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Published: Jun, 2017 | Pages:
68 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)
Familial Amyloid Neuropathies - Pipeline Review, H1 2017 Summary Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Familial Amyloid Neuropathies - Pipeline Review, H1 2017, provides an overview of the Familial Amyloid Neuropathies (Metabolic Disorders) pipeline landscape. Familial amyloid neuropathy is a slowly progressive condition characterized by the buildup of abnormal deposits of a protein called amyloid (amyloidosis) in the body's organs and tissues. Symptoms include numbness, tingling, pins and needles in the feet and hands, weakness and pain in the arms and legs, loss of sensation, urinary retention, reduced sweating, ankle swelling, fatigue, nausea, weight loss, dizziness and fainting. Supportive treatment includes diuretics, calcium channel blockers, beta blockers, angiotensin receptor blockers and angiotensin converting enzyme (ACE) inhibitors. Report Highlights Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Familial Amyloid Neuropathies - Pipeline Review, H1 2017, provides comprehensive information on the therapeutics under development for Familial Amyloid Neuropathies (Metabolic Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. The Familial Amyloid Neuropathies (Metabolic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Familial Amyloid Neuropathies and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Filing rejected/Withdrawn, Phase III, Phase II, Preclinical and Discovery stages are 1, 2, 2, 1 and 1 respectively. Similarly, the Universities portfolio in Phase II, Preclinical and Discovery stages comprises 1, 2 and 1 molecules, respectively. Familial Amyloid Neuropathies (Metabolic Disorders) pipeline guide 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. The guide 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. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis. Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data. Scope - The pipeline guide provides a snapshot of the global therapeutic landscape of Familial Amyloid Neuropathies (Metabolic Disorders). - The pipeline guide reviews pipeline therapeutics for Familial Amyloid Neuropathies (Metabolic Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources. - The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages. - The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities. - The pipeline guide reviews key companies involved in Familial Amyloid Neuropathies (Metabolic Disorders) therapeutics and enlists all their major and minor projects. - The pipeline guide evaluates Familial Amyloid Neuropathies (Metabolic Disorders) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type. - The pipeline guide encapsulates all the dormant and discontinued pipeline projects. - The pipeline guide reviews latest news related to pipeline therapeutics for Familial Amyloid Neuropathies (Metabolic Disorders) Reasons To Buy - Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies. - Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage. - Find and recognize significant and varied types of therapeutics under development for Familial Amyloid Neuropathies (Metabolic Disorders). - Classify potential new clients or partners in the target demographic. - Develop tactical initiatives by understanding the focus areas of leading companies. - Plan mergers and acquisitions meritoriously by identifying key players and it's most promising pipeline therapeutics. - Formulate corrective measures for pipeline projects by understanding Familial Amyloid Neuropathies (Metabolic Disorders) pipeline depth and focus of Indication therapeutics. - 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. - Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.
Table of Contents
