Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Pipeline Review, H2 2018
Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Pipeline Review, H2 2018
SUMMARY
Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Transthyretin is a transport protein. It transports thyroid hormones in the plasma and cerebrospinal fluid, and also transports retinol (vitamin A) in the plasma. The diseases caused by mutations include amyloidotic polyneuropathy, euthyroid hyperthyroxinaemia, amyloidotic vitreous opacities, cardiomyopathy, oculoleptomeningeal amyloidosis, meningocerebrovascular amyloidosis and carpal tunnel syndrome.
Transthyretin (ATTR or Prealbumin or TBPA or TTR) pipeline Target constitutes close to 14 molecules. Out of which approximately 13 molecules are developed by companies and remaining by the universities/institutes. The molecules developed by companies in Pre-Registration, Filing rejected/Withdrawn, Phase II, Phase I and Preclinical stages are 2, 1, 2, 4 and 4 respectively. Similarly, the universities portfolio in Phase II stages comprises 1 molecules, respectively.
Report covers products from therapy areas Metabolic Disorders, Central Nervous System and Cardiovascular which include indications Familial Amyloid Neuropathies, Amyloidosis, Amyloid Cardiomyopathy, Familial Amyloid Cardiomyopathy, Alzheimer's Disease, Cardiomyopathy and Neuropathy.
The latest report Transthyretin - Pipeline Review, H2 2018, outlays comprehensive information on the Transthyretin (ATTR or Prealbumin or TBPA or TTR) targeted therapeutics, complete with analysis by indications, stage of development, mechanism of action (MoA), route of administration (RoA) and molecule type. It also reviews key players involved in Transthyretin (ATTR or Prealbumin or TBPA or TTR) targeted therapeutics development with respective active and dormant or discontinued projects.
The report is built using data and information sourced from proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources.
Note: Certain content/sections in the pipeline guide may be removed or altered based on the availability and relevance of data.
SCOPE
SUMMARY
Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Transthyretin is a transport protein. It transports thyroid hormones in the plasma and cerebrospinal fluid, and also transports retinol (vitamin A) in the plasma. The diseases caused by mutations include amyloidotic polyneuropathy, euthyroid hyperthyroxinaemia, amyloidotic vitreous opacities, cardiomyopathy, oculoleptomeningeal amyloidosis, meningocerebrovascular amyloidosis and carpal tunnel syndrome.
Transthyretin (ATTR or Prealbumin or TBPA or TTR) pipeline Target constitutes close to 14 molecules. Out of which approximately 13 molecules are developed by companies and remaining by the universities/institutes. The molecules developed by companies in Pre-Registration, Filing rejected/Withdrawn, Phase II, Phase I and Preclinical stages are 2, 1, 2, 4 and 4 respectively. Similarly, the universities portfolio in Phase II stages comprises 1 molecules, respectively.
Report covers products from therapy areas Metabolic Disorders, Central Nervous System and Cardiovascular which include indications Familial Amyloid Neuropathies, Amyloidosis, Amyloid Cardiomyopathy, Familial Amyloid Cardiomyopathy, Alzheimer's Disease, Cardiomyopathy and Neuropathy.
The latest report Transthyretin - Pipeline Review, H2 2018, outlays comprehensive information on the Transthyretin (ATTR or Prealbumin or TBPA or TTR) targeted therapeutics, complete with analysis by indications, stage of development, mechanism of action (MoA), route of administration (RoA) and molecule type. It also reviews key players involved in Transthyretin (ATTR or Prealbumin or TBPA or TTR) targeted therapeutics development with respective active and dormant or discontinued projects.
The report is built using data and information sourced from proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources.
Note: Certain content/sections in the pipeline guide may be removed or altered based on the availability and relevance of data.
