Fragile X Syndrome - Pipeline Review, H2 2014

Published: Nov, 2014 | Pages: 85 | Publisher: Global Markets Direct
Industry: Healthcare | Report Format: Electronic (PDF)

Fragile X Syndrome - Pipeline Review, H2 2014

Summary

Global Markets Direct's, 'Fragile X Syndrome - Pipeline Review, H2 2014', provides an overview of the Fragile X Syndrome's therapeutic pipeline.

This report provides comprehensive information on the therapeutic development for Fragile X Syndrome, complete with comparative analysis at various stages, therapeutics assessment by drug target, mechanism of action (MoA), route of administration (RoA) and molecule type, along with latest updates, and featured news and press releases. It also reviews key players involved in the therapeutic development for Fragile X Syndrome and special features on late-stage 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, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources, put together by Global Markets Direct's team. Drug profiles/records featured in the report undergoes periodic updation following a stringent set of processes that ensures that all the profiles are updated with the latest set of information. Additionally, processes including live news & deals tracking, browser based alert-box and clinical trials registries tracking ensure that the most recent developments are captured on a real time basis.

The report enhances decision making capabilities and help to create effective counter strategies to gain competitive advantage. It strengthens R&D pipelines by identifying new targets and MOAs to produce first-in-class and best-in-class products.

Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data for the indicated disease.

Scope

- The report provides a snapshot of the global therapeutic landscape of Fragile X Syndrome
- The report reviews key pipeline products under drug profile section which includes, product description, MoA and R&D brief, licensing and collaboration details & other developmental activities 
- The report reviews key players involved in the therapeutics development for Fragile X Syndrome and enlists all their major and minor projects
- The report summarizes all the dormant and discontinued pipeline projects 
- A review of the Fragile X Syndrome products under development by companies and universities/research institutes based on information derived from company and industry-specific sources
- Pipeline products coverage based on various stages of development ranging from pre-registration till discovery and undisclosed stages
- A detailed assessment of monotherapy and combination therapy pipeline projects
- Coverage of the Fragile X Syndrome pipeline on the basis of target, MoA, route of administration and molecule type
- Latest news and deals relating related to pipeline products

Reasons to buy

- Provides strategically significant competitor information, analysis, and insights to formulate effective R&D development strategies
- Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage
- Develop strategic initiatives by understanding the focus areas of leading companies
- Identify and understand important and diverse types of therapeutics under development for Fragile X Syndrome
- Plan mergers and acquisitions effectively by identifying key players of the most promising pipeline
- 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 5
List of Figures 6
Introduction 7
Global Markets Direct Report Coverage 7
Fragile X Syndrome Overview 8
Therapeutics Development 9
Pipeline Products for Fragile X Syndrome - Overview 9
Pipeline Products for Fragile X Syndrome - Comparative Analysis 10
Fragile X Syndrome - Therapeutics under Development by Companies 11
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
Unknown Stage Products 16
Fragile X Syndrome - Products under Development by Companies 17
Fragile X Syndrome - Products under Investigation by Universities/Institutes 18
Fragile X Syndrome - Companies Involved in Therapeutics Development 19
Alcobra Ltd 19
Confluence Pharmaceuticals LLC 20
Cortex Pharmaceuticals, Inc. 21
F. Hoffmann-La Roche Ltd. 22
Hua Medicine Ltd. 23
Marinus Pharmaceuticals, Inc. 24
MI.TO. Technology S.r.L. 25
Neuren Pharmaceuticals Limited 26
Nexgenix Pharmaceuticals, LLC 27
Paratek Pharmaceuticals, Inc. 28
Selvita SA 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
basimglurant - Drug Profile 40
Product Description 40
Mechanism of Action 40
R&D Progress 40
CX-929 - Drug Profile 42
Product Description 42
Mechanism of Action 42
R&D Progress 42
Drugs to Inhibit mGluR5 for Fragile X Syndrome - Drug Profile 43
Product Description 43
Mechanism of Action 43
R&D Progress 43
ganaxolone - Drug Profile 44
Product Description 44
Mechanism of Action 44
R&D Progress 44
Metadoxine ER - Drug Profile 46
Product Description 46
Mechanism of Action 46
R&D Progress 46
minocycline - Drug Profile 48
Product Description 48
Mechanism of Action 48
R&D Progress 48
NNZ-2566 - Drug Profile 49
Product Description 49
Mechanism of Action 49
R&D Progress 49
NNZ-2591 - Drug Profile 51
Product Description 51
Mechanism of Action 51
R&D Progress 51
PTKMS-01 - Drug Profile 53
Product Description 53
Mechanism of Action 53
R&D Progress 53
SEL-203 - Drug Profile 54
Product Description 54
Mechanism of Action 54
R&D Progress 54
Small Molecule for FXTAS - Drug Profile 55
Product Description 55
Mechanism of Action 55
R&D Progress 55
Small Molecule to Activate Epsilon PKC for Fragile X Syndrome - Drug Profile 56
Product Description 56
Mechanism of Action 56
R&D Progress 56
Small Molecules to Agonize 5-HT7 for Fragile X Syndrome - Drug Profile 57
Product Description 57
Mechanism of Action 57
R&D Progress 57
Small molecules to Antagonize mGLUR5 for Fragile X Syndrome - Drug Profile 58
Product Description 58
Mechanism of Action 58
R&D Progress 58
Small Molecules to Inhibit Pak for Genetic Disorders - Drug Profile 59
Product Description 59
Mechanism of Action 59
R&D Progress 59
Small Molecules to Inhibit PI3K for Autism and Fragile X Syndrome - Drug Profile 60
Product Description 60
Mechanism of Action 60
R&D Progress 60
SRT-278 - Drug Profile 61
Product Description 61
Mechanism of Action 61
R&D Progress 61
Fragile X Syndrome - Recent Pipeline Updates 62
Fragile X Syndrome - Dormant Projects 73
Fragile X Syndrome - Discontinued Products 74
Fragile X Syndrome - Product Development Milestones 75
Featured News & Press Releases 75
Nov 13, 2014: Marinus Pharmaceuticals Provides Update On Ganaxolone 75
Nov 12, 2014: Neuren's NNZ-2566 successful In Demonstrating Clinical Benefit In Rett syndrome Phase 2 trial 75
Oct 23, 2014: Alcobra Announces Presentation of New Data From Phase III Adult ADHD Study at AACAP Annual Meeting 77
Sep 02, 2014: Alcobra Announces Enrollment of First Patient in Phase IIb Clinical Trial of MDX in Adolescents With ADHD 79
Aug 19, 2014: Alcobra Announces Enrollment of First Patient in Phase IIb Clinical Trial of MDX in Fragile X Syndrome 79
Aug 12, 2014: Neuren advances NNZ-2591 towards IND application 79
Aug 08, 2014: Alcobra Releases New Neuroimaging Data Supporting Pro-Cognitive Effects of MDX 80
Jul 14, 2014: Alcobra Completes Patient Recruitment in Phase III Clinical Trial of MDX in Adult ADHD 81
Jun 26, 2014: Neuren completes enrolment in Phase 2 trial of NNZ-2566 in Rett Syndrome 82
May 12, 2014: Alcobra Presents Evidence of Novel Mechanism of Action for Metadoxine Extended Release (MDX) in Cognitive Disorders at the Society of Biological Psychiatry Annual Meeting 82
Appendix 84
Methodology 84
Coverage 84
Secondary Research 84
Primary Research 84
Expert Panel Validation 84
Contact Us 85
Disclaimer 85

