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
Breast Cancer	2

Condition MeSH

Condition MeSH for TARGRETIN
Intervention	Trials
Lymphoma, T-Cell, Cutaneous	8
Lymphoma, T-Cell	8
Lymphoma	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
Israel	6
Canada	6
Australia	5

Trials by US State

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

Last updated: July 28, 2026

Targretin clinical trials update, market analysis, and projection (bexarotene)

Targretin (bexarotene) is an established, oncology-focused retinoid approved for cutaneous T-cell lymphoma (CTCL). Public clinical activity has narrowed to post-approval investigations and label-adjacent studies, with limited late-stage pivotal development visible versus current oncology pipelines. Market growth is constrained by mature penetration, specialty-channel competition, payer controls for high-cost oral oncology, and the long-tail nature of CTCL incidence. Near-term sales projections track underlying CTCL treatment demand and the company’s ability to defend pricing and formulary position, not a new wave of late-stage approvals.

Targretin (bexarotene) clinical trials update: what studies are still active and what endpoints matter?

Answer: Current trial visibility for Targretin is mostly post-approval, smaller cohorts, and combination or maintenance-style designs rather than large, definitive Phase 3 programs. The highest relevance trials for market impact are those that expand use beyond CTCL-only settings, improve response durability, or demonstrate clear differentiation versus contemporary CTCL regimens.

Which indications dominate Targretin trial activity (CTCL vs off-label oncology)?

• Primary labeled indication: CTCL (skin-directed and systemic treatment contexts in different approvals and schedules).
• Trial themes that most affect adoption:
  • Combination regimens in CTCL to improve overall response rate (ORR) or progression-free survival (PFS).
  • Biomarker-enriched studies to identify responders and reduce discontinuation.
  • Real-world effectiveness and tolerability studies to support payer coverage.

What endpoints most influence payer and guideline uptake for Targretin in ongoing studies?

• ORR and durable response rate (especially in skin manifestations where CTCL response drives continuation).
• PFS in systemic strategies.
• Time to treatment failure for combination settings.
• Safety/tolerability profiles that reduce dose interruptions, which directly impact adherence and real-world persistence.

Where combination therapy trials are most likely to move the market needle

Combination trials are the most probable vehicle for incremental uptake because they can:

• Target patients resistant to retinoid monotherapy.
• Provide a rationale for sequencing with newer CTCL options.
• Offer a differentiator on tolerability or response depth.

What to watch in trial readouts that change commercialization

• Any signal of improved response durability (durable CR/PR) compared with historical retinoid outcomes.
• Evidence that lower dosing schedules retain efficacy while reducing hallmark retinoid toxicities (lipids, thyroid effects).
• Expansion data that sustains or broadens systemic use patterns within CTCL.

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Targretin market analysis: who buys it, where does it sell, and what drives demand?

Answer: Targretin demand is concentrated in specialty oncology prescribing for CTCL patients, with sales determined by CTCL incidence, line-of-therapy patterns, payer authorization dynamics, and clinician comfort with retinoid toxicity management.

Demand drivers

• CTCL epidemiology and treatment pathways
  • CTCL is rare; patient counts drive revenue ceilings even when utilization is high.
  • Relapsed/refractory patients and those needing systemic options anchor long-term demand.
• Treatment sequencing
  • Retinoids can be used as systemic therapy in particular lines, but adoption depends on alternatives (e.g., newer agents, skin-directed therapies).
• Adherence and dose management
  • Retinoid-related lab monitoring and endocrine/lipid management affect persistence.

Pricing and payer controls

• High-cost oral oncology products face formulary tiering and prior authorization.
• Targretin’s ability to sustain volume depends on:
  • Formulary access.
  • Prescriber satisfaction with toxicity management protocols.
  • Competitive pressure from newer CTCL agents.

Competitive landscape in CTCL

Answer: The CTCL market is characterized by multi-agent options across skin-directed and systemic categories. Targretin competes on:

• Oral systemic delivery (convenience).
• Established retinoid efficacy history.
• Clinician familiarity and known lab-management requirements.

Key competitive pressures include:

• Newer systemic CTCL drugs that may shift sequencing earlier.
• Combination regimens that relegate retinoids to later lines unless superior efficacy or tolerability is shown in comparative data.

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How does Targretin compare with other CTCL oral and systemic therapies on value and usability?

Answer: Targretin’s differentiator is oral delivery and a known efficacy profile, but the value case is constrained by retinoid toxicities and the need for routine lipid/thyroid monitoring. Competitors with easier monitoring or more durable response data can gain earlier-line share if clinical guidance shifts.

What matters most to clinicians choosing among CTCL systemic options

• Response rate in skin and systemic disease.
• Treatment tolerability and frequency of lab monitoring.
• Evidence supporting sequencing in relapsed/refractory CTCL.
• Patient convenience and adherence.

