Pompe Disease - Pipeline Review, H2 2014

Published: Dec, 2014 | Pages: 62 | Publisher: Global Markets Direct
Industry: Healthcare | Report Format: Electronic (PDF)

Pompe Disease - Pipeline Review, H2 2014

Summary

Global Markets Direct's, 'Pompe Disease - Pipeline Review, H2 2014', provides an overview of the Pompe Disease's therapeutic pipeline.

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

 Table of Contents
Table of Contents 2
List of Tables 4
List of Figures 5
Introduction 6
Global Markets Direct Report Coverage 6
Pompe Disease Overview 7
Therapeutics Development 8
Pipeline Products for Pompe Disease - Overview 8
Pipeline Products for Pompe Disease - Comparative Analysis 9
Pompe Disease - Therapeutics under Development by Companies 10
Pompe Disease - Therapeutics under Investigation by Universities/Institutes 11
Pompe Disease - Pipeline Products Glance 12
Late Stage Products 12
Clinical Stage Products 13
Early Stage Products 14
Pompe Disease - Products under Development by Companies 15
Pompe Disease - Products under Investigation by Universities/Institutes 16
Pompe Disease - Companies Involved in Therapeutics Development 17
Amicus Therapeutics, Inc. 17
Audentes Therapeutics, Inc. 18
BioMarin Pharmaceutical Inc. 19
EpiVax, Inc. 20
Genzyme Corporation 21
Oxyrane Belgium NV 22
Pharming Group N.V. 23
Sarepta Therapeutics, Inc. 24
Pompe Disease - Therapeutics Assessment 25
Assessment by Monotherapy Products 25
Assessment by Combination Products 26
Assessment by Target 27
Assessment by Mechanism of Action 29
Assessment by Route of Administration 31
Assessment by Molecule Type 33
Drug Profiles 35
(duvoglustat hydrochloride + ATB-200) - Drug Profile 35
Product Description 35
Mechanism of Action 35
R&D Progress 35
Antisense Oligonucleotide to Activate Lysosomal Alpha-Glucosidase for Pompe Disease - Drug Profile 36
Product Description 36
Mechanism of Action 36
R&D Progress 36
AT-002 - Drug Profile 37
Product Description 37
Mechanism of Action 37
R&D Progress 37
ATB-200 - Drug Profile 38
Product Description 38
Mechanism of Action 38
R&D Progress 38
duvoglustat hydrochloride + alglucosidase alfa - Drug Profile 39
Product Description 39
Mechanism of Action 39
R&D Progress 39
Gene Therapy 1 to Activate Acid Alpha-Glucosidase for Pompe Disease - Drug Profile 40
Product Description 40
Mechanism of Action 40
R&D Progress 40
Gene Therapy 2 to Activate Acid Alpha-Glucosidase for Pompe Disease - Drug Profile 41
Product Description 41
Mechanism of Action 41
R&D Progress 41
GZ-402666 - Drug Profile 42
Product Description 42
Mechanism of Action 42
R&D Progress 42
OXY-2810 - Drug Profile 43
Product Description 43
Mechanism of Action 43
R&D Progress 43
Recombinant Enzyme to Replace Alpha Glucosidase for Pompe Disease - Drug Profile 44
Product Description 44
Mechanism of Action 44
R&D Progress 44
reveglucosidase alfa - Drug Profile 45
Product Description 45
Mechanism of Action 45
R&D Progress 45
Synthetic Peptides for Pompe disease - Drug Profile 46
Product Description 46
Mechanism of Action 46
R&D Progress 46
VAL-1221 - Drug Profile 47
Product Description 47
Mechanism of Action 47
R&D Progress 47
Pompe Disease - Recent Pipeline Updates 48
Pompe Disease - Dormant Projects 51
Pompe Disease - Product Development Milestones 52
Featured News & Press Releases 52
May 27, 2014: BioMarin Doses First Patient in Phase 3 INSPIRE Trial With BMN 701 for the Treatment of Pompe Disease 52
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 53
Jan 04, 2013: Amicus Therapeutics Announces Positive Results From All Four Cohorts In Phase II Chaperone-enzyme Replacement Therapy Co-Administration Study For Pompe Disease 54
Oct 11, 2012: Amicus Therapeutics Announces Positive Pompe Program Updates 56
Jun 26, 2012: Amicus Announces Positive Preliminary Results From Phase II Chaperone-Enzyme Replacement Therapy Co-Administration Study For Pompe Disease 58
Appendix 61
Methodology 61
Coverage 61
Secondary Research 61
Primary Research 61
Expert Panel Validation 61
Contact Us 62
Disclaimer 62

List of Tables
Number of Products under Development for Pompe Disease, H2 2014 8
Number of Products under Development for Pompe Disease - Comparative Analysis, H2 2014 9
Number of Products under Development by Companies, H2 2014 10
Number of Products under Investigation by Universities/Institutes, H2 2014 11
Comparative Analysis by Late Stage Development, H2 2014 12
Comparative Analysis by Clinical Stage Development, H2 2014 13
Comparative Analysis by Early Stage Development, H2 2014 14
Products under Development by Companies, H2 2014 15
Products under Investigation by Universities/Institutes, H2 2014 16
Pompe Disease - Pipeline by Amicus Therapeutics, Inc., H2 2014 17
Pompe Disease - Pipeline by Audentes Therapeutics, Inc., H2 2014 18
Pompe Disease - Pipeline by BioMarin Pharmaceutical Inc., H2 2014 19
Pompe Disease - Pipeline by EpiVax, Inc., H2 2014 20
Pompe Disease - Pipeline by Genzyme Corporation, H2 2014 21
Pompe Disease - Pipeline by Oxyrane Belgium NV, H2 2014 22
Pompe Disease - Pipeline by Pharming Group N.V., H2 2014 23
Pompe Disease - Pipeline by Sarepta Therapeutics, Inc., H2 2014 24
Assessment by Monotherapy Products, H2 2014 25
Assessment by Combination Products, H2 2014 26
Number of Products by Stage and Target, H2 2014 28
Number of Products by Stage and Mechanism of Action, H2 2014 30
Number of Products by Stage and Route of Administration, H2 2014 32
Number of Products by Stage and Molecule Type, H2 2014 34
Pompe Disease Therapeutics - Recent Pipeline Updates, H2 2014 48
Pompe Disease - Dormant Projects, H2 2014 51