Friedreich Ataxia - Pipeline Review, H2 2016

Date: September 21, 2016
Pages: 95
Price:
US$ 2,000.00
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Publisher: Global Markets Direct
Report type: Strategic Report
Delivery: E-mail Delivery (PDF)
ID: FB08A0E3991EN
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Friedreich Ataxia - Pipeline Review, H2 2016

Summary

Global Markets Direct’s, ‘Friedreich Ataxia - Pipeline Review, H2 2016’, provides an overview of the Friedreich Ataxia pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Friedreich Ataxia, 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 Friedreich Ataxia 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 Friedreich Ataxia
  • The report reviews pipeline therapeutics for Friedreich Ataxia 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 Friedreich Ataxia therapeutics and enlists all their major and minor projects
  • The report assesses Friedreich Ataxia 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 Friedreich Ataxia
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 Friedreich Ataxia
  • 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 Friedreich Ataxia 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
Introduction
Global Markets Direct Report Coverage
Friedreich Ataxia Overview
Therapeutics Development
Pipeline Products for Friedreich Ataxia - Overview
Pipeline Products for Friedreich Ataxia - Comparative Analysis
Friedreich Ataxia - Therapeutics under Development by Companies
Friedreich Ataxia - Therapeutics under Investigation by Universities/Institutes
Friedreich Ataxia - Pipeline Products Glance
Late Stage Products
Clinical Stage Products
Early Stage Products
Friedreich Ataxia - Products under Development by Companies
Friedreich Ataxia - Products under Investigation by Universities/Institutes
Friedreich Ataxia - Companies Involved in Therapeutics Development
Adverum Biotechnologies, Inc.
Almirall, S.A.
BioMarin Pharmaceutical Inc.
Biovista Inc.
Catabasis Pharmaceuticals, Inc.
Edison Pharmaceuticals, Inc.
Horizon Pharma Plc
Ixchel Pharma, LLC
ProQR Therapeutics N.V.
RaNA Therapeutics, Inc.
Reata Pharmaceuticals, Inc.
Retrotope, Inc
Shire Plc
STATegics, Inc.
Voyager Therapeutics, Inc.
Friedreich Ataxia - Therapeutics Assessment
Assessment by Monotherapy Products
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Drug Profiles
AGIL-FA - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
ANN-003 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
BVA-202 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
BVA-203 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
CAT-4001 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
dimethyl fumarate - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate Frataxin for Friedreich’s Ataxia - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate Frataxin for Friedreich’s Ataxia - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate Frataxin for Friedrich's Ataxia - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
interferon gamma-1b - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
interferon gamma-1b - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
IXC-101 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
JOT-101 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Oligonucleotide to Activate Frataxin for Friedreich's Ataxia - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
omaveloxolone - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
QRX-604 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Recombinant Protein for Friedreich Ataxia - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
RG-3250 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
RT-001 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
RT-002 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SHP-622 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
STSE-15 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
TAT-FXN - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
vatiquinone - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
VYFXN-01 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Friedreich Ataxia - Dormant Projects
Friedreich Ataxia - Discontinued Products
Friedreich Ataxia - Product Development Milestones
Featured News & Press Releases
Aug 02, 2016: Agilis Biotherapeutics Announces FDA Orphan Drug Designation for the Treatment of Friedreich’s Ataxia
May 05, 2016: Horizon Pharma Completes Target Enrollment of 90 Patients for Phase 3 Trial of ACTIMMUNE(R) (interferon gamma-1b) for the Treatment of People With Friedreich Ataxia
Jan 19, 2016: FARA Announces Catabasis Pharmaceuticals as the Recipient of the Kyle Bryant Translational Research Award to Evaluate CAT-4001 as a Potential Therapy for Friedreich’s Ataxia
Aug 05, 2015: Key Patent Granted For AAVLife’s Gene-Therapy Program to Treat Cardiomyopathy in Friedreich’s Ataxia
Jun 05, 2015: Horizon Pharma Initiates Phase 3 Trial of ACTIMMUNE for the Treatment of People With Friedreich's Ataxia
Apr 10, 2015: Horizon Pharma Receives FDA Fast Track Designation for ACTIMMUNE in the Treatment of Friedreich's Ataxia
Mar 24, 2015: Catabasis Pharmaceuticals to Present at International Ataxia Research Conference
Feb 13, 2015: Horizon Pharma Submits Investigational New Drug Application for ACTIMMUNE in the Treatment of Friedreich's Ataxia
Jan 29, 2015: Reata Enrolls First Patient in the MOXIe Study, a Phase 2/3 Study Examining RTA 408 in Friedreich's Ataxia Patients
Oct 09, 2014: Horizon Pharma Announces Presentation of ACTIMMUNE Phase 2 Data in Friedreich's Ataxia
Oct 03, 2014: Horizon Pharma Receives Orphan-Drug Designation for ACTIMMUNE (Interferon gamma-1b) in Friedreich's Ataxia
Sep 30, 2014: Reata Announces the Initiation of Phase 2 Studies Examining RTA 408 for the Treatment of Friedreich's Ataxia and Mitochondrial Myopathies
Mar 17, 2014: FDA Awards Fast Track Status to Edison Pharmaceuticals' EPI-743 for Friedreich's Ataxia
Feb 04, 2014: FDA Grants Edison Pharmaceuticals' EPI-743 Orphan Status for Friedreich's Ataxia
Nov 04, 2013: Edison Pharmaceuticals, FARA, and USF Announce Initiation of EPI-743 Phase 2 Friedreich's Ataxia Clinical Trial in Adults with Point Mutations
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer 95

