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OpportunityAnalyzer: Myelofibrosis - Opportunity Analysis and Forecasts to 2025

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
1.1 List of Tables
1.2 List of Figures
2 Introduction
2.1 Catalyst
2.2 Related Reports
2.3 Upcoming Related Reports
3 Disease Overview
3.1 Etiology and Pathophysiology
3.1.1 Etiology
3.1.2 Pathophysiology
3.2 Classification and Prognosis
3.3 Symptoms
3.4 Quality of Life
4 Epidemiology
4.1 Risk Factors and Comorbidities
4.2 Global Trends
4.2.1 US
4.2.2 5EU
4.2.3 Japan
4.3 Forecast Methodology
4.3.1 Sources Used
4.3.2 Forecast Assumptions and Methods
4.3.3 Sources Not Used
4.4 Epidemiological Forecast of Myelofibrosis (2015-2025)
4.4.1 Diagnosed Incident Cases
4.4.2 Diagnosed Prevalent Cases
4.5 Discussion
4.5.1 Epidemiological Forecast Insight
4.5.2 Limitations of the Analysis
4.5.3 Strengths of the Analysis
5 Current Treatment Options
5.1 Overview
5.2 Diagnosis and Treatment
5.2.1 Diagnosis
5.2.2 Treatment Guidelines and Leading Prescribed Drugs
5.2.3 Clinical Practice
5.3 Major Brands - JAK Inhibitors
5.3.1 Jakafi (Ruxolitinib)
5.4 Conventional Medical Therapy (Off-Label)
5.4.1 Cytoreductive Drugs
5.4.2 Androgen Therapies
5.4.3 Erythropoiesis-Stimulating Agents
5.4.4 Immunomodulatory Imide Drugs
5.4.5 Anti-fibrotic Agents
6 Unmet Needs Assessment and Opportunity Analysis
6.1 Overview
6.2 Development of Curative Treatments
6.2.1 Unmet Need
6.2.2 Gap Analysis
6.2.3 Opportunity
6.3 Treatments for MF Patients with Severe Thrombocytopenia
6.3.1 Unmet Need
6.3.2 Gap Analysis
6.3.3 Opportunity
6.4 Second-Line Treatments for MF Patients Refractory to Jakafi
6.4.1 Unmet Need
6.4.2 Gap Analysis
6.4.3 Opportunity
6.5 Approved Treatment for MF-Associated Anemia
6.5.1 Unmet Need
6.5.2 Gap Analysis
6.5.3 Opportunity
6.6 Effective Treatments with Better Long-Term Safety
6.6.1 Unmet Need
6.6.2 Gap Analysis
6.6.3 Opportunity
7 Research and Development Strategies
7.1 Overview
7.1.1 Targeting the JAK/STAT Signaling Pathway
7.1.2 Developing Drugs that Reverse Bone Marrow Fibrosis
7.1.3 Second-Line Therapies for Patients After Jakafi Treatment
7.1.4 Novel Drugs in Combination with Jakafi
7.1.5 Immuno-oncology Approach
7.2 Clinical Trial Design
7.2.1 Shifting Paradigm of Primary Endpoints Selection
7.2.2 Inclusion of MF Patients with Severe Thrombocytopenia
7.2.3 Selection of an Appropriate Comparator
8 Pipeline Assessment
8.1 Overview
8.2 Promising Drugs in Clinical Development
8.2.1 Momelotinib (GS-0387; CYT387)
8.2.2 Imetelstat (JNJ-63935937; GRN-163L)
8.2.3 PRM-151 (rhPTX-2)
8.2.4 Pacritinib
8.3 Innovative Early-Stage Approaches
8.3.1 Programmed Cell Death Receptor-1/Programmed Cell Death Receptor Ligand-1 Inhibitors
8.3.2 Hedgehog Pathway Inhibitors
8.3.3 Jakafi Combination Therapies
8.4 Other Drugs in Development
8.4.1 ASP-0113 (TransVax, VCL-CB01)
9 Pipeline Valuation Analysis
9.1 Clinical Benchmark of Key Pipeline Drugs
9.2 Commercial Benchmark of Key Pipeline Drugs
9.3 Competitive Assessment
9.4 Top-Line 10-Year Forecast
9.4.1 US
9.4.2 5EU
9.4.3 Japan
10 Appendix
10.1 Bibliography
10.2 Abbreviations
10.3 Methodology
10.4 Forecasting Methodology
10.4.1 General Forecast Assumptions
10.4.2 Drugs Included in Each Therapeutic Class
10.4.3 Key Launch Dates
10.4.4 General Pricing Assumptions
10.4.5 Individual Drug Assumptions
10.4.6 Generic Erosion
10.4.7 Pricing of Pipeline Agents
10.5 Primary Research - KOLs Interviewed for this Report
10.6 Primary Research - Prescriber Survey
10.7 About the Authors
10.7.1 Analyst
10.7.2 Reviewer
10.7.3 Therapy Area Director
10.7.4 Epidemiologist
10.7.5 Managing Epidemiologists
10.7.6 Global Director of Therapy Analysis and Epidemiology
