Homozygous Familial Hypercholesterolemia (HoFH) - Pipeline Review, H1 2018
Homozygous Familial Hypercholesterolemia (HoFH) - Pipeline Review, H1 2018
Summary
Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Homozygous Familial Hypercholesterolemia (HoFH) - Pipeline Review, H1 2018, 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, H1 2018, 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 III, Phase II, Phase I, Preclinical and Unknown stages are 3, 5, 1, 2 and 1 respectively.
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 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 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 it’s 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.
Companies Mentioned in the Report
Akcea Therapeutics Inc
CymaBay Therapeutics Inc
Daewoong Co Ltd
Gemphire Therapeutics Inc
LipimetiX Development Inc
Madrigal Pharmaceuticals Inc.
Regeneron Pharmaceuticals Inc
RegenxBio Inc
The Medicines Company
List of Tables
List of Figures
Introduction
Global Markets Direct Report Coverage
Homozygous Familial Hypercholesterolemia (HoFH) - Overview
Homozygous Familial Hypercholesterolemia (HoFH) - Therapeutics Development
Pipeline Overview
Pipeline by Companies
Products under Development by Companies
Homozygous Familial Hypercholesterolemia (HoFH) - Therapeutics Assessment
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Homozygous Familial Hypercholesterolemia (HoFH) - Companies Involved in Therapeutics Development
Akcea Therapeutics Inc
CymaBay Therapeutics Inc
Daewoong Co Ltd
Gemphire Therapeutics Inc
LipimetiX Development Inc
Madrigal Pharmaceuticals Inc.
Regeneron Pharmaceuticals Inc
RegenxBio Inc
The Medicines Company
Homozygous Familial Hypercholesterolemia (HoFH) - Drug Profiles
AEM-28 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
AEM-2814 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
AKCEA-ANGPTL3LRx - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
alirocumab - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
evinacumab - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
gemcabene calcium - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
inclisiran - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
MGL-3196 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
MGL-3745 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
RGX-501 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
seladelpar lysine - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
ST-103 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Homozygous Familial Hypercholesterolemia (HoFH) - Dormant Projects
Homozygous Familial Hypercholesterolemia (HoFH) - Discontinued Products
Homozygous Familial Hypercholesterolemia (HoFH) - Product Development Milestones
Featured News & Press Releases
Apr 30, 2018: The Medicines Company Presents New Data at the National Lipid Association Scientific Sessions
Mar 08, 2018: The Medicines Company Reaches Enrollment Target for ORION-10 Ahead of Schedule
Jan 25, 2018: Inclisiran’s First Pivotal Trial Achieves Target Enrollment Ahead of Schedule
Nov 13, 2017: Akcea Initiates AKCEA-ANGPTL3-LRx Phase 2 Program in Patients with Rare Hyperlipidemias
Sep 25, 2017: Gemphire to Present New COBALT-1 Clinical Data at the 2017 FH Global Summit
Jun 28, 2017: Gemphire Announces Top-Line Data from COBALT-1 Phase 2b Clinical Trial in HoFH Patients
May 24, 2017: Regeneron Announces ANGPTL3/Evinacumab Publication in New England Journal of Medicine and Positive Phase 2 Data in People with HoFH
Apr 06, 2017: Regeneron Announces Evinacumab has Received FDA Breakthrough Therapy Designation for Homozygous Familial Hypercholesterolemia
Mar 14, 2017: The Medicines Company to Webcast Presentation of ORION-1 Phase II Study of Inclisiran at ACC.17
Jan 30, 2017: Gemphire Announces Interim LDL-C Lowering Data from COBALT-1 Phase 2b Clinical Trial
Jan 05, 2017: Gemphire Therapeutics Provides Clinical Update
Dec 19, 2016: Capstone Therapeutics Announces Profound, Rapid LDL Cholesterol Reduction in AEM-28-14 Primate Study
Sep 26, 2016: Gemphire Therapeutics Enrolls First Patient in the COBALT-1 Trial Investigating Gemcabene in Homozygous Familial Hypercholesterolemia
May 31, 2016: Regeneron Presents Positive Interim Data from Phase 2 Proof-of-Concept Study of Evinacumab in Patients with Homozygous Familial Hypercholesterolemia
Mar 17, 2016: CymaBay Therapeutics Announces Positive Results from its Pilot Phase 2 Clinical Study of MBX-8025 in Patients with Homozygous Familial Hypercholesterolemia
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
DisclaimerList of Tables
Number of Products under Development for Homozygous Familial Hypercholesterolemia (HoFH), H1 2018
Number of Products under Development by Companies, H1 2018
Products under Development by Companies, H1 2018
Number of Products by Stage and Target, H1 2018
Number of Products by Stage and Mechanism of Action, H1 2018
Number of Products by Stage and Route of Administration, H1 2018
Number of Products by Stage and Molecule Type, H1 2018
Homozygous Familial Hypercholesterolemia (HoFH) - Pipeline by Akcea Therapeutics Inc, H1 2018
Homozygous Familial Hypercholesterolemia (HoFH) - Pipeline by CymaBay Therapeutics Inc, H1 2018
Homozygous Familial Hypercholesterolemia (HoFH) - Pipeline by Daewoong Co Ltd, H1 2018
Homozygous Familial Hypercholesterolemia (HoFH) - Pipeline by Gemphire Therapeutics Inc, H1 2018
Homozygous Familial Hypercholesterolemia (HoFH) - Pipeline by LipimetiX Development Inc, H1 2018
Homozygous Familial Hypercholesterolemia (HoFH) - Pipeline by Madrigal Pharmaceuticals Inc., H1 2018
Homozygous Familial Hypercholesterolemia (HoFH) - Pipeline by Regeneron Pharmaceuticals Inc, H1 2018
Homozygous Familial Hypercholesterolemia (HoFH) - Pipeline by RegenxBio Inc, H1 2018
Homozygous Familial Hypercholesterolemia (HoFH) - Pipeline by The Medicines Company, H1 2018
Homozygous Familial Hypercholesterolemia (HoFH) - Dormant Projects, H1 2018