LIST OF TABLES

Number of Products under Development for Friedreich Ataxia, H2 2016
Number of Products under Development for Friedreich Ataxia - Comparative Analysis, H2 2016
Number of Products under Development by Companies, H2 2016
Number of Products under Investigation by Universities/Institutes, H2 2016
Comparative Analysis by Late Stage Development, H2 2016
Comparative Analysis by Clinical Stage Development, H2 2016
Comparative Analysis by Early Stage Development, H2 2016
Products under Development by Companies, H2 2016
Products under Investigation by Universities/Institutes, H2 2016
Friedreich Ataxia - Pipeline by Adverum Biotechnologies, Inc., H2 2016
Friedreich Ataxia - Pipeline by Almirall, S.A., H2 2016
Friedreich Ataxia - Pipeline by BioMarin Pharmaceutical Inc., H2 2016
Friedreich Ataxia - Pipeline by Biovista Inc., H2 2016
Friedreich Ataxia - Pipeline by Catabasis Pharmaceuticals, Inc., H2 2016
Friedreich Ataxia - Pipeline by Edison Pharmaceuticals, Inc., H2 2016
Friedreich Ataxia - Pipeline by Horizon Pharma Plc, H2 2016
Friedreich Ataxia - Pipeline by Ixchel Pharma, LLC, H2 2016
Friedreich Ataxia - Pipeline by ProQR Therapeutics N.V., H2 2016
Friedreich Ataxia - Pipeline by RaNA Therapeutics, Inc., H2 2016
Friedreich Ataxia - Pipeline by Reata Pharmaceuticals, Inc., H2 2016
Friedreich Ataxia - Pipeline by Retrotope, Inc, H2 2016
Friedreich Ataxia - Pipeline by Shire Plc, H2 2016
Friedreich Ataxia - Pipeline by STATegics, Inc., H2 2016
Friedreich Ataxia - Pipeline by Voyager Therapeutics, Inc., H2 2016
Assessment by Monotherapy Products, H2 2016
Number of Products by Stage and Target, H2 2016
Number of Products by Stage and Mechanism of Action, H2 2016
Number of Products by Stage and Route of Administration, H2 2016
Number of Products by Stage and Molecule Type, H2 2016
Friedreich Ataxia - Dormant Projects, H2 2016
Friedreich Ataxia - Discontinued Products, H2 2016 83

LIST OF FIGURES

Number of Products under Development for Friedreich Ataxia, H2 2016
Number of Products under Development for Friedreich Ataxia - Comparative Analysis, H2 2016
Number of Products under Development by Companies, H2 2016
Number of Products under Investigation by Universities/Institutes, H2 2016
Comparative Analysis by Late Stage Development, H2 2016
Comparative Analysis by Clinical Stage Development, H2 2016
Comparative Analysis by Early Stage Products, H2 2016
Assessment by Monotherapy Products, H2 2016
Number of Products by Targets, H2 2016
Number of Products by Stage and Targets, H2 2016
Number of Products by Top 10 Mechanism of Actions, H2 2016
Number of Products by Stage and Top 10 Mechanism of Actions, H2 2016
Number of Products by Routes of Administration, H2 2016
Number of Products by Stage and Routes of Administration, H2 2016
Number of Products by Molecule Types, H2 2016
Number of Products by Stage and Molecule Types, H2 2016
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