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Epidermolysis Bullosa - Pipeline Insight, 2020

  • ID: 4330956
  • Drug Pipelines
  • October 2020
  • Region: Global
  • 90 pages
  • DelveInsight

FEATURED COMPANIES

  • Abeona Therapeutics
  • Aegle Therapeutics
  • Berg Pharma
  • Lenus Therapeutics
  • Onconova Therapeutics
  • Phoenix Tissue Repair
This “Epidermolysis Bullosa - Pipeline Insight, 2020,” report provides comprehensive insights about 30+ companies and 30+ pipeline drugs in Epidermolysis Bullosa pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Geography Covered
  • Global coverage
Epidermolysis Bullosa Understanding

Epidermolysis Bullosa: Overview

Epidermolysis bullosa (EB) is a genetic skin disorder characterized clinically by blister formation from mechanical trauma. There are four main types with additional sub-types identified. There is a spectrum of severity, and within each type, one may be either mildly or severely affected. EB ranges from being a minor inconvenience requiring modification of some activities, to being completely disabling and, in some cases, fatal. Friction causes blister formation. Blisters can form anywhere on the surface of the skin, within the oral cavity and in more severe forms may also involve the external surface of the eye, as well as the respiratory, gastrointestinal and genitourinary tracts. In some forms of the disease, disfiguring scars and disabling musculoskeletal deformities occur. Currently, there is no cure for EB. Supportive care includes daily wound care, bandaging, and pain management as needed.

Symptoms

The main symptoms of all types of EB include:
  • skin that blisters easily
  • blisters inside the mouth
  • blisters on the hands and soles of the feet
  • scarred skin, sometimes with small white spots called milia
  • thickened skin and nails
Diagnosis

Clinical diagnosis of the type of EB based on presentation in the neonatal period should be avoided as all types of EB may look alike in this age group. When EB is suspected, a skin biopsy should be obtained and sent to an appropriate laboratory to confirm the diagnosis with transmission electron microscopy (TEM) and/or immunofluorescent antibody/antigen mapping. Molecular genetic testing for mutations in most of the genes known to be associated with the various types of EB is clinically available.

Treatment

There's currently no cure for EB, so treatment aims to relieve symptoms and prevent complications developing, such as infection.

A team of medical specialists will help the patient decide what treatment is best and offer advice about living with the condition.

One can manage EB at home by:
  • Popping blisters with a sterile needle
  • Applying protective dressings
  • Avoiding things that make the condition worse
Medicines can be used to treat infection or to reduce pain. Surgery may be needed if EB causes narrowing of the food pipe or problems with the hands.

Epidermolysis Bullosa Emerging Drugs Chapters

This segment of the Epidermolysis Bullosa report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Epidermolysis Bullosa Emerging Drugs

Oleogel-S10: Amryt Pharma
Oleogel-S10 (AP101) is Amryt's lead development candidate, a potential treatment for Epidermolysis Bullosa, a rare and distressing genetic skin disorder affecting young children and adults for which there is currently no approved treatments available. Oleogel-S10 is currently in a phase III clinical trial and recently reported positive unblinded interim efficacy and safety analyses.

RGN-137: RegeneRx
RegeneRx has been developing RGN-137, a topical gel formulation of the peptide Tβ4, as a novel treatment to accelerate dermal wound healing. RGN-137 represents a novel approach to dermal wound healing. Unlike growth factors, RGN-137 has been shown in preclinical studies to attenuate multiple healing pathways, such as apoptosis, angiogenesis, collagen deposition, and tissue inflammation. The gene for Tβ4 has also been shown in preclinical studies to be one of the first to be upregulated when an injury occurs, suggesting that the peptide contributes to the healing process. It is currently in phase II stage of development.

BPM31510: RegeneRx
BPM31510 (ubidecarenone) is BERG's leading product candidate. BPM31510 works by correcting cancer cell metabolism, thereby reactivating apoptosis. It is currently in phase I stage of development.

Epidermolysis Bullosa: Therapeutic Assessment

This segment of the report provides insights about the different Epidermolysis Bullosa drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Epidermolysis Bullosa

There are approx. 30+ key companies which are developing the therapies for Epidermolysis Bullosa. The companies which have their Epidermolysis Bullosa drug candidates in the most advanced stage, i.e. phase III include Amryt Pharma and others
Phases

This report covers around 30+ products under different phases of clinical development like
  • Late-stage products (Phase III)
  • Mid-stage products (Phase II)
  • Early-stage products (Phase I) along with the details of
  • Pre-clinical and Discovery stage candidates
  • Discontinued & Inactive candidates
  • Route of Administration
Epidermolysis Bullosa pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as
  • Infusion
  • Intradermal
  • Intramuscular
  • Intravenous
  • Oral
  • Parenteral
  • Subcutaneous
  • Topical.
  • Molecule Type
Products have been categorized under various Molecule types such as
  • Gene therapies
  • Small molecule
  • Vaccines
  • Polymers
  • Peptides
  • Monoclonal antibodies
  • Product Type
The drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Epidermolysis Bullosa: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase III,II, I, preclinical and discovery stage. It also analyses Epidermolysis Bullosa therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Epidermolysis Bullosa drugs.

