Table of Contents
1 Table of Contents 2
1.1 List of Tables 4
1.2 List of Figures 4
2 Executive Summary 6
2.1 Small Market with Limited Treatment Options 6
2.2 Multiple Reasons for Investment 6
2.3 Significant Level of First-in-Class Innovation 6
2.4 Deal Activity Represents Investment Opportunity 6
3 The Case for Innovation in the Hematological Disorders Market 7
3.1 Growing Number of Opportunities for Biologic Products 8
3.2 Diversification of Molecular Targets 8
3.3 Innovative First-in-Class Product Developments Remain Attractive 8
3.4 Regulatory and Reimbursement Policy Shifts Favor First-in-Class Product Innovation 9
3.5 Sustained Innovation 9
3.6 GBI Research Report Guidance 9
4 Clinical and Commercial Landscape 11
4.1 Disease Overview 11
4.2 Signs and Symptoms 12
4.2.1 Sickle Cell Disease 12
4.2.2 Anemia 13
4.2.3 Paroxysmal Nocturnal Hemoglobinuria 13
4.2.4 Other Disorders 13
4.3 Diagnosis 14
4.3.1 Sickle Cell Disease 14
4.3.2 Anemia 15
4.3.3 Paroxysmal Nocturnal Hemoglobinuria 15
4.3.4 Other Disorders 15
4.4 Etiology and Pathophysiology 16
4.4.1 Overview of Normal Iron Circulation 16
4.4.2 Disease State 17
4.5 Epidemiology 21
4.5.1 Sickle Cell Disease 21
4.5.2 Anemia 21
4.5.3 Paroxysmal Nocturnal Hemoglobinuria 21
4.5.4 Other Disorders 22
4.6 Treatment 23
4.6.1 Sickle Cell Disease 23
4.6.2 Anemia 23
4.6.3 Paroxysmal Nocturnal Hemoglobinuria 24
4.6.4 Other Disorders 24
4.7 Overview of Marketed Products 24
4.8 Current Unmet Need in the Hematological disorders Market 26
5 Pipeline Landscape Assessment 27
5.1 Hematological Disorders Pipeline Overview 27
5.2 Pipeline Development Landscape 27
5.2.1 Pipeline by Indication 27
5.2.2 Pipeline by Stage of Development and Molecule Type 28
5.2.3 Molecular Targets in the Pipeline 30
5.3 Comparative Distribution of Programs between the Hematological Disorders Pipeline and Market by Molecular Target 33
5.4 First-in-Class Programs Acting Upon Novel Molecular Targets 33
5.5 Ratio of First-in-Class Programs to First-in-Class Targets 37
5.6 List of all First-in-Class Pipeline Programs 39
6 Hematological Disorders Signaling Network and Innovation Alignment 43
6.1 Complexity of Signaling Networks in Hematological Disorders 43
6.2 Signaling Pathways and First-in-Class Molecular Target Integration 44
6.3 First-in-Class Matrix Assessment 44
7 First-in-Class Target Evaluation 47
7.1 Sickle Cell Disease 47
7.1.1 Pipeline Programs Targeting XXX 47
7.1.2 Pipeline Programs Targeting XXX 48
7.1.3 Pipeline Programs Targeting XXX 50
7.1.4 Pipeline Programs Targeting XXX 51
7.2 Anemias 53
7.2.1 Pipeline Programs Targeting XXX 53
7.2.2 Pipeline Programs Targeting XXX 54
7.2.3 Pipeline Programs Targeting XXX 56
7.3 Paroxysmal Nocturnal Hemoglobinuria 57
7.3.1 Pipeline Programs Targeting XXX 57
7.3.2 Pipeline Programs Targeting XXX 59
7.3.3 Pipeline Programs Targeting XXX 60
7.4 Bleeding Disorders 61
7.5 Immune Cell Disorders 61
7.6 Conclusion 61
8 Deals and Strategic Consolidations 63
8.1 Industry-Wide First-in-Class Deals 63
8.2 Hematological Disorders Deals Landscape 64
8.3 Licensing Deals 64
8.3.1 Deals by Region, Year and Value 65
8.3.2 Deals by Stage of Development and Value 66
8.3.3 Deal Number and Value by Molecule Type and Molecular Target 66
8.4 Co-development Deals 66
8.4.1 Deals by Region, Year and Value 66
8.4.2 Deals by Stage of Development and Value 67
8.4.3 Molecule Type and Value 68
8.4.4 Molecular Target and Value 68
8.5 List of First-in-Class Pipeline Programs with and without Prior Deal Involvement 69
9 Appendix 72
9.1 Abbreviations 72
9.2 References 72
9.3 Research Methodology 77
9.3.1 Data integrity: 77
9.3.2 Innovative and meaningful analytical techniques and frameworks: 77
9.3.3 Evidence based analysis and insight: 77
9.4 Secondary Research 77
9.4.1 Market Analysis 77
9.4.2 Pipeline Analysis 78
9.4.3 Overall Pipeline 78
9.4.4 First-in-Class Analysis 78
9.4.5 First-in-Class Matrix Assessment 78
9.4.6 First-in-Class Target Profiles 78
9.4.7 Licensing and Co-development Deals 78
9.5 Contact Us 79
9.6 Disclaimer 79