Summary
Global Markets Direct’s, ‘Pompe Disease - Pipeline Review, H1 2016’, provides an overview of the Pompe Disease pipeline landscape.
The report provides comprehensive information on the therapeutics under development for Pompe Disease, 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 Pompe Disease 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 Pompe Disease
- The report reviews pipeline therapeutics for Pompe Disease 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 Pompe Disease therapeutics and enlists all their major and minor projects
- The report assesses Pompe Disease 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 Pompe Disease
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 Pompe Disease
- 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 Pompe Disease 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
Alexion Pharmaceuticals, Inc.
Amicus Therapeutics, Inc.
Audentes Therapeutics, Inc.
BioMarin Pharmaceutical Inc.
EpiVax, Inc.
Etubics Corporation
Genzyme Corporation
Greenovation Biotech GmbH
Oxyrane Belgium NV
Pharming Group N.V.
RespireRx Pharmaceuticals Inc.
Sarepta Therapeutics, Inc.
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Table of Contents
Table of Contents 2
List of Tables 5
List of Figures 6
Introduction 7
Global Markets Direct Report Coverage 7
Pompe Disease Overview 8
Therapeutics Development 9
Pipeline Products for Pompe Disease - Overview 9
Pipeline Products for Pompe Disease - Comparative Analysis 10
Pompe Disease - Therapeutics under Development by Companies 11
Pompe Disease - Therapeutics under Investigation by Universities/Institutes 12
Pompe Disease - Pipeline Products Glance 13
Late Stage Products 13
Clinical Stage Products 14
Early Stage Products 15
Pompe Disease - Products under Development by Companies 16
Pompe Disease - Products under Investigation by Universities/Institutes 17
Pompe Disease - Companies Involved in Therapeutics Development 18
Alexion Pharmaceuticals, Inc. 18
Amicus Therapeutics, Inc. 19
Audentes Therapeutics, Inc. 20
BioMarin Pharmaceutical Inc. 21
EpiVax, Inc. 22
Etubics Corporation 23
Genzyme Corporation 24
Greenovation Biotech GmbH 25
Oxyrane Belgium NV 26
Pharming Group N.V. 27
RespireRx Pharmaceuticals Inc. 28
Sarepta Therapeutics, Inc. 29
Pompe Disease - Therapeutics Assessment 30
Assessment by Monotherapy Products 30
Assessment by Combination Products 31
Assessment by Target 32
Assessment by Mechanism of Action 34
Assessment by Route of Administration 36
Assessment by Molecule Type 38
Drug Profiles 40
Antisense Oligonucleotide to Activate Lysosomal Alpha-Glucosidase for Pompe Disease - Drug Profile 40
Product Description 40
Mechanism of Action 40
R&D Progress 40
AT-982 - Drug Profile 41
Product Description 41
Mechanism of Action 41
R&D Progress 41
ATB-200 + miglustat - Drug Profile 42
Product Description 42
Mechanism of Action 42
R&D Progress 42
CX-1739 - Drug Profile 44
Product Description 44
Mechanism of Action 44
R&D Progress 44
CX-717 - Drug Profile 47
Product Description 47
Mechanism of Action 47
R&D Progress 47
Gene Therapy 1 to Activate Acid Alpha-Glucosidase for Pompe Disease - Drug Profile 49
Product Description 49
Mechanism of Action 49
R&D Progress 49
Gene Therapy 2 to Activate Acid Alpha-Glucosidase for Pompe Disease - Drug Profile 50
Product Description 50
Mechanism of Action 50
R&D Progress 50
Gene Therapy to Activate Acid Alpha-Glucosidase for Pompe Disease - Drug Profile 51
Product Description 51
Mechanism of Action 51
R&D Progress 51
GZ-402666 - Drug Profile 52
Product Description 52
Mechanism of Action 52
R&D Progress 52
MOSS-GAA - Drug Profile 53
Product Description 53
Mechanism of Action 53
R&D Progress 53
OXY-2810 - Drug Profile 54
Product Description 54
Mechanism of Action 54
R&D Progress 54
Recombinant Alpha Glucosidase Replacement for Pompe Disease - Drug Profile 55
Product Description 55
Mechanism of Action 55
R&D Progress 55
Recombinant Enzymes for Pompe, Fabry and Hunter Diseases - Drug Profile 56
Product Description 56
Mechanism of Action 56
R&D Progress 56
reveglucosidase alfa - Drug Profile 57
Product Description 57
Mechanism of Action 57
R&D Progress 57
Synthetic Peptides for Pompe disease - Drug Profile 58