SCOPE
- The report provides a snapshot of the global therapeutic landscape for Transthyretin (ATTR or Prealbumin or TBPA or TTR)
- The report reviews Transthyretin (ATTR or Prealbumin or TBPA or TTR) targeted therapeutics under development by companies and universities/research institutes based on information derived from company and industry-specific sources
- The report covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages
- The report features descriptive drug profiles for the pipeline products which includes, product description, descriptive MoA, R&D brief, licensing and collaboration details & other developmental activities
- The report reviews key players involved in Transthyretin (ATTR or Prealbumin or TBPA or TTR) targeted therapeutics and enlists all their major and minor projects
- The report assesses Transthyretin (ATTR or Prealbumin or TBPA or TTR) targeted therapeutics based on mechanism of action (MoA), route of administration (RoA) and molecule type
- The report summarizes all the dormant and discontinued pipeline projects
- The report reviews latest news and deals related to Transthyretin (ATTR or Prealbumin or TBPA or TTR) targeted therapeutics
- Gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies
- Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage
- Identify and understand the targeted therapy areas and indications for Transthyretin (ATTR or Prealbumin or TBPA or TTR)
- Identify the use of drugs for target identification and drug repurposing
- Identify potential new clients or partners in the target demographic
- Develop strategic initiatives by understanding the focus areas of leading companies
- Plan mergers and acquisitions effectively by identifying key players and it’s most promising pipeline therapeutics
- Devise corrective measures for pipeline projects by understanding Transthyretin (ATTR or Prealbumin or TBPA or TTR) development landscape
- Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope
Introduction
Global Markets Direct Report Coverage
Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Overview
Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Therapeutics Development
Products under Development by Stage of Development
Products under Development by Therapy Area
Products under Development by Indication
Products under Development by Companies
Products under Development by Universities/Institutes
Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Therapeutics Assessment
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Companies Involved in Therapeutics Development
Akcea Therapeutics Inc
Alnylam Pharmaceuticals Inc
Neurimmune Holding AG
Pfizer Inc
Prothena Corp Plc
Regeneron Pharmaceuticals Inc
Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Drug Profiles
AG-10 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
AKCEA-TTR-LRx - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
ALN-TTRsc02 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
doxycycline hyclate - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
EDE-1307 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
inotersen sodium - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
NI-301 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
NPT-189 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
NTLA-1001 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
patisiran - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Peptides to Inhibit TTR for Familial Amyloid Neuropathies - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
PRX-004 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
tafamidis meglumine - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
tolcapone - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Dormant Products
Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Discontinued Products
Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Product Development Milestones
Featured News & Press Releases
Oct 04, 2018: Akcea and Ionis announce approval of TEGSEDI (inotersen injection) in Canada
Oct 03, 2018: FDA grants Orphan Drug Designation to Eidos Therapeutics product candidate, AG10, for treatment of transthyretin amyloidosis
Oct 03, 2018: Kaskela Law LLC Announces Investor Class Action Lawsuit Against Alnylam Pharmaceuticals, Inc. and Encourages Investors with Losses in Excess of $100,000 to Contact the Firm
Oct 02, 2018: Alnylam launches ONPATTRO (patisiran), the first-ever RNAi therapeutic, in Germany
