Charcot-Marie-Tooth Disease Type I A – Pipeline Review, H2 2016

Global Markets Direct’s, ‘Charcot-Marie-Tooth Disease Type I A – Pipeline Review, H2 2016’, provides an overview of the Charcot-Marie-Tooth Disease Type I A pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Charcot-Marie-Tooth Disease Type I A, 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 Type I A 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 Type I A

The report reviews pipeline therapeutics for Charcot-Marie-Tooth Disease Type I A 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 Type I A therapeutics and enlists all their major and minor projects

The report assesses Charcot-Marie-Tooth Disease Type I A 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 Type I A

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 Type I A

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 Type I A 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

Addex Therapeutics Ltd

Affectis Pharmaceuticals AG

Genzyme Corp

Lead Discovery Center GmbH

Pharnext SA

Table of Contents

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 Type I A Overview 6

Therapeutics Development 7

Pipeline Products for Charcot-Marie-Tooth Disease Type I A - Overview 7

Pipeline Products for Charcot-Marie-Tooth Disease Type I A - Comparative Analysis 8

Charcot-Marie-Tooth Disease Type I A - Therapeutics under Development by Companies 9

Charcot-Marie-Tooth Disease Type I A - Therapeutics under Investigation by Universities/Institutes 10

Charcot-Marie-Tooth Disease Type I A - Pipeline Products Glance 11

Late Stage Products 11

Early Stage Products 12

Charcot-Marie-Tooth Disease Type I A - Products under Development by Companies 13

Charcot-Marie-Tooth Disease Type I A - Products under Investigation by Universities/Institutes 14

Charcot-Marie-Tooth Disease Type I A - Companies Involved in Therapeutics Development 15

Addex Therapeutics Ltd 15

Affectis Pharmaceuticals AG 16

Genzyme Corp 17

Lead Discovery Center GmbH 18

Pharnext SA 19

Charcot-Marie-Tooth Disease Type I A - Therapeutics Assessment 20

Assessment by Monotherapy Products 20

Assessment by Combination Products 21

Assessment by Target 22

Assessment by Mechanism of Action 24

Assessment by Route of Administration 26

Assessment by Molecule Type 27

Drug Profiles 29

(baclofen + naltrexone hydrochloride + sorbitol) - Drug Profile 29

Product Description 29

Mechanism Of Action 29

R&D Progress 29

ADX-71441 - Drug Profile 32

Product Description 32

Mechanism Of Action 32

R&D Progress 32

AFC-5128 - Drug Profile 36

Product Description 36

Mechanism Of Action 36

R&D Progress 36

Small Molecules for Charcot-Marie-Tooth Disease Type I A - Drug Profile 37

Product Description 37

Mechanism Of Action 37

R&D Progress 37

Small Molecules to Inhibit PMP22 for Charcot-Marie-Tooth Type 1 A - Drug Profile 38

Product Description 38

Mechanism Of Action 38

R&D Progress 38

Stem Cell Therapy for Charcot-Marie-Tooth Disease Type 1A - Drug Profile 39

Product Description 39

Mechanism Of Action 39

R&D Progress 39

Charcot-Marie-Tooth Disease Type I A - Dormant Projects 40

Charcot-Marie-Tooth Disease Type I A - Product Development Milestones 41

Featured News & Press Releases 41

Dec 20, 2016: Pharnext Completes Patient Enrollment in International Pivotal Phase 3 Trial of PXT3003 for Charcot-Marie-Tooth Disease Type 1A 41

Nov 17, 2016: Pharnext Announces that the Data Safety Monitoring Board Recommends Continuing the Ongoing Phase 3 Trial of PXT3003 for Charcot-Marie-Tooth Disease Type 1A 42

Oct 04, 2016: Pharnext Announces Support of the First Patient-Centered Charcot-Marie-Tooth Summit Organized by the Hereditary Neuropathy Foundation 42

Sep 01, 2016: Pharnext’s PXT3003 Featured in Multiple Presentations at the 6th International Charcot-Marie-Tooth and Related Neuropathy Consortium (CMTR) Meeting 43

