Global Markets Direct’s, ‘Myotonic Dystrophy – Pipeline Review, H2 2015′, provides an overview of the Myotonic Dystrophy’s therapeutic pipeline.

This report provides comprehensive information on the therapeutic development for Myotonic 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 Myotonic 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 Myotonic 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 Myotonic Dystrophy and enlists all their major and minor projects
• The report summarizes all the dormant and discontinued pipeline projects
• A review of the Myotonic 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 Myotonic 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 Myotonic Dystrophy
• Plan mergers and acquisitions effectively by identifying key players of the most promising pipeline
• 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

Table of Contents

Table of Contents 2
List of Tables 5
List of Figures 5
Introduction 6
Global Markets Direct Report Coverage 6
Myotonic Dystrophy Overview 7
Therapeutics Development 8
Pipeline Products for Myotonic Dystrophy - Overview 8
Pipeline Products for Myotonic Dystrophy - Comparative Analysis 9
Myotonic Dystrophy - Therapeutics under Development by Companies 10
Myotonic Dystrophy - Therapeutics under Investigation by Universities/Institutes 11
Myotonic Dystrophy - Pipeline Products Glance 12
Clinical Stage Products 12
Early Stage Products 13
Myotonic Dystrophy - Products under Development by Companies 14
Myotonic Dystrophy - Products under Investigation by Universities/Institutes 16
Myotonic Dystrophy - Companies Involved in Therapeutics Development 17
BioMarin Pharmaceutical Inc. 17
F. Hoffmann-La Roche Ltd. 18
Genzyme Corporation 19
Isis Pharmaceuticals, Inc. 20
Marina Biotech, Inc. 21
Valentia Biopharma S.L. 22
Myotonic Dystrophy - Therapeutics Assessment 23
Assessment by Monotherapy Products 23
Assessment by Target 24
Assessment by Mechanism of Action 26
Assessment by Route of Administration 28
Assessment by Molecule Type 30
Drug Profiles 32
Antisense Oligonucleotide for Spinal Muscular Atrophy and Myotonic Dystrophy - Drug Profile 32
Product Description 32
Mechanism of Action 32
R&D Progress 32
ISIS-DMPK2.5 - Drug Profile 33
Product Description 33
Mechanism of Action 33
R&D Progress 33
PRO-135 - Drug Profile 34
Product Description 34
Mechanism of Action 34
R&D Progress 34
RNAi Oligonucleotides for Myotonic Dystrophy - Drug Profile 35
Product Description 35
Mechanism of Action 35
R&D Progress 35
Small Molecule to Target CUG RNA for Myotonic Dystrophy 1 - Drug Profile 36
Product Description 36
Mechanism of Action 36
R&D Progress 36
Small Molecule to Target RNA for Myotonic Dystrophy - Drug Profile 37
Product Description 37
Mechanism of Action 37
R&D Progress 37
Small Molecules for Myotonic Dystrophy Type 1 - Drug Profile 38
Product Description 38
Mechanism of Action 38
R&D Progress 38
Small Molecules to Target RNA for Myotonic Dystrophy - Drug Profile 40
Product Description 40
Mechanism of Action 40
R&D Progress 40
SRT-149 - Drug Profile 41
Product Description 41
Mechanism of Action 41
R&D Progress 41
SRT-152 - Drug Profile 42
Product Description 42
Mechanism of Action 42
R&D Progress 42
VAL-0411 - Drug Profile 43
Product Description 43
Mechanism of Action 43
R&D Progress 43
VLT-001 - Drug Profile 44
Product Description 44
Mechanism of Action 44
R&D Progress 44
VLT-002 - Drug Profile 45
Product Description 45
Mechanism of Action 45
R&D Progress 45
VLT-003 - Drug Profile 46
Product Description 46
Mechanism of Action 46
R&D Progress 46
VLT-004 - Drug Profile 47
Product Description 47
Mechanism of Action 47
R&D Progress 47
VLT-005 - Drug Profile 48
Product Description 48
Mechanism of Action 48
R&D Progress 48
VLT-014 - Drug Profile 49
Product Description 49
Mechanism of Action 49
R&D Progress 49
VLT-015 - Drug Profile 50
Product Description 50
Mechanism of Action 50
R&D Progress 50
VLT-016 - Drug Profile 51
Product Description 51
Mechanism of Action 51
R&D Progress 51
VLT-017 - Drug Profile 52
Product Description 52
Mechanism of Action 52
R&D Progress 52
VLT-025 - Drug Profile 53
Product Description 53
Mechanism of Action 53
R&D Progress 53
Myotonic Dystrophy - Recent Pipeline Updates 54
Myotonic Dystrophy - Product Development Milestones 55
Featured News & Press Releases 55
Feb 25, 2014: VLT Biopharma gets the Orphan Drug designation for VLT015 55
Sep 19, 2013: AmorChem invests in the development of a therapeutic to treat myotonic dystrophy 55
Dec 20, 2012: Scripps Florida Scientists Create New Approach to Destroy Disease-Associated RNAs in Cells 56
Appendix 57
Methodology 57
Coverage 57
Secondary Research 57
Primary Research 57
Expert Panel Validation 57
Contact Us 57
Disclaimer 58

