Muscular Dystrophy – Pipeline Review, H2 2016

Global Markets Direct’s, ‘Muscular Dystrophy – Pipeline Review, H2 2016’, provides an overview of the Muscular Dystrophy pipeline landscape.

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

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

The report assesses 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 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 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 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

Companies mentioned

Acceleron Pharma, Inc.

AMO Pharma Limited

Asahi Kasei Pharma Corp.

aTyr Pharma, Inc.

Benitec Biopharma Limited

Bio Blast Pharma Ltd.

Biophytis SAS

Corcept Therapeutics Incorporated

Evotec AG

F. Hoffmann-La Roche Ltd.

Genethon

Ionis Pharmaceuticals, Inc.

Marina Biotech, Inc.

Medestea Research & Production S.p.A.

Novogen Limited

Pfizer Inc.

Prothelia, Inc.

SanBio, Inc.

Santhera Pharmaceuticals Holding AG

Sarepta Therapeutics, Inc.

Selecta Biosciences, Inc.

Strykagen Corporation

Takeda Pharmaceutical Company Limited

WAVE Life Sciences Ltd.

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

Muscular Dystrophy Overview 8

Therapeutics Development 9

Pipeline Products for Muscular Dystrophy - Overview 9

Pipeline Products for Muscular Dystrophy - Comparative Analysis 10

Muscular Dystrophy - Therapeutics under Development by Companies 11

Muscular Dystrophy - Therapeutics under Investigation by Universities/Institutes 13

Muscular Dystrophy - Pipeline Products Glance 14

Late Stage Products 14

Clinical Stage Products 15

Early Stage Products 16

Muscular Dystrophy - Products under Development by Companies 17

Muscular Dystrophy - Products under Investigation by Universities/Institutes 20

Muscular Dystrophy - Companies Involved in Therapeutics Development 21

Acceleron Pharma, Inc. 21

AMO Pharma Limited 22

Asahi Kasei Pharma Corp. 23

aTyr Pharma, Inc. 24

Benitec Biopharma Limited 25

Bio Blast Pharma Ltd. 26

Biophytis SAS 27

Corcept Therapeutics Incorporated 28

Evotec AG 29

F. Hoffmann-La Roche Ltd. 30

Genethon 31

Ionis Pharmaceuticals, Inc. 32

Marina Biotech, Inc. 33

Medestea Research & Production S.p.A. 34

Novogen Limited 35

Pfizer Inc. 36

Prothelia, Inc. 37

SanBio, Inc. 38

Santhera Pharmaceuticals Holding AG 39

Sarepta Therapeutics, Inc. 40

Selecta Biosciences, Inc. 41

Strykagen Corporation 42

Takeda Pharmaceutical Company Limited 43

WAVE Life Sciences Ltd. 44

Muscular Dystrophy - Therapeutics Assessment 45

Assessment by Monotherapy Products 45

Assessment by Target 46

Assessment by Mechanism of Action 48

Assessment by Route of Administration 50

Assessment by Molecule Type 52

Drug Profiles 54

ACE-083 - Drug Profile 54

Antisense Oligonucleotide to Inhibit DM1 Protein Kinase for Myotonic Dystrophy - Drug Profile 56

Antisense RNAi Oligonucleotides for Myotonic Dystrophy - Drug Profile 57

ATYR-1940 - Drug Profile 58

baliforsen - Drug Profile 61

BIO-103 - Drug Profile 62

domagrozumab - Drug Profile 63

Drugs for Merosin-Deficient Congenital Muscular Dystrophy Type 1A - Drug Profile 65

elcatonin - Drug Profile 66

Gene Therapy for Muscular Dystrophy and Liver Diseases - Drug Profile 67

Gene Therapy to Activate Dysferlin for Duchenne and Limb Girdle Muscular Dystrophies - Drug Profile 68

Gene Therapy to Activate Dysferlin for Dysferlinopathies - Drug Profile 69

Gene Therapy to Activate Dystrophin for Muscular Dystrophy - Drug Profile 70

IUCT-169 - Drug Profile 71

IUCT-290 - Drug Profile 72

IUCT-309 - Drug Profile 73

ketoprofen - Drug Profile 74

LR-08 - Drug Profile 75

MED-1101 - Drug Profile 76

mexiletine hydrochloride - Drug Profile 77

Oligonucleotide 1 to Target Dystrophia Myotonica Protein Kinase for Myotonic Dystrophy - Drug Profile 78

Oligonucleotides to Inhibit DM1 Protein Kinase for Myotonic Dystrophy - Drug Profile 79

omigapil - Drug Profile 80

Pabparna - Drug Profile 82

poloxamer - Drug Profile 83

PRT-01 - Drug Profile 85

Recombinant Protein to Activate Utrophin for Muscular Dystrophies - Drug Profile 87

RNAi Gene Therapy to Inhibit Myotilin for LGMD - Drug Profile 88

RP-33 - Drug Profile 89

SB-308 - Drug Profile 90

SIWA-318 - Drug Profile 91

Small Molecule to Inhibit DUX4 for Muscular Dystrophy - Drug Profile 92

Small Molecule to Target CUG RNA for Myotonic Dystrophy 1 - Drug Profile 93

Small Molecule to Target RNA for Myotonic Dystrophy - Drug Profile 94

Small Molecules for Dysferlinopathies - Drug Profile 95

Small Molecules for Facioscapulohumeral Muscular Dystrophy - Drug Profile 96

Small Molecules for Myotonic Dystrophy - Drug Profile 97

Small Molecules for Myotonic Dystrophy Type 1 - Drug Profile 98

Small Molecules to Activate SMCHD1 for Facioscapulohumeral Dystrophy - Drug Profile 99

