Pompe Disease - Pipeline Review, H2 2015 Is Released

Pompe Disease - Pipeline Review, H2 2015 Summary Global Markets Direct s, Pompe Disease - Pipeline
Review, H2 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 Directs 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 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 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 counterstrategies 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 outlicensing 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.

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Table of Contents

Introduction 8
Global Markets Direct Report Coverage 8
Pompe Disease Overview 9
Therapeutics Development 10
Pipeline Products for Pompe Disease - Overview 10
Pipeline Products for Pompe Disease - Comparative Analysis 11
Pompe Disease - Therapeutics under Development by Companies 12
Pompe Disease - Therapeutics under Investigation by Universities/Institutes 13
Pompe Disease - Pipeline Products Glance 14
Late Stage Products 14
Clinical Stage Products 15
Early Stage Products 16
Pompe Disease - Products under Development by Companies 17
Pompe Disease - Products under Investigation by Universities/Institutes 18
Pompe Disease - Companies Involved in Therapeutics Development 19
Alexion Pharmaceuticals, Inc. 19
Amicus Therapeutics, Inc. 20
Audentes Therapeutics, Inc. 21
BioMarin Pharmaceutical Inc. 22
Cortex Pharmaceuticals, Inc. 23
EpiVax, Inc. 24
Etubics Corporation 25
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Genzyme Corporation 26
greenovation Biotech GmbH 27
Oxyrane Belgium NV 28
Pharming Group N.V. 29
Sarepta Therapeutics, Inc. 30
Pompe Disease - Therapeutics Assessment 31
Assessment by Monotherapy Products 31
Assessment by Combination Products 32
Assessment by Target 33
Assessment by Mechanism of Action 35
Assessment by Route of Administration 37
Assessment by Molecule Type 39
Drug Profiles 41
Antisense Oligonucleotide to Activate Acid Alpha-Glucosidase for Pompe Disease - Drug Profile 41
Product Description 41
Mechanism of Action 41
R&D Progress 41
Antisense Oligonucleotide to Activate Lysosomal Alpha-Glucosidase for Pompe Disease - Drug Profile 42
Product Description 42
Mechanism of Action 42
R&D Progress 42
AT-002 - Drug Profile 43
Product Description 43
Mechanism of Action 43
R&D Progress 43
ATB200 + Chaperone - Drug Profile 44

Product Description 44
Mechanism of Action 44
R&D Progress 44
CX-1739 - Drug Profile 46
Product Description 46
Mechanism of Action 46
R&D Progress 46
CX-717 - Drug Profile 48
Product Description 48
Mechanism of Action 48
R&D Progress 48
duvoglustat hydrochloride + alglucosidase alfa - Drug Profile 50
Product Description 50
Mechanism of Action 50
R&D Progress 50
Gene Therapy 1 to Activate Acid Alpha-Glucosidase for Pompe Disease - Drug Profile 51
Product Description 51
Mechanism of Action 51
R&D Progress 51
Gene Therapy 2 to Activate Acid Alpha-Glucosidase for Pompe Disease - Drug Profile 52
Product Description 52
Mechanism of Action 52
R&D Progress 52
Gene Therapy to Activate Acid Alpha-Glucosidase for Pompe Disease - Drug Profile 53
Product Description 53
Mechanism of Action 53
R&D Progress 53
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GZ-402666 - Drug Profile 54

Product Description 54
Mechanism of Action 54
R&D Progress 54
MOSS-GAA - Drug Profile 55
Product Description 55
Mechanism of Action 55
R&D Progress 55
OXY-2810 - Drug Profile 56
Product Description 56
Mechanism of Action 56
R&D Progress 56
Recombinant Enzyme to Replace Alpha Glucosidase for Pompe Disease - Drug Profile 57
Product Description 57
Mechanism of Action 57
R&D Progress 57
Recombinant Enzymes for Pompe, Fabry and Hunter Diseases - Drug Profile 58
Product Description 58
Mechanism of Action 58
R&D Progress 58
reveglucosidase alfa - Drug Profile 59
Product Description 59
Mechanism of Action 59
R&D Progress 59
Synthetic Peptides for Pompe disease - Drug Profile 60
Product Description 60

Mechanism of Action 60
R&D Progress 60
VAL-1221 - Drug Profile 61
Product Description 61
Mechanism of Action 61
R&D Progress 61
Pompe Disease - Recent Pipeline Updates 62
Pompe Disease - Dormant Projects 67
Pompe Disease - Product Development Milestones 68
Featured News & Press Releases 68
Sep 03, 2015: Valerion Therapeutics Announces Positive Results Of Non-Clinical Research Study at SSIEM
Annual Symposium 68
May 11, 2015: Audentes Therapeutics To Present New Data On AT-002 at American Society of Gene and
Cell Therapy Annual Meeting 68
May 04, 2015: Cortex Pharmaceuticals Announces Publication of Research Results on the Use of
Ampakines CX1739 in the Treatment of Respiratory Disorders 70
Feb 10, 2015: Amicus Therapeutics Presents Preclinical Data on Next-Generation Pompe ERT at WORLD
Symposium 2015 70
May 27, 2014: BioMarin Doses First Patient in Phase 3 INSPIRE Trial with BMN 701 for the Treatment of
Pompe Disease 71
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 72
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 74
Oct 11, 2012: Amicus Therapeutics Announces Positive Pompe Program Updates 75
Jun 26, 2012: Amicus Announces Positive Preliminary Results From Phase II Chaperone-Enzyme
Replacement Therapy Co-Administration Study For Pompe Disease 77
Appendix 80
Methodology 80
Coverage 80
Secondary Research 80
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Primary Research 80
Expert Panel Validation 80
Contact Us 80
Disclaimer 81

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