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

Published By :

Global Markets Direct

Published Date : Dec 2016

Category :

Pharmaceutical

No. of Pages : 52 Pages

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

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Charcot-Marie-Tooth Disease Type I A Pipeline Review, H2 2016, provides an overview of the Charcot-Marie-Tooth Disease Type I A (Genetic Disorders) pipeline landscape.

Charcot-Marie-Tooth disease type 1A (CMT1A) is a type of inherited neurological disorder that affects the peripheral nerves. Symptoms include distal weakness and atrophy, sensory loss, foot deformities, and slow nerve conduction velocity. The disorder is caused by a duplication of the PMP22 gene on Chromosome 17. Symptomatic treatment includes pain medication, physical therapy and surgery.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Charcot-Marie-Tooth Disease Type I A Pipeline Review, H2 2016, provides comprehensive information on the therapeutics under development for Charcot-Marie-Tooth Disease Type I A (Genetic Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.

The Charcot-Marie-Tooth Disease Type I A (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Charcot-Marie-Tooth Disease Type I A and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase III, IND/CTA Filed, Preclinical and Discovery stages are 1, 1, 1 and 2 respectively. Similarly, the Universities portfolio in Preclinical stages comprises 1 molecules, respectively.

Charcot-Marie-Tooth Disease Type I A (Genetic Disorders) pipeline guide 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. The guide is built using data and information sourced from Global Markets Directs 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. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

Scope

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

Reasons to buy

- Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
- Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.
- Find and recognize significant and varied types of therapeutics under development for Charcot-Marie-Tooth Disease Type I A (Genetic Disorders).
- Classify potential new clients or partners in the target demographic.
- Develop tactical initiatives by understanding the focus areas of leading companies.
- Plan mergers and acquisitions meritoriously by identifying key players and its most promising pipeline therapeutics.
- Formulate corrective measures for pipeline projects by understanding Charcot-Marie-Tooth Disease Type I A (Genetic Disorders) 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.
- Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what 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 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: Pharnexts 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
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
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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