CLINICAL TRIALS PROFILE FOR VESANOID

All Clinical Trials for Vesanoid

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00005825 ↗	Combination Chemotherapy in Treating Patients With Stage IIIB or Stage IV Non-small Cell Lung Cancer	Unknown status	Raghu Nandan, M.D., Inc	Phase 2	1998-09-01	RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining more than one drug may kill more tumor cells. PURPOSE: Phase II trial to study the effectiveness of combination chemotherapy in treating patients who have stage IIIB or stage IV non-small cell lung cancer.
NCT00006220 ↗	Arsenic Trioxide With or Without Tretinoin in Treating Patients With Hematologic Cancer That Has Not Responded to Previous Therapy	Terminated	National Cancer Institute (NCI)	Phase 1/Phase 2	1999-06-01	RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Tretinoin may help hematologic cancer cells develop into normal white blood cells. PURPOSE: Phase I/II trial to study the effectiveness of arsenic trioxide with or without tretinoin in treating patients who have hematologic cancer that has not responded to previous therapy.
NCT00006220 ↗	Arsenic Trioxide With or Without Tretinoin in Treating Patients With Hematologic Cancer That Has Not Responded to Previous Therapy	Terminated	Washington University School of Medicine	Phase 1/Phase 2	1999-06-01	RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Tretinoin may help hematologic cancer cells develop into normal white blood cells. PURPOSE: Phase I/II trial to study the effectiveness of arsenic trioxide with or without tretinoin in treating patients who have hematologic cancer that has not responded to previous therapy.
NCT00326170 ↗	Phase II 5-Azacytidine Plus VPA Plus ATRA	Completed	Celgene Corporation	Phase 2	2005-07-01	5-aza is a chemotherapy drug with activity in leukemia and myelodysplastic syndromes (MDS). Researchers hope that valproic acid (VPA) and all-trans retinoic acid (ATRA)will increase the effects of 5-aza. The goal of this clinical research study is to find the highest safe dose of valproic acid (VPA) that can be given in combination with 5-azacytidine (5-aza) and all-trans retinoic acid (ATRA) in the treatment of AML and MDS. The safety and effectiveness of this combination therapy will also be studied. Additional blood and bone marrow samples will be requested. These samples will be used to evaluate the effect of the treatment on leukemic cells. In addition, any leftover blood and bone marrow samples that are collected at the start of the study and during the regularly scheduled evaluations to be sent for research studies. The research studies will examine changes in the blood and bone marrow cells that might help explain the causes of leukemia and MDS and how the combination of 5-aza, VPA, and ATRA works.
NCT00326170 ↗	Phase II 5-Azacytidine Plus VPA Plus ATRA	Completed	M.D. Anderson Cancer Center	Phase 2	2005-07-01	5-aza is a chemotherapy drug with activity in leukemia and myelodysplastic syndromes (MDS). Researchers hope that valproic acid (VPA) and all-trans retinoic acid (ATRA)will increase the effects of 5-aza. The goal of this clinical research study is to find the highest safe dose of valproic acid (VPA) that can be given in combination with 5-azacytidine (5-aza) and all-trans retinoic acid (ATRA) in the treatment of AML and MDS. The safety and effectiveness of this combination therapy will also be studied. Additional blood and bone marrow samples will be requested. These samples will be used to evaluate the effect of the treatment on leukemic cells. In addition, any leftover blood and bone marrow samples that are collected at the start of the study and during the regularly scheduled evaluations to be sent for research studies. The research studies will examine changes in the blood and bone marrow cells that might help explain the causes of leukemia and MDS and how the combination of 5-aza, VPA, and ATRA works.

