Pompe Disease - Pipeline Review, H2 2016

Published: Oct, 2016 | Pages: 70 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Pompe Disease - Pipeline Review, H2 2016, provides an overview of the Pompe Disease (Metabolic Disorders) pipeline landscape.

Pompe disease is an inherited disorder caused by defect in a gene called GAA. The GAA gene is responsible for the production of the GAA enzyme (acid alpha-glucosidase). This enzyme is needed to break down glycogen, a form of sugar stored in muscle cells. When too much glycogen builds up in the muscle cells, the cells become damaged and the muscles cannot function properly. Symptoms include respiratory failure, muscle pain, proximal muscle weakness, respiratory tract infections, headache and difficulty chewing or jaw muscle fatigue.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Pompe Disease - Pipeline Review, H2 2016, provides comprehensive information on the therapeutics under development for Pompe Disease (Metabolic Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.

The Pompe Disease (Metabolic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Pompe Disease and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase III, Phase II, Phase I, Preclinical and Discovery stages are 1, 1, 2, 8 and 2 respectively for Similarly, the Universities portfolio in Phase II and Phase I stages comprises 1 and 1 molecules, respectively for Pompe Disease.

Pompe Disease (Metabolic Disorders) pipeline guide 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. The guide 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. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

Scope

- The pipeline guide provides a snapshot of the global therapeutic landscape of Pompe Disease (Metabolic Disorders).
- The pipeline guide reviews pipeline therapeutics for Pompe Disease (Metabolic Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources.
- The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.
- The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.
- The pipeline guide reviews key companies involved in Pompe Disease (Metabolic Disorders) therapeutics and enlists all their major and minor projects.
- The pipeline guide evaluates Pompe Disease (Metabolic Disorders) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.
- The pipeline guide encapsulates all the dormant and discontinued pipeline projects.
- The pipeline guide reviews latest news related to pipeline therapeutics for Pompe Disease (Metabolic Disorders)

Reasons To Buy

- Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
- Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.
- Find and recognize significant and varied types of therapeutics under development for Pompe Disease (Metabolic Disorders).
- Classify potential new clients or partners in the target demographic.
- Develop tactical initiatives by understanding the focus areas of leading companies.
- Plan mergers and acquisitions meritoriously by identifying key players and it's most promising pipeline therapeutics.
- Formulate corrective measures for pipeline projects by understanding Pompe Disease (Metabolic Disorders) 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.
- Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.