Homozygous Familial Hypercholesterolemia (HoFH) - Discontinued Products, H1 2018List of Figures
Number of Products under Development for Homozygous Familial Hypercholesterolemia (HoFH), H1 2018
Number of Products under Development by Companies, H1 2018
Number of Products by Targets, H1 2018
Number of Products by Stage and Targets, H1 2018
Number of Products by Mechanism of Actions, H1 2018
Number of Products by Stage and Mechanism of Actions, H1 2018
Number of Products by Routes of Administration, H1 2018
Number of Products by Stage and Routes of Administration, H1 2018
Number of Products by Molecule Types, H1 2018
Number of Products by Stage and Molecule Types, H1 2018
Research Methodology
The research study released with title "Homozygous Familial Hypercholesterolemia (HoFH) - Pipeline Review, H1 2018" involved the extensive usage of both primary and secondary data sources. The research process involved the study of various factors affecting the industry, including the government policy, market environment, competitive landscape, historical data, present trends in the market, technological innovation, upcoming technologies and the technical progress in related industry, and market risks, opportunities, market barriers and challenges. The following illustrative figure shows the market research methodology applied in this report.Key Executives Interviewed
Research Programs/Design
| Historical Data (2017-2022E) |
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| Market Factor Analysis |
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Market Size Estimation
Top-down and bottom-up approaches are used to validate and estimate the global/regional market size by manufacturers, regional splits, product segments and application.
To establish Research coverage of the study with most relevant & functional players HTF MI follows industry standards such as NAICS/SIC/ICB/TRCB, furthermore the sorted list of companies is validated using Product Mapping and business activities and shortlisted by industry perspective.
HTF MI select experienced analyst and associate that are specialized in the domain for conducting research. They prepare a list of companies by region that helps to understand players penetration rate in the market. This companies are further analyses in what segment or application they serve in our market. The list of product and applications are evaluated for each companies dealing in the Homozygous Familial Hypercholesterolemia trend. The market study is conducted on basis of more than 200 companies dealing in the market regional as well as global areas with purpose to understand company’s positioning regarding market value, volume and their market share for regional as well as global. Further to bring relevance specific to any niche market we set and apply number of criteria like Geographic Footprints, Regional Segments of Revenue, Operational Centres, etc. The next step is to finalize a team (In-House + Data Online) who then starts collecting C & D level executives and profiles, Industry experts, Opinion leaders etc from Homozygous Familial Hypercholesterolemia trend who then work towards appointment generation.
Primary Data Collection
The primary research is performed by taking the interviews of executives of various companies dealing in the Homozygous Familial Hypercholesterolemia trend as well as using the survey reports, research institute, and latest research reports. Meanwhile, analyst team keeps preparing set of questionnaires and relevant list of appointee or target primary respondents; the target audience are then tapped and segregated with various mediums and channels that are feasible for making connection that includes email communication, telephonic, skype, LinkedIn Group & In Mail, Community Forums, Open Survey, SurveyMonkey etc.
Secondary Sources
The secondary resources considered in the Homozygous Familial Hypercholesterolemia trend study includes company annual reports, press releases, published articles of organizations and associations, and the survey taken through different mediums such as online website and forums, survey monkey, LinkedIn, etc. In addition, the valuable strategic information obtained from industry association, paid database, and investor presentations are also good tool for information to make appropriate iterative validation are used to modify market models and cross-verify primary research and insights.
Market Breakdown and Data Triangulation
To make market engineering data complete; extensive primary research is conducted to gather information and validate the critical numbers derived to be used for calculations of market statistics; market size estimations; forecasting; breakdown; and data triangulation. Both top-down and bottom-up approaches were applied, along with several data triangulation methods, to perform market estimation and market forecasting for the overall market segments and sub-segments listed in this report. Furthermore qualitative and further quantitative analysis is also done for all the numbers arrived at in the market estimation process to list relevant information in the final deliverable report "Homozygous Familial Hypercholesterolemia (HoFH) - Pipeline Review, H1 2018" .
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Key Information from Primary Sources
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| Format | Properties | |
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| 1-User Access | The report is sent in PDF format. The report will be emailed to you. | This is a single user access license, allowing one specific user to access the report. |
| Site User | The study would be provided in PDF format. The report will be emailed to you. | This is a site license, allowing 1-10 employees within your organisation to access the report. |
| Enterprise User | The format of deliverable would be PDF and Excel. | This is an enterprise or corporate license, allowing all employees within your organisation irrespective of geographic location to access the report. |
