Acute Myeloid Leukemia Therapeutics Market Size, Share, Growth Trend and Research Report 2020 Radiant Insights, Inc

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. 


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 


- 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 


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 


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 


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 


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 


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 


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


Pipeline, 2013 62 

Table 10: Acute Myeloid Leukemia Therapeutics, Therapies Targeting Wilm's Tumor 1 in Developmental 


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 




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 


List of Figures 

Figure 1: Acute Myeloid Leukemia Therapeutics, Efficacy Results for Key Parameters - Marketed 

Products, Intensive Remission Induction Therapy 30 


Products, Non-Intensive Remission Induction Therapy 36 


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), 2014- 

2020 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), 2016- 


Figure 15: Acute Myeloid Leukemia Therapeutics, Global, Forecast Revenues of StemEx ($m), 2018- 


Figure 16: Acute Myeloid Leukemia Therapeutics, Efficacy Results for Key Parameters - Pipeline (Blue) 

and Marketed Products Comparison. Intensive Remission Induction 94 


and Marketed Products Comparison. Non-Intensive Remission Induction 95 


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 


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, 2006- 

2013 121 

Figure 33: Acute Myeloid Leukemia Therapeutics, Global, Licensing Deals by Molecule Type and 

Mechanism of Action, 2006 - 2013 122 


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