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Fragile X Syndrome - Pipeline Review, H2 2015 Is Released

Press Release   •   Jan 29, 2016 16:45 IST

Fragile X Syndrome - Pipeline Review, H2 2015 Summary Global Markets Direct s, Fragile X Syndrome - Pipeline Review, H2 2015, provides an overview of the Fragile X Syndromes 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 Directs 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 Directs 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.

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Table of Contents

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 S.A.S. 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

Sage Therapeutics 27

Selvita SA 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

AMO-01 - Drug Profile 40

Product Description 40

Mechanism of Action 40

R&D Progress 40

basimglurant - Drug Profile 41

Product Description 41

Mechanism of Action 41

R&D Progress 41

Bryostatin-1 - Drug Profile 43

Product Description 43

Mechanism of Action 43

R&D Progress 43

cercosporamide - Drug Profile 45

Product Description 45

Mechanism of Action 45

R&D Progress 45

flindokalner - Drug Profile 47

Product Description 47

Mechanism of Action 47

R&D Progress 47

gaboxadol - Drug Profile 48

Product Description 48

Mechanism of Action 48

R&D Progress 48

ganaxolone - Drug Profile 50

Product Description 50

Mechanism of Action 50

R&D Progress 50

Metadoxine ER - Drug Profile 52

Product Description 52

Mechanism of Action 52

R&D Progress 52

minocycline - Drug Profile 54

Product Description 54

Mechanism of Action 54

R&D Progress 54

NNZ-2591 - Drug Profile 55

Product Description 55

Mechanism of Action 55

R&D Progress 55

SEL-203 - 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 65

Product Description 65

Mechanism of Action 65

R&D Progress 65

Fragile X Syndrome - Recent Pipeline Updates 66

Fragile X Syndrome - Dormant Projects 82

Fragile X Syndrome - Discontinued Products 83

Fragile X Syndrome - Product Development Milestones 84

Featured News & Press Releases 84

Nov 30, 2015: Neuren receives grant of composition patent in Europe for NNZ-2591 84

Sep 21, 2015: FDA Grants Fast Track Designation to Alcobra's MDX for Fragile X Syndrome 84

Aug 13, 2015: Neuren confirms orphan drug designation in Europe for Rett syndrome and Fragile X syndrome 85

Jun 24, 2015: Alcobra Announces Results From Phase 2 Clinical Trial Of MDX For Fragile X Syndrome 85

Jun 23, 2015: Alcobra to Present Results of Phase II Fragile X Trial in a Conference Call & Webcast Presentation on Wednesday, June 24 86

Apr 16, 2015: Neurotrope Receives Research Grant from the FRAXA Research Foundation to Fund Pre-Clinical Fragile X Syndrome Behavioral Studies 86

Apr 09, 2015: Neurotrope Receives FDA Orphan Drug Designation for Bryostatin in the Treatment of Fragile X Syndrome 86

Mar 02, 2015: Neuren receives FDA response to request for Breakthrough Therapy 87

Feb 16, 2015: Neuren receives Orphan Drug designation from FDA for trofinetide in Rett syndrome 88

Jan 28, 2015: Neuren Announces Trofinetide as Proposed International Nonproprietary Name for NNZ-2566 88

Appendix 90

Methodology 90

Coverage 90

Secondary Research 90

Primary Research 90

Expert Panel Validation 90

Contact Us 90

Disclaimer 91

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