Friedreich Ataxia - Pipeline Review, H1 2015
Summary
Global Markets Directs, Friedreich Ataxia - Pipeline Review, H1 2015, provides an overview of the Friedreich Ataxias therapeutic pipeline.
This report provides comprehensive information on the therapeutic development for Friedreich Ataxia, complete with comparative analysis at various stages, therapeutics assessment by drug target, mechanism of action (MoA), route of administration (RoA) and molecule type, along with latest updates, and featured news and press releases. It also reviews key players involved in the therapeutic development for Friedreich Ataxia and special features on late-stage and discontinued projects.
Global Markets Directs 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 Directs proprietary databases, Company/University websites, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources, put together by Global Markets Directs team. Drug profiles/records featured in the report undergoes periodic updation following a stringent set of processes that ensures that all the profiles are updated with the latest set of information. Additionally, processes including live news & deals tracking, browser based alert-box and clinical trials registries tracking ensure that the most recent developments are captured on a real time basis.
The report enhances decision making capabilities and help to create effective counter strategies to gain competitive advantage. It strengthens R&D pipelines by identifying new targets and MOAs to produce first-in-class and best-in-class products.
Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data for the indicated disease.
Scope
- The report provides a snapshot of the global therapeutic landscape of Friedreich Ataxia
- The report reviews key pipeline products under drug profile section which includes, product description, MoA and R&D brief, licensing and collaboration details & other developmental activities
- The report reviews key players involved in the therapeutics development for Friedreich Ataxia and enlists all their major and minor projects
- The report summarizes all the dormant and discontinued pipeline projects
- A review of the Friedreich Ataxia products under development by companies and universities/research institutes based on information derived from company and industry-specific sources
- Pipeline products coverage based on various stages of development ranging from pre-registration till discovery and undisclosed stages
- A detailed assessment of monotherapy and combination therapy pipeline projects
- Coverage of the Friedreich Ataxia pipeline on the basis of target, MoA, route of administration and molecule type
- Latest news and deals relating related to pipeline products
Reasons to buy
- Provides strategically significant competitor information, analysis, and insights to formulate effective R&D development strategies
- Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage
- Develop strategic initiatives by understanding the focus areas of leading companies
- Identify and understand important and diverse types of therapeutics under development for Friedreich Ataxia
- Plan mergers and acquisitions effectively by identifying key players of the most promising pipeline
- 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 13
Friedreich Ataxia - Pipeline Products Glance 14
Clinical Stage Products 14
Early Stage Products 15
Friedreich Ataxia - Products under Development by Companies 16
Friedreich Ataxia - Products under Investigation by Universities/Institutes 18
Friedreich Ataxia - Companies Involved in Therapeutics Development 19
AAVLife 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
Shire Plc 28
STATegics, Inc. 29
Varinel, Inc. 30
Friedreich Ataxia - Therapeutics Assessment 31
Assessment by Monotherapy Products 31
Assessment by Target 32
Assessment by Mechanism of Action 34
Assessment by Route of Administration 36
Assessment by Molecule Type 38
Drug Profiles 40
BB-FA - Drug Profile 40
Product Description 40
Mechanism of Action 40
R&D Progress 40
BVA-202 - Drug Profile 41
Product Description 41
Mechanism of Action 41
R&D Progress 41
BVA-203 - Drug Profile 42
Product Description 42
Mechanism of Action 42
R&D Progress 42
CAT-4001 - Drug Profile 43
Product Description 43
Mechanism of Action 43
R&D Progress 43
Cell Therapy for Friedreich’s Ataxia - Drug Profile 44
Product Description 44
Mechanism of Action 44
R&D Progress 44
diazoxide - Drug Profile 45
Product Description 45
Mechanism of Action 45
R&D Progress 45
Gene Therapy for Friedreich's ataxia - Drug Profile 46
Product Description 46
Mechanism of Action 46
R&D Progress 46
Gene Therapy for Friedreich’s Ataxia - Drug Profile 47
Product Description 47
Mechanism of Action 47
R&D Progress 47
Gene Therapy to Activate Frataxin for Friedreich’s Ataxia - Drug Profile 48
Product Description 48
Mechanism of Action 48
R&D Progress 48
Gene Therapy to Activate Frataxin for Friedrich's Ataxia - Drug Profile 49
Product Description 49
Mechanism of Action 49
R&D Progress 49
interferon gamma-1b (recombinant) - Drug Profile 50
Product Description 50
Mechanism of Action 50
R&D Progress 50
Oligonucleotide to Activate Frataxin for Friedreich's Ataxia - Drug Profile 52
