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Pompe Disease-Pipeline Review, H1 2015

Pompe Disease-Pipeline Review, H1 2015


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Executive Summary

Pompe Disease-Pipeline Review, H1 2015

Summary

Global Markets Direct's, 'Pompe Disease-Pipeline Review, H1 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 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, 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

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

Amicus Therapeutics, Inc. 18

Audentes Therapeutics, Inc. 19

BioMarin Pharmaceutical Inc. 20

Cortex Pharmaceuticals, Inc. 21

EpiVax, Inc. 22

Genzyme Corporation 23

Oxyrane Belgium NV 24

Pharming Group N.V. 25

Sarepta Therapeutics, Inc. 26

Synageva BioPharma Corp. 27

Pompe Disease-Therapeutics Assessment 28

Assessment by Monotherapy Products 28

Assessment by Combination Products 29

Assessment by Target 30

Assessment by Mechanism of Action 32

Assessment by Route of Administration 34

Assessment by Molecule Type 36

Drug Profiles 38

(duvoglustat hydrochloride + ATB-200)-Drug Profile 38

Product Description 38

Mechanism of Action 38

R&D Progress 38

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-002-Drug Profile 40

Product Description 40

Mechanism of Action 40

R&D Progress 40

ATB-200-Drug Profile 41

Product Description 41

Mechanism of Action 41

R&D Progress 41

CX-1739-Drug Profile 43

Product Description 43

Mechanism of Action 43

R&D Progress 43

CX-717-Drug Profile 45

Product Description 45

Mechanism of Action 45

R&D Progress 45

duvoglustat hydrochloride + alglucosidase alfa-Drug Profile 47

Product Description 47

Mechanism of Action 47

R&D Progress 47

Gene Therapy 1 to Activate Acid Alpha-Glucosidase for Pompe Disease-Drug Profile 48

Product Description 48

Mechanism of Action 48

R&D Progress 48

Gene Therapy 2 to Activate Acid Alpha-Glucosidase for Pompe Disease-Drug Profile 49

Product Description 49

Mechanism of Action 49

R&D Progress 49

GZ-402666-Drug Profile 50

Product Description 50

Mechanism of Action 50

R&D Progress 50

Next Generation biologics for Pompe, Fabry and Hunter-Drug Profile 51

Product Description 51

Mechanism of Action 51

R&D Progress 51

OXY-2810-Drug Profile 52

Product Description 52

Mechanism of Action 52

R&D Progress 52

Recombinant Enzyme to Replace Alpha Glucosidase for Pompe Disease-Drug Profile 53

Product Description 53

Mechanism of Action 53

R&D Progress 53

reveglucosidase alfa-Drug Profile 54

Product Description 54

Mechanism of Action 54

R&D Progress 54

Synthetic Peptides for Pompe disease-Drug Profile 55

Product Description 55

Mechanism of Action 55

R&D Progress 55

VAL-1221-Drug Profile 56

Product Description 56

Mechanism of Action 56

R&D Progress 56

Pompe Disease-Recent Pipeline Updates 57

Pompe Disease-Dormant Projects 62

Pompe Disease-Product Development Milestones 63

Featured News & Press Releases 63

May 11, 2015: Audentes Therapeutics To Present New Data On AT-002 at American Society of Gene and Cell Therapy Annual Meeting 63

May 04, 2015: Cortex Pharmaceuticals Announces Publication of Research Results on the Use of Ampakines CX1739 in the Treatment of Respiratory Disorders 64

May 04, 2015: Cortex Pharmaceuticals Announces Publication of Research Results on the Use of Ampakines CX717 in the Treatment of Respiratory Disorders 65

Feb 10, 2015: Amicus Therapeutics Presents Preclinical Data on Next-Generation Pompe ERT at WORLD Symposium 2015 65

May 27, 2014: BioMarin Doses First Patient in Phase 3 INSPIRE Trial With BMN 701 for the Treatment of Pompe Disease 66

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 67

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 68

Oct 11, 2012: Amicus Therapeutics Announces Positive Pompe Program Updates 70

Jun 26, 2012: Amicus Announces Positive Preliminary Results From Phase II Chaperone-Enzyme Replacement Therapy Co-Administration Study For Pompe Disease 72

