Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) – Pipeline Review, H2 2016

Global Markets Direct’s, ‘Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) – Pipeline Review, H2 2016’, provides an overview of the Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) and features dormant and discontinued projects.

Global Markets Direct’s report features investigational drugs from across globe covering over 20 therapy areas and nearly 3,000 indications. The report is built using data and information sourced from Global Markets Direct’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Drug profiles featured in the report undergoes periodic review following a stringent set of processes to ensure that all the profiles are updated with the latest set of information. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

The report helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage.

Note: Certain sections in the report may be removed or altered based on the availability and relevance of data.

Scope

The report provides a snapshot of the global therapeutic landscape of Mucopolysaccharidosis II (MPS II) (Hunter Syndrome )

The report reviews pipeline therapeutics for Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) by companies and universities/research institutes based on information derived from company and industry-specific sources

The report covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages

The report features descriptive drug profiles for the pipeline products which includes, product description, descriptive MoA, R&D brief, licensing and collaboration details & other developmental activities

The report reviews key players involved Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) therapeutics and enlists all their major and minor projects

The report assesses Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) therapeutics based on drug target, mechanism of action (MoA), route of administration (RoA) and molecule type

The report summarizes all the dormant and discontinued pipeline projects

The report reviews latest news related to pipeline therapeutics for Mucopolysaccharidosis II (MPS II) (Hunter Syndrome )

Reasons to buy

Gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies

Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage

Identify and understand important and diverse types of therapeutics under development for Mucopolysaccharidosis II (MPS II) (Hunter Syndrome )

Identify potential new clients or partners in the target demographic

Develop strategic initiatives by understanding the focus areas of leading companies

Plan mergers and acquisitions effectively by identifying key players and it’s most promising pipeline therapeutics

Devise corrective measures for pipeline projects by understanding Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) pipeline depth and focus of Indication therapeutics

Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope

Modify the therapeutic portfolio by identifying discontinued projects and understanding the factors that drove them from pipeline

Companies mentioned

Alexion Pharmaceuticals Inc

AngioChem Inc

ArmaGen Inc

Bioasis Technologies Inc

Green Cross Corp

Inventiva

JCR Pharmaceuticals Co Ltd

Laboratorios Del Dr Esteve SA

RegenxBio Inc

Sangamo BioSciences Inc

Shire Plc

Table of Contents

Table of Contents

Table of Contents 2

List of Tables 5

List of Figures 6

Introduction 7

Global Markets Direct Report Coverage 7

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) Overview 8

Therapeutics Development 9

Pipeline Products for Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Overview 9

Pipeline Products for Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Comparative Analysis 10

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Therapeutics under Development by Companies 11

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Therapeutics under Investigation by Universities/Institutes 12

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline Products Glance 13

Late Stage Products 13

Clinical Stage Products 14

Early Stage Products 15

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Products under Development by Companies 16

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Products under Investigation by Universities/Institutes 17

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Companies Involved in Therapeutics Development 18

Alexion Pharmaceuticals Inc 18

AngioChem Inc 19

ArmaGen Inc 20

Bioasis Technologies Inc 21

Green Cross Corp 22

Inventiva 23

JCR Pharmaceuticals Co Ltd 24

Laboratorios Del Dr Esteve SA 25

RegenxBio Inc 26

Sangamo BioSciences Inc 27

Shire Plc 28

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Therapeutics Assessment 29

Assessment by Monotherapy Products 29

Assessment by Target 30

Assessment by Mechanism of Action 32

Assessment by Route of Administration 34

Assessment by Molecule Type 36

Drug Profiles 38

AGT-182 - Drug Profile 38

Product Description 38

Mechanism Of Action 38

R&D Progress 38

DUOC-01 - Drug Profile 40

Product Description 40

Mechanism Of Action 40

R&D Progress 40

EGT-301 - Drug Profile 41

Product Description 41

Mechanism Of Action 41

R&D Progress 41

Gene Therapy to Activate Iduronate 2 Sulfatase for Mucopolysaccharidosis II - Drug Profile 42

Product Description 42

Mechanism Of Action 42

R&D Progress 42

idursulfase - Drug Profile 43

Product Description 43

Mechanism Of Action 43

R&D Progress 43

idursulfase beta - Drug Profile 45

Product Description 45

Mechanism Of Action 45

R&D Progress 45

JR-032 - Drug Profile 47

Product Description 47

Mechanism Of Action 47

R&D Progress 47

JR-141 - Drug Profile 48

Product Description 48

Mechanism Of Action 48

R&D Progress 48

MTf-I2S - Drug Profile 49

Product Description 49

Mechanism Of Action 49

R&D Progress 49

MTfp-I2S - Drug Profile 50

Product Description 50

Mechanism Of Action 50

R&D Progress 50

odiparcil - Drug Profile 51

Product Description 51

Mechanism Of Action 51

R&D Progress 51

pentosan polysulfate sodium - Drug Profile 52

Product Description 52

Mechanism Of Action 52

R&D Progress 52

Recombinant Enzymes for Pompe, Fabry and Hunter Diseases - Drug Profile 53

Product Description 53

Mechanism Of Action 53

R&D Progress 53

Recombinant Iduronate 2-Sulfatase Replacement for Mucopolysaccharidosis II - Drug Profile 54

