Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Pipeline Review, H1 2018

April 2018 | 80 pages | ID: TE1AF34D503EN
Global Markets Direct

Abstracts

Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Pipeline Review, H1 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 The molecules developed by companies in Pre-Registration, Filing rejected/Withdrawn, Phase II, Phase I and Preclinical stages are 2, 1, 1, 3 and 6 respectively.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, H1 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

REASONS TO BUY

• 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

Contents

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
Alnylam Pharmaceuticals Inc
Arcturus Therapeutics Ltd
Ionis 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
LUNAR-TTR - 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
Oligonucleotides to Inhibit Transthyretin for Amyloidosis - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
patisiran - 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
Apr 03, 2018: Silence Therapeutics: Litigation Update (Portugal and US)
Mar 29, 2018: Pfizer Announces Positive Topline Results From Phase 3 ATTR-ACT Study Of Tafamidis In Patients With Transthyretin Cardiomyopathy
Mar 28, 2018: Alnylam Presents New Clinical Results from the APOLLO Phase 3 Study of Patisiran at the 16th International Symposium on Amyloidosis
Mar 28, 2018: Alnylam Presents New data from the Phase 1 study of ALN-TTRsc02 at the 16th International Symposium on Amyloidosis
Mar 28, 2018: Proclara Biosciences Presents Preclinical Data Supporting the Development of GAIM-Based Therapies for Peripheral Amyloidoses at the 16th International Symposium on Amyloidosis
Mar 26, 2018: Ionis and Akcea Present New Data from ATTR Amyloidosis Program at 16th International Symposium on Amyloidosis
Mar 20, 2018: Eidos Therapeutics to Host Symposium on TTR Stabilization at the 16th International Symposium on Amyloidosis
Mar 14, 2018: Alnylam to Present Clinical Trial Data on Amyloidosis Drug Candidate ALN-TTRsc02 at the 16th International Symposium on Amyloidosis
Mar 14, 2018: Alnylam to Report New Clinical Results from the APOLLO Phase 3 Study of Patisiran at the 16th International Symposium on Amyloidosis
Feb 27, 2018: Intellia Therapeutics Announces Publication in Cell Reports of Preclinical Data Demonstrating Effective CRISPR/Cas9 Genome Editing Using Lipid Nanoparticle (LNP) Delivery Technology
Feb 01, 2018: Alnylam Announces FDA Acceptance of New Drug Application (NDA) and Priority Review Status for Patisiran, an Investigational RNAi Therapeutic for the Treatment of Hereditary ATTR (hATTR) Amyloidosis
Jan 25, 2018: Alnylam Announces EMA Acceptance of Marketing Authorisation Application (MAA) for Patisiran for the Treatment of Hereditary ATTR (hATTR) Amyloidosis
Jan 08, 2018: Ionis Inotersen NDA Accepted for Priority Review by the FDA
Dec 18, 2017: Sanofi and Alnylam Submit Marketing Authorization Application to the EMA for Patisiran for the Treatment of Hereditary ATTR Amyloidosis
Dec 12, 2017: Alnylam Completes Submission of New Drug Application to U.S. Food and Drug Administration (FDA) for Patisiran for the Treatment of Hereditary ATTR (hATTR) Amyloidosis
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer

List Of Tables

LIST OF TABLES

Number of Products under Development by Stage of Development, H1 2018
Number of Products under Development by Therapy Areas, H1 2018
Number of Products under Development by Indication, H1 2018
Number of Products under Development by Companies, H1 2018
Products under Development by Companies, H1 2018
Products under Development by Companies, H1 2018 (Contd.1), H1 2018
Number of Products under Investigation by Universities/Institutes, H1 2018
Products under Investigation by Universities/Institutes, H1 2018
Number of Products by Stage and Mechanism of Actions, H1 2018
Number of Products by Stage and Route of Administration, H1 2018
Number of Products by Stage and Molecule Type, H1 2018
Pipeline by Alnylam Pharmaceuticals Inc, H1 2018
Pipeline by Arcturus Therapeutics Ltd, H1 2018
Pipeline by Ionis Pharmaceuticals Inc, H1 2018
Pipeline by Neurimmune Holding AG, H1 2018
Pipeline by Pfizer Inc, H1 2018
Pipeline by Prothena Corp Plc, H1 2018
Pipeline by Regeneron Pharmaceuticals Inc, H1 2018
Dormant Projects, H1 2018
Discontinued Products, H1 2018

List Of Figures

LIST OF FIGURES

Number of Products under Development by Stage of Development, H1 2018
Number of Products under Development by Therapy Areas, H1 2018
Number of Products under Development by Indications, H1 2018
Number of Products by Mechanism of Actions, H1 2018
Number of Products by Stage and Mechanism of Actions, H1 2018
Number of Products by Routes of Administration, H1 2018
Number of Products by Stage and Routes of Administration, H1 2018
Number of Products by Molecule Types, H1 2018
Number of Products by Stage and Top 10 Molecule Types, H1 2018

COMPANIES MENTIONED

Alnylam Pharmaceuticals Inc
Arcturus Therapeutics Ltd
Ionis Pharmaceuticals Inc
Neurimmune Holding AG
Pfizer Inc
Prothena Corp Plc
Regeneron Pharmaceuticals Inc