Rett Syndrome – Pipeline Review, H1 2018

Global Markets Direct’s, ‘Rett Syndrome – Pipeline Review, H1 2018’, provides an overview of the Rett Syndrome pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Rett Syndrome, 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 Rett Syndrome 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 Rett Syndrome

– The report reviews pipeline therapeutics for Rett Syndrome 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 Rett Syndrome therapeutics and enlists all their major and minor projects

– The report assesses Rett Syndrome 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 Rett Syndrome

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 Rett Syndrome

- 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 Rett Syndrome 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

Companies mentioned

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

Table of Contents

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

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

Disclaimer

List of Tables

List 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 2018

List of Figures

List 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

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