CLINICAL TRIALS PROFILE FOR MYLOTARG

All Clinical Trials for MYLOTARG

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00038805 ↗	Nonmyeloablative Preparative Regimen Using Mylotarg for Patients With High Risk Acute Myeloid Leukemia (AML), Acute Lymphocytic Leukemia (ALL), Chronic Myeloid Leukemia (CML) and Myelodysplastic Syndrome (MDS)	Terminated	Wyeth is now a wholly owned subsidiary of Pfizer	Phase 2/Phase 3	2001-05-01	Primary Objective: To determine the safety and maximum tolerated dose of CMA-676 as part of an intensive but nonmyeloablative preparative regimen in older or medically infirm patients undergoing mini-allogeneic peripheral blood stem cell transplantation Secondary Objectives: 1. To evaluate response rates, engraftment kinetics and degree of chimerism achievable with this strategy. 2. To evaluate disease-free and overall survival and relapse rates. 3. To evaluate the need and ability to give multiple cycles of Mylotarg plus FA and mobilized DLI in patients not achieving complete remission.
NCT00006265 ↗	Gemtuzumab Ozogamicin and High-Dose Cytarabine in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia	Completed	National Cancer Institute (NCI)	Phase 2	2001-03-01	RATIONALE: Monoclonal antibodies, such as gemtuzumab ozogamicin, can locate cancer cells and either kill them or deliver cancer-killing substances to them without harming normal cells. Drugs used in chemotherapy, such as cytarabine, use different ways to stop cancer cells from dividing so they stop growing or die. Combining gemtuzumab ozogamicin with cytarabine may kill more cancer cells. PURPOSE: Phase II trial to study the effectiveness of combining gemtuzumab ozogamicin with high-dose cytarabine in treating patients who have relapsed or refractory acute myeloid leukemia.
NCT00006265 ↗	Gemtuzumab Ozogamicin and High-Dose Cytarabine in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia	Completed	Alliance for Clinical Trials in Oncology	Phase 2	2001-03-01	RATIONALE: Monoclonal antibodies, such as gemtuzumab ozogamicin, can locate cancer cells and either kill them or deliver cancer-killing substances to them without harming normal cells. Drugs used in chemotherapy, such as cytarabine, use different ways to stop cancer cells from dividing so they stop growing or die. Combining gemtuzumab ozogamicin with cytarabine may kill more cancer cells. PURPOSE: Phase II trial to study the effectiveness of combining gemtuzumab ozogamicin with high-dose cytarabine in treating patients who have relapsed or refractory acute myeloid leukemia.
NCT00017589 ↗	Oblimersen and Gemtuzumab Ozogamicin in Treating Older Patients With Relapsed Acute Myeloid Leukemia	Completed	Genta Incorporated	Phase 2	2000-12-01	RATIONALE: Monoclonal antibodies such as gemtuzumab ozogamicin can locate tumor cells and either kill them or deliver tumor-killing substances to them without harming normal cells. Oblimersen may help gemtuzumab ozogamicin kill more cancer cells by making cancer cells more sensitive to the drug. PURPOSE: Phase II trial to study the effectiveness of combining oblimersen and gemtuzumab ozogamicin in treating older patients who have relapsed acute myeloid leukemia.

Clinical Trial Conditions for MYLOTARG

Condition Name

Condition Name for MYLOTARG
Intervention	Trials
Acute Myeloid Leukemia	20
Leukemia	11
Myelodysplastic Syndrome	9
Acute Myelogenous Leukemia	7

Condition MeSH

Condition MeSH for MYLOTARG
Intervention	Trials
Leukemia	53
Leukemia, Myeloid, Acute	52
Leukemia, Myeloid	49
Myelodysplastic Syndromes	14

Clinical Trial Locations for MYLOTARG

Trials by Country

+

−

Trials by Country for MYLOTARG
Location	Trials
United States	285
Canada	24
Spain	17
Germany	6
France	6

Trials by US State

+

−

Trials by US State for MYLOTARG
Location	Trials
Texas	17
New York	13
California	13
Washington	12
North Carolina	11

Clinical Trial Progress for MYLOTARG

Clinical Trial Phase

Clinical Trial Phase for MYLOTARG
Clinical Trial Phase	Trials
Phase 4	3
Phase 3	8
Phase 2/Phase 3	3

Clinical Trial Status

Clinical Trial Status for MYLOTARG
Clinical Trial Phase	Trials
Completed	24
Recruiting	23
Active, not recruiting	4

Clinical Trial Sponsors for MYLOTARG

Sponsor Name

Sponsor Name for MYLOTARG
Sponsor	Trials
National Cancer Institute (NCI)	23
Pfizer	12
M.D. Anderson Cancer Center	10

Sponsor Type

Sponsor Type for MYLOTARG
Sponsor	Trials
Other	67
Industry	26
NIH	23

Mylotarg: Clinical Trials, Market Analysis, and Projections

Introduction

Mylotarg, also known as gemtuzumab ozogamicin, is an antibody-drug conjugate (ADC) that targets the CD33 antigen, which is commonly expressed on the surface of acute myeloid leukemia (AML) cells. Here, we will delve into the clinical trials, market analysis, and projections for this significant therapeutic agent.

