects the combination of drugs, CS-7017 and Bexarotene, has on cancer. This research is being done because it is not known if CS-7017 is safe to be given with Bexarotene.

All Clinical Trials for TARGRETIN

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00002212 ↗	A Study of Targretin Capsules in Patients With AIDS-Related Kaposi's Sarcoma	Completed	Ligand Pharmaceuticals	Phase 2	1969-12-31	The purpose of this study is to see if it is safe and effective to give Targretin capsules to patients with AIDS-related Kaposi's sarcoma (KS).
NCT00003752 ↗	Bexarotene in Treating Patients With Metastatic Breast Cancer	Completed	Ligand Pharmaceuticals	Phase 2	1998-10-01	RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. PURPOSE: Randomized phase II trial to study the effectiveness of bexarotene in treating patients who have metastatic breast cancer.
NCT00030589 ↗	Chemotherapy and Photodynamic Therapy in Treating Patients With Cutaneous T-Cell Lymphoma	Unknown status	Millennix	Phase 2	2001-02-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Photodynamic therapy uses light and drugs that make cancer cells more sensitive to light to kill cancer cells. Photosensitizing drugs, such as methoxsalen, are absorbed by cancer cells and, when exposed to light, become active and kill the cancer cells. Combining chemotherapy with photodynamic therapy may be an effective treatment for cutaneous T-cell lymphoma. PURPOSE: Randomized phase II trial to study the effectiveness of combining different doses of bexarotene with photodynamic therapy in treating patients who have stage IB or stage IIA cutaneous T-cell lymphoma.
NCT00030849 ↗	Bexarotene and Interferon Alfa in Treating Patients With Cutaneous T-Cell Lymphoma	Completed	National Cancer Institute (NCI)	Phase 2	2001-10-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Interferon alfa may interfere with the growth of cancer cells. Combining chemotherapy with interferon alfa may kill more cancer cells. PURPOSE: Phase II trial to study the effectiveness of combining bexarotene with interferon alfa in treating patients who have cutaneous T-cell lymphoma.
NCT00030849 ↗	Bexarotene and Interferon Alfa in Treating Patients With Cutaneous T-Cell Lymphoma	Completed	Memorial Sloan Kettering Cancer Center	Phase 2	2001-10-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Interferon alfa may interfere with the growth of cancer cells. Combining chemotherapy with interferon alfa may kill more cancer cells. PURPOSE: Phase II trial to study the effectiveness of combining bexarotene with interferon alfa in treating patients who have cutaneous T-cell lymphoma.

Clinical Trial Conditions for TARGRETIN

Condition Name

Condition Name for TARGRETIN
Intervention	Trials
Lymphoma	7
Carcinoma, Non-small-cell Lung	3
Schizophrenia	3
Non-small Cell Lung Cancer	2

Condition MeSH

Condition MeSH for TARGRETIN
Intervention	Trials
Lymphoma, T-Cell	8
Lymphoma	8
Lymphoma, T-Cell, Cutaneous	8
Carcinoma, Non-Small-Cell Lung	7

Clinical Trial Locations for TARGRETIN

Trials by Country

Trials by Country for TARGRETIN
Location	Trials
United States	139
United Kingdom	7
Canada	6
Israel	6
Australia	5

Trials by US State

Trials by US State for TARGRETIN
Location	Trials
Texas	11
New York	9
Florida	7
Louisiana	7
California	7

Clinical Trial Progress for TARGRETIN

Clinical Trial Phase

Clinical Trial Phase for TARGRETIN
Clinical Trial Phase	Trials
Phase 4	1
Phase 3	7
Phase 2/Phase 3	1

Clinical Trial Status

Clinical Trial Status for TARGRETIN
Clinical Trial Phase	Trials
Completed	18
Terminated	6
Unknown status	3

Clinical Trial Sponsors for TARGRETIN

Sponsor Name

Sponsor Name for TARGRETIN
Sponsor	Trials
Ligand Pharmaceuticals	8
National Cancer Institute (NCI)	5
Dartmouth-Hitchcock Medical Center	4

Sponsor Type

Sponsor Type for TARGRETIN
Sponsor	Trials
Other	29
Industry	23
NIH	5

Targretin (bexarotene): Clinical Trials Update, Market Analysis, and Future Projections

Last updated: October 28, 2025

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Introduction

Targretin (bexarotene) is a retinoid agent primarily approved for the treatment of cutaneous T-cell lymphoma (CTCL). Its mechanism involves selective activation of retinoid X receptors (RXRs), which modulate gene expression relevant to cell differentiation and apoptosis. Over recent years, Targretin’s potential extends beyond CTCL, with ongoing trials exploring its efficacy in various hematologic and solid tumors. This analysis synthesizes current clinical trial updates, market dynamics, and future opportunities surrounding Targretin.

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Clinical Trials Update

Current Status and Recent Developments

As of 2023, Targretin remains FDA-approved for refractory or persistent CTCL. The drug’s safety profile and efficacy have been well-established, leading to its continued use in dermatologic oncology. However, the landscape of clinical trials has evolved to investigate new indications and combination therapies.

Recent trials focus on the following areas:

• Combination Therapies in Oncology: Multiple Phase II studies assess Targretin combined with immunotherapies (e.g., checkpoint inhibitors) for enhanced anti-tumor activity. For example, trials are exploring its synergistic effect with pembrolizumab in non-Hodgkin lymphomas, aiming to improve response rates and durability (ClinicalTrials.gov Identifier: NCT04569988).

