シャルコー・マリー・トゥース病(Charcot-Marie-Tooth Disease)の治療薬パイプライン動向（2015年上半期版）...市場調査レポートについてご紹介

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 First–In-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