Ocular Cancer Drug Development Pipeline Review, 2018

This report provides an overview of the pipeline landscape for ocular cancer and comprehensive information on the therapeutics under development and key players involved in therapeutic development for retinoblastoma and uveal melanoma. It also features dormant and discontinued products.

Retinoblastoma is an eye cancer that begins in the retina. Symptoms include white color in the center circle of the eye (pupil), eye redness, vision problems, a different color in each iris and eye swelling.

The predisposing factors include age and heredity. There are seven products in active development for this indication and companies operating in this space include Ophthotech and VCN Biosciences.

Uveal melanoma, the most common intraocular tumor, is a disease in which melanocytes, found in the part of the eye called the uvea, become cancerous. Symptoms include dark spot on the iris, glaucoma, eye pain and eye redness.

Uveal melanoma is more common in people who have atypical mole syndrome, dysplastic nevus syndrome, and ocular or oculodermal melanocytosis. There are 35 products in active development for this indication and companies operating in this space include Novartis and Eli Lilly.

Molecular targets acted on by products in development for ocular cancer include tumor antigens, kinases and growth factor receptors.

Scope

– Which companies are the most active within each pipeline?

– Which pharmaceutical approaches are the most prominent at each stage of the pipeline and within each indication?

– To what extent do universities and institutions play a role within this pipeline, compared to pharmaceutical companies?

– What are the most important R&D milestones and data publications to have happened in this disease area?

Reasons to buy

- Understand the overall pipeline, with an at-a-glance overview of all products in therapeutic development for each indication

- Assess the products in development in granular detail, with an up-to-date overview of each individual pipeline program in each indication and a comprehensive picture of recent updates and milestones for each

- Analyze the companies, institutions and universities currently operating in the pipeline, and the products being fielded by each of these

- Understand the composition of the pipeline in terms of molecule type, molecular target, mechanism of action and route of administration

Companies mentioned

Aeglea BioTherapeutics Inc

APEIRON Biologics AG

Bellicum Pharmaceuticals Inc

Bristol-Myers Squibb Co

Celldex Therapeutics Inc

Cleveland BioLabs Inc

Delcath Systems Inc

Eli Lilly and Co

EyePoint Pharmaceuticals Inc

Iconic Therapeutics Inc

Immunocore Ltd

Innovation Pharmaceuticals Inc

Iovance Biotherapeutics Inc

Novartis AG

Ophthotech Corp

PEP-Therapy SAS

PepVax Inc

Pfizer Inc

Plexxikon Inc

Polaris Pharmaceuticals Inc

Syndax Pharmaceuticals Inc

Tesaro Inc

VCN Biosciences SL

Table of Contents

1 Table of Contents

1 Table of Contents 4

1.1 List of Tables 5

1.2 List of Figures 6

2 Introduction 7

2.1 Ocular Cancer Report Coverage 7

2.2 Retinoblastoma - Overview 7

2.3 Uveal Melanoma - Overview 7

3 Therapeutics Development 8

3.1 Retinoblastoma 8

3.2 Uveal Melanoma 10

4 Therapeutics Assessment 16

4.1 Retinoblastoma 16

4.2 Uveal Melanoma 22

5 Companies Involved in Therapeutics Development 31

5.1 Retinoblastoma 31

5.2 Uveal Melanoma 34

6 Dormant Projects 43

6.1 Retinoblastoma 43

6.2 Uveal Melanoma 43

7 Discontinued Products 44

7.1 Uveal Melanoma 44

8 Product Development Milestones 45

8.1 Retinoblastoma 45

8.2 Uveal Melanoma 49

9 Appendix 62

9.1 Methodology 62

9.2 Coverage 62

9.3 Secondary Research 62

9.4 Primary Research 62

9.5 Expert Panel Validation 62

9.6 Contact Us 62

9.7 Disclaimer 63

List of Tables

1.1 List of Tables

Table 1: Number of Products under Development for Retinoblastoma 8

Table 2: Number of Products under Development by Companies, Retinoblastoma 9

Table 3: Products under Development by Companies, Retinoblastoma 10

Table 4: Number of Products under Development for Uveal Melanoma 11

Table 5: Number of Products under Development by Companies, Uveal Melanoma 12

Table 6: Number of Products under Development by Universities/Institutes, Uveal Melanoma 13

Table 7: Products under Development by Companies, Uveal Melanoma 14

Table 8: Products under Development by Universities/Institutes, Uveal Melanoma 15

Table 9: Number of Products by Stage and Target, Retinoblastoma 17

Table 10: Number of Products by Stage and Mechanism of Action, Retinoblastoma 18

Table 11: Number of Products by Stage and Route of Administration, Retinoblastoma 20

