Duchenne Muscular Dystrophy-Pipeline Review, H2 2016

Duchenne Muscular Dystrophy-Pipeline Review, H2 2016


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Executive Summary

Duchenne Muscular Dystrophy-Pipeline Review, H2 2016

Summary

Global Markets Direct's, Duchenne Muscular Dystrophy-Pipeline Review, H2 2016', provides an overview of the Duchenne Muscular Dystrophy pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Duchenne Muscular Dystrophy, 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 Duchenne Muscular Dystrophy 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 Duchenne Muscular Dystrophy

The report reviews pipeline therapeutics for Duchenne Muscular Dystrophy 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 Duchenne Muscular Dystrophy therapeutics and enlists all their major and minor projects

The report assesses Duchenne Muscular Dystrophy 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 Duchenne Muscular Dystrophy

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 Duchenne Muscular Dystrophy

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 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



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Ankur Gupta, Head Marketing & Communications

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Table of Contents

Table of Contents 2

List of Tables 6

List of Figures 8

Introduction 9

Global Markets Direct Report Coverage 9

Duchenne Muscular Dystrophy Overview 10

Therapeutics Development 11

Pipeline Products for Duchenne Muscular Dystrophy-Overview 11

Pipeline Products for Duchenne Muscular Dystrophy-Comparative Analysis 12

Duchenne Muscular Dystrophy-Therapeutics under Development by Companies 13

Duchenne Muscular Dystrophy-Therapeutics under Investigation by Universities/Institutes 18

Duchenne Muscular Dystrophy-Pipeline Products Glance 19

Late Stage Products 19

Clinical Stage Products 20

Early Stage Products 21

Unknown Stage Products 22

Duchenne Muscular Dystrophy-Products under Development by Companies 23

Duchenne Muscular Dystrophy-Products under Investigation by Universities/Institutes 28

Duchenne Muscular Dystrophy-Companies Involved in Therapeutics Development 29

Acceleron Pharma, Inc. 29

Anagenesis Biotechnology 30

Asklepios BioPharmaceutical, Inc. 31

BioMarin Pharmaceutical Inc. 32

Bristol-Myers Squibb Company 33

Capricor Therapeutics, Inc. 34

Catabasis Pharmaceuticals, Inc. 35

Daiichi Sankyo Company, Limited 36

Debiopharm International SA 37

Editas Medicine, Inc. 38

EryDel SPA 39

FibroGen, Inc. 40

Galapagos NV 41

Genethon 42

GTx, Inc. 43

GW Pharmaceuticals Plc 44

Idera Pharmaceuticals, Inc. 45

Italfarmaco S.p.A. 46

La Jolla Pharmaceutical Company 47

Lexicon Pharmaceuticals, Inc. 48

Marathon Pharmaceuticals, LLC 49

Marina Biotech, Inc. 50

Merck KGaA 51

Milo Biotechnology LLC 52

Mitochon Pharmaceuticals, Inc. 53

MyoTherix Inc. 54

NicOx S.A. 55

Nippon Shinyaku Co., Ltd. 56

Nobelpharma Co., Ltd. 57

Pfizer Inc. 58

Pluristem Therapeutics Inc. 59

Prothelia, Inc. 60

PTC Therapeutics, Inc. 61

RASRx, LLC 62

ReveraGen BioPharma, Inc. 63

Santhera Pharmaceuticals Holding AG 64

Sarepta Therapeutics, Inc. 65

Summit Therapeutics Plc 66

Taiho Pharmaceutical Co., Ltd. 67

WAVE Life Sciences Ltd. 68

Zambon Company S.p.A. 69

Duchenne Muscular Dystrophy-Therapeutics Assessment 70

Assessment by Monotherapy Products 70

Assessment by Target 71

Assessment by Mechanism of Action 74

Assessment by Route of Administration 77

Assessment by Molecule Type 79

Drug Profiles 81

4-P001-Drug Profile 81

AAV1-Foliistatin-Drug Profile 82

ACE-083-Drug Profile 84

alisporivir-Drug Profile 86

Antisense Oligonucleotide 1 to Activate Dystrophin for Duchenne Muscular Dystrophy-Drug Profile 89

Antisense Oligonucleotide to Activate Dystrophin for Duchenne Muscular Dystrophy-Drug Profile 90

Antisense Oligonucleotide to Inhibit Myostatin for DMD and Becker MD-Drug Profile 91

Antisense Oligonucleotides to Inhibit ACVR2B for Duchenne Muscular Dystrophy-Drug Profile 92

