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Wilson Disease - Pipeline Review, H2 2016

Published By :

Global Markets Direct

Published Date : Dec 2016

Category :

Pharmaceutical

No. of Pages : 45 Pages

Wilson Disease - Pipeline Review, H2 2016

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Wilson Disease Pipeline Review, H2 2016, provides an overview of the Wilson Disease (Genetic Disorders) pipeline landscape.

Wilson disease is a rare autosomal recessive inherited disorder of copper metabolism that is characterized by excessive deposition of copper in the liver, brain, and other tissues. Symptoms include abdominal pain, jaundice, problems with speech, swallowing and muscle stiffness. Treatment includes chelators and Vitamin E supplements.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Wilson Disease Pipeline Review, H2 2016, provides comprehensive information on the therapeutics under development for Wilson Disease (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 Wilson Disease (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Wilson Disease and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase II, Phase I and Preclinical stages are 1, 1 and 2 respectively. Similarly, the Universities portfolio in Preclinical stages comprises 1 molecules, respectively.

Wilson Disease (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 Wilson Disease (Genetic Disorders).
- The pipeline guide reviews pipeline therapeutics for Wilson Disease (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 Wilson Disease (Genetic Disorders) therapeutics and enlists all their major and minor projects.
- The pipeline guide evaluates Wilson Disease (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 Wilson Disease (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 Wilson Disease (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 Wilson Disease (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
Wilson Disease Overview 6
Therapeutics Development 7
Pipeline Products for Wilson Disease - Overview 7
Pipeline Products for Wilson Disease - Comparative Analysis 8
Wilson Disease - Therapeutics under Development by Companies 9
Wilson Disease - Therapeutics under Investigation by Universities/Institutes 10
Wilson Disease - Pipeline Products Glance 11
Clinical Stage Products 11
Early Stage Products 12
Wilson Disease - Products under Development by Companies 13
Wilson Disease - Products under Investigation by Universities/Institutes 14
Wilson Disease - Companies Involved in Therapeutics Development 15
Dimension Therapeutics Inc 15
GMP-Orphan SAS 16
Revive Therapeutics Ltd 17
Wilson Therapeutics AB 18
Wilson Disease - Therapeutics Assessment 19
Assessment by Monotherapy Products 19
Assessment by Target 20
Assessment by Mechanism of Action 22
Assessment by Route of Administration 24
Assessment by Molecule Type 25
Drug Profiles 27
DTX-701 - Drug Profile 27
Product Description 27
Mechanism Of Action 27
R&D Progress 27
Gene Therapy to Activate ATP7B for Wilsons Disease - Drug Profile 28
Product Description 28
Mechanism Of Action 28
R&D Progress 28
REV-005 - Drug Profile 29
Product Description 29
Mechanism Of Action 29
R&D Progress 29
trientine tetrahydrochloride - Drug Profile 30
Product Description 30
Mechanism Of Action 30
R&D Progress 30
WTX-101 - Drug Profile 31
Product Description 31
Mechanism Of Action 31
R&D Progress 31
Wilson Disease - Dormant Projects 34
Wilson Disease - Discontinued Products 35
Wilson Disease - Product Development Milestones 36
Featured News & Press Releases 36
Dec 05, 2016: Kadmon Submits Abbreviated New Drug Application Filing for Trientine Hydrochloride to FDA 36
Dec 05, 2016: Wilson Therapeutics Announces That WTX101 Meets The Primary Endpoint In Phase 2 Study In Wilson Disease 36
Nov 11, 2016: Wilson Therapeutics Presents Updated Preliminary Clinical Data on WTX101 at the AASLD Liver Meeting 37
Jun 23, 2016: Wilson Therapeutics Presents Preliminary Clinical Data on Decuprate at the 20th International Congress of Parkinson's Disease and Movement Disorders 38
May 30, 2016: Wilson Therapeutics Presents Encouraging Clinical Data On Decuprate At The Congress Of The European Academy Of Neurology 39
May 18, 2016: Wilson Therapeutics Announces That the Ongoing Phase II Study Has Been Fully Enrolled 40
Apr 14, 2016: Wilson Therapeutics presents data on Decuprate (WTX101) from ongoing Phase 2 study at The International Liver Congress 2016 40
Apr 16, 2015: Wilson Therapeutics Announces Presentation Of WTX101-201 Phase 2 Study At The EASL 50th International Liver Congress 2015 41
Nov 17, 2014: Wilson Therapeutics Announces Start Of Phase 2 Study To Evaluate The Efficacy And Safety Of WTX101 In Newly Diagnosed Wilson Disease Patients 42
Jan 13, 2014: Wilson Therapeutics Announces Successful Filing of U.S. IND to Advance WTX101 Development Program for Wilson Disease 42
Appendix 44
Methodology 44
Coverage 44
Secondary Research 44
Primary Research 44
Expert Panel Validation 44
Contact Us 44
Disclaimer 45

List of Tables
Number of Products under Development for Wilson Disease, H2 2016 7
Number of Products under Development for Wilson Disease - 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 Clinical 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
Wilson Disease - Pipeline by Dimension Therapeutics Inc, H2 2016 15
Wilson Disease - Pipeline by GMP-Orphan SAS, H2 2016 16
Wilson Disease - Pipeline by Revive Therapeutics Ltd, H2 2016 17
Wilson Disease - Pipeline by Wilson Therapeutics AB, H2 2016 18
Assessment by Monotherapy Products, H2 2016 19
Number of Products by Stage and Target, H2 2016 21
Number of Products by Stage and Mechanism of Action, H2 2016 23
Number of Products by Stage and Route of Administration, H2 2016 24
Number of Products by Stage and Molecule Type, H2 2016 26
Wilson Disease - Dormant Projects, H2 2016 34
Wilson Disease - Discontinued Products, H2 2016 35

List of Figures
Number of Products under Development for Wilson Disease, H2 2016 7
Number of Products under Development for Wilson Disease - Comparative Analysis, H2 2016 8
Number of Products under Development by Companies, H2 2016 9
Comparative Analysis by Clinical Stage Development, H2 2016 11
Comparative Analysis by Early Stage Products, H2 2016 12
Assessment by Monotherapy Products, H2 2016 19
Number of Products by Targets, H2 2016 20
Number of Products by Stage and Targets, H2 2016 20
Number of Products by Mechanism of Actions, H2 2016 22
Number of Products by Stage and Mechanism of Actions, H2 2016 22
Number of Products by Molecule Types, H2 2016 25
Number of Products by Stage and Molecule Types, H2 2016 25

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