Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Pipeline Review, H2 2017
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Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Pipeline Review, H2 2017
Summary
According to the recently published report Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Pipeline Review, H2 2017; Transthyretin (ATTR or Prealbumin or TBPA or TTR) pipeline Target constitutes close to 15 molecules. Out of which approximately 12 molecules are developed by companies and remaining by the universities/institutes.
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.
The report Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Pipeline Review, H2 2017 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; that are being developed by Companies / Universities.
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. Currently, The molecules developed by companies in Filing rejected/Withdrawn, Phase III, Phase II, Phase I and Preclinical stages are 1, 2, 2, 2 and 5 respectively. Similarly, the universities portfolio in Phase II, Preclinical and Discovery stages comprises 1, 1 and 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, Alzheimers Disease, Cardiomyopathy and Neuropathy.
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 its 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
Summary
According to the recently published report Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Pipeline Review, H2 2017; Transthyretin (ATTR or Prealbumin or TBPA or TTR) pipeline Target constitutes close to 15 molecules. Out of which approximately 12 molecules are developed by companies and remaining by the universities/institutes.
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.
The report Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Pipeline Review, H2 2017 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; that are being developed by Companies / Universities.
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. Currently, The molecules developed by companies in Filing rejected/Withdrawn, Phase III, Phase II, Phase I and Preclinical stages are 1, 2, 2, 2 and 5 respectively. Similarly, the universities portfolio in Phase II, Preclinical and Discovery stages comprises 1, 1 and 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, Alzheimers Disease, Cardiomyopathy and Neuropathy.
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 its 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
TABLE OF CONTENTS
Table of Contents
Table of Contents 2
List of Tables 5
List of Figures 5
Introduction 6
Global Markets Direct Report Coverage 6
Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Overview 7
Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Therapeutics Development 8
Products under Development by Stage of Development 8
Products under Development by Therapy Area 9
Products under Development by Indication 10
Products under Development by Companies 11
Products under Development by Universities/Institutes 14
Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Therapeutics Assessment 16
Assessment by Mechanism of Action 16
Assessment by Route of Administration 18
Assessment by Molecule Type 20
Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Companies Involved in Therapeutics Development 22
Alnylam Pharmaceuticals Inc 22
Arcturus Therapeutics Inc 22
Ionis Pharmaceuticals Inc 23
Neurimmune Holding AG 23
Pfizer Inc 24
Prothena Corp Plc 24
Regeneron Pharmaceuticals Inc 24
Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Drug Profiles 26
AG-10 - Drug Profile 26
Product Description 26
Mechanism Of Action 26
R&D Progress 26
ALN-TTRsc02 - Drug Profile 27
Product Description 27
Mechanism Of Action 27
R&D Progress 27
CRX-1008 - Drug Profile 29
Product Description 29