Antisense RNAi Oligonucleotides for Duchenne Muscular Dystrophy-Drug Profile 93

Aptamer to Activate Calcineurin A for Genetic Disorders and Musculoskeletal Disorders-Drug Profile 94

arbekacin-Drug Profile 95

ARM-210-Drug Profile 96

AT-300-Drug Profile 98

ataluren-Drug Profile 100

Biostrophin-Drug Profile 108

BMBD-001-Drug Profile 109

BMS-986089-Drug Profile 110

CAP-1002-Drug Profile 111

CAP-2003-Drug Profile 116

CAT-1004-Drug Profile 117

DA-4210-Drug Profile 121

deflazacort-Drug Profile 122

dexamethasone sodium phosphate-Drug Profile 124

domagrozumab-Drug Profile 126

Drug for Duchenne Muscular Dystrophy-Drug Profile 128

Drugs for Duchenne Muscular Dystrophy-Drug Profile 129

DS-5141-Drug Profile 130

DT-200-Drug Profile 131

enobosarm-Drug Profile 132

eteplirsen-Drug Profile 139

ezutromid-Drug Profile 150

Gene Therapy for Duchenne Muscular Dystrophy-Drug Profile 156

Gene Therapy to Activate Dysferlin for Duchenne and Limb Girdle Muscular Dystrophies-Drug Profile 157

Gene Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy-Drug Profile 158

Gene Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy-Drug Profile 159

Gene Therapy to Activate Utrophin for Duchenne Muscular Dystrophy-Drug Profile 160

givinostat-Drug Profile 161

Gtx-027-Drug Profile 163

IB-DMD-Drug Profile 164

idebenone-Drug Profile 165

IMO-8400-Drug Profile 173

LJPC-30Sa-Drug Profile 179

LJPC-30Sb-Drug Profile 180

LX-2931-Drug Profile 181

MG-53-Drug Profile 183

MP-101-Drug Profile 185

Myomir-Drug Profile 186

naproxcinod-Drug Profile 187

NS-065-Drug Profile 190

Oligonucleotide to Activate Dystrophin for Duchenne Muscular Dystrophy-Drug Profile 191

Oligonucleotides 1 to Activate Dystrophin for Duchenne Muscular Dystrophy-Drug Profile 192

Oligonucleotides 2 to Activate Dystrophin for Duchenne Muscular Dystrophy-Drug Profile 193

Oligonucleotides 3 to Activate Dystrophin for Duchenne Muscular Dystrophy-Drug Profile 194

OSX-200-Drug Profile 195

pamrevlumab-Drug Profile 196

PLX-PAD-Drug Profile 202

poloxamer-Drug Profile 211

PRO-052-Drug Profile 213

PRO-055-Drug Profile 215

PRT-01-Drug Profile 217

Recombinant Protein for Muscular Dystrophy-Drug Profile 219

Recombinant Protein to Activate Utrophin for Muscular Dystrophies-Drug Profile 220

Recombinant Proteins for Duchenne Muscular Dystrophy-Drug Profile 221

rimeporide-Drug Profile 222

RTC-13-Drug Profile 223

SB-001-Drug Profile 225

SB-002-Drug Profile 226

SGT-001-Drug Profile 227

Small Molecule to Activate Dystrophin for Duchenne Muscular Dystrophy-Drug Profile 228

Small Molecules for Duchenne Muscular Dystrophy-Drug Profile 229

Small Molecules for Duchenne Muscular Dystrophy-Drug Profile 230

Small Molecules to Activate Utrophin for Duchenne Muscular Dystrophy-Drug Profile 231

Small Molecules to Agonize Cannabinoid Receptor for Duchenne Muscular Dystrophy-Drug Profile 232

SMT-022357-Drug Profile 233

SRP-4008-Drug Profile 235

SRP-4044-Drug Profile 236

SRP-4045-Drug Profile 237

SRP-4050-Drug Profile 238

SRP-4052-Drug Profile 239

SRP-4053-Drug Profile 240

SRP-4055-Drug Profile 241

Stem Cell Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy-Drug Profile 242

Stem Cell Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy-Drug Profile 244

Stem Cell Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy-Drug Profile 245

Stryka-516-Drug Profile 246

Stryka-969-Drug Profile 247

StrykaPro-1-Drug Profile 248

Synthetic Peptides for Duchene Muscle Dystrophy-Drug Profile 249

TAS-205-Drug Profile 250

TVN-102-Drug Profile 251

TXA-127-Drug Profile 252

vamorolone-Drug Profile 256

ZP-049-Drug Profile 258

Duchenne Muscular Dystrophy-Dormant Projects 259

Duchenne Muscular Dystrophy-Discontinued Products 263

Duchenne Muscular Dystrophy-Product Development Milestones 264

Featured News & Press Releases 264

Appendix 275

Methodology 275

Coverage 275

Secondary Research 275

Primary Research 275

Expert Panel Validation 275

Contact Us 275

Disclaimer 276

List of Tables

Number of Products under Development for Duchenne Muscular Dystrophy, H2 2016 18

Number of Products under Development for Duchenne Muscular Dystrophy-Comparative Analysis, H2 2016 19