Clinical Trial Conditions for Vesanoid

Condition Name

Condition Name for Vesanoid
Intervention	Trials
Acute Myelogenous Leukemia	3
Myelodysplastic Syndrome	3
Acute Myeloid Leukemia	2
Acute Promyelocytic Leukemia With PML-RARA	2

Condition MeSH

Condition MeSH for Vesanoid
Intervention	Trials
Leukemia	10
Leukemia, Myeloid, Acute	7
Myelodysplastic Syndromes	6
Preleukemia	6

Clinical Trial Locations for Vesanoid

Trials by Country

Trials by Country for Vesanoid
Location	Trials
United States	69
Canada	6
Australia	6
France	2
New Zealand	2

Trials by US State

Trials by US State for Vesanoid
Location	Trials
Texas	6
California	3
Colorado	3
Ohio	3
Oregon	3

Clinical Trial Progress for Vesanoid

Clinical Trial Phase

Clinical Trial Phase for Vesanoid
Clinical Trial Phase	Trials
PHASE3	1
Phase 3	2
Phase 2	10

Clinical Trial Status

Clinical Trial Status for Vesanoid
Clinical Trial Phase	Trials
Completed	8
Recruiting	6
Active, not recruiting	3

Clinical Trial Sponsors for Vesanoid

Sponsor Name

Sponsor Name for Vesanoid
Sponsor	Trials
Celgene Corporation	4
M.D. Anderson Cancer Center	4
National Cancer Institute (NCI)	3

Sponsor Type

Sponsor Type for Vesanoid
Sponsor	Trials
Other	37
Industry	9
NIH	4

Vesanoid Market Analysis and Financial Projection

Last updated: April 26, 2026

What is the latest clinical and market outlook for VESANOID (tretinoin) and what projections matter to investors?

Clinical status: what trials are actively shaping VESANOID’s evidence base?

VESANOID is tretinoin (all-trans retinoic acid, ATRA) for acute promyelocytic leukemia (APL). Its current clinical footprint is defined by (1) established efficacy in APL and (2) regimen evolution (ATRA plus anthracycline, and ATRA plus arsenic-based approaches). Post-approval development has largely taken the form of regimen optimization and real-world evidence rather than new pivotal monotherapy phase 3 programs.

Evidence anchor in APL

The clinical use of VESANOID in APL rests on ATRA’s ability to induce differentiation of promyelocytes. The standard-of-care logic is anchored by long-established trials from the 1990s, with subsequent randomized comparisons shaping combination positioning across risk strata.

Key landmark evidence (high-level):

• ATRA-based regimens in newly diagnosed APL have shown complete remission induction and improved outcomes versus historical controls, with differentiation syndrome as a known risk-management focus.
• ATRA plus anthracycline has been a foundational regimen design.
• ATRA plus arsenic trioxide (ATO) has gained broad adoption and is now a major reference standard in many settings, with regimen selection influenced by risk category and regional practice.

Publicly documented regulatory framing (package labeling):

• VESANOID’s labeled indication is treatment of acute promyelocytic leukemia (APL) in conjunction with appropriate chemotherapy and is guided by APL-specific safety and monitoring instructions, including differentiation syndrome risk management.
  Source: VESANOID prescribing information [1].

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Safety and regimen considerations: what dosing and risk controls drive clinical adoption?

Clinical trial activity for APL regimens is inseparable from safety protocol implementation. VESANOID labeling and standard APL practice define the key operational requirements.

Key labeled safety elements in APL

• Differentiation syndrome: requires prompt recognition and management.
• Monitoring and supportive care: reflect APL’s complication profile (coagulopathy, leukocytosis, cytopenias).
• Drug interactions: retinoids can have interaction patterns via metabolism and supportive medication use in induction regimens.

Source: VESANOID prescribing information [1].

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Market analysis: how does the VESANOID opportunity look by demand drivers and pricing reality?

VESANOID is an older, established oncology product with patent-driven dynamics largely replaced by generic availability in most mature markets and by historical purchasing patterns tied to APL treatment guidelines. For market modeling, the decisive factors are not “launch growth” but incumbent retention, generic share, and hospital regimen choice.

Demand drivers specific to APL

Market demand is driven by:

• Incidence of APL (a subset of AML).
• Treatment standard selection (ATRA-based induction).
• Speed of access (formulary decisions, supply reliability, and relative cost vs alternatives).
• Regional regimen preference (ATRA plus anthracycline vs ATRA plus ATO).

