Frontier Pharma: Transplantation Therapeutics – A Highly Innovative Pipeline with a Range of Adaptive and Innate Immune-Targeting Programs Focusing on Graft-Versus-Host Disease and Kidney Transplantation

In 2015 a total of 127,000 transplantations were performed worldwide, an increase of 5.8% from 2014, with 33,000 of these occurring in the EU and 32,000 in the US. Approximately 60% of these were kidney transplants, with liver, heart, lung, pancreas and small bowel transplantations accounting for the other most common procedures (Dominguez-Gil and Matesanz, 2017). When a graft is transplanted from a genetically non-identical individual, the recipient’s immune system recognizes the graft as foreign.

This leads to an anti-graft immune response that involves T cells invading the new tissue, multiplying, and recruiting more immune cells to the transplant site in order to remove this foreign body. Depending on the nature of the incompatibility and the immune response, and acute or chronic rejection process can occur. Conversely, if the graft consists of hematopoietic stem cells or immune cells, there is a risk that they will mount an immune response against the host, known as graft versus host disease (GVHD). There are several safe pharmacological treatment options for acute rejections, but long-term treatment options remain unsatisfactory.

The risk of infection limits the effectiveness of these therapies, and improvements to their efficacy are also needed. Specific transplantation tolerance, in which alloreactive T cells are inactivated while the broader immune response is left intact, removing the need for broad immunosuppressant therapies, can be considered to be the ultimate goal for clinical transplantation. Compared with the overall immunology pipeline, in which there are 1,915 products, the transplantation pipeline is small, with only 244 products. However, of the 145 products in the transplantation pipeline with a disclosed molecular target, there are 65 first-in-class products, acting on a total of 63 distinct first-in-class targets, highlighting the fact that this pipeline displays strong levels of innovation.

Scope

– There is a need for therapies that can achieve graft-specific immunosuppression, without having a general effect on the wider immune system. Which therapies and technologies currently in development are most likely to achieve this?

– There are 63 distinct first-in-class molecular targets in development for transplantation. Which of these hold the strongest potential in the clinic, and which are closest to reaching the market?

– How effective are current therapies for these indications, and how have they been able to improve the general prognosis in recent decades?

– Which molecule types and molecular targets are most prominent across transplantation therapy?

– Which specific types of transplantation are being most heavily studied across the pharmaceutical pipeline?

Reasons to buy

- Appreciate the current clinical and commercial landscapes by considering disease symptoms, pathogenesis, etiology, co-morbidities and complications, epidemiology, diagnosis, prognosis and treatment options for transplantation rejection.

- Identify leading products and key unmet needs within the market.

- Recognize trends in pipeline innovation by analyzing therapies by stage of development, molecule type and molecular target.

- Assess the therapeutic potential of first-in-class targets. Using a proprietary molecular target matrix assessment, first-in-class targets in the pipeline have been assessed and ranked according to clinical potential.

- Consider first-in-class pipeline products with no prior involvement in licensing and co-development deals, which may represent potential investment opportunities.

