CLINICAL TRIALS PROFILE FOR VINCASAR PFS

505(b)(2) Clinical Trials for VINCASAR PFS

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
OTC	NCT03742258 ↗	Combination Chemotherapy and TAK-659 as Front-Line Treatment in Treating Patients With High-Risk Diffuse Large B Cell Lymphoma	Active, not recruiting	National Cancer Institute (NCI)	Phase 1	2019-03-13	The purpose of this research study is to evaluate a new investigational drug, TAK-659, given in combination with standard chemotherapy, for the treatment of Diffuse Large B-cell Lymphoma (DLBCL). ?Investigational? means that TAK-659 has not been approved by the United States Food and Drug Administration (FDA) for use as a prescription or over-the-counter medication to treat a certain condition. The primary purpose of this study is to find the appropriate and safe dose of the study drug to be used in combination with standard chemotherapy for the treatment of your disease and to determine how well the drug works in treating the disease. Other objectives include measuring the amount of the study drug in the body at different times after taking the study drug. Participation in the study is expected to last for up to 3 years after receiving the last dose of the study drug. Patients will receive the study treatment for up to 18 weeks, as long as they are benefitting.
OTC	NCT03742258 ↗	Combination Chemotherapy and TAK-659 as Front-Line Treatment in Treating Patients With High-Risk Diffuse Large B Cell Lymphoma	Active, not recruiting	Northwestern University	Phase 1	2019-03-13	The purpose of this research study is to evaluate a new investigational drug, TAK-659, given in combination with standard chemotherapy, for the treatment of Diffuse Large B-cell Lymphoma (DLBCL). ?Investigational? means that TAK-659 has not been approved by the United States Food and Drug Administration (FDA) for use as a prescription or over-the-counter medication to treat a certain condition. The primary purpose of this study is to find the appropriate and safe dose of the study drug to be used in combination with standard chemotherapy for the treatment of your disease and to determine how well the drug works in treating the disease. Other objectives include measuring the amount of the study drug in the body at different times after taking the study drug. Participation in the study is expected to last for up to 3 years after receiving the last dose of the study drug. Patients will receive the study treatment for up to 18 weeks, as long as they are benefitting.

All Clinical Trials for VINCASAR PFS

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00004010 ↗	Combination Chemotherapy and Radiation Therapy in Treating Children With Previously Untreated Stage II, Stage III, or Stage IV Hodgkin's Disease	Completed	National Cancer Institute (NCI)	Phase 2	1999-10-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining more than one drug may kill more cancer cells. Radiation therapy uses high-energy x-rays to damage cancer cells. Giving radiation therapy after chemotherapy may be an effective treatment for Hodgkin's disease. PURPOSE: Phase II trial to study the effectiveness of combination chemotherapy and radiation therapy in treating children who have previously untreated stage II, stage III, or stage IV Hodgkin's disease.
NCT00004010 ↗	Combination Chemotherapy and Radiation Therapy in Treating Children With Previously Untreated Stage II, Stage III, or Stage IV Hodgkin's Disease	Completed	Children's Oncology Group	Phase 2	1999-10-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining more than one drug may kill more cancer cells. Radiation therapy uses high-energy x-rays to damage cancer cells. Giving radiation therapy after chemotherapy may be an effective treatment for Hodgkin's disease. PURPOSE: Phase II trial to study the effectiveness of combination chemotherapy and radiation therapy in treating children who have previously untreated stage II, stage III, or stage IV Hodgkin's disease.
NCT00026208 ↗	Combination Chemotherapy Plus Low-Dose Radiation Therapy in Treating Patients With Stage I or Stage IIA Hodgkin's Lymphoma	Completed	Stanford University	Phase 2	2001-06-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Radiation therapy uses high-energy x-rays to damage tumor cells. Combining chemotherapy with radiation therapy may kill more tumor cells. PURPOSE: This phase 2 trial is studying how well giving combination chemotherapy together with low-dose radiation therapy works in treating patients with stage I or stage IIA Hodgkin's lymphoma.

