Acute myeloid leukemia therapeutics market size, share, growth trend and research report 2020

Acute Myeloid Leukemia Therapeutics Market Size, Share, Growth Trend and Research Report 2020: Radiant Insights, Inc Summary GBI Research, has released its latest pharma report, "Acute Myeloid Leukemia Therapeutics Market to 2020 - Novel Therapies to Offer Clinical Benefit in Small Patient Cohorts" Treatment and prognosis in AML is strongly influenced by a patient's age, and their cytogenetic profile.

In the majority of cases these two prognostic influences are linked, with a higher frequency of unfavorable cytogenetic abnormalities observed in the elderly. Survival in this cohort of elderly patients is very poor, with a five year overall survival of 3-8% (Luger, 2010). Despite a relatively advanced understanding of genetic abnormalities associated with AML, the introduction of targeted therapies is lagging in this indication in comparison to other cancers such as breast and lung cancer, with no approved targeted therapies. Such slow development may be a reflection of AMLs status as an orphan indication.

View Full Report with TOC @ http://www.radiantinsights.com/research/acute-myeloid-leukemiatherapeutics-market-to-2020-novel-therapies-to-offer-clinical-benefit-in-small-patient-cohorts Intensive treatment in eligible patients (younger patients, and approximately 50% of diagnosed elderly patients) is typically the combination of the two chemotherapeutic agents cytarabine and daunorubicin, both of which were approved in the 1960s. In patients ineligible for intensive first-line chemotherapy, options are very poor, with the more recently approved Vidaza and Dacogen as the treatment options, which both offer unsatisfactory survival. Across all newly diagnosed patients that obtain complete remission, a stem cell transplant offers the highest chance of long-term survival. However, this procedure is risky, with a higher rate of treatment related mortality in the absence of better techniques to reduce the risk of graft-versus-host disease.

The majority of patients experience disease relapse, which is almost always fatal. Treatment options in these patients typically involve the off-label use of chemotherapeutic agents, whether in combination or as monotherapies.

There are clear gaps in the market for therapies to meet several unmet needs by increasing the initial length of remission; improving treatment options for newly-diagnosed patients, ineligible for relapsed disease treatments; improving the success of and reducing the side effects of stem cell transplantation; and improving survival, safety and quality of life in patients with relapsed disease. The current developmental pipeline addresses these gaps in the market, along with the significant lack of targeted therapies. Five of the eight pipeline products are under development as non-intensive therapies for the elderly, and six of the eight products are being investigated in relapsed disease.

Results so far have been mixed, with several drugs offering no overwhelming clinical benefit in Phase I and II clinical trials. Some drugs have demonstrated encouraging results - namely CPX-351, quizartinib, StemEx, treosulfan and midostaurin. All of these drugs are forecast to be approved within the forecast period, a result of clinical trial data that suggest these drugs can offer improved survival in comparison to the currently marketed products. It is important to note however, that these improvements and the

subsequent approval of these products is restricted to small patient cohorts - including patients with secondary AML, those with internal tandem repeats in Fms-like tyrosine kinase, and patients eligible for a stem cell transplant, but for whom a matched donor cannot be found. This fragmentation in the treatment algorithm is a reflection of the heterogeneity of AML, with continued fractionation likely to be essential for further effective treatments to be developed. This is reflected in the current developmental pipeline, with drugs targeting a high variety of molecule types and molecular targets currently under investigation in this disease.

The small patient cohorts the pipeline drugs are expected to be approved in will result in each having a minimal effect on any growth in market revenues over the forecast period. They will nevertheless stimulate market growth. An increase in prevalence and treatment populations, driven by aging populations across each territory will also have a positive effect on market revenues. However, as an orphan indication, the effect of population growth is also expected to have a minor effect. As a result, the global market revenues are forecast to rise at a limited CAGR of 4.8% from $632.4m in 2013 to $878.2m in 2020.