Table of Contents

1 Table of Contents

1 Table of Contents 2

1.1 List of Tables 3

1.2 List of Figures 3

2 Executive Summary 5

2.1 Strong Unmet need for Prevention of Chronic Rejection 5

2.2 Moderately-Sized by Highly Innovative Pipeline 5

2.3 Chemokines and Immune Surface Antigens Appear to Hold Promise as Novel Targets 5

3 The Case for Innovation 6

3.1 Growing Opportunities for Biologic Products 7

3.2 Diversification of Molecular Targets 7

3.3 Innovative First-in-Class Product Developments Remain Attractive 7

3.4 Regulatory and Reimbursement Policy Shifts Favor First-in-Class Product Innovation 8

3.5 Sustained Innovation 8

3.6 GBI Research Report Guidance 9

4 Clinical and Commercial Landscape 10

4.1 Disease Overview 10

4.2 Symptoms 11

4.2.1 Transplant Rejection 11

4.2.2 Graft-Versus-Host Disease 12

4.3 Diagnosis 12

4.4 Etiology and Pathophysiology 13

4.4.1 Major Histocompatibility Complex 13

4.4.2 Allogeneic Immune Response 13

4.4.3 Innate Immunity 15

4.4.4 The Role of Cytokines 15

4.4.5 Types of Rejection 15

4.5 Epidemiology and Prognosis 17

4.5.1 Transplant Rejection 17

4.5.2 Graft-Versus-Host Disease 18

4.6 Treatment Options 18

4.6.1 Antibody Therapies 19

4.6.2 Calcineurin Inhibitors 19

4.6.3 Anti-Proliferative Agents 19

4.6.4 mTOR Inhibitors 20

4.6.5 Corticosteroids 20

4.7 Overview of Marketed Products 21

4.8 Current Unmet Needs 23

5 Assessment of Pipeline Product Innovation 24

5.1 Pipeline by Stage of Development, Molecule Type and Molecular Target 24

5.2 First-in-Class Programs Targeting Novel Molecular Targets 28

6 Transplant Rejection Signaling Network, Disease Causation and Innovation Alignment 36

6.1 Complexity of Signaling Networks 36

6.2 Signaling Pathways and First-in-Class Molecular Target Integration 36

6.3 First-in-Class Matrix Assessment 36

7 First-in-Class Target and Pipeline Program Evaluation 39

7.1 Pipeline Programs Targeting C-X-C Chemokine Receptor Type 1 and C-X-C Chemokine Receptor Type 2 39

7.2 Pipeline Programs Targeting Tumor Necrosis Factor Receptor Superfamily Member 5 41

7.3 Pipeline Programs Targeting Stimulator of Interferon Genes Protein 43

7.4 Pipeline Programs Targeting Leukocyte Surface Antigen CD47 44

7.5 Pipeline Programs Targeting Tyrosine Protein Kinase SYK 46

7.6 Pipeline Programs Targeting Potassium Voltage Gated Channel Subfamily A Member 3 48

8 Strategic Consolidations 50

8.1 Industry-Wide First-in-Class Deals 50

8.2 Licensing Deals 52

8.2.1 Deals by Region, Year and Value 52

8.2.2 Deals by Stage of Development and Value 53

8.2.3 Deals by Molecule Type, Molecular Target and Value 54

8.2.4 List of Deals with Disclosed Deal Values 56

8.3 Co-development Deals 57

8.3.1 Deals by Region, Year and Value 57

8.3.2 Deals by Stage of Development and Value 59

8.3.3 Deals by Molecule Type, Molecular Target and Value 59

8.3.4 List of Deals with Disclosed Deal Values 61

8.4 List of First-in-Class Pipeline Products with and without Prior Deal Involvement 62

9 Appendix 64

9.1 Abbreviations 64

9.2 Disease List 64

9.3 References 65

9.4 Research Methodology 70

9.4.1 Data integrity 70

9.4.2 Innovative and meaningful analytical techniques and frameworks 70

9.4.3 Evidence based analysis and insight 70

9.5 Secondary Research 70

9.5.1 Market Analysis 70

9.5.2 Pipeline Analysis 70

9.5.3 Licensing and Co-development Deals 71

9.6 Contact Us 71

9.7 Disclaimer 72

List of Tables

1.1 List of Tables

Table 1: Transplantation Therapeutics, Global, Organ-Specific Symptoms of Transplant Rejection, 2017 11

Table 2: Transplantation Therapeutics, Global, Organ-Specific Symptoms of Acute GVHD, 2017 12

Table 3: Transplantation Therapeutics, Global, Organ-Specific Symptoms of Acute GVHD, 2017 12

Table 4: Transplantation Therapeutics, Global, Key Features of C-X-C Chemokine Receptor Type 1, 2017 40

Table 5: Transplantation Therapeutics, Global, Key Features of C-X-C Chemokine Receptor Type 2, 2017 40

Table 6: Transplantation Therapeutics, Global, Pipeline Programs Targeting C-X-C Chemokine Receptor Type 1 and C-X-C Chemokine Receptor Type 2, 2017 41

Table 7: Transplantation Therapeutics, Global, Key Features of Tumor Necrosis Factor Receptor Superfamily Member 5, 2017 42

Table 8: Transplantation Therapeutics, Global, Pipeline Programs Targeting Tumor Necrosis Factor Receptor Superfamily Member 5, 2017 43

Table 9: Transplantation Therapeutics, Global, Key Features of Stimulator of Interferon Genes Protein, 2017 44

Table 10: Transplantation Therapeutics, Global, Pipeline Programs Targeting Stimulator of Interferon Genes Protein, 2017 44

Table 11: Transplantation Therapeutics Global, Key Features of Leukocyte Surface Antigen CD47, 2017 45

Table 12: Transplantation Therapeutics, Global, Pipeline Programs Targeting Leukocyte Surface Antigen CD47, 2017 46

Table 13: Transplantation Therapeutics, Global, Key Features of Tyrosine Protein Kinase SYK, 2017 47

Table 14: Transplantation Therapeutics, Global, Pipeline Programs Targeting Tyrosine Protein Kinase SYK, 2017 47

