Fragile X Syndrome – Pipeline Review, H1 2017

Global Markets Direct’s, ‘Fragile X Syndrome – Pipeline Review, H1 2017’, 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

Companies mentioned

Aelis Farma SAS

AMO Pharma Ltd

Anavex Life Sciences Corp

Confluence Pharmaceuticals LLC

DRI Biosciences Corp

Eli Lilly and Company

Fulcrum Therapeutics Inc

GlaxoSmithKline Plc

GW Pharmaceuticals Plc

Marinus Pharmaceuticals Inc

Neuren Pharmaceuticals Ltd

Neuron Biopharma SA

Ovid Therapeutics Inc

Sage Therapeutics Inc

Zynerba Pharmaceuticals Inc

Table of Contents

Table of Contents

Table of Contents 2

List of Tables 4

List of Figures 4

Introduction 5

Global Markets Direct Report Coverage 5

Fragile X Syndrome - Overview 6

Fragile X Syndrome - Therapeutics Development 7

Pipeline Overview 7

Pipeline by Companies 8

Pipeline by Universities/Institutes 10

Products under Development by Companies 11

Products under Development by Universities/Institutes 13

Fragile X Syndrome - Therapeutics Assessment 14

Assessment by Target 14

Assessment by Mechanism of Action 16

Assessment by Route of Administration 18

Assessment by Molecule Type 20

Fragile X Syndrome - Companies Involved in Therapeutics Development 22

Aelis Farma SAS 22

AMO Pharma Ltd 22

Anavex Life Sciences Corp 23

Confluence Pharmaceuticals LLC 23

DRI Biosciences Corp 24

Eli Lilly and Company 24

Fulcrum Therapeutics Inc 25

GlaxoSmithKline Plc 25

GW Pharmaceuticals Plc 26

Marinus Pharmaceuticals Inc 26

Neuren Pharmaceuticals Ltd 27

Neuron Biopharma SA 27

Ovid Therapeutics Inc 28

Sage Therapeutics Inc 28

Zynerba Pharmaceuticals Inc 29

Fragile X Syndrome - Drug Profiles 30

acamprosate calcium - Drug Profile 30

ACT-01 - Drug Profile 31

AMO-01 - Drug Profile 32

ANAVEX-273 - Drug Profile 34

Bryostatin-1 - Drug Profile 44

cannabidiol - Drug Profile 49

cercosporamide - Drug Profile 54

Drug to Antagonize mGluR5 for Fragile X Syndrome and 16p11.2 Microdeletion Syndrome - Drug Profile 56

flindokalner - Drug Profile 57

gaboxadol - Drug Profile 58

ganaxolone - Drug Profile 60

JRP-655 - Drug Profile 67

NNZ-2591 - Drug Profile 68

NST-0076 - Drug Profile 70

SAGE-217 - Drug Profile 71

SGE-872 - Drug Profile 74

Small Molecule for Fragile X-Associated Tremor Ataxia Syndrome - Drug Profile 75

Small Molecule to Activate FMRP for Fragile X Syndrome - Drug Profile 76

Small Molecule to Antagonize CB1R for Fragile X Syndrome and Down Syndrome - Drug Profile 77

Small Molecules to Activate Slack Channels for Fragile X Syndrome - Drug Profile 78

Small Molecules to Agonize Cannabinoid Receptor 2 for Fragile X Syndrome, Pervasive Developmental Disorder and Rett Syndrome - Drug Profile 79

Small Molecules to Block Kv3.1 for Fragile X Syndrome - Drug Profile 80

Small Molecules to Inhibit PI3K for Autism and Fragile X Syndrome - Drug Profile 81

SRT-278 - Drug Profile 82

TC-2153 - Drug Profile 83

trofinetide - Drug Profile 84

Fragile X Syndrome - Dormant Projects 92

Fragile X Syndrome - Discontinued Products 94

Fragile X Syndrome - Product Development Milestones 95

Featured News & Press Releases 95

Appendix 105

Methodology 105

Coverage 105

Secondary Research 105

Primary Research 105

Expert Panel Validation 105

Contact Us 105

Disclaimer 106

List of Tables

List of Tables

Number of Products under Development for Fragile X Syndrome, H1 2017

Number of Products under Development by Companies, H1 2017

Number of Products under Development by Universities/Institutes, H1 2017

Products under Development by Companies, H1 2017

Products under Development by Companies, H1 2017 (Contd..1), H1 2017

Products under Development by Universities/Institutes, H1 2017

Number of Products by Stage and Target, H1 2017

Number of Products by Stage and Mechanism of Action, H1 2017

Number of Products by Stage and Route of Administration, H1 2017

Number of Products by Stage and Molecule Type, H1 2017

Fragile X Syndrome – Pipeline by Aelis Farma SAS, H1 2017

Fragile X Syndrome – Pipeline by AMO Pharma Ltd, H1 2017

Fragile X Syndrome – Pipeline by Anavex Life Sciences Corp, H1 2017

Fragile X Syndrome – Pipeline by Confluence Pharmaceuticals LLC, H1 2017

Fragile X Syndrome – Pipeline by DRI Biosciences Corp, H1 2017

Fragile X Syndrome – Pipeline by Eli Lilly and Company, H1 2017

Fragile X Syndrome – Pipeline by Fulcrum Therapeutics Inc, H1 2017

Fragile X Syndrome – Pipeline by GlaxoSmithKline Plc, H1 2017

Fragile X Syndrome – Pipeline by GW Pharmaceuticals Plc, H1 2017

Fragile X Syndrome – Pipeline by Marinus Pharmaceuticals Inc, H1 2017

Fragile X Syndrome – Pipeline by Neuren Pharmaceuticals Ltd, H1 2017

Fragile X Syndrome – Pipeline by Neuron Biopharma SA, H1 2017

Fragile X Syndrome – Pipeline by Ovid Therapeutics Inc, H1 2017

Fragile X Syndrome – Pipeline by Sage Therapeutics Inc, H1 2017

Fragile X Syndrome – Pipeline by Zynerba Pharmaceuticals Inc, H1 2017

Fragile X Syndrome – Dormant Projects, H1 2017

Fragile X Syndrome – Dormant Projects, H1 2017 (Contd..1), H1 2017

Fragile X Syndrome – Discontinued Products, H1 2017

List of Figures

List of Figures

Number of Products under Development for Fragile X Syndrome, H1 2017

Number of Products under Development by Companies, H1 2017

Number of Products under Development by Universities/Institutes, H1 2017

Number of Products by Top 10 Targets, H1 2017

Number of Products by Stage and Top 10 Targets, H1 2017

Number of Products by Top 10 Mechanism of Actions, H1 2017

Number of Products by Stage and Top 10 Mechanism of Actions, H1 2017

Number of Products by Routes of Administration, H1 2017

Number of Products by Stage and Routes of Administration, H1 2017

Number of Products by Molecule Types, H1 2017

Number of Products by Stage and Molecule Types, H1 2017

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