Rett Syndrome - Pipeline Review, H1 2018
Rett Syndrome - Pipeline Review, H1 2018
Summary
Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Rett Syndrome - Pipeline Review, H1 2018, provides an overview of the Rett Syndrome (Central Nervous System) pipeline landscape.
Rett syndrome is a neurodevelopmental disorder. This is caused by genetic mutation on MECP2 gene. Rett syndrome is described in four stages: Stage I or early onset, Stage II or rapid destruction, Stage III, or the plateau and Stage IV, or the late motor deterioration stage. Symptoms include slowed growth, loss of normal movement and coordination, loss of communication abilities, breathing problems, pain, irregular heartbeat and seizures. Risk factors include family history and gender. Treatment includes disease modifying drugs.
Report Highlights
Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Rett Syndrome - Pipeline Review, H1 2018, provides comprehensive information on the therapeutics under development for Rett Syndrome (Central Nervous System), 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 Rett Syndrome (Central Nervous System) pipeline guide also reviews of key players involved in therapeutic development for Rett Syndrome 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 Discovery stages are 1, 3, 4, 8 and 2 respectively. Similarly, the Universities portfolio in Preclinical stages comprises 5 molecules, respectively.
Rett Syndrome (Central Nervous System) 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 Rett Syndrome (Central Nervous System).
- The pipeline guide reviews pipeline therapeutics for Rett Syndrome (Central Nervous System) 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 Rett Syndrome (Central Nervous System) therapeutics and enlists all their major and minor projects.
- The pipeline guide evaluates Rett Syndrome (Central Nervous System) 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 Rett Syndrome (Central Nervous System)
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 Rett Syndrome (Central Nervous System).
- 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 Rett Syndrome (Central Nervous System) 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
AMO Pharma Ltd
Amorsa Therapeutics Inc
Anavex Life Sciences Corp
ArmaGen Inc
AveXis Inc
Biohaven Pharmaceutical Holding Company Ltd
Eloxx Pharmaceuticals Inc
GW Pharmaceuticals Plc
Mitochon Pharmaceuticals Inc
Mitsubishi Tanabe Pharma Corp
Neuren Pharmaceuticals Ltd
Neurolixis Inc
Newron Pharmaceuticals SpA
OPKO Health Inc
Ultragenyx Pharmaceutical Inc
List of Tables
List of Figures
Introduction
Global Markets Direct Report Coverage
Rett Syndrome - Overview
Rett Syndrome - Therapeutics Development
Pipeline Overview
Pipeline by Companies
Pipeline by Universities/Institutes
Products under Development by Companies
Products under Development by Universities/Institutes
Rett Syndrome - Therapeutics Assessment
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Rett Syndrome - Companies Involved in Therapeutics Development
AMO Pharma Ltd
Amorsa Therapeutics Inc
Anavex Life Sciences Corp
ArmaGen Inc
AveXis Inc
Biohaven Pharmaceutical Holding Company Ltd
Eloxx Pharmaceuticals Inc
GW Pharmaceuticals Plc
Mitochon Pharmaceuticals Inc
Mitsubishi Tanabe Pharma Corp
Neuren Pharmaceuticals Ltd
Neurolixis Inc
Newron Pharmaceuticals SpA
OPKO Health Inc
Ultragenyx Pharmaceutical Inc
Rett Syndrome - Drug Profiles
ANAVEX-273 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Antisense Oligonucleotide for Rett Syndrome - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
AVXS-201 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
BHV-5000 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Bryostatin-1 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
CPT-157633 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
ELX-02 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
fingolimod hydrochloride - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Fusion Protein for Rett Syndrome - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene therapy to Activate MECP2 for Rett Syndrome - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate MECP2 Protein for Rett Syndrome - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
GWP-42006 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
LM-22A4 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
MP-101 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
NLX-101 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
