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Charcot-Marie-Tooth Disease - Pipeline Review, H1 2016

  • February 2016
  • -
  • Global Markets Direct
  • -
  • 56 pages

Charcot-Marie-Tooth Disease - Pipeline Review, H1 2016

Summary

Global Markets Direct’s, ‘Charcot-Marie-Tooth Disease - Pipeline Review, H1 2016’, provides an overview of the Charcot-Marie-Tooth Disease pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Charcot-Marie-Tooth Disease, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Charcot-Marie-Tooth Disease and features dormant 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, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Drug profiles featured in the report undergoes periodic review following a stringent set of processes to ensure that all the profiles are updated with the latest set of information. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

The report helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage.

Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data.


Scope

- The report provides a snapshot of the global therapeutic landscape of Charcot-Marie-Tooth Disease
- The report reviews pipeline therapeutics for Charcot-Marie-Tooth Disease by companies and universities/research institutes based on information derived from company and industry-specific sources
- The report covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages
- The report features descriptive drug profiles for the pipeline products which includes, product description, descriptive MoA, R&D brief, licensing and collaboration details & other developmental activities
- The report reviews key players involved Charcot-Marie-Tooth Disease therapeutics and enlists all their major and minor projects
- The report assesses Charcot-Marie-Tooth Disease therapeutics based on drug target, mechanism of action (MoA), route of administration (RoA) and molecule type
- The report summarizes all the dormant and discontinued pipeline projects
- The report reviews latest news related to pipeline therapeutics for Charcot-Marie-Tooth Disease

Reasons to buy

- Gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies
- Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage
- Identify and understand important and diverse types of therapeutics under development for Charcot-Marie-Tooth Disease
- Identify potential new clients or partners in the target demographic
- Develop strategic initiatives by understanding the focus areas of leading companies
- Plan mergers and acquisitions effectively by identifying key players and it’s most promising pipeline therapeutics
- 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

Charcot-Marie-Tooth Disease - Pipeline Review, H1 2016
Table of Contents
Table of Contents 2
List of Tables 5
List of Figures 5
Introduction 6
Global Markets Direct Report Coverage 6
Charcot-Marie-Tooth Disease Overview 7
Therapeutics Development 8
Pipeline Products for Charcot-Marie-Tooth Disease - Overview 8
Pipeline Products for Charcot-Marie-Tooth Disease - Comparative Analysis 9
Charcot-Marie-Tooth Disease - Therapeutics under Development by Companies 10
Charcot-Marie-Tooth Disease - Therapeutics under Investigation by Universities/Institutes 11
Charcot-Marie-Tooth Disease - Pipeline Products Glance 12
Late Stage Products 12
Early Stage Products 13
Charcot-Marie-Tooth Disease - Products under Development by Companies 14
Charcot-Marie-Tooth Disease - Products under Investigation by Universities/Institutes 15
Charcot-Marie-Tooth Disease - Companies Involved in Therapeutics Development 16
Acetylon Pharmaceuticals, Inc. 16
Addex Therapeutics Ltd 17
Affectis Pharmaceuticals AG 18
Genzyme Corporation 19
InFlectis BioScience 20
PharmatrophiX, Inc. 21
Pharnext SAS 22
Charcot-Marie-Tooth Disease - Therapeutics Assessment 23
Assessment by Monotherapy Products 23
Assessment by Combination Products 24
Assessment by Target 25
Assessment by Mechanism of Action 27
Assessment by Route of Administration 29
Assessment by Molecule Type 30
Drug Profiles 32
(baclofen + naltrexone + sorbitol) - Drug Profile 32
Product Description 32
Mechanism of Action 32
RandD Progress 32
ADX-71441 - Drug Profile 34
Product Description 34
Mechanism of Action 34
RandD Progress 34
AFC-5128 - Drug Profile 35
Product Description 35
Mechanism of Action 35
RandD Progress 35
IFB-088 - Drug Profile 36
Product Description 36
Mechanism of Action 36
RandD Progress 36
Small Molecule to Inhibit HDAC6 for Charcot-Marie-Tooth Disease - Drug Profile 37
Product Description 37
Mechanism of Action 37
RandD Progress 37
Small Molecule to Inhibit Rab7 GTPase for Oncology, Neuropathy, Immunology, Neurodegenerative Diseases, Lipidoses and Charcot-Marie-Tooth Disease Type II - Drug Profile 38
Product Description 38
Mechanism of Action 38
RandD Progress 38
Small Molecules to Agonize TrkB and TrkC for Alzheimer's, Parkinson's and Charcot-Marie-Tooth Disease - Drug Profile 39
Product Description 39
Mechanism of Action 39
RandD Progress 39
Small Molecules to Inhibit HDAC-6 for Immunology, Oncology and Genetic Disorders - Drug Profile 40
Product Description 40
Mechanism of Action 40
RandD Progress 40
Small Molecules to Inhibit PMP22 for Charcot-Marie-Tooth Type 1 A - Drug Profile 41
Product Description 41
Mechanism of Action 41
RandD Progress 41
Stem Cell Therapy to Activate PMP22 for Charcot-Marie-Tooth Disease Type 1A - Drug Profile 42
Product Description 42
Mechanism of Action 42
RandD Progress 42
Charcot-Marie-Tooth Disease - Recent Pipeline Updates 43
Charcot-Marie-Tooth Disease - Dormant Projects 47
Charcot-Marie-Tooth Disease - Product Development Milestones 48
Featured News and Press Releases 48
Dec 15, 2015: Pharnext Initiates PLEO-CMT, an International Pivotal Phase 3 Trial, in Charcot-Marie-Tooth disease type 1A 48
Jun 30, 2015: Pharnext's PXT-3003 Featured in Multiple Presentations at 2015 Peripheral Nerve Society Biennial Meeting 48
Jan 13, 2015: InFlectis BioScience banks €1.75m to realise the potential of IFB-088 drug candidate, a breakthrough therapeutic approach to treat neurodegenerative diseases 49
Dec 17, 2014: Pharnext Announces Pleotherapy Proof of Concept in Charcot-Marie-Tooth Disease Type 1A through Two Peer-Reviewed Publications 50
Oct 21, 2014: Addex And The Charcot-Marie-Tooth Association Enter Collaboration To Advance ADX71441 In Charcot-Marie-Tooth 1A disorder 51
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 51
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 52
Apr 24, 2013: Addex Receives Approval To Initiate Phase I First-In-man Clinical Study For ADX71441 52
Jan 22, 2013: Addex Receives Broad US Composition Of Matter Patent For ADX71441 53
Jan 07, 2013: Addex Therapeutics Announces Positive Data With ADX71441 In Pre-Clinical Transgenic Model Of Charcot-Marie-Tooth 1A Disease 54
Appendix 55
Methodology 55
Coverage 55
Secondary Research 55
Primary Research 55
Expert Panel Validation 55
Contact Us 55
Disclaimer 56

