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Homozygous Familial Hypercholesterolemia (HoFH) - Pipeline Review, H2 2016

Published By :

Global Markets Direct

Published Date : Nov 2016

Category :

Pharmaceutical

No. of Pages : 53 Pages

Homozygous Familial Hypercholesterolemia (HoFH) - Pipeline Review, H2 2016

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Homozygous Familial Hypercholesterolemia (HoFH) Pipeline Review, H2 2016, provides an overview of the Homozygous Familial Hypercholesterolemia (HoFH) (Metabolic Disorders) pipeline landscape.

Homozygous familial hypercholesterolemia is a genetic disorder which is caused due to mutation from both parents. HoFH causes LDL cholesterol level to be very high. Symptoms include xanthelasmas, chest pain and sores on the toes that do not heal. Treatment includes diet changes and statin drugs.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Homozygous Familial Hypercholesterolemia (HoFH) Pipeline Review, H2 2016, provides comprehensive information on the therapeutics under development for Homozygous Familial Hypercholesterolemia (HoFH) (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 Homozygous Familial Hypercholesterolemia (HoFH) (Metabolic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Homozygous Familial Hypercholesterolemia (HoFH) and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase II, Phase I, Preclinical and Discovery stages are 5, 1, 3 and 1 respectively.Homozygous Familial Hypercholesterolemia (HoFH).

Homozygous Familial Hypercholesterolemia (HoFH) (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 Directs 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 Homozygous Familial Hypercholesterolemia (HoFH) (Metabolic Disorders).
- The pipeline guide reviews pipeline therapeutics for Homozygous Familial Hypercholesterolemia (HoFH) (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 Homozygous Familial Hypercholesterolemia (HoFH) (Metabolic Disorders) therapeutics and enlists all their major and minor projects.
- The pipeline guide evaluates Homozygous Familial Hypercholesterolemia (HoFH) (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 Homozygous Familial Hypercholesterolemia (HoFH) (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 Homozygous Familial Hypercholesterolemia (HoFH) (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 its most promising pipeline therapeutics.
- Formulate corrective measures for pipeline projects by understanding Homozygous Familial Hypercholesterolemia (HoFH) (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
Table of Contents 2
List of Tables 4
List of Figures 4
Introduction 5
Global Markets Direct Report Coverage 5
Homozygous Familial Hypercholesterolemia (HoFH) Overview 6
Therapeutics Development 7
Pipeline Products for Homozygous Familial Hypercholesterolemia (HoFH) - Overview 7
Homozygous Familial Hypercholesterolemia (HoFH) - Therapeutics under Development by Companies 8
Homozygous Familial Hypercholesterolemia (HoFH) - Pipeline Products Glance 10
Clinical Stage Products 10
Early Stage Products 11
Homozygous Familial Hypercholesterolemia (HoFH) - Products under Development by Companies 12
Homozygous Familial Hypercholesterolemia (HoFH) - Companies Involved in Therapeutics Development 13
ARMO Biosciences Inc 13
CymaBay Therapeutics Inc 14
Gemphire Therapeutics Inc 15
LipimetiX Development Inc 16
Regeneron Pharmaceuticals Inc 17
RegenxBio Inc 18
Homozygous Familial Hypercholesterolemia (HoFH) - Therapeutics Assessment 19
Assessment by Monotherapy Products 19
Assessment by Target 20
Assessment by Mechanism of Action 22
Assessment by Route of Administration 24
Assessment by Molecule Type 26
Drug Profiles 28
AEM-28 - Drug Profile 28
Product Description 28
Mechanism Of Action 28
R&D Progress 28
AEM-2802 - Drug Profile 30
Product Description 30
Mechanism Of Action 30
R&D Progress 30
AEM-2814 - Drug Profile 31
Product Description 31
Mechanism Of Action 31
R&D Progress 31
AM-0010 - Drug Profile 32
Product Description 32
Mechanism Of Action 32
R&D Progress 32
evinacumab - Drug Profile 35
Product Description 35
Mechanism Of Action 35
R&D Progress 35
gemcabene calcium - Drug Profile 37
Product Description 37
Mechanism Of Action 37
R&D Progress 37
MBX-8025 - Drug Profile 39
Product Description 39
Mechanism Of Action 39
R&D Progress 39
MGL-3196 - Drug Profile 42
Product Description 42
Mechanism Of Action 42
R&D Progress 42
MGL-3745 - Drug Profile 44
Product Description 44
Mechanism Of Action 44
R&D Progress 44
RGX-501 - Drug Profile 45
Product Description 45
Mechanism Of Action 45
R&D Progress 45
Homozygous Familial Hypercholesterolemia (HoFH) - Dormant Projects 47
Homozygous Familial Hypercholesterolemia (HoFH) - Product Development Milestones 48
Featured News & Press Releases 48
Sep 26, 2016: Gemphire Therapeutics Enrolls First Patient in the COBALT-1 Trial Investigating Gemcabene in Homozygous Familial Hypercholesterolemia 48
May 31, 2016: Regeneron Presents Positive Interim Data from Phase 2 Proof-of-Concept Study of Evinacumab in Patients with Homozygous Familial Hypercholesterolemia 48
Mar 17, 2016: CymaBay Therapeutics Announces Positive Results from its Pilot Phase 2 Clinical Study of MBX-8025 in Patients with Homozygous Familial Hypercholesterolemia 49
Apr 23, 2015: CymaBay Therapeutics Announces the Initiation of a Phase 2 Study of MBX-8025 in Patients With Homozygous Familial Hypercholesterolemia 50
Mar 25, 2015: CymaBay Therapeutics Announces U.S. Orphan Drug Designation for MBX-8025 in Homozygous Familial Hypercholesterolemia 51
Appendix 52
Methodology 52
Coverage 52
Secondary Research 52
Primary Research 52
Expert Panel Validation 52
Contact Us 52
Disclaimer 53

