Fragile X Syndrome - Pipeline Review, H1 2016

Published: Apr, 2016 | Pages: 95 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

Fragile X Syndrome - Pipeline Review, H1 2016

Summary

Global Markets Direct's, ‘Fragile X Syndrome - Pipeline Review, H1 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 Content
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 13
Fragile X Syndrome - Pipeline Products Glance 14
Clinical Stage Products 14
Early Stage Products 15
Fragile X Syndrome - Products under Development by Companies 16
Fragile X Syndrome - Products under Investigation by Universities/Institutes 17
Fragile X Syndrome - Companies Involved in Therapeutics Development 18
Aelis Farma SAS 18
Alcobra Ltd 19
AMO Pharma Limited 20
Confluence Pharmaceuticals LLC 21
Eli Lilly and Company 22
F. Hoffmann-La Roche Ltd. 23
Marinus Pharmaceuticals, Inc. 24
MI.TO. Technology S.r.L. 25
Neuren Pharmaceuticals Limited 26
Ovid Therapeutics Inc. 27
Sage Therapeutics, Inc. 28
Zynerba Pharmaceuticals, Inc. 29
Fragile X Syndrome - Therapeutics Assessment 30
Assessment by Monotherapy Products 30
Assessment by Target 31
Assessment by Mechanism of Action 33
Assessment by Route of Administration 35
Assessment by Molecule Type 37
Drug Profiles 39
acamprosate calcium - Drug Profile 39
Product Description 39
Mechanism of Action 39
R&D Progress 39
ACT-01 - Drug Profile 40
Product Description 40
Mechanism of Action 40
R&D Progress 40
AMO-01 - Drug Profile 41
Product Description 41
Mechanism of Action 41
R&D Progress 41
basimglurant - Drug Profile 42
Product Description 42
Mechanism of Action 42
R&D Progress 42
Bryostatin-1 - Drug Profile 44
Product Description 44
Mechanism of Action 44
R&D Progress 44
cercosporamide - Drug Profile 46
Product Description 46
Mechanism of Action 46
R&D Progress 46
flindokalner - Drug Profile 48
Product Description 48
Mechanism of Action 48
R&D Progress 48
gaboxadol - Drug Profile 49
Product Description 49
Mechanism of Action 49
R&D Progress 49
ganaxolone - Drug Profile 51
Product Description 51
Mechanism of Action 51
R&D Progress 51
Metadoxine ER - Drug Profile 53
Product Description 53
Mechanism of Action 53
R&D Progress 53
minocycline - Drug Profile 55
Product Description 55
Mechanism of Action 55
R&D Progress 55
NNZ-2591 - Drug Profile 56
Product Description 56
Mechanism of Action 56
R&D Progress 56
SGE-872 - Drug Profile 57
Product Description 57
Mechanism of Action 57
R&D Progress 57
Small Molecule for FXTAS - Drug Profile 58
Product Description 58
Mechanism of Action 58
R&D Progress 58
Small Molecule to Antagonize CB1R for Fragile X Syndrome and Down Syndrome - Drug Profile 59
Product Description 59
Mechanism of Action 59
R&D Progress 59
Small Molecules to Agonize 5-HT7 for Fragile X Syndrome - Drug Profile 60
Product Description 60
Mechanism of Action 60
R&D Progress 60
Small Molecules to Inhibit PI3K for Autism and Fragile X Syndrome - Drug Profile 61
Product Description 61
Mechanism of Action 61
R&D Progress 61
SRT-278 - Drug Profile 62
Product Description 62
Mechanism of Action 62
R&D Progress 62
trofinetide - Drug Profile 63
Product Description 63
Mechanism of Action 63
R&D Progress 63
ZYN-002 - Drug Profile 66
Product Description 66
Mechanism of Action 66
R&D Progress 66
Fragile X Syndrome - Recent Pipeline Updates 67
Fragile X Syndrome - Dormant Projects 83
Fragile X Syndrome - Discontinued Products 85
Fragile X Syndrome - Product Development Milestones 86
Featured News & Press Releases 86
Mar 08, 2016: Neurotrope Announces Publication Demonstrating Synaptic Regeneration with the PKC epsilon Activator Bryostatin, in a Fragile X Disease Late Brain Development Model 86
Feb 25, 2016: Zynerba Pharmaceuticals Announces ZYN002 Granted Orphan Drug Designation for Fragile X Syndrome 86
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 87
Dec 07, 2015: Neuren's trofinetide successful in proof of concept Phase 2 clinical trial in Fragile X syndrome 88
Nov 30, 2015: Neuren receives grant of composition patent in Europe for NNZ-2591 90
Sep 29, 2015: Neuren provides update on Fragile X and

List of Tables

Number of Products under Development for Fragile X Syndrome, H1 2016 10
Number of Products under Development for Fragile X Syndrome - Comparative Analysis, H1 2016 11
Number of Products under Development by Companies, H1 2016 12
Number of Products under Investigation by Universities/Institutes, H1 2016 13
Comparative Analysis by Clinical Stage Development, H1 2016 14
Comparative Analysis by Early Stage Development, H1 2016 15
Products under Development by Companies, H1 2016 16
Products under Investigation by Universities/Institutes, H1 2016 17
Fragile X Syndrome - Pipeline by Aelis Farma SAS, H1 2016 18
Fragile X Syndrome - Pipeline by Alcobra Ltd, H1 2016 19
Fragile X Syndrome - Pipeline by AMO Pharma Limited, H1 2016 20
Fragile X Syndrome - Pipeline by Confluence Pharmaceuticals LLC, H1 2016 21
Fragile X Syndrome - Pipeline by Eli Lilly and Company, H1 2016 22
Fragile X Syndrome - Pipeline by F. Hoffmann-La Roche Ltd., H1 2016 23
Fragile X Syndrome - Pipeline by Marinus Pharmaceuticals, Inc., H1 2016 24
Fragile X Syndrome - Pipeline by MI.TO. Technology S.r.L., H1 2016 25
Fragile X Syndrome - Pipeline by Neuren Pharmaceuticals Limited, H1 2016 26
Fragile X Syndrome - Pipeline by Ovid Therapeutics Inc., H1 2016 27
Fragile X Syndrome - Pipeline by Sage Therapeutics, Inc., H1 2016 28
Fragile X Syndrome - Pipeline by Zynerba Pharmaceuticals, Inc., H1 2016 29
Assessment by Monotherapy Products, H1 2016 30
Number of Products by Stage and Target, H1 2016 32
Number of Products by Stage and Mechanism of Action, H1 2016 34
Number of Products by Stage and Route of Administration, H1 2016 36
Number of Products by Stage and Molecule Type, H1 2016 38
Fragile X Syndrome Therapeutics - Recent Pipeline Updates, H1 2016 67
Fragile X Syndrome - Dormant Projects, H1 2016 83
Fragile X Syndrome - Dormant Projects (Contd..1), H1 2016 84
Fragile X Syndrome - Discontinued Products, H1 2016 85