Fragile X Syndrome - Pipeline Review, H1 2018

  • ID: 4491168
  • Drug Pipelines
  • 133 pages
  • Global Markets Direct
1 of 5

FEATURED COMPANIES

  • Aelis Farma SAS
  • Anavex Life Sciences Corp
  • Eli Lilly and Co
  • Fulcrum Therapeutics Inc
  • GW Pharmaceuticals Plc
  • Novartis AG
  • MORE
Fragile X Syndrome - Pipeline Review, H1 2018

Summary:

This latest Pharmaceutical and Healthcare disease pipeline guide Fragile X Syndrome - Pipeline Review, H1 2018, provides an overview of the Fragile X Syndrome (Genetic Disorders) pipeline landscape.

Fragile X syndrome is a genetic condition that causes a range of developmental problems including learning disabilities and cognitive impairment. Fragile X syndrome is caused by a change in a gene called FMR1. Fragile X syndrome occurs both in male and female. Symptoms include delay in crawling, walking, or twisting, hyperactive or impulsive behavior, speech and language delay and intellectual disability.

Report Highlights:

This Pharmaceutical and Healthcare latest pipeline guide Fragile X Syndrome - Pipeline Review, H1 2018, provides comprehensive information on the therapeutics under development for Fragile X Syndrome (Genetic Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.

The Fragile X Syndrome (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Fragile X Syndrome and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase III, Phase II, Phase I, Preclinical and Discovery stages are 1, 4, 5, 13 and 2 respectively. Similarly, the Universities portfolio in Preclinical and Discovery stages comprises 4 and 1 molecules, respectively.

Fragile X Syndrome (Genetic Disorders) pipeline guide 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.

The guide is built using data and information sourced from the publisher'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. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

Scope:
  • The pipeline guide provides a snapshot of the global therapeutic landscape of Fragile X Syndrome (Genetic Disorders).
  • The pipeline guide reviews pipeline therapeutics for Fragile X Syndrome (Genetic Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources.
  • The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.
  • The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.
  • The pipeline guide reviews key companies involved in Fragile X Syndrome (Genetic Disorders) therapeutics and enlists all their major and minor projects.
  • The pipeline guide evaluates Fragile X Syndrome (Genetic Disorders) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.
  • The pipeline guide encapsulates all the dormant and discontinued pipeline projects.
  • The pipeline guide reviews latest news related to pipeline therapeutics for Fragile X Syndrome (Genetic Disorders)
Reasons for Purchase:
  • Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
  • Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.
  • Find and recognize significant and varied types of therapeutics under development for Fragile X Syndrome (Genetic Disorders).
  • Classify potential new clients or partners in the target demographic.
  • Develop tactical initiatives by understanding the focus areas of leading companies.
  • Plan mergers and acquisitions meritoriously by identifying key players and it’s most promising pipeline therapeutics.
  • Formulate corrective measures for pipeline projects by understanding Fragile X Syndrome (Genetic Disorders) 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.
  • Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.
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Note: Product cover images may vary from those shown
2 of 5

FEATURED COMPANIES

  • Aelis Farma SAS
  • Anavex Life Sciences Corp
  • Eli Lilly and Co
  • Fulcrum Therapeutics Inc
  • GW Pharmaceuticals Plc
  • Novartis AG
  • MORE
Introduction

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 2

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

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

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
Note: Product cover images may vary from those shown
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  • 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
Note: Product cover images may vary from those shown
5 of 5
Note: Product cover images may vary from those shown
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