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Epidermolysis Bullosa-Pipeline Review, H1 2015

Epidermolysis Bullosa-Pipeline Review, H1 2015


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Executive Summary

Epidermolysis Bullosa-Pipeline Review, H1 2015

Summary

Global Markets Direct's, 'Epidermolysis Bullosa-Pipeline Review, H1 2015', provides an overview of the Epidermolysis Bullosa's therapeutic pipeline.

This report provides comprehensive information on the therapeutic development for Epidermolysis Bullosa, complete with comparative analysis at various stages, therapeutics assessment by drug target, mechanism of action (MoA), route of administration (RoA) and molecule type, along with latest updates, and featured news and press releases. It also reviews key players involved in the therapeutic development for Epidermolysis Bullosa and special features on late-stage 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, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources, put together by Global Markets Direct's team. Drug profiles/records featured in the report undergoes periodic updation following a stringent set of processes that ensures that all the profiles are updated with the latest set of information. Additionally, processes including live news & deals tracking, browser based alert-box and clinical trials registries tracking ensure that the most recent developments are captured on a real time basis.

The report enhances decision making capabilities and help to create effective counter strategies to gain competitive advantage. It strengthens R&D pipelines by identifying new targets and MOAs to produce first-in-class and best-in-class products.

Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data for the indicated disease.

Scope

The report provides a snapshot of the global therapeutic landscape of Epidermolysis Bullosa

The report reviews key pipeline products under drug profile section which includes, product description, MoA and R&D brief, licensing and collaboration details & other developmental activities

The report reviews key players involved in the therapeutics development for Epidermolysis Bullosa and enlists all their major and minor projects

The report summarizes all the dormant and discontinued pipeline projects

A review of the Epidermolysis Bullosa products under development by companies and universities/research institutes based on information derived from company and industry-specific sources

Pipeline products coverage based on various stages of development ranging from pre-registration till discovery and undisclosed stages

A detailed assessment of monotherapy and combination therapy pipeline projects

Coverage of the Epidermolysis Bullosa pipeline on the basis of target, MoA, route of administration and molecule type

Latest news and deals relating related to pipeline products

Reasons To Buy

Provides strategically significant competitor information, analysis, and insights to formulate effective R&D development strategies

Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage

Develop strategic initiatives by understanding the focus areas of leading companies

Identify and understand important and diverse types of therapeutics under development for Epidermolysis Bullosa

Plan mergers and acquisitions effectively by identifying key players of the most promising pipeline

Devise corrective measures for pipeline projects by understanding Epidermolysis Bullosa 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

Table of Contents

Table of Contents 2

List of Tables 4

List of Figures 5

Introduction 6

Global Markets Direct Report Coverage 6

Epidermolysis Bullosa Overview 7

Therapeutics Development 8

Pipeline Products for Epidermolysis Bullosa-Overview 8

Pipeline Products for Epidermolysis Bullosa-Comparative Analysis 9

Epidermolysis Bullosa-Therapeutics under Development by Companies 10

Epidermolysis Bullosa-Therapeutics under Investigation by Universities/Institutes 11

Epidermolysis Bullosa-Pipeline Products Glance 12

Late Stage Products 12

Clinical Stage Products 13

Early Stage Products 14

Epidermolysis Bullosa-Products under Development by Companies 15

Epidermolysis Bullosa-Products under Investigation by Universities/Institutes 16

Epidermolysis Bullosa-Companies Involved in Therapeutics Development 17

Fibrocell Science, Inc. 17

GlaxoSmithKline plc 18

InMed Pharmaceuticals Inc. 19

RegeneRx Biopharmaceuticals, Inc. 20

Scioderm, Inc. 21

Shire Plc 22

TWi Pharmaceuticals, Inc. 23

Epidermolysis Bullosa-Therapeutics Assessment 24

Assessment by Monotherapy Products 24

Assessment by Target 25

Assessment by Mechanism of Action 27

Assessment by Route of Administration 29

Assessment by Molecule Type 31

Drug Profiles 33

diacerein-Drug Profile 33

Product Description 33

Mechanism of Action 33

R&D Progress 33

Gene Therapy for Epidermolysis Bullosa-Drug Profile 34

Product Description 34

Mechanism of Action 34

R&D Progress 34

Gene Therapy for Epidermolysis Bullosa-Drug Profile 35

Product Description 35

Mechanism of Action 35

R&D Progress 35

Gene Therapy to Activate Collagen Type VII for Recessive Dystrophic Epidermolysis Bullosa-Drug Profile 36

Product Description 36

Mechanism of Action 36

R&D Progress 36

GMHDFCOL-7-Drug Profile 37

Product Description 37

Mechanism of Action 37

R&D Progress 37

ICX-RHY-Drug Profile 38

Product Description 38

Mechanism of Action 38

R&D Progress 38

INM-750-Drug Profile 40

Product Description 40

Mechanism of Action 40

R&D Progress 40

RGN-137-Drug Profile 41

Product Description 41

Mechanism of Action 41

R&D Progress 41

SD-101-Drug Profile 42

Product Description 42

Mechanism of Action 42

R&D Progress 42

SHP-608-Drug Profile 43

Product Description 43

Mechanism of Action 43

R&D Progress 43

Epidermolysis Bullosa-Recent Pipeline Updates 44

Epidermolysis Bullosa-Dormant Projects 49

Epidermolysis Bullosa-Discontinued Products 50

Epidermolysis Bullosa-Product Development Milestones 51

Featured News & Press Releases 51

Mar 19, 2015: Scioderm Initiates Phase 3 Registration Trial of Zorblisa (SD-101), a Novel Topical Therapy for Patients with Epidermolysis Bullosa 51