Number of Products under Development by Companies, H2 2016 21

Number of Products under Development by Companies, H2 2016 (Contd..1) 22

Number of Products under Development by Companies, H2 2016 (Contd..2) 23

Number of Products under Development by Companies, H2 2016 (Contd..3) 24

Number of Products under Investigation by Universities/Institutes, H2 2016 25

Comparative Analysis by Late Stage Development, H2 2016 26

Comparative Analysis by Clinical Stage Development, H2 2016 27

Comparative Analysis by Early Stage Development, H2 2016 28

Comparative Analysis by Unknown Stage Development, H2 2016 29

Products under Development by Companies, H2 2016 30

Products under Development by Companies, H2 2016 (Contd..1) 31

Products under Development by Companies, H2 2016 (Contd..2) 32

Products under Development by Companies, H2 2016 (Contd..3) 33

Products under Development by Companies, H2 2016 (Contd..4) 34

Products under Investigation by Universities/Institutes, H2 2016 35

Duchenne Muscular Dystrophy-Pipeline by Acceleron Pharma, Inc., H2 2016 36

Duchenne Muscular Dystrophy-Pipeline by Anagenesis Biotechnology, H2 2016 37

Duchenne Muscular Dystrophy-Pipeline by Asklepios BioPharmaceutical, Inc., H2 2016 38

Duchenne Muscular Dystrophy-Pipeline by BioMarin Pharmaceutical Inc., H2 2016 39

Duchenne Muscular Dystrophy-Pipeline by Bristol-Myers Squibb Company, H2 2016 40

Duchenne Muscular Dystrophy-Pipeline by Capricor Therapeutics, Inc., H2 2016 41

Duchenne Muscular Dystrophy-Pipeline by Catabasis Pharmaceuticals, Inc., H2 2016 42

Duchenne Muscular Dystrophy-Pipeline by Daiichi Sankyo Company, Limited, H2 2016 43

Duchenne Muscular Dystrophy-Pipeline by Debiopharm International SA , H2 2016 44

Duchenne Muscular Dystrophy-Pipeline by Editas Medicine, Inc., H2 2016 45

Duchenne Muscular Dystrophy-Pipeline by EryDel SPA, H2 2016 46

Duchenne Muscular Dystrophy-Pipeline by FibroGen, Inc., H2 2016 47

Duchenne Muscular Dystrophy-Pipeline by Galapagos NV, H2 2016 48

Duchenne Muscular Dystrophy-Pipeline by Genethon, H2 2016 49

Duchenne Muscular Dystrophy-Pipeline by GTx, Inc., H2 2016 50

Duchenne Muscular Dystrophy-Pipeline by GW Pharmaceuticals Plc, H2 2016 51

Duchenne Muscular Dystrophy-Pipeline by Idera Pharmaceuticals, Inc., H2 2016 52

Duchenne Muscular Dystrophy-Pipeline by Italfarmaco S.p.A., H2 2016 53

Duchenne Muscular Dystrophy-Pipeline by La Jolla Pharmaceutical Company, H2 2016 54

Duchenne Muscular Dystrophy-Pipeline by Lexicon Pharmaceuticals, Inc., H2 2016 55

Duchenne Muscular Dystrophy-Pipeline by Marathon Pharmaceuticals, LLC, H2 2016 56

Duchenne Muscular Dystrophy-Pipeline by Marina Biotech, Inc., H2 2016 57

Duchenne Muscular Dystrophy-Pipeline by Merck KGaA, H2 2016 58

Duchenne Muscular Dystrophy-Pipeline by Milo Biotechnology LLC, H2 2016 59

Duchenne Muscular Dystrophy-Pipeline by Mitochon Pharmaceuticals, Inc., H2 2016 60

