866-997-4948(US-Canada Toll Free)

Pompe Disease - Pipeline Review, H2 2013

Published By :

Global Markets Direct

Published Date : Dec 2013

Category :

Metabolic Disorders

No. of Pages : 64 Pages


Global Markets Directs, \'Pompe Disease Pipeline Review, H2 2013\', provides an overview of the indications 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 Directs 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 Directs 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.
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 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
Genzyme Corporation 18
BioMarin Pharmaceutical Inc. 19
Amicus Therapeutics, Inc. 20
EpiVax, Inc. 21
Valerion Therapeutics 22
Oxyrane Belgium NV 23
Audentes 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
Assessment by Therapeutic Class 35
Drug Profiles 37
BMN-701 - Drug Profile 37
Product Description 37
Mechanism of Action 37
R&D Progress 37
duvoglustat hydrochloride + alglucosidase alfa - Drug Profile 39
Product Description 39
Mechanism of Action 39
R&D Progress 39
rAAV1-CMV-GAA - Drug Profile 40
Product Description 40
Mechanism of Action 40
R&D Progress 40
GZ-402666 - Drug Profile 41
Product Description 41
Mechanism of Action 41
R&D Progress 41
alglucosidase alfa - Drug Profile 42
Product Description 42
Mechanism of Action 42
R&D Progress 42
BMN-103 - Drug Profile 43
Product Description 43
Mechanism of Action 43
R&D Progress 43
bortezomib - Drug Profile 44
Product Description 44
Mechanism of Action 44
R&D Progress 44
Tregitopes For Pompe disease - Drug Profile 45
Product Description 45
Mechanism of Action 45
R&D Progress 45
Pompe Disease Program - Drug Profile 46
Product Description 46
Mechanism of Action 46
R&D Progress 46
AT-002 - Drug Profile 47
Product Description 47
Mechanism of Action 47
R&D Progress 47
VAL-1221 - Drug Profile 48
Product Description 48
Mechanism of Action 48
R&D Progress 48
Pompe Disease - Recent Pipeline Updates 49
Pompe Disease - Dormant Projects 53
Pompe Disease - Product Development Milestones 54
Featured News & Press Releases 54
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 54
Feb 07, 2013: Amicus Therapeutics To Present Data On Its Pharmacological Chaperones At Lysosomal Disease Network WORLD Symposium 55
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 56
Oct 11, 2012: Amicus Therapeutics Announces Positive Pompe Program Updates 58
Jun 26, 2012: Amicus Announces Positive Preliminary Results From Phase II Chaperone-Enzyme Replacement Therapy Co-Administration Study For Pompe Disease 60
Jan 21, 2010: Genzyme Announces Six-Month FDA Review For Lumizyme BLA 62

Appendix 63
Methodology 63
Coverage 63
Secondary Research 63
Primary Research 63
Expert Panel Validation 63
Contact Us 64
Disclaimer 64

List of Table


Number of Products under Development for Pompe Disease, H2 2013 8
Number of Products under Development for Pompe Disease - Comparative Analysis, H2 2013 9
Number of Products under Development by Companies, H2 2013 11
Number of Products under Investigation by Universities/Institutes, H2 2013 12
Comparative Analysis by Late Stage Development, H2 2013 13
Comparative Analysis by Clinical Stage Development, H2 2013 14
Comparative Analysis by Early Stage Development, H2 2013 15
Products under Development by Companies, H2 2013 16
Products under Investigation by Universities/Institutes, H2 2013 17
Pompe Disease - Pipeline by Genzyme Corporation, H2 2013 18
Pompe Disease - Pipeline by BioMarin Pharmaceutical Inc., H2 2013 19
Pompe Disease - Pipeline by Amicus Therapeutics, Inc., H2 2013 20
Pompe Disease - Pipeline by EpiVax, Inc., H2 2013 21
Pompe Disease - Pipeline by Valerion Therapeutics, H2 2013 22
Pompe Disease - Pipeline by Oxyrane Belgium NV, H2 2013 23
Pompe Disease - Pipeline by Audentes Therapeutics, Inc., H2 2013 24
Assessment by Monotherapy Products, H2 2013 25
Assessment by Combination Products, H2 2013 26
Number of Products by Stage and Target, H2 2013 28
Number of Products by Stage and Mechanism of Action, H2 2013 30
Number of Products by Stage and Route of Administration, H2 2013 32
Number of Products by Stage and Molecule Type, H2 2013 34
Number of Products by Stage and Therapeutic Class, H2 2013 36
Pompe Disease Therapeutics - Recent Pipeline Updates, H2 2013 49
Pompe Disease - Dormant Projects, H2 2013 53

List of Chart


Number of Products under Development for Pompe Disease, H2 2013 8
Number of Products under Development for Pompe Disease - Comparative Analysis, H2 2013 9
Number of Products under Development by Companies, H2 2013 10
Number of Products under Investigation by Universities/Institutes, H2 2013 12
Comparative Analysis by Clinical Stage Development, H2 2013 14
Comparative Analysis by Early Stage Products, H2 2013 15
Assessment by Monotherapy Products, H2 2013 25
Number of Products by Top 10 Target, H2 2013 27
Number of Products by Stage and Top 10 Target, H2 2013 28
Number of Products by Top 10 Mechanism of Action, H2 2013 29
Number of Products by Stage and Top 10 Mechanism of Action, H2 2013 30
Number of Products by Top 10 Route of Administration, H2 2013 31
Number of Products by Stage and Top 10 Route of Administration, H2 2013 32
Number of Products by Top 10 Molecule Type, H2 2013 33
Number of Products by Stage and Top 10 Molecule Type, H2 2013 34
Number of Products by Top 10 Therapeutic Class, H2 2013 35
Number of Products by Stage and Top 10 Therapeutic Class, H2 2013 36

Make an enquiry before buying this Report

Please fill the enquiry form below.

  • Full Name *
  • Your Email *
  • Job Title *
  • Company *
  • Phone No. * (Pls. Affix Country Code)
  • Message
  • Security Code *