Appendix 75

Methodology 75

Coverage 75

Secondary Research 75

Primary Research 75

Expert Panel Validation 75

Contact Us 75

Disclaimer 76

List of Tables

Number of Products under Development for Pompe Disease, H1 2015 9

Number of Products under Development for Pompe Disease-Comparative Analysis, H1 2015 10

Number of Products under Development by Companies, H1 2015 11

Number of Products under Investigation by Universities/Institutes, H1 2015 12

Comparative Analysis by Late Stage Development, H1 2015 13

Comparative Analysis by Clinical Stage Development, H1 2015 14

Comparative Analysis by Early Stage Development, H1 2015 15

Products under Development by Companies, H1 2015 16

Products under Investigation by Universities/Institutes, H1 2015 17

Pompe Disease-Pipeline by Amicus Therapeutics, Inc., H1 2015 18

Pompe Disease-Pipeline by Audentes Therapeutics, Inc., H1 2015 19

Pompe Disease-Pipeline by BioMarin Pharmaceutical Inc., H1 2015 20

Pompe Disease-Pipeline by Cortex Pharmaceuticals, Inc., H1 2015 21

Pompe Disease-Pipeline by EpiVax, Inc., H1 2015 22

Pompe Disease-Pipeline by Genzyme Corporation, H1 2015 23

Pompe Disease-Pipeline by Oxyrane Belgium NV, H1 2015 24

Pompe Disease-Pipeline by Pharming Group N.V., H1 2015 25

Pompe Disease-Pipeline by Sarepta Therapeutics, Inc., H1 2015 26

Pompe Disease-Pipeline by Synageva BioPharma Corp., H1 2015 27

Assessment by Monotherapy Products, H1 2015 28

Assessment by Combination Products, H1 2015 29

Number of Products by Stage and Target, H1 2015 31

Number of Products by Stage and Mechanism of Action, H1 2015 33

Number of Products by Stage and Route of Administration, H1 2015 35

Number of Products by Stage and Molecule Type, H1 2015 37

Pompe Disease Therapeutics-Recent Pipeline Updates, H1 2015 57

Pompe Disease-Dormant Projects, H1 2015 62

List of Figures

Number of Products under Development for Pompe Disease, H1 2015 9

Number of Products under Development for Pompe Disease-Comparative Analysis, H1 2015 10

Number of Products under Development by Companies, H1 2015 11

Comparative Analysis by Clinical Stage Development, H1 2015 14

Comparative Analysis by Early Stage Products, H1 2015 15

Assessment by Monotherapy Products, H1 2015 28

Assessment by Combination Products, H1 2015 29

Number of Products by Top 10 Targets, H1 2015 30

Number of Products by Stage and Top 10 Targets, H1 2015 31

Number of Products by Top 10 Mechanism of Actions, H1 2015 32

Number of Products by Stage and Top 10 Mechanism of Actions, H1 2015 33

Number of Products by Top 10 Routes of Administration, H1 2015 34

Number of Products by Stage and Top 10 Routes of Administration, H1 2015 35

Number of Products by Top 10 Molecule Types, H1 2015 36

Number of Products by Stage and Top 10 Molecule Types, H1 2015 37

Single User License:
Report can be used by individual purchaser only

Site License:
Report can be shared by unlimited users within one corporate location, e.g. a regional office

Corporate User License:
Report can be shared globally by unlimited users within the purchasing corporation e.g. all employees of a single company

To know more information on Purchase by Section, please send a mail to query [@] kenresearch.com
 

Amicus Therapeutics, Inc.

Audentes Therapeutics, Inc.

BioMarin Pharmaceutical Inc.

Cortex Pharmaceuticals, Inc.

EpiVax, Inc.

Genzyme Corporation

Oxyrane Belgium NV

Pharming Group N.V.

Sarepta Therapeutics, Inc.

Synageva BioPharma Corp.

Pompe Disease Therapeutic Products under Development, Key Players in Pompe Disease Therapeutics, Pompe Disease Pipeline Overview, Pompe Disease Pipeline, Pompe Disease Pipeline Assessment


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