Small Molecules to Antagonize Glucocorticoid Receptor II for Muscular Dystrophy - Drug Profile 100

Small Molecules to Inhibit MBNL1 for Myotonic Dystrophy Type I - Drug Profile 101

Small Molecules to Target RNA for Myotonic Dystrophy - Drug Profile 102

SRT-149 - Drug Profile 103

SRT-152 - Drug Profile 104

Stem Cell Therapy for Muscular Dystrophy - Drug Profile 105

Stryka-232 - Drug Profile 106

Stryka-234 - Drug Profile 107

Stryka-425 - Drug Profile 108

Stryka-533 - Drug Profile 109

Stryka-978 - Drug Profile 110

tideglusib - Drug Profile 111

trehalose - Drug Profile 112

TXA-127 - Drug Profile 116

VAL-0411 - Drug Profile 119

VAL-1205 - Drug Profile 120

Muscular Dystrophy - Dormant Projects 121

Muscular Dystrophy - Discontinued Products 123

Muscular Dystrophy - Product Development Milestones 124

Featured News & Press Releases 124

Appendix 139

Methodology 139

Coverage 139

Secondary Research 139

Primary Research 139

Expert Panel Validation 139

Contact Us 139

Disclaimer 140

List of Tables

List of Tables

Number of Products under Development for Muscular Dystrophy, H2 2016 13

Number of Products under Development for Muscular Dystrophy – Comparative Analysis, H2 2016 14

Number of Products under Development by Companies, H2 2016 15

Number of Products under Development by Companies, H2 2016 (Contd..1) 16

Number of Products under Investigation by Universities/Institutes, H2 2016 17

Comparative Analysis by Late Stage Development, H2 2016 18

Comparative Analysis by Clinical Stage Development, H2 2016 19

Comparative Analysis by Early Stage Development, H2 2016 20

Products under Development by Companies, H2 2016 21

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

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

Products under Investigation by Universities/Institutes, H2 2016 24

Muscular Dystrophy – Pipeline by Acceleron Pharma, Inc., H2 2016 25

Muscular Dystrophy – Pipeline by AMO Pharma Limited, H2 2016 26

Muscular Dystrophy – Pipeline by Asahi Kasei Pharma Corp., H2 2016 27

Muscular Dystrophy – Pipeline by aTyr Pharma, Inc., H2 2016 28

Muscular Dystrophy – Pipeline by Benitec Biopharma Limited, H2 2016 29

Muscular Dystrophy – Pipeline by Bio Blast Pharma Ltd., H2 2016 30

Muscular Dystrophy – Pipeline by Biophytis SAS, H2 2016 31

Muscular Dystrophy – Pipeline by Corcept Therapeutics Incorporated, H2 2016 32

Muscular Dystrophy – Pipeline by Evotec AG, H2 2016 33

Muscular Dystrophy – Pipeline by F. Hoffmann-La Roche Ltd., H2 2016 34

Muscular Dystrophy – Pipeline by Genethon, H2 2016 35

Muscular Dystrophy – Pipeline by Ionis Pharmaceuticals, Inc., H2 2016 36

Muscular Dystrophy – Pipeline by Marina Biotech, Inc., H2 2016 37

Muscular Dystrophy – Pipeline by Medestea Research & Production S.p.A., H2 2016 38

Muscular Dystrophy – Pipeline by Novogen Limited, H2 2016 39

Muscular Dystrophy – Pipeline by Pfizer Inc., H2 2016 40

Muscular Dystrophy – Pipeline by Prothelia, Inc., H2 2016 41

Muscular Dystrophy – Pipeline by SanBio, Inc., H2 2016 42

Muscular Dystrophy – Pipeline by Santhera Pharmaceuticals Holding AG, H2 2016 43

Muscular Dystrophy – Pipeline by Sarepta Therapeutics, Inc., H2 2016 44

Muscular Dystrophy – Pipeline by Selecta Biosciences, Inc., H2 2016 45

Muscular Dystrophy – Pipeline by Strykagen Corporation, H2 2016 46

Muscular Dystrophy – Pipeline by Takeda Pharmaceutical Company Limited, H2 2016 47

Muscular Dystrophy – Pipeline by WAVE Life Sciences Ltd., H2 2016 48

Assessment by Monotherapy Products, H2 2016 49

Number of Products by Stage and Target, H2 2016 51

Number of Products by Stage and Mechanism of Action, H2 2016 53

Number of Products by Stage and Route of Administration, H2 2016 55

Number of Products by Stage and Molecule Type, H2 2016 57

Muscular Dystrophy – Dormant Projects, H2 2016 125

Muscular Dystrophy – Dormant Projects (Contd..1), H2 2016 126

Muscular Dystrophy – Discontinued Products, H2 2016 127

List of Figures

List of Figures

Number of Products under Development for Muscular Dystrophy, H2 2016 13

Number of Products under Development for Muscular Dystrophy – Comparative Analysis, H2 2016 14

Number of Products under Development by Companies, H2 2016 15

Number of Products under Investigation by Universities/Institutes, H2 2016 17

Comparative Analysis by Clinical Stage Development, H2 2016 19

Comparative Analysis by Early Stage Products, H2 2016 20

Assessment by Monotherapy Products, H2 2016 49

Number of Products by Top 10 Targets, H2 2016 50

Number of Products by Stage and Top 10 Targets, H2 2016 50

Number of Products by Top 10 Mechanism of Actions, H2 2016 52

Number of Products by Stage and Top 10 Mechanism of Actions, H2 2016 52

Number of Products by Routes of Administration, H2 2016 54

Number of Products by Stage and Routes of Administration, H2 2016 54

Number of Products by Top 10 Molecule Types, H2 2016 56

Number of Products by Stage and Top 10 Molecule Types, H2 2016 56

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