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Published: Jul, 2015 | Pages:
48 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)
Charcot-Marie-Tooth Disease - Pipeline Review, H2 2015 Summary Global Markets Direct's, 'Charcot-Marie-Tooth Disease - Pipeline Review, H2 2015', provides an overview of the Charcot-Marie-Tooth Disease's therapeutic pipeline. This report provides comprehensive information on the therapeutic development for Charcot-Marie-Tooth 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 Charcot-Marie-Tooth 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 Charcot-Marie-Tooth 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 Charcot-Marie-Tooth Disease and enlists all their major and minor projects - The report summarizes all the dormant and discontinued pipeline projects - A review of the Charcot-Marie-Tooth 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 Charcot-Marie-Tooth 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 Charcot-Marie-Tooth Disease - Plan mergers and acquisitions effectively by identifying key players of the most promising pipeline - Devise corrective measures for pipeline projects by understanding Charcot-Marie-Tooth 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 4 List of Figures 4 Introduction 5 Global Markets Direct Report Coverage 5 Charcot-Marie-Tooth Disease Overview 6 Therapeutics Development 7 Pipeline Products for Charcot-Marie-Tooth Disease - Overview 7 Pipeline Products for Charcot-Marie-Tooth Disease - Comparative Analysis 8 Charcot-Marie-Tooth Disease - Therapeutics under Development by Companies 9 Charcot-Marie-Tooth Disease - Therapeutics under Investigation by Universities/Institutes 10 Charcot-Marie-Tooth Disease - Pipeline Products Glance 11 Clinical Stage Products 11 Early Stage Products 12 Charcot-Marie-Tooth Disease - Products under Development by Companies 13 Charcot-Marie-Tooth Disease - Products under Investigation by Universities/Institutes 14 Charcot-Marie-Tooth Disease - Companies Involved in Therapeutics Development 15 Addex Therapeutics Ltd 15 Genzyme Corporation 16 PharmatrophiX, Inc. 17 Pharnext SAS 18 Charcot-Marie-Tooth Disease - Therapeutics Assessment 19 Assessment by Monotherapy Products 19 Assessment by Combination Products 20 Assessment by Target 21 Assessment by Mechanism of Action 23 Assessment by Route of Administration 25 Assessment by Molecule Type 26 Drug Profiles 28 (baclofen + naltrexone + sorbitol) - Drug Profile 28 Product Description 28 Mechanism of Action 28 R&D Progress 28 ADX-71441 - Drug Profile 30 Product Description 30 Mechanism of Action 30 R&D Progress 30 Small Molecule to Inhibit Rab7 GTPase for Oncology, Neuropathy, Immunology, Neurodegenerative Diseases, Lipidoses and Charcot-Marie-Tooth Disease Type II - Drug Profile 31 Product Description 31 Mechanism of Action 31 R&D Progress 31 Small Molecules to Agonize TrkB and TrkC for Alzheimer's, Parkinson's and Charcot-Marie-Tooth Disease - Drug Profile 32 Product Description 32 Mechanism of Action 32 R&D Progress 32 Small Molecules to Inhibit HDAC-6 for Immunology, Oncology and Genetic Disorders - Drug Profile 33 Product Description 33 Mechanism of Action 33 R&D Progress 33 Small Molecules to Inhibit PMP22 for Charcot-Marie-Tooth Type 1 A - Drug Profile 34 Product Description 34 Mechanism of Action 34 R&D Progress 34 Stem Cell Therapy to Activate PMP22 for Charcot-Marie-Tooth Disease Type 1A - Drug Profile 35 Product Description 35 Mechanism of Action 35 R&D Progress 35 Charcot-Marie-Tooth Disease - Recent Pipeline Updates 36 Charcot-Marie-Tooth Disease - Dormant Projects 39 Charcot-Marie-Tooth Disease - Product Development Milestones 40 Featured News & Press Releases 40 Jun 30, 2015: Pharnext's PXT-3003 Featured in Multiple Presentations at 2015 Peripheral Nerve Society Biennial Meeting 40 Dec 17, 2014: Pharnext Announces Pleotherapy Proof of Concept in Charcot-Marie-Tooth Disease Type 1A through Two Peer-Reviewed Publications 40 Oct 21, 2014: Addex And The Charcot-Marie-Tooth Association Enter Collaboration To Advance ADX71441 In Charcot-Marie-Tooth 1A disorder 41 Jun 23, 2014: The EMA and FDA Grant Orphan Drug Designation to Pharnext's PXT-3003 for the Treatment of Charcot-Marie-Tooth Disease Type 1A 43 Oct 03, 2013: Addex ADX71441 Dose Dependently Reduced PMP22 Expression Comparable to Baclofen in a Pre-Clinical Transgenic Model of Charcot-Marie-Tooth 1A Disease 43 Apr 24, 2013: Addex Receives Approval To Initiate Phase I First-In-man Clinical Study For ADX71441 44 Jan 22, 2013: Addex Receives Broad US Composition Of Matter Patent For ADX71441 45 Jan 07, 2013: Addex Therapeutics Announces Positive Data With ADX71441 In Pre-Clinical Transgenic Model Of Charcot-Marie-Tooth 1A Disease 45 Nov 22, 2011: Pharnext Completes Patient Recruitment For Phase II Study Of PXT3003 In Type 1A Charcot-Marie-Tooth Disease 46 Appendix 47 Methodology 47 Coverage 47 Secondary Research 47 Primary Research 47 Expert Panel Validation 47 Contact Us 47 Disclaimer 48
List of Tables
Number of Products under Development for Charcot-Marie-Tooth Disease, H2 2015 7 Number of Products under Development for Charcot-Marie-Tooth Disease - Comparative Analysis, H2 2015 8 Number of Products under Development by Companies, H2 2015 9 Number of Products under Investigation by Universities/Institutes, H2 2015 10 Comparative Analysis by Clinical Stage Development, H2 2015 11 Comparative Analysis by Early Stage Development, H2 2015 12 Products under Development by Companies, H2 2015 13 Products under Investigation by Universities/Institutes, H2 2015 14 Charcot-Marie-Tooth Disease - Pipeline by Addex Therapeutics Ltd, H2 2015 15 Charcot-Marie-Tooth Disease - Pipeline by Genzyme Corporation, H2 2015 16 Charcot-Marie-Tooth Disease - Pipeline by PharmatrophiX, Inc., H2 2015 17 Charcot-Marie-Tooth Disease - Pipeline by Pharnext SAS, H2 2015 18 Assessment by Monotherapy Products, H2 2015 19 Assessment by Combination Products, H2 2015 20 Number of Products by Stage and Target, H2 2015 22 Number of Products by Stage and Mechanism of Action, H2 2015 24 Number of Products by Stage and Route of Administration, H2 2015 25 Number of Products by Stage and Molecule Type, H2 2015 27 Charcot-Marie-Tooth Disease Therapeutics - Recent Pipeline Updates, H2 2015 36 Charcot-Marie-Tooth Disease - Dormant Projects, H2 2015 39
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