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Duchenne Muscular Dystrophy-Pipeline Review, H1 2015

Duchenne Muscular Dystrophy-Pipeline Review, H1 2015


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Executive Summary

Duchenne Muscular Dystrophy-Pipeline Review, H1 2015

Summary

Global Markets Direct's, 'Duchenne Muscular Dystrophy-Pipeline Review, H1 2015', provides an overview of the Duchenne Muscular Dystrophy's therapeutic pipeline.

This report provides comprehensive information on the therapeutic development for Duchenne Muscular Dystrophy, complete with comparative analysis at various stages, therapeutics assessment by drug target, mechanism of action (MoA), route of administration (RoA) and molecule type, along with latest updates, and featured news and press releases. It also reviews key players involved in the therapeutic development for Duchenne Muscular Dystrophy and special features on late-stage 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, 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. Drug profiles/records featured in the report undergoes periodic updation following a stringent set of processes that ensures that all the profiles are updated with the latest set of information. Additionally, processes including live news & deals tracking, browser based alert-box and clinical trials registries tracking ensure that the most recent developments are captured on a real time basis.

The report enhances decision making capabilities and help to create effective counter strategies to gain competitive advantage. It strengthens R&D pipelines by identifying new targets and MOAs to produce first-in-class and best-in-class products.

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

Scope

The report provides a snapshot of the global therapeutic landscape of Duchenne Muscular Dystrophy

The report reviews key pipeline products under drug profile section which includes, product description, MoA and R&D brief, licensing and collaboration details & other developmental activities

The report reviews key players involved in the therapeutics development for Duchenne Muscular Dystrophy and enlists all their major and minor projects

The report summarizes all the dormant and discontinued pipeline projects

A review of the Duchenne Muscular Dystrophy products under development by companies and universities/research institutes based on information derived from company and industry-specific sources

Pipeline products coverage based on various stages of development ranging from pre-registration till discovery and undisclosed stages

A detailed assessment of monotherapy and combination therapy pipeline projects

Coverage of the Duchenne Muscular Dystrophy pipeline on the basis of target, MoA, route of administration and molecule type

Latest news and deals relating related to pipeline products

Reasons To Buy

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

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

Develop strategic initiatives by understanding the focus areas of leading companies

Identify and understand important and diverse types of therapeutics under development for Duchenne Muscular Dystrophy

Plan mergers and acquisitions effectively by identifying key players of the most promising pipeline

Devise corrective measures for pipeline projects by understanding Duchenne Muscular Dystrophy 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

Table of Contents

Table of Contents 2

List of Tables 5

List of Figures 7

Introduction 8

Global Markets Direct Report Coverage 8

Duchenne Muscular Dystrophy Overview 9

Therapeutics Development 10

Pipeline Products for Duchenne Muscular Dystrophy-Overview 10

Pipeline Products for Duchenne Muscular Dystrophy-Comparative Analysis 11

Duchenne Muscular Dystrophy-Therapeutics under Development by Companies 12

Duchenne Muscular Dystrophy-Therapeutics under Investigation by Universities/Institutes 16

Duchenne Muscular Dystrophy-Pipeline Products Glance 17

Late Stage Products 17

Clinical Stage Products 18

Early Stage Products 19

Duchenne Muscular Dystrophy-Products under Development by Companies 20

Duchenne Muscular Dystrophy-Products under Investigation by Universities/Institutes 25

