Epidermolysis Bullosa – Pipeline Review, H2 2016

Global Markets Direct’s, ‘Epidermolysis Bullosa – Pipeline Review, H2 2016’, provides an overview of the Epidermolysis Bullosa pipeline landscape.

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

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

The report assesses Epidermolysis Bullosa 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 Epidermolysis Bullosa

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 Epidermolysis Bullosa

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 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

Companies mentioned

Abeona Therapeutics, Inc.

Amryt Pharma plc

Berg LLC

Fibrocell Science, Inc.

GlaxoSmithKline Plc

InMed Pharmaceuticals Inc.

Karus Therapeutics Limited

ProQR Therapeutics N.V.

RegeneRx Biopharmaceuticals, Inc.

Scioderm, Inc.

Stratatech Corp

TWi Pharmaceuticals, Inc.

WAVE Life Sciences Ltd.

Table of Contents

Table of Contents

Table of Contents 2

List of Tables 5

List of Figures 6

Introduction 7

Global Markets Direct Report Coverage 7

Epidermolysis Bullosa Overview 8

Therapeutics Development 9

Pipeline Products for Epidermolysis Bullosa - Overview 9

Pipeline Products for Epidermolysis Bullosa - Comparative Analysis 10

Epidermolysis Bullosa - Therapeutics under Development by Companies 11

Epidermolysis Bullosa - Therapeutics under Investigation by Universities/Institutes 13

Epidermolysis Bullosa - Pipeline Products Glance 14

Late Stage Products 14

Clinical Stage Products 15

Early Stage Products 16

Epidermolysis Bullosa - Products under Development by Companies 17

Epidermolysis Bullosa - Products under Investigation by Universities/Institutes 18

Epidermolysis Bullosa - Companies Involved in Therapeutics Development 19

Abeona Therapeutics, Inc. 19

Amryt Pharma plc 20

Berg LLC 21

Fibrocell Science, Inc. 22

GlaxoSmithKline Plc 23

InMed Pharmaceuticals Inc. 24

Karus Therapeutics Limited 25

ProQR Therapeutics N.V. 26

RegeneRx Biopharmaceuticals, Inc. 27

Scioderm, Inc. 28

Stratatech Corp 29

TWi Pharmaceuticals, Inc. 30

WAVE Life Sciences Ltd. 31

Epidermolysis Bullosa - Therapeutics Assessment 32

Assessment by Monotherapy Products 32

Assessment by Target 33

Assessment by Mechanism of Action 35

Assessment by Route of Administration 37

Assessment by Molecule Type 39

Drug Profiles 41

allantoin - Drug Profile 41

Product Description 41

Mechanism Of Action 41

R&D Progress 41

ALLO-ASC - Drug Profile 44

Product Description 44

Mechanism Of Action 44

R&D Progress 44

Antisense Oligonucleotide 1 to Inhibit Keratin for Epidermolysis Bullosa Simplex - Drug Profile 45

Product Description 45

Mechanism Of Action 45

R&D Progress 45

Antisense Oligonucleotide 2 to Inhibit Keratin for Epidermolysis Bullosa Simplex - Drug Profile 46

