Fragile X Syndrome - Pipeline Review, H2 2015 Is

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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 preregistration 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
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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
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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
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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 PreClinical Fragile X Syndrome Behavioral Studies 86
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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 NNZ2566 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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