Global Markets Direct’s, ‘Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) – Pipeline Review, H2 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 6
List of Figures 7
Introduction 8
Global Markets Direct Report Coverage 8
Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) Overview 9
Therapeutics Development 10
Pipeline Products for Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Overview 10
Pipeline Products for Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Comparative Analysis 11
Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Therapeutics under Development by Companies 12
Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Therapeutics under Investigation by Universities/Institutes 14
Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Pipeline Products Glance 15
Late Stage Products 15
Clinical Stage Products 16
Early Stage Products 17
Unknown Stage Products 18
Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Products under Development by Companies 19
Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Products under Investigation by Universities/Institutes 21
Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Companies Involved in Therapeutics Development 22
Aegerion Pharmaceuticals, Inc. 22
Cerenis Therapeutics Holding SA 23
CymaBay Therapeutics, Inc. 24
Gemphire Therapeutics Inc. 25
Merck & Co., Inc. 26
Pfizer Inc. 27
Regeneron Pharmaceuticals, Inc. 28
RegenxBio Inc. 29
Sanofi 30
Thetis Pharmaceuticals LLC 31
Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Therapeutics Assessment 32
Assessment by Monotherapy Products 32
Assessment by Combination Products 33
Assessment by Target 34
Assessment by Mechanism of Action 36
Assessment by Route of Administration 38
Assessment by Molecule Type 40
Drug Profiles 42
(ezetimibe + atorvastatin calcium) - Drug Profile 42
Product Description 42
Mechanism of Action 42
R&D Progress 42
AEM-28 - Drug Profile 43
Product Description 43
Mechanism of Action 43
R&D Progress 43
AEM-28-02 - Drug Profile 44
Product Description 44
Mechanism of Action 44
R&D Progress 44
AEM-2814 - Drug Profile 45
Product Description 45
Mechanism of Action 45
R&D Progress 45
alirocumab - Drug Profile 46
Product Description 46
Mechanism of Action 46
R&D Progress 46
AM-0010 - Drug Profile 50
Product Description 50
Mechanism of Action 50
R&D Progress 50
anacetrapib - Drug Profile 51
Product Description 51
Mechanism of Action 51
R&D Progress 51
Antisense Oligonucleotides to Inhibit ApoB100 for Familial Hypercholesterolemia - Drug Profile 53
Product Description 53
Mechanism of Action 53
R&D Progress 53
bococizumab - Drug Profile 54
Product Description 54
Mechanism of Action 54
R&D Progress 54
BSN-272 - Drug Profile 55
Product Description 55
Mechanism of Action 55
R&D Progress 55
CER-001 - Drug Profile 56
Product Description 56
Mechanism of Action 56
R&D Progress 56
evinacumab - Drug Profile 58
Product Description 58
Mechanism of Action 58
R&D Progress 58
gemcabene calcium - Drug Profile 59
Product Description 59
Mechanism of Action 59
R&D Progress 59
lomitapide mesylate - Drug Profile 60
Product Description 60
Mechanism of Action 60
R&D Progress 60
MBX-8025 - Drug Profile 63
Product Description 63
Mechanism of Action 63
R&D Progress 63
mipomersen sodium - Drug Profile 65
Product Description 65
Mechanism of Action 65
R&D Progress 65
RGX-501 - Drug Profile 67
Product Description 67
Mechanism of Action 67
R&D Progress 67
TP-452 - Drug Profile 68
Product Description 68
Mechanism of Action 68
R&D Progress 68
Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Recent Pipeline Updates 69
Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Dormant Projects 90
Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Discontinued Products 91
Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Product Development Milestones 92
Featured News & Press Releases 92
Sep 01, 2015: Sanofi And Regeneron Announce New Positive Praluent Phase 3 Data Presented At Esc Congress 2015 92
Aug 03, 2015: Isis Reports Positive Data from KYNAMRO (mipomersen sodium) FOCUS FH Phase 3 Study in Patients with Severe Heterozygous Familial Hypercholesterolemia 93
Jul 24, 2015: Sanofi And Regeneron Announce FDA Approval Of Praluent (Alirocumab) Injection, The First Pcsk9 Inhibitor In The U.S., For The Treatment Of High LDL Cholesterol In Adult Patients 94
May 26, 2015: Aegerion Pharmaceuticals Achieves Primary Endpoint in Phase III Study of Lomitapide in Japanese Patients With Homozygous Familial Hypercholesterolemia (HoFH) 96
Apr 30, 2015: AOP Orphan Launches Lojuxta (lomitapide) for Treatment of Homozygous Familial Hypercholesterolaemia to the Austrian Market 96
Apr 23, 2015: CymaBay Therapeutics Announces the Initiation of a Phase 2 Study of MBX-8025 in Patients With Homozygous Familial Hypercholesterolemia 98
Mar 25, 2015: CymaBay Therapeutics Announces U.S. Orphan Drug Designation for MBX-8025 in Homozygous Familial Hypercholesterolemia 99
Jan 28, 2015: Aegerion Investigated by Former Louisiana Attorney General and Special Prosecutor Richard P. Ieyoub and the Firm of Ieyoub O'Bell & Myers 99
Nov 18, 2014: Genzyme And Isis Present KYNAMRO Clinical Data At The American Heart Association 100
Sep 02, 2014: Isis Pharmaceuticals Presents Data On KYNAMRO At European Society of Cardiology Congress 2014 101
Appendix 103
Methodology 103
Coverage 103
Secondary Research 103
Primary Research 103
Expert Panel Validation 103
Contact Us 103
Disclaimer 104

