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OpportunityAnalyzer: Myelofibrosis - Opportunity Analysis and Forecasts to 2025

Published By :

GlobalData

Published Date : Nov 2016

Category :

Pharmaceutical

No. of Pages : 250 Pages

OpportunityAnalyzer: Myelofibrosis - Opportunity Analysis and Forecasts to 2025

Summary

Myelofibrosis (MF) is a rare blood disorder, which is characterized by bone marrow fibrosis. Currently, there is only one approved drug, Incyte/Novartis Jakafi (ruxolitinib), for the treatment of MF, and other conventional therapies used in MF are off-label. However, none of these drugs are curative, and the only potentially curative intervention is allogeneic stem cell transplant (allo-SCT), which is available to a very small percentage of eligible patients because of the high risk of morbidity and mortality. Therefore, there is a huge unmet need for the treatment of MF.

This report highlights the significant unmet need for novel drug treatment for MF, both to alleviate MF-associated complications and to reverse the disease course, across the seven major markets; it also discusses the associated commercial opportunities for new market entrants to gain a foothold in the market. GlobalData anticipate the MF market to almost double, from $545.2m to $1.01 billion, over the forecast period of 2015-2025. The key drivers wills be the launch of pipeline drugs, increasing incidence and an increase in the use of drugs for splenomegaly and constitutional symptoms in the 5EU and Japan.

Highlights

Key Questions Answered

- The MF market has high unmet need. What are the main unmet needs in this market? How will the drugs under development fulfil the unmet needs of the MF market?
- There are three middle- to late-stage MF pipeline drugs expected to launch during the forecast period. Will these drugs make a significant impact on the MF market? Which of these drugs will have the deepest patient penetration and highest peak sales, and why?
- The current MF market is dominated by one JAK inhibitor, Jakafi. How will the launch of pipeline drugs with novel mechanism of action change this? How will the way MF patients are treated change over the next years? What are the key drivers and barriers to this change?

Key Findings

- The launch of premium priced products, in particular second-line treatments for patients who are refractory to Jakafi, will be the main drivers of growth in he MF market.
- There are high unmet needs in MF. The biggest unmet need is for curative treatments. The unmet needs will only be partially addressed by the major pipeline drugs. In addition, there are currently no approved or major pipeline drugs for MF-associated anemia. Any drug that can get approved in this setting can expect a lucrative return.
- Key Opinion Leaders urged pharma companies to focus their R&D strategies on trying to reverse the disease course of MF. This will involve collaborating with academics to identify new molecular targets.
- One key R&D strategy will be developing drugs that reverse bone marrow fibrosis.

Scope

- Overview of MF, including epidemiology, etiology, pathophysiology, symptoms, diagnosis, treatment guidelines and disease management.
- Annualized MF therapeutics market revenue, average cost of therapy and treatment usage pattern data from 2015 and forecast for seven years to 2025.
- Key topics covered include strategic competitor assessment, market characterization, unmet needs, clinical trial mapping and implications for the MF therapeutics market.
- Pipeline analysis: comprehensive data split across different phases, emerging novel trends under development, and detailed analysis of middle- to late-stage pipeline drugs.
- Analysis of the current and future market competition in the global MF 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

