Dystrophin (DMD) - Pipeline Review, H2 2016

Publisher Name :
Date: 31-Aug-2016
No. of pages: 81

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*Certain sections in the report may be removed or altered based on the availability and relevance of data.

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Global Markets Direct's, ‘Dystrophin (DMD) - Pipeline Review, H2 2016', provides in depth analysis on Dystrophin (DMD) targeted pipeline therapeutics.

The report provides comprehensive information on the Dystrophin (DMD), targeted therapeutics, complete with analysis by indications, stage of development, 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 Dystrophin (DMD) targeted therapeutics development 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.

Scope

  • The report provides a snapshot of the global therapeutic landscape for Dystrophin (DMD)

  • The report reviews Dystrophin (DMD) targeted therapeutics under development 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 in Dystrophin (DMD) targeted therapeutics and enlists all their major and minor projects

  • The report assesses Dystrophin (DMD) targeted therapeutics based on 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 and deals related to Dystrophin (DMD) targeted therapeutics

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 the targeted therapy areas and indications for Dystrophin (DMD)

  • Identify the use of drugs for target identification and drug repurposing

  • 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 Dystrophin (DMD) development landscape

  • 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

Dystrophin (DMD) - Pipeline Review, H2 2016

Table of Contents

Table of Contents 2
List of Tables 6
List of Figures 6
Introduction 7
Global Markets Direct Report Coverage 7
Dystrophin (DMD) Overview 8
Therapeutics Development 9
Dystrophin (DMD) - Products under Development by Stage of Development 9
Dystrophin (DMD) - Products under Development by Therapy Area 10
Dystrophin (DMD) - Products under Development by Indication 11
Dystrophin (DMD) - Pipeline Products Glance 12
Late Stage Products 12
Early Stage Products 13
Dystrophin (DMD) - Products under Development by Companies 14
Dystrophin (DMD) - Products under Development by Universities/Institutes 16
Dystrophin (DMD) - Therapeutics Assessment 18
Assessment by Monotherapy/Combination Products 18
Assessment by Mechanism of Action 19
Assessment by Route of Administration 20
Assessment by Molecule Type 22
Dystrophin (DMD) - Companies Involved in Therapeutics Development 24
Asklepios BioPharmaceutical, Inc. 24
BioMarin Pharmaceutical Inc. 25
Daiichi Sankyo Company, Limited 26
Genethon 27
Nippon Shinyaku Co., Ltd. 28
PTC Therapeutics, Inc. 29
Sarepta Therapeutics, Inc. 30
WAVE Life Sciences Ltd. 31
Dystrophin (DMD) - Drug Profiles 32
Antisense Oligonucleotide 1 to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile 32
Product Description 32
Mechanism Of Action 32
R&D Progress 32
Antisense Oligonucleotide to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile 33
Product Description 33
Mechanism Of Action 33
R&D Progress 33
Antisense Oligonucleotide to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile 34
Product Description 34
Mechanism Of Action 34
R&D Progress 34
Biostrophin - Drug Profile 35
Product Description 35
Mechanism Of Action 35
R&D Progress 35
BMBD-001 - Drug Profile 36
Product Description 36
Mechanism Of Action 36
R&D Progress 36
DS-5141 - Drug Profile 37
Product Description 37
Mechanism Of Action 37
R&D Progress 37
eteplirsen - Drug Profile 38
Product Description 38
Mechanism Of Action 38
R&D Progress 38
Gene Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile 49
Product Description 49
Mechanism Of Action 49
R&D Progress 49
Gene Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile 50
Product Description 50
Mechanism Of Action 50
R&D Progress 50
Gene Therapy to Activate Dystrophin for Muscular Dystrophy - Drug Profile 51
Product Description 51
Mechanism Of Action 51
R&D Progress 51
NS-065 - Drug Profile 52
Product Description 52
Mechanism Of Action 52
R&D Progress 52
Oligonucleotide to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile 53
Product Description 53
Mechanism Of Action 53
R&D Progress 53
Oligonucleotides 1 to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile 54
Product Description 54
Mechanism Of Action 54
R&D Progress 54
Oligonucleotides 2 to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile 55
Product Description 55
Mechanism Of Action 55
R&D Progress 55
Oligonucleotides 3 to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile 56
Product Description 56
Mechanism Of Action 56
R&D Progress 56
PRO-055 - Drug Profile 57
Product Description 57
Mechanism Of Action 57
R&D Progress 57
SGT-001 - Drug Profile 59
Product Description 59
Mechanism Of Action 59
R&D Progress 59
Small Molecule to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile 60
Product Description 60
Mechanism Of Action 60
R&D Progress 60
SRP-4008 - Drug Profile 61
Product Description 61
Mechanism Of Action 61
R&D Progress 61
SRP-4044 - Drug Profile 62
Product Description 62
Mechanism Of Action 62
R&D Progress 62
SRP-4045 - Drug Profile 63
Product Description 63
Mechanism Of Action 63
R&D Progress 63
SRP-4050 - Drug Profile 64
Product Description 64
Mechanism Of Action 64
R&D Progress 64
SRP-4052 - Drug Profile 65
Product Description 65
Mechanism Of Action 65
R&D Progress 65
SRP-4053 - Drug Profile 66
Product Description 66
Mechanism Of Action 66
R&D Progress 66
SRP-4055 - Drug Profile 67
Product Description 67
Mechanism Of Action 67
R&D Progress 67
Stem Cell Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile 68
Product Description 68
Mechanism Of Action 68
R&D Progress 68
Dystrophin (DMD) - Dormant Projects 69
Dystrophin (DMD) - Discontinued Products 70
Dystrophin (DMD) - Featured News & Press Releases 71
Jun 06, 2016: Sarepta Therapeutics Announces FDA Request For Dystrophin Data Prior To Making A Decision on Eteplirsen NDA 71
May 25, 2016: Sarepta Therapeutics Announces FDA Will Not Complete the Review of the Eteplirsen New Drug Application By The PDUFA Date 71
Apr 25, 2016: MDA Chief Medical and Scientific Officer Testifies at Landmark FDA Hearing for Duchenne Drug 71
Apr 25, 2016: Sarepta Issues Statement on Advisory Committee Outcome for Use of Eteplirsen in the Treatment of Duchenne Muscular Dystrophy 72
Mar 14, 2016: Sarepta Therapeutics Announces FDA Advisory Committee Meeting to Review Eteplirsen 73
Mar 14, 2016: Sarepta Therapeutics Announces FDA Advisory Committee Meeting to Review Eteplirsen as a Treatment for Duchenne Muscular Dystrophy 73
Feb 25, 2016: Daiichi Sankyo Announces Commencement of Clinical Trials in Japan for Duchenne Muscular Dystrophy Treatment 73
Feb 08, 2016: Sarepta Therapeutics Receives Notification of PDUFA Extension for Eteplirsen 74
Jan 20, 2016: FDA Postpones Advisory Committee Meeting to Review Eteplirsen Due to Severe Weather Storm in the Washington D.C. Area 74
Dec 18, 2015: Sarepta Therapeutics Announces FDA Advisory Committee Meeting to Review Eteplirsen as a Treatment for Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping 75
Nov 17, 2015: Sarepta Therapeutics Announces Publication of Positive Long-Term Safety and Efficacy Data for Eteplirsen in the Annals of Neurology 75
Oct 22, 2015: Gene Therapy Treats All Muscles in the Body in Muscular Dystrophy Dogs 76
Oct 14, 2015: Sarepta Therapeutics Announces Tentative FDA Advisory Committee Meeting to Review Eteplirsen as a Treatment for Duchenne Muscular Dystrophy 77
Oct 01, 2015: Sarepta Therapeutics Announces Additional Long-Term Efficacy and Safety Data from Pivotal Phase IIb Program of Eteplirsen for Treatment of Duchenne Muscular Dystrophy 77
Aug 25, 2015: Sarepta Therapeutics Announces FDA Has Filed Eteplirsen NDA for the Potential Treatment of Duchenne Muscular Dystrophy for Patients Amenable to Exon 51 Skipping 79
Appendix 80
Methodology 80
Coverage 80
Secondary Research 80
Primary Research 80
Expert Panel Validation 80
Contact Us 80
Disclaimer 81

