+353-1-416-8900REST OF WORLD
+44-20-3973-8888REST OF WORLD
1-917-300-0470EAST COAST U.S
1-800-526-8630U.S. (TOLL FREE)

PRINTER FRIENDLY

Rett Syndrome - Pipeline Review, H2 2019

  • ID: 4894136
  • Drug Pipelines
  • December 2019
  • Region: Global
  • 165 pages
  • Global Markets Direct
1h Free Analyst Time

Speak directly to the analyst to clarify any post sales queries you may have.

1 of 3

FEATURED COMPANIES

  • AMO Pharma Ltd
  • Anavex Life Sciences Corp
  • AveXis Inc
  • DepYmed Inc
  • Epigen Biosciences Inc
  • Lucy Therapeutics Inc
  • MORE
Rett Syndrome - Pipeline Review, H2 2019

Summary

This latest Pharmaceutical and Healthcare disease pipeline guide Rett Syndrome - Pipeline Review, H2 2019, provides an overview of the Rett Syndrome (Central Nervous System) pipeline landscape.

Rett syndrome is a neurodevelopmental disorder. This is caused by genetic mutation on MECP2 gene. Rett syndrome is described in four stages: Stage I or early onset, Stage II or rapid destruction, Stage III, or the plateau and Stage IV, or the late motor deterioration stage. Symptoms include slowed growth, loss of normal movement and coordination, loss of communication abilities, breathing problems, pain, irregular heartbeat and seizures. Risk factors include family history and gender. Treatment includes disease modifying drugs.

Report Highlights

This Pharmaceutical and Healthcare latest pipeline guide Rett Syndrome - Pipeline Review, H2 2019, provides comprehensive information on the therapeutics under development for Rett Syndrome (Central Nervous System), 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 Rett Syndrome (Central Nervous System) pipeline guide also reviews of key players involved in therapeutic development for Rett 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 3, 6, 4, 8 and 4 respectively. Similarly, the Universities portfolio in Preclinical stages comprises 5 molecules, respectively.

Rett Syndrome (Central Nervous System) 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 Rett Syndrome (Central Nervous System).
  • The pipeline guide reviews pipeline therapeutics for Rett Syndrome (Central Nervous System) 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 Rett Syndrome (Central Nervous System) therapeutics and enlists all their major and minor projects.
  • The pipeline guide evaluates Rett Syndrome (Central Nervous System) 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 Rett Syndrome (Central Nervous System)
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 Rett Syndrome (Central Nervous System).
  • 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 Rett Syndrome (Central Nervous System) 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.
Note: Product cover images may vary from those shown
2 of 3

