Rett Syndrome – Pipeline Review, H2 2017

Global Markets Direct’s, ‘Rett Syndrome – Pipeline Review, H2 2017’, 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

Apteeus

ArmaGen Inc

AveXis Inc

BioElectron Technology Corp

Biohaven Pharmaceutical Holding Company Ltd

Eloxx Pharmaceuticals Ltd

GW Pharmaceuticals 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

Table of Contents

Table of Contents

Table of Contents 2

List of Tables 4

List of Figures 4

Introduction 5

Global Markets Direct Report Coverage 5

Rett Syndrome - Overview 6

Rett Syndrome - Therapeutics Development 7

Pipeline Overview 7

Pipeline by Companies 8

Pipeline by Universities/Institutes 11

Products under Development by Companies 12

Products under Development by Universities/Institutes 14

Rett Syndrome - Therapeutics Assessment 15

Assessment by Target 15

Assessment by Mechanism of Action 17

Assessment by Route of Administration 19

Assessment by Molecule Type 21

Rett Syndrome - Companies Involved in Therapeutics Development 23

AMO Pharma Ltd 23

Amorsa Therapeutics Inc 23

Anavex Life Sciences Corp 23

Apteeus 24

ArmaGen Inc 24

AveXis Inc 24

BioElectron Technology Corp 25

Biohaven Pharmaceutical Holding Company Ltd 25

Eloxx Pharmaceuticals Ltd 25

GW Pharmaceuticals Plc 26

Mitochon Pharmaceuticals Inc 26

Mitsubishi Tanabe Pharma Corp 27

Neuren Pharmaceuticals Ltd 27

Neurolixis Inc 28

Newron Pharmaceuticals SpA 28

OPKO Health Inc 28

Sage Therapeutics Inc 29

Ultragenyx Pharmaceutical Inc 29

Rett Syndrome - Drug Profiles 31

amlexanox - Drug Profile 31

AMO-04 - Drug Profile 32

ANAVEX-273 - Drug Profile 33

Antisense Oligonucleotide for Rett Syndrome - Drug Profile 43

BHV-5000 - Drug Profile 44

Bryostatin-1 - Drug Profile 45

CPT-157633 - Drug Profile 51

ELX-02 - Drug Profile 52

fingolimod hydrochloride - Drug Profile 54

Fusion Protein for Rett Syndrome - Drug Profile 61

Gene therapy to Activate MECP2 for Rett Syndrome - Drug Profile 62

Gene Therapy to Activate MECP2 for Rett Syndrome - Drug Profile 63

GWP-42006 - Drug Profile 64

LM-22A4 - Drug Profile 66

MP-101 - Drug Profile 68

NLX-101 - Drug Profile 69

SAGE-217 - Drug Profile 71

sarizotan hydrochloride - Drug Profile 75

Small Molecule to Agonize mGlu5 for Rett Syndrome - Drug Profile 78

Small Molecule to Agonize mGlu7 for Rett Syndrome - Drug Profile 79

Small Molecule to Agonize TrkB Receptor for Central Nervous System, Metabolic Disorders and ENT Disorders - Drug Profile 80

Small Molecules to Antagonize NMDA Receptor for Rett Syndrome - Drug Profile 81

trofinetide - Drug Profile 82

UX-007 - Drug Profile 90

vatiquinone - Drug Profile 97

Rett Syndrome - Dormant Projects 102

Rett Syndrome - Product Development Milestones 103

Featured News & Press Releases 103

Appendix 117

Methodology 117

Coverage 117

Secondary Research 117

Primary Research 117

Expert Panel Validation 117

Contact Us 117

Disclaimer 118

List of Tables

List of Tables

Number of Products under Development for Rett Syndrome, H2 2017

Number of Products under Development by Companies, H2 2017

Number of Products under Development by Companies, H2 2017 (Contd..1), H2 2017

Number of Products under Development by Universities/Institutes, H2 2017

Products under Development by Companies, H2 2017

Products under Development by Companies, H2 2017 (Contd..1), H2 2017

Products under Development by Universities/Institutes, H2 2017

Number of Products by Stage and Target, H2 2017

Number of Products by Stage and Mechanism of Action, H2 2017

Number of Products by Stage and Route of Administration, H2 2017

Number of Products by Stage and Molecule Type, H2 2017

Rett Syndrome – Pipeline by AMO Pharma Ltd, H2 2017

Rett Syndrome – Pipeline by Amorsa Therapeutics Inc, H2 2017

Rett Syndrome – Pipeline by Anavex Life Sciences Corp, H2 2017

Rett Syndrome – Pipeline by Apteeus, H2 2017

Rett Syndrome – Pipeline by ArmaGen Inc, H2 2017

Rett Syndrome – Pipeline by AveXis Inc, H2 2017

Rett Syndrome – Pipeline by BioElectron Technology Corp, H2 2017

Rett Syndrome – Pipeline by Biohaven Pharmaceutical Holding Company Ltd, H2 2017

Rett Syndrome – Pipeline by Eloxx Pharmaceuticals Ltd, H2 2017

Rett Syndrome – Pipeline by GW Pharmaceuticals Plc, H2 2017

Rett Syndrome – Pipeline by Mitochon Pharmaceuticals Inc, H2 2017

Rett Syndrome – Pipeline by Mitsubishi Tanabe Pharma Corp, H2 2017

Rett Syndrome – Pipeline by Neuren Pharmaceuticals Ltd, H2 2017

Rett Syndrome – Pipeline by Neurolixis Inc, H2 2017

Rett Syndrome – Pipeline by Newron Pharmaceuticals SpA, H2 2017

Rett Syndrome – Pipeline by OPKO Health Inc, H2 2017

Rett Syndrome – Pipeline by Sage Therapeutics Inc, H2 2017

Rett Syndrome – Pipeline by Ultragenyx Pharmaceutical Inc, H2 2017

Rett Syndrome – Dormant Projects, H2 2017

List of Figures

List of Figures

Number of Products under Development for Rett Syndrome, H2 2017

Number of Products under Development by Companies, H2 2017

Number of Products by Top 10 Targets, H2 2017

Number of Products by Stage and Top 10 Targets, H2 2017

Number of Products by Top 10 Mechanism of Actions, H2 2017

Number of Products by Stage and Top 10 Mechanism of Actions, H2 2017

Number of Products by Routes of Administration, H2 2017

Number of Products by Stage and Routes of Administration, H2 2017

Number of Products by Molecule Types, H2 2017

Number of Products by Stage and Molecule Types, H2 2017

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