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Pompe Disease - Pipeline Review, H2 2015 Is Released

Press Release   •   Jan 30, 2016 12:30 IST

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 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.

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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

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

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

Primary Research 80

Expert Panel Validation 80

Contact Us 80

Disclaimer 81

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