Fragile X Syndrome - Pipeline Review, H2 2016

Published: Sep, 2016 | Pages: 117 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

Fragile X Syndrome - Pipeline Review, H2 2016

Summary

Global Markets Direct's, 'Fragile X Syndrome - Pipeline Review, H2 2016', provides an overview of the Fragile X Syndrome pipeline landscape. 

The report provides comprehensive information on the therapeutics under development for Fragile X 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 Fragile X 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 Fragile X Syndrome
- The report reviews pipeline therapeutics for Fragile X 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 Fragile X Syndrome therapeutics and enlists all their major and minor projects
- The report assesses Fragile X 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 Fragile X 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 Fragile X 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 Fragile X 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

 Table of Contents

Table of Contents 2
List of Tables 6
List of Figures 7
Introduction 8
Global Markets Direct Report Coverage 8
Fragile X Syndrome Overview 9
Therapeutics Development 10
Pipeline Products for Fragile X Syndrome - Overview 10
Pipeline Products for Fragile X Syndrome - Comparative Analysis 11
Fragile X Syndrome - Therapeutics under Development by Companies 12
Fragile X Syndrome - Therapeutics under Investigation by Universities/Institutes 14
Fragile X Syndrome - Pipeline Products Glance 15
Late Stage Products 15
Clinical Stage Products 16
Early Stage Products 17
Fragile X Syndrome - Products under Development by Companies 18
Fragile X Syndrome - Products under Investigation by Universities/Institutes 20
Fragile X Syndrome - Companies Involved in Therapeutics Development 21
Aelis Farma SAS 21
Alcobra Ltd 22
AMO Pharma Limited 23
Anavex Life Sciences Corp. 24
Confluence Pharmaceuticals LLC 25
DRI Biosciences Corporation 26
Eli Lilly and Company 27
GlaxoSmithKline Plc 28
GW Pharmaceuticals Plc 29
Kareus Therapeutics, SA 30
Marinus Pharmaceuticals, Inc. 31
Neuren Pharmaceuticals Limited 32
Neuron Biopharma SA 33
Ovid Therapeutics Inc. 34
Sage Therapeutics, Inc. 35
Zynerba Pharmaceuticals, Inc. 36
Fragile X Syndrome - Therapeutics Assessment 37
Assessment by Monotherapy Products 37
Assessment by Target 38
Assessment by Mechanism of Action 40
Assessment by Route of Administration 42
Assessment by Molecule Type 44
Drug Profiles 46
acamprosate calcium - Drug Profile 46
Product Description 46
Mechanism Of Action 46
R&D Progress 46
ACT-01 - Drug Profile 47
Product Description 47
Mechanism Of Action 47
R&D Progress 47
AMO-01 - Drug Profile 48
Product Description 48
Mechanism Of Action 48
R&D Progress 48
ANAVEX-273 - Drug Profile 49
Product Description 49
Mechanism Of Action 49
R&D Progress 49
Bryostatin-1 - Drug Profile 57
Product Description 57
Mechanism Of Action 57
R&D Progress 57
cercosporamide - Drug Profile 61
Product Description 61
Mechanism Of Action 61
R&D Progress 61
Drug to Antagonize mGluR5 for Fragile X Syndrome and 16p11.2 Microdeletion Syndrome - Drug Profile 62
Product Description 62
Mechanism Of Action 62
R&D Progress 62
flindokalner - Drug Profile 63
Product Description 63
Mechanism Of Action 63
R&D Progress 63
gaboxadol - Drug Profile 64
Product Description 64
Mechanism Of Action 64
R&D Progress 64
ganaxolone - Drug Profile 66
Product Description 66
Mechanism Of Action 66
R&D Progress 66
JRP-655 - Drug Profile 71
Product Description 71
Mechanism Of Action 71
R&D Progress 71
KU-046 - Drug Profile 72
Product Description 72
Mechanism Of Action 72
R&D Progress 72
metadoxine ER - Drug Profile 73
Product Description 73
Mechanism Of Action 73
R&D Progress 73
NNZ-2591 - Drug Profile 79
Product Description 79
Mechanism Of Action 79
R&D Progress 79
NST-0076 - Drug Profile 81
Product Description 81
Mechanism Of Action 81
R&D Progress 81
SGE-872 - Drug Profile 82
Product Description 82
Mechanism Of Action 82
R&D Progress 82
Small Molecule for Fragile X-Associated Tremor Ataxia Syndrome - Drug Profile 83
Product Description 83
Mechanism Of Action 83
R&D Progress 83
Small Molecule to Activate FMRP for Fragile X Syndrome - Drug Profile 84
Product Description 84
Mechanism Of Action 84
R&D Progress 84
Small Molecule to Antagonize CB1R for Fragile X Syndrome and Down Syndrome - Drug Profile 85
Product Description 85
Mechanism Of Action 85
R&D Progress 85
Small Molecules to Activate Slack Channels for Fragile X Syndrome - Drug Profile 86
Product Description 86
Mechanism Of Action 86
R&D Progress 86
Small Molecules to Agonize Cannabinoid Receptor 2 for Fragile X Syndrome, Pervasive Developmental Disorder and Rett Syndrome - Drug Profile 87
Product Description 87
Mechanism Of Action 87
R&D Progress 87
Small Molecules to Block Kv3.1 for Fragile X Syndrome - Drug Profile 88
Product Description 88
Mechanism Of Action 88
R&D Progress 88
Small Molecules to Inhibit PI3K for Autism and Fragile X Syndrome - Drug Profile 89
Product Description 89
Mechanism Of Action 89
R&D Progress 89
SRT-278 - Drug Profile 90
Product Description 90
Mechanism Of Action 90
R&D Progress 90
TC-2153 - Drug Profile 91
Product Description 91
Mechanism Of Action 91
R&D Progress 91
trofinetide - Drug Profile 92
Product Description 92
Mechanism Of Action 92
R&D Progress 92
ZYN-002 - Drug Profile 98
Product Description 98
Mechanism Of Action 98
R&D Progress 98
Fragile X Syndrome - Dormant Projects 101
Fragile X Syndrome - Discontinued Products 103
Fragile X Syndrome - Product Development Milestones 104
Featured News & Press Releases 104
Jul 19, 2016: Alcobra Granted European Orphan Drug Designation for Metadoxine in Fragile X Syndrome 104
Jun 28, 2016: Results from Phase 2 Exploratory Clinical Study Support Continued Development of Ganaxolone in Fragile X Syndrome 104
Jun 27, 2016: Marinus Pharmaceuticals to Host Conference Call on Tuesday, June 28 105
Jun 27, 2016: Zynerba Pharmaceuticals Announces Positive Top Line Results from ZYN002 CBD Gel Phase 1 Multiple Rising Dose Trial and Initiation of Phase 2 Study in Adult Epilepsy Patients with Refractory Focal Seizures 106
Jun 22, 2016: Neuren receives grant of new Australian patent for trofinetide to treat Autism Spectrum Disorders 107
Jun 21, 2016: Bryostatin, a PKC epsilon Activator, Generates New Synapses Through Accumulation of the Synaptic Anchoring Protein PSD-95 at Neuronal Membranes 107
Jun 13, 2016: Neurotrope's Chief Scientific Officer Dr. Daniel Alkon Presents at the Gordon Conference: Drugs That Target Common Mechanisms for Synaptic Restoration in Mental Retardation and Alzheimer's Disease 108
Jun 07, 2016: AMO Pharma Presents Pre-clinical Data Showing AMO-01 Successfully Inhibits Activated Pathway Associated with Fragile X Syndrome 108
Jun 06, 2016: Anavex Announces Positive Preclinical Results with ANAVEX 2-73 in Fragile X - Autism-Related Disorders 109
Apr 12, 2016: Cannabis and Cannabinoid Research Publishes Data Demonstrating the Degradation of Cannabidiol to Psychoactive Cannabinoids when Exposed to Simulated Gastric Fluid 110
Mar 08, 2016: Neurotrope Announces Publication Demonstrating Synaptic Regeneration with the PKC epsilon Activator Bryostatin, in a Fragile X Disease Late Brain Development Model 111
Feb 25, 2016: Zynerba Pharmaceuticals Announces ZYN002 Granted Orphan Drug Designation for Fragile X Syndrome 111
Jan 06, 2016: Zynerba Pharmaceuticals Announces Positive Initial Results From ZYN002 CBD Gel Phase 1 Single Rising Dose Trial and Initiation of Phase 1 Multiple Rising Dose Trial 112
Dec 07, 2015: Neuren's trofinetide successful in proof of concept Phase 2 clinical trial in Fragile X syndrome 113
Nov 30, 2015: Neuren receives grant of composition patent in Europe for NNZ-2591 115
Appendix 116
Methodology 116
Coverage 116
Secondary Research 116
Primary Research 116
Expert Panel Validation 116
Contact Us 116
Disclaimer 117

