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.
NCT00050960 ↗	Evaluation of Efficacy, Safety and Tolerability of Targretin Capsules in Patients With Advanced or Metastatic Non-small Cell Lung Cancer	Completed	Eisai Inc.	Phase 3	2002-05-01	This study evaluates the use of Targretin capsules (bexarotene) in combination with standard chemotherapy for the treatment of metastatic Non-Small Cell Lung Cancer (NSCLC) in patients who have not yet received chemotherapy for their lung cancer.

Clinical Trial Conditions for Targretin

Condition Name

Condition Name for Targretin
Intervention	Trials
Lymphoma	7
Carcinoma, Non-small-cell Lung	3
Schizophrenia	3
Mycosis Fungoides	2

Condition MeSH

Condition MeSH for Targretin
Intervention	Trials
Lymphoma	8
Lymphoma, T-Cell, Cutaneous	8
Lymphoma, T-Cell	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: Clinical Trials, Market Analysis, and Projections

Introduction

Targretin, also known as bexarotene, is a retinoid X receptor (RXR)-selective agonist used primarily for the treatment of cutaneous manifestations of cutaneous T-cell lymphoma (CTCL). Here, we will delve into the clinical trials, market analysis, and future projections for this drug.

Clinical Trials Overview

Efficacy and Safety

Clinical trials for Targretin have been conducted to evaluate its efficacy and safety in patients with CTCL. Two major studies involving 193 patients who had received at least two previous treatments showed promising results. Approximately half of the patients treated with the starting dose of 300 mg/m²/day responded to the treatment, as assessed by the doctor’s grading and clinical skin scores[1][4].

In a phase 2 and 3 clinical trial, bexarotene was administered to 58 patients with biopsy-proven stage IA through IIA CTCL who were refractory to or intolerant of previous treatments. The trial demonstrated that bexarotene was well-tolerated and effective, with 54% of patients achieving a clinical complete response or partial remission at doses of 300 mg/m²/day[4].

Side Effects and Monitoring

While Targretin has shown efficacy, it also comes with side effects that require monitoring. Hypertriglyceridemia and hypothyroidism are notable side effects, but these are reversible and manageable with concomitant medication[4].

Market Analysis

Current Market Status

Targretin has been approved by the US FDA since 1999 with orphan designation and is currently available in 36 countries or regions, including recent approvals in Hong Kong and Macau. It is recommended in major treatment guidelines such as those from the National Comprehensive Cancer Network (NCCN) and the European Organisation for Research and Treatment of Cancer (EORTC)[5].

Regional Expansion

The marketing authorization in Hong Kong and Macau marks the first commercial availability of Targretin in China. This expansion is part of a broader strategy by Main Life Corp., Ltd., in collaboration with Minophagen Pharmaceutical Co., Ltd., to develop and commercialize the drug in Asia[5].

Market Projections

Global Cancer Market Trends

The global cancer market, particularly for targeted therapies, is expected to grow significantly. While Targretin is not as widely used as some other targeted therapies like Tagrisso (osimertinib) or Avastin (bevacizumab), its niche in treating CTCL ensures a stable market presence. The overall non-small cell lung cancer (NSCLC) market, which includes other targeted therapies, is projected to reach $26.8 billion by 2025, with targeted therapies contributing significantly to this growth[2].

Specific Projections for Targretin

Although specific financial projections for Targretin are not as detailed as those for more widely used drugs, its approval and marketing in new regions suggest a potential for increased sales. The drug's status as a standard-of-care for refractory CTCL ensures a consistent demand, particularly in regions where it has recently been approved.

Unmet Needs and Future Directions

Clinical Trials and Innovation

The clinical trials market, driven by factors such as the COVID-19 pandemic, technological evolution, and the rise of personalized medicine, is expected to grow significantly. The global clinical trials market is projected to increase from $81.90 billion in 2023 to $153.59 billion by 2033, with a CAGR of 6.49% from 2024 to 2033. This growth could lead to more innovative treatments and potentially new indications for Targretin or similar drugs[3].

Patient Quality of Life

Improving the quality of life for patients with CTCL remains a key focus. Targretin, with its selective binding to retinoid X receptors and its ability to induce apoptosis and cell cycle arrest, offers a valuable treatment option. Continued research and expansion into new markets will help more patients access this treatment, enhancing their quality of life[5].

Key Takeaways

• Clinical Efficacy: Targretin has shown significant efficacy in treating CTCL, with response rates of around 50% in clinical trials.
• Market Expansion: Recent approvals in Hong Kong and Macau mark the drug's entry into the Chinese market, expanding its global reach.
• Side Effects Management: Hypertriglyceridemia and hypothyroidism are manageable side effects that require monitoring.
• Market Trends: The global cancer market, particularly for targeted therapies, is expected to grow, ensuring a stable market presence for Targretin.
• Future Directions: Continued innovation in clinical trials and expansion into new markets will be crucial for the drug's future success.

FAQs

What is Targretin used for?

Targretin, or bexarotene, is used for the treatment of cutaneous manifestations of cutaneous T-cell lymphoma (CTCL) in patients who are refractory to at least one prior systemic therapy.

How effective is Targretin in clinical trials?

In clinical trials, Targretin has shown response rates of around 50% in patients treated with the starting dose of 300 mg/m²/day, as assessed by doctor’s grading and clinical skin scores.

What are the common side effects of Targretin?

Common side effects include hypertriglyceridemia and hypothyroidism, which are reversible and manageable with concomitant medication.

Where is Targretin currently available?

Targretin is currently available in 36 countries or regions, including recent approvals in Hong Kong and Macau, marking its first commercial availability in China.

What is the projected growth of the clinical trials market?

The global clinical trials market is projected to increase from $81.90 billion in 2023 to $153.59 billion by 2033, with a CAGR of 6.49% from 2024 to 2033.

How does Targretin work?

Targretin works by selectively binding to retinoid X receptors, inducing apoptosis and cell cycle arrest, which helps in suppressing tumor growth.

Sources

1. European Medicines Agency (EMA). Targretin | European Medicines Agency (EMA).
2. Drug Development. NSCLC MARKET - Global Drug Forecast & Market Analysis to 2025.
3. BioSpace. Clinical Trials Market Size to Increase USD 153.59 Billion by 2033.
4. JAMA Network. Phase 2 and 3 Clinical Trial of Oral Bexarotene (Targretin Capsules ...
5. BioSpace. Main Life received Targretin® (Bexarotene) Marketing Authorisation ...