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Published: Oct, 2017 | Pages:
74 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)
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 Currently, The molecules developed by companies in Filing rejected/Withdrawn, Phase III, Phase II, Phase I and Preclinical stages are 1, 2, 2, 2 and 5 respectively.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, Alzheimer's 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 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.
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 Alzheimer's 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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