Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Pipeline Review, H2 2016

Published: Jul, 2016 | Pages: 63 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Pipeline Review, H2 2016

Summary

Global Markets Direct's, 'Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20)  - Pipeline Review, H2 2016', provides in depth analysis on Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20)  targeted pipeline therapeutics. 

The report provides comprehensive information on the Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) , targeted therapeutics, complete with analysis by indications, stage of development, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Additionally, the report provides an overview of key players involved in Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20)  targeted therapeutics development and features dormant 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, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Drug profiles featured in the report undergoes periodic review following a stringent set of processes to ensure that all the profiles are updated with the latest set of information. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

The report 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.

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

Scope

- The report provides a snapshot of the global therapeutic landscape for Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) 
- The report reviews Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20)  targeted therapeutics under development by companies and universities/research institutes based on information derived from company and industry-specific sources 
- The report covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages 
- The report features descriptive drug profiles for the pipeline products which includes, product description, descriptive MoA, R&D brief, licensing and collaboration details & other developmental activities 
- The report reviews key players involved in Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20)  targeted therapeutics and enlists all their major and minor projects 
- The report assesses Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20)  targeted therapeutics based on mechanism of action (MoA), route of administration (RoA) and molecule type 
- The report summarizes all the dormant and discontinued pipeline projects 
- The report reviews latest news and deals related to Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20)  targeted therapeutics

Reasons To Buy

- Gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies
- Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage 
- Identify and understand the targeted therapy areas and indications for Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) 
- Identify the use of drugs for target identification and drug repurposing
- Identify potential new clients or partners in the target demographic
- Develop strategic initiatives by understanding the focus areas of leading companies 
- Plan mergers and acquisitions effectively by identifying key players and it's most promising pipeline therapeutics
- Devise corrective measures for pipeline projects by understanding Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20)  development landscape 
- 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