Duchenne Muscular Dystrophy-Companies Involved in Therapeutics Development 26

Acceleron Pharma, Inc. 26

Asklepios BioPharmaceutical, Inc. 27

Bristol-Myers Squibb Company 28

Catabasis Pharmaceuticals, Inc. 29

Daiichi Sankyo Company, Limited 30

Eli Lilly and Company 31

EryDel SPA 32

Fate Therapeutics, Inc. 33

Galapagos NV 34

Genethon 35

Idera Pharmaceuticals, Inc. 36

Italfarmaco S.p.A. 37

Marathon Pharmaceuticals, LLC 38

Marina Biotech, Inc. 39

miRagen Therapeutics, Inc. 40

NicOx S.A. 41

Nippon Shinyaku Co., Ltd. 42

nLife Therapeutics, S.L. 43

Nobelpharma Co., Ltd. 44

Pfizer Inc. 45

Prosensa Therapeutics B.V. 46

Prothelia, Inc. 47

PTC Therapeutics, Inc. 48

Reata Pharmaceuticals, Inc. 49

Retrophin Inc. 50

ReveraGen BioPharma, Inc. 51

Sanofi 52

Santhera Pharmaceuticals Holding AG 53

Sarepta Therapeutics, Inc. 54

Shenzhen Beike Biotechnology Co., Ltd. 55

Shire Plc 56

Summit Corporation plc 57

Taiho Pharmaceutical Co., Ltd. 58

Zambon Company S.p.A. 59

Duchenne Muscular Dystrophy-Therapeutics Assessment 60

Assessment by Monotherapy Products 60

Assessment by Combination Products 61

Assessment by Target 62

Assessment by Mechanism of Action 64

Assessment by Route of Administration 66

Assessment by Molecule Type 68

Drug Profiles 70

AAV1-FS-344-Drug Profile 70

ACE-083-Drug Profile 71

Antisense Oligonucleotide for Duchenne Muscular Dystrophy-Drug Profile 72

Antisense Oligonucleotide to Activate Dystrophin for Duchene Muscular Dystrophy-Drug Profile 73

Antisense Oligonucleotide to Inhibit Myostatin for DMD and Becker MD-Drug Profile 74

Aptamer to Activate Calcineurin A for Genetic Disorders and Musculoskeletal Disorders-Drug Profile 75

arbekacin-Drug Profile 76

ARM-210-Drug Profile 77

AT-300-Drug Profile 78

ataluren-Drug Profile 80

Biostrophin-Drug Profile 83

BMS-986089-Drug Profile 84

CAT-1004-Drug Profile 85

CAT-1040-Drug Profile 87

CAT-1041-Drug Profile 88

deflazacort-Drug Profile 89

dexamethasone sodium phosphate-Drug Profile 90

drisapersen-Drug Profile 92

Drugs for Duchenne Muscular Dystrophy-Drug Profile 94

DT-200-Drug Profile 95

eteplirsen-Drug Profile 97

Gene Therapy for Duchenne Muscular Dystrophy-Drug Profile 100

Gene Therapy to Activate Dysferlin for Duchenne and Limb Girdle Muscular Dystrophies-Drug Profile 101

Gene Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy-Drug Profile 102

givinostat-Drug Profile 103

halofuginone hydrobromide-Drug Profile 105

idebenone-Drug Profile 108

IMO-8400-Drug Profile 110

MG-53-Drug Profile 112

MGN-8107-Drug Profile 114

naproxcinod-Drug Profile 115

NBD Peptide-Drug Profile 117

NS-065-Drug Profile 119

NU-21101 + NU-21502-Drug Profile 120

Oligonucleotides for Ischemic Heart Disease and Duchenne Muscular Dystrophy-Drug Profile 121

OSX-200-Drug Profile 122

Peptides for Duchenne Muscular Dystrophy and Cardiac Disease-Drug Profile 123

PF-06252616-Drug Profile 124

poloxamer-Drug Profile 125

PRO-044-Drug Profile 127

PRO-045-Drug Profile 129

PRO-052-Drug Profile 131

PRO-053-Drug Profile 133

PRO-055-Drug Profile 135

PROSPECT-Drug Profile 136

PRT-01-Drug Profile 137

PRT-20-Drug Profile 138

PRT-300-Drug Profile 139

PRT-400-Drug Profile 140

PRT-500-Drug Profile 141

RE-001-Drug Profile 142

Recombinant Protein for Muscular Dystrophy-Drug Profile 143

Recombinant Proteins for Duchenne Muscular Dystrophy-Drug Profile 144

RNAi Oligonucleotides for Duchenne Muscular Dystrophy-Drug Profile 145

RTC-13-Drug Profile 146

SHP-619-Drug Profile 148

Small Molecule for Duchenne Muscular Dystrophy-Drug Profile 149

Small Molecule for Muscular Dystrophy and Other Muscle Disorders-Drug Profile 150

Small Molecule to Activate Dystrophin for Duchenne Muscular Dystrophy-Drug Profile 152

Small Molecule to Activate Utrophin for Duchenne Muscular Dystrophy-Drug Profile 153

Small Molecules to Activate Nrf2 for Duchenne Muscular Dystrophy-Drug Profile 154

Small Molecules to Activate Utrophin for Duchenne Muscular Dystrophy-Drug Profile 155

Small Molecules to Target Utrophin for Duchenne Muscular Dystrophy-Drug Profile 156