FEATURED COMPANIES

  • AMO Pharma Ltd
  • Anavex Life Sciences Corp
  • AveXis Inc
  • DepYmed Inc
  • Epigen Biosciences Inc
  • Lucy Therapeutics Inc
  • MORE
Introduction
Report Coverage
Rett Syndrome - Overview
Rett Syndrome - Therapeutics Development
Pipeline Overview
Pipeline by Companies
Pipeline by Universities/Institutes
Products under Development by Companies
Products under Development by Universities/Institutes
Rett Syndrome - Therapeutics Assessment
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Rett Syndrome - Companies Involved in Therapeutics Development
AMO Pharma Ltd
Amorsa Therapeutics Inc
Anavex Life Sciences Corp
ArmaGen Inc
AveXis Inc
BioElectron Technology Corp
Biohaven Pharmaceutical Holding Company Ltd
BioXcel Corp
DepYmed Inc
Eloxx Pharmaceuticals Inc
Epigen Biosciences Inc
GW Pharmaceuticals Plc
Lucy Therapeutics Inc
Mitochon Pharmaceuticals Inc
Mitsubishi Tanabe Pharma Corp
Neuren Pharmaceuticals Ltd
Neurolixis Inc
Neurotrope Bioscience Inc
Newron Pharmaceuticals SpA
PharmatrophiX Inc
Relmada Therapeutics Inc
Starwise Therapeutics LLC
Thiogenesis Therapeutics SARL
Ultragenyx Pharmaceutical Inc
Rett Syndrome - Drug Profiles
AVXS-201 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
BHV-5000 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
blarcamesine hydrochloride - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Bryostatin-1 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
BXCL-902 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
cannabidiol - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
CPT-157633 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Drugs for Rett Syndrome - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
ELX-02 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
fingolimod hydrochloride - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Fusion Protein for Rett Syndrome - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene therapy to Activate MECP2 for Rett Syndrome - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate MECP2 Protein for Rett Syndrome - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
GWP-42006 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
LM-22A4 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
MP-101 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
NLX-101 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
REL-1017 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
sarizotan hydrochloride - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecule to Agonize mGlu5 for Rett Syndrome - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecule to Agonize mGlu7 for Rett Syndrome - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecules to Agonize Sigma-1 Receptor for Metabolic and entral Nervous System Disorders - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecules to Antagonize NMDA Receptor for Rett Syndrome - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecules to Inhibit HDAC6 for Rett Syndrome - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecules to Inhibit PTP1B for Rett Syndrome - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
tianeptine - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
triheptanoin - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
trofinetide - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
TTI-0102 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
vatiquinone - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Rett Syndrome - Dormant Projects
Rett Syndrome - Product Development Milestones
Featured News & Press Releases
Nov 19, 2019: Newron receives FDA Rare Pediatric Disease Designation for Sarizotan for the treatment of Rett Syndrome
Nov 14, 2019: Anavex Life Sciences receives Rare Pediatric Disease Designation from FDA for ANAVEX2-73 (blarcamesine) for the treatment of Rett Syndrome
Nov 06, 2019: Anavex Life Sciences announces publication of foundational data for ANAVEX2-73 (blarcamesine) in Rett Syndrome
Oct 31, 2019: Acadia initiates Phase III study of trofinetide to treat Rett syndrome
Oct 02, 2019: Anavex Life Sciences Issued New U.S. Patent for Composition of Matter Covering ANAVEX2-73 for the Treatment of Alzheimer’s Disease
Sep 27, 2019: Anavex Life Sciences presented at the 6th Annual European Rett Syndrome Conference preliminary clinical efficacy data from the U.S. Rett Syndrome Phase 2 ANAVEX2-73 Study
Sep 16, 2019: Anavex Life Sciences announces preliminary clinical efficacy data of its U.S. phase 2 clinical trial of ANAVEX 2-73 in patients with Rett Syndrome
Sep 04, 2019: Anavex Life Sciences announces initiation of the EXCELLENCE ANAVEX 2-73-RS-003 clinical study in pediatric patients with Rett Syndrome
Jul 31, 2019: Anavex Life Sciences reports recent data review by the independent data safety monitoring board for its US phase 2 clinical trial of ANAVEX 2-73 in patients with Rett syndrome
Jul 29, 2019: Anavex Life Sciences receives positive opinion for orphan designation from the European Medicines Agency for ANAVEX2-73 for the treatment of rett syndrome
Jul 17, 2019: Anavex Life Sciences presents new clinical data identifying gut microbiota biomarkers asociated with improved clinical response in patients treated with ANAVEX2-73 at 2019 Alzheimer’s Association International Conference (AAIC)
Jun 25, 2019: Anavex Life Sciences announces first patient dosed in extension study to its U.S. phase 2 clinical trial of ANAVEX2-73 in patients for Rett Syndrome
Jun 06, 2019: Anavex Life Sciences announces first patient dosed in Phase 2 clinical trial of ANAVEX2-73 for the treatment of Rett Syndrome (AVATAR)
May 08, 2019: Rett Syndrome Association of Australia and Anavex Life Sciences announce initiation of the phase 2 AVATAR clinical study in patients with Rett Syndrome
Mar 27, 2019: Positive phase 2 study results of Trofinetide in pediatric Rett Syndrome published in Neurology, the medical journal of the American Academy of Neurology
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer

List of Tables
Number of Products under Development for Rett Syndrome, H2 2019
Number of Products under Development by Companies, H2 2019
Number of Products under Development by Companies, H2 2019 (Contd..1), H2 2019
Number of Products under Development by Universities/Institutes, H2 2019
Products under Development by Companies, H2 2019
Products under Development by Companies, H2 2019 (Contd..1), H2 2019
Products under Development by Universities/Institutes, H2 2019
Number of Products by Stage and Target, H2 2019
Number of Products by Stage and Mechanism of Action, H2 2019
Number of Products by Stage and Route of Administration, H2 2019
Number of Products by Stage and Molecule Type, H2 2019
Rett Syndrome - Pipeline by AMO Pharma Ltd, H2 2019
Rett Syndrome - Pipeline by Amorsa Therapeutics Inc, H2 2019
Rett Syndrome - Pipeline by Anavex Life Sciences Corp, H2 2019
Rett Syndrome - Pipeline by ArmaGen Inc, H2 2019
Rett Syndrome - Pipeline by AveXis Inc, H2 2019
Rett Syndrome - Pipeline by BioElectron Technology Corp, H2 2019
Rett Syndrome - Pipeline by Biohaven Pharmaceutical Holding Company Ltd, H2 2019
Rett Syndrome - Pipeline by BioXcel Corp, H2 2019
Rett Syndrome - Pipeline by DepYmed Inc, H2 2019
Rett Syndrome - Pipeline by Eloxx Pharmaceuticals Inc, H2 2019
Rett Syndrome - Pipeline by Epigen Biosciences Inc, H2 2019
Rett Syndrome - Pipeline by GW Pharmaceuticals Plc, H2 2019
Rett Syndrome - Pipeline by Lucy Therapeutics Inc, H2 2019
Rett Syndrome - Pipeline by Mitochon Pharmaceuticals Inc, H2 2019
Rett Syndrome - Pipeline by Mitsubishi Tanabe Pharma Corp, H2 2019
Rett Syndrome - Pipeline by Neuren Pharmaceuticals Ltd, H2 2019
Rett Syndrome - Pipeline by Neurolixis Inc, H2 2019
Rett Syndrome - Pipeline by Neurotrope Bioscience Inc, H2 2019
Rett Syndrome - Pipeline by Newron Pharmaceuticals SpA, H2 2019
Rett Syndrome - Pipeline by PharmatrophiX Inc, H2 2019
Rett Syndrome - Pipeline by Relmada Therapeutics Inc, H2 2019
Rett Syndrome - Pipeline by Starwise Therapeutics LLC, H2 2019
Rett Syndrome - Pipeline by Thiogenesis Therapeutics SARL, H2 2019
Rett Syndrome - Pipeline by Ultragenyx Pharmaceutical Inc, H2 2019
Rett Syndrome - Dormant Projects, H2 2019

List of Figures
Number of Products under Development for Rett Syndrome, H2 2019
Number of Products under Development by Companies, H2 2019
Number of Products by Top 10 Targets, H2 2019
Number of Products by Stage and Top 10 Targets, H2 2019
Number of Products by Top 10 Mechanism of Actions, H2 2019
Number of Products by Stage and Top 10 Mechanism of Actions, H2 2019
Number of Products by Routes of Administration, H2 2019
Number of Products by Stage and Routes of Administration, H2 2019
Number of Products by Molecule Types, H2 2019
Number of Products by Stage and Molecule Types, H2 2019
Note: Product cover images may vary from those shown
3 of 3

Loading
LOADING...

4 of 3
  • AMO Pharma Ltd
  • Amorsa Therapeutics Inc
  • Anavex Life Sciences Corp
  • ArmaGen Inc
  • AveXis Inc
  • BioElectron Technology Corp
  • Biohaven Pharmaceutical Holding Company Ltd
  • BioXcel Corp
  • DepYmed Inc
  • Eloxx Pharmaceuticals Inc
  • Epigen Biosciences Inc
  • GW Pharmaceuticals Plc
  • Lucy Therapeutics Inc
  • Mitochon Pharmaceuticals Inc
  • Mitsubishi Tanabe Pharma Corp
  • Neuren Pharmaceuticals Ltd
  • Neurolixis Inc
  • Neurotrope Bioscience Inc
  • Newron Pharmaceuticals SpA
  • PharmatrophiX Inc
  • Relmada Therapeutics Inc
  • Starwise Therapeutics LLC
  • Thiogenesis Therapeutics SARL
  • Ultragenyx Pharmaceutical Inc
Note: Product cover images may vary from those shown
Adroll
adroll