Friedreich Ataxia - Pipeline Review, H2 2016

Published: Sep, 2016 | Pages: 95 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

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

 Table of Contents

Table of Contents 2
List of Tables 6
List of Figures 7
Introduction 8
Global Markets Direct Report Coverage 8
Friedreich Ataxia Overview 9
Therapeutics Development 10
Pipeline Products for Friedreich Ataxia - Overview 10
Pipeline Products for Friedreich Ataxia - Comparative Analysis 11
Friedreich Ataxia - Therapeutics under Development by Companies 12
Friedreich Ataxia - Therapeutics under Investigation by Universities/Institutes 14
Friedreich Ataxia - Pipeline Products Glance 15
Late Stage Products 15
Clinical Stage Products 16
Early Stage Products 17
Friedreich Ataxia - Products under Development by Companies 18
Friedreich Ataxia - Products under Investigation by Universities/Institutes 19
Friedreich Ataxia - Companies Involved in Therapeutics Development 20
Adverum Biotechnologies, Inc. 20
Almirall, S.A. 21
BioMarin Pharmaceutical Inc. 22
Biovista Inc. 23
Catabasis Pharmaceuticals, Inc. 24
Edison Pharmaceuticals, Inc. 25
Horizon Pharma Plc 26
Ixchel Pharma, LLC 27
ProQR Therapeutics N.V. 28
RaNA Therapeutics, Inc. 29
Reata Pharmaceuticals, Inc. 30
Retrotope, Inc 31
Shire Plc 32
STATegics, Inc. 33
Voyager Therapeutics, Inc. 34
Friedreich Ataxia - Therapeutics Assessment 35
Assessment by Monotherapy Products 35
Assessment by Target 36
Assessment by Mechanism of Action 38
Assessment by Route of Administration 40
Assessment by Molecule Type 42
Drug Profiles 44
AGIL-FA - Drug Profile 44
Product Description 44
Mechanism Of Action 44
R&D Progress 44
ANN-003 - Drug Profile 45
Product Description 45
Mechanism Of Action 45
R&D Progress 45
BVA-202 - Drug Profile 47
Product Description 47
Mechanism Of Action 47
R&D Progress 47
BVA-203 - Drug Profile 48
Product Description 48
Mechanism Of Action 48
R&D Progress 48
CAT-4001 - Drug Profile 49
Product Description 49
Mechanism Of Action 49
R&D Progress 49
dimethyl fumarate - Drug Profile 50
Product Description 50
Mechanism Of Action 50
R&D Progress 50
Gene Therapy to Activate Frataxin for Friedreich's Ataxia - Drug Profile 51
Product Description 51
Mechanism Of Action 51
R&D Progress 51
Gene Therapy to Activate Frataxin for Friedreich's Ataxia - Drug Profile 52
Product Description 52
Mechanism Of Action 52
R&D Progress 52
Gene Therapy to Activate Frataxin for Friedrich's Ataxia - Drug Profile 53
Product Description 53
Mechanism Of Action 53
R&D Progress 53
interferon gamma-1b - Drug Profile 54
Product Description 54
Mechanism Of Action 54
R&D Progress 54
interferon gamma-1b - Drug Profile 58
Product Description 58
Mechanism Of Action 58
R&D Progress 58
IXC-101 - Drug Profile 59
Product Description 59
Mechanism Of Action 59
R&D Progress 59
JOT-101 - Drug Profile 60
Product Description 60
Mechanism Of Action 60
R&D Progress 60
Oligonucleotide to Activate Frataxin for Friedreich's Ataxia - Drug Profile 61
Product Description 61
Mechanism Of Action 61
R&D Progress 61
omaveloxolone - Drug Profile 62
Product Description 62
Mechanism Of Action 62
R&D Progress 62
QRX-604 - Drug Profile 65
Product Description 65
Mechanism Of Action 65
R&D Progress 65
Recombinant Protein for Friedreich Ataxia - Drug Profile 66
Product Description 66
Mechanism Of Action 66
R&D Progress 66
RG-3250 - Drug Profile 67
Product Description 67
Mechanism Of Action 67
R&D Progress 67
RT-001 - Drug Profile 68
Product Description 68
Mechanism Of Action 68
R&D Progress 68
RT-002 - Drug Profile 70
Product Description 70
Mechanism Of Action 70
R&D Progress 70
SHP-622 - Drug Profile 71
Product Description 71
Mechanism Of Action 71
R&D Progress 71
STSE-15 - Drug Profile 73
Product Description 73
Mechanism Of Action 73
R&D Progress 73
TAT-FXN - Drug Profile 75
Product Description 75
Mechanism Of Action 75
R&D Progress 75
vatiquinone - Drug Profile 76
Product Description 76
Mechanism Of Action 76
R&D Progress 76
VYFXN-01 - Drug Profile 81
Product Description 81
Mechanism Of Action 81
R&D Progress 81
Friedreich Ataxia - Dormant Projects 82
Friedreich Ataxia - Discontinued Products 83
Friedreich Ataxia - Product Development Milestones 84
Featured News & Press Releases 84
Aug 02, 2016: Agilis Biotherapeutics Announces FDA Orphan Drug Designation for the Treatment of Friedreich's Ataxia 84
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 84
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 85
Aug 05, 2015: Key Patent Granted For AAVLife's Gene-Therapy Program to Treat Cardiomyopathy in Friedreich's Ataxia 86
Jun 05, 2015: Horizon Pharma Initiates Phase 3 Trial of ACTIMMUNE for the Treatment of People With Friedreich's Ataxia 87
Apr 10, 2015: Horizon Pharma Receives FDA Fast Track Designation for ACTIMMUNE in the Treatment of Friedreich's Ataxia 87
Mar 24, 2015: Catabasis Pharmaceuticals to Present at International Ataxia Research Conference 88
Feb 13, 2015: Horizon Pharma Submits Investigational New Drug Application for ACTIMMUNE in the Treatment of Friedreich's Ataxia 88
Jan 29, 2015: Reata Enrolls First Patient in the MOXIe Study, a Phase 2/3 Study Examining RTA 408 in Friedreich's Ataxia Patients 89
Oct 09, 2014: Horizon Pharma Announces Presentation of ACTIMMUNE Phase 2 Data in Friedreich's Ataxia 89
Oct 03, 2014: Horizon Pharma Receives Orphan-Drug Designation for ACTIMMUNE (Interferon gamma-1b) in Friedreich's Ataxia 90
Sep 30, 2014: Reata Announces the Initiation of Phase 2 Studies Examining RTA 408 for the Treatment of Friedreich's Ataxia and Mitochondrial Myopathies 90
Mar 17, 2014: FDA Awards Fast Track Status to Edison Pharmaceuticals' EPI-743 for Friedreich's Ataxia 91
Feb 04, 2014: FDA Grants Edison Pharmaceuticals' EPI-743 Orphan Status for Friedreich's Ataxia 92
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 92
Appendix 94
Methodology 94
Coverage 94
Secondary Research 94
Primary Research 94
Expert Panel Validation 94
Contact Us 94
Disclaimer 95