The report will enable you to -
- 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. Additionally a list of acquisition targets included in the pipeline product company list.
- Develop business strategies by understanding the trends shaping and driving the global MF therapeutics market.
- Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the global MF therapeutics market in 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.
- Track drug sales in the global MF therapeutics market from 2015-2025.
- 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 19
2.1 Catalyst 19
2.2 Related Reports 19
2.3 Upcoming Related Reports 20
3 Disease Overview 21
3.1 Etiology and Pathophysiology 21
3.1.1 Etiology 22
3.1.2 Pathophysiology 23
3.2 Classification and Prognosis 25
3.3 Symptoms 27
3.4 Quality of Life 30
4 Epidemiology 31
4.1 Risk Factors and Comorbidities 32
4.2 Global Trends 34
4.2.1 US 35
4.2.2 5EU 37
4.2.3 Japan 37
4.3 Forecast Methodology 37
4.3.1 Sources Used 37
4.3.2 Forecast Assumptions and Methods 48
4.3.3 Sources Not Used 61
4.4 Epidemiological Forecast of Myelofibrosis (2015-2025) 62
4.4.1 Diagnosed Incident Cases 62
4.4.2 Diagnosed Prevalent Cases 77
4.5 Discussion 90
4.5.1 Epidemiological Forecast Insight 90
4.5.2 Limitations of the Analysis 91
4.5.3 Strengths of the Analysis 92
5 Current Treatment Options 93
5.1 Overview 93
5.2 Diagnosis and Treatment 94
5.2.1 Diagnosis 94
5.2.2 Treatment Guidelines and Leading Prescribed Drugs 99
5.2.3 Clinical Practice 100
5.3 Major Brands - JAK Inhibitors 108
5.3.1 Jakafi (Ruxolitinib) 108
5.4 Conventional Medical Therapy (Off-Label) 120
5.4.1 Cytoreductive Drugs 120
5.4.2 Androgen Therapies 121
5.4.3 Erythropoiesis-Stimulating Agents 124
5.4.4 Immunomodulatory Imide Drugs 124
5.4.5 Anti-fibrotic Agents 126
6 Unmet Needs Assessment and Opportunity Analysis 127
6.1 Overview 127
6.2 Development of Curative Treatments 128
6.2.1 Unmet Need 128
6.2.2 Gap Analysis 129
6.2.3 Opportunity 130
6.3 Treatments for MF Patients with Severe Thrombocytopenia 131
6.3.1 Unmet Need 131
6.3.2 Gap Analysis 131
6.3.3 Opportunity 132
6.4 Second-Line Treatments for MF Patients Refractory to Jakafi 132
6.4.1 Unmet Need 132
6.4.2 Gap Analysis 133
6.4.3 Opportunity 133
6.5 Approved Treatment for MF-Associated Anemia 134
6.5.1 Unmet Need 134
6.5.2 Gap Analysis 134
6.5.3 Opportunity 135
6.6 Effective Treatments with Better Long-Term Safety 135
6.6.1 Unmet Need 135
6.6.2 Gap Analysis 136
6.6.3 Opportunity 137
7 Research and Development Strategies 138
7.1 Overview 138
7.1.1 Targeting the JAK/STAT Signaling Pathway 138
7.1.2 Developing Drugs that Reverse Bone Marrow Fibrosis 139
7.1.3 Second-Line Therapies for Patients After Jakafi Treatment 139
7.1.4 Novel Drugs in Combination with Jakafi 140
7.1.5 Immuno-oncology Approach 141
7.2 Clinical Trial Design 142
7.2.1 Shifting Paradigm of Primary Endpoints Selection 144
7.2.2 Inclusion of MF Patients with Severe Thrombocytopenia 145
7.2.3 Selection of an Appropriate Comparator 146
8 Pipeline Assessment 147
8.1 Overview 147
8.2 Promising Drugs in Clinical Development 147
8.2.1 Momelotinib (GS-0387; CYT387) 151
8.2.2 Imetelstat (JNJ-63935937; GRN-163L) 160
8.2.3 PRM-151 (rhPTX-2) 168
8.2.4 Pacritinib 175
8.3 Innovative Early-Stage Approaches 181
8.3.1 Programmed Cell Death Receptor-1/Programmed Cell Death Receptor Ligand-1 Inhibitors 181
8.3.2 Hedgehog Pathway Inhibitors 182
8.3.3 Jakafi Combination Therapies 183
8.4 Other Drugs in Development 188
8.4.1 ASP-0113 (TransVax, VCL-CB01) 189
9 Pipeline Valuation Analysis 191
9.1 Clinical Benchmark of Key Pipeline Drugs 191
9.2 Commercial Benchmark of Key Pipeline Drugs 193
9.3 Competitive Assessment 195
9.4 Top-Line 10-Year Forecast 197
9.4.1 US 201
9.4.2 5EU 205
9.4.3 Japan 208
10 Appendix 211
10.1 Bibliography 211
10.2 Abbreviations 228
10.3 Methodology 233
10.4 Forecasting Methodology 233
10.4.1 General Forecast Assumptions 233
10.4.2 Drugs Included in Each Therapeutic Class 235
10.4.3 Key Launch Dates 235
10.4.4 General Pricing Assumptions 235
10.4.5 Individual Drug Assumptions 237
10.4.6 Generic Erosion 240
10.4.7 Pricing of Pipeline Agents 240
10.5 Primary Research - KOLs Interviewed for this Report 242
10.6 Primary Research - Prescriber Survey 244
10.7 About the Authors 245
10.7.1 Analyst 245
10.7.2 Reviewer 245
10.7.3 Therapy Area Director 246
10.7.4 Epidemiologist 247
10.7.5 Managing Epidemiologists 247
10.7.6 Global Director of Therapy Analysis and Epidemiology 248
10.8 About GlobalData 249
10.9 Disclaimer 249

