Fragile X Syndrome – Pipeline Review, H2 2016

Global Markets Direct’s, ‘Fragile X Syndrome – Pipeline Review, H2 2016’, 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

Alcobra Ltd

AMO Pharma Limited

Anavex Life Sciences Corp.

Confluence Pharmaceuticals LLC

DRI Biosciences Corporation

Eli Lilly and Company

GlaxoSmithKline Plc

GW Pharmaceuticals Plc

Kareus Therapeutics, SA

Marinus Pharmaceuticals, Inc.

Neuren Pharmaceuticals Limited

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 6

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 14

Fragile X Syndrome - Pipeline Products Glance 15

Late Stage Products 15

Clinical Stage Products 16

Early Stage Products 17

Fragile X Syndrome - Products under Development by Companies 18

Fragile X Syndrome - Products under Investigation by Universities/Institutes 20

Fragile X Syndrome - Companies Involved in Therapeutics Development 21

Aelis Farma SAS 21

Alcobra Ltd 22

AMO Pharma Limited 23

Anavex Life Sciences Corp. 24

Confluence Pharmaceuticals LLC 25

DRI Biosciences Corporation 26

Eli Lilly and Company 27

GlaxoSmithKline Plc 28

GW Pharmaceuticals Plc 29

Kareus Therapeutics, SA 30

Marinus Pharmaceuticals, Inc. 31

Neuren Pharmaceuticals Limited 32

Neuron Biopharma SA 33

Ovid Therapeutics Inc. 34

Sage Therapeutics, Inc. 35

Zynerba Pharmaceuticals, Inc. 36

Fragile X Syndrome - Therapeutics Assessment 37

Assessment by Monotherapy Products 37

Assessment by Target 38

Assessment by Mechanism of Action 40

Assessment by Route of Administration 42

Assessment by Molecule Type 44

Drug Profiles 46

acamprosate calcium - Drug Profile 46

Product Description 46

Mechanism Of Action 46

R&D Progress 46

ACT-01 - Drug Profile 47

Product Description 47

Mechanism Of Action 47

R&D Progress 47

AMO-01 - Drug Profile 48

Product Description 48

Mechanism Of Action 48

R&D Progress 48

ANAVEX-273 - Drug Profile 49

Product Description 49

Mechanism Of Action 49

R&D Progress 49

Bryostatin-1 - Drug Profile 57

Product Description 57

Mechanism Of Action 57

R&D Progress 57

cercosporamide - Drug Profile 61

Product Description 61

Mechanism Of Action 61

R&D Progress 61

Drug to Antagonize mGluR5 for Fragile X Syndrome and 16p11.2 Microdeletion Syndrome - Drug Profile 62

Product Description 62

Mechanism Of Action 62

R&D Progress 62

flindokalner - Drug Profile 63

Product Description 63

Mechanism Of Action 63

R&D Progress 63

gaboxadol - Drug Profile 64

Product Description 64

Mechanism Of Action 64

R&D Progress 64

ganaxolone - Drug Profile 66

Product Description 66

Mechanism Of Action 66

R&D Progress 66

JRP-655 - Drug Profile 71

Product Description 71

Mechanism Of Action 71

R&D Progress 71

KU-046 - Drug Profile 72

Product Description 72

Mechanism Of Action 72

R&D Progress 72

metadoxine ER - Drug Profile 73

Product Description 73

Mechanism Of Action 73

R&D Progress 73

NNZ-2591 - Drug Profile 79

Product Description 79

Mechanism Of Action 79

R&D Progress 79

NST-0076 - Drug Profile 81

Product Description 81

Mechanism Of Action 81

R&D Progress 81

SGE-872 - Drug Profile 82

Product Description 82

Mechanism Of Action 82

R&D Progress 82

Small Molecule for Fragile X-Associated Tremor Ataxia Syndrome - Drug Profile 83

Product Description 83

Mechanism Of Action 83

R&D Progress 83

Small Molecule to Activate FMRP for Fragile X Syndrome - Drug Profile 84

Product Description 84

Mechanism Of Action 84

R&D Progress 84

Small Molecule to Antagonize CB1R for Fragile X Syndrome and Down Syndrome - Drug Profile 85