10.8 About GlobalData
10.9 Disclaimer
1.1 List of Tables
Table 1: Risk Factors for MF Patient Survival
Table 2: Scoring Systems for Classifying MF Patients by Risk
Table 3: Symptoms of MF
Table 4: Risk Factors and Comorbidities of PMF
Table 5: 7MM, Diagnosed Incidence of PMF (Cases per 100,000 Population)
Table 6: 7MM, Sources Used to Forecast the Diagnosed Incident Cases of PMF
Table 7: 7MM, Sources Used to Forecast the Diagnosed Incident Cases of PMF by IPSS Risk Categorization
Table 8: 7MM, Sources Used to Forecast the Diagnosed Incident Cases of PMF by JAK2V617F Mutation
Table 9: 7MM, Sources Used to Forecast the Diagnosed Incident Cases of PMF by CALR/ASXL1 Mutations
Table 10: 7MM, Sources Used to Forecast the CALR/ASXL1 Mutation Cases by Molecular Risk
Table 11: 7MM, Sources Used to Forecast the Diagnosed Incident Cases of PET MF
Table 12: 7MM, Sources Used to Forecast the Diagnosed Incident Cases of PPV MF
Table 13: 7MM, Sources Used to Forecast the Diagnosed Prevalent Cases of PMF
Table 14: 7MM, Sources Used to Forecast the Diagnosed Prevalent Cases of PET MF
Table 15: 7MM, Sources Used to Forecast the Diagnosed Prevalent Cases of PPV MF
Table 16: 7MM, Diagnosed Incident Cases of PMF, Ages ≥40 Years, Both Sexes, N, Selected Years 2015-2025
Table 17: 7MM, Age-Specific Diagnosed Incident Cases of PMF, Both Sexes, N (Row %), 2015
Table 18: 7MM, Sex-Specific Diagnosed Incident Cases of PMF, Ages ≥40 Years, N (Row %), 2015
Table 19: 7MM, Diagnosed Incident Cases of PET MF, Ages ≥40 Years, Both Sexes, N, Selected Years 2015-2025
Table 20: 7MM, Sex-Specific Diagnosed Incident Cases of PET MF, Ages ≥40 Years, N (Row %), 2015
Table 21: 7MM, Diagnosed Incident Cases of PPV MF, Ages ≥40 Years, Both Sexes, N, Selected Years 2015-2025
Table 22: 7MM, Sex-Specific Diagnosed Incident Cases of PPV MF, Ages ≥40 Years, N (Row %), 2015
Table 23: 7MM, Diagnosed Prevalent Cases of PMF, Ages ≥40 Years, Both Sexes, N, Selected Years 2015-2025
Table 24: 7MM, Age-Specific Diagnosed Prevalent Cases of PMF, Both Sexes, N (Row %), 2015
Table 25: 7MM, Sex-Specific Diagnosed Prevalent Cases of PMF, Ages ≥40 Years, N (Row %), 2015
Table 26: 7MM, Diagnosed Prevalent Cases of PET MF, Ages ≥40 Years, Both Sexes, N, Selected Years 2015-2025
Table 27: 7MM, Sex-Specific Diagnosed Prevalent Cases of PET MF, Ages ≥40 Years, N (Row %), 2015
Table 28: 7MM, Diagnosed Prevalent Cases of PPV MF, Ages ≥40 Years, Both Sexes, N, Selected Years 2015-2025
Table 29: 7MM, Sex-Specific Diagnosed Prevalent Cases of PPV MF, Ages ≥40 Years, N (Row %), 2015
Table 30: Diagnostic Criteria for PMF, Post-PV MF, and Post-ET MF (WHO and IWG-MRT, 2008)
Table 31: WHO 2016 Diagnostic Criteria for PrePMF and Overt PMF
Table 32: Grading of MF
Table 33: Treatment Guidelines for MF
Table 34: Risk Factors for MF Patient Survival
Table 35: Leading Treatments for MF Used in Clinical Practice, 2016
Table 36: Product Profile - Jakafi
Table 37: Efficacy Data for Jakafi from the COMFORT-I Trial
Table 38: Efficacy Data from the Three-Year Followup of the COMFORT-I trial
Table 39: Efficacy Data for Patients Receiving Jakafi in the COMFORT-II Trial
Table 40: Key Safety Data for Jakafi from the COMFORT-I Trial
Table 41: Safety Data for Patients Receiving Jakafi from the Five-Year Follow-Up of the COMFORT-II Trial
Table 42: Jakafi SWOT Analysis, 2016
Table 43: Unmet Need and Opportunity in MF, 2015
Table 44: Key Phase III and Phase II Clinical Trials for MF
Table 45: Key Mid- and Late-Stage Pipeline Agents for MF, 2016
Table 46: Comparison of Promising Pipeline Agents in Development for MF, 2015-2025
Table 47: Product Profile - Momelotinib
Table 48: Efficacy Data for Momelotinib Based on a Phase I/II Trial
