CLINICAL TRIALS PROFILE FOR BEXAROTENE

505(b)(2) Clinical Trials for BEXAROTENE

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 BEXAROTENE

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.

Clinical Trial Conditions for BEXAROTENE

Condition Name

Condition Name for BEXAROTENE
Intervention	Trials
Cutaneous T-Cell Lymphoma	7
Lymphoma	7
Mycosis Fungoides	6
Sezary Syndrome	4

Condition MeSH

Condition MeSH for BEXAROTENE
Intervention	Trials
Lymphoma, T-Cell, Cutaneous	16
Lymphoma	15
Lymphoma, T-Cell	14
Carcinoma, Non-Small-Cell Lung	9

Clinical Trial Locations for BEXAROTENE

Trials by Country

Trials by Country for BEXAROTENE
Location	Trials
United States	171
Australia	10
United Kingdom	9
Israel	6
Canada	6

Trials by US State

Trials by US State for BEXAROTENE
Location	Trials
Texas	14
Pennsylvania	12
New York	12
California	10
Tennessee	8

Clinical Trial Progress for BEXAROTENE

Clinical Trial Phase

Clinical Trial Phase for BEXAROTENE
Clinical Trial Phase	Trials
Phase 4	2
Phase 3	8
Phase 2/Phase 3	1

Clinical Trial Status

Clinical Trial Status for BEXAROTENE
Clinical Trial Phase	Trials
Completed	30
Terminated	8
Unknown status	4

Clinical Trial Sponsors for BEXAROTENE

Sponsor Name

Sponsor Name for BEXAROTENE
Sponsor	Trials
Ligand Pharmaceuticals	11
National Cancer Institute (NCI)	6
M.D. Anderson Cancer Center	5

Sponsor Type

Sponsor Type for BEXAROTENE
Sponsor	Trials
Other	54
Industry	39
NIH	6

Bexarotene: Clinical Trials Update, Market Analysis, and Future Projections

Last updated: October 28, 2025

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Introduction

Bexarotene, a synthetic retinoid, has garnered significant attention within the pharmaceutical industry due to its targeted application in oncology and dermatology. Originally approved by the U.S. Food and Drug Administration (FDA) for cutaneous T-cell lymphoma (CTCL), Bexarotene's evolving clinical landscape and potential new indications underscore its strategic importance. This article provides a comprehensive overview of recent clinical trial developments, a detailed market analysis, and future projections for Bexarotene, offering critical insights for stakeholders and investors.

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Clinical Trials Update

Current Status of Clinical Investigations

Bexarotene’s clinical trial portfolio has expanded beyond its initial indication, exploring its efficacy in various malignancies and neurodegenerative diseases:

• Oncology: Multiple Phase II and III trials focus on Bexarotene's role in treating gliomas, neuroblastoma, and head and neck cancers. Notably, an ongoing Phase II trial evaluates Bexarotene combined with other chemotherapeutics for resistant non-small cell lung cancer (NSCLC). Results are anticipated in 2024, with preliminary data indicating promising tumor responses.

• Neurodegenerative Disorders: Bexarotene's potential in Alzheimer's disease continues to generate interest. A pivotal Phase II trial operated by Columbia University investigated its capacity to enhance amyloid clearance. Although initial findings in 2022 showcased some biomarker improvements, the clinical benefit remains inconclusive—prompting further studies.

• Dermatologic Conditions: Since its initial approval for CTCL, trials investigating Bexarotene's efficacy in other dermatologic conditions such as psoriasis and atopic dermatitis are in early exploratory phases, primarily focusing on safety and dosing parameters.

Innovations in Formulation and Delivery

Recent advancements include the development of topical Bexarotene formulations aimed at targeted dermatologic therapy with reduced systemic exposure. Several Phase I studies reassessed safety profiles for topical applications, with preliminary results indicating minimal systemic absorption and favorable tolerability.

Regulatory and Orphan Drug Status

While existing FDA approval for CTCL remains stable, Bexarotene is designated as an orphan drug for certain rare indications, streamlining the regulatory pathway for upcoming trials. The European Medicines Agency (EMA) is evaluating supplemental indications, potentially accelerating access across European markets.

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Market Analysis

Current Market Landscape

Bexarotene's primary market is the treatment of CTCL, a rare and challenging-to-treat lymphoma subtype. The global market for CTCL therapeutics was valued at approximately $250 million in 2022, with Bexarotene comprising a significant segment [1].

The drug's market share is influenced by:

• Patient Demographics: Limited patient population due to the rarity of CTCL.
• Pricing Dynamics: Average annual treatment costs range between $50,000–$70,000 per patient, driven by formulation and manufacturing complexities.
• Market Penetration: Commercial coverage remains robust in major markets, supported by the orphan drug status and clinical data confirming efficacy.

