OpportunityAnalyzer: Myelofibrosis – Opportunity Analysis and Forecasts to 2025

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.

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.

Key Highlights

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?

Companies mentioned

Incyte

Novartis

Bristol-Myers Squibb

Gilead

Johnson & Johnson

Geron

Promedior

CTI BioPharma

Shire

Amgen

Celgene

Merck & Co.

Roche

Acerta Pharma

Pfizer

MEI Pharma

Incyte

Novartis

Bristol-Myers Squibb

Gilead

Johnson & Johnson

Geron

Promedior

CTI BioPharma

Shire

Amgen

Celgene

Merck & Co.

Roche

Acerta Pharma

Pfizer

MEI Pharma

Table of Contents

1Table of Contents

1.1List of Tables

1.2List of Figures

2Introduction

2.1Catalyst

2.2Related Reports

2.3Upcoming Related Reports

3Disease Overview

3.1Etiology and Pathophysiology

3.1.1Etiology

3.1.2Pathophysiology

3.2Classification and ...

1Table of Contents

1.1List of Tables

1.2List of Figures

2Introduction

2.1Catalyst

2.2Related Reports

2.3Upcoming Related Reports

3Disease Overview

3.1Etiology and Pathophysiology

3.1.1Etiology

3.1.2Pathophysiology

3.2Classification and Prognosis

3.3Symptoms

3.4Quality of Life

4Epidemiology

4.1Risk Factors and Comorbidities

4.2Global Trends

4.2.1US

4.2.25EU

4.2.3Japan

4.3Forecast Methodology

4.3.1Sources Used

4.3.2Forecast Assumptions and Methods

4.3.3Sources Not Used

4.4Epidemiological Forecast of Myelofibrosis (2015–2025)

4.4.1Diagnosed Incident Cases

4.4.2Diagnosed Prevalent Cases

4.5Discussion

4.5.1Epidemiological Forecast Insight

4.5.2Limitations of the Analysis

4.5.3Strengths of the Analysis

5Current Treatment Options

5.1Overview

5.2Diagnosis and Treatment

5.2.1Diagnosis

5.2.2Treatment Guidelines and Leading Prescribed Drugs

5.2.3Clinical Practice

5.3Major Brands – JAK Inhibitors

5.3.1Jakafi (Ruxolitinib)

5.4Conventional Medical Therapy (Off-Label)

5.4.1Cytoreductive Drugs

5.4.2Androgen Therapies

5.4.3Erythropoiesis-Stimulating Agents

5.4.4Immunomodulatory Imide Drugs

5.4.5Anti-fibrotic Agents

6Unmet Needs Assessment and Opportunity Analysis

6.1Overview

6.2Development of Curative Treatments

6.2.1Unmet Need

6.2.2Gap Analysis

6.2.3Opportunity

6.3Treatments for MF Patients with Severe Thrombocytopenia

6.3.1Unmet Need

6.3.2Gap Analysis

6.3.3Opportunity

6.4Second-Line Treatments for MF Patients Refractory to Jakafi

6.4.1Unmet Need

6.4.2Gap Analysis

6.4.3Opportunity

6.5Approved Treatment for MF-Associated Anemia

6.5.1Unmet Need

6.5.2Gap Analysis

6.5.3Opportunity

6.6Effective Treatments with Better Long-Term Safety

6.6.1Unmet Need

6.6.2Gap Analysis

6.6.3Opportunity

7Research and Development Strategies

7.1Overview

7.1.1Targeting the JAK/STAT Signaling Pathway

7.1.2Developing Drugs that Reverse Bone Marrow Fibrosis

7.1.3Second-Line Therapies for Patients After Jakafi Treatment

7.1.4Novel Drugs in Combination with Jakafi

7.1.5Immuno-oncology Approach

7.2Clinical Trial Design

7.2.1Shifting Paradigm of Primary Endpoints Selection

7.2.2Inclusion of MF Patients with Severe Thrombocytopenia

7.2.3Selection of an Appropriate Comparator

8Pipeline Assessment

8.1Overview

8.2Promising Drugs in Clinical Development

8.2.1Momelotinib (GS-0387; CYT387)

8.2.2Imetelstat (JNJ-63935937; GRN-163L)

8.2.3PRM-151 (rhPTX-2)

8.2.4Pacritinib

8.3Innovative Early-Stage Approaches

8.3.1Programmed Cell Death Receptor-1/Programmed Cell Death Receptor Ligand-1 Inhibitors

8.3.2Hedgehog Pathway Inhibitors

8.3.3Jakafi Combination Therapies

8.4Other Drugs in Development

8.4.1ASP-0113 (TransVax, VCL-CB01)

9Pipeline Valuation Analysis

9.1Clinical Benchmark of Key Pipeline Drugs

9.2Commercial Benchmark of Key Pipeline Drugs

9.3Competitive Assessment

9.4Top-Line 10-Year Forecast

9.4.1US

9.4.25EU

9.4.3Japan

10Appendix

10.1Bibliography

10.2Abbreviations

10.3Methodology

10.4Forecasting Methodology

10.4.1General Forecast Assumptions

10.4.2Drugs Included in Each Therapeutic Class

10.4.3Key Launch Dates

10.4.4General Pricing Assumptions

10.4.5Individual Drug Assumptions

10.4.6Generic Erosion

10.4.7Pricing of Pipeline Agents

10.5Primary Research – KOLs Interviewed for this Report

10.6Primary Research – Prescriber Survey

10.7About the Authors

10.7.1Analyst

10.7.2Reviewer

10.7.3Therapy Area Director

10.7.4Epidemiologist

10.7.5Managing Epidemiologists

10.7.6Global Director of Therapy Analysis and Epidemiology

10.8About GlobalData

10.9Disclaimer

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: ...

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

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 ...

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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