What matters most to payers

• Cost per patient treated.
• Time on therapy (durability and discontinuation rates).
• Real-world evidence on efficacy and safety manageability.

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When does Targretin lose exclusivity and how does that affect the generic or biosimilar risk?

Answer: Targretin is a small-molecule retinoid (bexarotene). Generic and/or authorized alternatives are a central risk to revenue if exclusivity is exhausted and ANDA entry is feasible. However, whether that translates into material sales erosion depends on the presence of market-authorized generics, distribution channel switching costs, and formulary inertia.

Small-molecule reality check

• For small molecules like bexarotene, generics typically carry the main “exclusivity to entry” risk profile rather than biosimilar dynamics.

Key commercial implication

• Generic entry usually drives:
  • Revenue decline through price compression.
  • Lower gross-to-net ratio improvements.
  • Increased payer adoption but potentially reduced total profitability for remaining branded supply.

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What is the Orange Book status of Targretin and what patents cover it?

Answer: Orange Book status determines whether branded protections are still listed for bexarotene and which patent numbers block generic substitution. Patent barriers are commercially relevant because they control timing for ANDA approvals and “launch at risk” behavior.

Patent estate factors that change ANDA entry risk

• Expiration dates for active composition-of-matter and formulation patents.
• Method-of-use patent scope tied to CTCL dosing regimens and clinical outcomes.
• Exclusivity listings linked to FDA approval pathways.

(This section is intentionally data-light because no Orange Book dataset or patent list was provided in the source material available in this chat.)

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Targretin patent and litigation landscape: which challenges matter for market projections?

Answer: For branded oncology small molecules, litigation affects timing and certainty of generic entry. The main litigation question is whether Paragraph IV filings or settlement agreements lock in delayed entry.

What to track

• Paragraph IV ANDA filings referencing bexarotene.
• Delaware litigation (if present) and settlement terms.
• Court rulings that narrow claim coverage.

(This section is intentionally non-enumerative because no litigation docket inputs were provided in the source material available in this chat.)

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FDA regulatory status update for Targretin: what changes the label or clinical use?

Answer: For market impact, changes to:

• Indication scope,
• Dosing recommendations,
• Safety warnings,
• Monitoring requirements, are the highest-value FDA events.

What to watch

• Label updates that affect tolerability management.
• Risk communications that influence prescribing behavior.
• Any new REMS-like or monitoring requirements (rare for this product class but label-driven monitoring changes can still matter).

(This section is intentionally non-specific because no FDA label update timeline was provided.)

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Market projection for Targretin through 2028: base case, drivers, and downside risks

Answer: Targretin market trajectory is most likely to remain stable-to-declining in a mature specialty oncology market unless:

1. it secures a meaningful new clinical differentiation that changes sequencing, or
2. generic entry does not accelerate beyond current competition.

Base case projection logic (driver-based)

• CTCL patient demand stays steady but is structurally limited by incidence.
• Line-of-therapy competition caps share growth.
• Specialty pharmacy and payer management limit rapid volume expansion.
• Price erosion depends on generic availability and formulary bargaining outcomes.

Key upside scenarios

• Demonstrated improved durability or simplified toxicity management from follow-on clinical evidence supporting stronger guideline inclusion.
• Expansion into more systemic CTCL lines (earlier sequencing) if new readouts demonstrate clear benefit.

Key downside scenarios

• Faster-than-expected price compression from generic or authorized generic adoption.
• Increased competitor share shift if newer CTCL therapies show superior outcomes or simpler administration.

Projection range (directional)

• Most likely direction: modest decline to low-growth, driven by maturity and competitive pricing pressure.
• Magnitude: largely determined by generic availability and gross-to-net compression rather than trial-driven demand shocks.

(No quantitative revenue baseline, geography, or unit data was provided in the source material available in this chat, so no numeric forecast can be produced without fabricating facts.)

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Key Takeaways

• Targretin (bexarotene) is an established CTCL therapy with clinical trial activity that is likely post-approval and combination-focused rather than transformative Phase 3.
• Market demand is capped by CTCL rarity and driven by treatment sequencing, payer access, and lab-monitoring manageability.
• The dominant revenue swing factor is pricing and competitive entry, not a large new label expansion.
• Patent and Orange Book status determine ANDA entry risk timing; litigation can delay entry but requires dataset-backed confirmation.

FAQs

1. What CTCL lines of therapy does Targretin typically occupy and how does that affect sales?
2. Do combination regimens with bexarotene improve PFS or only ORR in CTCL trials?
3. How does bexarotene’s lipid and thyroid monitoring burden influence real-world persistence?
4. What Paragraph IV or ANDA events most influence generic entry timing for bexarotene?
5. What label or safety updates for Targretin have historically changed prescribing behavior?

References

(No external sources were provided in the prompt or available within this chat to cite.)