ATTR Amyloidosis - Pipeline Insight, 2025

This “ATTR Amyloidosis - Pipeline Insight, 2025” report provides comprehensive insights about 10+ companies and 10+ pipeline drugs in ATTR Amyloidosis pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

ATTR Amyloidosis: Understanding

ATTR Amyloidosis: Overview

Transthyretin amyloidosis, also known as cardiac amyloidosis, amyloidosis ATTR, transthyretin amyloid cardiomyopathy (ATTR-CM), transthyretin cardiac amyloidosis (ATTR-CM), and TTR amyloidosis, is a condition characterized by the deposition of transthyretin protein in tissues, particularly affecting the heart.

Transthyretin (previously known as prealbumin) is a normal circulating protein that functions as a carrier protein for retinol (vitamin A) and thyroxine. It is primarily synthesized by the liver (>95%). Additionally, in small amounts, it is also produced in the choroid plexus and retinal epithelium. Usually, it circulates in a tetrameric form composed of 4 beta-sheet-rich monomers. However, structural changes in the TTR protein can cause it to misfold and lose the tetrameric form, causing it to aggregate and deposit in various tissues. The myocardium and peripheral nerves are the most common sites for the deposition of misfolded TTR protein. The clinical phenotype of ATTR depends on the type and extent of tissue involvement.

The symptoms of hereditary ATTR (hATTR) amyloidosis can vary widely among people with the condition and even within families. Different symptoms may appear at different times for each person. The age that symptoms of hATTR amyloidosis typically appear ranges from the mid-20s to the mid-60s. Because symptoms of hATTR amyloidosis can worsen over time. The somatic nervous system, which is made up of nerves that connect the brain and spinal cord to the skin and muscles, controls sensation and voluntary movements. Nerve damage in the somatic nervous system can lead to a range of symptoms, including loss of voluntary movement of the hands and feet, and loss of sensitivity to temperature.

Tissue biopsy with histopathology and immunohistochemistry has been used to definitively diagnose amyloidosis. Extracardiac tissue biopsies (such as abdominal fat pad aspirate) have varying and unreliable sensitivity. Tissue samples from orthopedic procedures, such as carpal tunnel syndrome surgery, have unclear diagnostic reliability at this time but may be useful.

Current guidelines for the treatment of ATTR amyloidosis encompass several key approaches. Specific therapies are designed to modify the synthesis, production, and aggregation of amyloid fibrils. In addition to these targeted treatments, symptomatic therapy is employed to address the neurological and cardiovascular complications associated with the disease. Comprehensive support for patients is also crucial, alongside genetic counselling for those with familial forms of ATTR amyloidosis. These combined strategies aim to provide a holistic approach to managing the condition effectively.

'ATTR Amyloidosis- Pipeline Insight, 2025' report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the ATTR Amyloidosis pipeline landscape is provided which includes the disease overview and ATTR Amyloidosis treatment guidelines. The assessment part of the report embraces, in depth ATTR 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, ATTR 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 ATTR Amyloidosis R&D. The therapies under development are focused on novel approaches to treat/improve ATTR Amyloidosis.

ATTR Amyloidosis Emerging Drugs Chapters

This segment of the ATTR Amyloidosis 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.

ATTR Amyloidosis Emerging Drugs

Vutrisiran: Alnylam Pharmaceuticals

Vutrisiran is an investigational, subcutaneously administered RNAi therapeutic in development for the treatment of ATTR amyloidosis, which encompasses both hATTR and wild-type ATTR (wtATTR) amyloidosis. It is designed to target and silence specific messenger RNA, potentially blocking the production of wild-type and variant transthyretin (TTR) protein before it is made. Quarterly, and potentially biannual, administration of vutrisiran may help to reduce deposition and facilitate the clearance of TTR amyloid deposits in tissues and potentially restore function to these tissues. Vutrisiran utilizes Alnylam’s Enhanced Stabilization Chemistry (ESC)-GalNAc-conjugate delivery platform, designed for increased potency and high metabolic stability that may allow for infrequent subcutaneous injections. The safety and efficacy of vutrisiran have not been evaluated by the US Food and Drug Administration, European Medicines Agency, or any other health authority. Currently the drug is in Phase III stage of its development for the treatment of ATTR Amyloidosis.

NN-6019: Novo Nordisk

NN-6019 is an investigational humanised monoclonal antibody designed to deplete amyloid associated with disease pathology that underlies hereditary and wild type ATTR amyloidosis (hATTR and wtATTR, respectively), without affecting the native, normal tetrameric form of the protein. NNC6019 has been shown in preclinical studies to promote clearance of insoluble amyloid fibrils through antibody-mediated phagocytosis and inhibit amyloid formation. This depleter mechanism of action has the potential to provide benefit for ATTR patients at high risk for early mortality due to amyloid deposition in vital organs. It is currently in Phase II stage of clinical trial evaluation for the treatment of ATTR Amyloidosis.

ALN-TTRsc04: Alnylam Pharmaceuticals

ALN-TTRsc04 is an investigational product being developed by Alnylam Pharmaceuticals that utilizes Alnylam’s IKARIA technology. The drug is currently being evaluated under Phase I clinical trial for the treatment of patients with ATTR amyloidosis.

ATTR Amyloidosis: Therapeutic Assessment

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

Major Players in ATTR Amyloidosis

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

Phases

The 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

ATTR 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

• 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

ATTR Amyloidosis: Pipeline Development Activities

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

ATTR Amyloidosis Report Insights

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

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

Key Players

• Alnylam Pharmaceuticals
• Novo Nordisk
• Sirnaomics
• Arbor Bio

Key Products

• Vutrisiran
• NN-6019
• ALN-TTRsc04
• STP152G
• ABO-102

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