Frontier Pharma: Ophthalmology Therapeutics – Gene Therapies and First-in-Class Targets Offer Novel Treatments for Substantial Unmet Needs in a Pipeline Dominated by Angiogenic and Immune Signaling Drugs

The ophthalmologic therapy area encompasses a wide range of disorders affecting the eye and its supporting structures. These include glaucoma, age-related macular degeneration (AMD), diabetic retinopathy (DR), diabetic macular edema (DME) and dry eye syndrome (DES). The therapy area is characterized by vast unmet need, as patients have few treatment options and poor outcomes.

This report assesses first-in-class innovation in the ophthalmology pipeline, highlighting key trends in the distribution of these products. In-depth analysis reveals that the level of innovation within the therapy area pipeline is high, with strong potential for drugs to address unmet needs and be commercially lucrative. First-in-class drugs account for 38% of products with a disclosed molecular target, acting on 151 unique molecular targets, which is indicative of the approach being taken in the field.

In the past the ophthalmological disorders therapy area has suffered from a lack of full understanding of the pathophysiology and underlying mechanisms of the diseases. However, as this knowledge has improved vastly in recent years, potential targets have been identified for treatment. As well as the main disorders previously mentioned, there is also interest in developing treatments for indications such as allergic conjunctivitis and retinitis pigmentosa. The approval of a gene therapy for retinitis pigmentosa is indicative of this progress, and represents the first gene therapy approved for an inherited condition by the FDA.

The pipeline is varied in terms of molecular targets and molecule types. Products acting on the immune system and neurotransmitter signaling are the most prevalent, but no single target dominates by any clear margin. There are a total of 194 first-in class pipeline products in development for ophthalmologic indications – 38% of the 512 pipeline products for which there is a disclosed molecular target. Encompassing gene therapies, slowing of the visual cycle and antisense therapies, the pipeline appears robust and has huge potential to strengthen treatment options for ophthalmological disorders.

Scope

– The pipeline is large, with 865 products in active development. Does current pipeline innovation hold the potential to affect the future ophthalmology market?

– There are 194 first-in-class products in the ophthalmologic disorder pipeline. Which of these possess the greatest potential to improve disease outcome and be commercially successful, based on their target?

– Analysis of strategic consolidations and deals revealed a high level of activity in recent years for licensing agreements, but a low level of co-development. How do deal frequency and value compare between molecular targets and molecule types, and which first-in-class targets have not yet been involved in deals?

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 ophthalmological disorders market in terms of dominant molecule types and targets, and understand the current unmet needs and how they can be addressed. This knowledge allows a competitive understanding of gaps in the market.

- Analyze the ophthalmological 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 ophthalmological 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 ophthalmological disorders deals landscape by analyzing trends in licensing and co-development deals, and a curated list of ophthalmological 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 3

1.2 List of Figures 3

2 Executive Summary 5

2.1 Market Landscape Poised for Entry of Novel Products 5

2.2 Moderately Sized but Strong First-in-Class Pipeline 5

2.3 Strong Uptake of First-in-Class Products in Strategic Consolidations 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 Development Remains 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 Therapy Area Overview 10