Product Description 46

Mechanism Of Action 46

R&D Progress 46

Biologic for Epidermolysis Bullosa - Drug Profile 47

Product Description 47

Mechanism Of Action 47

R&D Progress 47

birch bark extract - Drug Profile 48

Product Description 48

Mechanism Of Action 48

R&D Progress 48

Cell Therapy for Dermatology and Immunology - Drug Profile 50

Product Description 50

Mechanism Of Action 50

R&D Progress 50

Cell Therapy for Epidermolysis Bullosa - Drug Profile 52

Product Description 52

Mechanism Of Action 52

R&D Progress 52

Cell Therapy for Recessive Dystrophic Epidermolysis Bullosa - Drug Profile 53

Product Description 53

Mechanism Of Action 53

R&D Progress 53

diacerein - Drug Profile 54

Product Description 54

Mechanism Of Action 54

R&D Progress 54

EB-101 - Drug Profile 57

Product Description 57

Mechanism Of Action 57

R&D Progress 57

EB-201 - Drug Profile 58

Product Description 58

Mechanism Of Action 58

R&D Progress 58

FCX-007 - Drug Profile 59

Product Description 59

Mechanism Of Action 59

R&D Progress 59

Gene Therapy for Epidermolysis Bullosa - Drug Profile 62

Product Description 62

Mechanism Of Action 62

R&D Progress 62

Gene Therapy for Epidermolysis Bullosa - Drug Profile 63

Product Description 63

Mechanism Of Action 63

R&D Progress 63

Gene Therapy to Activate Lysyl Hydroxylase-3 for Recessive Dystrophic Epidermolysis Bullosa - Drug Profile 64

Product Description 64

Mechanism Of Action 64

R&D Progress 64

ICX-RHY - Drug Profile 65

Product Description 65

Mechanism Of Action 65

R&D Progress 65

INM-750 - Drug Profile 67

Product Description 67

Mechanism Of Action 67

R&D Progress 67

KA-1463 - Drug Profile 68

Product Description 68

Mechanism Of Action 68

R&D Progress 68

QR-313 - Drug Profile 69

Product Description 69

Mechanism Of Action 69

R&D Progress 69

RGN-137 - Drug Profile 70

Product Description 70

Mechanism Of Action 70

R&D Progress 70

TXA-127 - Drug Profile 72

Product Description 72

Mechanism Of Action 72

R&D Progress 72

ubidecarenone - Drug Profile 75

Product Description 75

Mechanism Of Action 75

R&D Progress 75

Epidermolysis Bullosa - Dormant Projects 77

Epidermolysis Bullosa - Discontinued Products 78

Epidermolysis Bullosa - Product Development Milestones 79

Featured News & Press Releases 79

Oct 01, 2016: Castle Creek Pharmaceuticals Announces Late-Breaking Results at EADV of Phase 2 Topical Diacerein 1% Data in Patients with Epidermolysis Bullosa Simplex 79

Sep 26, 2016: Abeona Therapeutics Enrolls First Patient in Phase 2 for EB-101 Gene Therapy Clinical Trial for Epidermolysis Bullosa 80

Sep 19, 2016: Fibrocell Completes Enrollment in NC1+ Cohort of Phase I/II Clinical Trial of FCX-007 for Treatment of Recessive Dystrophic Epidermolysis Bullosa 80

Sep 13, 2016: Amryt Pharma announces US patent for Lead Drug, Episalvan 81

Sep 08, 2016: Abeona Therapeutics Enrolls 5th Patient in Phase 1/2 Gene Therapy Clinical Trial for Epidermolysis Bullosa 81

Sep 07, 2016: Tarix Orphan Receives Orphan Drug Status for TXA127 for Genetic Skin Disorder Recessive Dystrophic Epidermolysis Bullosa 82

Jul 27, 2016: Fibrocell Announces First Subjects Enrolled in Phase I/II Clinical Trial of FCX-007 for Treatment of Recessive Dystrophic Epidermolysis Bullosa 83

Jun 29, 2016: Fibrocell Announces Update on FCX-007 83

Jun 27, 2016: Tarix Orphan Announces Positive Results with TXA127 in Animal Model of Dystrophic Epidermolysis Bullosa 83

Jun 13, 2016: Fibrocell Announces Initiation of Patient Recruitment in Phase I/II Clinical Trial of FCX-007 for Treatment of Recessive Dystrophic Epidermolysis Bullosa 84

Apr 18, 2016: Fibrocell and Intrexon Announce Allowance to Commence Phase I/II Trial for FCX-007 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa 85

Dec 04, 2015: RegeneRx Receives Chinese Patent for Key TB4 Peptide Fragment 85

Oct 08, 2015: Fibrocell and Intrexon Announce Poster Presentation on FCX-007 for the Treatment of RDEB at American Society of Human Genetics Annual Meeting 86