1.1 List of Tables
Table 1: Risk Factors for MF Patient Survival 26
Table 2: Scoring Systems for Classifying MF Patients by Risk 26
Table 3: Symptoms of MF 29
Table 4: Risk Factors and Comorbidities of PMF 33
Table 5: 7MM, Diagnosed Incidence of PMF (Cases per 100,000 Population) 35
Table 6: 7MM, Sources Used to Forecast the Diagnosed Incident Cases of PMF 39
Table 7: 7MM, Sources Used to Forecast the Diagnosed Incident Cases of PMF by IPSS Risk Categorization 40
Table 8: 7MM, Sources Used to Forecast the Diagnosed Incident Cases of PMF by JAK2V617F Mutation 40
Table 9: 7MM, Sources Used to Forecast the Diagnosed Incident Cases of PMF by CALR/ASXL1 Mutations 41
Table 10: 7MM, Sources Used to Forecast the CALR/ASXL1 Mutation Cases by Molecular Risk 41
Table 11: 7MM, Sources Used to Forecast the Diagnosed Incident Cases of PET MF 42
Table 12: 7MM, Sources Used to Forecast the Diagnosed Incident Cases of PPV MF 44
Table 13: 7MM, Sources Used to Forecast the Diagnosed Prevalent Cases of PMF 45
Table 14: 7MM, Sources Used to Forecast the Diagnosed Prevalent Cases of PET MF 46
Table 15: 7MM, Sources Used to Forecast the Diagnosed Prevalent Cases of PPV MF 47
Table 16: 7MM, Diagnosed Incident Cases of PMF, Ages 40 Years, Both Sexes, N, Selected Years 2015-2025 62
Table 17: 7MM, Age-Specific Diagnosed Incident Cases of PMF, Both Sexes, N (Row %), 2015 64
Table 18: 7MM, Sex-Specific Diagnosed Incident Cases of PMF, Ages 40 Years, N (Row %), 2015 66
Table 19: 7MM, Diagnosed Incident Cases of PET MF, Ages 40 Years, Both Sexes, N, Selected Years 2015-2025 72
Table 20: 7MM, Sex-Specific Diagnosed Incident Cases of PET MF, Ages 40 Years, N (Row %), 2015 73
Table 21: 7MM, Diagnosed Incident Cases of PPV MF, Ages 40 Years, Both Sexes, N, Selected Years 2015-2025 75
Table 22: 7MM, Sex-Specific Diagnosed Incident Cases of PPV MF, Ages 40 Years, N (Row %), 2015 76
Table 23: 7MM, Diagnosed Prevalent Cases of PMF, Ages 40 Years, Both Sexes, N, Selected Years 2015-2025 78
Table 24: 7MM, Age-Specific Diagnosed Prevalent Cases of PMF, Both Sexes, N (Row %), 2015 79
Table 25: 7MM, Sex-Specific Diagnosed Prevalent Cases of PMF, Ages 40 Years, N (Row %), 2015 81
Table 26: 7MM, Diagnosed Prevalent Cases of PET MF, Ages 40 Years, Both Sexes, N, Selected Years 2015-2025 84
Table 27: 7MM, Sex-Specific Diagnosed Prevalent Cases of PET MF, Ages 40 Years, N (Row %), 2015 85
Table 28: 7MM, Diagnosed Prevalent Cases of PPV MF, Ages 40 Years, Both Sexes, N, Selected Years 2015-2025 87
