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Wilson Disease - Pipeline Review, H1 2017

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TABLE OF CONTENTS

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DESCRIPTION

Wilson Disease - Pipeline Review, H1 2017

Summary

Global Markets Directs latest Pharmaceutical and Healthcare disease pipeline guide Wilson Disease - Pipeline Review, H1 2017, 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 Directs Pharmaceutical and Healthcare latest pipeline guide Wilson Disease - Pipeline Review, H1 2017, 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 Pre-Registration, Phase II and Preclinical stages are 1, 1 and 4 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

Introduction
Global Markets Direct Report Coverage
Wilson Disease - Overview
Wilson Disease - Therapeutics Development
Pipeline Overview
Pipeline by Companies
Pipeline by Universities/Institutes
Products under Development by Companies
Products under Development by Universities/Institutes
Wilson Disease - Therapeutics Assessment
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Wilson Disease - Companies Involved in Therapeutics Development
Dimension Therapeutics Inc
Ionis Pharmaceuticals Inc
Vivet Therapeutics SAS
Wilson Therapeutics AB
Wilson Disease - Drug Profiles
Antisense Oligonucleotides to Inhibit CTR1 for Wilson Disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
CM-1186 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
DTX-701 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
methanobactin - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
trientine tetrahydrochloride - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
VTX-801 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
WTX-101 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Wilson Disease - Dormant Projects
Wilson Disease - Discontinued Products
Wilson Disease - Product Development Milestones
Featured News & Press Releases
Jun 08, 2017: Wilson Therapeutics presents Phase 2 data for WTX101 at MDS meeting
Apr 24, 2017: Wilson Therapeutics presented positive final Phase 2 data for WTX101 at EASL Annual Meeting
Apr 21, 2017: gmp-orphan Receives CHMP Positive Opinion For Cuprior
Apr 05, 2017: Final Phase 2 Results For WTX-101 Accepted As A Late-Breaker Presentation At EASL Annual Meeting
Dec 05, 2016: Wilson Therapeutics Announces That WTX101 Meets The Primary Endpoint In Phase 2 Study In Wilson Disease
Nov 11, 2016: Wilson Therapeutics Presents Updated Preliminary Clinical Data on WTX101 at the AASLD Liver Meeting
Jun 23, 2016: Wilson Therapeutics Presents Preliminary Clinical Data on Decuprate at the 20th International Congress of Parkinsons Disease and Movement Disorders
May 30, 2016: Wilson Therapeutics Presents Encouraging Clinical Data On Decuprate At The Congress Of The European Academy Of Neurology
May 18, 2016: Wilson Therapeutics Announces That the Ongoing Phase II Study Has Been Fully Enrolled
Apr 14, 2016: Wilson Therapeutics presents data on Decuprate (WTX101) from ongoing Phase 2 study at The International Liver Congress 2016
Apr 16, 2015: Wilson Therapeutics Announces Presentation Of WTX101-201 Phase 2 Study At The EASL 50th International Liver Congress 2015
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
Jan 13, 2014: Wilson Therapeutics Announces Successful Filing of U.S. IND to Advance WTX101 Development Program for Wilson Disease
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer

List Of Tables

Number of Products under Development for Wilson Disease, H1 2017
Number of Products under Development by Companies, H1 2017
Number of Products under Development by Universities/Institutes, H1 2017
Products under Development by Companies, H1 2017
Products under Development by Universities/Institutes, H1 2017
Number of Products by Stage and Target, H1 2017
Number of Products by Stage and Mechanism of Action, H1 2017
Number of Products by Stage and Route of Administration, H1 2017
Number of Products by Stage and Molecule Type, H1 2017
Wilson Disease - Pipeline by Dimension Therapeutics Inc, H1 2017
Wilson Disease - Pipeline by Ionis Pharmaceuticals Inc, H1 2017
Wilson Disease - Pipeline by Vivet Therapeutics SAS, H1 2017
Wilson Disease - Pipeline by Wilson Therapeutics AB, H1 2017
Wilson Disease - Dormant Projects, H1 2017
Wilson Disease - Discontinued Products, H1 2017

List Of Figures

Number of Products under Development for Wilson Disease, H1 2017
Number of Products under Development by Companies, H1 2017
Number of Products by Targets, H1 2017
Number of Products by Stage and Targets, H1 2017
Number of Products by Mechanism of Actions, H1 2017
Number of Products by Stage and Mechanism of Actions, H1 2017
Number of Products by Stage and Routes of Administration, H1 2017
Number of Products by Molecule Types, H1 2017
Number of Products by Stage and Molecule Types, H1 2017

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