List of Tables List of Figures Introduction Global Markets Direct Report Coverage Familial Amyloid Neuropathies - Overview Familial Amyloid Neuropathies - Therapeutics Development Pipeline Overview Pipeline by Companies Pipeline by Universities/Institutes Products under Development by Companies Products under Development by Universities/Institutes Familial Amyloid Neuropathies - Therapeutics Assessment Assessment by Target Assessment by Mechanism of Action Assessment by Route of Administration Assessment by Molecule Type Familial Amyloid Neuropathies - Companies Involved in Therapeutics Development Alnylam Pharmaceuticals Inc Bsim2 Ionis Pharmaceuticals Inc Pfizer Inc SOM Biotech SL Familial Amyloid Neuropathies - Drug Profiles AG-10 - Drug Profile Product Description Mechanism Of Action R&D Progress CLR-01 - 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 Monoclonal Antibody to Inhibit ATTR for Familial Amyloid Polyneuropathy - Drug Profile Product Description Mechanism Of Action R&D Progress patisiran - Drug Profile Product Description Mechanism Of Action R&D Progress Small Molecules to Inhibit Transthyretin for Familial Amyloid Polyneuropathy - Drug Profile Product Description Mechanism Of Action R&D Progress Small Molecules to Inhibit Transthyretin Related Amyloid Protein for Familial Amyloidotic Polyneuropathy - 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 Familial Amyloid Neuropathies - Dormant Projects Familial Amyloid Neuropathies - Discontinued Products Familial Amyloid Neuropathies - Product Development Milestones Featured News & Press Releases May 15, 2017: Ionis Pharmaceuticals Announces Phase 3 NEURO-TTR Study of Inotersen (IONIS-TTR Rx) Meets Both Primary Endpoints 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 Oct 10, 2016: Data Monitoring Committee Recommends Continuation of APOLLO Phase 3 Clinical Trial of Patisiran for Hereditary ATTR Amyloidosis with Polyneuropathy (hATTR-PN) Jul 07, 2016: Ionis Pharmaceuticals Reports Positive Clinical Data on IONIS-TTR Rx at the XV International Symposium on Amyloidosis Jul 01, 2016: Alnylam Reports New Results on patisiran at the XV International Symposium on Amyloidosis Jun 27, 2016: Alnylam to Report New Patisiran Results at the XV International Symposium on Amyloidosis May 26, 2016: Ionis Pharmaceuticals Provides Update on IONIS-TTR Rx Program Apr 20, 2016: Alnylam Reports Complete 18-Month Data from Ongoing Phase 2 Open-Label Extension Study of Patisiran, an Investigational RNAi Therapeutic Targeting Transthyretin for the Treatment of Hereditary ATTR Amyloidosis with Polyneuropathy (hATTR-PN) Apr 07, 2016: Ionis Pharmaceuticals Provides Update on IONIS-TTRRX Program Feb 01, 2016: Alnylam Completes Enrollment in APOLLO Phase 3 Study with Patisiran, an Investigational RNAi Therapeutic for Patients with Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis) Dec 17, 2015: Isis Pharmaceuticals Announces Initiation of an Investigator-Sponsored Phase 2 Study of ISIS-TTR Rx in Patients with TTR Cardiomyopathy Amyloidosis Nov 03, 2015: Isis Pharmaceuticals Reports an Update on ISIS-TTR Rx, Including Positive Data from Multiple Clinical Studies, Presented at the EC-ATTR Meeting Nov 03, 2015: Alnylam Reports Positive Results from Ongoing Phase 2 Open-Label Extension Studies for Patisiran in Development for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis) Oct 27, 2015: Isis Pharmaceuticals to Host Webcast to Review ISIS-TTR Data Presented at 1st European Congress of Hereditary ATTR Amyloidosis and Provide Phase 3 Development Plan Update Sep 28, 2015: Alnylam and Collaborators Report First-Ever Evidence that Patisiran Reduces Pathogenic, Misfolded Transthyretin Monomers and Oligomers in TTR-Mediated Amyloidosis Patients with Familial Amyloidotic Polyneuropathy Appendix Methodology Coverage Secondary Research Primary Research Expert Panel Validation Contact Us Disclaimer
List of Tables
Number of Products under Development for Familial Amyloid Neuropathies, H1 2017 Number of Products under Development by Companies, H1 2017 Number of Products under Development by Universities/Institutes, H1 2017 Products under Development by Companies, H1 2017 Products under Development by Universities/Institutes, H1 2017 Number of Products by Stage and Target, H1 2017 Number of Products by Stage and Mechanism of Action, H1 2017 Number of Products by Stage and Route of Administration, H1 2017 Number of Products by Stage and Molecule Type, H1 2017 Familial Amyloid Neuropathies - Pipeline by Alnylam Pharmaceuticals Inc, H1 2017 Familial Amyloid Neuropathies - Pipeline by Bsim2, H1 2017 Familial Amyloid Neuropathies - Pipeline by Ionis Pharmaceuticals Inc, H1 2017 Familial Amyloid Neuropathies - Pipeline by Pfizer Inc, H1 2017 Familial Amyloid Neuropathies - Pipeline by SOM Biotech SL, H1 2017 Familial Amyloid Neuropathies - Dormant Projects, H1 2017 Familial Amyloid Neuropathies - Discontinued Products, H1 2017
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