List of Tables

Number of Products under Development for Myotonic Dystrophy, H2 2015 8
Number of Products under Development for Myotonic Dystrophy - Comparative Analysis, H2 2015 9
Number of Products under Development by Companies, H2 2015 10
Number of Products under Investigation by Universities/Institutes, H2 2015 11
Comparative Analysis by Clinical Stage Development, H2 2015 12
Comparative Analysis by Early Stage Development, H2 2015 13
Products under Development by Companies, H2 2015 14
Products under Development by Companies, H2 2015 (Contd..1) 15
Products under Investigation by Universities/Institutes, H2 2015 16
Myotonic Dystrophy - Pipeline by BioMarin Pharmaceutical Inc., H2 2015 17
Myotonic Dystrophy - Pipeline by F. Hoffmann-La Roche Ltd., H2 2015 18
Myotonic Dystrophy - Pipeline by Genzyme Corporation, H2 2015 19
Myotonic Dystrophy - Pipeline by Isis Pharmaceuticals, Inc., H2 2015 20
Myotonic Dystrophy - Pipeline by Marina Biotech, Inc., H2 2015 21
Myotonic Dystrophy - Pipeline by Valentia Biopharma S.L., H2 2015 22
Assessment by Monotherapy Products, H2 2015 23
Number of Products by Stage and Target, H2 2015 25
Number of Products by Stage and Mechanism of Action, H2 2015 27
Number of Products by Stage and Route of Administration, H2 2015 29
Number of Products by Stage and Molecule Type, H2 2015 31
Myotonic Dystrophy Therapeutics - Recent Pipeline Updates, H2 2015 54

List of Figures

Number of Products under Development for Myotonic Dystrophy, H2 2015 8
Number of Products under Development for Myotonic Dystrophy - Comparative Analysis, H2 2015 9
Number of Products under Development by Companies, H2 2015 10
Comparative Analysis by Early Stage Products, H2 2015 13
Assessment by Monotherapy Products, H2 2015 23
Number of Products by Top 10 Targets, H2 2015 24
Number of Products by Stage and Top 10 Targets, H2 2015 24
Number of Products by Top 10 Mechanism of Actions, H2 2015 26
Number of Products by Stage and Top 10 Mechanism of Actions, H2 2015 26
Number of Products by Top 10 Routes of Administration, H2 2015 28
Number of Products by Stage and Top 10 Routes of Administration, H2 2015 28
Number of Products by Top 10 Molecule Types, H2 2015 30
Number of Products by Stage and Top 10 Molecule Types, H2 2015 30