Rett Syndrome - Pipeline Review, H2 2016

Published: Oct, 2016 | Pages: 122 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Rett Syndrome - Pipeline Review, H2 2016, 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, H2 2016, 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, Discovery and Unknown stages are 1, 4, 3, 8, 3 and 2 respectively for Similarly, the Universities portfolio in Preclinical stages comprises 3 molecules, respectively for Rett Syndrome.

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.

 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

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