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Mucopolysaccharidosis Disorders Drug Development Pipeline Review, 2018

Published By :

GBI Research

Published Date : Jul 2018

Category :

Pharmaceutical

No. of Pages : 98 Pages

Mucopolysaccharidosis Disorders Drug Development Pipeline Review, 2018

Summary

This report provides an overview of the pipeline landscape for mucopolysaccharidosis disorders, a group of inherited lysosomal storage disorders. It provides comprehensive information on the therapeutics under development and key players involved in therapeutic development for mucopolysaccharidosis I (MPS I) (Hurler syndrome), mucopolysaccharidosis II (MPS II) (Hunter syndrome) and mucopolysaccharidosis III (MPS III) (Sanfilippo syndrome), and features dormant and discontinued products.

MPS I is caused by a deficiency of alpha-L-iduronidase, a lysosomal enzyme normally required for the breakdown of certain complex carbohydrates known as glycosaminoglycans (GAGs). Symptoms include abnormal bones in the spine, claw hand, cloudy corneas, deafness and heart valve problems. Treatment includes bone marrow transplantation, enzyme therapy and gene therapy. There are 18 products in development for this indication.

MPS II is a condition that affects many different parts of the body and occurs almost exclusively in males. Signs and symptoms include claw-like hands, protruding tongue, changing facial features, including thickening of the lips, tongue and nostrils and delayed development. Treatment includes bone marrow transplantation, enzyme therapy and gene therapy. There are 15 products in development for this indication.

MPS III is caused by an absence or malfunctioning of GAGs. Symptoms include seizures, hyperactivity, liver and spleen enlargement, severe diarrhea or constipation and enlargement of tonsils and adenoids. Treatment includes enzyme replacement therapy (ERT). There are 18 products in development for this indication.

Companies operating in the mucopolysaccharidosis disorders pipeline space include ArmaGen, Sangamo and AngioChem.

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

1 Table of Contents
1 Table of Contents 4
1.1 List of Tables 5
1.2 List of Figures 6
2 Introduction 8
2.1 Mucopolysaccharidosis Disorders Report Coverage 8
2.2 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Overview 8
2.3 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) - Overview 8
2.4 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Overview 8
3 Therapeutics Development 9
3.1 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 9
3.2 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 14
3.3 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 19
4 Therapeutics Assessment 23
4.1 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 23
4.2 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 28
4.3 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 34
5 Companies Involved in Therapeutics Development 40
5.1 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 40
5.2 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 44
5.3 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 49
6 Dormant Projects 54
6.1 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 54
6.2 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 55
6.3 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 56
7 Discontinued Products 57
7.1 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 57
7.2 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 57
7.3 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 57
8 Product Development Milestones 58
8.1 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 58
8.2 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 70
8.3 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 82
9 Appendix 97
9.1 Methodology 97
9.2 Coverage 97
9.3 Secondary Research 97
9.4 Primary Research 97
9.5 Expert Panel Validation 97
9.6 Contact Us 98
9.7 Disclaimer 98