Sep 28, 2018: Alnylam announces submission of New Drug Application in Japan for ONPATTRO (patisiran sodium) for treatment of Hereditary ATTR Amyloidosis
Sep 28, 2018: Alnylam Announces Submission of New Drug Application in Japan for ONPATTRO™ (patisiran sodium) for Treatment of Hereditary ATTR Amyloidosis
Sep 24, 2018: Alnylam Pharmaceuticals receives Priority Review in Canada for Patisiran, an investigational RNAi Therapeutic for the treatment of Hereditary Transthyretin-mediated Amyloidosis (hATTR amyloidosis) with Polyneuropathy
Sep 18, 2018: New sub-group analyses from the Tafamidis phase 3 Transthyretin Amyloid Cardiomyopathy (ATTR-ACT) study presented at 2018 HFSA Annual Scientific Meeting
Sep 17, 2018: Eidos Therapeutics presents data from its Phase 1 Clinical Trial of AG10 at the 22nd Annual Scientific Meeting of the Heart Failure Society of America
Sep 14, 2018: Alnylam announces publication in circulation of exploratory cardiac endpoint data from APOLLO phase 3 study of Patisiran
Aug 30, 2018: Alnylam receives approval of ONPATTRO (patisiran) in Europe
Aug 28, 2018: Pfizer reports data from tafamidis’ rare heart disease trial
Aug 23, 2018: Intellia Therapeutics presents progress in lead in vivo program at cold spring harbor laboratory’s fourth meeting on genome engineering: the CRISPR-Cas revolution
Aug 23, 2018: Eidos Therapeutics announces publication of AG10 molecular design in journal of medicinal chemistry
Aug 20, 2018: Veristat congratulates Alnylam on the FDA approval of ONPATTRO (patisiran)
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer
Global Markets Direct Report Coverage
Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Overview
Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Therapeutics Development
Products under Development by Stage of Development
Products under Development by Therapy Area
Products under Development by Indication
Products under Development by Companies
Products under Development by Universities/Institutes
Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Therapeutics Assessment
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Companies Involved in Therapeutics Development
Akcea Therapeutics Inc
Alnylam Pharmaceuticals Inc
Neurimmune Holding AG
Pfizer Inc
Prothena Corp Plc
Regeneron Pharmaceuticals Inc
Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Drug Profiles
AG-10 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
AKCEA-TTR-LRx - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
ALN-TTRsc02 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
doxycycline hyclate - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
EDE-1307 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
inotersen sodium - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
NI-301 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
NPT-189 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
NTLA-1001 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
patisiran - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Peptides to Inhibit TTR for Familial Amyloid Neuropathies - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
PRX-004 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
tafamidis meglumine - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
tolcapone - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Dormant Products
Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Discontinued Products
Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Product Development Milestones
Featured News & Press Releases
Oct 04, 2018: Akcea and Ionis announce approval of TEGSEDI (inotersen injection) in Canada
Oct 03, 2018: FDA grants Orphan Drug Designation to Eidos Therapeutics product candidate, AG10, for treatment of transthyretin amyloidosis
Oct 03, 2018: Kaskela Law LLC Announces Investor Class Action Lawsuit Against Alnylam Pharmaceuticals, Inc. and Encourages Investors with Losses in Excess of $100,000 to Contact the Firm
Oct 02, 2018: Alnylam launches ONPATTRO (patisiran), the first-ever RNAi therapeutic, in Germany
Sep 28, 2018: Alnylam announces submission of New Drug Application in Japan for ONPATTRO (patisiran sodium) for treatment of Hereditary ATTR Amyloidosis
Sep 28, 2018: Alnylam Announces Submission of New Drug Application in Japan for ONPATTRO™ (patisiran sodium) for Treatment of Hereditary ATTR Amyloidosis
Sep 24, 2018: Alnylam Pharmaceuticals receives Priority Review in Canada for Patisiran, an investigational RNAi Therapeutic for the treatment of Hereditary Transthyretin-mediated Amyloidosis (hATTR amyloidosis) with Polyneuropathy
