Pompe Disease – Pipeline Review, H2 2016

Global Markets Direct’s, ‘Pompe Disease – Pipeline Review, H2 2016’, provides an overview of the Pompe Disease pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Pompe Disease, complete with analysis by stage of development, drug target, 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 therapeutic development for Pompe Disease 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 of Pompe Disease

The report reviews pipeline therapeutics for Pompe Disease 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 Pompe Disease therapeutics and enlists all their major and minor projects

The report assesses Pompe Disease therapeutics based on drug target, 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 related to pipeline therapeutics for Pompe Disease

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 important and diverse types of therapeutics under development for Pompe Disease

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 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

Companies mentioned

Alexion Pharmaceuticals Inc

Amicus Therapeutics, Inc.

Audentes Therapeutics, Inc.

EpiVax, Inc.

Etubics Corporation

Genzyme Corporation

greenovation Biotech GmbH

Oxyrane Belgium NV

Pharming Group N.V.

RespireRx Pharmaceuticals Inc.

Sarepta Therapeutics, Inc.

Table of Contents

Table of Contents

Table of Contents 2

List of Tables 5

List of Figures 6

Introduction 7

Global Markets Direct Report Coverage 7

Pompe Disease Overview 8

Therapeutics Development 9

Pipeline Products for Pompe Disease - Overview 9

Pipeline Products for Pompe Disease - Comparative Analysis 10

Pompe Disease - Therapeutics under Development by Companies 11

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

Alexion Pharmaceuticals Inc 18

Amicus Therapeutics, Inc. 19

Audentes Therapeutics, Inc. 20

EpiVax, Inc. 21

Etubics Corporation 22

Genzyme Corporation 23

greenovation Biotech GmbH 24

Oxyrane Belgium NV 25

Pharming Group N.V. 26

RespireRx Pharmaceuticals Inc. 27

Sarepta Therapeutics, Inc. 28

Pompe Disease - Therapeutics Assessment 29

Assessment by Monotherapy Products 29

Assessment by Combination Products 30

Assessment by Target 31

Assessment by Mechanism of Action 33

Assessment by Route of Administration 35

Assessment by Molecule Type 37

Drug Profiles 39

Antisense Oligonucleotide to Activate Lysosomal Alpha-Glucosidase for Pompe Disease - Drug Profile 39

Product Description 39

Mechanism Of Action 39

R&D Progress 39

AT-982 - Drug Profile 40

Product Description 40

Mechanism Of Action 40

R&D Progress 40

ATB-200 + miglustat - Drug Profile 42

Product Description 42

Mechanism Of Action 42

R&D Progress 42

CX-1739 - Drug Profile 44

Product Description 44

Mechanism Of Action 44

R&D Progress 44

CX-717 - Drug Profile 48

Product Description 48

Mechanism Of Action 48

R&D Progress 48

Gene Therapy 1 to Activate Acid Alpha-Glucosidase for Pompe Disease - Drug Profile 50

Product Description 50

Mechanism Of Action 50

R&D Progress 50

Gene Therapy 2 to Activate Acid Alpha-Glucosidase for Pompe Disease - Drug Profile 51

Product Description 51

Mechanism Of Action 51

R&D Progress 51

Gene Therapy to Activate Acid Alpha-Glucosidase for Pompe Disease - Drug Profile 52

Product Description 52

Mechanism Of Action 52

R&D Progress 52

GZ-402666 - Drug Profile 53

Product Description 53

Mechanism Of Action 53

R&D Progress 53

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 Alpha Glucosidase Replacement for Pompe Disease - Drug Profile 57

Product Description 57

Mechanism Of Action 57

R&D Progress 57

Recombinant Alpha Glucosidase Replacement for Pompe Disease - Drug Profile 58

Product Description 58

Mechanism Of Action 58

R&D Progress 58

Recombinant Enzymes for Pompe, Fabry and Hunter Diseases - 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 - Dormant Projects 62

