Epidermolysis Bullosa - Pipeline Review, H2 2016

Published: Oct, 2016 | Pages: 90 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Epidermolysis Bullosa - Pipeline Review, H2 2016, provides an overview of the Epidermolysis Bullosa (Dermatology) pipeline landscape.

Epidermolysis bullosa (EB) is a group of inherited bullous disorders characterized by blister formation in response to mechanical trauma. Symptoms include blistering of skin, deformity or loss of fingernails and toenails, internal blistering, including on the throat, esophagus, upper airway, stomach, intestines and urinary tract, skin thickening on palms and soles of the feet (hyperkeratosis) and scalp blistering, scarring and hair loss (scarring alopecia). Treatment includes antibiotics, antibiotic and surgery.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Epidermolysis Bullosa - Pipeline Review, H2 2016, provides comprehensive information on the therapeutics under development for Epidermolysis Bullosa (Dermatology), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.

The Epidermolysis Bullosa (Dermatology) pipeline guide also reviews of key players involved in therapeutic development for Epidermolysis Bullosa and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Pre-Registration, Phase II, Phase I, Preclinical and Discovery stages are 1, 7, 1, 8 and 4 respectively for Similarly, the Universities portfolio in Phase I and Preclinical stages comprises 1 and 1 molecules, respectively for Epidermolysis Bullosa.

Epidermolysis Bullosa (Dermatology) pipeline guide 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. The guide 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. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

Scope

- The pipeline guide provides a snapshot of the global therapeutic landscape of Epidermolysis Bullosa (Dermatology).
- The pipeline guide reviews pipeline therapeutics for Epidermolysis Bullosa (Dermatology) by companies and universities/research institutes based on information derived from company and industry-specific sources.
- The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.
- The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.
- The pipeline guide reviews key companies involved in Epidermolysis Bullosa (Dermatology) therapeutics and enlists all their major and minor projects.
- The pipeline guide evaluates Epidermolysis Bullosa (Dermatology) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.
- The pipeline guide encapsulates all the dormant and discontinued pipeline projects.
- The pipeline guide reviews latest news related to pipeline therapeutics for Epidermolysis Bullosa (Dermatology)

Reasons To Buy

- Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
- Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.
- Find and recognize significant and varied types of therapeutics under development for Epidermolysis Bullosa (Dermatology).
- Classify potential new clients or partners in the target demographic.
- Develop tactical initiatives by understanding the focus areas of leading companies.
- Plan mergers and acquisitions meritoriously by identifying key players and it's most promising pipeline therapeutics.
- Formulate corrective measures for pipeline projects by understanding Epidermolysis Bullosa (Dermatology) 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.
- Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.

 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

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