SMTC-1100-Drug Profile 157

SRP-4008-Drug Profile 159

SRP-4044-Drug Profile 160

SRP-4045-Drug Profile 161

SRP-4050-Drug Profile 162

SRP-4052-Drug Profile 163

SRP-4053-Drug Profile 164

SRP-4055-Drug Profile 165

Stem Cell Therapy for Duchenne Muscular Dystrophy-Drug Profile 166

Stem Cell Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy-Drug Profile 167

tadalafil-Drug Profile 169

TAS-205-Drug Profile 171

TVN-102-Drug Profile 172

TXA-127-Drug Profile 173

VBP-15-Drug Profile 175

ZP-049-Drug Profile 177

Duchenne Muscular Dystrophy-Recent Pipeline Updates 178

Duchenne Muscular Dystrophy-Dormant Projects 220

Duchenne Muscular Dystrophy-Discontinued Products 222

Duchenne Muscular Dystrophy-Product Development Milestones 223

Featured News & Press Releases 223

Appendix 230

Methodology 230

Coverage 230

Secondary Research 230

Primary Research 230

Expert Panel Validation 230

Contact Us 230

Disclaimer 231

List of Tables

Number of Products under Development for Duchenne Muscular Dystrophy, H1 2015 16

Number of Products under Development for Duchenne Muscular Dystrophy-Comparative Analysis, H1 2015 17

Number of Products under Development by Companies, H1 2015 19

Number of Products under Development by Companies, H1 2015 (Contd..1) 20

Number of Products under Development by Companies, H1 2015 (Contd..2) 21

Number of Products under Investigation by Universities/Institutes, H1 2015 22

Comparative Analysis by Late Stage Development, H1 2015 23

Comparative Analysis by Clinical Stage Development, H1 2015 24

Comparative Analysis by Early Stage Development, H1 2015 25

Products under Development by Companies, H1 2015 26

Products under Development by Companies, H1 2015 (Contd..1) 27

Products under Development by Companies, H1 2015 (Contd..2) 28

Products under Development by Companies, H1 2015 (Contd..3) 29

Products under Development by Companies, H1 2015 (Contd..4) 30

Products under Investigation by Universities/Institutes, H1 2015 31

Duchenne Muscular Dystrophy-Pipeline by Acceleron Pharma, Inc., H1 2015 32

Duchenne Muscular Dystrophy-Pipeline by Asklepios BioPharmaceutical, Inc., H1 2015 33

Duchenne Muscular Dystrophy-Pipeline by Bristol-Myers Squibb Company, H1 2015 34

Duchenne Muscular Dystrophy-Pipeline by Catabasis Pharmaceuticals, Inc., H1 2015 35

Duchenne Muscular Dystrophy-Pipeline by Daiichi Sankyo Company, Limited, H1 2015 36

Duchenne Muscular Dystrophy-Pipeline by Eli Lilly and Company, H1 2015 37

Duchenne Muscular Dystrophy-Pipeline by EryDel SPA, H1 2015 38

Duchenne Muscular Dystrophy-Pipeline by Fate Therapeutics, Inc., H1 2015 39

Duchenne Muscular Dystrophy-Pipeline by Galapagos NV, H1 2015 40

Duchenne Muscular Dystrophy-Pipeline by Genethon, H1 2015 41

Duchenne Muscular Dystrophy-Pipeline by Idera Pharmaceuticals, Inc., H1 2015 42

Duchenne Muscular Dystrophy-Pipeline by Italfarmaco S.p.A., H1 2015 43

Duchenne Muscular Dystrophy-Pipeline by Marathon Pharmaceuticals, LLC, H1 2015 44

Duchenne Muscular Dystrophy-Pipeline by Marina Biotech, Inc., H1 2015 45

Duchenne Muscular Dystrophy-Pipeline by miRagen Therapeutics, Inc., H1 2015 46

Duchenne Muscular Dystrophy-Pipeline by NicOx S.A., H1 2015 47

Duchenne Muscular Dystrophy-Pipeline by Nippon Shinyaku Co., Ltd., H1 2015 48

Duchenne Muscular Dystrophy-Pipeline by nLife Therapeutics, S.L., H1 2015 49

Duchenne Muscular Dystrophy-Pipeline by Nobelpharma Co., Ltd., H1 2015 50

Duchenne Muscular Dystrophy-Pipeline by Pfizer Inc., H1 2015 51

Duchenne Muscular Dystrophy-Pipeline by Prosensa Therapeutics B.V., H1 2015 52

Duchenne Muscular Dystrophy-Pipeline by Prothelia, Inc., H1 2015 53

Duchenne Muscular Dystrophy-Pipeline by PTC Therapeutics, Inc., H1 2015 54

Duchenne Muscular Dystrophy-Pipeline by Reata Pharmaceuticals, Inc., H1 2015 55

Duchenne Muscular Dystrophy-Pipeline by Retrophin Inc., H1 2015 56

Duchenne Muscular Dystrophy-Pipeline by ReveraGen BioPharma, Inc., H1 2015 57

Duchenne Muscular Dystrophy-Pipeline by Sanofi, H1 2015 58

Duchenne Muscular Dystrophy-Pipeline by Santhera Pharmaceuticals Holding AG, H1 2015 59

