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

  • ID: 3804687
  • Drug Pipelines
  • 60 pages
  • Global Markets Direct
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FEATURED COMPANIES

  • Addex Therapeutics Ltd
  • Genzyme Corporation
  • InFlectis BioScience
  • PharmatrophiX, Inc.
  • Pharnext SAS
  • MORE
Charcot-Marie-Tooth Disease - Pipeline Review, H2 2016

Summary

‘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.

Our 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 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
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Note: Product cover images may vary from those shown
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FEATURED COMPANIES

  • Addex Therapeutics Ltd
  • Genzyme Corporation
  • InFlectis BioScience
  • PharmatrophiX, Inc.
  • Pharnext SAS
  • MORE
List of Tables

List of Figures

Introduction

Report Coverage

Charcot-Marie-Tooth Disease Overview

Therapeutics Development

Pipeline Products for Charcot-Marie-Tooth Disease - Overview

Pipeline Products for Charcot-Marie-Tooth Disease - Comparative Analysis

Charcot-Marie-Tooth Disease - Therapeutics under Development by Companies

Charcot-Marie-Tooth Disease - Therapeutics under Investigation by Universities/Institutes

Charcot-Marie-Tooth Disease - Pipeline Products Glance

Late Stage Products

Early Stage Products

Charcot-Marie-Tooth Disease - Products under Development by Companies

Charcot-Marie-Tooth Disease - Products under Investigation by Universities/Institutes

Charcot-Marie-Tooth Disease - Companies Involved in Therapeutics Development

Acetylon Pharmaceuticals, Inc.

Addex Therapeutics Ltd

Affectis Pharmaceuticals AG

Genzyme Corporation

InFlectis BioScience

Lead Discovery Center GmbH

PharmatrophiX, Inc.

Pharnext SAS

Charcot-Marie-Tooth Disease - Therapeutics Assessment

Assessment by Monotherapy Products

Assessment by Combination Products

Assessment by Target

Assessment by Mechanism of Action

Assessment by Route of Administration

Assessment by Molecule Type

Drug Profiles

(baclofen + naltrexone + sorbitol) - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

ADX-71441 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

AFC-5128 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

IFB-088 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

LM-22B10 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

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

Product Description

Mechanism Of Action

R&D Progress

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

Product Description

Mechanism Of Action

R&D Progress

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

Product Description

Mechanism Of Action

R&D Progress

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

Product Description

Mechanism Of Action

R&D Progress

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

Product Description

Mechanism Of Action

R&D Progress

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

Product Description

Mechanism Of Action

R&D Progress

Charcot-Marie-Tooth Disease - Dormant Projects

Charcot-Marie-Tooth Disease - Product Development Milestones

Featured News & Press Releases

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer 60List of Tables

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

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

Number of Products under Development by Companies, H2 2016

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

Comparative Analysis by Late Stage Development, H2 2016

Comparative Analysis by Early Stage Development, H2 2016

Products under Development by Companies, H2 2016

Products under Investigation by Universities/Institutes, H2 2016

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

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

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

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

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

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

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

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

Assessment by Monotherapy Products, H2 2016

Assessment by Combination Products, H2 2016

Number of Products by Stage and Target, H2 2016

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

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

Number of Products by Stage and Molecule Type, H2 2016

Charcot-Marie-Tooth Disease - Dormant Projects, H2 2016 47List of Figures

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

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

Number of Products under Development by Companies, H2 2016

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

Comparative Analysis by Early Stage Products, H2 2016

Assessment by Monotherapy Products, H2 2016

Assessment by Combination Products, H2 2016

Number of Products by Targets, H2 2016

Number of Products by Stage and Targets, H2 2016

Number of Products by Mechanism of Actions, H2 2016

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

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

Number of Products by Molecule Types, H2 2016

Number of Products by Stage and Molecule Types, H2 2016
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FEATURED COMPANIES

  • Addex Therapeutics Ltd
  • Genzyme Corporation
  • InFlectis BioScience
  • PharmatrophiX, Inc.
  • Pharnext SAS
  • MORE
Charcot-Marie-Tooth Disease pipeline therapeutics constitutes close to 11 molecules. Out of which approximately 8 molecules are developed by Companies and remaining by the Universities/Institutes. Our latest report Charcot-Marie-Tooth Disease – Pipeline Review, H2 2016, outlays 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.

Charcot-Marie-Tooth Disease Charcot Marie Tooth disease is a group of hereditary disorders that damage the nerves in arms and legs (peripheral nerves). Symptoms include high foot arches, loss of muscle bulk in legs and feet, curled toes, frequent tripping or falling and decreased ability to run. Risk factors include diabetes and family history. The molecules developed by Companies in Phase III, IND/CTA Filed, Preclinical and Discovery stages are 1, 1, 3 and 3 respectively. Similarly, the Universities portfolio in Preclinical and Discovery stages comprises 2 and 1 molecules, respectively.

Furthermore, this report also reviews of key players involved in therapeutic development for Charcot-Marie-Tooth Disease and features dormant and discontinued projects. Driven by data 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.
Note: Product cover images may vary from those shown
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Acetylon Pharmaceuticals, Inc.
Addex Therapeutics Ltd
Affectis Pharmaceuticals AG
Genzyme Corporation
InFlectis BioScience
Lead Discovery Center GmbH
PharmatrophiX, Inc.
Pharnext SAS
Note: Product cover images may vary from those shown
6 of 6
Note: Product cover images may vary from those shown
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