Duchenne Muscular Dystrophy-Pipeline by MyoTherix Inc., H2 2016 61

Duchenne Muscular Dystrophy-Pipeline by NicOx S.A., H2 2016 62

Duchenne Muscular Dystrophy-Pipeline by Nippon Shinyaku Co., Ltd., H2 2016 63

Duchenne Muscular Dystrophy-Pipeline by Nobelpharma Co., Ltd., H2 2016 64

Duchenne Muscular Dystrophy-Pipeline by Pfizer Inc., H2 2016 65

Duchenne Muscular Dystrophy-Pipeline by Pluristem Therapeutics Inc., H2 2016 66

Duchenne Muscular Dystrophy-Pipeline by Prothelia, Inc., H2 2016 67

Duchenne Muscular Dystrophy-Pipeline by PTC Therapeutics, Inc., H2 2016 68

Duchenne Muscular Dystrophy-Pipeline by RASRx, LLC, H2 2016 69

Duchenne Muscular Dystrophy-Pipeline by ReveraGen BioPharma, Inc., H2 2016 70

Duchenne Muscular Dystrophy-Pipeline by Santhera Pharmaceuticals Holding AG, H2 2016 71

Duchenne Muscular Dystrophy-Pipeline by Sarepta Therapeutics, Inc., H2 2016 72

Duchenne Muscular Dystrophy-Pipeline by Summit Therapeutics Plc, H2 2016 73

Duchenne Muscular Dystrophy-Pipeline by Taiho Pharmaceutical Co., Ltd., H2 2016 74

Duchenne Muscular Dystrophy-Pipeline by WAVE Life Sciences Ltd., H2 2016 75

Duchenne Muscular Dystrophy-Pipeline by Zambon Company S.p.A., H2 2016 76

Assessment by Monotherapy Products, H2 2016 77

Number of Products by Stage and Target, H2 2016 79

Number of Products by Stage and Mechanism of Action, H2 2016 82

Number of Products by Stage and Route of Administration, H2 2016 85

Number of Products by Stage and Molecule Type, H2 2016 87

Duchenne Muscular Dystrophy-Dormant Projects, H2 2016 266

Duchenne Muscular Dystrophy-Dormant Projects (Contd..1), H2 2016 267

Duchenne Muscular Dystrophy-Dormant Projects (Contd..2), H2 2016 268

Duchenne Muscular Dystrophy-Dormant Projects (Contd..3), H2 2016 269

Duchenne Muscular Dystrophy-Discontinued Products, H2 2016 270

List of Figures

Number of Products under Development for Duchenne Muscular Dystrophy, H2 2016 18

Number of Products under Development for Duchenne Muscular Dystrophy-Comparative Analysis, H2 2016 19

Number of Products under Development by Companies, H2 2016 20

Number of Products under Investigation by Universities/Institutes, H2 2016 25

Comparative Analysis by Late Stage Development, H2 2016 26

Comparative Analysis by Clinical Stage Development, H2 2016 27

Comparative Analysis by Early Stage Products, H2 2016 28

Assessment by Monotherapy Products, H2 2016 77

Number of Products by Top 10 Targets, H2 2016 78

Number of Products by Stage and Top 10 Targets, H2 2016 78

Number of Products by Top 10 Mechanism of Actions, H2 2016 81

Number of Products by Stage and Top 10 Mechanism of Actions, H2 2016 81

Number of Products by Routes of Administration, H2 2016 84

Number of Products by Stage and Routes of Administration, H2 2016 84

Number of Products by Top 10 Molecule Types, H2 2016 86

Number of Products by Stage and Top 10 Molecule Types, H2 2016 86

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To know more information on Purchase by Section, please send a mail to sales [@] kenresearch.com
 

Acceleron Pharma, Inc.

Anagenesis Biotechnology

Asklepios BioPharmaceutical, Inc.

BioMarin Pharmaceutical Inc.

Bristol-Myers Squibb Company

Capricor Therapeutics, Inc.

Catabasis Pharmaceuticals, Inc.

Daiichi Sankyo Company, Limited

Debiopharm International SA

Editas Medicine, Inc.

EryDel SPA

FibroGen, Inc.

Galapagos NV

Genethon

GTx, Inc.

GW Pharmaceuticals Plc

Idera Pharmaceuticals, Inc.

Italfarmaco S.p.A.

La Jolla Pharmaceutical Company

Lexicon Pharmaceuticals, Inc.

Marathon Pharmaceuticals, LLC

Marina Biotech, Inc.

Merck KGaA

Milo Biotechnology LLC

Mitochon Pharmaceuticals, Inc.

MyoTherix Inc.

NicOx S.A.

Nippon Shinyaku Co., Ltd.

Nobelpharma Co., Ltd.

Pfizer Inc.

Pluristem Therapeutics Inc.

Prothelia, Inc.

PTC Therapeutics, Inc.

RASRx, LLC

ReveraGen BioPharma, Inc.

Santhera Pharmaceuticals Holding AG

Sarepta Therapeutics, Inc.

Summit Therapeutics Plc

Taiho Pharmaceutical Co., Ltd.

WAVE Life Sciences 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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