Mucopolysaccharidosis ' Pipeline Review, H2 2012

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Mucopolysaccharidosis ' Pipeline Review, H2 2012
Published on December 2012

Report Summary


Summary

Global Markets Direct's, 'Mucopolysaccharidosis - Pipeline Review, H2 2012', provides an overview of the indication's therapeutic
pipeline. This report provides information on the therapeutic development for Mucopolysaccharidosis, complete with latest updates,
and special features on late-stage and discontinued projects. It also reviews key players involved in the therapeutic development for
Mucopolysaccharidosis. Mucopolysaccharidosis - Pipeline Review, Half Year is built using data and information sourced from Global
Markets Direct's proprietary databases, Company/University websites, SEC filings, investor presentations and featured press releases
from company/university sites and industry-specific third party sources, put together by Global Markets Direct's team.

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

Scope

- A snapshot of the global therapeutic scenario for Mucopolysaccharidosis.
- A review of the Mucopolysaccharidosis products under development by companies and universities/research institutes based on
information derived from company and industry-specific sources.
- Coverage of products based on various stages of development ranging from discovery till registration stages.
- A feature on pipeline projects on the basis of monotherapy and combined therapeutics.
- Coverage of the Mucopolysaccharidosis pipeline on the basis of route of administration and molecule type.
- Key discontinued pipeline projects.
- Latest news and deals relating to the products.

Reasons to buy

- Identify and understand important and diverse types of therapeutics under development for Mucopolysaccharidosis.
- Identify emerging players with potentially strong product portfolio and design effective counter-strategies to gain competitive
advantage.
- Plan mergers and acquisitions effectively by identifying players of the most promising pipeline.
- Devise corrective measures for pipeline projects by understanding Mucopolysaccharidosis 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.

Mucopolysaccharidosis ' Pipeline Review, H2 2012 (From Slideshare) Page 1/8


Table of Content

Table of Contents
Table of Contents 2
List of Tables 6
List of Figures 7
Introduction 8
Global Markets Direct Report Coverage 8
Mucopolysaccharidosis Overview 9
Therapeutics Development 10
An Overview of Pipeline Products for Mucopolysaccharidosis 10
Mucopolysaccharidosis Therapeutics under Development by Companies 12
Mucopolysaccharidosis Therapeutics under Investigation by Universities/Institutes 15
Late Stage Products 16
Comparative Analysis 16
Mid Clinical Stage Products 17
Early Clinical Stage Products 18
Discovery and Pre-Clinical Stage Products 19
Mucopolysaccharidosis Therapeutics ' Products under Development by Companies 20
Mucopolysaccharidosis Therapeutics ' Products under Investigation by Universities/Institutes 22
Companies Involved in Mucopolysaccharidosis Therapeutics Development 23
Athersys, Inc. 23
Lentigen Corporation 24
BioMarin Pharmaceutical Inc. 25
Esteve Group 26
Shire Plc 27
OPKO Health, Inc. 28
ReGenX Biosciences, LLC 29
Green Cross Corporation 30
JCR Pharmaceuticals Co., Ltd. 31
Synageva BioPharma Corp. 32
Vivendy Therapeutics Ltd. 33
Zacharon Pharmaceuticals, Inc. 34
ArmaGen Technologies, Inc. 35
Mucopolysaccharidosis ' Therapeutics Assessment 36
Assessment by Monotherapy Products 36
Assessment by Combination Products 37
Assessment by Route of Administration 38
Assessment by Molecule Type 40
Drug Profiles 43
HGT-1410 - Drug Profile 43
Product Description 43
Mechanism of Action 43



R&D Progress 43
MultiStem - Drug Profile 45
R&D Progress 45
elosulfase alfa - Drug Profile 47
R&D Progress 47
elosulfase alfa - Drug Profile 49
R&D Progress 49
idursulfase - Drug Profile 51
R&D Progress 51
AGT-181 - Drug Profile 52
R&D Progress 52
Drug For Mucopolysaccharidosis I,II,IIIA,IIIB,IIIC - Drug Profile 53
R&D Progress 53
Campath-1H + Clofarabine + Melphalan + Hematopoietic Stem Cell Transplantation - Drug Profile 54
R&D Progress 55
Campath-1H + Cyclophosphamide + Busulfan + Allogeneic Stem Cell Transplantation - Drug Profile 56
R&D Progress 56
Busulfan + Cyclophosphamide + Antithymocyte Globulin + Hematopoietic Stem Cell Transplantation - Drug Profile 58
R&D Progress 59
laronidase - Drug Profile 60
R&D Progress 60
idursulfase - Drug Profile 61
R&D Progress 61
Aldurazyme + Hematopoietic Stem Cell Transplantation - Drug Profile 63



R&D Progress 63
GC-1111 - Drug Profile 65
R&D Progress 65
SBC-103 - Drug Profile 66
R&D Progress 66
AGT-182 - Drug Profile 67
R&D Progress 67
UX-003 - Drug Profile 68
R&D Progress 68
Drug For Hurler Syndrome - Drug Profile 69
R&D Progress 69
Drug For Maroteaux-Lamy Syndrome - Drug Profile 70
R&D Progress 70
Sanfilippo - Drug Profile 71
R&D Progress 71
Human Placental Derived Stem Cells - Drug Profile 72
R&D Progress 72
LG-1041 - Drug Profile 73
R&D Progress 73
HGT-3010 - Drug Profile 74
R&D Progress 74
Enzyme Replacement Therapy - Drug Profile 75
R&D Progress 75
Oligonucleotide For MPS-1 - Drug Profile 77