4.1.1 Glaucoma 10

4.1.2 Age-related Macular Degeneration 10

4.1.3 Diabetic Macular Edema 10

4.1.4 Diabetic Retinopathy 10

4.1.5 Dry Eye Syndrome 11

4.1.6 Inherited Genetic Disorders 11

4.2 Disease Symptoms 11

4.3 Epidemiology and Etiology 12

4.3.1 Glaucoma 13

4.3.2 Age-Related Macular Degeneration 13

4.3.3 Diabetic Retinopathy and Diabetic Macular Edema 13

4.3.4 Dry Eye Syndrome 13

4.4 Diagnosis 13

4.5 Pathophysiology 14

4.5.1 Healthy Eye Physiology 14

4.5.2 Glaucoma 15

4.5.3 Age-Related Macular Degeneration 16

4.5.4 Diabetic Macular Edema and Diabetic Retinopathy 17

4.5.5 Dry Eye Syndrome 17

4.5.6 Inherited Genetic Diseases 18

4.6 Prognosis 18

4.6.1 Glaucoma 18

4.6.2 Age-Related Macular Degeneration 19

4.6.3 Diabetic Retinopathy and Diabetic Macular Edema 19

4.6.4 Dry Eye Syndrome 19

4.7 Treatment Options 20

4.8 Overview of Marketed Products in Ophthalmology 22

4.8.1 Molecule Type and Target Analysis 22

5 Assessment of Pipeline Product Innovation 23

5.1 Ophthalmology Pipeline by Molecular Target 26

5.2 Comparative Distribution of Programs between the Ophthalmology Market and Pipeline by Therapeutic Target Family 28

5.3 First-in-Class Pipeline Programs Targeting Novel Molecular Targets 29

6 Signaling Network, Disease Causation and Innovation Alignment 40

6.1 Complexity of Signaling Networks in Ophthalmology 40

6.2 Signaling Pathways and First-in-Class Molecular Target Integration 40

6.3 First-in-Class Matrix Assessment 41

7 First-in-Class Target Evaluation 45

7.1 Pipeline Programs Targeting MER 45

7.2 Pipeline Programs Targeting Rhodopsin 46

7.3 Pipeline Programs Targeting NLRP3 47

7.4 Pipeline Programs Targeting ABCA4 48

7.5 Pipeline Programs Targeting Complement Factor B 48

7.6 Pipeline Programs Targeting RPE65 AND RBP4 50

7.7 Conclusion 52

8 Deals and Strategic Consolidations 53

8.1 Industry-Wide First-in-Class Deals 53

8.2 Licensing Deals 55

8.2.1 Deals by Region, Year and Value 55

8.2.2 Deals by Stage of Development and Value 56

8.2.3 Deals by Molecule Type, Mechanism of Action and Value 56

8.2.4 Table for Licensing Deals Valued Above $100m 58

8.3 Co-development Deals 58

8.3.1 Deals by Region, Year and Value 58

8.3.2 Deals by Stage of Development and Value 60

8.3.3 Deals by Molecule Type, Mechanism of Action and Value 60

8.3.4 Table for Co-development Deals Valued Above $100m 61

8.4 First-in-Class Programs With and Without Prior Deal Involvement 61

9 Appendix 65

9.1 Abbreviations 65

9.2 References 65

9.3 Research Methodology 69

9.4 Primary Research 70

9.5 Secondary Research 70

9.5.1 Market Analysis 70

9.5.2 Pipeline Analysis 70

9.5.3 First-in-Class Matrix Assessment 71

9.5.4 First-in-Class Target Profiles 71

9.5.5 Licensing and Co-development Deals 71

9.6 Contact Us 71

9.7 Disclaimer 71

List of Tables

1.1 List of Tables

Table 1: Ophthalmology Therapeutics, Global, Main Symptoms of Glaucoma, AMD, DR and DES, 2017 12

Table 2: Ophthalmology Therapeutics, Global, Prevalence of Major Ophthalmologic Disorders, 2017 13

Table 3: Ophthalmology Therapeutics, Global, Treatment Options Available for Glaucoma, 2017 21

Table 4: Ophthalmology Therapeutics, Global, Data for MERTK as a Molecular Target, 2017 46

Table 5: Ophthalmology Therapeutics, Global, Data for RHO as a Molecular Target, 2017 47

Table 6: Ophthalmology Therapeutics, Global, Data for NLRP3 as a Molecular Target, 2017 47

Table 7: Ophthalmology Therapeutics, Global, Data for ABCA4 as a Molecular Target, 2017 48

Table 8: Ophthalmology Therapeutics, Global, Data for Cfb as a Molecular Target, 2017 50

Table 9: Ophthalmology Therapeutics, Global, Data for Serine Protease RPE65 as a Molecular Target, 2017 51

Table 10: Ophthalmology Therapeutics, Global, Data for Serine Protease RBP4 as a Molecular Target, 2017 52

List of Figures

1.2 List of Figures

Figure 1: Ophthalmology Therapeutics, Global, Innovation Trends in Product Approvals, Number of Product Approvals by FDA and Five-Year Moving Average of Products Approvals (%), 1987-2012 6

Figure 2: Ophthalmology Therapeutics, Global, First-in-Class and Non-First-in-Class Products, Sales Performance After Marketing Approval ($m) 8

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

Figure 4: Ophthalmology Therapeutics, Global, Overview of Pipeline Products, 2017 23

Figure 5: Ophthalmology Therapeutics, Global, Pipeline by Stage of Development and Molecule Type, 2017 24

Figure 6: Ophthalmology Therapeutics, Global, Pipeline by Key Indications and Stage of Development, 2017 25

Figure 7: Ophthalmology Therapeutics, Global, Pipeline by Key Indications and Molecule Type, 2017 26

Figure 8: Ophthalmology Therapeutics, Global, Pipeline by Molecular Target, 2017 27

Figure 9: Ophthalmology Therapeutics, Global, Pipeline by Key Indications and Molecular Target, 2017 28

Figure 10: Ophthalmology Therapeutics, Global, Molecular Target Family Comparison, Pipeline and Marketed Products, 2017 28

Figure 11: Ophthalmology Therapeutics, Global, Molecular Target Family Comparison, Pipeline First-in-Class and Established Molecular Targets, 2017 29

Figure 12: Ophthalmology Therapeutics, Global, Percentage of First-in-Class Products in Pipeline by Stage of Development (%), 2017 30

Figure 13: Ophthalmology Therapeutics, Global, Percentage of First-in-Class Products in Pipeline by Molecular Target Family (%), 2017 30

