Fragile X Syndrome – Pipeline Review, H1 2018

Global Markets Direct’s, ‘Fragile X Syndrome – Pipeline Review, H1 2018’, 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 Co

Epigen Biosciences Inc

Fulcrum Therapeutics Inc

GlaxoSmithKline Plc

GW Pharmaceuticals Plc

Marinus Pharmaceuticals Inc

Neuren Pharmaceuticals Ltd

Neuron Biopharma SA

Novartis AG

Ovid Therapeutics Inc

Sage Therapeutics Inc

Sentinel Oncology Ltd

Tetra Discovery Partners LLC

Zynerba Pharmaceuticals Inc

Table of Contents

Table of Contents

List of Tables

List of Figures

Introduction

Global Markets Direct Report Coverage

Fragile X Syndrome Overview

Fragile X Syndrome Therapeutics Development

Pipeline Overview

Pipeline by Companies

Pipeline by Universities/Institutes

Products under Development by Companies

Products under Development by Universities/Institutes

Fragile X Syndrome Therapeutics Assessment

Assessment by Target

Assessment by Mechanism of Action

Assessment by Route of Administration

Assessment by Molecule Type

Fragile X Syndrome Companies Involved in Therapeutics Development

Aelis Farma SAS

AMO Pharma Ltd

Anavex Life Sciences Corp

Confluence Pharmaceuticals LLC

DRI Biosciences Corp

Eli Lilly and Co

Epigen Biosciences Inc

Fulcrum Therapeutics Inc

GlaxoSmithKline Plc

GW Pharmaceuticals Plc

Marinus Pharmaceuticals Inc

Neuren Pharmaceuticals Ltd

Neuron Biopharma SA

Novartis AG

Ovid Therapeutics Inc

Sage Therapeutics Inc

Sentinel Oncology Ltd

Tetra Discovery Partners LLC

Zynerba Pharmaceuticals Inc

Fragile X Syndrome Drug Profiles

acamprosate calcium Drug Profile

Product Description

Mechanism Of Action

R&D Progress

ACT-01 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

AMO-01 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

ANAVEX-273 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

arbaclofen Drug Profile

Product Description

Mechanism Of Action

R&D Progress

AUT-00206 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

BPN-14770 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Bryostatin-1 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

cannabidiol Drug Profile

Product Description

Mechanism Of Action

R&D Progress

cercosporamide Drug Profile

Product Description

Mechanism Of Action

R&D Progress

EPGN-1370 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

flindokalner Drug Profile

Product Description

Mechanism Of Action

R&D Progress

gaboxadol Drug Profile

Product Description

Mechanism Of Action

R&D Progress

ganaxolone Drug Profile

Product Description

Mechanism Of Action

R&D Progress

GWP-42006 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

JRP-655 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

mavoglurant Drug Profile

Product Description

Mechanism Of Action

R&D Progress

NNZ-2591 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

NST-0076 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

SERx-519 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

SGE-872 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

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

Product Description

Mechanism Of Action

R&D Progress

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

Product Description

Mechanism Of Action

R&D Progress

Small Molecules to Activate FMRP for Fragile X Syndrome Drug Profile

Product Description

Mechanism Of Action

R&D Progress

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

Product Description

Mechanism Of Action

R&D Progress

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

Product Description

Mechanism Of Action

R&D Progress

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

Product Description

Mechanism Of Action

R&D Progress

SOL-101 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

TC-2153 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

trofinetide Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Fragile X Syndrome Dormant Projects

Fragile X Syndrome Discontinued Products

Fragile X Syndrome Product Development Milestones

Featured News & Press Releases

Mar 05, 2018: Zynerba Pharmaceuticals Announces Positive Meeting with U.S. Food and Drug Administration and Plans to Conduct a Single Pivotal Study of ZYN002 in Fragile X Syndrome to Support an NDA Filing

Mar 01, 2018: Ovid Therapeutics Announces Multiple Poster Presentations of OV101 at the American Academy of Neurology 70th Annual Meeting

Jan 03, 2018: Zynerba Pharmaceuticals Provides Update on cannabidiol

Nov 28, 2017: Ovid Therapeutics Announces OV101 Shows Comparable PK Profile in Phase 1 Study of Adolescent Patients and Amends Phase 2 STARS Clinical Trial Protocol to Include Adolescents

Nov 07, 2017: New Preclinical Research Suggests Utility of Tetra Discovery Partners' BPN14770 to Provide Therapeutic Benefit in Fragile X Syndrome

Oct 16, 2017: Ovid Therapeutics Announces Positive Preclinical OV101 Data Demonstrating Behavioral Improvements in Fragile X Syndrome Model

Oct 10, 2017: Ovid Therapeutics Receives Orphan Drug Designation from the U.S. FDA for OV101 for Treatment of Fragile X Syndrome

