Frontier Pharma: Hematological Disorders – Iron Regulators and Immune Response Targeted Programs Within Anemias and other Red Blood Cell Disorders Hold Potential to Transform Therapy Area with Significant Unmet Need

The hematological disorders therapy area encompasses non-malignant disorders of the blood such as anemia, sickle cell disease, neutropenia and hemophilia. These diseases can be grouped into the three categories- red blood cell disorders, bleeding disorders and immune cell disorders – of which red blood cell disorders are characterized by significant unmet need related to few effective treatment options and poor prognosis. As such, they are the primary focus of the report.

Historically, the hematological disorders therapy area has suffered from a lack of funding for research, which has contributed to the level of unmet need, although there have been instances in which funding for an indication has resulted in a strong return on investment. A notable example is the approval of Soliris in 2007, which is used to treat the orphan disease paroxysmal nocturnal hemoglobinuria. Soliris is one of only two marketed treatment options for PNH and is forecast to generate annual revenue of over $3 billion by the end of 2017, rising to $5.5 billion in 2023.

The report assesses first-in-class innovation in the hematological disorders pipeline, highlighting key trends in the pipeline, and emerging treatment classes. There are 92 first-in-class products in development in this therapy area; which account for 28% of products in the overall pipeline for which there is a disclosed molecular target, and act upon 71 unique individual molecular targets.

These first-in-class targets in the pipeline are numerous and varied, with many of them having been shown to be promising in early stage studies. Immune components are the most common, accounting for a total of 21 first-in-class developmental programs, closely followed by DNA regulators. These target categories combined account for the majority of red blood cell disorder pipeline therapies, although iron regulator programs alone also appear frequently.

Scope

– With 488 products in active development, the pipeline for hematological disorders is modestly sized. Does current pipeline innovation hold the potential to affect the future hematological disorders market?

– There are 92 first-in-class products in the hematological disorders pipeline, which act on a novel molecular target which is not present in an approved product across any indication the pharmaceutical industry. Which of these hold the greatest potential to improve future disease treatment with regard to their molecular target?

– Analysis of the history of strategic consolidations revealed a modest level of deal activity in recent years and a large number of first-in-class products not yet involved in any deals. How do deal frequency and value compare between target families and molecule types, and which first-in-class programs which have not yet been involved in a licensing or co-development deal appear to be particularly promising?

Reasons to buy

- Understand the current clinical and commercial landscape. This includes a comprehensive study of disease pathogenesis, diagnosis and prognosis, and the treatment options available.

- Visualize the composition of the hematological disorders market in terms of dominant molecule types and targets, highlighting what the current unmet needs are and how they can be addressed. This knowledge allows a competitive understanding of gaps in the current market.

- Analyze the hematological disorders pipeline and stratify by stage of development, molecule type and molecular target. There are strong signs in the pipeline that the industry is seeking novel approaches to treating hematological disorders to overcome the overwhelming level of unmet need.

- Assess the therapeutic potential of first-in-class targets. Using a proprietary matrix, first-in-class products have been assessed and ranked according to clinical potential. Promising early-stage targets have been reviewed in greater detail.

- Identify commercial opportunities in the hematological disorders deals landscape by analyzing trends in licensing and co-development deals and analyzing a curated list of hematological disorder therapies that have not yet been involved in deals, and may offer potential investment opportunities.

Table of Contents

1 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 Hemopexin 47

7.1.2 Pipeline Programs Targeting RAC-Alpha Serine/threonine-Protein Kinase (AKT1) 48

7.1.3 Pipeline Programs Targeting P-Selectin 50

7.1.4 Pipeline Programs Targeting Ferroportin (Solute Carrier Family 40 Member 1) 51

7.2 Anemias 53

7.2.1 Pipeline Programs Targeting Transferrin Receptor Protein 1 53

7.2.2 Pipeline Programs Targeting Hepcidin 54

7.2.3 Pipeline Programs Targeting Natural Resistance Associated Macrophage Protein-2 56

7.3 Paroxysmal Nocturnal Hemoglobinuria 57

7.3.1 Pipeline Programs Targeting Complement factor-D (Cfd) 57

7.3.2 Pipeline Programs Targeting Mannan Binding Lectin Serine Protease-1 (Masp2) 59

7.3.3 Pipeline Programs Targeting Complement Component C6 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

List of Tables

1.1 List of Tables

Table 1: Hematological Disorders Therapeutics Market, Global, Summary of Signs and Symptoms of Key Hematological Disorders, 2017 14

