Pompe Disease - Pipeline Review, H2 2012

Global Markets Direct, December 2012, Pages: 43

Pompe Disease – Pipeline Review, H2 2012

Summary

Global Markets Direct’s, 'Pompe Disease - Pipeline Review, H2 2012', provides an overview of the indication’s therapeutic pipeline. This report provides information on the therapeutic development for Pompe Disease, complete with latest updates, and special features on late-stage and discontinued projects. It also reviews key players involved in the therapeutic development for Pompe Disease. Pompe Disease - Pipeline Review, Half Year 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.

Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data for the indicated disease.

Scope

- A snapshot of the global therapeutic scenario for Pompe Disease.
- 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.
- Coverage of products based on various stages of development ranging from discovery till registration stages.
- A feature on pipeline projects on the basis of monotherapy and combined therapeutics.
- Coverage of the Pompe Disease pipeline on the basis of route of administration and molecule type.
- Key discontinued pipeline projects.
- Latest news and deals relating to the products.

Reasons to buy

- Identify and understand important and diverse types of therapeutics under development for Pompe Disease.
- Identify emerging players with potentially strong product portfolio and design effective counter-strategies to gain competitive advantage.
- Plan mergers and acquisitions effectively by identifying 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.

2
List of Tables 4
List of Figures 4
Introduction 5
REPORT COVERAGE 5
Pompe Disease Overview 6
Therapeutics Development 7
An Overview of Pipeline Products for Pompe Disease 7
Pompe Disease Therapeutics under Development by Companies 9
Pompe Disease Therapeutics under Investigation by Universities/Institutes 10
Mid Clinical Stage Products 11
Comparative Analysis 11
Pre-Clinical Stage Products 12
Comparative Analysis 12
Pompe Disease Therapeutics – Products under Development by Companies 13
Pompe Disease Therapeutics – Products under Investigation by Universities/Institutes 14
Companies Involved in Pompe Disease Therapeutics Development 15
Genzyme Corporation 15
Kaiser Permanente 16
BioMarin Pharmaceutical Inc. 17
Amicus Therapeutics, Inc. 18
Pompe Disease – Therapeutics Assessment 19
Assessment by Monotherapy Products 19
Assessment by Combination Products 20
Assessment by Route of Administration 21
Assessment by Molecule Type 23
Drug Profiles 25
duvoglustat hydrochloride - Drug Profile 25
Product Description 25
Mechanism of Action 25
R&D Progress 25
BMN-701 - Drug Profile 27
Product Description 27
Mechanism of Action 27
R&D Progress 27
rAAV1-CMV-GAA - Drug Profile 29
Product Description 29
Mechanism of Action 29
R&D Progress 29
Neo-rhGAA - Drug Profile 30
Product Description 30
Mechanism of Action 30
R&D Progress 30
AT-2220 + Alglucosidase Alfa (ERT) - Drug Profile 31
Product Description 31
Mechanism of Action 31
R&D Progress 31
BMN-103 - Drug Profile 32
Product Description 32
Mechanism of Action 32
R&D Progress 32
bortezomib - Drug Profile 33
Product Description 33
Mechanism of Action 33
R&D Progress 33
Pompe Disease Therapeutics – Drug Profile Updates 34
Pompe Disease Therapeutics - Dormant Products 36
Pompe Disease – Product Development Milestones 37
Featured News & Press Releases 37
Oct 11, 2012: Amicus Therapeutics Announces Positive Pompe Program Updates 37
Jun 26, 2012: Amicus Announces Positive Preliminary Results From Phase II Chaperone-Enzyme Replacement Therapy Co-Administration Study For Pompe Disease 39
Jan 21, 2010: Genzyme Announces Six-Month FDA Review For Lumizyme BLA 41
Appendix 42
Methodology 42
Coverage 42
Secondary Research 42
Primary Research 42
Expert Panel Validation 42
Contact Us 43
Disclaimer 43

List of Tables
Number of Products Under Development for Pompe Disease, H2 2012 7
Products under Development for Pompe Disease – Comparative Analysis, H2 2012 8
Number of Products under Development by Companies, H2 2012 9
Number of Products under Investigation by Universities/Institutes, H2 2012 10
Comparative Analysis by Mid Clinical Stage Development, H2 2012 11
Comparative Analysis by Pre-Clinical Stage Development, H2 2012 12
Products under Development by Companies, H2 2012 13
Products under Investigation by Universities/Institutes, H2 2012 14
Genzyme Corporation, H2 2012 15
Kaiser Permanente, H2 2012 16
BioMarin Pharmaceutical Inc., H2 2012 17
Amicus Therapeutics, Inc., H2 2012 18
Assessment by Monotherapy Products, H2 2012 19
Assessment by Combination Products, H2 2012 20
Assessment by Stage and Route of Administration, H2 2012 22
Assessment by Stage and Molecule Type, H2 2012 24
Pompe Disease Therapeutics – Drug Profile Updates 34
Pompe Disease Therapeutics – Dormant Products 36

List of Figures
Number of Products under Development for Pompe Disease, H2 2012 7
Products under Development for Pompe Disease – Comparative Analysis, H2 2012 8
Products under Development by Companies, H2 2012 9
Products under Investigation by Universities/Institutes, H2 2012 10
Mid Clinical Stage Products, H2 2012 11
Pre-Clinical Stage Products, H2 2012 12
Assessment by Monotherapy Products, H2 2012 19
Assessment by Combination Products, H2 2012 20
Assessment by Route of Administration, H2 2012 21
Assessment by Stage and Route of Administration, H2 2012 22
Assessment by Molecule Type, H2 2012 23
Assessment by Stage and Molecule Type, H2 2012 24

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