Wilson Disease – Pipeline Review, H2 2017

Global Markets Direct’s, ‘Wilson Disease – Pipeline Review, H2 2017’, provides an overview of the Wilson Disease pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Wilson 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 Wilson Disease 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 Wilson Disease

The report reviews pipeline therapeutics for Wilson 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 Wilson Disease therapeutics and enlists all their major and minor projects

The report assesses Wilson 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 Wilson 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 Wilson 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 Wilson 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

Companies mentioned

Ultragenyx Pharmaceutical Inc

Vivet Therapeutics SAS

Wilson Therapeutics AB

Table of Contents

Table of Contents

List of Tables

List of Figures

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

Ultragenyx Pharmaceutical Inc

Vivet Therapeutics SAS

Wilson Therapeutics AB

Wilson Disease Drug Profiles

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 Product Development Milestones

Featured News & Press Releases

Dec 14, 2017: WTX101 Granted Fast Track Designation by the U.S. FDA for the Treatment of Wilson Disease

Oct 23, 2017: Wilson Therapeutics reaches agreement with the FDA and EMA to initiate pivotal Phase 3 FOCuS study with WTX101 in Wilson Disease

Oct 20, 2017: Promising Preliminary Long-term Data For WTX101 In Wilson Disease Highlighted at The Liver Meeting

Oct 06, 2017: Phase 2 Clinical Trial Data for WTX101 Published in The Lancet Gastroenterology & Hepatology

Oct 01, 2017: Promising Preliminary Long-term Data from WTX101 Phase 2 Extension Study to be Presented at AASLD Annual Meeting

Sep 26, 2017: Vivet’s First Gene Therapy Product, VTX 801 for Wilson’s Disease, Receives European and US Orphan Drug Designation

Jun 08, 2017: Wilson Therapeutics presents Phase 2 data for WTX101 at MDS meeting

Apr 25, 2017: Wilson Therapeutics presents promising neurological Phase 2 data for WTX101 at AAN 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 Parkinson's Disease and Movement Disorders

May 30, 2016: Wilson Therapeutics Presents Encouraging Clinical Data On Decuprate At The Congress Of The European Academy Of Neurology

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer

List of Tables

List of Tables

Number of Products under Development for Wilson Disease, H2 2017

Number of Products under Development by Companies, H2 2017

Number of Products under Development by Universities/Institutes, H2 2017

Products under Development by Companies, H2 2017

Products under Development by Universities/Institutes, H2 2017

Number of Products by Stage and Target, H2 2017

Number of Products by Stage and Mechanism of Action, H2 2017

Number of Products by Stage and Route of Administration, H2 2017

Number of Products by Stage and Molecule Type, H2 2017

Wilson Disease Pipeline by Ultragenyx Pharmaceutical Inc, H2 2017

Wilson Disease Pipeline by Vivet Therapeutics SAS, H2 2017

Wilson Disease Pipeline by Wilson Therapeutics AB, H2 2017

Wilson Disease Dormant Projects, H2 2017

List of Figures

List of Figures

Number of Products under Development for Wilson Disease, H2 2017

Number of Products under Development by Companies, H2 2017

Number of Products by Targets, H2 2017

Number of Products by Stage and Targets, H2 2017

Number of Products by Mechanism of Actions, H2 2017

Number of Products by Stage and Mechanism of Actions, H2 2017

Number of Products by Stage and Routes of Administration, H2 2017

Number of Products by Molecule Types, H2 2017

Number of Products by Stage and Molecule Types, H2 2017

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