List of Tables

Number of Products under Development for, H2 2016 9
Number of Products under Development by Therapy Area, H2 2016 10
Number of Products under Development by Indication, H2 2016 11
Comparative Analysis by Late Stage Development, H2 2016 12
Comparative Analysis by Early Stage Products, H2 2016 13
Number of Products under Development by Companies, H2 2016 14
Products under Development by Companies, H2 2016 15
Number of Products under Investigation by Universities/Institutes, H2 2016 16
Products under Investigation by Universities/Institutes, H2 2016 17
Assessment by Monotherapy/Combination Products, H2 2016 18
Number of Products by Stage and Mechanism of Action, H2 2016 19
Number of Products by Stage and Route of Administration, H2 2016 21
Number of Products by Stage and Molecule Type, H2 2016 23
Pipeline by Asklepios BioPharmaceutical, Inc., H2 2016 24
Pipeline by BioMarin Pharmaceutical Inc., H2 2016 25
Pipeline by Daiichi Sankyo Company, Limited, H2 2016 26
Pipeline by Genethon, H2 2016 27
Pipeline by Nippon Shinyaku Co., Ltd., H2 2016 28
Pipeline by PTC Therapeutics, Inc., H2 2016 29
Pipeline by Sarepta Therapeutics, Inc., H2 2016 30
Pipeline by WAVE Life Sciences Ltd., H2 2016 31
Dormant Projects, H2 2016 69
Discontinued Products, H2 2016 70

List of Figures

Number of Products under Development for, H2 2016 9
Number of Products under Development by Therapy Area, H2 2016 10
Number of Products under Development by Indication, H2 2016 11
Comparative Analysis by Late Stage Development, H2 2016 12
Comparative Analysis by Early Stage Products, H2 2016 13
Assessment by Monotherapy/Combination Products, H2 2016 18
Number of Products by Stage and Mechanism of Actions, H2 2016 19
Number of Products by Routes of Administration, H2 2016 20
Number of Products by Stage and Routes of Administration, H2 2016 20
Number of Products by Molecule Types, H2 2016 22
Number of Products by Stage and Molecule Type, H2 2016 22
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