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Friedreich Ataxia Treatment Pipeline Review H2 2016

Wednesday, October 19, 2016 23:47
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‘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.

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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 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.


- 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

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Key points in 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 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

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