Table 2: Hematological Disorders Therapeutics Market, Global and US, Estimates of Prevalent Cases of Key Disorders, 2017 22

Table 3: Hematological Disorders Therapeutics Market, Global, Data for Hemopexin as a Molecular Target, 2017 48

Table 4: Hematological Disorders Therapeutics Market, Global, Data for RAC-Alpha Serine/threonine-Protein Kinase as a Molecular Target, 2017 50

Table 5: Hematological Disorders Therapeutics Market, Global, Data for P-Selectin as a Molecular Target, 2017 51

Table 6: Hematological Disorders Therapeutics Market, Global, Data for Ferroportin (Solute Carrier Family 40 Member 1) as a Molecular Target, 2017 53

Table 7: Hematological Disorders Therapeutics Market, Global, Data for Transferrin Receptor Protein 1 as a Molecular Target, 2017 54

Table 8: Hematological Disorders Therapeutics Market, Global, Data for Hepcidin as a Molecular Target, 2017 56

Table 9: Hematological Disorders Therapeutics Market, Global, Data for Natural Resistance Associated Macrophage Protein-2 as a Molecular Target, 2017 57

Table 10: Hematological Disorders Therapeutics Market, Global, Data for Complement factor-D as a Molecular Target, 2017 59

Table 11: Hematological Disorders Therapeutics Market, Global, Data for Mannan Binding Lectin Serine Protease-2 as a Molecular Target, 2017 60

Table 12: Hematological Disorders Therapeutics Market, Global, Pipeline Programs Targeting Complement Component C6, 2017 61

List of Figures

1.1 List of Figures

Figure 1: Overall Pharmaceutical Industry, Innovation Trends in Product Approvals, Number of Product Approvals by FDA and Five-Year Moving Average of Products Approvals (%), 1987-2016 7

Figure 2: Hematological Disorders Therapeutics Market, First-in-Class and Non-First-in-Class Products, Sales Performance After Marketing Approval, 2006-2013 ($m) 9

Figure 3: Hematological Disorders Therapeutics Market, Global, Complement Pathway Overview, 2017 17

Figure 4: Hematological Disorders Therapeutics Market, Global, Marketed Product Overview, 2017 25

Figure 5: Overall Pharmaceutical Market, Global, Developmental Pipeline Overview, 2017 27

Figure 6: Hematological Disorders Therapeutics Market, Global, Pipeline by Indication, 2017 28

Figure 7: Hematological Disorders Therapeutics Market, Global, Pipeline by Stage of Development and Molecule Type, 2017 30

Figure 8: Hematological Disorders Therapeutics Market, Global, Pipeline by Molecular Target, 2017 32

Figure 9: Hematological Disorders Therapeutics Market, Global, Overall Pharmaceutical Industry First-in-Class Pipeline by Therapy Area, 2017 33

Figure 10: Hematological Disorders Therapeutics Market, Global, Overall Pharmaceutical Industry First-in-Class Pipeline by Therapy Area, 2017 34

Figure 11: Hematological Disorders Therapeutics Market, Global, First-in-Class Pipeline for Hematological disorders by Stage of Development and Molecule Type, 2017 35

Figure 12: Hematological Disorders Therapeutics Market, Global, First-in-Class Pipeline for Hematological disorders by Stage of Development and Molecule Type, 2017 36

Figure 13: Hematological Disorders Therapeutics Market, Global, Molecular Target Family Comparison, Pipeline First-in-Class and Established Molecular Targets, 2017 37

Figure 14: Hematological Disorders Therapeutics Market, Global, Molecular Target Family Comparison, Pipeline First-in-Class and Established Molecular Targets, 2017 38

Figure 15: Hematological Disorders Therapeutics Market, Global, Molecular Target Family Comparison, Pipeline First-in-Class and Established Molecular Targets, 2017 39

Figure 16: Hematological Disorders Therapeutics Market, Global, First-in-Class Developmental Programs in Sickle Cell Disease Pipeline, 2017 40

Figure 17: Hematological Disorders Therapeutics Market, Global, First-in-Class Developmental Programs in Anemias Pipeline, 2017 40

Figure 18: Hematological Disorders Therapeutics Market, Global, First-in-Class Developmental Programs in Paroxysmal Nocturnal Hemoglobinuria Pipeline, 2017 41