Mechanism Of Action 29
R&D Progress 29
doxycycline hyclate - Drug Profile 31
Product Description 31
Mechanism Of Action 31
R&D Progress 31
EDE-1307 - Drug Profile 32
Product Description 32
Mechanism Of Action 32
R&D Progress 32
inotersen sodium - Drug Profile 33
Product Description 33
Mechanism Of Action 33
R&D Progress 33
LUNAR-TTR - Drug Profile 38
Product Description 38
Mechanism Of Action 38
R&D Progress 38
Monoclonal Antibody to Inhibit ATTR for Familial Amyloid Polyneuropathy - Drug Profile 39
Product Description 39
Mechanism Of Action 39
R&D Progress 39
NI-301 - Drug Profile 40
Product Description 40
Mechanism Of Action 40
R&D Progress 40
NPT-189 - Drug Profile 41
Product Description 41
Mechanism Of Action 41
R&D Progress 41
Oligonucleotides to Inhibit Transthyretin for Amyloidosis - Drug Profile 42
Product Description 42
Mechanism Of Action 42
R&D Progress 42
patisiran - Drug Profile 43
Product Description 43
Mechanism Of Action 43
R&D Progress 43
PRX-004 - Drug Profile 52
Product Description 52
Mechanism Of Action 52
R&D Progress 52
Small Molecules to Inhibit Transthyretin for Familial Amyloid Polyneuropathy - Drug Profile 53
Product Description 53
Mechanism Of Action 53
R&D Progress 53
tafamidis meglumine - Drug Profile 54
Product Description 54
Mechanism Of Action 54
R&D Progress 54
Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Dormant Products 58
Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Discontinued Products 59
Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Product Development Milestones 60
Featured News & Press Releases 60
Sep 28, 2017: Eidos Therapeutics Initiates First Clinical Study for AG10 Targeting Transthyretin Amyloidosis, Appoints Camille Landis as Chief Business Officer 60
Sep 20, 2017: Arbutus LNP Licensee Alnylam Announces Positive Phase 3 Results for LNP-Enabled Patisiran Program 60
Sep 20, 2017: Sanofi and Alnylam Report Positive Topline Results from APOLLO Phase 3 Study of Patisiran in Hereditary ATTR (hATTR) Amyloidosis Patients with Polyneuropathy 61
Aug 31, 2017: Amyloid Publishes Long-term Data Analysis From Pfizer Suggesting Tafamidis Delays Progression of TTR-FAP, a Rare Disease 62
Jul 18, 2017: Proclara Biosciences Presents New Preclinical Data Supporting the Development of NPT189 at Alzheimers Association International Conference (AAIC) 2017 64
Jul 17, 2017: Proclara Biosciences Provides Update on NPT189 65
Jul 10, 2017: New Data Presented at Peripheral Nerve Society Meeting Further Support Potential Benefit of Inotersen 65
Jun 06, 2017: Pfizer Receives FDA Fast Track Designation for Tafamidis for Transthyretin Cardiomyopathy 66
May 15, 2017: Ionis Pharmaceuticals Announces Phase 3 NEURO-TTR Study of Inotersen (IONIS-TTR Rx) Meets Both Primary Endpoints 67
Apr 27, 2017: BridgeBio Pharma Launches Eidos Therapeutics to Develop Targeted Therapy for Fatal Heart Disease 68
Apr 26, 2017: Alnylam Reports Final 24-Month Results from Phase 2 Open-Label Extension Study of Patisiran, an Investigational RNAi Therapeutic in Development for the Treatment of Hereditary ATTR (hATTR) Amyloidosis 69
Mar 10, 2017: Proclara Biosciences Australia, a Subsidiary of Proclara Biosciences, Awarded Grant by Biopharmaceuticals Australia to Help Develop New Biotherapy with Blockbuster Potential 70
Nov 07, 2016: VYNDAQEL (tafamidis) Receives Approval in Brazil by ANVISA for the Treatment of Transthyretin Familial Amyloid Polyneuropathy 70
Oct 10, 2016: Data Monitoring Committee Recommends Continuation of APOLLO Phase 3 Clinical Trial of Patisiran for Hereditary ATTR Amyloidosis with Polyneuropathy (hATTR-PN) 71
Aug 08, 2016: Pfizer Announces Publication of New Analysis Showing Long-Term Therapy with VYNDAQEL (tafamidis) Slowed Progression of Rare Neurodegenerative Disease 71
Appendix 73
Methodology 73
