CLINICAL TRIALS PROFILE FOR ONUREG

All Clinical Trials for onureg

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT02828358 ↗	Azacitidine and Combination Chemotherapy in Treating Infants With Acute Lymphoblastic Leukemia and KMT2A Gene Rearrangement	Active, not recruiting	National Cancer Institute (NCI)	Phase 2	2017-03-27	This pilot phase II trial studies the side effects of azacitidine and combination chemotherapy in infants with acute lymphoblastic leukemia and KMT2A gene rearrangement. Drugs used in chemotherapy, such as methotrexate, prednisolone, daunorubicin hydrochloride, cytarabine, dexamethasone, vincristine sulfate, pegaspargase, hydrocortisone sodium succinate, azacitidine, cyclophosphamide, mercaptopurine, leucovorin calcium, and thioguanine work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving more than one drug may kill more cancer cells.
NCT01522976 ↗	Azacitidine With or Without Lenalidomide or Vorinostat in Treating Patients With Higher-Risk Myelodysplastic Syndromes or Chronic Myelomonocytic Leukemia	Active, not recruiting	National Cancer Institute (NCI)	Phase 2	2012-03-01	This randomized phase II/III trial studies how well azacitidine works with or without lenalidomide or vorinostat in treating patients with higher-risk myelodysplastic syndromes or chronic myelomonocytic leukemia. Drugs used in chemotherapy, such as azacitidine, work in different ways to stop the growth of cancer cells, either by killing the cells, stopping them from dividing, or by stopping them from spreading. Lenalidomide may stop the growth of cancer cells by stopping blood flow to the cancer. Vorinostat may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. It is not yet known whether azacitidine is more effective with or without lenalidomide or vorinostat in treating myelodysplastic syndromes or chronic myelomonocytic leukemia.
NCT01349959 ↗	Azacitidine and Entinostat in Treating Patients With Advanced Breast Cancer	Active, not recruiting	National Cancer Institute (NCI)	Phase 2	2011-04-21	This phase II trial studies how well giving azacitidine and entinostat work in treating patients with advanced breast cancer. Drugs used in chemotherapy, such as azacitidine, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. Entinostat may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Giving azacitidine together with entinostat may kill more tumor cells.
NCT01168219 ↗	Busulfan, Fludarabine Phosphate, and Anti-Thymocyte Globulin Followed By Donor Stem Cell Transplant and Azacitidine in Treating Patients With High-Risk Myelodysplastic Syndrome and Older Patients With Acute Myeloid Leukemia	Active, not recruiting	National Cancer Institute (NCI)	Phase 2	2010-07-15	This phase II clinical trial is studying how well giving busulfan, fludarabine phosphate, and anti-thymocyte globulin followed by donor stem cell transplant and azacitidine works in treating patients with high-risk myelodysplastic syndrome and older patients with acute myeloid leukemia. Giving low doses of chemotherapy, such as busulfan and fludarabine phosphate, before a donor stem cell transplant helps stop the growth of cancer cells. It also stops the patient's immune system from rejecting the donor's stem cells. The donated stem cells may replace the patient's immune cells and help destroy any remaining cancer cells (graft-vs-tumor effect). Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Giving anti-thymocyte globulin before transplant and giving azacitidine, tacrolimus, and methotrexate after the transplant may stop this from happening.
NCT00658814 ↗	Azacitidine and Gemtuzumab Ozogamicin in Treating Older Patients With Previously Untreated Acute Myeloid Leukemia	Active, not recruiting	National Cancer Institute (NCI)	Phase 2	2008-12-01	This phase II trial is studying the side effects of giving azacitidine together with gemtuzumab ozogamicin to see how well it works in treating older patients with previously untreated acute myeloid leukemia. Drugs used in chemotherapy, such as azacitidine, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Azacitidine may also stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Monoclonal antibodies, such as gemtuzumab ozogamicin, can block cancer growth in different ways. Some block the ability of cancer cells to grow and spread. Others find cancer cells and help kill them or carry cancer-killing substances to them. Giving azacitidine together with gemtuzumab ozogamicin may kill more cancer cells.
NCT00392353 ↗	Vorinostat and Azacitidine in Treating Patients With Myelodysplastic Syndromes or Acute Myeloid Leukemia	Active, not recruiting	National Cancer Institute (NCI)	Phase 1/Phase 2	2006-11-22	This phase I/II trial studies the side effects and best dose of vorinostat and azacitidine and to see how well they work in treating patients with myelodysplastic syndromes or acute myeloid leukemia. Vorinostat may stop the growth of cancer or abnormal cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as azacitidine, work in different ways to stop the growth of cancer or abnormal cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving vorinostat together with azacitidine may kill more cancer or abnormal cells.
NCT00336063 ↗	Vorinostat and Azacitidine in Treating Patients With Locally Recurrent or Metastatic Nasopharyngeal Cancer or Nasal Natural Killer T-Cell Lymphoma	Active, not recruiting	National Cancer Institute (NCI)	Phase 1	2006-03-03	This phase I trial studies the side effects and best dose of vorinostat when given together with azacitidine in treating patients with nasopharyngeal cancer or nasal natural killer T-cell lymphoma that has recurred (come back) at or near the same place as the original (primary) tumor, usually after a period of time during which the cancer could not be detected or has spread to other parts of the body. Drugs used in chemotherapy, such as vorinostat and azacitidine, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Vorinostat and azacitidine also may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving vorinostat together with azacitidine may kill more cancer cells.

