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Charcot-Marie-Tooth Disease - Pipeline Review, H2 2014

Published: Dec-2014 | Format: PDF | Global Markets Direct | Number of pages: 48 | Code: MRS - 18556


Global Markets Directs, Charcot-Marie-Tooth Disease Pipeline Review, H2 2014, provides an overview of the Charcot-Marie-Tooth Diseases 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 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 Directs 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.

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 Content 

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
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 42
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 42
Apr 24, 2013: Addex Receives Approval To Initiate Phase I FirstIn-man Clinical Study For ADX71441 43
Jan 22, 2013: Addex Receives Broad US Composition Of Matter Patent For ADX71441 44
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 45

Appendix 47
Methodology 47
Coverage 47
Secondary Research 47
Primary Research 47
Expert Panel Validation 47
Contact Us 48
Disclaimer 48

List of Tables

Number of Products under Development for Charcot-Marie-Tooth Disease, H2 2014 7
Number of Products under Development for Charcot-Marie-Tooth Disease - Comparative Analysis, H2 2014 8
Number of Products under Development by Companies, H2 2014 9
Number of Products under Investigation by Universities/Institutes, H2 2014 10
Comparative Analysis by Clinical Stage Development, H2 2014 11
Comparative Analysis by Early Stage Development, H2 2014 12
Products under Development by Companies, H2 2014 13
Products under Investigation by Universities/Institutes, H2 2014 14
Charcot-Marie-Tooth Disease - Pipeline by Addex Therapeutics Ltd, H2 2014 15
Charcot-Marie-Tooth Disease - Pipeline by Genzyme Corporation, H2 2014 16
Charcot-Marie-Tooth Disease - Pipeline by PharmatrophiX, Inc., H2 2014 17
Charcot-Marie-Tooth Disease - Pipeline by Pharnext SAS, H2 2014 18
Assessment by Monotherapy Products, H2 2014 19
Assessment by Combination Products, H2 2014 20
Number of Products by Stage and Target, H2 2014 22
Number of Products by Stage and Mechanism of Action, H2 2014 24
Number of Products by Stage and Route of Administration, H2 2014 25
Number of Products by Stage and Molecule Type, H2 2014 27
Charcot-Marie-Tooth Disease Therapeutics - Recent Pipeline Updates, H2 2014 36
Charcot-Marie-Tooth Disease - Dormant Projects, H2 2014 39

List of Figures

Number of Products under Development for Charcot-Marie-Tooth Disease, H2 2014 7
Number of Products under Development for Charcot-Marie-Tooth Disease - Comparative Analysis, H2 2014 8
Number of Products under Development by Companies, H2 2014 9
Number of Products under Investigation by Universities/Institutes, H2 2014 10
Comparative Analysis by Clinical Stage Development, H2 2014 11
Comparative Analysis by Early Stage Products, H2 2014 12
Assessment by Monotherapy Products, H2 2014 19
Number of Products by Top 10 Targets, H2 2014 21
Number of Products by Stage and Top 10 Targets, H2 2014 21
Number of Products by Top 10 Mechanism of Actions, H2 2014 23
Number of Products by Stage and Top 10 Mechanism of Actions, H2 2014 23
Number of Products by Stage and Top 10 Routes of Administration, H2 2014 25
Number of Products by Top 10 Molecule Types, H2 2014 26
Number of Products by Stage and Top 10 Molecule Types, H2 2014 26

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