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Published: Mar, 2015 | Pages:
101 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)
Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Pipeline Review, H1 2015 Summary Global Markets Direct's, 'Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Pipeline Review, H1 2015', provides an overview of the Familial Hypercholesterolemia (Type II Hyperlipoproteinemia)'s therapeutic pipeline. This report provides comprehensive information on the therapeutic development for Familial Hypercholesterolemia (Type II Hyperlipoproteinemia), complete with comparative analysis at various stages, therapeutics assessment by drug target, mechanism of action (MoA), route of administration (RoA) and molecule type, along with latest updates, and featured news and press releases. It also reviews key players involved in the therapeutic development for Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) and special features on late-stage and discontinued projects. 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, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources, put together by Global Markets Direct's team. Drug profiles/records featured in the report undergoes periodic updation following a stringent set of processes that ensures that all the profiles are updated with the latest set of information. Additionally, processes including live news & deals tracking, browser based alert-box and clinical trials registries tracking ensure that the most recent developments are captured on a real time basis. The report enhances decision making capabilities and help to create effective counter strategies to gain competitive advantage. It strengthens R&D pipelines by identifying new targets and MOAs to produce first-in-class and best-in-class products. Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data for the indicated disease. Scope - The report provides a snapshot of the global therapeutic landscape of Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - The report reviews key pipeline products under drug profile section which includes, product description, MoA and R&D brief, licensing and collaboration details & other developmental activities - The report reviews key players involved in the therapeutics development for Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) and enlists all their major and minor projects - The report summarizes all the dormant and discontinued pipeline projects - A review of the Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) products under development by companies and universities/research institutes based on information derived from company and industry-specific sources - Pipeline products coverage based on various stages of development ranging from pre-registration till discovery and undisclosed stages - A detailed assessment of monotherapy and combination therapy pipeline projects - Coverage of the Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) pipeline on the basis of target, MoA, route of administration and molecule type - Latest news and deals relating related to pipeline products Reasons To Buy - Provides strategically significant competitor information, analysis, and insights to formulate effective R&D development strategies - Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage - Develop strategic initiatives by understanding the focus areas of leading companies - Identify and understand important and diverse types of therapeutics under development for Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Plan mergers and acquisitions effectively by identifying key players of the most promising pipeline - Devise corrective measures for pipeline projects by understanding Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) 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 - Modify the therapeutic portfolio by identifying discontinued projects and understanding the factors that drove them from pipeline
Table of Contents Table of Contents 2 List of Tables 5 List of Figures 6 Introduction 7 Global Markets Direct Report Coverage 7 Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) Overview 8 Therapeutics Development 9 Pipeline Products for Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Overview 9 Pipeline Products for Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Comparative Analysis 10 Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Therapeutics under Development by Companies 11 Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Therapeutics under Investigation by Universities/Institutes 13 Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Pipeline Products Glance 14 Late Stage Products 14 Clinical Stage Products 15 Early Stage Products 16 Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Products under Development by Companies 17 Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Products under Investigation by Universities/Institutes 19 Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Companies Involved in Therapeutics Development 20 Aegerion Pharmaceuticals, Inc. 20 Amgen Inc. 21 Cerenis Therapeutics Holding SA 22 CymaBay Therapeutics, Inc. 23 Merck & Co., Inc. 24 Pfizer Inc. 25 Regeneron Pharmaceuticals, Inc. 26 ReGenX Biosciences, LLC 27 Sanofi 28 Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Therapeutics Assessment 29 Assessment by Monotherapy Products 29 Assessment by Target 30 Assessment by Mechanism of Action 32 Assessment by Route of Administration 34 Assessment by Molecule Type 36 