 Table of Contents

Table of Contents 2
List of Tables 5
List of Figures 5
Introduction 6
Global Markets Direct Report Coverage 6
Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) Overview 7
Therapeutics Development 8
Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Products under Development by Stage of Development 8
Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Products under Development by Therapy Area 9
Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Products under Development by Indication 10
Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Pipeline Products Glance 11
Late Stage Products 11
Early Stage Products 12
Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Products under Development by Companies 13
Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Products under Development by Universities/Institutes 15
Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Therapeutics Assessment 17
Assessment by Monotherapy/Combination Products 17
Assessment by Mechanism of Action 18
Assessment by Route of Administration 20
Assessment by Molecule Type 22
Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Companies Involved in Therapeutics Development 24
Amicus Therapeutics, Inc. 24
Audentes Therapeutics, Inc. 25
BioMarin Pharmaceutical Inc. 26
Etubics Corporation 27
Genzyme Corporation 28
greenovation Biotech GmbH 29
Oxyrane Belgium NV 30
Pharming Group N.V. 31
Sarepta Therapeutics, Inc. 32
Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Drug Profiles 33
Antisense Oligonucleotide to Activate Lysosomal Alpha-Glucosidase for Pompe Disease - Drug Profile 33
Product Description 33
Mechanism Of Action 33
R&D Progress 33
AT-982 - Drug Profile 34
Product Description 34
Mechanism Of Action 34
R&D Progress 34
ATB-200 + miglustat - Drug Profile 35
Product Description 35
Mechanism Of Action 35
R&D Progress 35
Gene Therapy 1 to Activate Acid Alpha-Glucosidase for Pompe Disease - Drug Profile 37
Product Description 37
Mechanism Of Action 37
R&D Progress 37
Gene Therapy 2 to Activate Acid Alpha-Glucosidase for Pompe Disease - Drug Profile 38
Product Description 38
Mechanism Of Action 38
R&D Progress 38
Gene Therapy to Activate Acid Alpha-Glucosidase for Pompe Disease - Drug Profile 39
Product Description 39
Mechanism Of Action 39
R&D Progress 39
GZ-402666 - Drug Profile 40
Product Description 40
Mechanism Of Action 40
R&D Progress 40
MOSS-GAA - 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 Alpha Glucosidase Replacement 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
VAL-1221 - Drug Profile 47
Product Description 47
Mechanism Of Action 47
R&D Progress 47
Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Dormant Projects 48
Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Featured News & Press Releases 49
Mar 03, 2016: Sanofi Genzyme Presents Results from Phase 1/2 Study of Investigational Second-Generation Therapy for Pompe Disease 49
Mar 01, 2016: Amicus Therapeutics Highlights Preclinical Pompe Data at WORLDSymposium 2016 50
Feb 10, 2016: Amicus Therapeutics Announces Oral and Poster Presentations on Chaperone AT2221 at 12th Annual WORLDSymposium 2016 51
Jan 11, 2016: BioMarin Announces Interim Analysis of INSPIRE Clinical Trial in Pompe Disease at 34th Annual J.P. Morgan Annual Healthcare Conference 51
Jan 11, 2016: Amicus Therapeutics Provides Update On Novel ERT for Pompe Disease (ATB200 + Chaperone) 52
Dec 22, 2015: Amicus Therapeutics Commences Phase 1/2 Study of Novel ERT for Treatment of Pompe Disease 53
Sep 30, 2015: BioMarin to Present Research Results on Duchenne Muscular Dystrophy and Pompe Disease Programs at the 20th International Congress of the World Muscle Society 54
Sep 03, 2015: Valerion Therapeutics Announces Positive Results Of Non-Clinical Research Study at SSIEM Annual Symposium 55
May 11, 2015: Audentes Therapeutics To Present New Data On AT-002 at American Society of Gene and Cell Therapy Annual Meeting 56
Feb 10, 2015: Amicus Therapeutics Presents Preclinical Data on Next-Generation Pompe ERT at WORLD Symposium 2015 56
May 27, 2014: BioMarin Doses First Patient in Phase 3 INSPIRE Trial With BMN 701 for the Treatment of Pompe Disease 57
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 58
Jan 19, 2011: BioMarin Initiates Phase I/II Trial For BMN 701 For Treatment Of Pompe Disease 59
Aug 30, 2010: BioMarin Receives FDA Orphan Drug Designation For BMN-701 For Treatment Of Pompe Disease 60
Aug 18, 2010: BioMarin Pharmaceutical Acquires ZyStor Therapeutics, Furthers Development Of BMN-701 (ZC-701) For Pompe Disease 60
Appendix 62
Methodology 62
Coverage 62
Secondary Research 62
Primary Research 62
Expert Panel Validation 62
Contact Us 62
Disclaimer 63

List of Tables

Number of Products under Development for, H2 2016 8
Number of Products under Development by Therapy Area, H2 2016 9
Number of Products under Development by Indication, H2 2016 10
Comparative Analysis by Late Stage Development, H2 2016 11
Comparative Analysis by Early Stage Products, H2 2016 12
Number of Products under Development by Companies, H2 2016 13
Products under Development by Companies, H2 2016 14
Number of Products under Investigation by Universities/Institutes, H2 2016 15
Products under Investigation by Universities/Institutes, H2 2016 16
Assessment by Monotherapy/Combination Products, H2 2016 17
Number of Products by Stage and Mechanism of Action, H2 2016 19
Number of Products by Stage and Route of Administration, H2 2016 21
Number of Products by Stage and Molecule Type, H2 2016 23
Pipeline by Amicus Therapeutics, Inc., H2 2016 24
Pipeline by Audentes Therapeutics, Inc., H2 2016 25
Pipeline by BioMarin Pharmaceutical Inc., H2 2016 26
Pipeline by Etubics Corporation, H2 2016 27
Pipeline by Genzyme Corporation, H2 2016 28
Pipeline by greenovation Biotech GmbH, H2 2016 29
Pipeline by Oxyrane Belgium NV, H2 2016 30
Pipeline by Pharming Group N.V., H2 2016 31
Pipeline by Sarepta Therapeutics, Inc., H2 2016 32
Dormant Projects, H2 2016 48