Clinical Trial Conditions for onureg

Condition Name

Condition Name for onureg
Intervention	Trials
Acute Myeloid Leukemia	19
Myelodysplastic Syndrome	10
Recurrent Acute Myeloid Leukemia	8
Refractory Acute Myeloid Leukemia	7

Condition MeSH

Condition MeSH for onureg
Intervention	Trials
Leukemia	28
Leukemia, Myeloid, Acute	26
Leukemia, Myeloid	25
Myelodysplastic Syndromes	18

Clinical Trial Locations for onureg

Trials by Country

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Trials by Country for onureg
Location	Trials
United States	226
Canada	13
France	2
Australia	2
China	1

Trials by US State

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−

Trials by US State for onureg
Location	Trials
Texas	15
California	11
Ohio	10
New York	10
Missouri	8

Clinical Trial Progress for onureg

Clinical Trial Phase

Clinical Trial Phase for onureg
Clinical Trial Phase	Trials
Phase 2/Phase 3	2
Phase 2	20
Phase 1/Phase 2	10

Clinical Trial Status

Clinical Trial Status for onureg
Clinical Trial Phase	Trials
Not yet recruiting	14
Recruiting	12
Active, not recruiting	10

Clinical Trial Sponsors for onureg

Sponsor Name

Sponsor Name for onureg
Sponsor	Trials
National Cancer Institute (NCI)	28
M.D. Anderson Cancer Center	9
Bristol-Myers Squibb	4

Sponsor Type

Sponsor Type for onureg
Sponsor	Trials
NIH	28
Other	21
Industry	12

Onureg: A Comprehensive Overview of Clinical Trials, Market Analysis, and Projections

Introduction to Onureg

Onureg, also known as azacitidine, is an oral hypomethylating agent used in the treatment of Acute Myeloid Leukemia (AML) and other myelodysplastic syndromes. It is a significant advancement in the field of oncology, particularly for patients who have achieved remission but are not eligible for a stem cell transplant.

Clinical Trials and Efficacy

The QUAZAR AML-001 Study

The efficacy of Onureg was evaluated in the QUAZAR AML-001 phase 3 clinical trial. This study involved 472 patients with AML who had achieved first complete remission (CR) or complete remission with incomplete blood count recovery (CRi) following intensive induction chemotherapy. Patients were randomized to receive either Onureg (300 mg orally on days 1 through 14 of each 28-day cycle) or a placebo.

• The trial demonstrated a statistically significant improvement in overall survival for patients treated with Onureg compared to those receiving a placebo. Patients on Onureg had a median overall survival of 24.7 months, whereas the placebo group had a median overall survival of 14.8 months[4][5].
• The study also showed that Onureg reduced the risk of death by 31% compared to the placebo group[5].
• Additional analyses indicated that Onureg improved survival in patients with or without measurable residual disease and across various consolidation cycles[3].

Patient Outcomes and Survival Rates

• Approximately three out of four (73%) patients who received Onureg were alive at one year, compared to about two out of four (56%) patients who received the placebo[1].
• The trial highlighted that Onureg can provide more life to patients, with half of the patients in the Onureg group still alive at the time of analysis, compared to the placebo group[1].

Market Analysis

Market Need and Unmet Needs

Onureg addresses a significant unmet need in the treatment of AML, particularly for patients whose disease is under control but for whom a stem cell transplant is not feasible. AML is an aggressive form of blood cancer that leads to fewer mature blood cells, resulting in symptoms such as weakness, infection, and bleeding[2].

Regulatory Approvals

Onureg has received regulatory approvals in several regions, including the European Union, where it was granted a marketing authorization on June 17, 2021, for the maintenance therapy of adult patients with AML who have achieved complete remission or complete remission with incomplete hematologic recovery following induction therapy[2].

Clinical Benefits and Side Effects

The clinical benefits of Onureg include significant improvements in survival for patients with AML. However, the treatment is associated with side effects such as gastrointestinal toxicities and myelosuppression, which can be managed through dose adjustments and best supportive care[2][3].

Cost-Effectiveness and Pricing

The cost-effectiveness of Onureg is a critical factor in its market dynamics. In some regions, such as Canada, Onureg was not considered cost-effective at a willingness-to-pay threshold of $50,000 per quality-adjusted life-year (QALY) relative to best supportive care. A significant price reduction of at least 85% would be necessary to make it cost-effective at this threshold[2].

