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Published: Dec, 2015 | Pages:
81 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)
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 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 6 List of Figures 7 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
List of Tables
Number of Products under Development for Pompe Disease, H2 2015 10 Number of Products under Development for Pompe Disease - Comparative Analysis, H2 2015 11 Number of Products under Development by Companies, H2 2015 12 Number of Products under Investigation by Universities/Institutes, H2 2015 13 Comparative Analysis by Late Stage Development, H2 2015 14 Comparative Analysis by Clinical Stage Development, H2 2015 15 Comparative Analysis by Early Stage Development, H2 2015 16 Products under Development by Companies, H2 2015 17 Products under Investigation by Universities/Institutes, H2 2015 18 Pompe Disease - Pipeline by Alexion Pharmaceuticals, Inc., H2 2015 19 Pompe Disease - Pipeline by Amicus Therapeutics, Inc., H2 2015 20 Pompe Disease - Pipeline by Audentes Therapeutics, Inc., H2 2015 21 Pompe Disease - Pipeline by BioMarin Pharmaceutical Inc., H2 2015 22 Pompe Disease - Pipeline by Cortex Pharmaceuticals, Inc., H2 2015 23 Pompe Disease - Pipeline by EpiVax, Inc., H2 2015 24 Pompe Disease - Pipeline by Etubics Corporation, H2 2015 25 Pompe Disease - Pipeline by Genzyme Corporation, H2 2015 26 Pompe Disease - Pipeline by greenovation Biotech GmbH, H2 2015 27 Pompe Disease - Pipeline by Oxyrane Belgium NV, H2 2015 28 Pompe Disease - Pipeline by Pharming Group N.V., H2 2015 29 Pompe Disease - Pipeline by Sarepta Therapeutics, Inc., H2 2015 30 Assessment by Monotherapy Products, H2 2015 31 Assessment by Combination Products, H2 2015 32 Number of Products by Stage and Target, H2 2015 34 Number of Products by Stage and Mechanism of Action, H2 2015 36 Number of Products by Stage and Route of Administration, H2 2015 38 Number of Products by Stage and Molecule Type, H2 2015 40 Pompe Disease Therapeutics - Recent Pipeline Updates, H2 2015 62 Pompe Disease - Dormant Projects, H2 2015 67
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