OpportunityAnalyzer: Acute Myeloid Leukemia (AML) - Opportunity Analysis and Forecasts to 2017

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OpportunityAnalyzer: Acute Myeloid Leukemia (AML) - Opportunity Analysis and Forecasts to 2017

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OpportunityAnalyzer: Acute Myeloid Leukemia (AML) - Opportunity Analysis and Forecasts to 2017

Summary

Acute myeloid (myelogenous, myelocytic) leukemia is a rapidly progressing blood cancer with a poor overall prognosis. AML is relatively rare, and predominantly affects older adults. AML is a heterogeneous disease, and many subtypes have been identified. The current standard of care consists of cytarabine-based chemotherapy, which can often achieve remission. However, the majority of patients relapse, and survival rates are grim. There are clear unmet needs for treatments that can prolong the overall survival of these patients, but drug manufacturers have struggled to gain regulatory approval for new therapies.

This report focuses on the remaining opportunity in AML exclusive of the acute promyleocytic leukemia (APL) subtype.

Highlights

Key Questions Answered

- Which AML patients have the greatest unmet needs?

- What are the R&D strategies drug makers are pursuing in the AML space?

- Why has it been so difficult for Pharma to develop successful therapies for AML?

- How are players aiming to circumvent difficulties that have historically plagued AML drug development?

- What are the most promising pipeline agents for AML? How do their clinical and commercial attributes compare to one another?

- What opportunities will remain following the launch of these pipeline agents?

- Do KOLs see a role for targeted therapies and immunotherapies in the treatment of AML?

Key Findings

- GlobalData forecasts the AML therapeutics market in the 6MM to grow from $151.0m in 2012 to $430.7m in 2017, at a CAGR of 23.3%. The main driver of growth in the global AML market will be the uptake of premium-priced pipeline agents.

- Despite high levels of unmet need, uptake of new drugs will be limited, as KOLs are relatively unimpressed by these agents. Therefore, ample market opportunity will remain at the end of the forecast period.

- To circumvent historical difficulties with regulatory approval, companies are designing early- and late-stage clinical trials more rigorously. Randomization of early-stage trials is critical, and the designation of overall survival (OS) as the primary endpoint of pivotal studies is mandatory.

- Key R&D trends include developing therapies for the patients with greatest unmet need, particularly the elderly, developing combination therapies, and investigating new targets (i.e. FLT3) involved in the pathogenesis of AML.

- Despite the hype around FLT3 inhibitors, KOLs are unconvinced that these drugs will be successful as monotherapies in AML.

Scope

- Overview of AML, including epidemiology, etiology, pathophysiology, symptoms, diagnosis, and disease management.

- Topline AML therapeutics market revenue from 2012–2017. Annual cost of therapy, and peak pipeline drug sales of drugs launching during this forecast period are included.

- Key topics covered include strategic competitor assessment, market characterization, unmet needs, R&D strategies, and clinical trial design for the AML therapeutics market.

- Pipeline analysis: comprehensive data split across different phases, emerging novel trends under development, synopses of innovative early-stage projects, and detailed analysis of late-stage pipeline drugs. An interactive clinical and commercial analyzer tool is available.

- Analysis of the current and future market competition in the global AML therapeutics market. Insightful review of the key industry drivers, restraints and challenges. Each trend is independently researched to provide qualitative analysis of its implications.

Reasons to buy

- Develop and design your in-licensing and out-licensing strategies through a review of pipeline products and technologies, and by identifying the companies with the most robust pipeline.

- Develop business strategies by understanding the trends shaping and driving the global AML therapeutics market.

- Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the global AML therapeutics market in the future.

- Formulate effective sales and marketing strategies by understanding the competitive landscape and by analysing the performance of various competitors.

- Identify emerging players with potentially strong product portfolios and create effective counter-strategies to gain a competitive advantage.

- Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments and strategic partnerships.

