Friedreich Ataxia - Pipeline Review, H2 2014
Summary
Global Markets Direct’s, ‘Friedreich Ataxia - Pipeline Review, H2 2014’, provides an overview of the Friedreich Ataxia’s 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 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, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources, put together by Global Markets Direct’s 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 5
List of Figures 6
Introduction 7
Global Markets Direct Report Coverage 7
Friedreich Ataxia Overview 8
Therapeutics Development 9
Pipeline Products for Friedreich Ataxia - Overview 9
Pipeline Products for Friedreich Ataxia - Comparative Analysis 10
Friedreich Ataxia - Therapeutics under Development by Companies 11
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
BioMarin Pharmaceutical Inc. 20
Biovista Inc. 21
Edison Pharmaceuticals, Inc. 22
Horizon Pharma plc 23
MI.TO. Technology S.r.L. 24
Reata Pharmaceuticals, Inc. 25
Shire Plc 26
STATegics, Inc. 27
Varinel, Inc. 28
Friedreich Ataxia - Therapeutics Assessment 29
Assessment by Monotherapy Products 29
Assessment by Target 30
Assessment by Mechanism of Action 32
Assessment by Route of Administration 34
Assessment by Molecule Type 36
Drug Profiles 38
BB-FA - Drug Profile 38
Product Description 38
Mechanism of Action 38
R&D Progress 38
BVA-202 - Drug Profile 39
Product Description 39
Mechanism of Action 39
R&D Progress 39
BVA-203 - Drug Profile 40
Product Description 40
Mechanism of Action 40
R&D Progress 40
Cell Therapy for Friedreich’s Ataxia - Drug Profile 41
Product Description 41
Mechanism of Action 41
R&D Progress 41
diazoxide - Drug Profile 42
Product Description 42
Mechanism of Action 42
R&D Progress 42
Gene Therapy for Friedreich's ataxia - Drug Profile 43
Product Description 43
Mechanism of Action 43
R&D Progress 43
Gene Therapy for Friedreich’s Ataxia - Drug Profile 44
Product Description 44
Mechanism of Action 44
R&D Progress 44
Gene Therapy to Activate Frataxin for Friedreich’s Ataxia - Drug Profile 45
Product Description 45
Mechanism of Action 45
R&D Progress 45
Gene Therapy to Activate Frataxin for Friedrich's Ataxia - Drug Profile 46
Product Description 46
Mechanism of Action 46
R&D Progress 46
interferon gamma-1b (recombinant) - Drug Profile 47
Product Description 47
Mechanism of Action 47
R&D Progress 47
Oligonucleotide to Activate Frataxin for Friedreich's Ataxia - Drug Profile 49
Product Description 49
Mechanism of Action 49
R&D Progress 49
RG-2833 - Drug Profile 50
Product Description 50
Mechanism of Action 50
R&D Progress 50
RG-3250 - Drug Profile 52
Product Description 52
Mechanism of Action 52
R&D Progress 52
RTA-408 - Drug Profile 53
Product Description 53
Mechanism of Action 53
R&D Progress 53
SHP-622 - Drug Profile 55
Product Description 55
Mechanism of Action 55
R&D Progress 55
STSE-15 - Drug Profile 56
Product Description 56
Mechanism of Action 56
R&D Progress 56
VAR-10100 - Drug Profile 57
Product Description 57
Mechanism of Action 57
R&D Progress 57
VAR-10300 - Drug Profile 58
Product Description 58
Mechanism of Action 58
R&D Progress 58
vatiquinone - Drug Profile 60
Product Description 60
Mechanism of Action 60
R&D Progress 60
VYFXNO-1 - Drug Profile 62
Product Description 62
Mechanism of Action 62
R&D Progress 62
Friedreich Ataxia - Recent Pipeline Updates 63
Friedreich Ataxia - Dormant Projects 70
Friedreich Ataxia - Discontinued Products 71
Friedreich Ataxia - Product Development Milestones 72
Featured News & Press Releases 72
Oct 09, 2014: Horizon Pharma Announces Presentation of ACTIMMUNE Phase 2 Data in Friedreich's Ataxia 72
Oct 03, 2014: Horizon Pharma Receives Orphan-Drug Designation for ACTIMMUNE (Interferon gamma-1b) in Friedreich's Ataxia 73
