This “Transthyretin Amyloid Cardiomyopathy - Pipeline Insight, 2021,” report provides comprehensive insights about 8+ companies and 8+ pipeline drugs in Transthyretin Amyloid Cardiomyopathy 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 Understanding
Transthyretin Amyloid Cardiomyopathy (ATTR-CM) is an underdiagnosed and potentially fatal disease. It's characterized by deposits of amyloid protein fibrils in the walls of the left ventricle, the main pumping chamber of the heart. In ATTR-CM, the amyloid protein is made of transthyretin. The amyloid protein deposits cause the heart walls to become stiff, resulting in the inability of the left ventricle to: 1. properly relax and fill with blood 2. Adequately squeeze to pump blood out of the heart. Due to genetic mutation or aging, the transthyretin doesn't assemble normally these abnormal proteins then clump together and shape themselves into amyloid fibrils. The fibrils travel through the bloodstream and are deposited in many organs, including the heart. As fibrils accumulate in the tissue, they thicken and stiffen the myocardium or wall of the heart. This causes a cardiomyopathy and, ultimately, heart failure. The diagnosis may be suspected because of typical symptoms and the results of a routine cardiac test - an electrocardiogram or echocardiogram. Once suspected, more specialized tests are needed to confirm the diagnosis. There are several promising new therapies for ATTR-CM on the horizon. Medications are approved for hereditary transthyretin amyloidosis affecting the nerves, causing a condition called neuropathy.
"Transthyretin Amyloid Cardiomyopathy - Pipeline Insight, 2021" report by the publisher outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Transthyretin Amyloid Cardiomyopathy pipeline landscape is provided which includes the disease overview and Transthyretin Amyloid Cardiomyopathy treatment guidelines. The assessment part of the report embraces, in depth Transthyretin Amyloid Cardiomyopathy 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 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 R&D. The therapies under development are focused on novel approaches to treat/improve Transthyretin Amyloid Cardiomyopathy.
This segment of the Transthyretin Amyloid Cardiomyopathy 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.
AKCEA-TTR-LRx is an antisense drug developed using Ionis' proprietary LIgand Conjugated Antisense (LICA) technology platform and is designed to inhibit production of TTR. It was discovered by Ionis and is being co-developed by Ionis and Akcea. CARDIO-TTRansform is a global, double-blind, randomized, placebo-controlled Phase III cardiovascular outcome study. It is designed to compare AKCEA-TTR-LRx to placebo in patients with both wild type and hereditary ATTR cardiomyopathy who are on the current available standard of care.
Alexion holds an exclusive license to develop and commercialize ALXN2060 (AG10) in Japan. Eidos is currently evaluating AG10 in two Phase III studies in the US and Europe - one for ATTR cardiomyopathy (ATTR-CM) and one for ATTR polyneuropathy (ATTR-PN). Alexion is conducting a Phase III bridging study of ALXN2060 for patients with ATTR-CM in Japan.
This segment of the report provides insights about the different Transthyretin Amyloid Cardiomyopathy drugs segregated based on following parameters that define the scope of the report, such as:
There are approx. 8+ key companies which are developing the therapies for Transthyretin Amyloid Cardiomyopathy. The companies which have their Transthyretin Amyloid Cardiomyopathy drug candidates in the most advanced stage, i.e. Phase III include, Ionis Pharmaceuticals.
The drugs have been categorized under various product types like Mono, Combination and Mono/Combination.
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Transthyretin Amyloid Cardiomyopathy therapeutic drugs key players involved in developing key drugs.
The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Transthyretin Amyloid Cardiomyopathy drugs.