Table 12: Number of Products by Stage and Molecule Type, Retinoblastoma 21

Table 13: Number of Products by Stage and Target, Uveal Melanoma 23

Table 14: Number of Products by Stage and Mechanism of Action, Uveal Melanoma 26

Table 15: Number of Products by Stage and Route of Administration, Uveal Melanoma 29

Table 16: Number of Products by Stage and Molecule Type, Uveal Melanoma 30

Table 17: Retinoblastoma – Pipeline by APEIRON Biologics AG, H1 2018 31

Table 18: Retinoblastoma – Pipeline by EyePoint Pharmaceuticals Inc, H1 2018 31

Table 19: Retinoblastoma – Pipeline by Innovation Pharmaceuticals Inc, H1 2018 32

Table 20: Retinoblastoma – Pipeline by Ophthotech Corp, H1 2018 32

Table 21: Retinoblastoma – Pipeline by PepVax Inc, H1 2018 33

Table 22: Retinoblastoma – Pipeline by VCN Biosciences SL, H1 2018 33

Table 23: Uveal Melanoma – Pipeline by Aeglea BioTherapeutics Inc, H1 2018 34

Table 24: Uveal Melanoma – Pipeline by Bellicum Pharmaceuticals Inc, H1 2018 34

Table 25: Uveal Melanoma – Pipeline by Bristol-Myers Squibb Co, H1 2018 35

Table 26: Uveal Melanoma – Pipeline by Celldex Therapeutics Inc, H1 2018 35

Table 27: Uveal Melanoma – Pipeline by Cleveland BioLabs Inc, H1 2018 36

Table 28: Uveal Melanoma – Pipeline by Delcath Systems Inc, H1 2018 36

Table 29: Uveal Melanoma – Pipeline by Eli Lilly and Co, H1 2018 37

Table 30: Uveal Melanoma – Pipeline by Iconic Therapeutics Inc, H1 2018 37

Table 31: Uveal Melanoma – Pipeline by Immunocore Ltd, H1 2018 38

Table 32: Uveal Melanoma – Pipeline by Iovance Biotherapeutics Inc, H1 2018 38

Table 33: Uveal Melanoma – Pipeline by Novartis AG, H1 2018 39

Table 34: Uveal Melanoma – Pipeline by PEP-Therapy SAS, H1 2018 39

Table 35: Uveal Melanoma – Pipeline by Pfizer Inc, H1 2018 40

Table 36: Uveal Melanoma – Pipeline by Plexxikon Inc, H1 2018 40

Table 37: Uveal Melanoma – Pipeline by Polaris Pharmaceuticals Inc, H1 2018 41

Table 38: Uveal Melanoma – Pipeline by Syndax Pharmaceuticals Inc, H1 2018 41

Table 39: Uveal Melanoma – Pipeline by Tesaro Inc, H1 2018 42

Table 40: Uveal Melanoma – Pipeline by Viralytics Ltd, H1 2018 42

Table 41: Retinoblastoma – Dormant Projects, H1 2018 43

Table 42: Uveal Melanoma – Dormant Projects, H1 2018 43

Table 43: Uveal Melanoma – Discontinued Products, H1 2018 44

List of Figures

1.2 List of Figures

Figure 1: Number of Products under Development for Retinoblastoma 8

Figure 2: Number of Products under Development by Companies, Retinoblastoma 9

Figure 3: Number of Products under Development for Uveal Melanoma 10

Figure 4: Number of Products under Development by Companies, Uveal Melanoma 11

Figure 5: Number of Products under Development by Universities/Institutes, Uveal Melanoma 13

Figure 6: Number of Products by Targets, Retinoblastoma 16

Figure 7: Number of Products by Stage and Targets, Retinoblastoma 16

Figure 8: Number of Products by Mechanism of Actions, Retinoblastoma 17

Figure 9: Number of Products by Stage and Mechanism of Actions, Retinoblastoma 18

Figure 10: Number of Products by Routes of Administration, Retinoblastoma 19

Figure 11: Number of Products by Stage and Routes of Administration, Retinoblastoma 19

Figure 12: Number of Products by Molecule Types, Retinoblastoma 20

Figure 13: Number of Products by Stage and Molecule Types, Retinoblastoma 21

Figure 14: Number of Products by Top 10 Targets, Uveal Melanoma 22

Figure 15: Number of Products by Stage and Top 10 Targets, Uveal Melanoma 22

Figure 16: Number of Products by Top 10 Mechanism of Actions, Uveal Melanoma 24

Figure 17: Number of Products by Stage and Top 10 Mechanism of Actions, Uveal Melanoma 25

Figure 18: Number of Products by Routes of Administration, Uveal Melanoma 28

Figure 19: Number of Products by Stage and Routes of Administration, Uveal Melanoma 28

Figure 20: Number of Products by Top 10 Molecule Types, Uveal Melanoma 29

Figure 21: Number of Products by Stage and Top 10 Molecule Types, Uveal Melanoma 30

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