Sep 30, 2014: Reata Announces the Initiation of Phase 2 Studies Examining RTA 408 for the Treatment of Friedreich's Ataxia and Mitochondrial Myopathies 73
Mar 17, 2014: FDA Awards Fast Track Status to Edison Pharmaceuticals' EPI-743 for Friedreich's Ataxia 74
Feb 04, 2014: FDA Grants Edison Pharmaceuticals' EPI-743 Orphan Status for Friedreich's Ataxia 75
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 75
Jan 07, 2013: Edison Pharma Initiates Phase IIb Clinical Trial Of EPI-743 In Friedreich Ataxia 76
Mar 15, 2012: Repligen Initiates Phase I Clinical Trial Of RG2833 In Patients With Friedreich's Ataxia 76
May 24, 2010: Repligen Receives FDA Orphan Drug Designation For RG2833 For Treatment Of Friedreich's Ataxia 78
May 13, 2010: Repligen Files Investigational New Drug Application with FDA for First Drug Targeting The Core Genetic Defect Of Friedreich’s Ataxia 78
Appendix 80
Methodology 80
Coverage 80
Secondary Research 80
Primary Research 80
Expert Panel Validation 80
Contact Us 81
Disclaimer 81
List Of Tables
List of Tables
Number of Products under Development for Friedreich Ataxia, H2 2014 9
Number of Products under Development for Friedreich Ataxia - Comparative Analysis, H2 2014 10
Number of Products under Development by Companies, H2 2014 12
Number of Products under Investigation by Universities/Institutes, H2 2014 13
Comparative Analysis by Clinical Stage Development, H2 2014 14
Comparative Analysis by Early Stage Development, H2 2014 15
Products under Development by Companies, H2 2014 16
Products under Development by Companies, H2 2014 (Contd..1) 17
Products under Investigation by Universities/Institutes, H2 2014 18
Friedreich Ataxia - Pipeline by AAVLife, H2 2014 19
Friedreich Ataxia - Pipeline by BioMarin Pharmaceutical Inc., H2 2014 20
Friedreich Ataxia - Pipeline by Biovista Inc., H2 2014 21
Friedreich Ataxia - Pipeline by Edison Pharmaceuticals, Inc., H2 2014 22
Friedreich Ataxia - Pipeline by Horizon Pharma plc, H2 2014 23
Friedreich Ataxia - Pipeline by MI.TO. Technology S.r.L., H2 2014 24
Friedreich Ataxia - Pipeline by Reata Pharmaceuticals, Inc., H2 2014 25
Friedreich Ataxia - Pipeline by Shire Plc, H2 2014 26
Friedreich Ataxia - Pipeline by STATegics, Inc., H2 2014 27
Friedreich Ataxia - Pipeline by Varinel, Inc., H2 2014 28
Assessment by Monotherapy Products, H2 2014 29
Number of Products by Stage and Target, H2 2014 31
Number of Products by Stage and Mechanism of Action, H2 2014 33
Number of Products by Stage and Route of Administration, H2 2014 35
Number of Products by Stage and Molecule Type, H2 2014 37
Friedreich Ataxia Therapeutics - Recent Pipeline Updates, H2 2014 63
Friedreich Ataxia - Dormant Projects, H2 2014 70
Friedreich Ataxia - Discontinued Products, H2 2014 71
List Of Figures
List of Figures
Number of Products under Development for Friedreich Ataxia, H2 2014 9
Number of Products under Development for Friedreich Ataxia - Comparative Analysis, H2 2014 10
Number of Products under Development by Companies, H2 2014 11
Comparative Analysis by Clinical Stage Development, H2 2014 14
Comparative Analysis by Early Stage Products, H2 2014 15
Assessment by Monotherapy Products, H2 2014 29
Number of Products by Top 10 Targets, H2 2014 30
Number of Products by Stage and Top 10 Targets, H2 2014 31
Number of Products by Top 10 Mechanism of Actions, H2 2014 32
Number of Products by Stage and Top 10 Mechanism of Actions, H2 2014 33
Number of Products by Top 10 Routes of Administration, H2 2014 34
Number of Products by Stage and Top 10 Routes of Administration, H2 2014 35
Number of Products by Top 10 Molecule Types, H2 2014 36
Number of Products by Stage and Top 10 Molecule Types, H2 2014 37
