Transthyretin Amyloid Cardiomyopathy (ATTR-CM) - Pipeline Insight, 2025

This “Transthyretin Amyloid Cardiomyopathy (ATTR-CM) - Pipeline Insight, 2025” report provides comprehensive insights about 3+ companies and 3+ pipeline drugs in Transthyretin Amyloid Cardiomyopathy (ATTR-CM) 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.

Transthyretin Amyloid Cardiomyopathy (ATTR-CM): Understanding

Transthyretin Amyloid Cardiomyopathy (ATTR-CM): Overview

Transthyretin amyloid cardiomyopathy (ATTR-CM) is a form of systemic amyloidosis characterized by the deposition of misfolded transthyretin (TTR) protein in the myocardium, leading to progressive cardiac dysfunction. It is distinct from light-chain (AL) amyloidosis, another major cause of cardiac amyloid deposition, and while other amyloidogenic proteins may affect various organs, they rarely target the heart. ATTR-CM can present with or without extracardiac symptoms, as amyloid deposits may accumulate systemically or be isolated to the heart. Historically underdiagnosed due to subtle early symptoms and limited diagnostic tools, ATTR-CM is now more readily detected thanks to advances in bone-avid radiotracer scintigraphy and emerging therapies. Improved diagnostics have revealed a greater prevalence of ATTR-CM, particularly among patients with heart failure with preserved ejection fraction (HFpEF), underscoring the need for heightened clinical awareness.

Transthyretin amyloid cardiomyopathy (ATTR-CM) presents with a range of cardiac and non-cardiac symptoms. Common cardiovascular manifestations include signs of heart failure such as fatigue, shortness of breath (particularly when lying down), swelling in the legs and ankles, and reduced exercise tolerance. Patients may also experience arrhythmias like palpitations or atrial fibrillation, and symptoms such as syncope or lightheadedness due to impaired cardiac output. Non-cardiac symptoms are often related to systemic amyloid deposition and include carpal tunnel syndrome, peripheral neuropathy causing numbness or pain in the extremities, and autonomic dysfunction presenting as gastrointestinal issues, orthostatic hypotension, erectile dysfunction, or bladder problems. Other reported symptoms may include eye floaters, cognitive difficulties, and unexplained weight loss, all contributing to diagnostic complexity.

In transthyretin amyloid cardiomyopathy (ATTR-CM), misfolded transthyretin (TTR) proteins form insoluble amyloid fibers that accumulate in the interstitial spaces of the myocardium, leading to increased stiffness and rigidity of the heart muscle. This deposition results in myocardial thickening and fibrosis, which impair the heart's mechanical function. Initially, the reduced ventricular compliance leads to diastolic dysfunction, and as the disease progresses, it can result in global systolic dysfunction. Elevated left ventricular end-diastolic and atrial pressures contribute to atrial enlargement and a higher risk of atrial arrhythmias. Additionally, misfolded TTR deposits can affect the autonomic and peripheral nervous systems, with hereditary ATTR (hATTR) more frequently involving the nervous system, while wild-type ATTR (wATTR) more commonly presents with predominant cardiac involvement.

Supportive treatment of transthyretin amyloid cardiomyopathy (ATTR-CM) focuses on managing heart failure and arrhythmias. For heart failure, maintaining euvolemia is essential and achieved through sodium restriction and potent diuretics like torsemide or bumetanide, often with aldosterone antagonists. Standard therapies such as beta-blockers and ACE inhibitors are usually poorly tolerated due to hypotension and autonomic dysfunction. In arrhythmia management, rhythm control is preferred for atrial fibrillation, with amiodarone being the antiarrhythmic of choice. Lifelong anticoagulation is recommended due to a high risk of thrombus. Conduction system disease is common and may require pacemaker placement, while ICDs are used mainly for secondary prevention.
"Transthyretin Amyloid Cardiomyopathy (ATTR-CM) - Pipeline Insight, 2025" report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Transthyretin Amyloid Cardiomyopathy (ATTR-CM) pipeline landscape is provided which includes the disease overview and Transthyretin Amyloid Cardiomyopathy (ATTR-CM) treatment guidelines. The assessment part of the report embraces, in depth Transthyretin Amyloid Cardiomyopathy (ATTR-CM) 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, Transthyretin Amyloid Cardiomyopathy (ATTR-CM) 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 Transthyretin Amyloid Cardiomyopathy (ATTR-CM) R&D. The therapies under development are focused on novel approaches to treat/improve Transthyretin Amyloid Cardiomyopathy (ATTR-CM).

Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Emerging Drugs Chapters

This segment of the Transthyretin Amyloid Cardiomyopathy (ATTR-CM) 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.

Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Emerging Drugs

NTLA-2001: Intellia Therapeutics

Based on Nobel Prize-winning CRISPR/Cas9 technology, nex-z has the potential to become the first one-time treatment for transthyretin (ATTR) amyloidosis. Nex-z is an investigational in vivo CRISPR-based therapy designed to inactivate the TTR gene that encodes for the mutated transthyretin (TTR) protein causing the polyneuropathy. Interim clinical data showed the administration of nex-z led to consistent, deep and long-lasting TTR reduction. Nex-z has been granted Regenerative Medicine Advanced Therapy designations by the US FDA for both cardiomyopathy and polyneuropathy. Nex-z has also been granted Orphan Drug Designation by the US FDA and European Commission. Intellia leads development and commercialization of nex-z as part of a multi-target discovery, development and commercialization collaboration with Regeneron. Currently, the drug is in Phase III stage of its development for the treatment of Transthyretin Amyloid Cardiomyopathy (ATTR-CM).

Transthyretin Amyloid Cardiomyopathy (ATTR-CM): Therapeutic Assessment

This segment of the report provides insights about the different Transthyretin Amyloid Cardiomyopathy (ATTR-CM) drugs segregated based on following parameters that define the scope of the report.

Major Players in Transthyretin Amyloid Cardiomyopathy (ATTR-CM)

• There are approx. 3+ key companies which are developing the therapies for Transthyretin Amyloid Cardiomyopathy (ATTR-CM). The companies which have their Transthyretin Amyloid Cardiomyopathy (ATTR-CM) drug candidates in the most advanced stage, i.e. Phase III include, Intellia Therapeutics.

Phases
The report covers around 3+ 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
Transthyretin Amyloid Cardiomyopathy (ATTR-CM) 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.

Transthyretin Amyloid Cardiomyopathy (ATTR-CM): Pipeline Activities

The report provides insights into different therapeutic candidates in Phase III, II, I, preclinical and discovery stage. It also analyses Transthyretin Amyloid Cardiomyopathy (ATTR-CM) therapeutic drugs key players involved in developing key drugs.

Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Transthyretin Amyloid Cardiomyopathy (ATTR-CM) drugs.

Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Report Insights

• Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• Intellia Therapeutics
• YolTech Therapeutics Co., Ltd
• Alexion Pharmaceuticals, Inc.

Key Products

• NTLA-2001
• YOLT-201
• ALXN2220

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