List of Tables

Number of Products under Development for Friedreich Ataxia, H2 2016 10
Number of Products under Development for Friedreich Ataxia - Comparative Analysis, H2 2016 11
Number of Products under Development by Companies, H2 2016 13
Number of Products under Investigation by Universities/Institutes, H2 2016 14
Comparative Analysis by Late Stage Development, H2 2016 15
Comparative Analysis by Clinical Stage Development, H2 2016 16
Comparative Analysis by Early Stage Development, H2 2016 17
Products under Development by Companies, H2 2016 18
Products under Investigation by Universities/Institutes, H2 2016 19
Friedreich Ataxia - Pipeline by Adverum Biotechnologies, Inc., H2 2016 20
Friedreich Ataxia - Pipeline by Almirall, S.A., H2 2016 21
Friedreich Ataxia - Pipeline by BioMarin Pharmaceutical Inc., H2 2016 22
Friedreich Ataxia - Pipeline by Biovista Inc., H2 2016 23
Friedreich Ataxia - Pipeline by Catabasis Pharmaceuticals, Inc., H2 2016 24
Friedreich Ataxia - Pipeline by Edison Pharmaceuticals, Inc., H2 2016 25
Friedreich Ataxia - Pipeline by Horizon Pharma Plc, H2 2016 26
Friedreich Ataxia - Pipeline by Ixchel Pharma, LLC, H2 2016 27
Friedreich Ataxia - Pipeline by ProQR Therapeutics N.V., H2 2016 28
Friedreich Ataxia - Pipeline by RaNA Therapeutics, Inc., H2 2016 29
Friedreich Ataxia - Pipeline by Reata Pharmaceuticals, Inc., H2 2016 30
Friedreich Ataxia - Pipeline by Retrotope, Inc, H2 2016 31
Friedreich Ataxia - Pipeline by Shire Plc, H2 2016 32
Friedreich Ataxia - Pipeline by STATegics, Inc., H2 2016 33
Friedreich Ataxia - Pipeline by Voyager Therapeutics, Inc., H2 2016 34
Assessment by Monotherapy Products, H2 2016 35
Number of Products by Stage and Target, H2 2016 37
Number of Products by Stage and Mechanism of Action, H2 2016 39
Number of Products by Stage and Route of Administration, H2 2016 41
Number of Products by Stage and Molecule Type, H2 2016 43
Friedreich Ataxia - Dormant Projects, H2 2016 82
Friedreich Ataxia - Discontinued Products, H2 2016 83