R&D Progress 77
AAV-NaGlu - Drug Profile 78
R&D Progress 78
Mucopolysaccharidosis Therapeutics ' Drug Profile Updates 79
Mucopolysaccharidosis Therapeutics ' Discontinued Products 88
Mucopolysaccharidosis Therapeutics - Dormant Products 89
Mucopolysaccharidosis ' Product Development Milestones 90
Featured News & Press Releases 90
Nov 05, 2012: BioMarin's Phase III Study Of GALNS For Treatment Of Morquio A Syndrome Meets Primary Endpoint 90
Jul 10, 2012: Athersys Receives Orphan Drug Designation For MultiStem In Hurler's Syndrome 91
Jun 18, 2012: Lysogene Completes Enrollment In Phase I/II Clinical Trial Of SAF-301 In Sanfilippo Type A Syndrome 92
Apr 20, 2012: Athersys Announces Publication Of Results Of Preclinical Study Of MultiStem In Cell Transplant 92
Mar 28, 2012: Ultragenyx Receives Orphan Drug Designation From EMA For UX003 For Treatment Of Mucopolysaccharidosis Type
7 93
Mar 08, 2012: BioMarin Completes Enrollment In Phase III Trial For GALNS For Treatment Of Morquio A Syndrome 93
Feb 28, 2012: Ultragenyx Receives Orphan Drug Designation For UX003 From FDA For Treatment Of Mucopolysaccharidosis Type 7
94
Jan 30, 2012: Synageva To Present Preclinical Data Of SBC-103 At Lysosomal Disease Network World Symposium 94
Nov 29, 2011: BioMarin Initiates Phase II Study For GALNS In Patients Under Five Years Of Age With MPS IVA 95
Nov 29, 2011: BioMarin Initiates Phase II Study For GALNS In Patients Under Five Years Of Age With MPS IVA 95
Appendix 96
Methodology 96
Coverage 96
Secondary Research 96
Primary Research 96
Expert Panel Validation 96
Contact Us 97
Disclaimer 97

List of Tables

Number of Products Under Development for Mucopolysaccharidosis, H2 2012 10
Products under Development for Mucopolysaccharidosis ' Comparative Analysis, H2 2012 11
Number of Products under Development by Companies, H2 2012 13
Number of Products under Development by Companies, H2 2012 (Contd..1) 14
Number of Products under Investigation by Universities/Institutes, H2 2012 15
Comparative Analysis by Late Stage Development, H2 2012 16
Comparative Analysis by Mid Clinical Stage Development, H2 2012 17
Comparative Analysis by Early Clinical Stage Development, H2 2012 18
Comparative Analysis by Discovery and Pre-Clinical Stage Development, H2 2012 19
Products under Development by Companies, H2 2012 20
Products under Development by Companies, H2 2012 (Contd..1) 21
Products under Investigation by Universities/Institutes, H2 2012 22
Athersys, Inc., H2 2012 23



Lentigen Corporation, H2 2012 24
BioMarin Pharmaceutical Inc., H2 2012 25
Esteve Group, H2 2012 26
Shire Plc, H2 2012 27
OPKO Health, Inc., H2 2012 28
ReGenX Biosciences, LLC, H2 2012 29
Green Cross Corporation, H2 2012 30
JCR Pharmaceuticals Co., Ltd., H2 2012 31
Synageva BioPharma Corp., H2 2012 32
Vivendy Therapeutics Ltd., H2 2012 33
Zacharon Pharmaceuticals, Inc., H2 2012 34
ArmaGen Technologies, Inc., H2 2012 35
Assessment by Monotherapy Products, H2 2012 36
Assessment by Combination Products, H2 2012 37
Assessment by Stage and Route of Administration, H2 2012 39
Assessment by Stage and Molecule Type, H2 2012 42
Mucopolysaccharidosis Therapeutics ' Drug Profile Updates 79
Mucopolysaccharidosis Therapeutics ' Discontinued Products 88
Mucopolysaccharidosis Therapeutics ' Dormant Products 89

List of Figures

Number of Products under Development for Mucopolysaccharidosis, H2 2012 10
Products under Development for Mucopolysaccharidosis ' Comparative Analysis, H2 2012 11
Products under Development by Companies, H2 2012 12
Products under Investigation by Universities/Institutes, H2 2012 15
Late Stage Products, H2 2012 16
Mid Clinical Stage Products, H2 2012 17
Early Clinical Stage Products, H2 2012 18
Discovery and Pre-Clinical Stage Products, H2 2012 19
Assessment by Monotherapy Products, H2 2012 36
Assessment by Combination Products, H2 2012 37
Assessment by Route of Administration, H2 2012 38
Assessment by Stage and Route of Administration, H2 2012 39
Assessment by Molecule Type, H2 2012 40
Assessment by Stage and Molecule Type, H2 2012 41


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