View Full Report with TOC @ http://www.radiantinsights.com/research/acute-myeloid-leukemiatherapeutics-market-to-2020-novel-therapies-to-offer-clinical-benefit-in-small-patient-cohorts Scope The report analyzes treatment usage patterns, marketed and pipeline drugs, and market forecasts across indications for AML.The report covers and includes - A brief introduction to AML, including the disease's pathogenesis, risk factors and diagnosis - An in-depth analysis of the drug combinations used in the treatment of AML, including analyses of their safety, efficacy, and place in the disease treatment algorithm. This includes a heat map comparing the drug combinations in terms of safety and efficacy - A comprehensive review of the pipeline for AML therapies, including individual analysis of a number of late-stage pipeline drugs that have the potential to enter the market during the forecast period. The pipeline is analyzed on the basis of Phase distribution, molecule types and molecular targets, as well as administration routes - An additional in-depth analysis of pipeline drug clinical trials by phase, trial size, trial duration and program failure rate analyses for each molecule type and molecular target - A multi-scenario forecast data for the market to 2020, taking into account how it will be affected by the introduction of new drugs, the expiry of key patents on current drugs and the changes in disease epidemiology across the key developed markets including the US, Canada, Japan, Germany, the UK, France, Italy and Spain - A discussion of the drivers and barriers for market growth

- An in-depth analysis of licensing and co-development deals involving drugs indicated in AML, including an in-depth outline of the key deals Reasons to buy The report will assist business development and enable marketing executives to strategize their product launches, by allowing them to - Understand the efficacy and safety of the current monotherapies and drug combinations used in the treatment of AML, with in-depth analysis of the disease treatment algorithm - Understand the key signaling pathways and molecular targets currently under investigation in drug development for AML View Full Report with TOC @ http://www.radiantinsights.com/research/acute-myeloid-leukemiatherapeutics-market-to-2020-novel-therapies-to-offer-clinical-benefit-in-small-patient-cohorts - Understand the vast Scope of the pipeline, including which molecule types and molecular targets are most prominent - Observe the trends in clinical trial duration and size by clinical phase and molecule type, and use the clinical trial failure rate analysis to assess the risk profiles of current and/or future developmental programs for AML cancer therapeutics - Assess the potential clinical and commercial impact of current late-stage pipeline molecules in the AML therapeutics market - Assess the location of involved companies, and the value of both licensing and co-development deals involving drugs under investigation for the treatment of AML Table of Contents Table of Contents 4 1.1 List of Tables 8 1.2 List of Figures 9 2 Introduction 11 2.1 Disease Introduction 12 2.2 Epidemiology 12 2.3 Symptoms 12 2.4 Risk Factors 13 2.4.1 Age 13

2.4.2 Gender 13 2.4.3 Smoking 13 2.4.4 Chemotherapy or Radiation Therapy 13 2.4.5 Benzene 13 2.4.6 Previous Myelodysplastic Syndrome 13 2.4.7 Chromosomal Disorders or Genetic Mutations 13 2.5 Diagnostic Techniques 14 2.5.1 Blood Tests and Immunophenotyping 14 2.5.2 Flow-Cytometry 14 View Full Report with TOC @ http://www.radiantinsights.com/research/acute-myeloid-leukemiatherapeutics-market-to-2020-novel-therapies-to-offer-clinical-benefit-in-small-patient-cohorts 2.5.3 Cytogenetic Analysis 14 2.5.4 Molecular Diagnostics 14 2.6 Pathophysiology 14 2.7 Diagnostic Criteria 16 2.8 Prognosis and Survival 18 3 Treatment Algorithm 21 3.1 Treatment of Acute Promyelocytic (M3) Leukemia is Effective, with High Cure Rates 22 3.2 Clinical Trial Response Criteria 22 3.3 Remission Induction Therapy 23 3.3.1 Intensive Remission Induction Therapy 23 3.3.2 Non-intensive Remission Induction Therapy 31 3.4 Consolidation Therapy 37 3.4.1 High- or Low-dose Cytarabine-based Therapy 37 3.4.2 Stem Cell Transplantation 39 3.5 Remission Re-induction in Relapsed Disease 41

