Myelodysplastic Syndromes – Opportunity Analysis and Forecasts to 2028

1 Table of Contents

1.1 List of Tables

1.2 List of Figures

2 Myelodysplastic Syndromes: Executive Summary

2.1 Moderate Growth Expected in MDS Market Between 2018 and 2028

2.2 Drug Developers Position Agents to Fill Key Vacancies in the Treatment Paradigms for Higher- and Lower-Risk MDS

2.3 Unmet Needs Persist in Difficult-to-Treat MDS Subgroups, Leaving Substantial Opportunity for Future Players

2.4 Current Late-Stage MDS Pipeline Offers a Range of New Options for Patients in the Front-Line and Later Treatment Settings

2.5 What Do Physicians Think?

3 Introduction

3.1 Catalyst

3.2 Related Reports

3.3 Upcoming Related Reports

4 Disease Overview

4.1 Etiology and Pathophysiology

4.1.1 Etiology

4.1.2 Pathophysiology

4.2 Clinical Presentation and Diagnosis

4.3 Classification

4.3.1 WHO Classification Criteria for MDS

4.3.2 IPSS-R Classification

5 Epidemiology

5.1 Disease Background

5.2 Risk Factors and Comorbidities

5.3 Global and Historical Trends

5.4 Forecast Methodology

5.4.1 Sources

5.4.2 Forecast Assumptions and Methods

5.5 Epidemiological Forecast for MDS (2018–2028)

5.5.1 Diagnosed Incident Cases of MDS

5.5.2 Age-Specific Diagnosed Incident Cases of MDS

5.5.3 Sex Specific Diagnosed Incident Cases of MDS

5.5.4 Diagnosed Incident Cases of MDS/MPN

5.5.5 Diagnosed Incident Cases of MDS by Subtype

5.5.6 Diagnosed Incident Cases of Primary/Secondary MDS

5.5.7 Diagnosed Incident Cases of MDS by Risk Group

5.5.8 Diagnosed Incident Cases of MDS by Mutations

5.5.9 Five-Year Diagnosed Prevalent Cases of MDS

5.6 Discussion

5.6.1 Epidemiological Forecast Insight

5.6.2 Limitations of the Analysis

5.6.3 Strengths of the Analysis

6 Current Treatment Options

6.1 Overview

6.1.1 HR-MDS

6.1.2 LR-MDS

6.2 Treatment Algorithm

6.2.1 HR-MDS

6.2.2 LR-MDS

7 Unmet Needs and Opportunity Assessment

7.1 Overview

7.2 Novel Strategies to Address Standard of Care Failure

7.2.1 HMA Failure

7.2.2 ESA Failure

7.3 Therapeutic Options for LR-MDS Patients with Thrombocytopenia and Neutropenia

7.4 Safer Curative Therapy Options

8 R&D Strategies

8.1 Overview

8.1.1 Second-Generation HMAs to Combat Genericization of Vidaza and Dacogen

8.1.2 Novel Mechanisms to Promote Erythropoiesis in LR-MDS with Anemia

8.1.3 Front-Line Combination Therapy Regimens that Synergize with Azacitidine

8.1.4 Targeted Therapy Based on Mutational Status

8.1.5 Label Expansions of AML Therapeutics

8.2 Clinical Trial Design

8.2.1 HR-MDS

8.2.2 LR-MDS

8.2.3 Considerations for Future Clinical Trial Design

9 Pipeline Assessment

9.1 Overview

9.1.1 HR-MDS

9.1.2 LR-MDS

9.2 Innovative Early Stage Approaches

10 Pipeline Valuation Analysis

10.1 Clinical Benchmark of Key Pipeline Drugs

10.1.1 HR-MDS

10.1.2 LR-MDS

10.2 Commercial Benchmark of Key Pipeline Drugs

10.2.1 HR-MDS

10.2.2 LR-MDS

10.3 Competitive Assessment

10.3.1 HR-MDS

10.3.2 LR-MDS

10.4 Top-Line 10-Year Forecast

10.4.1 US

10.4.2 5EU

10.4.3 Japan

11 Appendix

11.1 Bibliography

11.2 Abbreviations

11.3 Methodology

11.3.1 Forecasting Methodology

11.3.2 Diagnosed Patients

11.3.3 Percent Drug-Treated Patients

11.3.4 Drugs Included in Each Therapeutic Class

11.3.5 Launch and Patent Expiry Dates

11.3.6 General Pricing Assumptions

11.3.7 Individual Drug Assumptions

11.3.8 Generic and Biosimilar Erosion

11.3.9 Pricing of Pipeline Agents

11.3.10 Other General Assumptions

11.4 Primary Research – Key Opinion Leaders Interviewed for This Report

11.4.1 Key Opinion Leaders

11.4.2 Payers

11.5 Primary Research – Prescriber Survey

11.6 About the Authors

11.6.1 Analyst

11.6.2 Therapy Area Director

11.6.3 Epidemiologists

11.6.4 Epidemiology Reviewers

11.6.5 Global Director of Therapy Analysis and Epidemiology

11.6.6 Global Head and EVP of Healthcare Operations and Strategy

11.7 About GlobalData

11.8 Contact Us

11.9 Disclaimer

Table 1: Myelodysplastic Syndromes: Key Metrics in the 7MM

Table 2: Common cytogenetic abnormalities in MDS

Table 3: Frequent mutations in MDS

Table 4: Tests used in the Diagnosis of MDS

Table 5: 2016 WHO Classification Criteria for MDS

Table 6: Cytopenia-defining values for MDS

Table 7: IPSS-R Prognostic Risk Categories/Scores and Clinical Outcomes

Table 8: IPSS-R Prognostic Score Values

Table 9: MDS Cytogenetic Scoring System

Table 10: Risk Factors and Comorbidities for MDS

Table 11: Leading Treatments for MDS and Country-Specific Launch Dates, 2020

Table 12: Treatment Guidelines for MDS

Table 13: IWG 2006 Proposed Modified Response Criteria for Altering the Natural History of MDS