List of Tables

Number of Products under Development for Familial Hypercholesterolemia (Type II Hyperlipoproteinemia), H2 2015 10
Number of Products under Development for Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Comparative Analysis, H2 2015 11
Number of Products under Development by Companies, H2 2015 13
Number of Products under Investigation by Universities/Institutes, H2 2015 14
Comparative Analysis by Late Stage Development, H2 2015 15
Comparative Analysis by Clinical Stage Development, H2 2015 16
Comparative Analysis by Early Stage Development, H2 2015 17
Comparative Analysis by Unknown Stage Development, H2 2015 18
Products under Development by Companies, H2 2015 19
Products under Development by Companies, H2 2015 (Contd...1) 20
Products under Investigation by Universities/Institutes, H2 2015 21
Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Pipeline by Aegerion Pharmaceuticals, Inc., H2 2015 22
Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Pipeline by Cerenis Therapeutics Holding SA, H2 2015 23
Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Pipeline by CymaBay Therapeutics, Inc., H2 2015 24
Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Pipeline by Gemphire Therapeutics Inc., H2 2015 25
Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Pipeline by Merck & Co., Inc., H2 2015 26
Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Pipeline by Pfizer Inc., H2 2015 27
Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Pipeline by Regeneron Pharmaceuticals, Inc., H2 2015 28
Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Pipeline by RegenxBio Inc., H2 2015 29
Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Pipeline by Sanofi, H2 2015 30
Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Pipeline by Thetis Pharmaceuticals LLC, H2 2015 31
Assessment by Monotherapy Products, H2 2015 32
Assessment by Combination Products, H2 2015 33
Number of Products by Stage and Target, H2 2015 35
Number of Products by Stage and Mechanism of Action, H2 2015 37
Number of Products by Stage and Route of Administration, H2 2015 39
Number of Products by Stage and Molecule Type, H2 2015 41
Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) Therapeutics - Recent Pipeline Updates, H2 2015 69
Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Dormant Projects, H2 2015 90
Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Discontinued Products, H2 2015 91

List of Figures

Number of Products under Development for Familial Hypercholesterolemia (Type II Hyperlipoproteinemia), H2 2015 10
Number of Products under Development for Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) - Comparative Analysis, H2 2015 11
Number of Products under Development by Companies, H2 2015 12
Comparative Analysis by Late Stage Development, H2 2015 15
Comparative Analysis by Early Stage Products, H2 2015 17
Assessment by Monotherapy Products, H2 2015 32
Number of Products by Top 10 Targets, H2 2015 34
Number of Products by Stage and Top 10 Targets, H2 2015 34
Number of Products by Top 10 Mechanism of Actions, H2 2015 36
Number of Products by Stage and Top 10 Mechanism of Actions, H2 2015 36
Number of Products by Routes of Administration, H2 2015 38
Number of Products by Stage and Routes of Administration, H2 2015 38
Number of Products by Molecule Types, H2 2015 40
Number of Products by Stage and Molecule Types, H2 2015 40