3.5.1 Salvage Chemotherapy 42 3.5.2 HSCT in Relapsed Disease 43 4 Commercial and Clinical Prospects of Marketed Products 47 4.1 Cytarabine 47 4.2 Daunorubicin 48 4.3 Idarubicin 48 4.4 Vidaza 49 4.5 Dacogen 50 4.6 Mitoxantrone 51 4.7 Etoposide 52 4.8 Fludarabine 53 4.9 Busulfan 54 4.10 Cyclophosphamide 54 4.11 Conclusion 55 View Full Report with TOC @ http://www.radiantinsights.com/research/acute-myeloid-leukemiatherapeutics-market-to-2020-novel-therapies-to-offer-clinical-benefit-in-small-patient-cohorts 5 Pipeline for Acute Myeloid Leukemia Therapeutics 56 5.1 Overview by Phase and Route of Administration 57 5.2 Overview by Molecule Type and Molecular Target 58 5.3 Key Molecular Targets in the Developmental Pipeline 59 5.3.1 DNA Machinery Targets 60 5.3.2 Targeted Therapies 60 5.3.3 CD123 61 5.3.4 CD33 61 5.3.5 Wilm's Tumor 1 63 5.3.6 Fms-Like Tyrosine Kinase 3 and its Downstream Pathway Components 63

5.3.7 Discussion 67 5.4 Clinical Trials 68 5.4.1 Clinical Trial Duration 68 5.4.2 Clinical Trial Size 70 5.4.3 Clinical Trial Failure Rates 71 5.4.4 Discussion 72 5.5 Promising Drugs in AML Developmental Pipeline 74 5.5.1 New Formulations 74 5.5.2 Nucleoside analogues 77 5.5.3 Stem Cell Therapy 88 5.5.4 Conclusions 92 View Full Report with TOC @ http://www.radiantinsights.com/research/acute-myeloid-leukemiatherapeutics-market-to-2020-novel-therapies-to-offer-clinical-benefit-in-small-patient-cohorts 6 Market Forecasts 97 6.1 Global 97 6.1.1 Treatment Usage Patterns 97 6.1.2 Market Forecasts 98 6.2 North America 101 6.2.1 US 101 6.2.2 Canada 104 6.3 Top Five European Markets 106 6.3.1 Epidemiology and Treatment Usage Patterns 106 6.3.2 Annual Cost of Therapy 108 6.3.3 Market Revenues 109 6.4 Japan 111 6.4.1 Epidemiology and Treatment Usage Patterns 111

6.4.2 Annual Cost of Therapy 111 6.4.3 Market Forecast 113 7 Drivers and Barriers 114 7.1 Market Drivers 114 7.1.1 High Level of Understanding of Genetic and Epigenetic Factors Underlying AML 114 7.1.2 High Number of Candidates in Drug Development 114 7.1.3 Aging Population 114 7.1.4 Orphan Designation 115 View Full Report with TOC @ http://www.radiantinsights.com/research/acute-myeloid-leukemiatherapeutics-market-to-2020-novel-therapies-to-offer-clinical-benefit-in-small-patient-cohorts 7.1.5 High Degree of Clinical Trial Participation 115 7.2 Market Barriers 115 7.2.1 Increasing Rates of Stem Cell Transplant 115 7.2.2 High Heterogeneity of the Disease 116 7.2.3 Lack of Standardized Treatment 116 7.2.4 Market Heavily Dominated by Generics 116 8 Strategic Consolidations 117 8.1 Co-development Deals 117 8.1.1 Ambit Biosciences Enters Co-development Agreement with Astellas 118 8.1.2 Equity Financing of BioTheryX by Leukemia and Lymphoma Society 118 8.1.3 Leukemia and Lymphoma Society Provide Funding for Celator's CPX-351 119 8.1.4 Ascenta Therapeutics Enter Agreement with Leukemia and Lymphoma Society 119 8.1.5 Stemline Therapeutics and MD Anderson Cancer Centre Enter Collaboration 119 8.2 Licensing Deals 119 8.2.1 EpiCept Sells European Rights of Ceplene to Meda 123 8.2.2 Biokine Therapeutics Out-License Development of BL-8040 to BioLineRx 123

