Transthyretin (ATTR or Prealbumin or TBPA or TTR) Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update

September 2022 | 85 pages | ID: TC087ADCB8E3EN
Global Markets Direct

Abstracts

Transthyretin (ATTR or Prealbumin or TBPA or TTR) Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update

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 17 molecules. Out of which approximately 16 molecules are developed by companies and remaining by the universities/institutes. The molecules developed by companies in Pre-Registration, Phase III, Phase II, Phase I, Preclinical and Discovery stages are 1, 3, 3, 4, 3 and 2 respectively. Similarly, the universities portfolio in Phase III stages comprises 1 molecules, respectively. Report covers products from therapy areas Metabolic Disorders, Ophthalmology, Central Nervous System, Gastrointestinal, Immunology and Infectious Disease which include indications Familial Amyloid Neuropathies, Familial Amyloid Cardiomyopathy, Amyloid Cardiomyopathy, Amyloidosis, Coronavirus Disease 2019 (COVID-19) Associated Acute Respiratory Distress Syndrome, Crohn's Disease (Regional Enteritis), Glaucoma, Juvenile Macular Degeneration (Stargardt Disease), Keratoconjunctivitis Sicca (Dry Eye), Multiple Sclerosis, Rheumatoid Arthritis, Ulcerative Colitis and Unspecified Ophthalmological Disorders.

The latest report Transthyretin - Drugs In Development, 2022, 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
ADRx Inc
Akcea Therapeutics Inc
Alexion Pharmaceuticals Inc
Alnylam Pharmaceuticals Inc
Autotac Bio Inc
BridgeBio Pharma Inc
BSIM Therapeutics SA
Corino Therapeutics Inc
Covalent Bioscience Inc
Galmed Pharmaceuticals Ltd
Novo Nordisk AS
Proclara Biosciences Inc
Regeneron Pharmaceuticals Inc
Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Drug Profiles
acoramidis hydrochloride - Drug Profile
Product Description
Mechanism Of Action
History of Events
ALNTTRSC-04 - Drug Profile
Product Description
Mechanism Of Action
History of Events
Amilo-5MER - Drug Profile
Product Description
Mechanism Of Action
History of Events
Antibody to Inhibit TTR for Familial Transthyretin Amyloidosis - Drug Profile
Product Description
Mechanism Of Action
History of Events
ATC-202 - Drug Profile
Product Description
Mechanism Of Action
doxycycline hyclate - Drug Profile
Product Description
Mechanism Of Action
History of Events
eplontersen sodium - Drug Profile
Product Description
Mechanism Of Action
History of Events
inotersen sodium - Drug Profile
Product Description
Mechanism Of Action
History of Events
Monoclonal Antibody for Familial Amyloid Cardiomyopathy - Drug Profile
Product Description
Mechanism Of Action
History of Events
NI-006 - Drug Profile
Product Description
Mechanism Of Action
History of Events
NPT-189 - Drug Profile
Product Description
Mechanism Of Action
History of Events
NTLA-2001 - Drug Profile
Product Description
Mechanism Of Action
History of Events
patisiran - Drug Profile
Product Description
Mechanism Of Action
History of Events
PRX-004 - Drug Profile
Product Description
Mechanism Of Action
History of Events
Small Molecules to Inhibit Transthyretin for Ophthalmology - Drug Profile
Product Description
Mechanism Of Action
tafamidis meglumine - Drug Profile
Product Description
Mechanism Of Action
History of Events
tolcapone - Drug Profile
Product Description
Mechanism Of Action
History of Events
vutrisiran - Drug Profile
Product Description
Mechanism Of Action
History of Events
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
Aug 18, 2022: Alnylam to present data from the APOLLO-B phase 3 study of patisiran in patients with ATTR amyloidosis with cardiomyopathy at the 18th International Symposium on amyloidosis
Aug 03, 2022: Alnylam reports positive topline results from APOLLO-B phase 3 study of patisiran in patients with ATTR amyloidosis with cardiomyopathy
Jul 22, 2022: Alnylam receives positive CHMP opinion for vutrisiran for the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy
Jul 20, 2022: Orsini Specialty Pharmacy selected as limited distribution partner for AMVUTTRA (vutrisiran)
Jun 24, 2022: Intellia and Regeneron present updated interim data from phase 1 Study of CRISPR-based NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis demonstrating that deep serum TTR reductions remained durable after a single dose
Jun 22, 2022: AstraZeneca-Ionis’ eplontersen meets endpoints in Phase III ATTRv-PN trial
Jun 13, 2022: Alnylam announces FDA approval of AMVUTTRA (vutrisiran), an RNAi therapeutic for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults
Jun 08, 2022: Intellia Therapeutics to present updated interim clinical data from ongoing phase 1 study of NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis at the International Liver Congress 2022
May 23, 2022: Alnylam presents new 18-month results from exploratory cardiac endpoints in HELIOS-A phase 3 study of investigational vutrisiran
Apr 04, 2022: Alnylam announces 3-Month extension of review period for New Drug Application for Vutrisiran
Apr 03, 2022: BridgeBio Pharma presents updated rsults from phase 2 open-label extension study of Acoramidis in transthyretin amyloid cardiomyopathy (ATTR-CM)
Mar 28, 2022: BridgeBio Pharma to present updated results from phase 2 open-label extension study of Acoramidis in Transthyretin Amyloid Cardiomyopathy (ATTR-CM) at the American College of Cardiology (ACC) Annual Scientific Session & Expo
Mar 01, 2022: Intellia, Regeneron report positive Phase I transthyretin amyloidosis data
Feb 17, 2022: Intellia Therapeutics announces two upcoming investor events in February 2022
Jan 24, 2022: Eplontersen granted Orphan Drug Designation in the US for transthyretin 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, 2022
Number of Products under Development by Therapy Areas, 2022
Number of Products under Development by Indication, 2022
Number of Products under Development by Companies, 2022
Products under Development by Companies, 2022
Products under Development by Companies, 2022 (Contd..1)
Products under Development by Companies, 2022 (Contd..2)
Number of Products under Investigation by Universities/Institutes, 2022
Products under Investigation by Universities/Institutes, 2022
Number of Products by Stage and Mechanism of Actions, 2022
Number of Products by Stage and Route of Administration, 2022
Number of Products by Stage and Molecule Type, 2022
Pipeline by ADRx Inc, 2022
Pipeline by Akcea Therapeutics Inc, 2022
Pipeline by Alexion Pharmaceuticals Inc, 2022
Pipeline by Alnylam Pharmaceuticals Inc, 2022
Pipeline by Autotac Bio Inc, 2022
Pipeline by BridgeBio Pharma Inc, 2022
Pipeline by BSIM Therapeutics SA, 2022
Pipeline by Corino Therapeutics Inc, 2022
Pipeline by Covalent Bioscience Inc, 2022
Pipeline by Galmed Pharmaceuticals Ltd, 2022
Pipeline by Novo Nordisk AS, 2022
Pipeline by Proclara Biosciences Inc, 2022
Pipeline by Regeneron Pharmaceuticals Inc, 2022
Dormant Projects, 2022
Discontinued Products, 2022

List Of Figures

LIST OF FIGURES

Number of Products under Development by Stage of Development, 2022
Number of Products under Development by Therapy Areas, 2022
Number of Products under Development by Top 10 Indications, 2022
Number of Products by Mechanism of Actions, 2022
Number of Products by Stage and Mechanism of Actions, 2022
Number of Products by Routes of Administration, 2022
Number of Products by Stage and Routes of Administration, 2022
Number of Products by Molecule Types, 2022
Number of Products by Stage and Molecule Types, 2022