Table 29: 7MM, Sex-Specific Diagnosed Prevalent Cases of PPV MF, Ages 40 Years, N (Row %), 2015 89
Table 30: Diagnostic Criteria for PMF, Post-PV MF, and Post-ET MF (WHO and IWG-MRT, 2008) 96
Table 31: WHO 2016 Diagnostic Criteria for PrePMF and Overt PMF 97
Table 32: Grading of MF 97
Table 33: Treatment Guidelines for MF 99
Table 34: Risk Factors for MF Patient Survival 104
Table 35: Leading Treatments for MF Used in Clinical Practice, 2016 107
Table 36: Product Profile - Jakafi 108
Table 37: Efficacy Data for Jakafi from the COMFORT-I Trial 112
Table 38: Efficacy Data from the Three-Year Followup of the COMFORT-I trial 113
Table 39: Efficacy Data for Patients Receiving Jakafi in the COMFORT-II Trial 114
Table 40: Key Safety Data for Jakafi from the COMFORT-I Trial 117
Table 41: Safety Data for Patients Receiving Jakafi from the Five-Year Follow-Up of the COMFORT-II Trial 118
Table 42: Jakafi SWOT Analysis, 2016 120
Table 43: Unmet Need and Opportunity in MF, 2015 128
Table 44: Key Phase III and Phase II Clinical Trials for MF 143
Table 45: Key Mid- and Late-Stage Pipeline Agents for MF, 2016 148
Table 46: Comparison of Promising Pipeline Agents in Development for MF, 2015-2025 150
Table 47: Product Profile - Momelotinib 152
Table 48: Efficacy Data for Momelotinib Based on a Phase I/II Trial 156
Table 49: Treatment-Emergent AEs Attributed to Momelotinib 159
Table 50: Momelotinib SWOT Analysis, 2016 160
Table 51: Product Profile - Imetelstat 161
Table 52: Efficacy of Imetelstat 165
Table 53: Safety of Imetelstat 167
Table 54: Pipeline Drug Imetelstat SWOT Analysis, 2016 168
Table 55: Product Profile - PRM-151 (rhPTX-2) 169
Table 56: Efficacy of PRM-151 172
Table 57: Pipeline Drug PRM-151 SWOT Analysis, 2016 175
Table 58: Product Profile - Pacritinib 176
Table 59: Efficacy of Pacritinib (PERSIST-1) 180
Table 60: Safety of Pacritinib (PERSIST-1) 181
Table 61: Other Drugs in Development for MF, 2016 188
Table 62: Clinical Benchmark of Key Pipeline Drugs - MF 191
Table 63: Commercial Benchmark of Key Pipeline Drugs - MF 193
Table 64: Top-Line Sales Forecasts ($m) for MF, 2015-2025 198
Table 65: Key Events Impacting Sales for MF, 2015-2025 200
Table 66: MF Market - Global Drivers and Barriers, 20152025 201
Table 67: Sales Forecasts ($m) for MF in the US, 2015-2025 203
Table 68: Sales Forecasts ($m) for MF in the 5EU, 2015-2025 206
Table 69: Sales Forecasts ($m) for Myelofibrosis in the Japan, 2015-2025 209
Table 70: Key Historical and Projected Launch Dates for MF 235
Table 71: High-Prescribing Physicians (non-KOLs) Surveyed, By Country 244

