Dermatomyositis - Pipeline Insight, 2024

This “Dermatomyositis - Pipeline Insight, 2024” report provides comprehensive insights about 10+ companies and 10+ pipeline drugs in Dermatomyositis 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

Dermatomyositis: Understanding

Dermatomyositis: Overview

Dermatomyositis (DM) is a rare, chronic inflammatory disease characterized by muscle weakness and distinctive skin manifestations. This idiopathic inflammatory myopathy primarily affects the proximal muscles, including the hips, thighs, shoulders, and neck, leading to progressive and symmetrical weakness that can significantly impair daily activities. Skin symptoms include Gottron's papules, which are raised, scaly bumps on the knuckles, elbows, and knees, and a heliotrope rash, characterized by a purplish discoloration and swelling around the eyes. Other notable rashes include the V-sign and shawl sign, which appear over the chest and shoulders respectively, as well as mechanic's hands, which feature rough, cracked skin on the sides of the fingers.

The precise cause of dermatomyositis remains unknown, though it is believed to involve a combination of genetic predisposition, immune system abnormalities, and environmental triggers such as infections or certain medications. The disease predominantly affects women and is most common in adults aged 40-60 and children aged 5-15. A family history of autoimmune diseases may also increase the risk of developing DM.

Diagnosing dermatomyositis involves a comprehensive clinical evaluation supported by various laboratory tests and imaging studies. Elevated muscle enzyme levels in the blood, such as creatine kinase, indicate muscle inflammation. Autoantibody tests, like those detecting anti-Jo-1 antibodies, are also critical. Electromyography (EMG) assesses abnormal electrical activity in affected muscles, while muscle biopsy provides direct evidence of muscle tissue inflammation and damage. Magnetic resonance imaging (MRI) helps visualize the extent of muscle inflammation.

Treatment of dermatomyositis aims to alleviate symptoms, enhance muscle strength, and prevent complications. Corticosteroids, such as prednisone, are commonly used to reduce inflammation. Immunosuppressive agents, including methotrexate and azathioprine, help modulate the immune response. In severe cases, biologic agents like rituximab may be administered. Physical therapy plays a vital role in improving muscle strength and function, while proper skin care, including sun protection and topical treatments, addresses skin symptoms.

Despite treatment, dermatomyositis can lead to significant complications if not managed appropriately. These include interstitial lung disease, cardiac issues like myocarditis or arrhythmias, and calcinosis, particularly in children. Additionally, adults with DM have an increased risk of malignancies, underscoring the importance of regular cancer screenings. While early diagnosis and treatment can lead to substantial improvement or remission, ongoing research is essential to further enhance patient outcomes and develop targeted therapies.

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

Dermatomyositis Emerging Drugs Chapters

This segment of the Dermatomyositis 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.

Dermatomyositis Emerging Drugs

EFG PH20: argenx

Efgartigimod is designed as a first-in-class investigational antibody fragment to target the neonatal Fc receptor (FcRn). Efgartigimod is being evaluated for the treatment of patients with severe autoimmune diseases with confirmed presence of pathogenic immunoglobulin G, IgG autoantibodies, where a severe unmet medical need exists. A Phase II/III study (NCT05523167) is evaluating the efficacy and safety of efgartigimod PH20 SC compared to placebo in adults with active IIMs, including DM, immune-mediated necrotizing myopathy (IMNM), polymyositis (PM), and antisynthetase syndrome. The primary endpoint is treatment response measured by Total Improvement Score (TIS). Another study (NCT05979441) is assessing the long-term safety and efficacy of subcutaneous efgartigimod in adults with IIMs. The drug is currently in Phase III stage of development to treat Dermatomyositis.

KZR-616: Kezar Life Sciences

KZR-616 offers a novel approach to harmonizing the immune system via selective immunoproteasome inhibition. Playing a critical role in the body's immune system, the immunoproteasome is abundantly expressed in immune cells and acts as a master regulator of multiple cellular functions. By selectively inhibiting the immunoproteasome, KZR-616 has the potential to affect multiple drivers of immune-mediated diseases.

Dermatomyositis: Therapeutic Assessment

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

Major Players in Dermatomyositis

• There are approx. 10+ key companies which are developing the therapies for Dermatomyositis. The companies which have their Dermatomyositis drug candidates in the most advanced stage, i.e. Phase III include, argenx.

Phases

This report covers around 10+ 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

Dermatomyositis 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.

Dermatomyositis: Pipeline Development Activities

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

Dermatomyositis Report Insights

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

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

Key Players

• Kezar Life Sciences
• Argenx
• Pfizer
• CSL Behring
• Viela Bio
• PAEAN Biotechnology
• Alexion Pharmaceuticals

Key Products

• KZR-616
• EFG PH20
• PF 06823859
• Hizentra
• Daxdilimab
• PN 101
• Ravulizumab

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