Charcot-Marie-Tooth Disease - Pipeline Review, H2 2016

Global Markets Direct’s, ‘Charcot-Marie-Tooth Disease - Pipeline Review, H2 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

Companies mentioned

Acetylon Pharmaceuticals, Inc.

Addex Therapeutics Ltd

Affectis Pharmaceuticals AG

Genzyme Corporation

InFlectis BioScience

Lead Discovery Center GmbH

PharmatrophiX, Inc.

Pharnext SAS

Acetylon Pharmaceuticals, Inc.

Addex Therapeutics Ltd

Affectis Pharmaceuticals AG

Genzyme Corporation

InFlectis BioScience

Lead Discovery Center GmbH

PharmatrophiX, Inc.

Pharnext SAS

Table of Contents

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 ...

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

Lead Discovery Center GmbH 21

PharmatrophiX, Inc. 22

Pharnext SAS 23

Charcot-Marie-Tooth Disease - Therapeutics Assessment 24

Assessment by Monotherapy Products 24

Assessment by Combination Products 25

Assessment by Target 26

Assessment by Mechanism of Action 28

Assessment by Route of Administration 30

Assessment by Molecule Type 31

Drug Profiles 33

(baclofen + naltrexone + sorbitol) - Drug Profile 33

Product Description 33

Mechanism Of Action 33

R&D Progress 33

ADX-71441 - Drug Profile 35

Product Description 35

Mechanism Of Action 35

R&D Progress 35

AFC-5128 - Drug Profile 38

Product Description 38

Mechanism Of Action 38

R&D Progress 38

IFB-088 - Drug Profile 39

Product Description 39

Mechanism Of Action 39

R&D Progress 39

LM-22B10 - Drug Profile 40

Product Description 40

Mechanism Of Action 40

R&D Progress 40

Small Molecule to Inhibit HDAC6 for Charcot-Marie-Tooth Disease - Drug Profile 41

Product Description 41

Mechanism Of Action 41

R&D Progress 41

Small Molecule to Inhibit Rab7 GTPase for Oncology, Neuropathy, Immunology, Neurodegenerative Diseases, Lipidoses and Charcot-Marie-Tooth Disease Type II - Drug Profile 42

Product Description 42

Mechanism Of Action 42

R&D Progress 42

Small Molecules for Charcot-Marie-Tooth Disease Type I A - Drug Profile 43

Product Description 43

Mechanism Of Action 43

R&D Progress 43

Small Molecules to Inhibit HDAC-6 for Immunology, Oncology and Genetic Disorders - Drug Profile 44

Product Description 44

Mechanism Of Action 44

R&D Progress 44

Small Molecules to Inhibit PMP22 for Charcot-Marie-Tooth Type 1 A - Drug Profile 45

Product Description 45

Mechanism Of Action 45

R&D Progress 45

Stem Cell Therapy to Activate PMP22 for Charcot-Marie-Tooth Disease Type 1A - Drug Profile 46

Product Description 46

Mechanism Of Action 46

R&D Progress 46

Charcot-Marie-Tooth Disease - Dormant Projects 47

Charcot-Marie-Tooth Disease - Product Development Milestones 48

Featured News & Press Releases 48

Apr 25, 2016: Pharnext Announces the Opening of the First U.S. Study Site for its PLEO-CMT International Pivotal Phase 3 Trial in Charcot-Marie-Tooth Disease Type 1A 48

Feb 16, 2016: Pharnext Supports the Fight Against Rare Diseases through Development of Pleotherapy-based Treatments 49

Jan 25, 2016: IFB-088 (Sephin1) receives orphan drug designation in the European Union for the treatment of Charcot-Marie-Tooth disease 49

Dec 15, 2015: Pharnext Initiates PLEO-CMT, an International Pivotal Phase 3 Trial, in Charcot-Marie-Tooth disease type 1A 50

Oct 27, 2015: FDA grants orphan drug designation to IFB-088 (Sephin1) for the treatment of Charcot-Marie-Tooth disease 51

Jun 30, 2015: Pharnext's PXT-3003 Featured in Multiple Presentations at 2015 Peripheral Nerve Society Biennial Meeting 51

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 52

Dec 17, 2014: Pharnext Announces Pleotherapy Proof of Concept in Charcot-Marie-Tooth Disease Type 1A through Two Peer-Reviewed Publications 52