Competitive landscape

For tretinoin in APL, “competition” is mainly about:

• Generic tretinoin versus branded VESANOID in pricing terms.
• Alternative APL induction strategies centered on ATO-based regimens that still use ATRA as a core component.
• Hospital formularies that treat ATRA as a standard backbone drug rather than a differentiated biologic or targeted therapy.

Because ATO-based regimens often still require ATRA, tretinoin competes more on cost and supply than on clinical differentiation.

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Projection model: what is the realistic market path for VESANOID over the next 3 to 5 years?

Without a brand-level revenue series and without a current, explicit “tretinoin brand sales” dataset in the provided source corpus, the only defensible projection is scenario logic grounded in structurally stable drivers: APL incidence stability, guideline persistence of ATRA-containing regimens, and cost compression from generics.

Projection logic (scenario constraints)

1. Therapy persistence: APL treatment guidelines continue to use ATRA-based regimens in most regions, limiting demand destruction.
2. Pricing pressure: generics reduce branded share and compress margins unless supply constraints or formulary stickiness protect the brand.
3. Utilization stability: APL is rare; volumes do not scale quickly even if the standard shifts.
4. Regimen mix shifts: if ATO-based regimens expand, total ATRA usage can remain stable because ATRA is still included, so brand impact depends on which ATRA product form wins formulary.

Bottom line: the realistic outlook for VESANOID is low growth with possible share erosion where generic coverage is strong, with revenue tracking more to formulary mix than to new-patient growth.

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What should investors and R&D teams watch next for VESANOID (tretinoin) in 2024 to 2026?

Even if the product itself is mature, investor-relevant signals exist in the APL regimen ecosystem:

1) ATO-based regimen share versus ATRA plus anthracycline

Shift toward ATO-including regimens tends to preserve ATRA demand but can alter which ATRA form is preferred on hospital budgets and procurement contracts.

2) Safety management protocols and differentiation syndrome incidence management

Sites that standardize differentiation syndrome monitoring can reduce early discontinuations and support consistent regimen completion. That matters for adoption metrics and treatment success rates.

3) Supply continuity and pricing

Generic penetration and tender cycles can swing branded uptake. Supply disruptions and pricing changes can briefly change brand share without any new science.

Sources: VESANOID prescribing information [1].

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Key Takeaways

• VESANOID (tretinoin) is a mature, APL-specific ATRA backbone where clinical evidence and regulatory labeling focus on differentiation syndrome risk management and use in combination with APL-standard chemotherapy. [1]
• Market demand is structurally tied to APL incidence and persistence of ATRA-containing induction regimens; growth is constrained by rarity of APL and by generic pricing pressure.
• Projections should model stability with margin compression and potential branded share erosion, not brand-like growth from new indications or phase 3 pivots, because the development path is regimen optimization rather than brand-new endpoints.

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FAQs

1) What is VESANOID’s approved role in APL therapy?

VESANOID is used for acute promyelocytic leukemia (APL) as ATRA and is indicated in conjunction with appropriate chemotherapy per prescribing information. [1]

2) What is the most operationally important safety risk with VESANOID?

Differentiation syndrome is the key APL-specific risk that requires prompt recognition and management. [1]

3) Does ATO replace VESANOID in modern APL regimens?

ATRA remains part of widely used ATO-containing approaches; ATO can change regimen mix but does not remove the need for ATRA in standard practice. (Clinical practice evolution reflected in long-standing APL regimen framework; prescribing info supports ATRA-based combination use.) [1]

4) Why is branded VESANOID growth limited even if APL therapy remains ATRA-based?

Generic tretinoin competition compresses pricing and typically drives brand share erosion, making growth more about formulary and procurement cycles than about new clinical expansion.

5) What type of “trial activity” should be expected for VESANOID at this stage?

At this stage, most activity centers on regimen optimization, risk stratification, and supportive care protocols rather than new monotherapy pivotal phase programs.

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References

[1] VESANOID (tretinoin) prescribing information. (n.d.). Accessed from FDA label text on the Drugs@FDA platform.