List of Tables
Number of Products under Development for Charcot-Marie-Tooth Disease, H1 2016 8
Number of Products under Development for Charcot-Marie-Tooth Disease - Comparative Analysis, H1 2016 9
Number of Products under Development by Companies, H1 2016 10
Number of Products under Investigation by Universities/Institutes, H1 2016 11
Comparative Analysis by Late Stage Development, H1 2016 12
Comparative Analysis by Early Stage Development, H1 2016 13
Products under Development by Companies, H1 2016 14
Products under Investigation by Universities/Institutes, H1 2016 15
Charcot-Marie-Tooth Disease - Pipeline by Acetylon Pharmaceuticals, Inc., H1 2016 16
Charcot-Marie-Tooth Disease - Pipeline by Addex Therapeutics Ltd, H1 2016 17
Charcot-Marie-Tooth Disease - Pipeline by Affectis Pharmaceuticals AG, H1 2016 18
Charcot-Marie-Tooth Disease - Pipeline by Genzyme Corporation, H1 2016 19
Charcot-Marie-Tooth Disease - Pipeline by InFlectis BioScience, H1 2016 20
Charcot-Marie-Tooth Disease - Pipeline by PharmatrophiX, Inc., H1 2016 21
Charcot-Marie-Tooth Disease - Pipeline by Pharnext SAS, H1 2016 22
Assessment by Monotherapy Products, H1 2016 23
Assessment by Combination Products, H1 2016 24
Number of Products by Stage and Target, H1 2016 26
Number of Products by Stage and Mechanism of Action, H1 2016 28
Number of Products by Stage and Route of Administration, H1 2016 29
Number of Products by Stage and Molecule Type, H1 2016 31
Charcot-Marie-Tooth Disease Therapeutics - Recent Pipeline Updates, H1 2016 43
Charcot-Marie-Tooth Disease - Dormant Projects, H1 2016 47

List of Figures
Number of Products under Development for Charcot-Marie-Tooth Disease, H1 2016 8
Number of Products under Development for Charcot-Marie-Tooth Disease - Comparative Analysis, H1 2016 9
Number of Products under Development by Companies, H1 2016 10
Number of Products under Investigation by Universities/Institutes, H1 2016 11
Comparative Analysis by Early Stage Products, H1 2016 13
Assessment by Monotherapy Products, H1 2016 23
Assessment by Combination Products, H1 2016 24
Number of Products by Targets, H1 2016 25
Number of Products by Stage and Targets, H1 2016 25
Number of Products by Mechanism of Actions, H1 2016 27
Number of Products by Stage and Mechanism of Actions, H1 2016 27
Number of Products by Stage and Routes of Administration, H1 2016 29
Number of Products by Molecule Types, H1 2016 30
Number of Products by Stage and Molecule Types, H1 2016 30

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