Product Description 85

Mechanism Of Action 85

R&D Progress 85

Small Molecules to Activate Slack Channels for Fragile X Syndrome - Drug Profile 86

Product Description 86

Mechanism Of Action 86

R&D Progress 86

Small Molecules to Agonize Cannabinoid Receptor 2 for Fragile X Syndrome, Pervasive Developmental Disorder and Rett Syndrome - Drug Profile 87

Product Description 87

Mechanism Of Action 87

R&D Progress 87

Small Molecules to Block Kv3.1 for Fragile X Syndrome - Drug Profile 88

Product Description 88

Mechanism Of Action 88

R&D Progress 88

Small Molecules to Inhibit PI3K for Autism and Fragile X Syndrome - Drug Profile 89

Product Description 89

Mechanism Of Action 89

R&D Progress 89

SRT-278 - Drug Profile 90

Product Description 90

Mechanism Of Action 90

R&D Progress 90

TC-2153 - Drug Profile 91

Product Description 91

Mechanism Of Action 91

R&D Progress 91

trofinetide - Drug Profile 92

Product Description 92

Mechanism Of Action 92

R&D Progress 92

ZYN-002 - Drug Profile 98

Product Description 98

Mechanism Of Action 98

R&D Progress 98

Fragile X Syndrome - Dormant Projects 101

Fragile X Syndrome - Discontinued Products 103

Fragile X Syndrome - Product Development Milestones 104

Featured News & Press Releases 104

Jul 19, 2016: Alcobra Granted European Orphan Drug Designation for Metadoxine in Fragile X Syndrome 104

Jun 28, 2016: Results from Phase 2 Exploratory Clinical Study Support Continued Development of Ganaxolone in Fragile X Syndrome 104

Jun 27, 2016: Marinus Pharmaceuticals to Host Conference Call on Tuesday, June 28 105

Jun 27, 2016: Zynerba Pharmaceuticals Announces Positive Top Line Results from ZYN002 CBD Gel Phase 1 Multiple Rising Dose Trial and Initiation of Phase 2 Study in Adult Epilepsy Patients with Refractory Focal Seizures 106

Jun 22, 2016: Neuren receives grant of new Australian patent for trofinetide to treat Autism Spectrum Disorders 107

Jun 21, 2016: Bryostatin, a PKC epsilon Activator, Generates New Synapses Through Accumulation of the Synaptic Anchoring Protein PSD-95 at Neuronal Membranes 107

Jun 13, 2016: Neurotrope's Chief Scientific Officer Dr. Daniel Alkon Presents at the Gordon Conference: Drugs That Target Common Mechanisms for Synaptic Restoration in Mental Retardation and Alzheimer's Disease 108

Jun 07, 2016: AMO Pharma Presents Pre-clinical Data Showing AMO-01 Successfully Inhibits Activated Pathway Associated with Fragile X Syndrome 108

Jun 06, 2016: Anavex Announces Positive Preclinical Results with ANAVEX 2-73 in Fragile X - Autism-Related Disorders 109

Apr 12, 2016: Cannabis and Cannabinoid Research Publishes Data Demonstrating the Degradation of Cannabidiol to Psychoactive Cannabinoids when Exposed to Simulated Gastric Fluid 110

Mar 08, 2016: Neurotrope Announces Publication Demonstrating Synaptic Regeneration with the PKC epsilon Activator Bryostatin, in a Fragile X Disease Late Brain Development Model 111

Feb 25, 2016: Zynerba Pharmaceuticals Announces ZYN002 Granted Orphan Drug Designation for Fragile X Syndrome 111

Jan 06, 2016: Zynerba Pharmaceuticals Announces Positive Initial Results From ZYN002 CBD Gel Phase 1 Single Rising Dose Trial and Initiation of Phase 1 Multiple Rising Dose Trial 112

Dec 07, 2015: Neuren’s trofinetide successful in proof of concept Phase 2 clinical trial in Fragile X syndrome 113

Nov 30, 2015: Neuren receives grant of composition patent in Europe for NNZ-2591 115