1 Table of Contents

1 Table of Contents 9

1.1 List of Tables 14

1.2 List of Figures 17

2 Introduction 18

2.1 Catalyst 18

2.2 Upcoming Related Reports 18

3 Disease Overview 19

3.1 Etiology and Pathophysiology 19

3.1.1 Etiology 19

3.1.2 Pathophysiology 20

3.1.3 Classification 21

3.1.4 Cytogenetic and Molecular Abnormalities 23

3.1.5 Prognosis 30

3.1.6 Quality of Life 30

3.2 Symptoms 31

4 Epidemiology 32

4.1 Risk Factors and Comorbidities 33

4.1.1 Increased age is associated with increased risk and worsened prognosis 34

4.1.2 Smoking increases risk of M2 AML 35

4.1.3 APL is more common among Spanish and Italian origins 36

4.1.4 Chemotherapy and radiation therapy increases risk of AML 36

4.1.5 Benzene increases risk of AML 37

4.2 Global Trends 38

4.2.1 Incidence 38

4.2.2 Survival and Prevalence 40

4.2.3 Subtypes and Mutations 41

4.2.4 Risk Groups 43

4.3 Forecast Methodology 46

4.3.1 Sources Used 47

4.3.2 Forecast Assumptions and Methods 53

4.3.3 Sources Not Used 57

4.4 Epidemiological Forecast of Acute Myeloid Leukemia (2012–2022) 58

4.4.1 Incident Cases of AML 58

4.4.2 Incident Cases of AML by Age 59

4.4.3 Incident Cases of AML by Sex 61

4.4.4 Age-Standardized Incidence of AML 63

4.4.5 Incident Cases of AML Subtypes 64

4.4.6 Incident Cases of AML Subtypes by Age 68

4.4.7 Incident Cases of AML with Mutations in the FLT3 Gene 69

4.4.8 Incident Cases of AML by Risk Group Classifications 71

4.4.9 Five-Year Prevalent Cases of AML 75

4.4.10 Five-Year Prevalent Cases of AML by Age 78

4.4.11 Five-Year Prevalent Cases of APL and MDS/Therapy-Related AML 79

4.4.12 Five-Year Prevalent Cases of APL and MDS/Therapy-Related AML by Age 81

4.5 Discussion 83

4.5.1 Epidemiological Forecast Insight 83

4.5.2 Limitations of the Analysis 84

4.5.3 Strengths of the Analysis 84

5 Disease Management 85

5.1 Diagnosis 85

5.2 Response Criteria 86

5.3 General Treatment Overview 87

5.3.1 Younger AML Patients 89

5.3.2 Older AML Patients 91

5.4 Hematopoietic Stem Cell Transplant 93

5.5 Monitoring for Residual Disease 95

5.6 APL 96

6 Current Treatment Options 98

6.1 Overview 98

6.2 Product Profiles – Major Brands 101

6.2.1 Cytarabine 101

6.2.2 Dacogen (decitabine) 105

6.2.3 Vidaza (azacitidine) 110

6.2.4 Other Therapeutic Agents 115

7 Unmet Needs Assessment and Opportunity Analysis 117

7.1 Overview 117

7.2 Unmet Needs Analysis 118

7.2.1 Unmet Need: Therapies that Prolong the OS of AML Patients 118

7.2.2 Unmet Need: The Elimination of Residual Disease 120

7.2.3 Unmet Need: Therapies that Achieve Durable CR in AML Patients 121

7.2.4 Unmet Need: Safety 121

7.2.5 Unmet Need: Maintenance Therapies 122

7.2.6 Unmet Need: Guidelines Directing the Optimum Course of Therapy 123

7.2.7 Unmet Need: Therapies with More Convenient Dosing Regimens 123

7.2.8 Unmet Need: Lower Cost of Therapy 124

7.3 Opportunity Analysis 124

7.3.1 Opportunity: Targeting Minimal Residual Disease (Leukemic Stem Cells) 124

7.3.2 Opportunity: Targeting Driver Molecular Mutations with Combination Therapy 125

7.3.3 Opportunity: Development of Immunotherapies for AML 126

7.3.4 Opportunity: Differentiation Agents 126

7.3.5 Opportunity: Development of Oral Maintenance Therapies 127

8 R&D Strategies 128

8.1 Overview 128

8.1.1 Targeting Multiple Patient Segments 128

8.1.2 Developing Novel Drugs in Combination with Established Therapies 129

8.1.3 Targeting Elderly AML Patients 130

8.1.4 Investigating New Targets Involved in the Pathogenesis of AML 130