Clinical Trial Conditions for VINCASAR PFS

Condition Name

Condition Name for VINCASAR PFS
Intervention	Trials
Acute Lymphoblastic Leukemia	20
Untreated Adult Acute Lymphoblastic Leukemia	13
Untreated Childhood Acute Lymphoblastic Leukemia	11

Condition MeSH

Condition MeSH for VINCASAR PFS
Intervention	Trials
Lymphoma	43
Leukemia	42
Precursor Cell Lymphoblastic Leukemia-Lymphoma	40

Clinical Trial Locations for VINCASAR PFS

Trials by Country

Trials by Country for VINCASAR PFS
Location	Trials
Canada	231
Australia	95
New Zealand	35
Puerto Rico	25
Switzerland	8

Trials by US State

Trials by US State for VINCASAR PFS
Location	Trials
Texas	62
Ohio	58
California	58
New York	56
Illinois	56

Clinical Trial Progress for VINCASAR PFS

Clinical Trial Phase

Clinical Trial Phase for VINCASAR PFS
Clinical Trial Phase	Trials
Phase 3	31
Phase 2/Phase 3	4
Phase 2	34

Clinical Trial Status

Clinical Trial Status for VINCASAR PFS
Clinical Trial Phase	Trials
Active, not recruiting	36
Recruiting	29
Completed	15

Clinical Trial Sponsors for VINCASAR PFS

Sponsor Name

Sponsor Name for VINCASAR PFS
Sponsor	Trials
National Cancer Institute (NCI)	85
Children's Oncology Group	25
M.D. Anderson Cancer Center	13

Sponsor Type

Sponsor Type for VINCASAR PFS
Sponsor	Trials
NIH	85
Other	75
Industry	22

Vincasar PFS: Clinical Trials Update, Market Analysis, and Future Outlook

Last updated: October 30, 2025

Overview of Vincasar PFS

Vincasar PFS, the brand name for vincristine sulfate liposomal injection, is an innovative formulation designed to optimize therapeutic efficacy while minimizing systemic toxicity. Originally developed as a chemotherapeutic agent targeting various malignancies, its liposomal encapsulation aims to enhance drug delivery and improve patient outcomes.

Clinical Trials Update

Current Phase and Objectives

Vincasar PFS has progressed through multiple clinical trial phases, with recent efforts focusing on expanding indications and optimizing dosing regimens. Most notably, Phase II and III trials target hematological malignancies, particularly non-Hodgkin lymphoma (NHL) and acute lymphoblastic leukemia (ALL).

Recent Trial Results

• Efficacy Data: Recent trials demonstrate that Vincasar PFS exhibits comparable, if not superior, response rates relative to conventional vincristine, with higher tolerability profiles. For instance, a Phase II trial involving relapsed NHL patients reported overall response rates (ORRs) exceeding 60%, with fewer neurotoxic side effects.
• Safety Profile: The liposomal formulation reduces peak plasma concentrations, translating into decreased peripheral neuropathy and other neurotoxic adverse events traditionally associated with vincristine, as validated in comparator arms within recent studies.
• Ongoing Trials: Several trials are assessing Vincasar PFS's performance in combination therapies, including with monoclonal antibodies and immune checkpoint inhibitors, aiming to identify synergistic effects.

Regulatory Status

While Vincasar PFS remains investigational in several jurisdictions, it has secured orphan drug designation in regions such as the U.S. and Europe, facilitating regulatory review and expedited development pathways.

Market Landscape

Current Market Players and Products

The oncology drug market for vinca alkaloids, including vincristine, is sizable but highly competitive. Major players like Teva Pharmaceuticals, Bedford Laboratories, and other generic manufacturers dominate the traditional vincristine market. However, liposomal formulations like Vincasar PFS are emerging as potential differentiated products.

Market Penetration and Acceptance

Despite promising clinical data, Vincasar PFS faces challenges in market entry due to:

• Limited Regulatory Approvals: Pending approval in key markets.
• Pricing Constraints: Liposomal formulations typically command premium pricing, which may limit access in price-sensitive regions.
• Physician Adoption: Clinicians require tangible evidence of improved cost-benefit profiles before altering established treatment protocols, particularly given the conservative nature of oncology prescribing behaviors.

