Hemochromatosis – Pipeline Review, H2 2017

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

The report provides comprehensive information on the therapeutics under development for Hemochromatosis, 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 Hemochromatosis 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 Hemochromatosis

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

The report assesses Hemochromatosis 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 Hemochromatosis

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 Hemochromatosis

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 Hemochromatosis 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

Ionis Pharmaceuticals Inc

La Jolla Pharmaceutical Company

Protagonist Therapeutics Inc

Silence Therapeutics Plc

Vifor Pharma AG

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

Hemochromatosis - Overview 6

Hemochromatosis - Therapeutics Development 7

Pipeline Overview 7

Pipeline by Companies 8

Pipeline by Universities/Institutes 9

Products under Development by Companies 10

Products under Development by Universities/Institutes 11

Hemochromatosis - Therapeutics Assessment 12

Assessment by Target 12

Assessment by Mechanism of Action 14

Assessment by Route of Administration 16

Assessment by Molecule Type 18

Hemochromatosis - Companies Involved in Therapeutics Development 20

Ionis Pharmaceuticals Inc 20

La Jolla Pharmaceutical Company 20

Protagonist Therapeutics Inc 21

Silence Therapeutics Plc 21

Vifor Pharma AG 21

Hemochromatosis - Drug Profiles 23

Antisense RNAi Oligonucleiotide for Hemochromatosis - Drug Profile 23

Product Description 23

Mechanism Of Action 23

R&D Progress 23

CN-128 - Drug Profile 24

Product Description 24

Mechanism Of Action 24

R&D Progress 24

Fusion Protein to Replace Hepcidin for Iron Overload Disorders - Drug Profile 25

Product Description 25

Mechanism Of Action 25

R&D Progress 25

IONISTMPRSS-6LRx - Drug Profile 26

Product Description 26

Mechanism Of Action 26

R&D Progress 26

LJPC-401 - Drug Profile 27

Product Description 27

Mechanism Of Action 27

R&D Progress 27

NSC-8679 - Drug Profile 29

Product Description 29

Mechanism Of Action 29

R&D Progress 29

PTG-300 - Drug Profile 30

Product Description 30

Mechanism Of Action 30

R&D Progress 30

VIT-2763 - Drug Profile 32

Product Description 32

Mechanism Of Action 32

R&D Progress 32

Hemochromatosis - Dormant Projects 33

Hemochromatosis - Product Development Milestones 34

Featured News & Press Releases 34

Sep 07, 2016: La Jolla Pharmaceutical Company Reports Positive Results from Phase 1 Study of LJPC-401 34

Jul 28, 2016: Protagonist Therapeutics Receives SBIR Funding for the Development of Injectable Hepcidin Mimetics for Treatment of Iron Overload Disorders 34

Oct 19, 2015: La Jolla Pharmaceutical Doses First Patient in Phase 1 Clinical Trial of LJPC-401 in Patients at Risk of Iron Overload 35

Aug 11, 2015: La Jolla Pharmaceutical Company Announces FDA Acceptance of IND for LJPC-401 35

Appendix 37

Methodology 37

Coverage 37

Secondary Research 37

Primary Research 37

Expert Panel Validation 37

Contact Us 37

Disclaimer 38

List of Tables

List of Tables

Number of Products under Development for Hemochromatosis, H2 2017 7

Number of Products under Development by Companies, H2 2017 8

Number of Products under Development by Universities/Institutes, H2 2017 9

Products under Development by Companies, H2 2017 10

Products under Development by Universities/Institutes, H2 2017 11

Number of Products by Stage and Target, H2 2017 13

Number of Products by Stage and Mechanism of Action, H2 2017 15

Number of Products by Stage and Route of Administration, H2 2017 17

Number of Products by Stage and Molecule Type, H2 2017 19

Hemochromatosis – Pipeline by Ionis Pharmaceuticals Inc, H2 2017 20

Hemochromatosis – Pipeline by La Jolla Pharmaceutical Company, H2 2017 20

Hemochromatosis – Pipeline by Protagonist Therapeutics Inc, H2 2017 21

Hemochromatosis – Pipeline by Silence Therapeutics Plc, H2 2017 21

Hemochromatosis – Pipeline by Vifor Pharma AG, H2 2017 22

Hemochromatosis – Dormant Projects, H2 2017 33

List of Figures

List of Figures

Number of Products under Development for Hemochromatosis, H2 2017 7

Number of Products under Development by Companies, H2 2017 8

Number of Products by Targets, H2 2017 12

Number of Products by Stage and Targets, H2 2017 12

Number of Products by Mechanism of Actions, H2 2017 14

Number of Products by Stage and Mechanism of Actions, H2 2017 14

Number of Products by Routes of Administration, H2 2017 16

Number of Products by Stage and Routes of Administration, H2 2017 16

Number of Products by Molecule Types, H2 2017 18

Number of Products by Stage and Molecule Types, H2 2017 18

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