Global Rett Syndrome Drug Pipeline Trends 2019: Discovery, Pre-clinical, Clinical, In Approval Therapeutics, Companies and Markets
High levels of pipeline activity are being observed in Rett Syndrome treatment during 2019. Clinical development activities are being undertaken by more than 20 companies including Amicus Therapeutics, AMO Pharma Ltd, Amorsa Therapeutics Inc, Anavex Life Sciences Corp, ArmaGen Inc and others.
A Significant contribution to the Rett Syndrome pipeline is being observed from emerging companies opting for new approaches to clinical development strategy. The early stage and late-stage Rett Syndrome pipeline included 22 compounds, driven by increasing number of targeted therapies. Progress is being made in accelerating compounds into advanced stages. However, fluctuating trends are being observed in average duration across three phases.
Increased understanding of underlying causes of Rett Syndrome condition and increased access to investments is encouraging growth of Rett Syndrome drug pipeline. However, despite high levels of pipeline activity, increasing clinical trial costs and decreasing drug approval rates is requiring companies to focus more on understanding clinical development pipeline compounds.
The research study is structured to provide clear and actionable insights into Rett Syndrome drug pipeline for companies to optimize spending levels by understanding competition, their pipeline products and prospect of further advances over the next few years.
The report addresses the discovery, research and pre-clinical stage active Rett Syndrome therapeutic candidates. Further, drugs in clinical stage including phase 1, phase 2, phase 3 and pre-registration phases are also analyzed.
The current report examines the current Rett Syndrome pipeline along with current development status, regulatory progress, mechanism of action, route of administration, collaborating companies, sponsors, molecule type, and research/discovery details.
In addition to drug overview, recent developments, the report also provides mechanism of action, Pre-clinical trial details and Clinical trial details for each of the drug candidates of Rett Syndrome. Further, orphan drug status, fast track designation, grants awarded and other special status for Rett Syndrome pipeline compounds are also included.
This study was produced independently by VPAResearch from our proprietary databases, primarily to assist users to develop in-depth understanding of 2019 Rett Syndrome pipeline and formulate effective research and development strategies.
RESEARCH METHODOLOGY
Our integrated data reports are built through robust research methodology to ensure our users can build on the insights and data accuracy for their expansion plans. Both primary and secondary research methods are used with stringent quality checks at each stage to evaluate trends, analysis and forecasts.
Scope of the Research
Rett Syndrome Pipeline candidates-
A Significant contribution to the Rett Syndrome pipeline is being observed from emerging companies opting for new approaches to clinical development strategy. The early stage and late-stage Rett Syndrome pipeline included 22 compounds, driven by increasing number of targeted therapies. Progress is being made in accelerating compounds into advanced stages. However, fluctuating trends are being observed in average duration across three phases.
Increased understanding of underlying causes of Rett Syndrome condition and increased access to investments is encouraging growth of Rett Syndrome drug pipeline. However, despite high levels of pipeline activity, increasing clinical trial costs and decreasing drug approval rates is requiring companies to focus more on understanding clinical development pipeline compounds.
The research study is structured to provide clear and actionable insights into Rett Syndrome drug pipeline for companies to optimize spending levels by understanding competition, their pipeline products and prospect of further advances over the next few years.
The report addresses the discovery, research and pre-clinical stage active Rett Syndrome therapeutic candidates. Further, drugs in clinical stage including phase 1, phase 2, phase 3 and pre-registration phases are also analyzed.
The current report examines the current Rett Syndrome pipeline along with current development status, regulatory progress, mechanism of action, route of administration, collaborating companies, sponsors, molecule type, and research/discovery details.
In addition to drug overview, recent developments, the report also provides mechanism of action, Pre-clinical trial details and Clinical trial details for each of the drug candidates of Rett Syndrome. Further, orphan drug status, fast track designation, grants awarded and other special status for Rett Syndrome pipeline compounds are also included.
This study was produced independently by VPAResearch from our proprietary databases, primarily to assist users to develop in-depth understanding of 2019 Rett Syndrome pipeline and formulate effective research and development strategies.
RESEARCH METHODOLOGY
Our integrated data reports are built through robust research methodology to ensure our users can build on the insights and data accuracy for their expansion plans. Both primary and secondary research methods are used with stringent quality checks at each stage to evaluate trends, analysis and forecasts.