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 43

Feb 16, 2016: Pharnext Supports the Fight Against Rare Diseases through Development of Pleotherapy-based Treatments 44

Dec 15, 2015: Pharnext Initiates PLEO-CMT, an International Pivotal Phase 3 Trial, in Charcot-Marie-Tooth disease type 1A 44

Jun 30, 2015: Pharnext's PXT-3003 Featured in Multiple Presentations at 2015 Peripheral Nerve Society Biennial Meeting 45

Dec 17, 2014: Pharnext Announces Pleotherapy Proof of Concept in Charcot-Marie-Tooth Disease Type 1A through Two Peer-Reviewed Publications 46

Oct 21, 2014: Addex And The Charcot-Marie-Tooth Association Enter Collaboration To Advance ADX71441 In Charcot-Marie-Tooth 1A disorder 47

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 48

Apr 24, 2013: Addex Receives Approval To Initiate Phase I First-In-man Clinical Study For ADX71441 48

Jan 22, 2013: Addex Receives Broad US Composition Of Matter Patent For ADX71441 49

Jan 07, 2013: Addex Therapeutics Announces Positive Data With ADX71441 In Pre-Clinical Transgenic Model Of Charcot-Marie-Tooth 1A Disease 49

Nov 22, 2011: Pharnext Completes Patient Recruitment For Phase II Study Of PXT3003 In Type 1A Charcot-Marie-Tooth Disease 50

Appendix 51

Methodology 51

Coverage 51

Secondary Research 51

Primary Research 51

Expert Panel Validation 51

Contact Us 51

Disclaimer 5

List of Tables

List of Tables

Number of Products under Development for Charcot-Marie-Tooth Disease Type I A, H2 2016 7

Number of Products under Development for Charcot-Marie-Tooth Disease Type I A – Comparative Analysis, H2 2016 8

Number of Products under Development by Companies, H2 2016 9

Number of Products under Investigation by Universities/Institutes, H2 2016 10

Comparative Analysis by Late Stage Development, H2 2016 11

Comparative Analysis by Early Stage Development, H2 2016 12

Products under Development by Companies, H2 2016 13

Products under Investigation by Universities/Institutes, H2 2016 14

Charcot-Marie-Tooth Disease Type I A – Pipeline by Addex Therapeutics Ltd, H2 2016 15

Charcot-Marie-Tooth Disease Type I A – Pipeline by Affectis Pharmaceuticals AG, H2 2016 16

Charcot-Marie-Tooth Disease Type I A – Pipeline by Genzyme Corp, H2 2016 17

Charcot-Marie-Tooth Disease Type I A – Pipeline by Lead Discovery Center GmbH, H2 2016 18

Charcot-Marie-Tooth Disease Type I A – Pipeline by Pharnext SA, H2 2016 19

Assessment by Monotherapy Products, H2 2016 20

Assessment by Combination Products, H2 2016 21

Number of Products by Stage and Target, H2 2016 23

Number of Products by Stage and Mechanism of Action, H2 2016 25

Number of Products by Stage and Route of Administration, H2 2016 26

Number of Products by Stage and Molecule Type, H2 2016 28

Charcot-Marie-Tooth Disease Type I A – Dormant Projects, H2 2016 40

List of Figures

List of Figures

Number of Products under Development for Charcot-Marie-Tooth Disease Type I A, H2 2016 7

Number of Products under Development for Charcot-Marie-Tooth Disease Type I A – Comparative Analysis, H2 2016 8

Number of Products under Development by Companies, H2 2016 9

Comparative Analysis by Early Stage Products, H2 2016 12

Assessment by Monotherapy Products, H2 2016 20

Assessment by Combination Products, H2 2016 21

Number of Products by Targets, H2 2016 22

Number of Products by Stage and Targets, H2 2016 22

Number of Products by Mechanism of Actions, H2 2016 24

Number of Products by Stage and Mechanism of Actions, H2 2016 24

Number of Products by Stage and Routes of Administration, H2 2016 26

Number of Products by Molecule Types, H2 2016 27

Number of Products by Stage and Molecule Types, H2 2016 27

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