Product Description 52
Mechanism of Action 52
R&D Progress 52
RG-2833 - Drug Profile 53
Product Description 53
Mechanism of Action 53
R&D Progress 53
RG-3250 - Drug Profile 55
Product Description 55
Mechanism of Action 55
R&D Progress 55
RTA-408 - Drug Profile 56
Product Description 56
Mechanism of Action 56
R&D Progress 56
SHP-622 - Drug Profile 59
Product Description 59
Mechanism of Action 59
R&D Progress 59
STSE-15 - Drug Profile 60
Product Description 60
Mechanism of Action 60
R&D Progress 60
VAR-10100 - Drug Profile 61
Product Description 61
Mechanism of Action 61
R&D Progress 61
VAR-10300 - Drug Profile 62
Product Description 62
Mechanism of Action 62
R&D Progress 62
vatiquinone - Drug Profile 64
Product Description 64
Mechanism of Action 64
R&D Progress 64
VYFXNO-1 - Drug Profile 67
Product Description 67
Mechanism of Action 67
R&D Progress 67
Friedreich Ataxia - Recent Pipeline Updates 68
Friedreich Ataxia - Dormant Projects 76
Friedreich Ataxia - Discontinued Products 77
Friedreich Ataxia - Product Development Milestones 78
Featured News & Press Releases 78
Apr 10, 2015: Horizon Pharma Receives FDA Fast Track Designation for ACTIMMUNE in the Treatment of Friedreich's Ataxia 78
Mar 26, 2015: BioBlast Pharma Announces Positive Preclinical Proof-of-Concept Results of Its Novel Mitochondrial Protein Replacement Platform in Friedreich's Ataxia 78
Mar 24, 2015: Catabasis Pharmaceuticals to Present at International Ataxia Research Conference 79
Feb 13, 2015: Horizon Pharma Submits Investigational New Drug Application for ACTIMMUNE in the Treatment of Friedreich's Ataxia 79
Jan 29, 2015: Reata Enrolls First Patient in the MOXIe Study, a Phase 2/3 Study Examining RTA 408 in Friedreich's Ataxia Patients 80
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 80
Oct 09, 2014: Horizon Pharma Announces Presentation of ACTIMMUNE Phase 2 Data in Friedreich's Ataxia 80
Oct 03, 2014: Horizon Pharma Receives Orphan-Drug Designation for ACTIMMUNE (Interferon gamma-1b) in Friedreich's Ataxia 81
Sep 30, 2014: Reata Announces the Initiation of Phase 2 Studies Examining RTA 408 for the Treatment of Friedreich's Ataxia and Mitochondrial Myopathies 82
Mar 17, 2014: FDA Awards Fast Track Status to Edison Pharmaceuticals' EPI-743 for Friedreich's Ataxia 82
Appendix 84
Methodology 84
Coverage 84
Secondary Research 84
Primary Research 84
Expert Panel Validation 84
Contact Us 84
Disclaimer 85
List Of Tables
Number of Products under Development for Friedreich Ataxia, H1 2015 10
Number of Products under Development for Friedreich Ataxia - Comparative Analysis, H1 2015 11
Number of Products under Development by Companies, H1 2015 12
Number of Products under Investigation by Universities/Institutes, H1 2015 13
Comparative Analysis by Clinical Stage Development, H1 2015 14
Comparative Analysis by Early Stage Development, H1 2015 15
Products under Development by Companies, H1 2015 16
Products under Development by Companies, H1 2015 (Contd..1) 17
Products under Investigation by Universities/Institutes, H1 2015 18
Friedreich Ataxia - Pipeline by AAVLife, H1 2015 19
Friedreich Ataxia - Pipeline by Bioblast Pharma Ltd., H1 2015 20
Friedreich Ataxia - Pipeline by BioMarin Pharmaceutical Inc., H1 2015 21
Friedreich Ataxia - Pipeline by Biovista Inc., H1 2015 22
Friedreich Ataxia - Pipeline by Catabasis Pharmaceuticals, Inc., H1 2015 23
Friedreich Ataxia - Pipeline by Edison Pharmaceuticals, Inc., H1 2015 24
Friedreich Ataxia - Pipeline by Horizon Pharma plc, H1 2015 25
Friedreich Ataxia - Pipeline by MI.TO. Technology S.r.L., H1 2015 26
Friedreich Ataxia - Pipeline by Reata Pharmaceuticals, Inc., H1 2015 27
Friedreich Ataxia - Pipeline by Shire Plc, H1 2015 28
Friedreich Ataxia - Pipeline by STATegics, Inc., H1 2015 29
Friedreich Ataxia - Pipeline by Varinel, Inc., H1 2015 30
Assessment by Monotherapy Products, H1 2015 31
Number of Products by Stage and Target, H1 2015 33
Number of Products by Stage and Mechanism of Action, H1 2015 35
Number of Products by Stage and Route of Administration, H1 2015 37
Number of Products by Stage and Molecule Type, H1 2015 39
Friedreich Ataxia Therapeutics - Recent Pipeline Updates, H1 2015 68
Friedreich Ataxia - Dormant Projects, H1 2015 76
Friedreich Ataxia - Discontinued Products, H1 2015 77
List Of Figures
Number of Products under Development for Friedreich Ataxia, H1 2015 10
Number of Products under Development for Friedreich Ataxia - Comparative Analysis, H1 2015 11
Number of Products under Development by Companies, H1 2015 12
Comparative Analysis by Clinical Stage Development, H1 2015 14
Comparative Analysis by Early Stage Products, H1 2015 15
Assessment by Monotherapy Products, H1 2015 31
Number of Products by Top 10 Targets, H1 2015 32
Number of Products by Stage and Top 10 Targets, H1 2015 33
Number of Products by Top 10 Mechanism of Actions, H1 2015 34
Number of Products by Stage and Top 10 Mechanism of Actions, H1 2015 35
Number of Products by Top 10 Routes of Administration, H1 2015 36
Number of Products by Stage and Top 10 Routes of Administration, H1 2015 37
Number of Products by Top 10 Molecule Types, H1 2015 38
Number of Products by Stage and Top 10 Molecule Types, H1 2015 39
