Summary
Global Markets Direct's 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
Global Markets Direct's 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 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. 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 to buy
- 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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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
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 2018