Appendix 116

Methodology 116

Coverage 116

Secondary Research 116

Primary Research 116

Expert Panel Validation 116

Contact Us 116

Disclaimer 117

List of Tables

List of Tables

Number of Products under Development for Fragile X Syndrome, H2 2016 10

Number of Products under Development for Fragile X Syndrome – Comparative Analysis, H2 2016 11

Number of Products under Development by Companies, H2 2016 13

Number of Products under Investigation by Universities/Institutes, H2 2016 14

Comparative Analysis by Late Stage Development, H2 2016 15

Comparative Analysis by Clinical Stage Development, H2 2016 16

Comparative Analysis by Early Stage Development, H2 2016 17

Products under Development by Companies, H2 2016 18

Products under Development by Companies, H2 2016 (Contd..1) 19

Products under Investigation by Universities/Institutes, H2 2016 20

Fragile X Syndrome – Pipeline by Aelis Farma SAS, H2 2016 21

Fragile X Syndrome – Pipeline by Alcobra Ltd, H2 2016 22

Fragile X Syndrome – Pipeline by AMO Pharma Limited, H2 2016 23

Fragile X Syndrome – Pipeline by Anavex Life Sciences Corp., H2 2016 24

Fragile X Syndrome – Pipeline by Confluence Pharmaceuticals LLC, H2 2016 25

Fragile X Syndrome – Pipeline by DRI Biosciences Corporation, H2 2016 26

Fragile X Syndrome – Pipeline by Eli Lilly and Company, H2 2016 27

Fragile X Syndrome – Pipeline by GlaxoSmithKline Plc, H2 2016 28

Fragile X Syndrome – Pipeline by GW Pharmaceuticals Plc, H2 2016 29

Fragile X Syndrome – Pipeline by Kareus Therapeutics, SA, H2 2016 30

Fragile X Syndrome – Pipeline by Marinus Pharmaceuticals, Inc., H2 2016 31

Fragile X Syndrome – Pipeline by Neuren Pharmaceuticals Limited, H2 2016 32

Fragile X Syndrome – Pipeline by Neuron Biopharma SA, H2 2016 33

Fragile X Syndrome – Pipeline by Ovid Therapeutics Inc., H2 2016 34

Fragile X Syndrome – Pipeline by Sage Therapeutics, Inc., H2 2016 35

Fragile X Syndrome – Pipeline by Zynerba Pharmaceuticals, Inc., H2 2016 36

Assessment by Monotherapy Products, H2 2016 37

Number of Products by Stage and Target, H2 2016 39

Number of Products by Stage and Mechanism of Action, H2 2016 41

Number of Products by Stage and Route of Administration, H2 2016 43

Number of Products by Stage and Molecule Type, H2 2016 45

Fragile X Syndrome – Dormant Projects, H2 2016 101

Fragile X Syndrome – Dormant Projects (Contd..1), H2 2016 102

Fragile X Syndrome – Discontinued Products, H2 2016 103

List of Figures

List of Figures

Number of Products under Development for Fragile X Syndrome, H2 2016 10

Number of Products under Development for Fragile X Syndrome – Comparative Analysis, H2 2016 11

Number of Products under Development by Companies, H2 2016 12

Number of Products under Investigation by Universities/Institutes, H2 2016 14

Comparative Analysis by Clinical Stage Development, H2 2016 16

Comparative Analysis by Early Stage Products, H2 2016 17

Assessment by Monotherapy Products, H2 2016 37

Number of Products by Top 10 Targets, H2 2016 38

Number of Products by Stage and Top 10 Targets, H2 2016 38

Number of Products by Top 10 Mechanism of Actions, H2 2016 40

Number of Products by Stage and Top 10 Mechanism of Actions, H2 2016 40

Number of Products by Routes of Administration, H2 2016 42

Number of Products by Stage and Routes of Administration, H2 2016 42

Number of Products by Molecule Types, H2 2016 44

Number of Products by Stage and Molecule Types, H2 2016 44

    Pricing

Discounts available for multiple purchases.

reportstore@globalmarketsdirect.com
+44 20 7947 2745

Saved reports