Sep 18, 2018: New sub-group analyses from the Tafamidis phase 3 Transthyretin Amyloid Cardiomyopathy (ATTR-ACT) study presented at 2018 HFSA Annual Scientific Meeting
Sep 17, 2018: Eidos Therapeutics presents data from its Phase 1 Clinical Trial of AG10 at the 22nd Annual Scientific Meeting of the Heart Failure Society of America
Sep 14, 2018: Alnylam announces publication in circulation of exploratory cardiac endpoint data from APOLLO phase 3 study of Patisiran
Aug 30, 2018: Alnylam receives approval of ONPATTRO (patisiran) in Europe
Aug 28, 2018: Pfizer reports data from tafamidis’ rare heart disease trial
Aug 23, 2018: Intellia Therapeutics presents progress in lead in vivo program at cold spring harbor laboratory’s fourth meeting on genome engineering: the CRISPR-Cas revolution
Aug 23, 2018: Eidos Therapeutics announces publication of AG10 molecular design in journal of medicinal chemistry
Aug 20, 2018: Veristat congratulates Alnylam on the FDA approval of ONPATTRO (patisiran)
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer
LIST OF TABLES
Number of Products under Development by Stage of Development, H2 2018
Number of Products under Development by Therapy Areas, H2 2018
Number of Products under Development by Indication, H2 2018
Number of Products under Development by Companies, H2 2018
Products under Development by Companies, H2 2018
Products under Development by Companies, H2 2018 (Contd.1), H2 2018
Number of Products under Investigation by Universities/Institutes, H2 2018
Products under Investigation by Universities/Institutes, H2 2018
Number of Products by Stage and Mechanism of Actions, H2 2018
Number of Products by Stage and Route of Administration, H2 2018
Number of Products by Stage and Molecule Type, H2 2018
Pipeline by Akcea Therapeutics Inc, H2 2018
Pipeline by Alnylam Pharmaceuticals Inc, H2 2018
Pipeline by Neurimmune Holding AG, H2 2018
Pipeline by Pfizer Inc, H2 2018
Pipeline by Prothena Corp Plc, H2 2018
Pipeline by Regeneron Pharmaceuticals Inc, H2 2018
Dormant Projects, H2 2018
Discontinued Products, H2 2018
Number of Products under Development by Stage of Development, H2 2018
Number of Products under Development by Therapy Areas, H2 2018
Number of Products under Development by Indication, H2 2018
Number of Products under Development by Companies, H2 2018
Products under Development by Companies, H2 2018
Products under Development by Companies, H2 2018 (Contd.1), H2 2018
Number of Products under Investigation by Universities/Institutes, H2 2018
Products under Investigation by Universities/Institutes, H2 2018
Number of Products by Stage and Mechanism of Actions, H2 2018
Number of Products by Stage and Route of Administration, H2 2018
Number of Products by Stage and Molecule Type, H2 2018
Pipeline by Akcea Therapeutics Inc, H2 2018
Pipeline by Alnylam Pharmaceuticals Inc, H2 2018
Pipeline by Neurimmune Holding AG, H2 2018
Pipeline by Pfizer Inc, H2 2018
Pipeline by Prothena Corp Plc, H2 2018
Pipeline by Regeneron Pharmaceuticals Inc, H2 2018
Dormant Projects, H2 2018
Discontinued Products, H2 2018
LIST OF FIGURES
Number of Products under Development by Stage of Development, H2 2018
Number of Products under Development by Therapy Areas, H2 2018
Number of Products under Development by Top 10 Indications, H2 2018
Number of Products by Mechanism of Actions, H2 2018
Number of Products by Stage and Mechanism of Actions, H2 2018
Number of Products by Routes of Administration, H2 2018
Number of Products by Stage and Routes of Administration, H2 2018
Number of Products by Molecule Types, H2 2018
Number of Products by Stage and Molecule Types, H2 2018
COMPANIES MENTIONED
Akcea Therapeutics Inc
Alnylam Pharmaceuticals Inc
Neurimmune Holding AG
Pfizer Inc
Prothena Corp Plc
Regeneron Pharmaceuticals Inc
Number of Products under Development by Stage of Development, H2 2018
Number of Products under Development by Therapy Areas, H2 2018
Number of Products under Development by Top 10 Indications, H2 2018
Number of Products by Mechanism of Actions, H2 2018
Number of Products by Stage and Mechanism of Actions, H2 2018
Number of Products by Routes of Administration, H2 2018
Number of Products by Stage and Routes of Administration, H2 2018
Number of Products by Molecule Types, H2 2018
Number of Products by Stage and Molecule Types, H2 2018
COMPANIES MENTIONED
Akcea Therapeutics Inc
Alnylam Pharmaceuticals Inc
Neurimmune Holding AG
Pfizer Inc
Prothena Corp Plc
Regeneron Pharmaceuticals Inc
