Wilson Disease – Pipeline Review, H2 2016

Global Markets Direct’s, ‘Wilson Disease – Pipeline Review, H2 2016’, 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

Dimension Therapeutics Inc

GMP-Orphan SAS

Revive Therapeutics Ltd

Wilson Therapeutics AB

Table of Contents

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 Wilson’s 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

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

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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