Oct 05, 2017: Ovid Therapeutics to Present Preclinical Data Demonstrating OV101 Normalizes Behavioral Abnormalities at the 18th International Fragile X and Related Neurodevelopmental Disorders Workshop

Sep 28, 2017: Zynerba Pharmaceuticals Announces Positive Top Line Results in ZYN002 Open Label Phase 2 FAB-C Study in Children with Fragile X Syndrome

Jul 10, 2017: Autifony Therapeutics receives Orphan Drug Designation for AUT00206 for Fragile X Syndrome

Jun 08, 2017: Zynerba Pharmaceuticals Achieves Target Enrollment in Exploratory Phase 2 Trial of ZYN002 in Fragile X Syndrome

May 31, 2017: Neuren announces grant of two new patents for trofinetide to 2032

May 22, 2017: Anavex Life Sciences Reports New Data for ANAVEX 2-73 in Neurodevelopmental Disorders Including Angelman Syndrome-Data Presented at Antiepileptic Drug Trials XIV 2017 Conference

Apr 18, 2017: Marinus to present Ganaxolone DATA at American academy of neurology annual meeting

Apr 10, 2017: Ovid Therapeutics Announces Initiation of Phase 1 Clinical Trial of OV101 for Adolescents with Angelman Syndrome or Fragile X Syndrome

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer

List of Tables

List of Tables

Number of Products under Development for Fragile X Syndrome, H1 2018

Number of Products under Development by Companies, H1 2018

Number of Products under Development by Companies, H1 2018 (Contd..1), H1 2018

Number of Products under Development by Universities/Institutes, H1 2018

Products under Development by Companies, H1 2018

Products under Development by Companies, H1 2018 (Contd..1), H1 2018

Products under Development by Universities/Institutes, H1 2018

Number of Products by Stage and Target, H1 2018

Number of Products by Stage and Target, H1 2018 (Contd..1), H1 2018

Number of Products by Stage and Mechanism of Action, H1 2018

Number of Products by Stage and Mechanism of Action, H1 2018 (Contd..1), H1 2018

Number of Products by Stage and Route of Administration, H1 2018

Number of Products by Stage and Molecule Type, H1 2018

Fragile X Syndrome Pipeline by Aelis Farma SAS, H1 2018

Fragile X Syndrome Pipeline by AMO Pharma Ltd, H1 2018

Fragile X Syndrome Pipeline by Anavex Life Sciences Corp, H1 2018

Fragile X Syndrome Pipeline by Confluence Pharmaceuticals LLC, H1 2018

Fragile X Syndrome Pipeline by DRI Biosciences Corp, H1 2018

Fragile X Syndrome Pipeline by Eli Lilly and Co, H1 2018

Fragile X Syndrome Pipeline by Epigen Biosciences Inc, H1 2018

Fragile X Syndrome Pipeline by Fulcrum Therapeutics Inc, H1 2018

Fragile X Syndrome Pipeline by GlaxoSmithKline Plc, H1 2018

Fragile X Syndrome Pipeline by GW Pharmaceuticals Plc, H1 2018

Fragile X Syndrome Pipeline by Marinus Pharmaceuticals Inc, H1 2018

Fragile X Syndrome Pipeline by Neuren Pharmaceuticals Ltd, H1 2018

Fragile X Syndrome Pipeline by Neuron Biopharma SA, H1 2018

Fragile X Syndrome Pipeline by Novartis AG, H1 2018

Fragile X Syndrome Pipeline by Ovid Therapeutics Inc, H1 2018

Fragile X Syndrome Pipeline by Sage Therapeutics Inc, H1 2018

Fragile X Syndrome Pipeline by Sentinel Oncology Ltd, H1 2018

Fragile X Syndrome Pipeline by Tetra Discovery Partners LLC, H1 2018

Fragile X Syndrome Pipeline by Zynerba Pharmaceuticals Inc, H1 2018

Fragile X Syndrome Dormant Projects, H1 2018

Fragile X Syndrome Dormant Projects, H1 2018 (Contd..1), H1 2018

Fragile X Syndrome Discontinued Products, H1 2018

List of Figures

List of Figures

Number of Products under Development for Fragile X Syndrome, H1 2018

Number of Products under Development by Companies, H1 2018

Number of Products under Development by Universities/Institutes, H1 2018

Number of Products by Top 10 Targets, H1 2018

Number of Products by Stage and Top 10 Targets, H1 2018

Number of Products by Top 10 Mechanism of Actions, H1 2018

Number of Products by Stage and Top 10 Mechanism of Actions, H1 2018

Number of Products by Routes of Administration, H1 2018

Number of Products by Stage and Routes of Administration, H1 2018

Number of Products by Molecule Types, H1 2018

Number of Products by Stage and Molecule Types, H1 2018

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