List of Tables

Number of Products under Development for Fragile X Syndrome, H2 2016 10
Number of Products under Development for Fragile X 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
Products under Development by Companies, H2 2016 18
Products under Development by Companies, H2 2016 (Contd..1) 19
Products under Investigation by Universities/Institutes, H2 2016 20
Fragile X Syndrome - Pipeline by Aelis Farma SAS, H2 2016 21
Fragile X Syndrome - Pipeline by Alcobra Ltd, H2 2016 22
Fragile X Syndrome - Pipeline by AMO Pharma Limited, H2 2016 23
Fragile X Syndrome - Pipeline by Anavex Life Sciences Corp., H2 2016 24
Fragile X Syndrome - Pipeline by Confluence Pharmaceuticals LLC, H2 2016 25
Fragile X Syndrome - Pipeline by DRI Biosciences Corporation, H2 2016 26
Fragile X Syndrome - Pipeline by Eli Lilly and Company, H2 2016 27
Fragile X Syndrome - Pipeline by GlaxoSmithKline Plc, H2 2016 28
Fragile X Syndrome - Pipeline by GW Pharmaceuticals Plc, H2 2016 29
Fragile X Syndrome - Pipeline by Kareus Therapeutics, SA, H2 2016 30
Fragile X Syndrome - Pipeline by Marinus Pharmaceuticals, Inc., H2 2016 31
Fragile X Syndrome - Pipeline by Neuren Pharmaceuticals Limited, H2 2016 32
Fragile X Syndrome - Pipeline by Neuron Biopharma SA, H2 2016 33
Fragile X Syndrome - Pipeline by Ovid Therapeutics Inc., H2 2016 34
Fragile X Syndrome - Pipeline by Sage Therapeutics, Inc., H2 2016 35
Fragile X Syndrome - Pipeline by Zynerba Pharmaceuticals, Inc., H2 2016 36
Assessment by Monotherapy Products, H2 2016 37
Number of Products by Stage and Target, H2 2016 39
Number of Products by Stage and Mechanism of Action, H2 2016 41
Number of Products by Stage and Route of Administration, H2 2016 43
Number of Products by Stage and Molecule Type, H2 2016 45
Fragile X Syndrome - Dormant Projects, H2 2016 101
Fragile X Syndrome - Dormant Projects (Contd..1), H2 2016 102
Fragile X Syndrome - Discontinued Products, H2 2016 103