Scope of the Research
Rett Syndrome Pipeline candidates-
- Pre-clinical Phase: Discovery, research, pre-clinical
- Early Phase: Phase 1 and Phase 2 Rett Syndrome drugs
- Late phase: Phase 3 and in-approval Rett Syndrome drugs
- Company overview
- Snapshot
- Rett Syndrome therapeutic treatment activities
- Snapshot
- Drug Name
- Alternative Names
- Company
- Originator
- Phase
- Molecule Type
- Orphan Drug Status
- Drug Overview
- Mechanism of Action
- Current Status
- Trial Details
- Pre-clinical trials for each drug candidate
- Clinical trials for each drug candidate
1 TABLE OF CONTENTS
1.1 List of Tables
1.2 List of Figures
2 EXECUTIVE SUMMARY
2.1 Rett Syndrome- Disease Overview
2.2 Rett Syndrome- Pipeline Snapshot
2.3 Rett Syndrome- Pipeline Drugs by Phase
2.4 Rett Syndrome- Pipeline Drugs by Company
2.5 Rett Syndrome- Pipeline Drugs by Mechanism of Action
3 COMPANY WISE PIPELINE ANALYSIS
3.1 Amicus Therapeutics Rett Syndrome Drug Pipeline, H2- 2019
3.2 AMO Pharma Ltd Rett Syndrome Drug Pipeline, H2- 2019
3.3 Amorsa Therapeutics Inc Rett Syndrome Drug Pipeline, H2- 2019
3.4 Anavex Life Sciences Corp Rett Syndrome Drug Pipeline, H2- 2019
3.5 ArmaGen Inc Rett Syndrome Drug Pipeline, H2- 2019
3.6 AveXis Inc Rett Syndrome Drug Pipeline, H2- 2019
3.7 Biohaven Pharmaceutical Holding Company Ltd Rett Syndrome Drug Pipeline, H2- 2019
3.8 Cold Spring Harbor Laboratory Rett Syndrome Drug Pipeline, H2- 2019
3.9 Eloxx Pharmaceuticals Inc Rett Syndrome Drug Pipeline, H2- 2019
3.10 GW Pharmaceuticals Plc Rett Syndrome Drug Pipeline, H2- 2019
3.11 Mitochon Pharmaceuticals Inc Rett Syndrome Drug Pipeline, H2- 2019
3.12 Mitsubishi Tanabe Pharma Corp Rett Syndrome Drug Pipeline, H2- 2019
3.13 Neuren Pharmaceuticals Ltd Rett Syndrome Drug Pipeline, H2- 2019
3.14 Neurolixis Inc Rett Syndrome Drug Pipeline, H2- 2019
3.15 Neurotrope Bioscience Inc Rett Syndrome Drug Pipeline, H2- 2019
3.16 Newron Pharmaceuticals SpA Rett Syndrome Drug Pipeline, H2- 2019
3.17 OPKO Health Inc Rett Syndrome Drug Pipeline, H2- 2019
3.18 Orpheris Inc Rett Syndrome Drug Pipeline, H2- 2019
3.19 PharmatrophiX Inc Rett Syndrome Drug Pipeline, H2- 2019
3.20 Relmada Therapeutics Inc Rett Syndrome Drug Pipeline, H2- 2019
3.21 Starwise Therapeutics LLC Rett Syndrome Drug Pipeline, H2- 2019
3.22 Ultragenyx Pharmaceutical Inc Rett Syndrome Drug Pipeline, H2- 2019
4 PHASE 1 PIPELINE DRUG DETAILS
4.1 Rett Syndrome- Phase 1 Drug Details
4.2 Rett Syndrome- Phase 1 Drug Overview
4.3 Rett Syndrome- Phase 1 Mechanism of Action
4.4 Current Status
4.5 Trial Details
5 PHASE 2 PIPELINE DRUG DETAILS
5.1 Rett Syndrome- Phase 2 Drug Details
5.2 Rett Syndrome- Phase 2 Drug Overview
5.3 Rett Syndrome- Phase 2 Mechanism of Action
5.4 Current Status
5.5 Trial Details
6 PHASE 3 PIPELINE DRUG DETAILS
6.1 Rett Syndrome- Phase 3 Drug Details
6.2 Rett Syndrome- Phase 3 Drug Overview
6.3 Rett Syndrome- Phase 3 Drug Mechanism of Action
6.4 Current Status
6.5 Trial Details
7 PRE-CLINICAL PHASE PIPELINE DRUG DETAILS
