Rett Syndrome – Pipeline Review, H2 2016

Global Markets Direct’s, ‘Rett Syndrome – Pipeline Review, H2 2016’, 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

Anavex Life Sciences Corp.

ArmaGen Inc.

Edison Pharmaceuticals, Inc.

Eloxx Pharmaceuticals, Ltd.

GW Pharmaceuticals Plc

Mitochon Pharmaceuticals, Inc.

Mitsubishi Tanabe Pharma Corporation

Neuren Pharmaceuticals Limited

Neurolixis Inc.

Newron Pharmaceuticals S.p.A.

OPKO Health, Inc.

Oryzon Genomics SA

PharmatrophiX, Inc.

Raptor Pharmaceutical Corp.

Recursion Pharmaceuticals, LLC

Sage Therapeutics, Inc.

Ultragenyx Pharmaceutical Inc.

Table of Contents

Table of Contents

Table of Contents 2

List of Tables 6

List of Figures 6

Introduction 8

Global Markets Direct Report Coverage 8

Rett Syndrome Overview 9

Therapeutics Development 10

Pipeline Products for Rett Syndrome - Overview 10

Pipeline Products for Rett Syndrome - Comparative Analysis 11

Rett Syndrome - Therapeutics under Development by Companies 12

Rett Syndrome - Therapeutics under Investigation by Universities/Institutes 14

Rett Syndrome - Pipeline Products Glance 15

Late Stage Products 15

Clinical Stage Products 16

Early Stage Products 17

Unknown Stage Products 18

Rett Syndrome - Products under Development by Companies 19

Rett Syndrome - Products under Investigation by Universities/Institutes 20

Rett Syndrome - Companies Involved in Therapeutics Development 21

Anavex Life Sciences Corp. 21

ArmaGen Inc. 22

Edison Pharmaceuticals, Inc. 23

Eloxx Pharmaceuticals, Ltd. 24

GW Pharmaceuticals Plc 25

Mitochon Pharmaceuticals, Inc. 26

Mitsubishi Tanabe Pharma Corporation 27

Neuren Pharmaceuticals Limited 28

Neurolixis Inc. 29

Newron Pharmaceuticals S.p.A. 30

OPKO Health, Inc. 31

Oryzon Genomics SA 32

PharmatrophiX, Inc. 33

Raptor Pharmaceutical Corp. 34

Recursion Pharmaceuticals, LLC 35

Sage Therapeutics, Inc. 36

Ultragenyx Pharmaceutical Inc. 37

Rett Syndrome - Therapeutics Assessment 38

Assessment by Monotherapy Products 38

Assessment by Target 39

Assessment by Mechanism of Action 41

Assessment by Route of Administration 43

Assessment by Molecule Type 45

Drug Profiles 47

amlexanox - Drug Profile 47

Product Description 47

Mechanism Of Action 47

R&D Progress 47

ANAVEX-273 - Drug Profile 48

Product Description 48

Mechanism Of Action 48

R&D Progress 48

Bryostatin-1 - Drug Profile 57

Product Description 57

Mechanism Of Action 57

R&D Progress 57

CPT-157633 - Drug Profile 61

Product Description 61

Mechanism Of Action 61

R&D Progress 61

cysteamine DR - Drug Profile 62

Product Description 62

Mechanism Of Action 62

R&D Progress 62

desipramine hydrochloride - Drug Profile 70

Product Description 70

Mechanism Of Action 70

R&D Progress 70

ELX-02 - Drug Profile 71

Product Description 71

Mechanism Of Action 71

R&D Progress 71

fingolimod hydrochloride - Drug Profile 72

Product Description 72

Mechanism Of Action 72

R&D Progress 72

Gene therapy to Activate MECP2 for Rett Syndrome - Drug Profile 78

Product Description 78

Mechanism Of Action 78

R&D Progress 78

LM-22A4 - Drug Profile 79

Product Description 79

Mechanism Of Action 79

R&D Progress 79

