Rett Syndrome-Pipeline Review, H1 2017

Rett Syndrome-Pipeline Review, H1 2017


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  • Pages: 119
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Executive Summary

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 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.



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Ankur Gupta, Head Marketing & Communications

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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 Neuren's 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 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

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

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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

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