7.1 Rett Syndrome- Pre-clinical Phase Drug Details
7.2 Rett Syndrome- Pre-clinical Phase Drug Overview
7.3 Rett Syndrome- Pre-clinical Phase Drug Mechanism of Action
7.4 Current Status
7.5 Trial Details
8 RECENT NEWS AND DEVELOPMENTS
9 APPENDIX
9.1 About VPAResearch
9.2 Sources and Research Methodology
1.1 List of Tables
1.2 List of Figures
2 EXECUTIVE SUMMARY
2.1 Rett Syndrome- Disease Overview
2.2 Rett Syndrome- Pipeline Snapshot
2.3 Rett Syndrome- Pipeline Drugs by Phase
2.4 Rett Syndrome- Pipeline Drugs by Company
2.5 Rett Syndrome- Pipeline Drugs by Mechanism of Action
3 COMPANY WISE PIPELINE ANALYSIS
3.1 Amicus Therapeutics Rett Syndrome Drug Pipeline, H2- 2019
3.2 AMO Pharma Ltd Rett Syndrome Drug Pipeline, H2- 2019
3.3 Amorsa Therapeutics Inc Rett Syndrome Drug Pipeline, H2- 2019
3.4 Anavex Life Sciences Corp Rett Syndrome Drug Pipeline, H2- 2019
3.5 ArmaGen Inc Rett Syndrome Drug Pipeline, H2- 2019
3.6 AveXis Inc Rett Syndrome Drug Pipeline, H2- 2019
3.7 Biohaven Pharmaceutical Holding Company Ltd Rett Syndrome Drug Pipeline, H2- 2019
3.8 Cold Spring Harbor Laboratory Rett Syndrome Drug Pipeline, H2- 2019
3.9 Eloxx Pharmaceuticals Inc Rett Syndrome Drug Pipeline, H2- 2019
3.10 GW Pharmaceuticals Plc Rett Syndrome Drug Pipeline, H2- 2019
3.11 Mitochon Pharmaceuticals Inc Rett Syndrome Drug Pipeline, H2- 2019
3.12 Mitsubishi Tanabe Pharma Corp Rett Syndrome Drug Pipeline, H2- 2019
3.13 Neuren Pharmaceuticals Ltd Rett Syndrome Drug Pipeline, H2- 2019
3.14 Neurolixis Inc Rett Syndrome Drug Pipeline, H2- 2019
3.15 Neurotrope Bioscience Inc Rett Syndrome Drug Pipeline, H2- 2019
3.16 Newron Pharmaceuticals SpA Rett Syndrome Drug Pipeline, H2- 2019
3.17 OPKO Health Inc Rett Syndrome Drug Pipeline, H2- 2019
3.18 Orpheris Inc Rett Syndrome Drug Pipeline, H2- 2019
3.19 PharmatrophiX Inc Rett Syndrome Drug Pipeline, H2- 2019
3.20 Relmada Therapeutics Inc Rett Syndrome Drug Pipeline, H2- 2019
3.21 Starwise Therapeutics LLC Rett Syndrome Drug Pipeline, H2- 2019
3.22 Ultragenyx Pharmaceutical Inc Rett Syndrome Drug Pipeline, H2- 2019
4 PHASE 1 PIPELINE DRUG DETAILS
4.1 Rett Syndrome- Phase 1 Drug Details
4.2 Rett Syndrome- Phase 1 Drug Overview
4.3 Rett Syndrome- Phase 1 Mechanism of Action
4.4 Current Status
4.5 Trial Details
5 PHASE 2 PIPELINE DRUG DETAILS
5.1 Rett Syndrome- Phase 2 Drug Details
5.2 Rett Syndrome- Phase 2 Drug Overview
5.3 Rett Syndrome- Phase 2 Mechanism of Action
5.4 Current Status
5.5 Trial Details
6 PHASE 3 PIPELINE DRUG DETAILS
6.1 Rett Syndrome- Phase 3 Drug Details
6.2 Rett Syndrome- Phase 3 Drug Overview
6.3 Rett Syndrome- Phase 3 Drug Mechanism of Action
6.4 Current Status
6.5 Trial Details
7 PRE-CLINICAL PHASE PIPELINE DRUG DETAILS
7.1 Rett Syndrome- Pre-clinical Phase Drug Details
7.2 Rett Syndrome- Pre-clinical Phase Drug Overview
7.3 Rett Syndrome- Pre-clinical Phase Drug Mechanism of Action
7.4 Current Status
7.5 Trial Details
8 RECENT NEWS AND DEVELOPMENTS
9 APPENDIX
9.1 About VPAResearch
9.2 Sources and Research Methodology
