Systemic Amyloidosis - Pipeline Insight, 2024

This “Systemic Amyloidosis - Pipeline Insight, 2024” report provides comprehensive insights about 7+ companies and 10+ pipeline drugs in Systemic Amyloidosis 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

Systemic Amyloidosis: Understanding

Systemic Amyloidosis: Overview

Systemic amyloidosis is a rare and complex disorder characterized by the deposition of misfolded protein aggregates in various organs, leading to progressive dysfunction and potentially life-threatening complications. The disease can be acquired or hereditary, with over 15 different types identified, each caused by a distinct precursor protein. Accurate diagnosis and typing of the amyloid subtype is crucial, as it determines the specific management approach. Epidemiological data on systemic amyloidosis primarily originate from high-income countries, with limited information from middle-income regions. The non-specific clinical presentation often results in a prolonged diagnostic journey, with patients experiencing multiple misdiagnoses before the final diagnosis is made.

Systemic amyloidosis is caused by the abnormal deposition of misfolded protein aggregates in various organs, leading to progressive dysfunction. There are several types of systemic amyloidosis, each caused by a distinct precursor protein:

AL amyloidosis (immunoglobulin light chain): the most common type, caused by abnormal antibodies linked to plasma c

ell disorders like multiple myeloma.

AA amyloidosis (serum amyloid A): triggered by chronic inflammatory diseases such as rheumatoid arthriti

s or inflammatory bowel disease.

ATTR amyloidosis (transthyretin): can be hereditary (ATTRv) or age-related (ATTRwt), caused by abnormal

transthyretin protein.

Dialysis-related amyloidosis (beta-2 microglobulin): common in long-term dialysis patients, affecting bones, joints and ten

dons.

Hereditary amyloidosis: rare forms passed down through families, often affecting the liver, ne

rves, heart and kidneys.The specific type determines the clinical presentation, treatment approach and prognosis.

Common signs and symptoms include fatigue, weight loss, paresthesias, hoarseness, and edema. Mucocutaneous involvement is common, manifesting as skin lesions, petechiae, and ecchymoses. Cardiac infiltration can cause angina, infarction, arrhythmias, or orthostatic hypotension, while renal amyloidosis often leads to proteinuria and nephrotic syndrome. Other symptoms include numbness and pain in hands and feet, shortness of breath, and swallowing difficulties.

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

Systemic Amyloidosis Emerging Drugs Chapters

This segment of the Systemic Amyloidosis 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.

Systemic Amyloidosis Emerging Drugs

Anselamimab: AstraZeneca

Anselamimab (CAEL-101) is a potentially first-in-class mAb designed to improve organ function by reducing or eliminating amyloid deposits in the tissues and organs of patients with AL amyloidosis. The antibody is designed to bind to misfolded light chain proteins and amyloid and shows binding to both kappa and lambda subtypes. CAEL-101 has received Orphan Drug Designation from both the US Food and Drug Administration (FDA) and the European Commission as a potential therapy for patients with AL amyloidosis. The drug is currently being evaluated under Phase III clinical trial for the treatment of patients with Systemic Amyloidosis.

AT02: Attralus

AT-02 is the company’s lead pan-amyloid removal (PAR) therapeutic candidate for systemic amyloidosis. AT-02 is a humanized IgG1 monoclonal antibody genetically fused with the company’s proprietary pan-amyloid binding peptide, enabling binding to multiple types of amyloid deposits. The drug is currently being evaluated under Phase II clinical trial for the treatment of patients with Systemic Amyloidosis.

Systemic Amyloidosis: Therapeutic Assessment

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

Major Players in Systemic Amyloidosis

There are approx. 7+ key companies which are developing the therapies for Systemic Amyloidosis. The companies which have their Systemic Amyloidosis drug candidates in the most advanced stage, i.e. Phase III include, AstraZeneca.

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

Systemic Amyloidosis 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.

Systemic Amyloidosis: Pipeline Development Activities

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

Systemic Amyloidosis Report Insights

• Systemic Amyloidosis Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• Attralus
• AstraZeneca
• Nexcella
• Prothena
• HaemaLogiX

Key Products

• AT02
• Anselamimab
• NXC-201
• Birtamimab
• KappaMab

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