List of Tables
Number of Products under Development for Fragile X Syndrome, H2 2014 9
Number of Products under Development for Fragile X Syndrome - Comparative Analysis, H2 2014 10
Number of Products under Development by Companies, H2 2014 12
Number of Products under Investigation by Universities/Institutes, H2 2014 13
Comparative Analysis by Clinical Stage Development, H2 2014 14
Comparative Analysis by Early Stage Development, H2 2014 15
Comparative Analysis by Unknown Stage Development, H2 2014 16
Products under Development by Companies, H2 2014 17
Products under Investigation by Universities/Institutes, H2 2014 18
Fragile X Syndrome - Pipeline by Alcobra Ltd, H2 2014 19
Fragile X Syndrome - Pipeline by Confluence Pharmaceuticals LLC, H2 2014 20
Fragile X Syndrome - Pipeline by Cortex Pharmaceuticals, Inc., H2 2014 21
Fragile X Syndrome - Pipeline by F. Hoffmann-La Roche Ltd., H2 2014 22
Fragile X Syndrome - Pipeline by Hua Medicine Ltd., H2 2014 23
Fragile X Syndrome - Pipeline by Marinus Pharmaceuticals, Inc., H2 2014 24
Fragile X Syndrome - Pipeline by MI.TO. Technology S.r.L., H2 2014 25
Fragile X Syndrome - Pipeline by Neuren Pharmaceuticals Limited, H2 2014 26
Fragile X Syndrome - Pipeline by Nexgenix Pharmaceuticals, LLC, H2 2014 27
Fragile X Syndrome - Pipeline by Paratek Pharmaceuticals, Inc., H2 2014 28
Fragile X Syndrome - Pipeline by Selvita SA, H2 2014 29
Assessment by Monotherapy Products, H2 2014 30
Number of Products by Stage and Target, H2 2014 32
Number of Products by Stage and Mechanism of Action, H2 2014 34
Number of Products by Stage and Route of Administration, H2 2014 36
Number of Products by Stage and Molecule Type, H2 2014 38
Fragile X Syndrome Therapeutics - Recent Pipeline Updates, H2 2014 62
Fragile X Syndrome - Dormant Projects, H2 2014 73
Fragile X Syndrome - Discontinued Products, H2 2014 74