Epidermolysis Bullosa - Pipeline Insight, 2024

This “Epidermolysis Bullosa - Pipeline Insight, 2024” report provides comprehensive insights about 20+ companies and 21+ 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, is a group of rare diseases that cause fragile, blistering skin. The blisters may appear in response to minor injury, even from heat, rubbing, scratching or adhesive tape. 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.

Symptoms of Epidermolysis Bullosa includes: skin that blisters easily, blisters inside the mouth, blisters on the hands and soles of the feet, scarred skin, thickened skin and nails, skin that looks thin, tiny pimple-like bumps (milia), dental problems, such as tooth decay, and difficulty swallowing.

The type reflects where on the body the blistering takes place and which layer of skin is affected. There are mainly five types of epidermolysis bullosa:

• Epidermolysis bullosa simplex (EBS): It is the most common type, which tends to be milder with a low risk of serious complications.

Dystrophic epidermolysis bullosa (DEB): This type of EB can range from mild to severe.

Junctional epidermolysis bullosa (JEB): This type of EB is the rarest and is the most severe type.

• Kindler syndrome: This is a mixed condition, since blisters appear across different skin layers. It can also cause patchy changes in your skin coloring when it’s exposed to the sun.

The diagnosis of EB is done through clinical diagnosis and skin biopsy. Confirmation of the diagnosis is done 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.

There is no cure for Epidermolysis Bullosa. The current treatments for Epidermolysis Bullosa is are symptomatic cure. The primary aim is to protect the skin and stop blister formation, promote healing, and prevent complications. Research, including gene therapy and cell-based therapy are in clinical development to improve quality of life.

"Epidermolysis Bullosa - Pipeline Insight, 2024" report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Epidermolysis Bullosa pipeline landscape is provided which includes the disease overview and Epidermolysis Bullosa treatment guidelines. The assessment part of the report embraces, in depth Epidermolysis Bullosa commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Epidermolysis Bullosa collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

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.

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/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

EB-101: Abeona Therapeutics

EB-101 is an autologous, engineered cell therapy for RDEB, a rare connective tissue disorder without an approved treatment in which patients suffer with severe epidermal wounds that impact the length and quality of their lives. People with RDEB have a defect in the COL7A1 gene, leaving them unable to produce Type VII collagen that helps anchor the dermal and epidermal layers of the skin. In November 2024, Abeona Therapeutics' biologics license application (BLA) for prademagene zamikeracel (pz-cel; EB-101), an investigational autologous gene-corrected epidermal sheet therapy for the treatment of patients with recessive dystrophic epidermolysis bullosa (RDEB), has been accepted for filing by the FDA. The agency has set the Prescription Drug User Fee Act (PDUFA) action date for the BLA for April 29, 2025.

INM-755: InMed Pharmaceuticals

INM-755 is a cannabinol (CBN) cream intended as a topical therapy to treat symptoms associated with epidermolysis bullosa (EB) and potentially other dermatological diseases. Preclinical data demonstrate that INM-755 (cannabinol) cream may help relieve hallmark EB symptoms, such as pain, inflammation, wound healing and itch, as well potentially restore the integrity of the skin in a subset of EB Simplex patients. The drug is currently being evaluated under Phase II clinical trial for the treatment of patients with Epidermolysis Bullosa.

BM-3103: BioMendics

The company’s lead product in development, BM-3103, is a patented topical gel. It is currently being studied in a Phase I/IIa trial for epidermolysis bullosa simplex, with additional indications in the pipeline. MTORX technology is an investigational platform encompassing therapeutic candidate, BM-3103.

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. 20+ 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. Registered include, Abeona Therapeutics.

Phases

The report covers around 21+ products under different phases of clinical development like:

• Late stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (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:

• Intravenous
• Subcutaneous
• Oral
• Intramuscular

Molecule Type

Products have been categorized under various Molecule types such as:

• Monoclonal antibody
• Small molecule
• Peptide

Product Type

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 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.

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 Players

• Abeona Therapeutics
• InMed Pharmaceuticals
• BioMendics
• RHEACELL
• Castle Creek Biosciences
• BPGbio
• Eloxx Pharmaceuticals
• Phoenicis Therapeutics
• Aegle Therapeutics
• Amryt Pharma

Key Products

• EB-101
• INM-755
• BM-3103
• APZ 2
• FCX-007
• BP 31510
• ZKN-013
• PTD-003
• AGLE-102
• AP-103

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