8.1.5 Alliances with Academic Groups 131

8.2 Clinical Trial Design 133

8.2.1 Selection of the Appropriate Efficacy Endpoints to Support Regulatory Approval 133

8.2.2 Selection of Active Comparator 136

8.2.3 Randomization and Stratification of Patients 137

8.2.4 Current Clinical Trial Design 139

8.2.5 Future Trends in Clinical Trial Design 141

9 Pipeline Assessment 144

9.1 Overview 144

9.2 Promising Drugs in Clinical Development 145

9.2.1 CC-486 (oral azacitidine) 146

9.2.2 CPX-351 149

9.2.3 Midostaurin 154

9.2.4 Quizartinib 158

9.2.5 Sapacitabine 164

9.2.6 Volasertib 168

9.2.7 Vosaroxin 172

9.3 Innovative Early-stage Approaches 176

9.3.1 Biologic-Targeted Therapy 177

9.3.2 Immunotherapies 179

9.3.3 Epigenetic Approaches 181

10 Pipeline Valuation Analysis 183

10.1 Clinical Benchmark of Key Pipeline Drugs 184

10.2 Commercial Benchmark of Key Pipeline Drugs 187

10.3 Competitive Assessment 189

10.4 Top Line Five-Year Forecast 190

10.4.1 US 192

10.4.2 5EU 194

11 Appendix 197

11.1 Bibliography 197

11.2 Abbreviations 215

11.3 Methodology 217

11.4 Forecasting Methodology 217

11.4.1 Diagnosed AML Patients 217

11.4.2 Percent Drug-treated Patients 218

11.4.3 Drugs Included in Each Therapeutic Class 218

11.4.4 Launch and Patent Expiry Dates 218

11.4.5 General Pricing Assumptions 219

11.4.6 Individual Drug Assumptions 220

11.4.7 Generic Erosion 223

11.4.8 Pricing of Pipeline Agents 223

11.5 Physicians and Specialists Included in this Study 224

11.6 About the Authors 226

11.6.1 Authors 226

11.6.2 Epidemiologist 227

11.6.3 Global Head of Healthcare 227

11.7 About GlobalData 228

11.8 Disclaimer 228

1.1 List of Tables

Table 1: Classification of AML Subtypes – WHO System 22

Table 2: Common Cytogenetic Abnormalities in AML 24

Table 3: Symptoms of AML 31

Table 4: Risk Factors and Comorbidities for AML in Adults 34

Table 5: Estimated Frequencies of Cytogenetic Abnormalities in AML 44

Table 6: Risk Group Classification Guidelines 45

Table 7: 6MM, Sources of AML Incidence 46

Table 8: 6MM, Incident Cases of AML, Men and Women Aged ?20 Years, N, Selected Years, 2012–2022 58

Table 9: 6MM, Incident Cases of AML by Age, Men and Women, N (row %), 2012 60

Table 10: 6MM, Incident Cases of AML by Sex, N (Row %), 2012 62

Table 11: 6MM, Incident Cases of APL, Men and Women Aged ?20 Years, N (Column %), Selected Years, 2012–2022 65

Table 12: 6MM, Incident Cases of MDS/Therapy-related AML, Men and Women Aged ?20 Years, N (Column %), Selected Years 2012–2022 66

Table 13: 6MM, Incident Cases of AML Subtypes by Age, Men and Women, N (Row % in Each Subtype), 2012 68

Table 14: 6MM, Incident Cases of AML with FLT3 Mutations, Men and Women Aged ?20, N (Column %), Selected Years 2012–2022 70

Table 15: 6MM, Risk Group Classification of AML Incident Cases, Men and Women Aged ?20 Years, N (Row %), 2012 72

Table 16: US, Risk Group Classification of AML Incident Cases by Age, Men and Women, N (Row %), 2012 74

Table 17: 6MM, Five-Year Prevalent Cases of AML, Men and Women Aged ?20 Years, N (Column %), Selected Years, 2012–2022 76

Table 18: 6MM, Five-Year Prevalent Cases of AML by Age, Men and Women, N (Row %), 2012 78

Table 19: 6MM, Five-Year Prevalent Cases of APL, Men and Women Aged ?20 Years, N (Column %), Selected Years 2012–2022 79

Table 20: 6MM, Five-Year Prevalent Cases of MDS/Therapy-Related AML, Men and Women Aged ?20 Years, N (Column %), Selected Years 2012–2022 80