Figure 19: Hematological Disorders Therapeutics Market, Global, First-in-Class Developmental Programs in Bleeding Disorders Pipeline, 2017 41

Figure 20: Hematological Disorders Therapeutics Market, Global, First-in-Class Developmental Programs in Immune Cell Disorders Pipeline, 2017 42

Figure 21: Hematological Disorders Therapeutics Market, Global, First-in-Class Molecular Target Analysis Matrix for Sickle Cell Disease, 2017 45

Figure 22: Hematological Disorders Therapeutics Market, Global, First-in-Class Molecular Target Analysis Matrix for Anemias, 2017 45

Figure 23: Hematological Disorders Therapeutics Market, Global, First-in-Class Molecular Target Analysis Matrix for Paroxysmal Nocturnal Hemoglobinuria, 2017 45

Figure 24: Hematological Disorders Therapeutics Market, Global, First-in-Class Molecular Target Analysis Matrix for Bleeding Disorders, 2017 46

Figure 25: Hematological Disorders Therapeutics Market, Global, First-in-Class Molecular Target Analysis Matrix for Immune Cell Disorders, 2017 46

Figure 26: Hematological Disorders Therapeutics Market, Global, Pipeline Programs Targeting Hemopexin, 2017 48

Figure 27: Hematological Disorders Therapeutics Market, Global, Pipeline Programs Targeting RAC Alpha Serine/Threonine Protein Kinase, 2017 50

Figure 28: Hematological Disorders Therapeutics Market, Global, Pipeline Programs Targeting P-Selectin, 2017 51

Figure 29: Hematological Disorders Therapeutics Market, Global, Pipeline Programs Targeting Ferroportin (Solute Carrier Family 40 Member 1), 2017 53

Figure 30: Hematological Disorders Therapeutics Market, Global, Pipeline Programs Targeting Transferrin Receptor Protein 1, 2017 54

Figure 31: Hematological Disorders Therapeutics Market, Global, Pipeline Programs Targeting Hepcidin, 2017 56

Figure 32: Hematological Disorders Therapeutics Market, Global, Pipeline Programs Targeting Natural Resistance Associated Macrophage Protein-2, 2017 57

Figure 33: Hematological Disorders Therapeutics Market, Global, Pipeline Programs Targeting Complement factor-D, 2017 59

Figure 34: Hematological Disorders Therapeutics Market, Global, Pipeline Programs Targeting Mannan Binding Lectin Serine Protease-2, 2017 60

Figure 35: Hematological Disorders Therapeutics Market, Global, Pipeline Programs Targeting Complement Component C6, 2017 61

Figure 36: Hematological Disorders Therapeutics Market, Global, Pipeline Programs Targeting Tissue Factor, 2017 61

Figure 37: Hematological Disorders Therapeutics Market, Global, Pipeline Programs Targeting IL5RA, 2017 61

Figure 38: Pharmaceutical Market, Global, Industry-Wide Deals by Stage of Development, 2006-2014 63

Figure 39: Pharmaceutical Market, Global, Industry Licensing Deal Values by Stage of Development, 2006-2014 64

Figure 40: Hematological Disorders Therapeutics Market, Global, Licensing Deals by Region and Value, 2006-2017 65

Figure 41: Hematological Disorders Therapeutics Market, Global, Licensing Deals by Stage of Development, 2006-2017 66

Figure 42: Hematological Disorders Therapeutics Market, Global, Licensing Deals Valued Above $25m, 2006-2017 66

Figure 43: Hematological Disorders Therapeutics Market, Global, Co-Development Deals by Region and Value, 2006-2017 67

Figure 44: Hematological Disorders Therapeutics Market, Global, Co-Development Deals by Stage of Development, 2006-2017 68

Figure 45: Hematological Disorders Therapeutics Market, Global, Co-Development Deals by Molecule Type, 2006-2017 68

Figure 46: Hematological Disorders Therapeutics Market, Global, Co-Development Deals by Molecular Target, 2006-2017 69

Figure 47: Hematological Disorders Therapeutics Market, Global, Co-Development Deals Valued Above $25m, 2006-2017 69

Figure 48: Hematological Disorders Therapeutics Market, Global, First-in-class Programs in Active Development Involved in Previous Deals, 2017 70

Figure 49: Hematological Disorders Therapeutics Market, Global, First-in-class Programs in Active Development Without Recorded Prior Deal Involvement, 2017 71

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