Current Treatment Scenario and Emerging Therapies:
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Geography Covered
- Global coverage
Transthyretin Amyloid Cardiomyopathy Understanding
Transthyretin Amyloid Cardiomyopathy: Overview
Transthyretin Amyloid Cardiomyopathy (ATTR-CM) is an underdiagnosed and potentially fatal disease. It's characterized by deposits of amyloid protein fibrils in the walls of the left ventricle, the main pumping chamber of the heart. In ATTR-CM, the amyloid protein is made of transthyretin. The amyloid protein deposits cause the heart walls to become stiff, resulting in the inability of the left ventricle to: 1. properly relax and fill with blood 2. Adequately squeeze to pump blood out of the heart. Due to genetic mutation or aging, the transthyretin doesn't assemble normally these abnormal proteins then clump together and shape themselves into amyloid fibrils. The fibrils travel through the bloodstream and are deposited in many organs, including the heart. As fibrils accumulate in the tissue, they thicken and stiffen the myocardium or wall of the heart. This causes a cardiomyopathy and, ultimately, heart failure. The diagnosis may be suspected because of typical symptoms and the results of a routine cardiac test - an electrocardiogram or echocardiogram. Once suspected, more specialized tests are needed to confirm the diagnosis. There are several promising new therapies for ATTR-CM on the horizon. Medications are approved for hereditary transthyretin amyloidosis affecting the nerves, causing a condition called neuropathy.
"Transthyretin Amyloid Cardiomyopathy - Pipeline Insight, 2021" report by the publisher outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Transthyretin Amyloid Cardiomyopathy pipeline landscape is provided which includes the disease overview and Transthyretin Amyloid Cardiomyopathy treatment guidelines. The assessment part of the report embraces, in depth Transthyretin Amyloid Cardiomyopathy 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 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 R&D. The therapies under development are focused on novel approaches to treat/improve Transthyretin Amyloid Cardiomyopathy.
Transthyretin Amyloid Cardiomyopathy Emerging Drugs Chapters
This segment of the Transthyretin Amyloid Cardiomyopathy 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.
Transthyretin Amyloid Cardiomyopathy Emerging Drugs
AKCEA-TTR-LRx: Ionis Pharmaceuticals
AKCEA-TTR-LRx is an antisense drug developed using Ionis' proprietary LIgand Conjugated Antisense (LICA) technology platform and is designed to inhibit production of TTR. It was discovered by Ionis and is being co-developed by Ionis and Akcea. CARDIO-TTRansform is a global, double-blind, randomized, placebo-controlled Phase III cardiovascular outcome study. It is designed to compare AKCEA-TTR-LRx to placebo in patients with both wild type and hereditary ATTR cardiomyopathy who are on the current available standard of care.
ALXN2060: Alexion Pharmaceuticals/Eidos Therapeutics
Alexion holds an exclusive license to develop and commercialize ALXN2060 (AG10) in Japan. Eidos is currently evaluating AG10 in two Phase III studies in the US and Europe - one for ATTR cardiomyopathy (ATTR-CM) and one for ATTR polyneuropathy (ATTR-PN). Alexion is conducting a Phase III bridging study of ALXN2060 for patients with ATTR-CM in Japan.
Transthyretin Amyloid Cardiomyopathy: Therapeutic Assessment
This segment of the report provides insights about the different Transthyretin Amyloid Cardiomyopathy drugs segregated based on following parameters that define the scope of the report, such as:
Major Players in Transthyretin Amyloid Cardiomyopathy
There are approx. 8+ key companies which are developing the therapies for Transthyretin Amyloid Cardiomyopathy. The companies which have their Transthyretin Amyloid Cardiomyopathy drug candidates in the most advanced stage, i.e. Phase III include, Ionis Pharmaceuticals.
Phases
This report covers around 8+ 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 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
- Parenteral
- Intravenous
- Subcutaneous
- Topical.
- Molecule Type
Products have been categorized under various Molecule types such as
- Monoclonal Antibody
- Peptides
- Polymer
- Small molecule
- Gene therapy
- Product Type
The drugs have been categorized under various product types like Mono, Combination and Mono/Combination.
Transthyretin Amyloid Cardiomyopathy: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Transthyretin Amyloid Cardiomyopathy 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 Transthyretin Amyloid Cardiomyopathy drugs.
