Myotonic Dystrophy – Pipeline Review, H2 2016

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

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

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

The report assesses Myotonic 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 Myotonic 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 Myotonic 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 Myotonic 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

AMO Pharma Limited

BioMarin Pharmaceutical Inc.

F. Hoffmann-La Roche Ltd.

Ionis Pharmaceuticals, Inc.

Marina Biotech, Inc.

Sarepta Therapeutics, Inc.

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

Myotonic Dystrophy Overview 8

Therapeutics Development 9

Pipeline Products for Myotonic Dystrophy - Overview 9

Pipeline Products for Myotonic Dystrophy - Comparative Analysis 10

Myotonic Dystrophy - Therapeutics under Development by Companies 11

Myotonic Dystrophy - Therapeutics under Investigation by Universities/Institutes 12

Myotonic Dystrophy - Pipeline Products Glance 13

Clinical Stage Products 13

Early Stage Products 14

Myotonic Dystrophy - Products under Development by Companies 15

Myotonic Dystrophy - Products under Investigation by Universities/Institutes 16

Myotonic Dystrophy - Companies Involved in Therapeutics Development 17

AMO Pharma Limited 17

BioMarin Pharmaceutical Inc. 18

F. Hoffmann-La Roche Ltd. 19

Ionis Pharmaceuticals, Inc. 20

Marina Biotech, Inc. 21

Sarepta Therapeutics, Inc. 22

WAVE Life Sciences Ltd. 23

Myotonic Dystrophy - Therapeutics Assessment 24

Assessment by Monotherapy Products 24

Assessment by Target 25

Assessment by Mechanism of Action 27

Assessment by Route of Administration 29

Assessment by Molecule Type 31

Drug Profiles 33

Antisense Oligonucleotide to Inhibit DM1 Protein Kinase for Myotonic Dystrophy - Drug Profile 33

Product Description 33

Mechanism Of Action 33

R&D Progress 33

Antisense RNAi Oligonucleotides for Myotonic Dystrophy - Drug Profile 34

Product Description 34

Mechanism Of Action 34

R&D Progress 34

IONISDMPK-2.5Rx - Drug Profile 35

Product Description 35

Mechanism Of Action 35

R&D Progress 35

IUCT-169 - Drug Profile 36

Product Description 36

Mechanism Of Action 36

R&D Progress 36

IUCT-290 - Drug Profile 37

Product Description 37

Mechanism Of Action 37

R&D Progress 37

IUCT-309 - Drug Profile 38

Product Description 38

Mechanism Of Action 38

R&D Progress 38

LR-08 - Drug Profile 39

Product Description 39

Mechanism Of Action 39

R&D Progress 39

mexiletine hydrochloride - Drug Profile 40

Product Description 40

Mechanism Of Action 40

R&D Progress 40

Oligonucleotide 1 to Target Dystrophia Myotonica Protein Kinase for Myotonic Dystrophy - Drug Profile 41

Product Description 41

Mechanism Of Action 41

R&D Progress 41

Oligonucleotides to Inhibit DM1 Protein Kinase for Myotonic Dystrophy - Drug Profile 42

Product Description 42

Mechanism Of Action 42

R&D Progress 42

PRO-135 - Drug Profile 43

Product Description 43

Mechanism Of Action 43

R&D Progress 43

RP-33 - Drug Profile 44

Product Description 44

Mechanism Of Action 44

R&D Progress 44

Small Molecule to Target CUG RNA for Myotonic Dystrophy 1 - Drug Profile 45

Product Description 45

Mechanism Of Action 45

R&D Progress 45

Small Molecule to Target RNA for Myotonic Dystrophy - Drug Profile 46

Product Description 46

Mechanism Of Action 46

R&D Progress 46

Small Molecules for Myotonic Dystrophy - Drug Profile 47

Product Description 47

Mechanism Of Action 47

R&D Progress 47

Small Molecules for Myotonic Dystrophy Type 1 - Drug Profile 48

Product Description 48

Mechanism Of Action 48

R&D Progress 48

Small Molecules to Inhibit MBNL1 for Myotonic Dystrophy Type I - Drug Profile 49

Product Description 49

Mechanism Of Action 49

R&D Progress 49

Small Molecules to Target RNA for Myotonic Dystrophy - Drug Profile 50

Product Description 50

Mechanism Of Action 50

R&D Progress 50

SRT-149 - Drug Profile 51

Product Description 51

Mechanism Of Action 51

R&D Progress 51

SRT-152 - Drug Profile 52

Product Description 52

Mechanism Of Action 52

R&D Progress 52

tideglusib - Drug Profile 53

Product Description 53

Mechanism Of Action 53

R&D Progress 53

VAL-0411 - Drug Profile 54

Product Description 54

Mechanism Of Action 54

R&D Progress 54

Myotonic Dystrophy - Dormant Projects 55

Myotonic Dystrophy - Discontinued Products 56

Myotonic Dystrophy - Product Development Milestones 57

Featured News & Press Releases 57

Sep 19, 2013: AmorChem invests in the development of a therapeutic to treat myotonic dystrophy 57

Dec 20, 2012: Scripps Florida Scientists Create New Approach to Destroy Disease-Associated RNAs in Cells 57

Appendix 59

Methodology 59

Coverage 59

Secondary Research 59

Primary Research 59

Expert Panel Validation 59

Contact Us 59

Disclaimer 60

List of Tables

List of Tables

Number of Products under Development for Myotonic Dystrophy, H2 2016 9

Number of Products under Development for Myotonic Dystrophy – 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 13

Comparative Analysis by Early Stage Development, H2 2016 14

Products under Development by Companies, H2 2016 15

Products under Investigation by Universities/Institutes, H2 2016 16

Myotonic Dystrophy – Pipeline by AMO Pharma Limited, H2 2016 17

Myotonic Dystrophy – Pipeline by BioMarin Pharmaceutical Inc., H2 2016 18

Myotonic Dystrophy – Pipeline by F. Hoffmann-La Roche Ltd., H2 2016 19

Myotonic Dystrophy – Pipeline by Ionis Pharmaceuticals, Inc., H2 2016 20

Myotonic Dystrophy – Pipeline by Marina Biotech, Inc., H2 2016 21

Myotonic Dystrophy – Pipeline by Sarepta Therapeutics, Inc., H2 2016 22

Myotonic Dystrophy – Pipeline by WAVE Life Sciences Ltd., H2 2016 23

Assessment by Monotherapy Products, H2 2016 24

Number of Products by Stage and Target, H2 2016 26

Number of Products by Stage and Mechanism of Action, H2 2016 28

Number of Products by Stage and Route of Administration, H2 2016 30

Number of Products by Stage and Molecule Type, H2 2016 32

Myotonic Dystrophy – Dormant Projects, H2 2016 55

Myotonic Dystrophy – Discontinued Products, H2 2016 56

List of Figures

List of Figures

Number of Products under Development for Myotonic Dystrophy, H2 2016 9

Number of Products under Development for Myotonic Dystrophy – 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 13

Comparative Analysis by Early Stage Products, H2 2016 14

Assessment by Monotherapy Products, H2 2016 24

Number of Products by Targets, H2 2016 25

Number of Products by Stage and Targets, H2 2016 25

Number of Products by Mechanism of Actions, H2 2016 27

Number of Products by Stage and Mechanism of Actions, H2 2016 27

Number of Products by Routes of Administration, H2 2016 29

Number of Products by Stage and Routes of Administration, H2 2016 29

Number of Products by Molecule Types, H2 2016 31

Number of Products by Stage and Molecule Types, H2 2016 31

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