Duchenne Muscular Dystrophy-Pipeline by Sarepta Therapeutics, Inc., H1 2015 60

Duchenne Muscular Dystrophy-Pipeline by Shenzhen Beike Biotechnology Co., Ltd., H1 2015 61

Duchenne Muscular Dystrophy-Pipeline by Shire Plc, H1 2015 62

Duchenne Muscular Dystrophy-Pipeline by Summit Corporation plc, H1 2015 63

Duchenne Muscular Dystrophy-Pipeline by Taiho Pharmaceutical Co., Ltd., H1 2015 64

Duchenne Muscular Dystrophy-Pipeline by Zambon Company S.p.A., H1 2015 65

Assessment by Monotherapy Products, H1 2015 66

Assessment by Combination Products, H1 2015 67

Number of Products by Stage and Target, H1 2015 69

Number of Products by Stage and Mechanism of Action, H1 2015 71

Number of Products by Stage and Route of Administration, H1 2015 73

Number of Products by Stage and Molecule Type, H1 2015 75

Duchenne Muscular Dystrophy Therapeutics-Recent Pipeline Updates, H1 2015 184

Duchenne Muscular Dystrophy-Dormant Projects, H1 2015 226

Duchenne Muscular Dystrophy-Dormant Projects (Contd..1), H1 2015 227

Duchenne Muscular Dystrophy-Discontinued Products, H1 2015 228

List of Figures

Number of Products under Development for Duchenne Muscular Dystrophy, H1 2015 16

Number of Products under Development for Duchenne Muscular Dystrophy-Comparative Analysis, H1 2015 17

Number of Products under Development by Companies, H1 2015 18

Number of Products under Investigation by Universities/Institutes, H1 2015 22

Comparative Analysis by Late Stage Development, H1 2015 23

Comparative Analysis by Clinical Stage Development, H1 2015 24

Comparative Analysis by Early Stage Products, H1 2015 25

Assessment by Monotherapy Products, H1 2015 66

Number of Products by Top 10 Targets, H1 2015 68

Number of Products by Stage and Top 10 Targets, H1 2015 68

Number of Products by Top 10 Mechanism of Actions, H1 2015 70

Number of Products by Stage and Top 10 Mechanism of Actions, H1 2015 70

Number of Products by Top 10 Routes of Administration, H1 2015 72

Number of Products by Stage and Top 10 Routes of Administration, H1 2015 72

Number of Products by Top 10 Molecule Types, H1 2015 74

Number of Products by Stage and Top 10 Molecule Types, H1 2015 74

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Acceleron Pharma, Inc.

Asklepios BioPharmaceutical, Inc.

Bristol-Myers Squibb Company

Catabasis Pharmaceuticals, Inc.

Daiichi Sankyo Company, Limited

Eli Lilly and Company

EryDel SPA

Fate Therapeutics, Inc.

Galapagos NV

Genethon

Idera Pharmaceuticals, Inc.

Italfarmaco S.p.A.

Marathon Pharmaceuticals, LLC

Marina Biotech, Inc.

miRagen Therapeutics, Inc.

NicOx S.A.

Nippon Shinyaku Co., Ltd.

nLife Therapeutics, S.L.

Nobelpharma Co., Ltd.

Pfizer Inc.

Prosensa Therapeutics B.V.

Prothelia, Inc.

PTC Therapeutics, Inc.

Reata Pharmaceuticals, Inc.

Retrophin Inc.

ReveraGen BioPharma, Inc.

Sanofi

Santhera Pharmaceuticals Holding AG

Sarepta Therapeutics, Inc.

Shenzhen Beike Biotechnology Co., Ltd.

Shire Plc

Summit Corporation plc

Taiho Pharmaceutical Co., Ltd.

Zambon Company S.p.A.

Duchenne Muscular Dystrophy Therapeutic Products under Development, Key Players in Duchenne Muscular Dystrophy Therapeutics, Duchenne Muscular Dystrophy Pipeline Overview, Duchenne Muscular Dystrophy Pipeline, Duchenne Muscular Dystrophy Pipeline Assessment


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