1.2 List of Figures
Figure 1: 7MM, Diagnosed Incident Cases of PMF, Ages 40 Years, Both Sexes, N, 2015-2025 63
Figure 2: 7MM, Age-Specific Diagnosed Incident Cases of PMF, Both Sexes, N, 2015 65
Figure 3: 7MM, Sex-Specific Diagnosed Incident Cases of PMF, Ages 40 Years, 2015 67
Figure 4: 7MM, Age-Standardized Diagnosed Incidence of PMF, Ages 40 Years, Cases per 100,000 Population, 2015 68
Figure 5: 7MM, Diagnosed Incident Cases of PMF by IPSS Risk Categorization, Both Sexes, Ages 40 Years, N, 2015 69
Figure 6: 7MM, Diagnosed Incident Cases of PMF by JAK2V617F and CALR/ASXL1 Mutations, Both Sexes, Ages 40 Years, N, 2015 70
Figure 7: 7MM, CALR/ASXL1 Mutation Cases by Molecular Risk, Both Sexes, Ages 40 Years, N, 2015 71
Figure 8: 7MM, Diagnosed Incident Cases of PET MF, Ages 40 Years, Both Sexes, N, 2015-2025 72
Figure 9: 7MM, Sex-Specific Diagnosed Incident Cases of PET MF, Ages 40 Years, 2015 74
Figure 10: 7MM, Diagnosed Incident Cases of PPV MF, Ages 40 Years, Both Sexes, N, 2015-2025 75
Figure 11: 7MM, Sex-Specific Diagnosed Incident Cases of PPV MF, Ages 40 Years, 2015 77
Figure 12: 7MM, Diagnosed Prevalent Cases of PMF, Ages 40 Years, Both Sexes, N, Selected Years 2015-2025 78
Figure 13: 7MM, Age-Specific Diagnosed Prevalent Cases of PMF, Both Sexes, N, 2015 80
Figure 14: 7MM, Sex-Specific Diagnosed Prevalent Cases of PMF, Ages 40 Years, 2015 82
Figure 15: 7MM, Age-Standardized Diagnosed Prevalence of PMF, Ages 40 Years, %, 2015 83
Figure 16: 7MM, Diagnosed Prevalent Cases of PET MF, Ages 40 Years, Both Sexes, N, Selected Years 2015-2025 84
Figure 17: 7MM, Sex-Specific Diagnosed Prevalent Cases of PET MF, Ages 40 Years, 2015 86
Figure 18: 7MM, Diagnosed Prevalent Cases of PPV MF, Ages 40 Years, Both Sexes, N, Selected Years 2015-2025 88
Figure 19: 7MM, Sex-Specific Diagnosed Prevalent Cases of PPV MF, Ages 40 Years, 2015 90
Figure 20: MF Treatment Flow 105
Figure 21: Jakafis Development in MF 111
Figure 22: MF - Phase II-III Pipeline, 2016 151
Figure 23: Momelotinib: Clinical Development in MF 155
Figure 24: Imetelstat: Clinical Development in MF 164
Figure 25: PRM-151: Phase II Development in MF 171
Figure 26: Competitive Assessment of Marketed and Pipeline Agents in MF, 2015-2025 197
Figure 27: Top-Line Sales for MF by Region, 2015 and 2025 199
Figure 28: Top-Line Sales for MF by Region, 20152025 200
Figure 29: Sales for MF by Drug Class in the US, 2015 and 2025 204
Figure 30: Sales for MF by Drug Class in the 5EU, 2015, and 2025 207
Figure 31: Sales for MF by Drug Class in the Japan, 2015 and 2025 210

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