Product Description 58
Mechanism of Action 58
R&D Progress 58
VAL-1221 - Drug Profile 59
Product Description 59
Mechanism of Action 59
R&D Progress 59
Pompe Disease - Recent Pipeline Updates 60
Pompe Disease - Dormant Projects 66
Pompe Disease - Product Development Milestones 67
Featured News & Press Releases 67
Jan 11, 2016: BioMarin Announces Interim Analysis of INSPIRE Clinical Trial in Pompe Disease at 34th Annual J.P. Morgan Annual Healthcare Conference 67
Sep 03, 2015: Valerion Therapeutics Announces Positive Results Of Non-Clinical Research Study at SSIEM Annual Symposium 68
May 11, 2015: Audentes Therapeutics To Present New Data On AT-002 at American Society of Gene and Cell Therapy Annual Meeting 68
May 04, 2015: Cortex Pharmaceuticals Announces Publication of Research Results on the Use of Ampakines CX1739 in the Treatment of Respiratory Disorders 70
May 04, 2015: Cortex Pharmaceuticals Announces Publication of Research Results on the Use of Ampakines CX717 in the Treatment of Respiratory Disorders 70
May 27, 2014: BioMarin Doses First Patient in Phase 3 INSPIRE Trial with BMN 701 for the Treatment of Pompe Disease 71
Mar 19, 2013: BioMarin Announces Results From Phase I/II Trial Of BMN-701 For Treatment Of Pompe Disease, Plans To Initiate Phase II/III Trial 72
Aug 18, 2010: BioMarin Pharmaceutical Acquires ZyStor Therapeutics, Furthers Development Of BMN-701 (ZC-701) For Pompe Disease 73
Appendix 75
Methodology 75
Coverage 75
Secondary Research 75
Primary Research 75
Expert Panel Validation 75
Contact Us 75
Disclaimer 76
List of Tables
Number of Products under Development for Pompe Disease, H1 2016 9
Number of Products under Development for Pompe Disease - Comparative Analysis, H1 2016 10
Number of Products under Development by Companies, H1 2016 11
Number of Products under Investigation by Universities/Institutes, H1 2016 12
Comparative Analysis by Late Stage Development, H1 2016 13
Comparative Analysis by Clinical Stage Development, H1 2016 14
Comparative Analysis by Early Stage Development, H1 2016 15
Products under Development by Companies, H1 2016 16
Products under Investigation by Universities/Institutes, H1 2016 17
Pompe Disease - Pipeline by Alexion Pharmaceuticals, Inc., H1 2016 18
Pompe Disease - Pipeline by Amicus Therapeutics, Inc., H1 2016 19
Pompe Disease - Pipeline by Audentes Therapeutics, Inc., H1 2016 20
Pompe Disease - Pipeline by BioMarin Pharmaceutical Inc., H1 2016 21
Pompe Disease - Pipeline by EpiVax, Inc., H1 2016 22
Pompe Disease - Pipeline by Etubics Corporation, H1 2016 23
Pompe Disease - Pipeline by Genzyme Corporation, H1 2016 24
Pompe Disease - Pipeline by greenovation Biotech GmbH, H1 2016 25
Pompe Disease - Pipeline by Oxyrane Belgium NV, H1 2016 26
Pompe Disease - Pipeline by Pharming Group N.V., H1 2016 27
Pompe Disease - Pipeline by RespireRx Pharmaceuticals Inc., H1 2016 28
Pompe Disease - Pipeline by Sarepta Therapeutics, Inc., H1 2016 29
Assessment by Monotherapy Products, H1 2016 30
Assessment by Combination Products, H1 2016 31
Number of Products by Stage and Target, H1 2016 33
Number of Products by Stage and Mechanism of Action, H1 2016 35
Number of Products by Stage and Route of Administration, H1 2016 37
Number of Products by Stage and Molecule Type, H1 2016 39
Pompe Disease Therapeutics - Recent Pipeline Updates, H1 2016 60
Pompe Disease - Dormant Projects, H1 2016 66
List of Figures
Number of Products under Development for Pompe Disease, H1 2016 9
Number of Products under Development for Pompe Disease - Comparative Analysis, H1 2016 10
Number of Products under Development by Companies, H1 2016 11
Comparative Analysis by Clinical Stage Development, H1 2016 14
Comparative Analysis by Early Stage Products, H1 2016 15
Assessment by Monotherapy Products, H1 2016 30
Number of Products by Targets, H1 2016 32
Number of Products by Stage and Targets, H1 2016 32
Number of Products by Mechanism of Actions, H1 2016 34
Number of Products by Stage and Mechanism of Actions, H1 2016 34
Number of Products by Routes of Administration, H1 2016 36
Number of Products by Stage and Routes of Administration, H1 2016 36
Number of Products by Molecule Types, H1 2016 38
Number of Products by Stage and Molecule Types, H1 2016 38