Table 14: IWG 2006 Proposed Modified Response Criteria for Hematologic Improvement

Table 15: IWG 2018 Suggested Modified HI-E Criteria for Response Evaluation

Table 16: IWG 2018 Suggested Modified HI-P and HI-N Criteria for Response Evaluation

Table 17: Comparison of Therapies in Development for HR-MDS and LR-MDS, 2018–2028

Table 18: Innovative Early Stage Approaches for MDS, 2020

Table 19: Clinical Benchmark of Key Pipeline Drugs – HR-MDS

Table 20: Clinical Benchmark of Key Pipeline Drugs – HR-MDS

Table 21: Clinical Benchmark of Key Pipeline Drugs – HR-MDS – Allogeneic HSCT

Table 22: Clinical Benchmark of Key Pipeline Drugs – LR-MDS

Table 23: Clinical Benchmark of Key Pipeline Drugs – LR-MDS

Table 24: Commercial Benchmark of Key Pipeline Drugs – HR-MDS

Table 25: Commercial Benchmark of Key Pipeline Drugs – HR-MDS

Table 26: Commercial Benchmark of Key Pipeline Drugs – HR-MDS –HSCT

Table 27: Commercial Benchmark of Key Pipeline Drugs – LR-MDS

Table 28: Commercial Benchmark of Key Pipeline Drugs – LR-MDS

Table 29: MDS Market – Global Drivers and Barriers, 2018–2028

Table 30: Key Events Impacting Sales for MDS in the US, 2018–2028

Table 31: Key Events Impacting Sales for MDS in the 5EU, 2018–2028

Table 32: Key Events Impacting Sales for MDS in Japan, 2018–2028

Table 33: Key Historical and Projected Launch Dates for MDS

Table 34: Key Historical and Projected Patent/Exclusivity Expiry Dates for MDS

Table 35: High-Prescribing Physicians (non-KOLs) Surveyed, By Country

Figure 1: Global Sales Forecast by Country for MDS in 2018 and 2028

Figure 2: Competitive Assessment of the Marketed and Pipeline Drugs Benchmarked Against the Standard of Care, HR-MDS

Figure 3: Competitive Assessment of Omidubicel Against the Standard of Care, HR-MDS

Figure 4: Competitive Assessment of the Marketed and Pipeline Drugs Benchmarked Against the Standard of Care, LR-MDS

Figure 5: Key Therapeutic Mechanisms in MDS

Figure 6: 8MM, Diagnosed Incidence Rates of MDS, Men and Women, Ages ≥18 Years, Cases per 100,000 Population, 2008‒2028

Figure 7: Sources Used for Diagnosed Incident Cases of MDS

Figure 8: Sources Used for Diagnosed Incident Cases of MDS/MPN

Figure 9: Sources Used for Diagnosed Incident Cases of MDS Subtypes

Figure 10: Sources Used for Diagnosed Incident Cases of Primary/Secondary MDS

Figure 11: Sources Used for Five-Year Diagnosed Prevalent Cases of MDS

Figure 12: 8MM, Diagnosed Incident Cases of MDS, Men and Women, Ages ≥18 Years, N, 2018

Figure 13: 8MM, Age-Specific Diagnosed Incident Cases of MDS, Men and Women, Ages ≥18 Years, N, 2018

Figure 14: 8MM, Sex-Specific Diagnosed Incident Cases of MDS, Men and Women, Ages ≥18 Years, N, 2018

Figure 15: 8MM, Diagnosed Incident Cases MDS/MPN, Men and Women, Ages ≥18 Years, N, 2018

Figure 16: 8MM, Proportion of Diagnosed Incident Cases of MDS by Subtype, Men and Women, Ages ≥18 Years, N, 2018

Figure 17: 8MM, Proportion of Diagnosed Incident Cases of Primary/Secondary MDS, Men and Women, Ages ≥18 Years, 2018

Figure 18: 8MM, Diagnosed Incident Cases of MDS by Risk Group, Men and Women, Ages ≥18 Years, N, 2018

Figure 19: 8MM, Diagnosed Incident Cases of MDS by Mutation, Men and Women, Ages ≥18 Years, N, 2018

Figure 20: 8MM, Five-Year Diagnosed Prevalent Cases of MDS, Men and Women, Ages ≥18 Years, N, 2018

Figure 21: NCCN Treatment Flow for HR-MDS

Figure 22: NCCN Treatment Flow for LR-MDS

Figure 23: Unmet Needs and Opportunities in MDS

Figure 24: Overview of the Development Pipeline in MDS

Figure 25: Key Phase II/III Trials for Promising Pipeline Agents that GlobalData Expects be Licensed for MDS in the 7MM During the Forecast Period

Figure 26:Competitive Assessment of Marketed and Pipeline Drugs Benchmarked Against the SOC, HR-MDS

Figure 27:Competitive Assessment of Omidubicel Against the SOC, HR-MDS

Figure 28:Competitive Assessment of Marketed and Pipeline Drugs Benchmarked Against the SOC, LR-MDS

Figure 29: Global Sales Forecast by Country for MDS in 2018 and 2028

Figure 30: Global MDS Sales Forecast by Class, 2018 and 2028

Figure 31: US MDS Sales Forecast by Class, 2018 and 2028

Figure 32: 5EU MDS Sales Forecast by Class, 2018 and 2028

Figure 33: Japan MDS Sales Forecast by Class, 2018 and 2028