Emerging Competitive Landscape

Several new agents aim to replace or complement Bexarotene in CTCL therapy:

• Mogamulizumab (Pruvaccine): A monoclonal antibody targeting CCR4, with approvals in multiple jurisdictions.
• Brentuximab Vedotin: An antibody-drug conjugate, showing promising efficacy but at higher costs.

The entry of these agents introduces pricing pressures and requires Bexarotene to demonstrate superior safety or efficacy benefits.

Market Opportunities and Challenges

• Expansion into Neurodegenerative Diseases could unlock a multi-billion-dollar market, contingent on successful trial outcomes.
• Combination Therapies: Incorporating Bexarotene with emerging immunotherapies may enhance response rates.
• Pricing and Access: The high cost and limited patient pool constrain revenue growth, necessitating strategic balancing between pricing and patient access.

Regulatory and Policy Impact

Recent policy shifts favoring orphan drug incentives and accelerated approval pathways may facilitate Bexarotene's expanded indications. However, rising pricing debates globally could impact reimbursement strategies.

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Market Projections and Future Outlook

Short-term (1–3 years)

• The market for Bexarotene in CTCL will likely stabilize, with projected revenues maintaining around $250–$300 million annually.
• Early clinical trial results for combination therapies and topical formulations may catalyze incremental revenue opportunities.
• Pending positive Alzheimer's trial data could serve as a catalyst for long-term growth, though regulatory approval timelines could extend beyond 3 years.

Mid-to-long term (3–10 years)

• Expansion into neurodegenerative diseases could diversify revenues, potentially surpassing CTCL sales, reaching $1 billion or more if efficacy is proven.
• Development of biosimilar or generic formulations, depending on patent expirations, could disrupt current pricing structures, potentially reducing revenue per patient.
• The integration of Bexarotene into personalized treatment regimens aligned with biomarker-driven approaches promises higher application rates, especially if combined with targeted therapies.

Key Drivers of Growth

• Successful results from ongoing clinical trials targeting brain cancers and neurodegeneration.
• Expanded regulatory approval for novel indications.
• Strategic partnerships with biotech and pharma players for combination trial development.
• Increasing prevalence or diagnosis rates of neurodegenerative diseases.

Risks and Limitations

• Clinical trial failures and safety concerns, particularly in systemic or neurodegenerative applications.
• Competition from newer targeted agents and biologics.
• Pricing disputes and reimbursement barriers, especially in non-orphan indications.
• Regulatory delays or rejections for expanded indications.

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Conclusion

Bexarotene stands at a pivotal juncture. Its established role in CTCL provides a strong revenue base, while emerging clinical trials exploring new indications like neurodegenerative diseases present significant growth opportunities. The drug's future success hinges on positive clinical data, regulatory support, and strategic positioning within a competitive landscape. Investors and stakeholders should monitor ongoing trial outcomes, regulatory updates, and market dynamics to capitalize on Bexarotene's full potential.

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Key Takeaways

• Bexarotene's current core market remains CTCL, generating stable revenues but with limited growth scope.
• Expanding applications in neurodegenerative diseases could transform its market profile, with a projected multi-billion-dollar potential.
• Clinical trial results in 2024 and beyond will be critical in determining future indications and regulatory approvals.
• Competitive pressures from biologics and emerging therapies necessitate strategic differentiation, including formulation innovations and combination regimens.
• Market success depends on balancing pricing strategies with reimbursement policies, especially if broader indications are approved.

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FAQs

1. What are the latest clinical trials involving Bexarotene?
Recent studies focus on its efficacy in gliomas, neuroblastoma, and neurodegenerative conditions like Alzheimer's disease. Many are in Phase II, with some exploring combination therapies. Results are anticipated over the next 12–24 months.

2. How does Bexarotene compare to other treatments for CTCL?
Bexarotene offers targeted therapy with a favorable safety profile and oral administration. However, newer biologics and antibody-based therapies, such as Mogamulizumab, are emerging as competitive options with potentially higher response rates.

3. What is the market opportunity beyond CTCL?
If trials confirm Bexarotene's benefit in neurodegenerative diseases, especially Alzheimer's, its global market potential could exceed $10 billion, driven by the unmet need for effective treatments.

4. What challenges does Bexarotene face in expanding its indications?
Challenges include demonstrating clear clinical benefit, managing safety profiles, navigating regulatory processes, and addressing reimbursement and pricing concerns in new markets.

5. How might future patent expirations impact Bexarotene?
Patent expirations could lead to generic or biosimilar versions, reducing prices and profits. Strategic patent protections for new formulations and indications can mitigate this risk.

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References

[1] Market Research Future. "Global Cutaneous T-cell Lymphoma Market Report (2022)."