sarizotan hydrochloride - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecule to Agonize mGlu5 for Rett Syndrome - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecule to Agonize mGlu7 for Rett Syndrome - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecules for Pervasive Developmental Disorder and Rett Syndrome - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecules to Antagonize NMDA Receptor for Rett Syndrome - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
tianeptine - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
trofinetide - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
UX-007 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Rett Syndrome - Dormant Projects
Rett Syndrome - Product Development Milestones
Featured News & Press Releases
Feb 24, 2018: Patent published on novel serotonin 5-HT1A receptor agonists NLX-101
Jan 18, 2018: Biohaven Announces Initiation Of Clinical Development For BHV-5000, A Novel Low-Trapping NMDA Antagonist
Nov 04, 2017: Anavex Life Sciences Presents New Clinical Data of Investigational Alzheimer’s Disease Treatment ANAVEX2-73 at 2017 Clinical Trials on Alzheimer’s Disease Meeting
Oct 25, 2017: Newron Pharmaceuticals to Present at European Rett Syndrome Congress and Host Advisory Board Meeting
Oct 13, 2017: Neuren confirms Phase 3 plan for Rett syndrome at FDA Meeting
Oct 05, 2017: Presentations and Publication for Neuren’s Rett Syndrome Clinical Trials
Jul 27, 2017: Drug Improves Brain Performance in Rett Syndrome Mice
Jul 20, 2017: Anavex Life Sciences Appoints Distinguished Researcher in Neurology and Epilepsy to Scientific Advisory Board
Jul 20, 2017: Neuren granted Type B Meeting with FDA for Rett syndrome
Jul 06, 2017: Biohavens BHV-5000 Receives Orphan Drug Designation From The FDA In Rett Syndrome
Jun 29, 2017: Neuren secures funding for key preparatory activities for Rett syndrome Phase 3 trial
May 31, 2017: Neuren announces grant of two new patents for trofinetide to 2032
May 22, 2017: Anavex Life Sciences Reports New Data for ANAVEX 2-73 in Neurodevelopmental Disorders Including Angelman Syndrome-Data Presented at Antiepileptic Drug Trials XIV 2017 Conference
May 17, 2017: Newron Pharmaceuticals Announces Expansion of STARS Study to Include Patients Under 13 Years of Age
Mar 22, 2017: Neuren's Phase 2 trial of trofinetide demonstrates significant clinical benefit in pediatric Rett syndrome
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
DisclaimerList of Tables
Number of Products under Development for Rett Syndrome, H1 2018
Number of Products under Development by Companies, H1 2018
Number of Products under Development by Universities/Institutes, H1 2018
Products under Development by Companies, H1 2018
Products under Development by Companies, H1 2018 (Contd..1), H1 2018
Products under Development by Universities/Institutes, 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
Rett Syndrome - Pipeline by AMO Pharma Ltd, H1 2018
Rett Syndrome - Pipeline by Amorsa Therapeutics Inc, H1 2018
Rett Syndrome - Pipeline by Anavex Life Sciences Corp, H1 2018
Rett Syndrome - Pipeline by ArmaGen Inc, H1 2018
Rett Syndrome - Pipeline by AveXis Inc, H1 2018
Rett Syndrome - Pipeline by Biohaven Pharmaceutical Holding Company Ltd, H1 2018
Rett Syndrome - Pipeline by Eloxx Pharmaceuticals Inc, H1 2018
Rett Syndrome - Pipeline by GW Pharmaceuticals Plc, H1 2018
Rett Syndrome - Pipeline by Mitochon Pharmaceuticals Inc, H1 2018
Rett Syndrome - Pipeline by Mitsubishi Tanabe Pharma Corp, H1 2018
Rett Syndrome - Pipeline by Neuren Pharmaceuticals Ltd, H1 2018
Rett Syndrome - Pipeline by Neurolixis Inc, H1 2018
Rett Syndrome - Pipeline by Newron Pharmaceuticals SpA, H1 2018
Rett Syndrome - Pipeline by OPKO Health Inc, H1 2018
Rett Syndrome - Pipeline by Ultragenyx Pharmaceutical Inc, H1 2018
Rett Syndrome - Dormant Projects, H1 2018List of Figures
Number of Products under Development for Rett Syndrome, H1 2018
Number of Products under Development by Companies, H1 2018
Number of Products by Top 10 Targets, H1 2018
Number of Products by Stage and Top 10 Targets, H1 2018
Number of Products by Top 10 Mechanism of Actions, H1 2018
Number of Products by Stage and Top 10 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 "Rett Syndrome - 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
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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 Rett Syndrome Pipeline Review outlook. 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 Rett Syndrome Pipeline Review outlook 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 Rett Syndrome Pipeline Review outlook 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 Rett Syndrome Pipeline Review outlook 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 "Rett Syndrome - 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. |