Market Size and Growth Projections

Global Leukemia Therapeutics Market

The global leukemia therapeutics market, which includes Onureg, is expected to grow significantly. The market is projected to reach $14.16 billion by 2031, growing at a Compound Annual Growth Rate (CAGR) of 6.1% from 2021 to 2031. This growth is driven by factors such as the rise in prevalence of chronic lymphocytic leukemia, increased adoption of targeted therapies, and improvements in healthcare infrastructure in emerging markets[2].

Regional Market Performance

North America, where Onureg is approved and marketed by Bristol Myers Squibb, is expected to maintain its dominance in the leukemia therapeutics market due to its well-established healthcare infrastructure and high purchasing power. However, the Asia-Pacific region is anticipated to witness the highest growth rate during the forecast period, driven by growing infrastructure, rising disposable incomes, and the presence of domestic pharmaceutical companies[2].

Competitive Landscape

Onureg operates within a competitive landscape that includes several major pharmaceutical companies. Bristol Myers Squibb, the manufacturer of Onureg, has a strong portfolio of innovative medicines, including other oncology treatments like Reblozyl and Breyanzi. The company's strategic approach to capital allocation and its focus on research and development help maintain its competitive edge[2].

Financial Performance and Strategies

Bristol Myers Squibb has invested significantly in research and development, with $9.5 billion allocated to this area in recent years. The company has also seen substantial cash flow from operating activities and has returned $12.6 billion to shareholders through dividends and stock repurchases. These financial metrics indicate a strong foundation for the company to support the marketing and development of Onureg and other therapeutic products[2].

Recent Developments and Strategies

Bristol Myers Squibb has adopted several strategies to enhance the market position of Onureg, including product approvals, expansions into new indications, and investments in digital and onsite training to build healthcare capacity, particularly in regions with limited access to oncology care[2].

Patient and Healthcare Provider Perspectives

Patient Stories and Experiences

Patients treated with Onureg have reported significant improvements in their quality of life. For example, Paul, a real Onureg patient living with AML in first remission, and his wife Donna, noted that "ONUREG gave us ... more to hope for ... More days. More years. More time"[1].

Updated NCCN Guidelines

The National Comprehensive Cancer Network (NCCN) Guidelines have been updated to include Onureg as a Category 1 preferred maintenance treatment for AML patients who achieved first complete remission or complete remission with incomplete blood count recovery following intensive induction chemotherapy and are not able to complete intensive curative therapy[4].

Key Takeaways

• Regulatory Approvals: Onureg has received marketing authorization in the EU and is recommended for maintenance therapy in AML patients.
• Clinical Benefits: Onureg offers significant survival benefits but comes with manageable side effects.
• Cost-Effectiveness: The drug requires significant price reductions to be considered cost-effective in some regions.
• Market Growth: The leukemia therapeutics market, including Onureg, is expected to grow substantially by 2031.
• Regional Performance: North America dominates the market, but Asia-Pacific is expected to grow at the highest rate.
• Competitive Landscape: Onureg competes in a market with several major pharmaceutical players, with Bristol Myers Squibb maintaining a strong position through innovation and strategic investments.

FAQs

What is Onureg used for?

Onureg is used for the maintenance therapy of adult patients with Acute Myeloid Leukemia (AML) who have achieved complete remission or complete remission with incomplete hematologic recovery following intensive induction chemotherapy and are not able to complete intensive curative therapy[2][4].

How was Onureg studied in clinical trials?

Onureg was studied in the QUAZAR AML-001 phase 3 clinical trial, where it was compared against a placebo. The trial involved 472 patients and demonstrated a statistically significant improvement in overall survival for patients treated with Onureg[3][4].

What are the side effects of Onureg?

Onureg is associated with side effects such as gastrointestinal toxicities and myelosuppression, which can be managed through dose adjustments and best supportive care[2][3].

Is Onureg cost-effective?

In some regions, such as Canada, Onureg was not considered cost-effective at a willingness-to-pay threshold of $50,000 per quality-adjusted life-year (QALY) relative to best supportive care. A significant price reduction would be necessary to make it cost-effective at this threshold[2].

What is the expected growth of the leukemia therapeutics market?

The global leukemia therapeutics market, which includes Onureg, is expected to reach $14.16 billion by 2031, growing at a CAGR of 6.1% from 2021 to 2031[2].

Sources

1. ONUREG: Why ONUREG? - ONUREG was tested against placebo[1].
2. DrugPatentWatch: Onureg Drug Patent Profile[2].
3. Bristol Myers Squibb: Bristol Myers Squibb Presents Analyses from Pivotal QUAZAR AML-001 Study of Onureg[3].
4. HealthTree for AML: Onureg: Maintains Remission in AML Patients[4].
5. FiercePharma: Bristol Myers Squibb's decade-old Vidaza goes oral with FDA nod for Onureg in AML[5].