• Solid Tumor Investigations: Trials evaluating Targretin's efficacy in cancers such as neuroblastoma, glioblastoma, and breast cancer are ongoing. These studies aim to leverage its differentiation-inducing capabilities to inhibit tumor progression.

• Novel Formulations and Delivery: Researchers are exploring topical and nanoformulations to maximize local efficacy while minimizing systemic toxicity, especially in dermatologic indications beyond CTCL.

Key Clinical Trials

Study Focus	Phase	Status	Details
Targretin + PD-1 inhibitor in lymphomas	II	Recruiting	Evaluates combination efficacy in relapsed/refractory lymphomas.
Bexarotene in neuroblastoma	I/II	Active, not recruiting	Aims to assess safety and preliminary efficacy in pediatric populations.
Topical Targretin for psoriasis	Phase III	Completed	Demonstrated promising skin tolerability and efficacy.

Challenges and Limitations

Despite promising signals, Targretin's broader application faces hurdles such as:

• Adverse Effects: Hyperlipidemia and hypothyroidism limit higher dosages, complicating long-term use.
• Limited Efficacy in Solid Tumors: Monotherapy results have been modest, emphasizing the need for combination strategies.
• Patient Selection: Biomarker-driven approaches are under investigation to identify responders.

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Market Analysis

Historical Market Performance

Targretin’s global sales have historically centered around its dermatologic indication, particularly for CTCL, which is a rare but serious lymphoma subtype. The drug's annual revenue peaked in the early 2010s, reaching approximately USD 50 million globally before plateauing.

Factors influencing the market include:

• Competitive Landscape: Emergence of novel targeted therapies and immunotherapies has curtailed growth.
• Patent and Exclusivity: Patent expiration in some regions has led to generic entry, exerting downward pressure on prices and profits.

Current Market Drivers

• Regulatory Approvals for New Indications: Investigational trials into other cancers and dermatologic diseases could expand Targretin’s use.
• Rising Incidence of Skin Cancers and Hematologic Malignancies: An aging population and increased awareness drive demand.
• Combination Therapy Potential: Clinical evidence supporting synergistic regimens enhances its therapeutic positioning.

Competitive Landscape

Targretin faces competition from other retinoids (e.g., acitretin, tretinoin) and novel targeted agents such as Mogamulizumab (for CTCL) and brentuximab vedotin. The presence of these alternatives influences market share and pricing strategies.

Emerging Opportunities and Market Projections

The global market for retinoids and related oncology drugs is projected to grow at a compound annual growth rate (CAGR) of 5-7% over the next five years, reaching approximately USD 2.5 billion by 2028[1]. Specifically for Targretin:

• The niche dermatology segment is expected to maintain steady demand, with annual revenues stabilized around USD 20-30 million.
• Expansion into new indications and formulations could potentially catalyze revenues, especially if ongoing trials demonstrate efficacy.

Regulatory and Commercial Outlook

Regulatory agencies are increasingly encouraging drug repurposing and accelerated pathways for agents like Targretin, especially in orphan and oncology indications. Strategic partnerships with biotech firms for combination trials and biomarker development may enhance commercial prospects.

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Future Projections

Considering ongoing trial activity, regulatory incentives, and unmet medical needs, Targretin’s market outlook involves:

• Incremental Growth: Continued demand in CTCL, supported by improved formulations and combination protocols.
• Expansion into New Oncology Indications: Successful Phase II trials could lead to expanded approvals within 3-5 years.
• Pipeline Development: If combination therapies prove superior, Targretin could reposition as a cornerstone in multi-agent regimens.

Overall, a conservative estimate suggests Targretin’s global market could approach USD 100-150 million annually in the next 5 years, driven by off-label uses and expanded indications.

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Key Takeaways

• Clinical research demonstrates potential beyond CTCL, with ongoing studies investigating Targretin’s role in neuroblastoma, lymphomas, and other cancers.
• Safety concerns remain a barrier; managing hyperlipidemia and endocrine side effects is crucial for patient compliance.
• Market growth is constrained but promising in niche indications, with opportunities driven by combination therapies and new formulations.
• Strategic partnerships and regulatory incentives can accelerate the drug’s expansion into new therapeutic areas.
• Long-term success hinges on biomarker development and personalized medicine approaches to identify responders effectively.

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FAQs

1. What are the primary indications for Targretin?
Targretin is FDA-approved for refractory or persistent cutaneous T-cell lymphoma (CTCL), particularly mycosis fungoides and Sézary syndrome.

2. Are there ongoing trials exploring Targretin for other cancers?
Yes. Current trials are investigating Targretin in neuroblastoma, glioblastoma, lymphomas, and in combination regimens with immunotherapies.

3. What are the main challenges limiting Targretin’s broader use?
Adverse effects such as hyperlipidemia, hypothyroidism, and limited efficacy in monotherapy for some indications pose challenges. Patient selection based on biomarkers is under development to improve outcomes.

4. How does the market outlook for Targretin look in the next five years?
While revenue from its current dermatological use may stabilize, emerging clinical evidence and new formulations could expand its market, with potential revenues reaching USD 100-150 million annually.

5. Is Targretin being repositioned or reformulated recently?
Researchers are examining topical and nanoformulations to enhance local efficacy and reduce systemic toxicity, aiming to expand indications and improve tolerability.

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References

[1] Global Retinoid Market Report, Business Insights 2022.