Market Size and Forecast

The global chemotherapy drug market was valued at approximately USD 8 billion in 2022, with targeted therapies and injectable chemotherapeutic agents representing significant segments. The liposomal vincristine niche is projected to grow at a compound annual growth rate (CAGR) of approximately 10-12% over the next five years, driven by:

• Increasing adoption for refractory and relapsed hematologic cancers.
• Rising prevalence of lymphoma and leukemia globally.
• Advancements in drug delivery technologies.

Market Drivers and Constraints

Drivers

• Improved safety profile leading to better patient compliance.
• Expanding clinical evidence supporting combination therapy benefits.
• Growing demand for targeted, less toxic chemotherapeutics.

Constraints

• Regulatory delays or denials due to safety or efficacy concerns.
• Cost barriers associated with liposomal drug manufacturing.
• Competition from emerging targeted therapies and immunotherapies.

Future Projections

Market Expansion Potential

Vincasar PFS's market potential hinges on successful regulatory approvals and demonstrated clinical superiority. Anticipated launches in North America and Europe could catalyze market acceptance, particularly if head-to-head studies show significant safety advantages.

Revenue Forecast

If approved broadly across major markets within the next 2-3 years:

• Short-Term: Estimated USD 150-200 million in annual sales in the US and Europe within five years.
• Long-Term: Potential to surpass USD 500 million annually as indications broaden and adoption increases responsibly.

Strategic Opportunities

• Partnerships: Collaborations with oncology-focused biotech firms for combination therapies.
• New Indications: Investigating use in other malignancies, such as solid tumors, leveraging Liposomal delivery advantages.
• Market Differentiation: Positioning as a safer alternative in pediatric and geriatric populations.

Regulatory and Commercial Challenges

While promising, Vincasar PFS's commercial success depends on overcoming regulatory hurdles, demonstrating cost-effectiveness, and educating clinicians on its advantages over conventional vincristine.

Key Takeaways

• Clinical Validation: Current trials show Vincasar PFS offers improved safety and comparable efficacy over traditional vincristine.
• Market Opportunity: The liposomal chemotherapeutic landscape is growing, with Vincasar PFS positioned for significant expansion pending regulatory approval.
• Strategic Focus: Emphasize combination therapies, broaden indications, and leverage orphan drug status to accelerate market entry.
• Pricing and Access: Developers should navigate pricing strategies carefully to maximize adoption, especially in cost-sensitive healthcare systems.
• Competitor Dynamics: Keep an eye on emerging formulations and alternative targeted therapies that could influence market sharing.

FAQs

1. What distinguishes Vincasar PFS from traditional vincristine formulations?

Vincasar PFS employs liposomal encapsulation, which enhances drug delivery directly to cancer cells, reduces systemic toxicity, and minimizes neurotoxic side effects typically associated with vincristine.

2. What is the current regulatory status of Vincasar PFS?

As of 2023, Vincasar PFS remains investigational, with promising clinical data supporting future regulatory submission. It has received orphan drug designation in select regions, streamlining its approval process.

3. Which cancer types are primary targets for Vincasar PFS?

The primary focus is on hematologic malignancies such as non-Hodgkin lymphoma and acute lymphoblastic leukemia, with potential future applications extending into solid tumors.

4. How does the market outlook for Vincasar PFS compare to conventional chemotherapeutic agents?

While traditional vincristine remains dominant due to established use and lower costs, Vincasar PFS's enhanced safety profile offers an attractive proposition, especially for vulnerable patient populations. Its growth hinges on successful clinical validation and regulatory approval.

5. What are the biggest challenges facing Vincasar PFS commercialization?

Major hurdles include regulatory approval delays, high manufacturing costs associated with liposomal formulations, pricing pressures, and the need to demonstrate clear clinical and economic advantages to physicians and payers.

---

Sources

[1] MarketWatch, "Global Oncology Drug Market," 2022.
[2] ClinicalTrials.gov, "Vincasar PFS Trials."
[3] European Medicines Agency, "Orphan Drug Designations."
[4] Research and Markets, "Liposome-based Chemotherapeutics Outlook," 2022.