8.2.3 Tolero In-License alvocidib from Sanofi 123 8.2.4 Bristol-Myers Squibb Enter Licensing Agreement with Innate Pharma for Lirilumab 124 8.2.5 Cell Therapeutics Enters Licensing Agreement with Chroma 124 8.2.6 Erytech Pharma Enters Licensing Agreement with Orphan Europe for Graspa 124 9 Appendix 125 9.1 All Pipeline Drugs by Phase 125 9.1.1 Discovery 125 9.1.2 Preclinical 127 9.1.3 IND/CTA-Filed 131 9.1.4 Phase I 132 9.1.5 Phase II 135 View Full Report with TOC @ http://www.radiantinsights.com/research/acute-myeloid-leukemiatherapeutics-market-to-2020-novel-therapies-to-offer-clinical-benefit-in-small-patient-cohorts 9.1.6 Phase III 141 9.2 Market Forecasts to 2020 142 9.2.1 Global 142 9.2.2 The US 142 9.2.3 Canada 142 9.2.4 The UK 143 9.2.5 France 143 9.2.6 Germany 144 9.2.7 Italy 144 9.2.8 Spain 145 9.2.9 Japan 145 9.3 References 146 9.4 Abbreviations 157

9.5 Research Methodology 159 9.5.1 Secondary Research 160 9.5.2 Marketed Product Profiles 160 9.5.3 Late-Stage Pipeline Candidates 160 9.5.4 Comparative Efficacy and Safety Heat Map for Marketed and Pipeline Products 160 9.5.5 Product Competitiveness Framework 161 9.5.6 Pipeline Analysis 161 9.5.7 Forecasting Model 162 9.5.8 Deals Data Analysis 163 9.6 Contact Us 163 9.7 Disclaimer 163 View Full Report with TOC @ http://www.radiantinsights.com/research/acute-myeloid-leukemiatherapeutics-market-to-2020-novel-therapies-to-offer-clinical-benefit-in-small-patient-cohorts List of Tables Table 1: Acute Myeloid Leukemia Therapeutics, French-American-British Classification, Incidence (%) and Prognosis, 1976-2014 16 Table 2: Acute Myeloid Leukemia Therapeutics, World Health Organization Classification, 2008 17 Table 3: Acute Myeloid Leukemia Therapeutics, Cytogenetic and Molecular Genetic Alterations and their Implications in Disease Prognosis 19 Table 4: Acute Myeloid Leukemia Therapeutics, Response Criteria (Remission or Treatment Failure) 23 Table 5: Acute Myeloid Leukemia Therapeutics, Survival and Relapse Rates by Risk Category, Prognosis 28 Table 6: Acute Myeloid Leukemia Therapeutics, Dosing Regimens of Conventional Care Regimes of Comparator Arm of One Phase III Clinical Trial of Vidaza, 2010 34 Table 7: Acute Myeloid Leukemia Therapeutics, Hazard Ratio for Allogeneic Stem Cell Transplants vs Chemotherapy and Autologous Stem Cell Transplant in Patients in First Remission, 2009 39 Table 8: Acute Myeloid Leukemia Therapeutics, Therapies Targeting CD123 in the Developmental Pipeline, 2013 61

