Transthyretin – Pipeline Review, H2 2019

“Global Markets Direct’s, ‘Transthyretin (ATTR or Prealbumin or TBPA or TTR)- Pipeline Review, H2 2019’, provides in depth analysis on Transthyretin (ATTR or Prealbumin or TBPA or TTR) targeted pipeline therapeutics. The report provides comprehensive information complete with analysis by indications, stage of development, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Additionally, the report provides an overview of key players involved in Transthyretin (ATTR or Prealbumin or TBPA or TTR) targeted therapeutics development and features dormant and discontinued projects. The report analyses the pipeline products from therapy areas Metabolic Disorders and Central Nervous System under development targeting Transthyretin (ATTR or Prealbumin or TBPA or TTR).

Global Markets Direct’s report features investigational drugs from across globe covering over 20 therapy areas and nearly 3,000 indications. The report is built using data and information sourced from Global Markets Direct’s 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. Drug profiles featured in the report undergoes periodic review following a stringent set of processes to ensure that all the profiles are updated with the latest set of information. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

The report helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage.

Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data.

*Updated report will be delivered in 48 hours of order confirmation.”

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

Companies mentioned

“ADRx Inc

Akcea Therapeutics Inc

Alnylam Pharmaceuticals Inc

Corino Therapeutics Inc

Covalent Bioscience Inc

Ebelle Pharmaceuticals Inc

Eidos Therapeutics Inc

Neurimmune Holding AG

Pfizer Inc

Precision Biosciences Inc

Proclara Biosciences Inc

Prothena Corp Plc

Regeneron Pharmaceuticals Inc

Trucode Gene Repair Inc”

Table of Contents

Table of Contents

List of Tables

List of Figures

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 ONPATTROâ„¢ (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

“List of Tables

Number of Products under Development by Stage of Development, H2 2019

Number of Products under Development by Therapy Areas, H2 2019

Number of Products under Development by Indication, H2 2019

Number of Products under Development by Companies, H2 2019

Products under Development by Companies, H2 2019

Products under Development by Companies, H2 2019 (Contd..1), H2 2019

Number of Products under Investigation by Universities/Institutes, H2 2019

Products under Investigation by Universities/Institutes, H2 2019

Number of Products by Stage and Mechanism of Actions, H2 2019

Number of Products by Stage and Route of Administration, H2 2019

Number of Products by Stage and Molecule Type, H2 2019

Pipeline by ADRx Inc, H2 2019

Pipeline by Akcea Therapeutics Inc, H2 2019

Pipeline by Alnylam Pharmaceuticals Inc, H2 2019

Pipeline by Corino Therapeutics Inc, H2 2019

Pipeline by Covalent Bioscience Inc, H2 2019

Pipeline by Ebelle Pharmaceuticals Inc, H2 2019

Pipeline by Eidos Therapeutics Inc, H2 2019

Pipeline by Neurimmune Holding AG, H2 2019

Pipeline by Pfizer Inc, H2 2019

Pipeline by Precision Biosciences Inc, H2 2019

Pipeline by Proclara Biosciences Inc, H2 2019

Pipeline by Prothena Corp Plc, H2 2019

Pipeline by Regeneron Pharmaceuticals Inc, H2 2019

Pipeline by Trucode Gene Repair Inc, H2 2019

Dormant Projects, H2 2019

Discontinued Products, H2 2019”

List of Figures

“List of Figures

Number of Products under Development by Stage of Development, H2 2019

Number of Products under Development by Therapy Areas, H2 2019

Number of Products under Development by Top 10 Indications, H2 2019

Number of Products by Mechanism of Actions, H2 2019

Number of Products by Stage and Mechanism of Actions, H2 2019

Number of Products by Routes of Administration, H2 2019

Number of Products by Stage and Routes of Administration, H2 2019

Number of Products by Molecule Types, H2 2019

Number of Products by Stage and Molecule Types, H2 2019”

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