Homozygous Familial Hypercholesterolemia (HoFH) - Pipeline Review, H1 2016

Published: May, 2016 | Pages: 71 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

Homozygous Familial Hypercholesterolemia (HoFH) - Pipeline Review, H1 2016

Summary

Global Markets Direct's, 'Homozygous Familial Hypercholesterolemia (HoFH) - Pipeline Review, H1 2016', provides an overview of the Homozygous Familial Hypercholesterolemia (HoFH) pipeline landscape. 

The report provides comprehensive information on the therapeutics under development for Homozygous Familial Hypercholesterolemia (HoFH), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Homozygous Familial Hypercholesterolemia (HoFH) and features dormant 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, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Drug profiles featured in the report undergoes periodic review following a stringent set of processes to ensure that all the profiles are updated with the latest set of information. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

The report 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.

Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data.

Scope

- The report provides a snapshot of the global therapeutic landscape of Homozygous Familial Hypercholesterolemia (HoFH)
- The report reviews pipeline therapeutics for Homozygous Familial Hypercholesterolemia (HoFH) 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 Homozygous Familial Hypercholesterolemia (HoFH) therapeutics and enlists all their major and minor projects
- The report assesses Homozygous Familial Hypercholesterolemia (HoFH) therapeutics based on drug target, 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 related to pipeline therapeutics for Homozygous Familial Hypercholesterolemia (HoFH)


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 important and diverse types of therapeutics under development for Homozygous Familial Hypercholesterolemia (HoFH)
- 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 Homozygous Familial Hypercholesterolemia (HoFH) 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 5
Introduction 6
Global Markets Direct Report Coverage 6
Homozygous Familial Hypercholesterolemia (HoFH) Overview 7
Therapeutics Development 8
Pipeline Products for Homozygous Familial Hypercholesterolemia (HoFH) - Overview 8
Homozygous Familial Hypercholesterolemia (HoFH) - Therapeutics under Development by Companies 9
Homozygous Familial Hypercholesterolemia (HoFH) - Pipeline Products Glance 10
Late Stage Products 10
Clinical Stage Products 11
Early Stage Products 12
Homozygous Familial Hypercholesterolemia (HoFH) - Products under Development by Companies 13
Homozygous Familial Hypercholesterolemia (HoFH) - Companies Involved in Therapeutics Development 14
Aegerion Pharmaceuticals, Inc. 14
CymaBay Therapeutics, Inc. 15
Kastle Therapeutics LLC 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 29
Product Description 29
Mechanism of Action 29
R&D Progress 29
AEM-2814 - Drug Profile 30
Product Description 30
Mechanism of Action 30
R&D Progress 30
AM-0010 - Drug Profile 31
Product Description 31
Mechanism of Action 31
R&D Progress 31
evinacumab - Drug Profile 33
Product Description 33
Mechanism of Action 33
R&D Progress 33
gemcabene calcium - Drug Profile 34
Product Description 34
Mechanism of Action 34
R&D Progress 34
lomitapide mesylate - Drug Profile 35
Product Description 35
Mechanism of Action 35
R&D Progress 35
MBX-8025 - Drug Profile 38
Product Description 38
Mechanism of Action 38
R&D Progress 38
MGL-3196 - Drug Profile 40
Product Description 40
Mechanism of Action 40
R&D Progress 40
MGL-3745 - Drug Profile 42
Product Description 42
Mechanism of Action 42
R&D Progress 42
mipomersen sodium - Drug Profile 43
Product Description 43
Mechanism of Action 43
R&D Progress 43
RGX-501 - Drug Profile 45
Product Description 45
Mechanism of Action 45
R&D Progress 45
Homozygous Familial Hypercholesterolemia (HoFH) - Recent Pipeline Updates 47
Homozygous Familial Hypercholesterolemia (HoFH) - Dormant Projects 61
Homozygous Familial Hypercholesterolemia (HoFH) - Product Development Milestones 62
Featured News & Press Releases 62
Mar 17, 2016: CymaBay Therapeutics Announces Positive Results from its Pilot Phase 2 Clinical Study of MBX-8025 in Patients with Homozygous Familial Hypercholesterolemia 62
Nov 20, 2015: JUXTAPID (lomitapide) Capsules Available on the Regie de l'Assurance Maladie du Quebec (RAMQ) for the Treatment of Homozygous Familial Hypercholesterolemia (HoFH) 63
Nov 10, 2015: Aegerion Pharmaceuticals Announces Results From First Year of LOWER Study at AHA Scientific Sessions 2015 64
May 26, 2015: Aegerion Pharmaceuticals Achieves Primary Endpoint in Phase III Study of Lomitapide in Japanese Patients With Homozygous Familial Hypercholesterolemia (HoFH) 64
Apr 30, 2015: AOP Orphan Launches Lojuxta (lomitapide) for Treatment of Homozygous Familial Hypercholesterolaemia to the Austrian Market 65
Apr 23, 2015: CymaBay Therapeutics Announces the Initiation of a Phase 2 Study of MBX-8025 in Patients With Homozygous Familial Hypercholesterolemia 66
Mar 25, 2015: CymaBay Therapeutics Announces U.S. Orphan Drug Designation for MBX-8025 in Homozygous Familial Hypercholesterolemia 67
Jan 28, 2015: Aegerion Investigated by Former Louisiana Attorney General and Special Prosecutor Richard P. Ieyoub and the Firm of Ieyoub O'Bell & Myers 68
Aug 27, 2014: Aegerion Pharmaceuticals Announces Resolution of Warning Letter for JUXTAPID Capsules 68
Apr 09, 2014: Aegerion Pharmaceuticals Initiates Phase 3 Clinical Trial in Japan to Evaluate Lomitapide for Treatment of Homozygous Familial Hypercholesterolemia 68
Appendix 70
Methodology 70
Coverage 70
Secondary Research 70
Primary Research 70
Expert Panel Validation 70
Contact Us 70
Disclaimer 71

List of Tables

Number of Products under Development for Homozygous Familial Hypercholesterolemia (HoFH), H1 2016 8
Number of Products under Development by Companies, H1 2016 9
Comparative Analysis by Late Stage Development, H1 2016 10
Comparative Analysis by Clinical Stage Development, H1 2016 11
Comparative Analysis by Early Stage Development, H1 2016 12
Products under Development by Companies, H1 2016 13
Homozygous Familial Hypercholesterolemia (HoFH) - Pipeline by Aegerion Pharmaceuticals, Inc., H1 2016 14
Homozygous Familial Hypercholesterolemia (HoFH) - Pipeline by CymaBay Therapeutics, Inc., H1 2016 15
Homozygous Familial Hypercholesterolemia (HoFH) - Pipeline by Kastle Therapeutics LLC, H1 2016 16
Homozygous Familial Hypercholesterolemia (HoFH) - Pipeline by Regeneron Pharmaceuticals, Inc., H1 2016 17
Homozygous Familial Hypercholesterolemia (HoFH) - Pipeline by RegenxBio Inc., H1 2016 18
Assessment by Monotherapy Products, H1 2016 19
Number of Products by Stage and Target, H1 2016 21
Number of Products by Stage and Mechanism of Action, H1 2016 23
Number of Products by Stage and Route of Administration, H1 2016 25
Number of Products by Stage and Molecule Type, H1 2016 27
Homozygous Familial Hypercholesterolemia (HoFH) Therapeutics - Recent Pipeline Updates, H1 2016 47
Homozygous Familial Hypercholesterolemia (HoFH) - Dormant Projects, H1 2016 61