Product Description 54

Mechanism Of Action 54

R&D Progress 54

RGX-121 - Drug Profile 55

Product Description 55

Mechanism Of Action 55

R&D Progress 55

SB-913 - Drug Profile 57

Product Description 57

Mechanism Of Action 57

R&D Progress 57

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Dormant Projects 59

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Discontinued Products 60

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) - Product Development Milestones 61

Featured News & Press Releases 61

Oct 26, 2016: National Center for Child Health and Development Initiates Phase 1/2 Trial of Green Cross' idursulfase-beta ICV for the Treatment of Hunter Syndrome with Neurocognitive Decline 61

Sep 13, 2016: REGENXBIO Publishes Data from Ongoing Preclinical Studies of NAV Gene Therapy RGX-121 61

Jul 05, 2016: REGENXBIO Provides Update On Gene Therapy Development Program RGX-121 62

Jun 20, 2016: Sangamo BioSciences Announces FDA Clearance of Investigational New Drug Application for ZFN-Mediated Genome Editing Treatment of MPS II 62

Apr 19, 2016: Preclinical Data from REGENXBIO’s RGX-121 Gene Therapy Program to be Presented at the American Society of Gene & Cell Therapy 19th Annual Meeting 63

Feb 25, 2016: ESTEVE Provides Update On EGT-301 For Hunter Syndrome 64

Jul 31, 2015: biOasis Announces the Successful Delivery to the CNS of an Enzyme used to Treat Hunter Syndrome 65

Sep 18, 2014: biOasis Initiates MPS II Study with Renowned Lysosomal Storage Disease Expert Dr. Maurizio Scarpa and The Brains for Brain Foundation 65

Oct 02, 2013: Green Cross Exports Orphan Disease Drug Hunterase to Algeria 66

Apr 08, 2013: Green Cross Announces Publication Of Clinical Trial Paper On Hunter Syndrome Drug Hunterase In International Scientific Press 67

Feb 18, 2013: Green Cross Receives FDA Orphan Drug Designation For Hunterase For Treatment Of Hunter Syndrome 68

Jan 10, 2012: Green Cross Receives Korean Approval For Hunterase 68

Oct 12, 2011: Green Cross Files Application For Korean Approval Of Hunterase For Treatment Of Hunter Syndrome 69

Appendix 70

Methodology 70

Coverage 70

Secondary Research 70

Primary Research 70

Expert Panel Validation 70

Contact Us 70

Disclaimer 71

List of Tables

List of Tables

Number of Products under Development for Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ), H2 2016 9

Number of Products under Development for Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) – Comparative Analysis, H2 2016 10

Number of Products under Development by Companies, H2 2016 11

Number of Products under Investigation by Universities/Institutes, H2 2016 12

Comparative Analysis by Late Stage Development, H2 2016 13

Comparative Analysis by Clinical Stage Development, H2 2016 14

Comparative Analysis by Early Stage Development, H2 2016 15

Products under Development by Companies, H2 2016 16

Products under Investigation by Universities/Institutes, H2 2016 17

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) – Pipeline by Alexion Pharmaceuticals Inc, H2 2016 18

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) – Pipeline by AngioChem Inc, H2 2016 19

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) – Pipeline by ArmaGen Inc, H2 2016 20

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) – Pipeline by Bioasis Technologies Inc, H2 2016 21

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) – Pipeline by Green Cross Corp, H2 2016 22

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) – Pipeline by Inventiva, H2 2016 23

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) – Pipeline by JCR Pharmaceuticals Co Ltd, H2 2016 24

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) – Pipeline by Laboratorios Del Dr Esteve SA, H2 2016 25

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) – Pipeline by RegenxBio Inc, H2 2016 26

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) – Pipeline by Sangamo BioSciences Inc, H2 2016 27

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) – Pipeline by Shire Plc, H2 2016 28

Assessment by Monotherapy Products, H2 2016 29

Number of Products by Stage and Target, H2 2016 31

Number of Products by Stage and Mechanism of Action, H2 2016 33

Number of Products by Stage and Route of Administration, H2 2016 35

Number of Products by Stage and Molecule Type, H2 2016 37

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) – Dormant Projects, H2 2016 59

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) – Discontinued Products, H2 2016 60

List of Figures

List of Figures

Number of Products under Development for Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ), H2 2016 9

Number of Products under Development for Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) – Comparative Analysis, H2 2016 10

Number of Products under Development by Companies, H2 2016 11

Number of Products under Investigation by Universities/Institutes, H2 2016 12

Comparative Analysis by Clinical Stage Development, H2 2016 14

Comparative Analysis by Early Stage Products, H2 2016 15

Assessment by Monotherapy Products, H2 2016 29

Number of Products by Targets, H2 2016 30

Number of Products by Stage and Targets, H2 2016 30

Number of Products by Mechanism of Actions, H2 2016 32

Number of Products by Stage and Mechanism of Actions, H2 2016 32

Number of Products by Routes of Administration, H2 2016 34

Number of Products by Stage and Routes of Administration, H2 2016 34

Number of Products by Molecule Types, H2 2016 36

Number of Products by Stage and Molecule Types, H2 2016 36

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