List of Tables
Number of Products under Development for Homozygous Familial Hypercholesterolemia (HoFH), H2 2016 7
Number of Products under Development by Companies, H2 2016 9
Comparative Analysis by Clinical Stage Development, H2 2016 10
Comparative Analysis by Early Stage Development, H2 2016 11
Products under Development by Companies, H2 2016 12
Homozygous Familial Hypercholesterolemia (HoFH) - Pipeline by ARMO Biosciences Inc, H2 2016 13
Homozygous Familial Hypercholesterolemia (HoFH) - Pipeline by CymaBay Therapeutics Inc, H2 2016 14
Homozygous Familial Hypercholesterolemia (HoFH) - Pipeline by Gemphire Therapeutics Inc, H2 2016 15
Homozygous Familial Hypercholesterolemia (HoFH) - Pipeline by LipimetiX Development Inc, H2 2016 16
Homozygous Familial Hypercholesterolemia (HoFH) - Pipeline by Regeneron Pharmaceuticals Inc, H2 2016 17
Homozygous Familial Hypercholesterolemia (HoFH) - Pipeline by RegenxBio Inc, H2 2016 18
Assessment by Monotherapy Products, H2 2016 19
Number of Products by Stage and Target, H2 2016 21
Number of Products by Stage and Mechanism of Action, H2 2016 23
Number of Products by Stage and Route of Administration, H2 2016 25
Number of Products by Stage and Molecule Type, H2 2016 27
Homozygous Familial Hypercholesterolemia (HoFH) - Dormant Projects, H2 2016 47

List of Figures
Number of Products under Development for Homozygous Familial Hypercholesterolemia (HoFH), H2 2016 7
Number of Products under Development by Companies, H2 2016 8
Comparative Analysis by Clinical Stage Development, H2 2016 10
Comparative Analysis by Early Stage Products, H2 2016 11
Assessment by Monotherapy Products, H2 2016 19
Number of Products by Targets, H2 2016 20
Number of Products by Stage and Targets, H2 2016 20
Number of Products by Mechanism of Actions, H2 2016 22
Number of Products by Stage and Mechanism of Actions, H2 2016 22
Number of Products by Routes of Administration, H2 2016 24
Number of Products by Stage and Routes of Administration, H2 2016 24
Number of Products by Molecule Types, H2 2016 26
Number of Products by Stage and Molecule Types, H2 2016 26

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