1.1 List of Tables
Table 1: Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 9
Table 2: Number of Products under Development by Companies Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 10
Table 3: Number of Products under Development by Universities/Institutes Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 11
Table 4: Products under Development by Companies Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 12
Table 5: Products under Development by Universities/Institutes Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 13
Table 6: Number of Products under Development for Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 14
Table 7: Number of Products under Development by Companies Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 15
Table 8: Number of Products under Development by Universities/Institutes Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 16
Table 9: Products under Development by Companies Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 17
Table 10: Products under Development by Universities/Institutes Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 18
Table 11: Number of Products under Development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 19
Table 12: Number of Products under Development by Companies Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 20
Table 13: Products under Development by Companies Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 21
Table 14: Number of Products by Stage and Target Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 23
Table 15: Number of Products by Stage and Mechanism of Action Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 25
Table 16: Number of Products by Stage and Route of Administration Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 26
Table 17: Number of Products by Stage and Molecule Type Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 28
Table 18: Number of Products by Stage and Target Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 29
Table 19: Number of Products by Stage and Mechanism of Action Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 30
Table 20: Number of Products by Stage and Route of Administration Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 32
Table 21: Number of Products by Stage and Molecule Type Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 33
Table 22: Number of Products by Stage and Target Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 35
Table 23: Number of Products by Stage and Mechanism of Action Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 36
Table 24: Number of Products by Stage and Route of Administration Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 38
Table 25: Number of Products by Stage and Molecule Type Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 39
Table 26: Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by AngioChem Inc 40
Table 27: Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by ArmaGen Inc 40
Table 28: Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by CRISPR Therapeutics 41
Table 29: Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by Eloxx Pharmaceuticals Inc 41
Table 30: Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by Immusoft Corp 42
Table 31: Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by JCR Pharmaceuticals Co Ltd 42
Table 32: Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by OPKO Health Inc 43
Table 33: Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by RegenxBio Inc 43
Table 34: Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by Sangamo Therapeutics Inc 44
Table 35: Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) - Pipeline by AngioChem Inc 44
Table 36: Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) - Pipeline by ArmaGen Inc 45
Table 37: Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) - Pipeline by Denali Therapeutics Inc 45
Table 38: Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) - Pipeline by GC Pharma 46
Table 39: Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) - Pipeline by JCR Pharmaceuticals Co Ltd 47
Table 40: Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) - Pipeline by Laboratorios Del Dr Esteve SA 47
Table 41: Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) - Pipeline by RegenxBio Inc 48
Table 42: Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) - Pipeline by Sangamo Therapeutics Inc 48
Table 43: Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) - Pipeline by Shire Plc 49
Table 44: Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Pipeline by Abeona Therapeutics Inc 50
Table 45: Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Pipeline by ArmaGen Inc 50
Table 46: Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Pipeline by Axcentua Pharmaceuticals AB 51
Table 47: Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Pipeline by BioMarin Pharmaceutical Inc 51
Table 48: Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Pipeline by Laboratorios Del Dr Esteve SA 52
Table 49: Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Pipeline by Lysogene SAS 52
Table 50: Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Pipeline by Swedish Orphan Biovitrum AB 53
Table 51: Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Dormant Projects 54
Table 52: Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) - Dormant Projects 55
Table 53: Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Dormant Projects 56
Table 54: Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Discontinued Products 57
Table 55: Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) - Discontinued Products 57
Table 56: Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Discontinued Products 57

1.2 List of Figures
Figure 1: Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 9
Figure 2: Number of Products under Development by Companies Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 10
Figure 3: Number of Products under Development by Universities/Institutes Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 11
Figure 4: Number of Products under Development for Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 14
Figure 5: Number of Products under Development by Companies Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 15
Figure 6: Number of Products under Development by Universities/Institutes Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 16
Figure 7: Number of Products under Development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 19
Figure 8: Number of Products under Development by Companies Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 20
Figure 9: Number of Products by Stage and Targets Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 23
Figure 10: Number of Products by Mechanism of Actions Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 24
Figure 11: Number of Products by Stage and Mechanism of Actions Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 24
Figure 12: Number of Products by Routes of Administration Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 25
Figure 13: Number of Products by Stage and Routes of Administration Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 26
Figure 14: Number of Products by Molecule Types Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 27
Figure 15: Number of Products by Stage and Molecule Types Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 27
Figure 16: Number of Products by Stage and Targets Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 28
Figure 17: Number of Products by Mechanism of Actions Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 29
Figure 18: Number of Products by Stage and Mechanism of Actions Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 30
Figure 19: Number of Products by Routes of Administration Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 31
Figure 20: Number of Products by Stage and Routes of Administration Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 31
Figure 21: Number of Products by Molecule Types Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 32
Figure 22: Number of Products by Stage and Molecule Types Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) 33
Figure 23: Number of Products by Targets Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 34
Figure 24: Number of Products by Stage and Targets Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 34
Figure 25: Number of Products by Mechanism of Actions Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 35
Figure 26: Number of Products by Stage and Mechanism of Actions Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 36
Figure 27: Number of Products by Routes of Administration Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 37
Figure 28: Number of Products by Stage and Routes of Administration Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 37
Figure 29: Number of Products by Molecule Types Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 38
Figure 30: Number of Products by Stage and Molecule Types Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 39

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