Figure 14: Ophthalmology Therapeutics, Global, Ratio of First-in-Class Products to First-in-Class Targets by Stage of Development, 2017 30

Figure 15: Ophthalmology Therapeutics, Global, Percentage of First-in-Class Products in Pipeline by Stage of Development (%), 2017 31

Figure 16: Ophthalmology Therapeutics, Global, First-in-Class Products in Pipeline (Part 1), 2017 31

Figure 17: Ophthalmology Therapeutics, Global, First-in-Class Products in Pipeline (Part 2), 2017 32

Figure 18: Ophthalmology Therapeutics, Global, First-in-Class Products in Pipeline (Part 3), 2017 32

Figure 19: Ophthalmology Therapeutics, Global, First-in-Class Products in Pipeline (Part 4), 2017 33

Figure 20: Ophthalmology Therapeutics, Global, First-in-Class Products in Pipeline (Part 5), 2017 33

Figure 21: Ophthalmology Therapeutics, Global, First-in-Class Products in Pipeline (Part 6), 2017 34

Figure 22: Ophthalmology Therapeutics, Global, First-in-Class Products in Pipeline (Part 7), 2017 35

Figure 23: Ophthalmology Therapeutics, Global, First-in-Class Products in Pipeline (Part 8), 2017 35

Figure 24: Ophthalmology Therapeutics, Global, First-in-Class Products in Pipeline (Part 9), 2017 36

Figure 25: Ophthalmology Therapeutics, Global, First-in-Class Products in Pipeline (Part 10), 2017 36

Figure 26: Ophthalmology Therapeutics, Global, First-in-Class Products in Pipeline (Part 11), 2017 37

Figure 27: Ophthalmology Therapeutics, Global, First-in-Class Products in Pipeline (Part 12), 2017 38

Figure 28: Ophthalmology Therapeutics, Global, First-in-Class Products in Pipeline (Part 13), 2017 39

Figure 29: Ophthalmology Therapeutics, Global, Target Matrix Assessment (Part 1), 2017 42

Figure 30: Ophthalmology Therapeutics, Global, Target Matrix Assessment (Part 2), 2017 43

Figure 31: Ophthalmology Therapeutics, Global, Target Matrix Assessment (Part 3), 2017 44

Figure 32: Ophthalmology Therapeutics, Global, Pipeline Programs Targeting MERTK, 2017 46

Figure 33: Ophthalmology Therapeutics, Global, Pipeline Programs Targeting RHO, 2017 47

Figure 34: Ophthalmology Therapeutics, Global, Pipeline Programs Targeting NLRP3, 2017 48

Figure 35: Ophthalmology Therapeutics, Global, Pipeline Programs Targeting ABCA4, 2017 48

Figure 36: Ophthalmology Therapeutics, Global, Pipeline Programs Targeting Cfb, 2017 50

Figure 37: Ophthalmology Therapeutics, Global, Pipeline Programs Targeting Serine Protease RPE65, 2017 52

Figure 38: Ophthalmology Therapeutics, Global, Pipeline Programs Targeting Serine Protease RBP4, 2017 52

Figure 39: Pharmaceutical Industry, Global, Licensing Deals by Stage of Development, 2006-2015 53

Figure 40: Pharmaceutical Industry, Global, Licensing Deal Values by Stage of Development ($m), 2006-2014 54

Figure 41: Ophthalmology Therapeutics, Global, Licensing Deals, 2006-2017 55

Figure 42: Ophthalmology Therapeutics, Global, Licensing Deals by Indication and Value, 2006-2017 56

Figure 43: Ophthalmology Therapeutics, Global, Licensing Deals by Stage of Development, Deal Value and Upfront Payment Value, 2006-2017 56

Figure 44: Ophthalmology Therapeutics, Global, Licensing Deals by Molecule Type and Mechanism of Action, 2006-2017 57

Figure 45: Ophthalmology Therapeutics, Global, Licensing Deals Valued Above $100m 58

Figure 46: Ophthalmology Therapeutics, Global, Co-development Deals, 2006-2017 59

Figure 47: Ophthalmology Therapeutics, Global, Co-development Deals by Indication and Value, 2006-2017 59

Figure 48: Ophthalmology Therapeutics, Global, Co-development Deals by Stage of Development, Deal Value and Upfront Payment Value, 2006-2017 60

Figure 49: Ophthalmology Therapeutics, Global, Co-development Deals by Molecule Type and Mechanism of Action, 2006-2017 61

Figure 50: Ophthalmology Therapeutics, Global, Licensing Deals Valued Above $100m 61

Figure 51: Ophthalmology Therapeutics, Global, First-in-Class Therapies Involved in Previous Deals, 2017 62

Figure 52: Ophthalmology Therapeutics, Global, First-in-Class Therapies Not Involved in Previous Deals (Part 1), 2017 63

Figure 53: Ophthalmology Therapeutics, Global, First-in-Class Therapies Not Involved in Previous Deals (Part 2), 2017 64

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