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Fragile X Syndrome - Pipeline Review, H1 2015

Published: May, 2015 | Pages: 89 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

Fragile X Syndrome - Pipeline Review, H1 2015

Summary

Global Markets Direct's, 'Fragile X Syndrome - Pipeline Review, H1 2015', 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 12 Fragile X Syndrome - Pipeline Products Glance 13 Clinical Stage Products 13 Early Stage Products 14 Unknown 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 S.A.S. 18 Alcobra Ltd 19 Confluence Pharmaceuticals LLC 20 Cortex Pharmaceuticals, Inc. 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 Selvita SA 27 Fragile X Syndrome - Therapeutics Assessment 28 Assessment by Monotherapy Products 28 Assessment by Target 29 Assessment by Mechanism of Action 31 Assessment by Route of Administration 33 Assessment by Molecule Type 35 Drug Profiles 37 acamprosate calcium - Drug Profile 37 Product Description 37 Mechanism of Action 37 R&D Progress 37 basimglurant - Drug Profile 38 Product Description 38 Mechanism of Action 38 R&D Progress 38 Bryostatin-1 - Drug Profile 40 Product Description 40 Mechanism of Action 40 R&D Progress 40 cercosporamide - Drug Profile 42 Product Description 42 Mechanism of Action 42 R&D Progress 42 CX-929 - Drug Profile 44 Product Description 44 Mechanism of Action 44 R&D Progress 44 Drugs to Inhibit mGluR5 for Fragile X Syndrome - Drug Profile 45 Product Description 45 Mechanism of Action 45 R&D Progress 45 flindokalner - Drug Profile 46 Product Description 46 Mechanism of Action 46 R&D Progress 46 gaboxadol - Drug Profile 47 Product Description 47 Mechanism of Action 47 R&D Progress 47 ganaxolone - Drug Profile 49 Product Description 49 Mechanism of Action 49 R&D Progress 49 Metadoxine ER - Drug Profile 51 Product Description 51 Mechanism of Action 51 R&D Progress 51 minocycline - Drug Profile 53 Product Description 53 Mechanism of Action 53 R&D Progress 53 NNZ-2591 - Drug Profile 54 Product Description 54 Mechanism of Action 54 R&D Progress 54 SEL-203 - Drug Profile 55 Product Description 55 Mechanism of Action 55 R&D Progress 55 Small Molecule for FXTAS - Drug Profile 56 Product Description 56 Mechanism of Action 56 R&D Progress 56 Small Molecule to Activate Epsilon PKC for Fragile X Syndrome - Drug Profile 57 Product Description 57 Mechanism of Action 57 R&D Progress 57 Small Molecule to Antagonize CB1R for Fragile X Syndrome and Down Syndrome - Drug Profile 58 Product Description 58 Mechanism of Action 58 R&D Progress 58 Small Molecules to Agonize 5-HT7 for Fragile X Syndrome - 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 trofinetide - Drug Profile 62 Product Description 62 Mechanism of Action 62 R&D Progress 62 Fragile X Syndrome - Recent Pipeline Updates 64 Fragile X Syndrome - Dormant Projects 78 Fragile X Syndrome - Discontinued Products 80 Fragile X Syndrome - Product Development Milestones 81 Featured News & Press Releases 81 Apr 16, 2015: Neurotrope Receives Research Grant From the FRAXA Research Foundation to Fund Pre-Clinical Fragile X Syndrome Behavioral Studies 81 Apr 09, 2015: Neurotrope Receives FDA Orphan Drug Designation for Bryostatin in the Treatment of Fragile X Syndrome 81 Mar 02, 2015: Neuren receives FDA response to request for Breakthrough Therapy 82 Feb 16, 2015: Neuren receives Orphan Drug designation from FDA for trofinetide in Rett syndrome 83 Jan 28, 2015: Neuren Announces Trofinetide as Proposed International Nonproprietary Name for NNZ-2566 83 Jan 27, 2015: Marinus Pharmaceuticals to Participate in Canaccord Genuity Rare Disease Biopharma Day 84 Dec 30, 2014: Neuren submits Orphan Drug and Breakthrough Therapy applications to the FDA 84 Nov 13, 2014: Marinus Pharmaceuticals Provides Update On Ganaxolone 84 Oct 23, 2014: Alcobra Announces Presentation of New Data From Phase III Adult ADHD Study at AACAP Annual Meeting 85 Sep 02, 2014: Alcobra Announces Enrollment of First Patient in Phase IIb Clinical Trial of MDX in Adolescents With ADHD 87 Appendix 88 Methodology 88 Coverage 88 Secondary Research 88 Primary Research 88 Expert Panel Validation 88 Contact Us 88 Disclaimer 89
List of Tables
Number of Products under Development for Fragile X Syndrome, H1 2015 9 Number of Products under Development for Fragile X Syndrome - Comparative Analysis, H1 2015 10 Number of Products under Development by Companies, H1 2015 11 Number of Products under Investigation by Universities/Institutes, H1 2015 12 Comparative Analysis by Clinical Stage Development, H1 2015 13 Comparative Analysis by Early Stage Development, H1 2015 14 Comparative Analysis by Unknown Stage Development, H1 2015 15 Products under Development by Companies, H1 2015 16 Products under Investigation by Universities/Institutes, H1 2015 17 Fragile X Syndrome - Pipeline by Aelis Farma S.A.S., H1 2015 18 Fragile X Syndrome - Pipeline by Alcobra Ltd, H1 2015 19 Fragile X Syndrome - Pipeline by Confluence Pharmaceuticals LLC, H1 2015 20 Fragile X Syndrome - Pipeline by Cortex Pharmaceuticals, Inc., H1 2015 21 Fragile X Syndrome - Pipeline by Eli Lilly and Company, H1 2015 22 Fragile X Syndrome - Pipeline by F. Hoffmann-La Roche Ltd., H1 2015 23 Fragile X Syndrome - Pipeline by Marinus Pharmaceuticals, Inc., H1 2015 24 Fragile X Syndrome - Pipeline by MI.TO. Technology S.r.L., H1 2015 25 Fragile X Syndrome - Pipeline by Neuren Pharmaceuticals Limited, H1 2015 26 Fragile X Syndrome - Pipeline by Selvita SA, H1 2015 27 Assessment by Monotherapy Products, H1 2015 28 Number of Products by Stage and Target, H1 2015 30 Number of Products by Stage and Mechanism of Action, H1 2015 32 Number of Products by Stage and Route of Administration, H1 2015 34 Number of Products by Stage and Molecule Type, H1 2015 36 Fragile X Syndrome Therapeutics - Recent Pipeline Updates, H1 2015 64 Fragile X Syndrome - Dormant Projects, H1 2015 78 Fragile X Syndrome - Dormant Projects (Contd..1), H1 2015 79 Fragile X Syndrome - Discontinued Products, H1 2015 80



                                

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