 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
EpiVax, Inc. 21
Etubics Corporation 22
Genzyme Corporation 23
greenovation Biotech GmbH 24
Oxyrane Belgium NV 25
Pharming Group N.V. 26
RespireRx Pharmaceuticals Inc. 27
Sarepta Therapeutics, Inc. 28
Pompe Disease - Therapeutics Assessment 29
Assessment by Monotherapy Products 29
Assessment by Combination Products 30
Assessment by Target 31
Assessment by Mechanism of Action 33
Assessment by Route of Administration 35
Assessment by Molecule Type 37
Drug Profiles 39
Antisense Oligonucleotide to Activate Lysosomal Alpha-Glucosidase for Pompe Disease - Drug Profile 39
Product Description 39
Mechanism Of Action 39
R&D Progress 39
AT-982 - Drug Profile 40
Product Description 40
Mechanism Of Action 40
R&D Progress 40
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 48
Product Description 48
Mechanism Of Action 48
R&D Progress 48
Gene Therapy 1 to Activate Acid Alpha-Glucosidase for Pompe Disease - Drug Profile 50
Product Description 50
Mechanism Of Action 50
R&D Progress 50
Gene Therapy 2 to Activate Acid Alpha-Glucosidase for Pompe Disease - Drug Profile 51
Product Description 51
Mechanism Of Action 51
R&D Progress 51
Gene Therapy to Activate Acid Alpha-Glucosidase for Pompe Disease - Drug Profile 52
Product Description 52
Mechanism Of Action 52
R&D Progress 52
GZ-402666 - Drug Profile 53
Product Description 53
Mechanism Of Action 53
R&D Progress 53
MOSS-GAA - Drug Profile 55
Product Description 55
Mechanism Of Action 55
R&D Progress 55
OXY-2810 - Drug Profile 56
Product Description 56
Mechanism Of Action 56
R&D Progress 56
Recombinant Alpha Glucosidase Replacement for Pompe Disease - Drug Profile 57
Product Description 57
Mechanism Of Action 57
R&D Progress 57
Recombinant Alpha Glucosidase Replacement for Pompe Disease - Drug Profile 58
Product Description 58
Mechanism Of Action 58
R&D Progress 58
Recombinant Enzymes for Pompe, Fabry and Hunter Diseases - Drug Profile 59
Product Description 59
Mechanism Of Action 59
R&D Progress 59
Synthetic Peptides for Pompe disease - Drug Profile 60
Product Description 60
Mechanism Of Action 60
R&D Progress 60
VAL-1221 - Drug Profile 61
Product Description 61
Mechanism Of Action 61
R&D Progress 61
Pompe Disease - Dormant Projects 62
Pompe Disease - Discontinued Products 63
Pompe Disease - Product Development Milestones 64
Featured News & Press Releases 64
Mar 03, 2016: Sanofi Genzyme Presents Results from Phase 1/2 Study of Investigational Second-Generation Therapy for Pompe Disease 64
Sep 03, 2015: Valerion Therapeutics Announces Positive Results Of Non-Clinical Research Study at SSIEM Annual Symposium 65
May 11, 2015: Audentes Therapeutics To Present New Data On AT-002 at American Society of Gene and Cell Therapy Annual Meeting 66
May 04, 2015: Cortex Pharmaceuticals Announces Publication of Research Results on the Use of Ampakines CX1739 in the Treatment of Respiratory Disorders 66
May 04, 2015: Cortex Pharmaceuticals Announces Publication of Research Results on the Use of Ampakines CX717 in the Treatment of Respiratory Disorders 67
Appendix 69
Methodology 69
Coverage 69
Secondary Research 69
Primary Research 69
Expert Panel Validation 69
Contact Us 69
Disclaimer 70

List of Tables

Number of Products under Development for Pompe Disease, H2 2016 9
Number of Products under Development for Pompe Disease - 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 Late Stage Development, H2 2016 13
Comparative Analysis by Clinical Stage Development, H2 2016 14
Comparative Analysis by Early Stage Development, H2 2016 15
Products under Development by Companies, H2 2016 16
Products under Investigation by Universities/Institutes, H2 2016 17
Pompe Disease - Pipeline by Alexion Pharmaceuticals Inc, H2 2016 18
Pompe Disease - Pipeline by Amicus Therapeutics, Inc., H2 2016 19
Pompe Disease - Pipeline by Audentes Therapeutics, Inc., H2 2016 20
Pompe Disease - Pipeline by EpiVax, Inc., H2 2016 21
Pompe Disease - Pipeline by Etubics Corporation, H2 2016 22
Pompe Disease - Pipeline by Genzyme Corporation, H2 2016 23
Pompe Disease - Pipeline by greenovation Biotech GmbH, H2 2016 24
Pompe Disease - Pipeline by Oxyrane Belgium NV, H2 2016 25
Pompe Disease - Pipeline by Pharming Group N.V., H2 2016 26
Pompe Disease - Pipeline by RespireRx Pharmaceuticals Inc., H2 2016 27
Pompe Disease - Pipeline by Sarepta Therapeutics, Inc., H2 2016 28
Assessment by Monotherapy Products, H2 2016 29
Assessment by Combination Products, H2 2016 30
Number of Products by Stage and Target, H2 2016 32
Number of Products by Stage and Mechanism of Action, H2 2016 34
Number of Products by Stage and Route of Administration, H2 2016 36
Number of Products by Stage and Molecule Type, H2 2016 38
Pompe Disease - Dormant Projects, H2 2016 62
Pompe Disease - Discontinued Products, H2 2016 63