Friedreich Ataxia - Pipeline Review, H1 2016

Published: Apr, 2016 | Pages: 91 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

Friedreich Ataxia - Pipeline Review, H1 2016

Summary

Global Markets Direct's, ‘Friedreich Ataxia - Pipeline Review, H1 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 Content
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 13
Friedreich Ataxia - Pipeline Products Glance 14
Late Stage Products 14
Clinical Stage Products 15
Early Stage Products 16
Friedreich Ataxia - Products under Development by Companies 17
Friedreich Ataxia - Products under Investigation by Universities/Institutes 18
Friedreich Ataxia - Companies Involved in Therapeutics Development 19
Annapurna Therapeutics 19
Bioblast Pharma Ltd. 20
BioMarin Pharmaceutical Inc. 21
Biovista Inc. 22
Catabasis Pharmaceuticals, Inc. 23
Edison Pharmaceuticals, Inc. 24
Horizon Pharma Plc 25
MI.TO. Technology S.r.L. 26
Reata Pharmaceuticals, Inc. 27
Retrotope, Inc 28
Shire Plc 29
STATegics, Inc. 30
Voyager Therapeutics, Inc. 31
Friedreich Ataxia - Therapeutics Assessment 32
Assessment by Monotherapy Products 32
Assessment by Target 33
Assessment by Mechanism of Action 35
Assessment by Route of Administration 37
Assessment by Molecule Type 39
Drug Profiles 41
AGIL-FA - Drug Profile 41
Product Description 41
Mechanism of Action 41
R&D Progress 41
ANN-003 - Drug Profile 42
Product Description 42
Mechanism of Action 42
R&D Progress 42
BB-FA - Drug Profile 44
Product Description 44
Mechanism of Action 44
R&D Progress 44
BVA-202 - Drug Profile 45
Product Description 45
Mechanism of Action 45
R&D Progress 45
BVA-203 - Drug Profile 46
Product Description 46
Mechanism of Action 46
R&D Progress 46
CAT-4001 - Drug Profile 47
Product Description 47
Mechanism of Action 47
R&D Progress 47
diazoxide - Drug Profile 48
Product Description 48
Mechanism of Action 48
R&D Progress 48
Gene Therapy to Activate Frataxin for Friedreich's Ataxia - Drug Profile 49
Product Description 49
Mechanism of Action 49
R&D Progress 49
Gene Therapy to Activate Frataxin for Friedreich's Ataxia - Drug Profile 50
Product Description 50
Mechanism of Action 50
R&D Progress 50
Gene Therapy to Activate Frataxin for Friedrich's Ataxia - Drug Profile 51
Product Description 51
Mechanism of Action 51
R&D Progress 51
interferon gamma-1b - Drug Profile 52
Product Description 52
Mechanism of Action 52
R&D Progress 52
IXC-101 - Drug Profile 55
Product Description 55
Mechanism of Action 55
R&D Progress 55
IXC-103 - Drug Profile 56
Product Description 56
Mechanism of Action 56
R&D Progress 56
IXC-105 - Drug Profile 57
Product Description 57
Mechanism of Action 57
R&D Progress 57
Oligonucleotide to Activate Frataxin for Friedreich's Ataxia - Drug Profile 58
Product Description 58
Mechanism of Action 58
R&D Progress 58
omaveloxolone - Drug Profile 59
Product Description 59
Mechanism of Action 59
R&D Progress 59
RG-3250 - Drug Profile 62
Product Description 62
Mechanism of Action 62
R&D Progress 62
RT-001 - Drug Profile 63
Product Description 63
Mechanism of Action 63
R&D Progress 63
SHP-622 - Drug Profile 64
Product Description 64
Mechanism of Action 64
R&D Progress 64
STSE-15 - Drug Profile 66
Product Description 66
Mechanism of Action 66
R&D Progress 66
vatiquinone - Drug Profile 68
Product Description 68
Mechanism of Action 68
R&D Progress 68
VYFXN-01 - Drug Profile 71
Product Description 71
Mechanism of Action 71
R&D Progress 71
Friedreich Ataxia - Recent Pipeline Updates 72
Friedreich Ataxia - Dormant Projects 82
Friedreich Ataxia - Discontinued Products 83
Friedreich Ataxia - Product Development Milestones 84
Featured News & Press Releases 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 84
Jun 18, 2015: BioBlast Pharma to Present Preclinical Proofs of Concept in Friedreich's Ataxia at Two Leading Scientific Meetings in June 84
Jun 05, 2015: Horizon Pharma Initiates Phase 3 Trial of ACTIMMUNE for the Treatment of People With Friedreich's Ataxia 85
Apr 10, 2015: Horizon Pharma Receives FDA Fast Track Designation for ACTIMMUNE in the Treatment of Friedreich's Ataxia 86
Mar 26, 2015: BioBlast Pharma Announces Positive Preclinical Proof-of-Concept Results of Its Novel Mitochondrial Protein Replacement Platform in Friedreich's Ataxia 86
Mar 24, 2015: Catabasis Pharmaceuticals to Present at International Ataxia Research Conference 87
Feb 13, 2015: Horizon Pharma Submits Investigational New Drug Application for ACTIMMUNE in the Treatment of Friedreich's Ataxia 87
Dec 17, 2014: BioBlast Receives Notice of Patent Issuance From USPTO for Its Novel Fusion Protein-Based Platform for the Treatment of Friedrich's Ataxia 88
Oct 09, 2014: Horizon Pharma Announces Presentation of ACTIMMUNE Phase 2 Data in Friedreich's Ataxia 88
Oct 03, 2014: Horizon Pharma Receives Orphan-Drug Designation for ACTIMMUNE (Interferon gamma-1b) in Friedreich's Ataxia 89
Appendix 90
Methodology 90
Coverage 90
Secondary Research 90
Primary Research 90
Expert Panel Validation 90
Contact Us 90
Disclaimer 91

