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Rett Syndrome - Pipeline Review, H1 2017

Published By :

Global Markets Direct

Published Date : May 2017

Category :

Diseases & Conditions

No. of Pages : 119 Pages

Rett Syndrome - Pipeline Review, H1 2017

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Rett Syndrome - Pipeline Review, H1 2017, 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 2017, 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, 4, 7, 6 and 3 respectively. Similarly, the Universities portfolio in Preclinical stages comprises 3 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 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 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 its 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.
Table of Contents
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
Amorsa Therapeutics Inc
Anavex Life Sciences Corp
Apteeus
ArmaGen Inc
Edison Pharmaceuticals Inc
Eloxx Pharmaceuticals Ltd
GW Pharmaceuticals Plc
Horizon Pharma Plc
Mitochon Pharmaceuticals Inc
Mitsubishi Tanabe Pharma Corp
Neuren Pharmaceuticals Ltd
Neurolixis Inc
Newron Pharmaceuticals SpA
OPKO Health Inc
Sage Therapeutics Inc
Ultragenyx Pharmaceutical Inc
Rett Syndrome - Drug Profiles
amlexanox - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
ANAVEX-273 - 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
cysteamine DR - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
desipramine hydrochloride - 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
Gene therapy to Activate MECP2 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
Oligonucleotide for Rett Syndrome - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Recombinant Protein for Rett Syndrome - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SAGE-217 - 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 TrkB Receptor for Central Nervous System, Metabolic Disorders and ENT Disorders - 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 Antagonise NMDA Receptor for Rett Syndrome - 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
vatiquinone - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Rett Syndrome - Dormant Projects
Rett Syndrome - Product Development Milestones
Featured News & Press Releases
Mar 22, 2017: Neuren's Phase 2 trial of trofinetide demonstrates significant clinical benefit in pediatric Rett syndrome
Jan 30, 2017: Neuren completes Phase 2 trial in pediatric Rett syndrome
Jan 19, 2017: Anavex Life Sciences Receives Grant From Rettsyndrome.org to Commence U.S. Phase 2 Trial in 2017
Nov 14, 2016: Neuren Completes Enrolment into Phase 2 Trial in Pediatric Rett Syndrome
Aug 25, 2016: Neuren expands Phase 2 Rett syndrome pediatric clinical trial
Jul 21, 2016: Newron Pharmaceuticals Announces Initiation of the STARS Study in the United States
Jun 24, 2016: Neuren presents at the Rettsyndrome.org 2016 Research Symposium
Jun 22, 2016: Neuren receives grant of new Australian patent for trofinetide to treat Autism Spectrum Disorders
Jun 16, 2016: Newron Pharmaceuticals to Present STARS Trial Design at US Rett Syndrome Symposium
Jun 06, 2016: Anavex Announces Positive Preclinical Results with ANAVEX 2-73 in Fragile X - Autism-Related Disorders
May 20, 2016: Anavex Announces U.S. FDA Orphan Drug Designation to ANAVEX 2-73 for the Treatment of Rett Syndrome
May 17, 2016: Mitochon Pharmaceuticals Raises $1.6M for Clinical Stage Development
May 17, 2016: Newron Announces IND Approval for Sarizotan for Treatment of Rett Syndrome
Apr 07, 2016: First subject commences Neurens pediatric Rett syndrome Phase 2 trial
Feb 25, 2016: Anavex Presents Preclinical Results of ANAVEX 2-73 in Rett Syndrome
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer

List of Tables
Number of Products under Development for Rett Syndrome, H1 2017
Number of Products under Development by Companies, H1 2017
Number of Products under Development by Companies, H1 2017 (Contd..1), H1 2017
Number of Products under Development by Universities/Institutes, H1 2017
Products under Development by Companies, H1 2017
Products under Development by Companies, H1 2017 (Contd..1), H1 2017
Products under Development by Universities/Institutes, H1 2017
Number of Products by Stage and Target, H1 2017
Number of Products by Stage and Mechanism of Action, H1 2017
Number of Products by Stage and Route of Administration, H1 2017
Number of Products by Stage and Molecule Type, H1 2017
Rett Syndrome - Pipeline by Amorsa Therapeutics Inc, H1 2017
Rett Syndrome - Pipeline by Anavex Life Sciences Corp, H1 2017
Rett Syndrome - Pipeline by Apteeus, H1 2017
Rett Syndrome - Pipeline by ArmaGen Inc, H1 2017
Rett Syndrome - Pipeline by Edison Pharmaceuticals Inc, H1 2017
Rett Syndrome - Pipeline by Eloxx Pharmaceuticals Ltd, H1 2017
Rett Syndrome - Pipeline by GW Pharmaceuticals Plc, H1 2017
Rett Syndrome - Pipeline by Horizon Pharma Plc, H1 2017
Rett Syndrome - Pipeline by Mitochon Pharmaceuticals Inc, H1 2017
Rett Syndrome - Pipeline by Mitsubishi Tanabe Pharma Corp, H1 2017
Rett Syndrome - Pipeline by Neuren Pharmaceuticals Ltd, H1 2017
Rett Syndrome - Pipeline by Neurolixis Inc, H1 2017
Rett Syndrome - Pipeline by Newron Pharmaceuticals SpA, H1 2017
Rett Syndrome - Pipeline by OPKO Health Inc, H1 2017
Rett Syndrome - Pipeline by Sage Therapeutics Inc, H1 2017
Rett Syndrome - Pipeline by Ultragenyx Pharmaceutical Inc, H1 2017
Rett Syndrome - Dormant Projects, H1 2017

List of Figures
Number of Products under Development for Rett Syndrome, H1 2017
Number of Products under Development by Companies, H1 2017
Number of Products by Top 10 Targets, H1 2017
Number of Products by Stage and Top 10 Targets, H1 2017
Number of Products by Top 10 Mechanism of Actions, H1 2017
Number of Products by Stage and Top 10 Mechanism of Actions, H1 2017
Number of Products by Routes of Administration, H1 2017
Number of Products by Stage and Routes of Administration, H1 2017
Number of Products by Molecule Types, H1 2017
Number of Products by Stage and Molecule Types, H1 2017

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