Pompe Disease - Discontinued Products 63

Pompe Disease - Product Development Milestones 64

Featured News & Press Releases 64

Mar 03, 2016: Sanofi Genzyme Presents Results from Phase 1/2 Study of Investigational Second-Generation Therapy for Pompe Disease 64

Sep 03, 2015: Valerion Therapeutics Announces Positive Results Of Non-Clinical Research Study at SSIEM Annual Symposium 65

May 11, 2015: Audentes Therapeutics To Present New Data On AT-002 at American Society of Gene and Cell Therapy Annual Meeting 66

May 04, 2015: Cortex Pharmaceuticals Announces Publication of Research Results on the Use of Ampakines CX1739 in the Treatment of Respiratory Disorders 66

May 04, 2015: Cortex Pharmaceuticals Announces Publication of Research Results on the Use of Ampakines CX717 in the Treatment of Respiratory Disorders 67

Appendix 69

Methodology 69

Coverage 69

Secondary Research 69

Primary Research 69

Expert Panel Validation 69

Contact Us 69

Disclaimer 70

List of Tables

List of Tables

Number of Products under Development for Pompe Disease, H2 2016 9

Number of Products under Development for Pompe Disease – Comparative Analysis, H2 2016 10

Number of Products under Development by Companies, H2 2016 11

Number of Products under Investigation by Universities/Institutes, H2 2016 12

Comparative Analysis by Late Stage Development, H2 2016 13

Comparative Analysis by Clinical Stage Development, H2 2016 14

Comparative Analysis by Early Stage Development, H2 2016 15

Products under Development by Companies, H2 2016 16

Products under Investigation by Universities/Institutes, H2 2016 17

Pompe Disease – Pipeline by Alexion Pharmaceuticals Inc, H2 2016 18

Pompe Disease – Pipeline by Amicus Therapeutics, Inc., H2 2016 19

Pompe Disease – Pipeline by Audentes Therapeutics, Inc., H2 2016 20

Pompe Disease – Pipeline by EpiVax, Inc., H2 2016 21

Pompe Disease – Pipeline by Etubics Corporation, H2 2016 22

Pompe Disease – Pipeline by Genzyme Corporation, H2 2016 23

Pompe Disease – Pipeline by greenovation Biotech GmbH, H2 2016 24

Pompe Disease – Pipeline by Oxyrane Belgium NV, H2 2016 25

Pompe Disease – Pipeline by Pharming Group N.V., H2 2016 26

Pompe Disease – Pipeline by RespireRx Pharmaceuticals Inc., H2 2016 27

Pompe Disease – Pipeline by Sarepta Therapeutics, Inc., H2 2016 28

Assessment by Monotherapy Products, H2 2016 29

Assessment by Combination Products, H2 2016 30

Number of Products by Stage and Target, H2 2016 32

Number of Products by Stage and Mechanism of Action, H2 2016 34

Number of Products by Stage and Route of Administration, H2 2016 36

Number of Products by Stage and Molecule Type, H2 2016 38

Pompe Disease – Dormant Projects, H2 2016 62

Pompe Disease – Discontinued Products, H2 2016 63

List of Figures

List of Figures

Number of Products under Development for Pompe Disease, H2 2016 9

Number of Products under Development for Pompe Disease – Comparative Analysis, H2 2016 10

Number of Products under Development by Companies, H2 2016 11

Number of Products under Investigation by Universities/Institutes, H2 2016 12

Comparative Analysis by Clinical Stage Development, H2 2016 14

Comparative Analysis by Early Stage Products, H2 2016 15

Assessment by Monotherapy Products, H2 2016 29

Number of Products by Targets, H2 2016 31

Number of Products by Stage and Targets, H2 2016 31

Number of Products by Mechanism of Actions, H2 2016 33

Number of Products by Stage and Mechanism of Actions, H2 2016 33

Number of Products by Routes of Administration, H2 2016 35

Number of Products by Stage and Routes of Administration, H2 2016 35

Number of Products by Molecule Types, H2 2016 37

Number of Products by Stage and Molecule Types, H2 2016 37

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