Table 9: Acute Myeloid Leukemia Therapeutics, Therapies Targeting CD33 in the Developmental Pipeline, 2013 62 Table 10: Acute Myeloid Leukemia Therapeutics, Therapies Targeting Wilm's Tumor 1 in Developmental Pipeline, 2013 63 Table 11: Acute Myeloid Leukemia Therapeutics, Upstream and Downstream Pathway Components of FLT3, 2013 64 Table 12: Acute Myeloid Leukemia Therapeutics, Therapies Targeting FLT3 in Developmental Pipeline, 2013 65 Table 13: AML Therapeutics, Combination Therapies* in Current Developmental Pipeline 67 Table 14: Acute Myeloid Leukemia Therapeutics, Dosing Schedules in One Phase II Trial of Qinprezo, 2010 79 Table 15: Acute Myeloid Leukemia Therapeutics, Results of a Phase II Clinical Trial of Midostaurin, 2009 83 View Full Report with TOC @ http://www.radiantinsights.com/research/acute-myeloid-leukemiatherapeutics-market-to-2020-novel-therapies-to-offer-clinical-benefit-in-small-patient-cohorts Table 16: Acute Myeloid Leukemia Therapeutics, Therapies Targeting CD123 in the Developmental Pipeline, 2013 106 Table 17: Acute Myeloid Leukemia Therapeutics, Top Five European Markets, Annual Cost of Therapy, ($), 2013-2020 108 Table 18: Market for AML, Global, Developmental Pipeline, Discovery, 2013 - 2020 125 Table 19: Market for AML, Global, Developmental Pipeline, Preclinical, 2013 - 2020 127 Table 20: Market for AML, Global, Developmental Pipeline, IND/CTA-Filed, 2013 - 2020 131 Table 21: Market for AML, Global, Developmental Pipeline, Phase I, 2013 - 2020 132 Table 22: Market for AML, Global, Developmental Pipeline, Phase II, 2013 - 2020 135 Table 23: Market for AML, Global, Developmental Pipeline, Phase III, 2013 - 2020 141 Table 24: Acute Myeloid Leukemia Market, Global, Market Forecasts, 2013-2020 142 Table 25: Acute Myeloid Leukemia Market, US, Market Forecasts, 2013-2020 142 Table 26: Acute Myeloid Leukemia Market, Canada, Market Forecasts, 2013-2020 142 Table 27: Acute Myeloid Leukemia Market, UK, Market Forecasts, 2013-2020 143

Table 28: Acute Myeloid Leukemia Market, France, Market Forecasts, 2013-2020 143 Table 29: Acute Myeloid Leukemia Market, Germany, Market Forecasts, 2013-2020 144 Table 30: Acute Myeloid Leukemia Market, Italy, Market Forecasts, 2013-2020 144 Table 31: Acute Myeloid Leukemia Market, Spain, Market Forecasts, 2013-2020 145 Table 32: Acute Myeloid Leukemia Market, Japan, Market Forecasts, 2013-2020 14 View Full Report with TOC @ http://www.radiantinsights.com/research/acute-myeloid-leukemiatherapeutics-market-to-2020-novel-therapies-to-offer-clinical-benefit-in-small-patient-cohorts List of Figures Figure 1: Acute Myeloid Leukemia Therapeutics, Efficacy Results for Key Parameters - Marketed Products, Intensive Remission Induction Therapy 30 Figure 2: Acute Myeloid Leukemia Therapeutics, Efficacy Results for Key Parameters - Marketed Products, Non-Intensive Remission Induction Therapy 36 Figure 3: Acute Myeloid Leukemia Therapeutics, Efficacy Results for Key Parameters - Marketed Products, Salvage Therapy in Relapsed Disease 45 Figure 4: Acute Myeloid Leukemia Therapeutics, Treatment Algorithm 46 Figure 5: Acute Myeloid Leukemia Therapeutics, Global, Pipeline Distribution by Stage, Program Type and Route of Administration, 2013 58 Figure 6: Acute Myeloid Leukemia Therapeutics, Global, Pipeline by Molecule Type and Molecular Target, 2013 59 Figure 7: Acute Myeloid Leukemia Therapeutics, Global, Molecular Targets of Developmental Pipeline, 2013 66 Figure 8: Acute Myeloid Leukemia Therapeutics, Global, Clinical Trial Duration (Months), 2006-2013 69 Figure 9: AML Therapeutics, Global, Clinical Trial Size (Participants), 2006-2013 71 Figure 10: AML Therapeutics, Global, Clinical Trial Failure Rate and Reasons for Failure, 2006-2013 72 Figure 11: Acute Myeloid Leukemia Therapeutics, Clinical Trial Heat Map 73 Figure 12: Acute Myeloid Leukemia Therapeutics, Global, Forecast Revenues of CPX-351 ($m), 20142020 76 Figure 13: Acute Myeloid Leukemia Therapeutics, Global, Forecast Revenues of Midostaurin ($m), 2014-2020 85

