Scleroderma - Pipeline Insight, 2024

This “Scleroderma - Pipeline Insight, 2024” report provides comprehensive insights about 45+ companies and 50+ pipeline drugs in Scleroderma 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

Scleroderma: Understanding

Scleroderma: Overview

Scleroderma is a connective tissue disorder characterized primarily by the thickening and hardening of the skin. The combining form “sclero” means "hard" in Greek, and the word “dermis” means skin. There are two primary types of scleroderma: localized and systemic (also called systemic sclerosis). In localized scleroderma, the disease affects mainly the skin and may have an impact on the muscles and bones. In systemic scleroderma, there is an involvement of the internal organs, such as the digestive tract, heart, lungs, and kidneys, among others. The severity and outcome of scleroderma are variable. Three primary mechanisms contribute to the development of the scleroderma: vascular anomalies, excess fibrosis, and autoimmune phenomenon. Abnormal interactions between endothelial cells, fibroblasts, and lymphocytes (B and T) lead to microcirculatory vascular involvement. The endothelial cells produce large amounts of endothelin 1, causing vasoconstriction and fibroblast activation. Also, fibroblasts and activated endothelial cells produce reactive oxygen species that speed up vascular remodeling, leading to the obliteration of small vessels. Activated fibroblasts differentiate easily into myofibroblasts, which have increased collagen synthesis ability.

Scleroderma is diagnosed through a clinical evaluation that includes a thorough examination, medical history review, and physical tests like skin biopsy and blood tests to assess organ involvement Common diagnostic procedures include nailfold capillaroscopy, ANA test, pulmonary function tests, CT scans, EKGs, echocardiograms, X-rays, and motility studies. The diagnosis is often made by dermatologists or rheumatologists who specialize in skin and autoimmune disorders. Treatment for scleroderma focuses on managing symptoms and complications, involving medications to suppress the immune system, physical therapy, pain relief, skin care, and emotional support early intervention is crucial for effective treatment and improving quality of life in scleroderma patients.

Scleroderma treatment aims to manage symptoms and prevent complications since there is currently no cure for the condition. Treatment strategies are tailored to individual needs, considering factors like symptoms, type of scleroderma, age, and overall health. Common treatment approaches include pain relief using nonsteroidal anti-inflammatory medications or corticosteroids, managing skin itchiness with lotions and moisturizers, and slowing skin thickening with immune system-suppressing medications. Additionally, physical therapy helps maintain muscle strength, while medications control blood pressure, improve blood flow, and address specific symptoms like heartburn and Raynaud's phenomenon. Surgery may be necessary in cases where complications cannot be resolved with less invasive therapies. Regular monitoring and early intervention are key to improving quality of life for individuals with scleroderma.

"Scleroderma- Pipeline Insight, 2024" report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Scleroderma pipeline landscape is provided which includes the disease overview and Scleroderma treatment guidelines. The assessment part of the report embraces, in depth Scleroderma 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, Scleroderma 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 Scleroderma R&D. The therapies under development are focused on novel approaches to treat/improve Scleroderma.

Scleroderma Emerging Drugs Chapters

This segment of the Scleroderma 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.

Scleroderma Emerging Drugs

MT-0551: Mitsubishi Tanabe Pharma Corporation

UPLIZNA (inebilizumab) is a humanized, affinity-optimized, and afucosylated IgG1 kappa (IgG1κ) monoclonal antibody developed by Mitsubishi Tanabe Pharma Corporation (MTPC). It binds to the B cell-specific surface antigen CD19, which is expressed on B cells including antibody-producing plasmablasts and plasma cells. Currently, the drug is in Phase III stage of its clinical trial for the treatment of Systemic Sclerosis.

FT011: Certa Therapeutics

FT011 is a novel drug that inhibits this GPCR, offering the potential to treat chronic fibrosis in multiple organs. Breakthrough research conducted by Certa Therapeutics has identified a novel biologic target in the fibrosis mechanism as a defined G protein-coupled receptor (GPCR). This GPCR is silent in healthy tissue but activates following injury or disease. Evidence demonstrates the role this GPCR has in multiple downstream pathways causing inflammation and fibrosis. Currently, the drug is in the Phase II stage of its development for the treatment of scleroderma.

AM1476: AnaMar AB

AM1476, an oral, small molecule, selective 5-HT2B receptor antagonist, offers a unique approach to treating systemic sclerosis by modulating key fibrotic pathways like TGF-β and Wnt. Acting as an antagonist, AM1476 interferes with serotonin (5-HT) pathways, particularly affecting molecules downstream like TGF-β only when 5-HT is active, thus minimizing adverse events. Demonstrating anti-fibrotic efficacy in preclinical models, AM1476 has shown the ability to halt and reverse fibrosis progression in skin and lung tissues, along with ameliorating processes related to fibroblast and macrophage activation. AM1476 has been granted orphan drug designation for treatment of systemic sclerosis in both the US and EU. Currently, the drug is in Phase I stage of its clinical trial for the treatment of Systemic Sclerosis.

Scleroderma: Therapeutic Assessment

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

Major Players in Scleroderma

• There are approx. 45+ key companies which are developing the therapies for Scleroderma. The companies which have their Scleroderma drug candidates in the most advanced stage, i.e. Phase III include, GlaxoSmithKline.

Phases

DelveInsight’s report covers around 50+ 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

Scleroderma pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Oral
• Intravenous
• Subcutaneous
• Parenteral
• Topical
• Molecule Type

Products have been categorized under various Molecule types such as

• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy
• Product Type

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

Scleroderma: Pipeline Development Activities

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

Scleroderma Report Insights

• Scleroderma Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• Genentech
• Maruho
• Mitsubishi Tanabe Pharma America
• GlaxoSmithKline
• Gesynta Pharma
• Certa Therapeutics
• ChemomAb
• Boehringer Ingelheim
• Bioray Laboratories
• Cabaletta Bio
• AnaMar AB

Key Products

• Vixarelimab
• Nemolizumab
• MT-7117
• Belimumab
• GS-248
• FT011
• CM-101
• Avenciguat
• BRL-301
• CABA 201
• AM1476

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