Table 21: 6MM, Prevalent Cases of AML Subtypes by Age, Men and Women, N (Row % in Each Subtype), 2012 82

Table 22: Types of Responses – AML 87

Table 23: Induction and Consolidation Regimens for APL by Patient Risk Group 97

Table 24: Leading Treatments for AML 100

Table 25: Product Profile – Cytarabine (generic) 103

Table 26: Cytarabine SWOT Analysis 105

Table 27: Product Profile – Dacogen 107

Table 28: Dacogen SWOT Analysis 109

Table 29: Product Profile – Vidaza 112

Table 30: Vidaza SWOT Analysis 114

Table 31: Other Therapeutic Agents Prescribed for AML 116

Table 32: Overall Unmet Needs in AML – Current Level of Attainment 118

Table 33: Design of Current Phase III Trials in AML 139

Table 34: AML – Late-Stage Pipeline, 2013 145

Table 35: Product Profile – CC-486 147

Table 36: CC-486 SWOT Analysis 148

Table 37: Product Profile – CPX-351 150

Table 38: CPX-351 SWOT Analysis 153

Table 39: Product Profile – Midostaurin 155

Table 40: Midostaurin SWOT Analysis 157

Table 41: Product Profile – Quizartinib 160

Table 42: Quizartinib SWOT Analysis 163

Table 43: Product Profile – Sapacitabine 165

Table 44: Sapacitabine SWOT Analysis 167

Table 45: Product Profile – Volasertib 169

Table 46: Volasertib SWOT Analysis 171

Table 47: Product Profile – Vosaroxin 173

Table 48: Vosaroxin SWOT Analysis 175

Table 49: Early-stage Pipeline Products in AML 176

Table 50: Clinical Benchmark of Key Pipeline Drugs – Newly Diagnosed AML 184

Table 51: Clinical Benchmark of Key Pipeline Drugs – Relapsed/Refractory AML 185

Table 52: Clinical Benchmark of Key Pipeline Drugs - Newly Diagnosed AML 187

Table 53: Top Line Sales Forecasts ($m) for AML, 2012–2017 190

Table 54: Key Events Impacting Sales for AML, 2013 192

Table 55: AML Market in the US – Drivers and Barriers, 2013 194

Table 56: The 5EU AML Market – Drivers and Barriers, 2013 196

Table 57: Key Launch Dates 218

Table 58: Key Patent Expiries 219

1.2 List of Figures

Figure 1: Comparison of Normal and Leukemia Blood Cells 21

Figure 2: 6MM, Incident Cases of AML, Men and Women Aged ?20 Years, N, 2012 and 2022 33

Figure 3: 6MM, Incident Cases of AML, Men and Women Aged ?20 Years, N, 2012–2022 59

Figure 4: 6MM, Incident Cases of AML by Age, Men and Women Aged ?20 Years, N, 2012 61

Figure 5: 6MM, Incident Cases of AML by Sex, Aged ?20, N, 2012 63

Figure 6: 6MM, Age-Standardized Incidence of AML, Aged ?20 Years, 2012 64

Figure 7: 6MM, Incident Cases of APL and MDS/Therapy-Related AML, Men and Women Aged ?20, N, 2012 and 2022 67

Figure 8: 6MM, Incident Cases of AML Subtypes by Age, Men and Women, N, 2012 69

Figure 9: 6MM, Incident Cases of AML with FLT3 Mutations, Men and Women Aged ?20, N, 2012–2022 71

Figure 10: 6MM, Risk Group Classification of AML Incident Cases, Men and Women Aged ?20 Years, N, 2012 73

Figure 11: US, Risk Group Classification of AML Incident Cases by Age, Men and Women, N, 2012 74

Figure 12: 6MM, Five-Year Prevalent Cases of AML, Men and Women Aged ?20 Years, N, 2012–2022 77

Figure 13: 6MM, Prevalent Cases of AML Subtypes by Age, Men and Women, N, 2012 83

Figure 14: Competitive Assessment of Late-Stage Pipeline Agents in AML, 2012–2017 189

Figure 15: Global Sales for AML by Region, 2012–2017 191

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Pathology Industry: OpportunityAnalyzer: Acute Myeloid Leukemia (AML) - Opportunity Analysis and Forecasts to 2017

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