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
List of Figures
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 Comparative Analysis by Clinical Stage Development, H1 2016 15 Comparative Analysis by Early Stage Products, H1 2016 16 Assessment by Monotherapy Products, H1 2016 32 Number of Products by Targets, H1 2016 33 Number of Products by Stage and Targets, H1 2016 33 Number of Products by Top 10 Mechanism of Actions, H1 2016 35 Number of Products by Stage and Top 10 Mechanism of Actions, H1 2016 35 Number of Products by Routes of Administration, H1 2016 37 Number of Products by Stage and Routes of Administration, H1 2016 37 Number of Products by Molecule Types, H1 2016 39 Number of Products by Stage and Molecule Types, H1 2016 39
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