Thursday 22 January 2015

Charcot-Marie-Tooth Disease - Pipeline Review, Study And Forecast

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

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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.

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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

Introduction
Global Markets Direct Report Coverage
Charcot-Marie-Tooth Disease Overview
Therapeutics Development
Pipeline Products for Charcot-Marie-Tooth Disease - Overview
Pipeline Products for Charcot-Marie-Tooth Disease - Comparative Analysis
Charcot-Marie-Tooth Disease - Therapeutics under Development by Companies
Charcot-Marie-Tooth Disease - Therapeutics under Investigation by Universities/Institutes
Charcot-Marie-Tooth Disease - Pipeline Products Glance
Clinical Stage Products
Early Stage Products
Charcot-Marie-Tooth Disease - Products under Development by Companies
Charcot-Marie-Tooth Disease - Products under Investigation by Universities/Institutes
Charcot-Marie-Tooth Disease - Companies Involved in Therapeutics Development
Addex Therapeutics Ltd
Genzyme Corporation
PharmatrophiX, Inc.
Pharnext SAS
Charcot-Marie-Tooth Disease - Therapeutics Assessment
Assessment by Monotherapy Products
Assessment by Combination Products
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Drug Profiles
(baclofen + naltrexone + sorbitol) - Drug Profile
Product Description
Mechanism of Action
R&D Progress
ADX-71441 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Small Molecule to Inhibit Rab7 GTPase for Oncology, Neuropathy, Immunology, Neurodegenerative Diseases, Lipidoses and Charcot-Marie-Tooth Disease Type II - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Small Molecules to Agonize TrkB and TrkC for Alzheimer's, Parkinson's and Charcot-Marie-Tooth Disease - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Small Molecules to Inhibit HDAC-6 for Immunology, Oncology and Genetic Disorders - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Small Molecules to Inhibit PMP22 for Charcot-Marie-Tooth Type 1 A - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Stem Cell Therapy to Activate PMP22 for Charcot-Marie-Tooth Disease Type 1A - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Charcot-Marie-Tooth Disease - Recent Pipeline Updates
Charcot-Marie-Tooth Disease - Dormant Projects
Charcot-Marie-Tooth Disease - Product Development Milestones
Featured News & Press Releases
Dec 17, 2014: Pharnext Announces Pleotherapy Proof of Concept in Charcot-Marie-Tooth Disease Type 1A through Two Peer-Reviewed Publications
Oct 21, 2014: Addex And The Charcot-Marie-Tooth Association Enter Collaboration To Advance ADX71441 In Charcot-Marie-Tooth 1A disorder
Jun 23, 2014: The EMA and FDA Grant Orphan Drug Designation to Pharnex's PXT-3003 for the Treatment of Charcot-Marie-Tooth Disease Type 1A
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
Apr 24, 2013: Addex Receives Approval To Initiate Phase I FirstIn-man Clinical Study For ADX71441
Jan 22, 2013: Addex Receives Broad US Composition Of Matter Patent For ADX71441
Jan 07, 2013: Addex Therapeutics Announces Positive Data With ADX71441 In Pre-Clinical Transgenic Model Of Charcot-Marie-Tooth 1A Disease
Nov 22, 2011: Pharnext Completes Patient Recruitment For Phase II Study Of PXT3003 In Type 1A Charcot-Marie-Tooth Disease
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer

List of Tables
Number of Products under Development for Charcot-Marie-Tooth Disease, H2 2014
Number of Products under Development for Charcot-Marie-Tooth Disease - Comparative Analysis, H2 2014
Number of Products under Development by Companies, H2 2014
Number of Products under Investigation by Universities/Institutes, H2 2014
Comparative Analysis by Clinical Stage Development, H2 2014
Comparative Analysis by Early Stage Development, H2 2014
Products under Development by Companies, H2 2014
Products under Investigation by Universities/Institutes, H2 2014
Charcot-Marie-Tooth Disease - Pipeline by Addex Therapeutics Ltd, H2 2014
Charcot-Marie-Tooth Disease - Pipeline by Genzyme Corporation, H2 2014
Charcot-Marie-Tooth Disease - Pipeline by PharmatrophiX, Inc., H2 2014
Charcot-Marie-Tooth Disease - Pipeline by Pharnext SAS, H2 2014
Assessment by Monotherapy Products, H2 2014
Assessment by Combination Products, H2 2014
Number of Products by Stage and Target, H2 2014
Number of Products by Stage and Mechanism of Action, H2 2014
Number of Products by Stage and Route of Administration, H2 2014
Number of Products by Stage and Molecule Type, H2 2014
Charcot-Marie-Tooth Disease Therapeutics - Recent Pipeline Updates, H2 2014
Charcot-Marie-Tooth Disease - Dormant Projects, H2 2014

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