Figure 14: Acute Myeloid Leukemia Therapeutics, Global, Forecast Revenues of Quizartinib ($m), 20162020 88 Figure 15: Acute Myeloid Leukemia Therapeutics, Global, Forecast Revenues of StemEx ($m), 20182020 91 Figure 16: Acute Myeloid Leukemia Therapeutics, Efficacy Results for Key Parameters - Pipeline (Blue) and Marketed Products Comparison. Intensive Remission Induction 94 Figure 17: Acute Myeloid Leukemia Therapeutics, Efficacy Results for Key Parameters - Pipeline (Blue) and Marketed Products Comparison. Non-Intensive Remission Induction 95 Figure 18: Acute Myeloid Leukemia Therapeutics, Efficacy Results for Key Parameters - Pipeline (Blue) and Marketed Products Comparison. Salvage Therapy for Relapsed Disease 96 Figure 19: Acute Myeloid Leukemia Therapeutics, Global, Treatment Usage Patterns ('000) and Market Revenues ($m), 2013-2020 100 View Full Report with TOC @ http://www.radiantinsights.com/research/acute-myeloid-leukemiatherapeutics-market-to-2020-novel-therapies-to-offer-clinical-benefit-in-small-patient-cohorts Figure 20: Acute Myeloid Leukemia Therapeutics, US, Treatment Usage Patterns ('000) and Annual Cost of Therapy ($), 2013-2020 102 Figure 21: Acute Myeloid Leukemia Therapeutics, US, Market Revenues ($m), 2013-2020 103 Figure 22: Acute Myeloid Leukemia Therapeutics, Canada, Treatment Usage Patterns and ACoT ('000; $), 2013-2020 105 Figure 23: Acute Myeloid Leukemia Therapeutics, Canada, Market Revenues ($m), 2013-2020 106 Figure 24: Acute Myeloid Leukemia Therapeutics, Top Five European Markets, Treatment Usage Patterns ('000), 2013-2020 107 Figure 25: Acute Myeloid Leukemia Therapeutics, Top Five European Markets, Annual Cost of Therapy ($), 2013-2020 109 Figure 26: Acute Myeloid Leukemia Therapeutics,Top Five European Markets, Market Revenues ($m), 2013-2020 110 Figure 27: Acute Myeloid Leukemia Therapeutics, Japan, Treatment Usage Patterns and Annual Cost of Therapy ('000; $), 2013-2020 112 Figure 28: Acute Myeloid Leukemia Therapeutics, Japan, Market Revenues ($m), 2013-2020 113 Figure 29: Acute Myeloid Leukemia Therapeutics, Global, Co-development Deals, 2006-2013 117

Figure 30: Acute Myeloid Leukemia Therapeutics, Global, Co-development Deals (Molecule Type and Mechanism of Action), 2006-2013 118 Figure 31: Acute Myeloid Leukemia Therapeutics, Global, Licensing Deals by Region, 2006-2013 120 Figure 32: Acute Myeloid Leukemia Therapeutics, Global, Licensing Deals by Value and Year, 20062013 121 Figure 33: Acute Myeloid Leukemia Therapeutics, Global, Licensing Deals by Molecule Type and Mechanism of Action, 2006 - 2013 122 View Full Report with TOC @ http://www.radiantinsights.com/research/acute-myeloid-leukemiatherapeutics-market-to-2020-novel-therapies-to-offer-clinical-benefit-in-small-patient-cohorts About Radiant Insights Radiant Insights is a platform for companies looking to meet their market research and business intelligence requirements. We assist and facilitate organizations and individuals procure market research reports, helping them in the decision making process. We have a comprehensive collection of reports, covering over 40 key industries and a host of micro markets. In addition to over extensive database of reports, our experienced research coordinators also offer a host of ancillary services such as, research partnerships/ tie-ups and customized research solutions.

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