Pompe Disease - Pipeline Review, H1 2015

Published: May, 2015 | Pages: 76 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

Pompe Disease - Pipeline Review, H1 2015

Summary

Global Markets Direct's, 'Pompe Disease - Pipeline Review, H1 2015', 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

Reason 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 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
Amicus Therapeutics, Inc. 18
Audentes Therapeutics, Inc. 19
BioMarin Pharmaceutical Inc. 20
Cortex Pharmaceuticals, Inc. 21
EpiVax, Inc. 22
Genzyme Corporation 23
Oxyrane Belgium NV 24
Pharming Group N.V. 25
Sarepta Therapeutics, Inc. 26
Synageva BioPharma Corp. 27
Pompe Disease - Therapeutics Assessment 28
Assessment by Monotherapy Products 28
Assessment by Combination Products 29
Assessment by Target 30
Assessment by Mechanism of Action 32
Assessment by Route of Administration 34
Assessment by Molecule Type 36
Drug Profiles 38
(duvoglustat hydrochloride + ATB-200) - Drug Profile 38
Product Description 38
Mechanism of Action 38
R&D Progress 38
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-002 - Drug Profile 40
Product Description 40
Mechanism of Action 40
R&D Progress 40
ATB-200 - Drug Profile 41
Product Description 41
Mechanism of Action 41
R&D Progress 41
CX-1739 - Drug Profile 43
Product Description 43
Mechanism of Action 43
R&D Progress 43
CX-717 - Drug Profile 45
Product Description 45
Mechanism of Action 45
R&D Progress 45
duvoglustat hydrochloride + alglucosidase alfa - 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 48
Product Description 48
Mechanism of Action 48
R&D Progress 48
Gene Therapy 2 to Activate Acid Alpha-Glucosidase for Pompe Disease - Drug Profile 49
Product Description 49
Mechanism of Action 49
R&D Progress 49
GZ-402666 - Drug Profile 50
Product Description 50
Mechanism of Action 50
R&D Progress 50
Next Generation biologics for Pompe, Fabry and Hunter - Drug Profile 51
Product Description 51
Mechanism of Action 51
R&D Progress 51
OXY-2810 - Drug Profile 52
Product Description 52
Mechanism of Action 52
R&D Progress 52
Recombinant Enzyme to Replace Alpha Glucosidase for Pompe Disease - Drug Profile 53
Product Description 53
Mechanism of Action 53
R&D Progress 53
reveglucosidase alfa - Drug Profile 54
Product Description 54
Mechanism of Action 54
R&D Progress 54
Synthetic Peptides for Pompe disease - Drug Profile 55
Product Description 55
Mechanism of Action 55
R&D Progress 55
VAL-1221 - Drug Profile 56
Product Description 56
Mechanism of Action 56
R&D Progress 56
Pompe Disease - Recent Pipeline Updates 57
Pompe Disease - Dormant Projects 62
Pompe Disease - Product Development Milestones 63
Featured News & Press Releases 63
May 11, 2015: Audentes Therapeutics To Present New Data On AT-002 at American Society of Gene and Cell Therapy Annual Meeting 63
May 04, 2015: Cortex Pharmaceuticals Announces Publication of Research Results on the Use of Ampakines CX1739 in the Treatment of Respiratory Disorders 64
May 04, 2015: Cortex Pharmaceuticals Announces Publication of Research Results on the Use of Ampakines CX717 in the Treatment of Respiratory Disorders 65
Feb 10, 2015: Amicus Therapeutics Presents Preclinical Data on Next-Generation Pompe ERT at WORLD Symposium 2015 65
May 27, 2014: BioMarin Doses First Patient in Phase 3 INSPIRE Trial With BMN 701 for the Treatment of Pompe Disease 66
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 67
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 68
Oct 11, 2012: Amicus Therapeutics Announces Positive Pompe Program Updates 70
Jun 26, 2012: Amicus Announces Positive Preliminary Results From Phase II Chaperone-Enzyme Replacement Therapy Co-Administration Study For Pompe Disease 72
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 2015 9
Number of Products under Development for Pompe Disease - Comparative Analysis, H1 2015 10
Number of Products under Development by Companies, H1 2015 11
Number of Products under Investigation by Universities/Institutes, H1 2015 12
Comparative Analysis by Late Stage Development, H1 2015 13
Comparative Analysis by Clinical Stage Development, H1 2015 14
Comparative Analysis by Early Stage Development, H1 2015 15
Products under Development by Companies, H1 2015 16
Products under Investigation by Universities/Institutes, H1 2015 17
Pompe Disease - Pipeline by Amicus Therapeutics, Inc., H1 2015 18
Pompe Disease - Pipeline by Audentes Therapeutics, Inc., H1 2015 19
Pompe Disease - Pipeline by BioMarin Pharmaceutical Inc., H1 2015 20
Pompe Disease - Pipeline by Cortex Pharmaceuticals, Inc., H1 2015 21
Pompe Disease - Pipeline by EpiVax, Inc., H1 2015 22
Pompe Disease - Pipeline by Genzyme Corporation, H1 2015 23
Pompe Disease - Pipeline by Oxyrane Belgium NV, H1 2015 24
Pompe Disease - Pipeline by Pharming Group N.V., H1 2015 25
Pompe Disease - Pipeline by Sarepta Therapeutics, Inc., H1 2015 26
Pompe Disease - Pipeline by Synageva BioPharma Corp., H1 2015 27
Assessment by Monotherapy Products, H1 2015 28
Assessment by Combination Products, H1 2015 29
Number of Products by Stage and Target, H1 2015 31
Number of Products by Stage and Mechanism of Action, H1 2015 33
Number of Products by Stage and Route of Administration, H1 2015 35
Number of Products by Stage and Molecule Type, H1 2015 37
Pompe Disease Therapeutics - Recent Pipeline Updates, H1 2015 57
Pompe Disease - Dormant Projects, H1 2015 62