Mar 10, 2015: TWi Biotechnology Receives Rare Disease Drug Designation for its Drug Candidate AC-203 in Taiwan 51

Feb 03, 2015: InMed Expands Pipeline with Initiation of Program Targeting Epidermolysis bullosa simplex 52

Jan 02, 2015: Scioderm to Present at 33rd Annual J.P. Morgan Healthcare Conference 52

Oct 21, 2014: TWi Biotechnology Receives Orphan Drug Designation for its Lead Drug Candidate AC-201 In United States 53

Oct 06, 2014: Scioderm to Present Data from Phase 2b Study of Zorblisa (SD-101) at the 13th Annual BIO Investor Forum 53

Sep 09, 2014: Scioderm to Present Data from Phase 2b Study of Zorblisa (SD-101) at the debra International Congress 53

Jul 23, 2014: Fibrocell Science Takes Active Role to Support Dystrophic Epidermolysis Bullosa Patient Advocacy Group 53

Jun 13, 2014: Fibrocell Science Receives Orphan Drug Designation for Genetically-Modified Autologous Human Fibroblasts to Advance Treatment Development for Recessive Dystrophic Epidermolysis Bullosa (RDEB) 54

May 28, 2014: Fibrocell Science Announces Plans for Process Development and Manufacturing of Genetically-modified Cell Therapy for Rare Disease, RDEB 55

Appendix 56

Methodology 56

Coverage 56

Secondary Research 56

Primary Research 56

Expert Panel Validation 56

Contact Us 56

Disclaimer 57

List of Tables

Number of Products under Development for Epidermolysis Bullosa, H1 2015 8

Number of Products under Development for Epidermolysis Bullosa-Comparative Analysis, H1 2015 9

Number of Products under Development by Companies, H1 2015 10

Number of Products under Investigation by Universities/Institutes, H1 2015 11

Comparative Analysis by Late Stage Development, H1 2015 12

Comparative Analysis by Clinical Stage Development, H1 2015 13

Comparative Analysis by Early Stage Development, H1 2015 14

Products under Development by Companies, H1 2015 15

Products under Investigation by Universities/Institutes, H1 2015 16

Epidermolysis Bullosa-Pipeline by Fibrocell Science, Inc., H1 2015 17

Epidermolysis Bullosa-Pipeline by GlaxoSmithKline plc, H1 2015 18

Epidermolysis Bullosa-Pipeline by InMed Pharmaceuticals Inc., H1 2015 19

Epidermolysis Bullosa-Pipeline by RegeneRx Biopharmaceuticals, Inc., H1 2015 20

Epidermolysis Bullosa-Pipeline by Scioderm, Inc., H1 2015 21

Epidermolysis Bullosa-Pipeline by Shire Plc, H1 2015 22

Epidermolysis Bullosa-Pipeline by TWi Pharmaceuticals, Inc., H1 2015 23

Assessment by Monotherapy Products, H1 2015 24

Number of Products by Stage and Target, H1 2015 26

Number of Products by Stage and Mechanism of Action, H1 2015 28

Number of Products by Stage and Route of Administration, H1 2015 30

Number of Products by Stage and Molecule Type, H1 2015 32

Epidermolysis Bullosa Therapeutics-Recent Pipeline Updates, H1 2015 44

Epidermolysis Bullosa-Dormant Projects, H1 2015 49

Epidermolysis Bullosa-Discontinued Products, H1 2015 50

List of Figures

Number of Products under Development for Epidermolysis Bullosa, H1 2015 8

Number of Products under Development for Epidermolysis Bullosa-Comparative Analysis, H1 2015 9

Number of Products under Development by Companies, H1 2015 10

Comparative Analysis by Early Stage Products, H1 2015 14

Assessment by Monotherapy Products, H1 2015 24

Number of Products by Top 10 Targets, H1 2015 25

Number of Products by Stage and Top 10 Targets, H1 2015 26

Number of Products by Top 10 Mechanism of Actions, H1 2015 27

Number of Products by Stage and Top 10 Mechanism of Actions, H1 2015 28

Number of Products by Top 10 Routes of Administration, H1 2015 29

Number of Products by Stage and Top 10 Routes of Administration, H1 2015 30

Number of Products by Top 10 Molecule Types, H1 2015 31

Number of Products by Stage and Top 10 Molecule Types, H1 2015 32

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Fibrocell Science, Inc.

GlaxoSmithKline plc

InMed Pharmaceuticals Inc.

RegeneRx Biopharmaceuticals, Inc.

Scioderm, Inc.

Shire Plc

TWi Pharmaceuticals, Inc.

Epidermolysis Bullosa Therapeutic Products under Development, Key Players in Epidermolysis Bullosa Therapeutics, Epidermolysis Bullosa Pipeline Overview, Epidermolysis Bullosa Pipeline, Epidermolysis Bullosa Pipeline Assessment


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