Table 49: Treatment-Emergent AEs Attributed to Momelotinib
Table 50: Momelotinib SWOT Analysis, 2016
Table 51: Product Profile - Imetelstat
Table 52: Efficacy of Imetelstat
Table 53: Safety of Imetelstat
Table 54: Pipeline Drug Imetelstat SWOT Analysis, 2016
Table 55: Product Profile - PRM-151 (rhPTX-2)
Table 56: Efficacy of PRM-151
Table 57: Pipeline Drug PRM-151 SWOT Analysis, 2016
Table 58: Product Profile - Pacritinib
Table 59: Efficacy of Pacritinib (PERSIST-1)
Table 60: Safety of Pacritinib (PERSIST-1)
Table 61: Other Drugs in Development for MF, 2016
Table 62: Clinical Benchmark of Key Pipeline Drugs - MF
Table 63: Commercial Benchmark of Key Pipeline Drugs - MF
Table 64: Top-Line Sales Forecasts ($m) for MF, 2015-2025
Table 65: Key Events Impacting Sales for MF, 2015-2025
Table 66: MF Market - Global Drivers and Barriers, 2015‒2025
Table 67: Sales Forecasts ($m) for MF in the US, 2015-2025
Table 68: Sales Forecasts ($m) for MF in the 5EU, 2015-2025
Table 69: Sales Forecasts ($m) for Myelofibrosis in the Japan, 2015-2025
Table 70: Key Historical and Projected Launch Dates for MF
Table 71: High-Prescribing Physicians (non-KOLs) Surveyed, By Country
1.2 List of Figures
Figure 1: 7MM, Diagnosed Incident Cases of PMF, Ages ≥40 Years, Both Sexes, N, 2015-2025
Figure 2: 7MM, Age-Specific Diagnosed Incident Cases of PMF, Both Sexes, N, 2015
Figure 3: 7MM, Sex-Specific Diagnosed Incident Cases of PMF, Ages ≥40 Years, 2015
Figure 4: 7MM, Age-Standardized Diagnosed Incidence of PMF, Ages ≥40 Years, Cases per 100,000 Population, 2015
Figure 5: 7MM, Diagnosed Incident Cases of PMF by IPSS Risk Categorization, Both Sexes, Ages ≥40 Years, N, 2015
Figure 6: 7MM, Diagnosed Incident Cases of PMF by JAK2V617F and CALR/ASXL1 Mutations, Both Sexes, Ages ≥40 Years, N, 2015
Figure 7: 7MM, CALR/ASXL1 Mutation Cases by Molecular Risk, Both Sexes, Ages ≥40 Years, N, 2015
Figure 8: 7MM, Diagnosed Incident Cases of PET MF, Ages ≥40 Years, Both Sexes, N, 2015-2025
Figure 9: 7MM, Sex-Specific Diagnosed Incident Cases of PET MF, Ages ≥40 Years, 2015
Figure 10: 7MM, Diagnosed Incident Cases of PPV MF, Ages ≥40 Years, Both Sexes, N, 2015-2025
Figure 11: 7MM, Sex-Specific Diagnosed Incident Cases of PPV MF, Ages ≥40 Years, 2015
Figure 12: 7MM, Diagnosed Prevalent Cases of PMF, Ages ≥40 Years, Both Sexes, N, Selected Years 2015-2025
Figure 13: 7MM, Age-Specific Diagnosed Prevalent Cases of PMF, Both Sexes, N, 2015
Figure 14: 7MM, Sex-Specific Diagnosed Prevalent Cases of PMF, Ages ≥40 Years, 2015
Figure 15: 7MM, Age-Standardized Diagnosed Prevalence of PMF, Ages ≥40 Years, %, 2015
Figure 16: 7MM, Diagnosed Prevalent Cases of PET MF, Ages ≥40 Years, Both Sexes, N, Selected Years 2015-2025
Figure 17: 7MM, Sex-Specific Diagnosed Prevalent Cases of PET MF, Ages ≥40 Years, 2015
Figure 18: 7MM, Diagnosed Prevalent Cases of PPV MF, Ages ≥40 Years, Both Sexes, N, Selected Years 2015-2025
Figure 19: 7MM, Sex-Specific Diagnosed Prevalent Cases of PPV MF, Ages ≥40 Years, 2015
Figure 20: MF Treatment Flow
Figure 21: Jakafi’s Development in MF
Figure 22: MF - Phase II-III Pipeline, 2016
Figure 23: Momelotinib: Clinical Development in MF
Figure 24: Imetelstat: Clinical Development in MF
Figure 25: PRM-151: Phase II Development in MF
Figure 26: Competitive Assessment of Marketed and Pipeline Agents in MF, 2015-2025
Figure 27: Top-Line Sales for MF by Region, 2015 and 2025
Figure 28: Top-Line Sales for MF by Region, 2015‒2025
Figure 29: Sales for MF by Drug Class in the US, 2015 and 2025
Figure 30: Sales for MF by Drug Class in the 5EU, 2015, and 2025
Figure 31: Sales for MF by Drug Class in the Japan, 2015 and 2025

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