Transthyretin Amyloid Cardiomyopathy Report Insights
- Transthyretin Amyloid Cardiomyopathy Pipeline Analysis
- Therapeutic Assessment
- Unmet Needs
- Impact of Drugs
Transthyretin Amyloid Cardiomyopathy Report Assessment
- Pipeline Product Profiles
- Therapeutic Assessment
- Pipeline Assessment
- Inactive drugs assessment
- Unmet Needs
Key Questions Answered
Current Treatment Scenario and Emerging Therapies:
- How many companies are developing Transthyretin Amyloid Cardiomyopathy drugs?
- How many Transthyretin Amyloid Cardiomyopathy 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?
- What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Transthyretin Amyloid Cardiomyopathy 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 and their status?
- What are the key designations that have been granted to the emerging drugs?
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Table of Contents
IntroductionExecutive Summary
Transthyretin Amyloid Cardiomyopathy: Overview
- Causes
- Mechanism of Action
- Signs and Symptoms
- Diagnosis
- Disease Management
Pipeline Therapeutics
- Comparative Analysis
Therapeutic Assessment
- Assessment by Product Type
- Assessment by Stage and Product Type
- Assessment by Route of Administration
- Assessment by Stage and Route of Administration
- Assessment by Molecule Type
- Assessment by Stage and Molecule Type
Late Stage Products (Phase III)
- Comparative Analysis
AKCEA-TTR-LRx: Ionis Pharmaceuticals
- Product Description
- Research and Development
- Product Development Activities
Mid Stage Products (Phase II)
- Comparative Analysis
Drug Name: Company Name
- Product Description
- Research and Development
- Product Development Activities
Early stage products (Phase I/II)
- Comparative Analysis
NI006: Neurimmune AG
- Product Description
- Research and Development
- Product Development Activities
Inactive Products
- Comparative Analysis
Transthyretin Amyloid Cardiomyopathy Key Companies
Transthyretin Amyloid Cardiomyopathy Key Products
Transthyretin Amyloid Cardiomyopathy- Unmet Needs
Transthyretin Amyloid Cardiomyopathy- Market Drivers and Barriers
Transthyretin Amyloid Cardiomyopathy- Future Perspectives and Conclusion
Transthyretin Amyloid Cardiomyopathy Analyst Views
Transthyretin Amyloid Cardiomyopathy Key Companies
AppendixList of Tables
Table 1 Total Products for Transthyretin Amyloid Cardiomyopathy
Table 2 Late Stage Products
Table 3 Mid Stage Products
Table 4 Early Stage Products
Table 5 Pre-clinical & Discovery Stage Products
Table 6 Assessment by Product Type
Table 7 Assessment by Stage and Product Type
Table 8 Assessment by Route of Administration
Table 9 Assessment by Stage and Route of Administration
Table 10 Assessment by Molecule Type
Table 11 Assessment by Stage and Molecule Type
Table 12 Inactive ProductsList of Figures
Figure 1 Total Products for Transthyretin Amyloid Cardiomyopathy
Figure 2 Late Stage Products
Figure 3 Mid Stage Products
Figure 4 Early Stage Products
Figure 5 Preclinical and Discovery Stage Products
Figure 6 Assessment by Product Type
Figure 7 Assessment by Stage and Product Type
Figure 8 Assessment by Route of Administration
Figure 9 Assessment by Stage and Route of Administration
Figure 10 Assessment by Molecule Type
Figure 11 Assessment by Stage and Molecule Type
Figure 12 Inactive Products
Companies Mentioned (Partial List)
A selection of companies mentioned in this report includes, but is not limited to:
- Alexion Pharmaceuticals
- Ionis Pharmaceuticals
- Edios therapeutics
- Alnylam Pharmaceuticals
- Neurimmune AG
- Corino Therapeutics
- Novo Nordisk
- Intellia Therapeutics
