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Charcot-Marie-Tooth Disease-Pipeline Review, H1 2015

Charcot-Marie-Tooth Disease-Pipeline Review, H1 2015


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Executive Summary

Charcot-Marie-Tooth Disease-Pipeline Review, H1 2015

Summary

Global Markets Direct's, 'Charcot-Marie-Tooth Disease-Pipeline Review, H1 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

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, H1 2015 7

Number of Products under Development for Charcot-Marie-Tooth Disease-Comparative Analysis, H1 2015 8

Number of Products under Development by Companies, H1 2015 9

Number of Products under Investigation by Universities/Institutes, H1 2015 10

Comparative Analysis by Clinical Stage Development, H1 2015 11

Comparative Analysis by Early Stage Development, H1 2015 12

Products under Development by Companies, H1 2015 13

Products under Investigation by Universities/Institutes, H1 2015 14

Charcot-Marie-Tooth Disease-Pipeline by Addex Therapeutics Ltd, H1 2015 15

Charcot-Marie-Tooth Disease-Pipeline by Genzyme Corporation, H1 2015 16

Charcot-Marie-Tooth Disease-Pipeline by PharmatrophiX, Inc., H1 2015 17

Charcot-Marie-Tooth Disease-Pipeline by Pharnext SAS, H1 2015 18

Assessment by Monotherapy Products, H1 2015 19

Assessment by Combination Products, H1 2015 20

Number of Products by Stage and Target, H1 2015 22

Number of Products by Stage and Mechanism of Action, H1 2015 24

Number of Products by Stage and Route of Administration, H1 2015 25

Number of Products by Stage and Molecule Type, H1 2015 27

Charcot-Marie-Tooth Disease Therapeutics-Recent Pipeline Updates, H1 2015 36

Charcot-Marie-Tooth Disease-Dormant Projects, H1 2015 39

List of Figures

Number of Products under Development for Charcot-Marie-Tooth Disease, H1 2015 7

Number of Products under Development for Charcot-Marie-Tooth Disease-Comparative Analysis, H1 2015 8

Number of Products under Development by Companies, H1 2015 9

Number of Products under Investigation by Universities/Institutes, H1 2015 10

Comparative Analysis by Clinical Stage Development, H1 2015 11

Comparative Analysis by Early Stage Products, H1 2015 12

Assessment by Monotherapy Products, H1 2015 19

Number of Products by Top 10 Targets, H1 2015 21

Number of Products by Stage and Top 10 Targets, H1 2015 22

Number of Products by Top 10 Mechanism of Actions, H1 2015 23

Number of Products by Stage and Top 10 Mechanism of Actions, H1 2015 24

Number of Products by Stage and Top 10 Routes of Administration, H1 2015 25

Number of Products by Top 10 Molecule Types, H1 2015 26

Number of Products by Stage and Top 10 Molecule Types, H1 2015 27

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Addex Therapeutics Ltd

Genzyme Corporation

PharmatrophiX, Inc.

Pharnext SAS

Charcot-Marie-Tooth Disease Therapeutic Products under Development, Key Players in Charcot-Marie-Tooth Disease Therapeutics, Charcot-Marie-Tooth Disease Pipeline Overview, Charcot-Marie-Tooth Disease Pipeline, Charcot-Marie-Tooth Disease Pipeline Assessment


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