Sep 25, 2015: Fibrocell and Intrexon Provide Regulatory Update for FCX-007 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa 86

Jul 22, 2015: RegeneRx Receives Notice of Acceptance for Australian Patent for Key TB4 Peptide Fragment 87

Appendix 88

Methodology 88

Coverage 88

Secondary Research 88

Primary Research 88

Expert Panel Validation 88

Contact Us 88

Disclaimer 89

List of Tables

List of Tables

Number of Products under Development for Epidermolysis Bullosa, H2 2016 9

Number of Products under Development for Epidermolysis Bullosa – Comparative Analysis, H2 2016 10

Number of Products under Development by Companies, H2 2016 12

Number of Products under Investigation by Universities/Institutes, H2 2016 13

Comparative Analysis by Late Stage Development, H2 2016 14

Comparative Analysis by Clinical Stage Development, H2 2016 15

Comparative Analysis by Early Stage Development, H2 2016 16

Products under Development by Companies, H2 2016 17

Products under Investigation by Universities/Institutes, H2 2016 18

Epidermolysis Bullosa – Pipeline by Abeona Therapeutics, Inc. , H2 2016 19

Epidermolysis Bullosa – Pipeline by Amryt Pharma plc, H2 2016 20

Epidermolysis Bullosa – Pipeline by Berg LLC, H2 2016 21

Epidermolysis Bullosa – Pipeline by Fibrocell Science, Inc., H2 2016 22

Epidermolysis Bullosa – Pipeline by GlaxoSmithKline Plc, H2 2016 23

Epidermolysis Bullosa – Pipeline by InMed Pharmaceuticals Inc., H2 2016 24

Epidermolysis Bullosa – Pipeline by Karus Therapeutics Limited, H2 2016 25

Epidermolysis Bullosa – Pipeline by ProQR Therapeutics N.V., H2 2016 26

Epidermolysis Bullosa – Pipeline by RegeneRx Biopharmaceuticals, Inc., H2 2016 27

Epidermolysis Bullosa – Pipeline by Scioderm, Inc., H2 2016 28

Epidermolysis Bullosa – Pipeline by Stratatech Corp, H2 2016 29

Epidermolysis Bullosa – Pipeline by TWi Pharmaceuticals, Inc., H2 2016 30

Epidermolysis Bullosa – Pipeline by WAVE Life Sciences Ltd., H2 2016 31

Assessment by Monotherapy Products, H2 2016 32

Number of Products by Stage and Target, H2 2016 34

Number of Products by Stage and Mechanism of Action, H2 2016 36

Number of Products by Stage and Route of Administration, H2 2016 38

Number of Products by Stage and Molecule Type, H2 2016 40

Epidermolysis Bullosa – Dormant Projects, H2 2016 77

Epidermolysis Bullosa – Discontinued Products, H2 2016 78

List of Figures

List of Figures

Number of Products under Development for Epidermolysis Bullosa, H2 2016 9

Number of Products under Development for Epidermolysis Bullosa – Comparative Analysis, H2 2016 10

Number of Products under Development by Companies, H2 2016 11

Number of Products under Investigation by Universities/Institutes, H2 2016 13

Comparative Analysis by Clinical Stage Development, H2 2016 15

Comparative Analysis by Early Stage Products, H2 2016 16

Assessment by Monotherapy Products, H2 2016 32

Number of Products by Targets, H2 2016 33

Number of Products by Stage and Targets, H2 2016 33

Number of Products by Mechanism of Actions, H2 2016 35

Number of Products by Stage and Mechanism of Actions, H2 2016 35

Number of Products by Routes of Administration, H2 2016 37

Number of Products by Stage and Routes of Administration, H2 2016 37

Number of Products by Molecule Types, H2 2016 39

Number of Products by Stage and Molecule Types, H2 2016 39

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