Pemphigus Vulgaris - Pipeline Insight, 2024

This “Pemphigus Vulgaris (PV) - Pipeline Insight, 2024,” report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in Pemphigus Vulgaris 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

Pemphigus Vulgaris Understanding

Pemphigus Vulgaris (PV): Overview

Pemphigus vulgaris is an autoimmune disorder that involves blistering and erosion of the skin and mucous membranes. It occurs almost exclusively in middle-aged or older people. The primary lesion of pemphigus vulgaris is a soft blister filled with clear fluid that appears on healthy or irritated skin. Many cases begin with blisters in the mouth, followed by skin blisters that may come and go. The blisters inside the mouth can make it hard for the person to eat. The rupture of blisters on the skin may be painful and limit the person's daily activities. Complications due to infections can be serious and the damaging nature of the blisters can cause loss of body fluids and protein. The exact cause of pemphigus vulgaris is unknown, but the blisters in pemphigus vulgaris are associated with the binding of antibodies to the skin cells.

Symptoms
The major symptom of pemphigus vulgaris is the development of clear, soft, and painful (sometimes tender) blisters of various sizes. In addition, the top layer of skin may detach from the lower layers in response to slight pinching or rubbing, causing it to peel off in sheets and to leave painful areas of open skin (erosions). Blisters can form on the skin and rupture, leaving raw, painful, crusted wounds. The person feels generally ill.

Diagnosis
Doctors usually recognize pemphigus vulgaris by its characteristic blisters, but the disorder is diagnosed with certainty by examining a sample of skin under a microscope (skin biopsy). Sometimes doctors use special chemical stains that allow antibody deposits to be seen under the microscope (called an immunofluorescence assay).

Treatment

• Corticosteroids (oral or intravenous)
• Antibiotics and protective dressings for ruptured blisters
• Often immunosuppressants or immune globulin to reduce corticosteroid use

High doses of corticosteroids are the mainstay of treatment. They are taken by mouth (orally) or, if the person is hospitalized, may be given by vein (intravenously). If the disease is controlled, the dose of corticosteroids is gradually reduced (tapered).

Pemphigus Vulgaris Emerging Drugs Chapters

This segment of the Pemphigus Vulgaris report encloses its detailed analysis of various drugs in different stages of clinical development, including phase 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.

Pemphigus Vulgaris Emerging Drugs

Efgartigimod: argenx Efgartigimod (ARGX-113) is an investigational therapy for IgG-mediated autoimmune diseases and was designed to exploit the natural interaction between IgG antibodies and the recycling receptor FcRn. Efgartigimod is the Fc-portion of an antibody that has been modified by the argenx proprietary ABDEG™ technology to increase its affinity for FcRn beyond that of normal IgG antibodies. As a result, efgartigimod blocks antibody recycling through FcRn binding and leads to fast depletion of the autoimmune disease-causing IgG autoantibodies. The development work on efgartigimod is conducted in close collaboration with Prof. E. Sally Ward (University of Texas Southwestern Medical and Texas A&M University Health Science Center, a part of Texas A&M University(TAMHSC)).

Efgartigimod is being evaluated for the treatment of patients with Pemphigus Vulgaris in an open-label, non-controlled, phase II study. The proposed study is an open-label, non-controlled, adaptive-design Phase II study to evaluate the safety, pharmacodynamics, pharmacokinetics, efficacy, and conditions of use (dosage, frequency of administration at maintenance) of ARGX-113 in patients with mild to moderate Pemphigus (Vulgaris or Foliaceus), either newly diagnosed or relapsing.

Pemphigus Vulgaris: Therapeutic Assessment

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

Major Players in Pemphigus Vulgaris

There are approx. 5+ key companies which are developing the therapies for Pemphigus Vulgaris. The companies which have their Pemphigus Vulgaris drug candidates in the mid stage, i.e. Phase II include, argenx etc.

Phases

This report covers around 5+ products under different phases of clinical development like

• Mid-stage products (Phase II and Phase I/II)
• Early-stage products (Phase I/II and Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

Route of Administration

Pemphigus Vulgaris 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
• Intramuscular

Molecule Type

Products have been categorized under various Molecule types such as

• Monoclonal antibodies
• T lymphocyte cell therapies
• Gene therapies

Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Pemphigus Vulgaris: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Pemphigus Vulgaris 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 Pemphigus Vulgaris drugs.

Report Highlights

The companies and academics are working to assess challenges and seek opportunities that could influence Pemphigus Vulgaris R&D. The therapies under development are focused on novel approaches to treat/improve Pemphigus Vulgaris.

May 2020: Argenx reported interim data from first cohort of Phase 2 proof-of-concept clinical trial of efgartigimod for the treatment of pemphigus vulgaris.

Pemphigus Vulgaris Report Insights

• Pemphigus Vulgaris Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Pemphigus Vulgaris 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 Pemphigus Vulgaris drugs?
• How many Pemphigus Vulgaris drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Pemphigus Vulgaris?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Pemphigus Vulgaris 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 Pemphigus Vulgaris and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• argenx BVBA
• Cabaletta Bio

Key Products

• Efgartigimod
• DSG3 CAART