Table 15: Transplantation Therapeutics, Global, Key Features of Potassium Voltage Gated Channel Subfamily A Member 3, 2017 49

Table 16: Transplantation Therapeutics, Global, Pipeline Programs Targeting Potassium Voltage Gated Channel Subfamily A Member 3, 2017 49

List of Figures

1.2 List of Figures

Figure 1: Transplantation Therapeutics, US, Innovation Trends in Product Approvals, 1987-2014 6

Figure 2: Transplantation Therapeutics, US, Sales Performance of First-in-Class and Non-First-in-Class Products Post Marketing Approval, 2006-2013 8

Figure 3: Transplantation Therapeutics, Global, Overview of Marketed Products, 2017 22

Figure 4: Transplantation Therapeutics, Global, Overall Pharmaceutical Industry Pipeline by Therapy Area, 2017 24

Figure 5: Transplantation Therapeutics, Global, Pipeline by Stage of Development and Molecule Type, 2017 25

Figure 6: Transplantation Therapeutics, Global, Key Indications by Stage of Development and Molecule Type, 2017 26

Figure 7: Transplantation Therapeutics, Global, Pipeline by Molecular Target, 2017 27

Figure 8: Transplantation Therapeutics, Global, Key Indications by Molecular Target, 2017 28

Figure 9: Transplantation Therapeutics, Global, Distribution of Pipeline First-in-Class and Non-First-in-Class Products by Stage of Development and Molecular Target, 2017 29

Figure 10: Transplantation Therapeutics, Global, Percentage Distribution of First-in-Class and Non-First-in-Class Pipeline Products with a Disclosed Target by Stage of Development (%), 2017 29

Figure 11: Transplantation Therapeutics, Global, Percentage Distribution of First-in-Class and Non-First-in-Class Pipeline Products by Molecular Target (%), 2017 30

Figure 12: Transplantation Therapeutics, Global, Ratio of First-in-Class Products to First-in-Class Targets by Stage of Development, 2017 30

Figure 13: Transplantation Therapeutics, Global, Ratio of First-in-Class Products to First-in-Class Targets by Molecular Target, 2017 31

Figure 14: Transplantation Therapeutics, Global, Pipeline Products, 2017 (Part 1) 31

Figure 15: Transplantation Therapeutics, Global, Pipeline Products, 2017 (Part 2) 32

Figure 16: Transplantation Therapeutics, Global, Pipeline Products, 2017 (Part 3) 33

Figure 17: Transplantation Therapeutics, Global, Pipeline Products, 2017 (Part 4) 34

Figure 18: Transplantation Therapeutics, Global, Pipeline Products, 2017 (Part 5) 35

Figure 19: Transplantation Therapeutics, Global, First-in-Class Matrix Assessment, 2017 38

Figure 20: Pharmaceutical Industry, Global, Licensing Deals by Stage of Development, 2006-2015 50

Figure 21: Pharmaceutical Industry, Global, Industry-Wide Licensing Deals by Deal Value, Upfront Payment Value, Stage of Development and First-in-Class Status ($m), 2006-2015 51

Figure 22: Transplantation Therapeutics, Global, Licensing Deals by Region, Value and Year, 2006-2017 53

Figure 23: Transplantation Therapeutics, Global, Licensing Deals by Stage of Development, Deal Value and Upfront Payment Value, 2006-2017 54

Figure 24: Transplantation Therapeutics, Global, Number and Aggregate Deal Value of Licensing Deals by Molecule Type and Molecular Target, 2006-2017 55

Figure 25: Transplantation Therapeutics, Global, Licensing Deals with Disclosed Deal Values, 2006-2017 56

Figure 26: Transplantation Therapeutics, Global, Co-development Deals by Region, Value and Year, 2006-2017 58

Figure 27: Transplantation Therapeutics, Global, Co-development Deals by Stage of Development, Deal Value and Upfront Payment Value, 2006-2017 59

Figure 28: Transplantation Therapeutics, Global, Number and Aggregate Deal Value of Co-development Deals by Molecule Type and Molecular Target, 2006-2017 60

Figure 29: Transplantation Therapeutics, Global, Co-development Deals with Disclosed Deal Values, 2006-2017 61

Figure 30: Transplantation Therapeutics, Global, First-in-class Programs in Active Development Without Recorded Prior Deal Involvement, 2017 (Part 1) 62

Figure 31: Transplantation Therapeutics, Global, First-in-class Programs in Active Development Without Recorded Prior Deal Involvement, 2017 (Part 2) 63

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