Clinical Trials and Efficacy

ALFA-0701 Trial

One of the pivotal trials for Mylotarg is the ALFA-0701 study, a Phase III, randomized, open-label clinical trial. This trial compared the efficacy of Mylotarg in combination with daunorubicin (DNR) and cytarabine (AraC) versus DNR and AraC alone in adult patients with previously untreated de novo AML. The primary endpoint was event-free survival (EFS), which showed a clinically meaningful improvement with the addition of Mylotarg. The median EFS was 13.6 months for the Mylotarg + DA arm versus 8.8 months for the DA alone arm, with a hazard ratio (HR) of 0.68 (95% CI: 0.51–0.91)[1][3].

AAML0531 Study

The AAML0531 study also provided supportive evidence for the efficacy of Mylotarg. This study measured EFS from the date of study entry until induction failure, relapse, or death. The EFS hazard ratio was 0.84 (95% CI: 0.71–0.99), indicating a significant benefit of adding Mylotarg to chemotherapy. The estimated percentage of patients free of induction failure, relapse, or death at five years was 48% in the Mylotarg + chemotherapy arm versus 40% in the chemotherapy alone arm[1].

MyloFrance-1 Study

In the MyloFrance-1 phase 2 study, Mylotarg was evaluated as a single agent in adults with CD33-positive AML in first relapse. The study showed that 26% of patients achieved complete remission (CR) following a single course of Mylotarg, with a median relapse-free survival of 11.6 months[1].

AML-19 Study

The AML-19 study compared Mylotarg to best supportive care (BSC) in patients with newly diagnosed AML who were either over 75 years old or between 61-75 years old with poor performance status or unwilling to receive intensive chemotherapy. The study demonstrated a significant improvement in overall survival (OS) with Mylotarg, with a median OS of 4.9 months versus 3.6 months for BSC, and a hazard ratio for OS of 0.69 (95% CI: 0.53–0.90)[1].

Safety Profile

The safety profile of Mylotarg has been extensively evaluated in these clinical trials. Common adverse events include hemorrhage, veno-occlusive disease, and various bleeding events such as epistaxis, purpura, and thrombocytopenia. Despite these risks, the overall safety profile is considered acceptable, and the benefits of Mylotarg therapy are deemed to outweigh the potential risks[3].

Market Analysis

Market Size and Forecast

The global Mylotarg market was valued at US$ 214.3 million in 2023 and is anticipated to reach US$ 179.5 million by 2030, with a compound annual growth rate (CAGR) of -2.5% during the forecast period 2024-2030. This decline is likely due to the limited patient population and the availability of other therapeutic options[2].

Regional Market Segmentation

The market is segmented by region, with detailed analyses provided for various countries and regions, including North America, Europe, Asia-Pacific, Latin America, and the Middle East & Africa. This segmentation helps in understanding the regional dynamics and growth potential of the Mylotarg market[2].

Competitive Landscape

The report on the global Mylotarg market also provides profiles of key competitors and their market ranks. It discusses technological trends and new product developments, which are crucial for manufacturers, new entrants, and industry chain-related companies to navigate the market effectively[2].

Projections and Future Outlook

Ongoing Research and Development

Vor Bio is currently conducting trials combining Mylotarg with their engineered hematopoietic stem cell product, trem-cel. This approach aims to enhance the therapeutic index of Mylotarg by shielding blood cells from on-target toxicity. Early data have shown promising results, including reliable engraftment and robust platelet recovery, suggesting potential future benefits for AML patients[4].

Regulatory Approvals

Mylotarg has been approved by regulatory bodies such as the FDA and the European Medicines Agency (EMA) for the treatment of AML. Health Canada has also granted approval based on the favorable benefit-risk profile demonstrated in clinical trials[3].

Key Takeaways

• Clinical Efficacy: Mylotarg has shown significant improvements in event-free survival and overall survival in various clinical trials, particularly when combined with standard chemotherapy regimens.
• Safety Profile: While Mylotarg is associated with certain adverse events, its safety profile is considered acceptable, and the benefits outweigh the risks.
• Market Analysis: The global Mylotarg market is expected to decline slightly due to a limited patient population and the emergence of other therapies.
• Future Outlook: Ongoing research, such as the combination with trem-cel, holds promise for enhancing the therapeutic efficacy and safety of Mylotarg.

FAQs

What is Mylotarg used for?

Mylotarg is used to treat acute myeloid leukemia (AML), specifically targeting CD33-positive AML cells.

What are the key clinical trials for Mylotarg?

The ALFA-0701, AAML0531, MyloFrance-1, and AML-19 studies are some of the pivotal trials that have evaluated the efficacy and safety of Mylotarg.

What is the safety profile of Mylotarg?

Mylotarg is associated with adverse events such as hemorrhage, veno-occlusive disease, and various bleeding events, but its overall safety profile is considered acceptable.

What is the current market size and forecast for Mylotarg?

The global Mylotarg market was valued at US$ 214.3 million in 2023 and is expected to reach US$ 179.5 million by 2030, with a CAGR of -2.5%.

Are there any ongoing research and development activities involving Mylotarg?

Yes, Vor Bio is conducting trials combining Mylotarg with their engineered hematopoietic stem cell product, trem-cel, to enhance the therapeutic index of Mylotarg.

Sources

1. Pfizer Medical Information: MYLOTARG™ (gemtuzumab ozogamicin) Clinical Studies - US.
2. QYResearch: Global Mylotarg Market Research Report 2024.
3. Health Canada: Summary Basis of Decision for Mylotarg.
4. CRISPR Medicine News: Vor Bio Announces Positive Clinical Data From Trial of Trem-cel Followed by Mylotarg in Acute Myeloid Leukaemia.