Drug Profiles 38 AEM-28 - Drug Profile 38 Product Description 38 Mechanism of Action 38 R&D Progress 38 alirocumab - Drug Profile 39 Product Description 39 Mechanism of Action 39 R&D Progress 39 AM-0010 - Drug Profile 43 Product Description 43 Mechanism of Action 43 R&D Progress 43 anacetrapib - Drug Profile 44 Product Description 44 Mechanism of Action 44 R&D Progress 44 Antisense Oligonucleotides to Inhibit ApoB100 for Familial Hypercholesterolemia - Drug Profile 46 Product Description 46 Mechanism of Action 46 R&D Progress 46 bococizumab - Drug Profile 47 Product Description 47 Mechanism of Action 47 R&D Progress 47 BSN-272 - Drug Profile 48 Product Description 48 Mechanism of Action 48 R&D Progress 48 CER-001 - Drug Profile 49 Product Description 49 Mechanism of Action 49 R&D Progress 49 evinacumab - Drug Profile 51 Product Description 51 Mechanism of Action 51 R&D Progress 51 evolocumab - Drug Profile 52 Product Description 52 Mechanism of Action 52 R&D Progress 52 Gene Therapy to Activate LDL Receptor for Homozygous Familial Hypercholesterolemia - Drug Profile 55 Product Description 55 Mechanism of Action 55 R&D Progress 55 lomitapide mesylate - Drug Profile 56 Product Description 56 Mechanism of Action 56 R&D Progress 56 MBX-8025 - Drug Profile 58 Product Description 58 Mechanism of Action 58 R&D Progress 58 mipomersen sodium - Drug Profile 60 Product Description 60 Mechanism of Action 60 R&D Progress 60 Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Recent Pipeline Updates 62 Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Dormant Projects 87 Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Discontinued Products 88 Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Product Development Milestones 89 Featured News & Press Releases 89 Jan 28, 2015: Aegerion Investigated by Former Louisiana Attorney General and Special Prosecutor Richard P. Ieyoub and the Firm of Ieyoub O'Bell & Myers 89 Nov 18, 2014: Genzyme And Isis Present KYNAMRO Clinical Data At The American Heart Association 89 Sep 02, 2014: Isis Pharmaceuticals Presents Data On KYNAMRO At European Society of Cardiology Congress 2014 90 Sep 02, 2014: Isis Pharmaceuticals Presents Data On KYNAMRO At European Society of Cardiology Congress 2014 91 Aug 27, 2014: Aegerion Pharmaceuticals Announces Resolution of Warning Letter for JUXTAPID Capsules 92 Jun 02, 2014: Cerenis Reports Two Positive Phase II Studies for HDL Mimetic CER-001 at EAS 92 May 08, 2014: Genzyme and Isis Receive Corporate Award from the National Organization for Rare Disorders for the Development of KYNAMRO 93 Apr 09, 2014: Aegerion Pharmaceuticals Initiates Phase 3 Clinical Trial in Japan to Evaluate Lomitapide for Treatment of Homozygous Familial Hypercholesterolemia 94 Mar 30, 2014: Data From Phase 3 Pivotal Studies Show Amgen's Novel Investigational Cholesterol-Lowering Medication Evolocumab Significantly Reduced LDL Cholesterol In Statin Intolerant Patients And In Patients On Statins 95 Mar 27, 2014: Sanofi and Regeneron To Present Alirocumab Clinical Data at the American College of Cardiology's 63rd Annual Scientific Session 98 Appendix 100 Methodology 100 Coverage 100 Secondary Research 100 Primary Research 100 Expert Panel Validation 100 Contact Us 100 Disclaimer 101
List of Tables Number of Products under Development for Familial Hypercholesterolemia (Type II Hyperlipoproteinemia), H1 2015 9 Number of Products under Development for Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Comparative Analysis, H1 2015 10 Number of Products under Development by Companies, H1 2015 12 Number of Products under Investigation by Universities/Institutes, H1 2015 13 Comparative Analysis by Late Stage Development, H1 2015 14 Comparative Analysis by Clinical Stage Development, H1 2015 15 Comparative Analysis by Early Stage Development, H1 2015 16 Products under Development by Companies, H1 2015 17 Products under Development by Companies, H1 2015 (Contd..1) 18 Products under Investigation by Universities/Institutes, H1 2015 19 Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Pipeline by Aegerion Pharmaceuticals, Inc., H1 2015 20 Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Pipeline by Amgen Inc., H1 2015 21 Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Pipeline by Cerenis Therapeutics Holding SA, H1 2015 22 Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Pipeline by CymaBay Therapeutics, Inc., H1 2015 23 Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Pipeline by Merck & Co., Inc., H1 2015 24 Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Pipeline by Pfizer Inc., H1 2015 25 Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Pipeline by Regeneron Pharmaceuticals, Inc., H1 2015 26 Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Pipeline by ReGenX Biosciences, LLC, H1 2015 27 Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Pipeline by Sanofi, H1 2015 28 Assessment by Monotherapy Products, H1 2015 29 Number of Products by Stage and Target, H1 2015 31 Number of Products by Stage and Mechanism of Action, H1 2015 33 Number of Products by Stage and Route of Administration, H1 2015 35 Number of Products by Stage and Molecule Type, H1 2015 37 Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) Therapeutics - Recent Pipeline Updates, H1 2015 62 Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Dormant Projects, H1 2015 87 Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Discontinued Products, H1 2015 88
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