MP-101 - Drug Profile 80

Product Description 80

Mechanism Of Action 80

R&D Progress 80

NLX-101 - Drug Profile 81

Product Description 81

Mechanism Of Action 81

R&D Progress 81

Oligonucleotide For Rett Syndrome - Drug Profile 82

Product Description 82

Mechanism Of Action 82

R&D Progress 82

ORY-2001 - Drug Profile 83

Product Description 83

Mechanism Of Action 83

R&D Progress 83

Recombinant Protein for Rett Syndrome - Drug Profile 85

Product Description 85

Mechanism Of Action 85

R&D Progress 85

REV-003 - Drug Profile 86

Product Description 86

Mechanism Of Action 86

R&D Progress 86

SAGE-217 - Drug Profile 87

Product Description 87

Mechanism Of Action 87

R&D Progress 87

sarizotan hydrochloride - Drug Profile 89

Product Description 89

Mechanism Of Action 89

R&D Progress 89

Small Molecule to Activate TrkB Receptor for Central Nervous System, Metabolic Disorders and ENT Disorders - Drug Profile 92

Product Description 92

Mechanism Of Action 92

R&D Progress 92

Small Molecules for Rett Syndrome - Drug Profile 93

Product Description 93

Mechanism Of Action 93

R&D Progress 93

Small Molecules to Agonize Cannabinoid Receptor 2 for Fragile X Syndrome, Pervasive Developmental Disorder and Rett Syndrome - Drug Profile 94

Product Description 94

Mechanism Of Action 94

R&D Progress 94

trofinetide - Drug Profile 95

Product Description 95

Mechanism Of Action 95

R&D Progress 95

UX-007 - Drug Profile 101

Product Description 101

Mechanism Of Action 101

R&D Progress 101

vatiquinone - Drug Profile 106

Product Description 106

Mechanism Of Action 106

R&D Progress 106

Rett Syndrome - Dormant Projects 110

Rett Syndrome - Product Development Milestones 111

Featured News & Press Releases 111

Jun 24, 2016: Neuren presents at the Rettsyndrome.org 2016 Research Symposium 111

Jun 22, 2016: Neuren receives grant of new Australian patent for trofinetide to treat Autism Spectrum Disorders 111

Jun 06, 2016: Anavex Announces Positive Preclinical Results with ANAVEX 2-73 in Fragile X - Autism-Related Disorders 112

May 20, 2016: Anavex Announces U.S. FDA Orphan Drug Designation to ANAVEX 2-73 for the Treatment of Rett Syndrome 113

May 17, 2016: Mitochon Pharmaceuticals Raises $1.6M for Clinical Stage Development 113

Apr 07, 2016: First subject commences Neuren’s pediatric Rett syndrome Phase 2 trial 113

Feb 25, 2016: Anavex Presents Preclinical Results of ANAVEX 2-73 in Rett Syndrome 114

Oct 13, 2015: Rettsyndrome.org confirms financial support for Neuren’s clinical trials of trofinetide 115

Oct 12, 2015: Neuren receives Notice of Allowance for new US patent covering the use of trofinetide to treat Rett syndrome 116

Aug 14, 2015: “trofinetide” confirmed as the International Nonproprietary Name for Neuren’s NNZ-2566 116

Aug 13, 2015: Neuren confirms orphan drug designation in Europe for Rett syndrome and Fragile X syndrome 116

Jul 29, 2015: Neuren reports progress in orphan drug programs 117

Nov 12, 2014: Neuren’s NNZ-2566 successful In Demonstrating Clinical Benefit In Rett syndrome Phase 2 trial 118

Dec 24, 2012: Neuren Pharma Receives FDA Approval For Phase II trial Of NNZ-2566 In Rett Syndrome 119

