Scleroderma - Pipeline Insight, 2025

This “Scleroderma - Pipeline Insight, 2025” report provides comprehensive insights about 40+ 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

Scleroderma: Understanding

Scleroderma: Overview

Scleroderma, also known as systemic sclerosis, is a rare and complex autoimmune connective tissue disease characterized by the progressive fibrosis and hardening of the skin and internal organs due to excessive collagen production. The pathogenesis of scleroderma involves a combination of immune dysregulation, abnormal vascular function, and fibrosis, leading to tissue damage and compromised organ function. The condition commonly presents with hallmark features such as skin thickening, Raynaud's phenomenon (reduced blood flow to extremities triggered by cold or stress), and involvement of multiple organ systems, including the lungs, heart, kidneys, and gastrointestinal tract. There are two main forms of scleroderma: limited cutaneous systemic sclerosis (lcSSc), which primarily affects the skin distal to the elbows and knees, and diffuse cutaneous systemic sclerosis (dcSSc), which involves more widespread skin thickening and is often associated with more severe internal organ complications, including pulmonary fibrosis and heart issues.

The etiology of scleroderma, or systemic sclerosis, is complex and not yet fully understood, although it is believed to result from a combination of genetic and environmental factors. Genetic predisposition plays a significant role, with certain human leukocyte antigen (HLA) alleles, such as DRB11104 and DQA10501, as well as non-HLA genes like PTPN22 and STAT4, being associated with an increased risk of developing the disease. These genetic factors contribute to the immune dysregulation seen in scleroderma, leading to the body's immune system attacking its own tissues. Environmental factors are also considered important in triggering or exacerbating the condition. Exposure to infectious agents, such as cytomegalovirus and Epstein-Barr virus, as well as chemicals like silica dust and organic solvents, have been linked to the onset of scleroderma. Hormonal influences may also play a role, with the disease being more common in women, suggesting a possible connection with sex hormones.

The clinical presentation of scleroderma, or systemic sclerosis, can vary widely among individuals and depends on the extent of skin and organ involvement. One of the primary features is skin thickening and hardening, which is classified into two main forms: limited cutaneous systemic sclerosis and diffuse cutaneous systemic sclerosis. In limited cutaneous systemic sclerosis, skin changes are typically confined to the fingers, hands, and face, with the progression of thickening being relatively slow. In contrast, diffuse cutaneous systemic sclerosis involves more widespread skin thickening that can extend to the trunk and upper limbs, and may progress more rapidly. A hallmark symptom of scleroderma is Raynaud's phenomenon, a condition where blood flow to the extremities, particularly the fingers and toes, is impaired in response to cold or stress, leading to color changes, numbness, and pain. Additional common manifestations include the appearance of small, dilated blood vessels on the skin, the development of calcium deposits under the skin, and gastrointestinal issues such as difficulty swallowing and acid reflux, often due to involvement of the esophagus.

The treatment of scleroderma, or systemic sclerosis, is personalized based on the individual's symptoms and the extent of organ involvement, as there is currently no cure for the disease. The primary focus of treatment is to alleviate symptoms, prevent complications, and improve the patient's quality of life. Immunosuppressive therapies, such as mycophenolate mofetil, methotrexate, and cyclophosphamide, are commonly used to address skin thickening and internal organ complications. For patients with early diffuse cutaneous scleroderma, autologous hematopoietic stem cell transplantation may be considered, particularly in those at high risk of mortality. Pulmonary complications, including interstitial lung disease and pulmonary hypertension, require specific management strategies. Mycophenolate mofetil is often the first-line treatment for interstitial lung disease, while pulmonary hypertension is typically treated with vasodilators, such as bosentan or sildenafil, to improve blood flow and reduce pressure in the lungs.

"Scleroderma- Pipeline Insight, 2025" 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 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.

Scleroderma Emerging Drugs

Inebilizumab: Mitsubishi Tanabe Pharma Corporation

Inebilizumab, also callled MEDI-551 is a humanized, anti-CD19 monoclonal antibody that binds to CD19, leading to antibody-dependent, cell-mediated cytotoxicity of B cells. CD19 is involved in B-cell signaling, intervening in the balance between humoral, antigen-induced response and tolerance induction. Currently, the drug is in Phase III stage of its clinical trial for the treatment of Scleroderma.

• HZN-825: Amgen

HZN-825 (Fipaxalparant) is a molecule that blocks lysophosphatidic acid receptor 1 (LPAR1). It is being investigated for the treatment of diffuse cutaneous systemic sclerosis. It is a small molecule acting as a negative allosteric modulator of LPAR1. Currently, the drug is in Phase II stage of its clinical trial for the treatment of Scleroderma.

MT-7117: Mitsubishi Tanabe Pharma America Inc.

Dersimelagon (MT-7117), a novel synthetic, oral, selective melanocortin-1 receptor agonist, demonstrated disease-modifying effects in preclinical models of systemic sclerosis (SSc). MT-7117 reduces inflammation, vascular dysfunction, and fibrosis through its pleiotropic effects on inflammatory cells, endothelial cells, and fibroblastsis, and is as a potential therapeutic agent for the treatment of SSc. Currently, the drug is in the Phase II stage of development to treat Scleroderma.

• RO7303509: Genentech, Inc.

RG-6315 (RO-7303509) is under development for the treatment of inflammation, fibrosis and systemic sclerosis. The drug candidate is administered through subcutaneous or intravenous routes. It is a new molecular entity (NME). Currently, the drug is in the Phase I stage of development to treat Scleroderma.

Soquelitinib: Corvus Pharmaceuticals

Soquelitinib (formerly CPI-818) is an investigational small molecule drug given orally designed to selectively inhibit ITK (interleukin-2-inducible T cell kinase), an enzyme that is expressed predominantly in T cells and plays a role in T cell and natural killer (NK) cell immune function.. Currently, the drug is in the Preclinical stage of development to treat Scleroderma.

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. 40+ 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, Mitsubishi Tanabe Pharma Corporation.

Phases

The 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

• AstraZeneca
• Mitsubishi Tanabe Pharma Corporation
• GlaxoSmithKline
• Boehringer Ingelheim
• Novartis Pharmaceuticals
• Certa Therapeutics
• Argenx
• Cumberland Pharmaceuticals
• Sanofi
• Amgen
• Genentech, Inc.
• Cabaletta Bio
• Kyverna Therapeutics
• ChemomAb Ltd.
• Corvus Pharmaceuticals
• Quoin Pharmaceuticals Ltd.
• aTyr Pharma
• APIE Therapeutics
• 4P Pharma
• Rubedo Life Sciences, Inc.
• Nobelpharma Co., Ltd.
• The Scripps Research Institute

Key Products

• Anifrolumab
• Inebilizumab
• Belimumab
• Avenciguat
• Rapcabtagene autoleucel
• FT011
• Efgartigimod PH20 SC
• Ifetroban
• Amlitelimab
• MT-7117
• HZN-825
• Vixarelimab
• CABA-201
• KYV-101
• CM-101
• Soquelitinib
• QRX008
• Efzofitimod
• APT-101
• Nerandomilast
• 4P022
• RLS-1496
• Rapalimus
• Novel Anti-fibrotic
• VAY736

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