Coverage 73
Secondary Research 73
Primary Research 73
Expert Panel Validation 73
Contact Us 73
Disclaimer 74
Number of Products under Development by Stage of Development, H2 2017 8
Number of Products under Development by Therapy Areas, H2 2017 9
Number of Products under Development by Indication, H2 2017 10
Number of Products under Development by Companies, H2 2017 11
Products under Development by Companies, H2 2017 12
Products under Development by Companies, H2 2017 (Contd..1), H2 2017 13
Number of Products under Investigation by Universities/Institutes, H2 2017 14
Products under Investigation by Universities/Institutes, H2 2017 15
Number of Products by Stage and Mechanism of Actions, H2 2017 17
Number of Products by Stage and Route of Administration, H2 2017 19
Number of Products by Stage and Molecule Type, H2 2017 21
Pipeline by Alnylam Pharmaceuticals Inc, H2 2017 22
Pipeline by Arcturus Therapeutics Inc, H2 2017 22
Pipeline by Ionis Pharmaceuticals Inc, H2 2017 23
Pipeline by Neurimmune Holding AG, H2 2017 23
Pipeline by Pfizer Inc, H2 2017 24
Pipeline by Prothena Corp Plc, H2 2017 24
Pipeline by Regeneron Pharmaceuticals Inc, H2 2017 25
Dormant Projects, H2 2017 58
Discontinued Products, H2 2017 59
Table of Contents 2
List of Tables 5
List of Figures 5
Introduction 6
Global Markets Direct Report Coverage 6
Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Overview 7
Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Therapeutics Development 8
Products under Development by Stage of Development 8
Products under Development by Therapy Area 9
Products under Development by Indication 10
Products under Development by Companies 11
Products under Development by Universities/Institutes 14
Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Therapeutics Assessment 16
Assessment by Mechanism of Action 16
Assessment by Route of Administration 18
Assessment by Molecule Type 20
Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Companies Involved in Therapeutics Development 22
Alnylam Pharmaceuticals Inc 22
Arcturus Therapeutics Inc 22
Ionis Pharmaceuticals Inc 23
Neurimmune Holding AG 23
Pfizer Inc 24
Prothena Corp Plc 24
Regeneron Pharmaceuticals Inc 24
Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Drug Profiles 26
AG-10 - Drug Profile 26
Product Description 26
Mechanism Of Action 26
R&D Progress 26
ALN-TTRsc02 - Drug Profile 27
Product Description 27
Mechanism Of Action 27
R&D Progress 27
CRX-1008 - Drug Profile 29
Product Description 29
Mechanism Of Action 29
R&D Progress 29
doxycycline hyclate - Drug Profile 31
Product Description 31
Mechanism Of Action 31
R&D Progress 31
EDE-1307 - Drug Profile 32
Product Description 32
Mechanism Of Action 32
R&D Progress 32
inotersen sodium - Drug Profile 33
Product Description 33
Mechanism Of Action 33
R&D Progress 33
LUNAR-TTR - Drug Profile 38
Product Description 38
Mechanism Of Action 38
R&D Progress 38
Monoclonal Antibody to Inhibit ATTR for Familial Amyloid Polyneuropathy - Drug Profile 39
Product Description 39
Mechanism Of Action 39
R&D Progress 39
NI-301 - Drug Profile 40
Product Description 40
Mechanism Of Action 40
R&D Progress 40
NPT-189 - Drug Profile 41
Product Description 41
Mechanism Of Action 41
R&D Progress 41
Oligonucleotides to Inhibit Transthyretin for Amyloidosis - Drug Profile 42
Product Description 42
Mechanism Of Action 42
R&D Progress 42
patisiran - Drug Profile 43
Product Description 43
Mechanism Of Action 43
R&D Progress 43
PRX-004 - Drug Profile 52
Product Description 52
Mechanism Of Action 52
R&D Progress 52
Small Molecules to Inhibit Transthyretin for Familial Amyloid Polyneuropathy - Drug Profile 53
Product Description 53
Mechanism Of Action 53
R&D Progress 53
tafamidis meglumine - Drug Profile 54
Product Description 54
Mechanism Of Action 54
