OpportunityAnalyzer: Myelofibrosis - Opportunity Analysis and Forecasts to 2025

  • ID: 4051394
  • Drug Pipelines
  • 250 pages
  • GlobalData
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Myelofibrosis Market will Exceed $1 Billion by 2025

FEATURED COMPANIES

  • Acerta Pharma
  • Bristol-Myers Squibb
  • CTI BioPharma
  • Gilead
  • Johnson & Johnson
  • Merck & Co.
  • MORE
Myelofibrosis (MF, also called osteomyelofibrosis) is a rare blood disorder that causes bone marrow (BM) fibrosis. MF is classified as a chronic myeloproliferative neoplasm (MPN), a group of heterogeneous hematopoietic stem cell malignancies, characterized by excessive production of mature blood cells of myeloid lineage, presence of somatic mutations, dysregulated kinase signaling, and release of abnormal cytokines (Barcelos and Santos-Silva, 2010; Cervantes, 2014; NORD, 2016). MF that develops de novo, in the absence of any other conditions, from clonal proliferation of an abnormal pluripotent stem cell in the BM, is called primary myelofibrosis (PMF), also known as chronic idiopathic myelofibrosis.

MF market in the 7MM is valued at US$545.2m. The market is defined as sales from approved branded drugs as well as off-label drugs used for the treatment of MF. The majority of 2015 sales, US$405.5m (74.4%), were generated in the US, while sales in the 5EU and Japan were estimated to be US$133.2m (24.4%) and USD6.5m (1.2%), respectively.

By 2025, the MF market is expected to grow to US$1.02 billion across the 7MM at a Compound Annual Growth Rate (CAGR) of 6.4%. At the end of the forecast period, the majority of sales, US$737m (72.5%), will still be generated in the US, and sales in the 5EU and Japan are forecast to be US$265.6m (26.1%) and US$13.4m (1.3%), respectively.

Major drivers of the growth of the MF market in the 7MM over the forecast period will include:

- The launch of pipeline agents, including Gilead’s momelotinib, Promedior’s PRM-151, and Johnson & Johnson(J&J)/Geron’s imetelstat. These pipeline drugs are expected to command a premium price compared to existing products and off-label drugs, and their uptake is expected to drive the overall growth of the MF market. The availability of new drugs will also increase the number of lines of therapy that MF patients receive.

- An increase in the incidence of MF from 3,791 new cases in 2015 to just over 4,300 new cases in 2025, at an Annual Growth Rate (AGR) of 1.5%, as forecast by the epidemiology team. This increase, coupled with an anticipated increase in branded therapy prescription, will drive growth of the global MF market over the forecast period.

- An increase in the use of drugs for the treatment of splenomegaly and constitutional symptoms in the 5EU and Japan over the forecast period. The author’s primary research showed that this market segment has not been saturated in the base year.

As per James Beggs, Analyst, explains: “Incyte/Novartis’ Jakafi is currently the only drug approved for the treatment of myelofibrosis-associated splenomegaly and constitutional symptoms in the 7MM. Currently, there are no drugs approved for patients who are refractory or become unresponsive to Jakafi treatment, and momelotinib and imetelstat are expected to contribute towards addressing this issue with launches in the second-line setting in 2017 and 2021, respectively.”

The report “OpportunityAnalyzer: Myelofibrosis - Opportunity Analysis and Forecasts to 2025” provides an overview of MF, including epidemiology, etiology, pathophysiology, symptoms, diagnosis, treatment guidelines and disease management.

In depth, this report provides the following analysis:
- Detailed analysis of MF therapeutics market revenue, average cost of therapy and treatment usage pattern data from 2015 and forecast for seven years to 2025.
- Covers strategic competitor assessment, market characterization, unmet needs, clinical trial mapping and implications for the MF therapeutics market.
- Provides 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.

Companies mentioned in this report: Incyte, Novartis, Bristol-Myers Squibb, Gilead, Johnson & Johnson, Geron, Promedior, CTI BioPharma, Shire, Amgen, Celgene, Merck & Co., Roche, Acerta Pharma, Pfizer, MEI Pharma.
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FEATURED COMPANIES

  • Acerta Pharma
  • Bristol-Myers Squibb
  • CTI BioPharma
  • Gilead
  • Johnson & Johnson
  • Merck & Co.
  • MORE
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

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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FEATURED COMPANIES

  • Acerta Pharma
  • Bristol-Myers Squibb
  • CTI BioPharma
  • Gilead
  • Johnson & Johnson
  • Merck & Co.
  • MORE
Myelofibrosis market will exceed $1 billion by 2025.

The myelofibrosis market is expected to almost double in value from $545.2 million in 2015 to $1.02 billion by 2025, representing a compound annual growth rate of 6.4%, according to research.

The company’s latest report states that the strength of this growth, which covers the seven major markets (7MM) of the US, France, Germany, Italy, Spain, the UK, and Japan, will primarily be driven by the launch of pipeline agents, including Gilead’s momelotinib, Promedior’s PRM-151, and Johnson & Johnson/Geron’s imetelstat. Other factors include an increase in the incidence of myelofibrosis, and a rise in the use of drugs for the treatment of splenomegaly and constitutional symptoms.

James Beggs, Ph.D., the Analyst covering Oncology and Hematology, explains: “Incyte/Novartis’ Jakafi is currently the only drug approved for the treatment of myelofibrosis-associated splenomegaly and constitutional symptoms in the 7MM. Currently, there are no drugs approved for patients who are refractory or become unresponsive to Jakafi treatment, and momelotinib and imetelstat are expected to contribute towards addressing this issue with launches in the second-line setting in 2017 and 2021, respectively.”

Despite this rising competition from pipeline agents, Jakafi is expected to retain its leading spot in the market, with just over 50% of the market share in 2025. The author expects that momelotinib can ultimately only capture about 25% of Jakafi’s patient share in the first-line setting, as Jakafi has the first-to-market advantage, and long-term efficacy and safety data are available.

Beggs continues: “Imetelstat, a first-in-class telomerase inhibitor, is expected to struggle for market penetration due to late market entry and the high level of toxicity shown in previous clinical trials. The author estimates sales of the drug in 2025 to be only $21.2 million, representing just 2% of the myelofibrosis market in the 7MM.

“PRM-151, however, looks more promising in terms of expected sales. If the drug manages to demonstrate anti-fibrotic effects in myelofibrosis patients in a pivotal trial, it may reduce patients’ dependency on treatments for anemia, splenomegaly, and constitutional symptoms, thereby fulfilling unmet needs. In effect, it will potentially limit the growth of other drug classes in the myelofibrosis landscape.”

- Comments provided by James Beggs, Ph.D., the Analyst covering Oncology and Hematology.
- Information based on the report: Opportunity Analyzer: Myelofibrosis - Opportunity Analysis and Forecasts to 2025.
- This report was built using data and information sourced from proprietary databases, primary and secondary research, and in-house analysis conducted by a team of industry experts.
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  • Incyte
  • Novartis
  • Bristol-Myers Squibb
  • Gilead
  • Johnson & Johnson
  • Geron
  • Promedior
  • CTI BioPharma
  • Shire
  • Amgen
  • Celgene
  • Merck & Co.
  • Roche
  • Acerta Pharma
  • Pfizer
  • MEI Pharma
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