Oct 21, 2014: Addex And The Charcot-Marie-Tooth Association Enter Collaboration To Advance ADX71441 In Charcot-Marie-Tooth 1A disorder 53

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 54

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 55

Apr 24, 2013: Addex Receives Approval To Initiate Phase I First-In-man Clinical Study For ADX71441 55

Jan 22, 2013: Addex Receives Broad US Composition Of Matter Patent For ADX71441 56

Jan 07, 2013: Addex Therapeutics Announces Positive Data With ADX71441 In Pre-Clinical Transgenic Model Of Charcot-Marie-Tooth 1A Disease 57

Nov 22, 2011: Pharnext Completes Patient Recruitment For Phase II Study Of PXT3003 In Type 1A Charcot-Marie-Tooth Disease 57

Appendix 59

Methodology 59

Coverage 59

Secondary Research 59

Primary Research 59

Expert Panel Validation 59

Contact Us 59

Disclaimer 60

List of Tables

List of Tables

Number of Products under Development for Charcot-Marie-Tooth Disease, H2 2016 8

Number of Products under Development for Charcot-Marie-Tooth Disease - Comparative Analysis, H2 ...

List of Tables

Number of Products under Development for Charcot-Marie-Tooth Disease, H2 2016 8

Number of Products under Development for Charcot-Marie-Tooth Disease - Comparative Analysis, H2 2016 9

Number of Products under Development by Companies, H2 2016 10

Number of Products under Investigation by Universities/Institutes, H2 2016 11

Comparative Analysis by Late Stage Development, H2 2016 12

Comparative Analysis by Early Stage Development, H2 2016 13

Products under Development by Companies, H2 2016 14

Products under Investigation by Universities/Institutes, H2 2016 15

Charcot-Marie-Tooth Disease - Pipeline by Acetylon Pharmaceuticals, Inc., H2 2016 16

Charcot-Marie-Tooth Disease - Pipeline by Addex Therapeutics Ltd, H2 2016 17

Charcot-Marie-Tooth Disease - Pipeline by Affectis Pharmaceuticals AG, H2 2016 18

Charcot-Marie-Tooth Disease - Pipeline by Genzyme Corporation, H2 2016 19

Charcot-Marie-Tooth Disease - Pipeline by InFlectis BioScience, H2 2016 20

Charcot-Marie-Tooth Disease - Pipeline by Lead Discovery Center GmbH, H2 2016 21

Charcot-Marie-Tooth Disease - Pipeline by PharmatrophiX, Inc., H2 2016 22

Charcot-Marie-Tooth Disease - Pipeline by Pharnext SAS, H2 2016 23

Assessment by Monotherapy Products, H2 2016 24

Assessment by Combination Products, H2 2016 25

Number of Products by Stage and Target, H2 2016 27

Number of Products by Stage and Mechanism of Action, H2 2016 29

Number of Products by Stage and Route of Administration, H2 2016 30

Number of Products by Stage and Molecule Type, H2 2016 32

Charcot-Marie-Tooth Disease - Dormant Projects, H2 2016 47

List of Figures

List of Figures

Number of Products under Development for Charcot-Marie-Tooth Disease, H2 2016 8

Number of Products under Development for Charcot-Marie-Tooth Disease - Comparative Analysis, H2 ...

List of Figures

Number of Products under Development for Charcot-Marie-Tooth Disease, H2 2016 8

Number of Products under Development for Charcot-Marie-Tooth Disease - Comparative Analysis, H2 2016 9

Number of Products under Development by Companies, H2 2016 10

Number of Products under Investigation by Universities/Institutes, H2 2016 11

Comparative Analysis by Early Stage Products, H2 2016 13

Assessment by Monotherapy Products, H2 2016 24

Assessment by Combination Products, H2 2016 25

Number of Products by Targets, H2 2016 26

Number of Products by Stage and Targets, H2 2016 26

Number of Products by Mechanism of Actions, H2 2016 28

Number of Products by Stage and Mechanism of Actions, H2 2016 28

Number of Products by Stage and Routes of Administration, H2 2016 30

Number of Products by Molecule Types, H2 2016 31

Number of Products by Stage and Molecule Types, H2 2016 31

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