Report Highlights
  • The companies and academics are working to assess challenges and seek opportunities that could influence Epidermolysis Bullosa R&D. The therapies under development are focused on novel approaches to treat/improve Epidermolysis Bullosa.
  • In September 2019, Amryt Pharma announced that the US Food and Drug Administration (FDA) has designated the investigation of ‘AP101' - Oleogel-S10 - for the treatment of epidermolysis bullosa (EB) as a ‘Fast Track' development programme.
  • RGN-137, also designated an orphan drug in the US, is the Company's Tβ4-based dermal gel formulation that is being developed for epidermolysis bullosa, a rare skin condition.
Epidermolysis Bullosa Report Insights
  • Epidermolysis Bullosa Pipeline Analysis
  • Therapeutic Assessment
  • Unmet Needs
  • Impact of Drugs
Epidermolysis Bullosa Report Assessment
  • Pipeline Product Profiles
  • Therapeutic Assessment
  • Pipeline Assessment
  • Inactive drugs assessment
  • Unmet Needs
Key Questions

Current Treatment Scenario and Emerging Therapies:
  • How many companies are developing Epidermolysis Bullosa drugs?
  • How many Epidermolysis Bullosa drugs are developed by each company?
  • How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Epidermolysis Bullosa?
  • What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Epidermolysis Bullosa therapeutics?
  • What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
  • What are the clinical studies going on for Epidermolysis Bullosa and their status?
  • What are the key designations that have been granted to the emerging drugs?
Key Questions
  • Castle Creek Pharmaceuticals
  • Shire Regenerative Medicine
  • Lenus Therapeutics
  • Aegle Therapeutics
  • Berg Pharma
  • Abeona Therapeutics
  • Onconova Therapeutics
  • Phoenix Tissue Repair
Key Questions
  • diacerein
  • ABH001
  • RGN-137
  • AGLE 102
  • Ubidecarenone
  • Topical beremagene
  • EB-101
  • Rigosertib Sodium
  • PTR-01


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Note: Product cover images may vary from those shown

FEATURED COMPANIES

  • Abeona Therapeutics
  • Aegle Therapeutics
  • Berg Pharma
  • Lenus Therapeutics
  • Onconova Therapeutics
  • Phoenix Tissue Repair
Introduction

Executive Summary

Epidermolysis Bullosa: Overview
  • Causes
  • Mechanism of Action
  • Signs and Symptoms
  • Diagnosis
  • Disease Management
Pipeline Therapeutics
  • Comparative Analysis
Therapeutic Assessment
  • Assessment by Product Type
  • Assessment by Stage and Product Type
  • Assessment by Route of Administration
  • Assessment by Stage and Route of Administration
  • Assessment by Molecule Type
  • Assessment by Stage and Molecule Type
Epidermolysis Bullosa - Analytical Perspective

In-depth Commercial Assessment
  • Epidermolysis Bullosa companies’ collaborations, Licensing, Acquisition -Deal Value Trends
Epidermolysis Bullosa Collaboration Deals
  • Company-Company Collaborations (Licensing / Partnering) Analysis
  • Company-University Collaborations (Licensing / Partnering) Analysis
Late Stage Products (Phase III)
  • Comparative Analysis
Oleogel-S10: Amryt Pharma
  • Product Description
  • Research and Development
  • Product Development Activities
Mid Stage Products (Phase II)
  • Comparative Analysis
RGN-137: RegeneRx
  • Product Description
  • Research and Development
  • Product Development Activities
Early Stage Products (Phase I)
  • Comparative Analysis
BPM31510: RegeneRx
  • Product Description
  • Research and Development
  • Product Development Activities
Pre-clinical and Discovery Stage Products
  • Comparative Analysis
Drug Name: Company Name
  • Product Description
  • Research and Development
  • Product Development Activities
Inactive Products
  • Comparative Analysis
Epidermolysis Bullosa Key Companies

Epidermolysis Bullosa Key Products

Epidermolysis Bullosa- Unmet Needs

Epidermolysis Bullosa- Market Drivers and Barriers

Epidermolysis Bullosa- Future Perspectives and Conclusion

Epidermolysis Bullosa Analyst Views

Epidermolysis Bullosa Key Companies

Appendix

List of Tables
Table 1 Total Products for Epidermolysis Bullosa
Table 2 Late Stage Products
Table 3 Mid Stage Products
Table 4 Early Stage Products
Table 5 Pre-clinical & Discovery Stage Products
Table 6 Assessment by Product Type
Table 7 Assessment by Stage and Product Type
Table 8 Assessment by Route of Administration
Table 9 Assessment by Stage and Route of Administration
Table 10 Assessment by Molecule Type
Table 11 Assessment by Stage and Molecule Type
Table 12 Inactive Products

List of Figures
Figure 1 Total Products for Epidermolysis Bullosa
Figure 2 Late Stage Products
Figure 3 Mid Stage Products
Figure 4 Early Stage Products
Figure 5 Preclinical and Discovery Stage Products
Figure 6 Assessment by Product Type
Figure 7 Assessment by Stage and Product Type
Figure 8 Assessment by Route of Administration
Figure 9 Assessment by Stage and Route of Administration
Figure 10 Assessment by Molecule Type
Figure 11 Assessment by Stage and Molecule Type
Figure 12 Inactive Products
Note: Product cover images may vary from those shown
  • Castle Creek Pharmaceuticals
  • Shire Regenerative Medicine
  • Lenus Therapeutics
  • Aegle Therapeutics
  • Berg Pharma
  • Abeona Therapeutics
  • Onconova Therapeutics
  • Phoenix Tissue Repair
Note: Product cover images may vary from those shown
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