R&D Progress 54
Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Dormant Products 58
Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Discontinued Products 59
Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Product Development Milestones 60
Featured News & Press Releases 60
Sep 28, 2017: Eidos Therapeutics Initiates First Clinical Study for AG10 Targeting Transthyretin Amyloidosis, Appoints Camille Landis as Chief Business Officer 60
Sep 20, 2017: Arbutus LNP Licensee Alnylam Announces Positive Phase 3 Results for LNP-Enabled Patisiran Program 60
Sep 20, 2017: Sanofi and Alnylam Report Positive Topline Results from APOLLO Phase 3 Study of Patisiran in Hereditary ATTR (hATTR) Amyloidosis Patients with Polyneuropathy 61
Aug 31, 2017: Amyloid Publishes Long-term Data Analysis From Pfizer Suggesting Tafamidis Delays Progression of TTR-FAP, a Rare Disease 62
Jul 18, 2017: Proclara Biosciences Presents New Preclinical Data Supporting the Development of NPT189 at Alzheimers Association International Conference (AAIC) 2017 64
Jul 17, 2017: Proclara Biosciences Provides Update on NPT189 65
Jul 10, 2017: New Data Presented at Peripheral Nerve Society Meeting Further Support Potential Benefit of Inotersen 65
Jun 06, 2017: Pfizer Receives FDA Fast Track Designation for Tafamidis for Transthyretin Cardiomyopathy 66
May 15, 2017: Ionis Pharmaceuticals Announces Phase 3 NEURO-TTR Study of Inotersen (IONIS-TTR Rx) Meets Both Primary Endpoints 67
Apr 27, 2017: BridgeBio Pharma Launches Eidos Therapeutics to Develop Targeted Therapy for Fatal Heart Disease 68
Apr 26, 2017: Alnylam Reports Final 24-Month Results from Phase 2 Open-Label Extension Study of Patisiran, an Investigational RNAi Therapeutic in Development for the Treatment of Hereditary ATTR (hATTR) Amyloidosis 69
Mar 10, 2017: Proclara Biosciences Australia, a Subsidiary of Proclara Biosciences, Awarded Grant by Biopharmaceuticals Australia to Help Develop New Biotherapy with Blockbuster Potential 70
Nov 07, 2016: VYNDAQEL (tafamidis) Receives Approval in Brazil by ANVISA for the Treatment of Transthyretin Familial Amyloid Polyneuropathy 70
Oct 10, 2016: Data Monitoring Committee Recommends Continuation of APOLLO Phase 3 Clinical Trial of Patisiran for Hereditary ATTR Amyloidosis with Polyneuropathy (hATTR-PN) 71
Aug 08, 2016: Pfizer Announces Publication of New Analysis Showing Long-Term Therapy with VYNDAQEL (tafamidis) Slowed Progression of Rare Neurodegenerative Disease 71
Appendix 73
Methodology 73
Coverage 73
Secondary Research 73
Primary Research 73
Expert Panel Validation 73
Contact Us 73
Disclaimer 74
List Of Tables
Number of Products under Development by Stage of Development, H2 2017 8
Number of Products under Development by Therapy Areas, H2 2017 9
Number of Products under Development by Indication, H2 2017 10
Number of Products under Development by Companies, H2 2017 11
Products under Development by Companies, H2 2017 12
Products under Development by Companies, H2 2017 (Contd..1), H2 2017 13
Number of Products under Investigation by Universities/Institutes, H2 2017 14
Products under Investigation by Universities/Institutes, H2 2017 15
Number of Products by Stage and Mechanism of Actions, H2 2017 17
Number of Products by Stage and Route of Administration, H2 2017 19
Number of Products by Stage and Molecule Type, H2 2017 21
Pipeline by Alnylam Pharmaceuticals Inc, H2 2017 22
Pipeline by Arcturus Therapeutics Inc, H2 2017 22
Pipeline by Ionis Pharmaceuticals Inc, H2 2017 23
Pipeline by Neurimmune Holding AG, H2 2017 23
Pipeline by Pfizer Inc, H2 2017 24
Pipeline by Prothena Corp Plc, H2 2017 24
Pipeline by Regeneron Pharmaceuticals Inc, H2 2017 25
Dormant Projects, H2 2017 58
Discontinued Products, H2 2017 59
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