May 17, 2012: Neuren Holds Pre-IND Meeting On Proposed Rett Syndrome Clinical Trials With FDA 120

Appendix 121

Methodology 121

Coverage 121

Secondary Research 121

Primary Research 121

Expert Panel Validation 121

Contact Us 121

Disclaimer 122

List of Tables

List of Tables

Number of Products under Development for Rett Syndrome, H2 2016 10

Number of Products under Development for Rett Syndrome – Comparative Analysis, H2 2016 11

Number of Products under Development by Companies, H2 2016 13

Number of Products under Investigation by Universities/Institutes, H2 2016 14

Comparative Analysis by Late Stage Development, H2 2016 15

Comparative Analysis by Clinical Stage Development, H2 2016 16

Comparative Analysis by Early Stage Development, H2 2016 17

Comparative Analysis by Unknown Stage Development, H2 2016 18

Products under Development by Companies, H2 2016 19

Products under Investigation by Universities/Institutes, H2 2016 20

Rett Syndrome – Pipeline by Anavex Life Sciences Corp., H2 2016 21

Rett Syndrome – Pipeline by ArmaGen Inc., H2 2016 22

Rett Syndrome – Pipeline by Edison Pharmaceuticals, Inc., H2 2016 23

Rett Syndrome – Pipeline by Eloxx Pharmaceuticals, Ltd., H2 2016 24

Rett Syndrome – Pipeline by GW Pharmaceuticals Plc, H2 2016 25

Rett Syndrome – Pipeline by Mitochon Pharmaceuticals, Inc., H2 2016 26

Rett Syndrome – Pipeline by Mitsubishi Tanabe Pharma Corporation, H2 2016 27

Rett Syndrome – Pipeline by Neuren Pharmaceuticals Limited, H2 2016 28

Rett Syndrome – Pipeline by Neurolixis Inc., H2 2016 29

Rett Syndrome – Pipeline by Newron Pharmaceuticals S.p.A., H2 2016 30

Rett Syndrome – Pipeline by OPKO Health, Inc., H2 2016 31

Rett Syndrome – Pipeline by Oryzon Genomics SA, H2 2016 32

Rett Syndrome – Pipeline by PharmatrophiX, Inc., H2 2016 33

Rett Syndrome – Pipeline by Raptor Pharmaceutical Corp., H2 2016 34

Rett Syndrome – Pipeline by Recursion Pharmaceuticals, LLC, H2 2016 35

Rett Syndrome – Pipeline by Sage Therapeutics, Inc., H2 2016 36

Rett Syndrome – Pipeline by Ultragenyx Pharmaceutical Inc., H2 2016 37

Assessment by Monotherapy Products, H2 2016 38

Number of Products by Stage and Target, H2 2016 40

Number of Products by Stage and Mechanism of Action, H2 2016 42

Number of Products by Stage and Route of Administration, H2 2016 44

Number of Products by Stage and Molecule Type, H2 2016 46

Rett Syndrome – Dormant Projects, H2 2016 110

List of Figures

List of Figures

Number of Products under Development for Rett Syndrome, H2 2016 10

Number of Products under Development for Rett Syndrome – Comparative Analysis, H2 2016 11

Number of Products under Development by Companies, H2 2016 12

Comparative Analysis by Clinical Stage Development, H2 2016 16

Comparative Analysis by Early Stage Products, H2 2016 17

Assessment by Monotherapy Products, H2 2016 38

Number of Products by Top 10 Targets, H2 2016 39

Number of Products by Stage and Top 10 Targets, H2 2016 39

Number of Products by Top 10 Mechanism of Actions, H2 2016 41

Number of Products by Stage and Top 10 Mechanism of Actions, H2 2016 41

Number of Products by Routes of Administration, H2 2016 43

Number of Products by Stage and Routes of Administration, H2 2016 43

Number of Products by Molecule Types, H2 2016 45

Number of Products by Stage and Molecule Types, H2 2016 45

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