List of Tables

Number of Products under Development for Friedreich Ataxia, H1 2016 10
Number of Products under Development for Friedreich Ataxia - Comparative Analysis, H1 2016 11
Number of Products under Development by Companies, H1 2016 12
Number of Products under Investigation by Universities/Institutes, H1 2016 13
Comparative Analysis by Late Stage Development, H1 2016 14
Comparative Analysis by Clinical Stage Development, H1 2016 15
Comparative Analysis by Early Stage Development, H1 2016 16
Products under Development by Companies, H1 2016 17
Products under Investigation by Universities/Institutes, H1 2016 18
Friedreich Ataxia - Pipeline by Annapurna Therapeutics, H1 2016 19
Friedreich Ataxia - Pipeline by Bioblast Pharma Ltd., H1 2016 20
Friedreich Ataxia - Pipeline by BioMarin Pharmaceutical Inc., H1 2016 21
Friedreich Ataxia - Pipeline by Biovista Inc., H1 2016 22
Friedreich Ataxia - Pipeline by Catabasis Pharmaceuticals, Inc., H1 2016 23
Friedreich Ataxia - Pipeline by Edison Pharmaceuticals, Inc., H1 2016 24
Friedreich Ataxia - Pipeline by Horizon Pharma Plc, H1 2016 25
Friedreich Ataxia - Pipeline by MI.TO. Technology S.r.L., H1 2016 26
Friedreich Ataxia - Pipeline by Reata Pharmaceuticals, Inc., H1 2016 27
Friedreich Ataxia - Pipeline by Retrotope, Inc, H1 2016 28
Friedreich Ataxia - Pipeline by Shire Plc, H1 2016 29
Friedreich Ataxia - Pipeline by STATegics, Inc., H1 2016 30
Friedreich Ataxia - Pipeline by Voyager Therapeutics, Inc., H1 2016 31
Assessment by Monotherapy Products, H1 2016 32
Number of Products by Stage and Target, H1 2016 34
Number of Products by Stage and Mechanism of Action, H1 2016 36
Number of Products by Stage and Route of Administration, H1 2016 38
Number of Products by Stage and Molecule Type, H1 2016 40
Friedreich Ataxia Therapeutics - Recent Pipeline Updates, H1 2016 72
Friedreich Ataxia - Dormant Projects, H1 2016 82
Friedreich Ataxia - Discontinued Products, H1 2016 83