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Friedreich Ataxia-Pipeline Review, H1 2015

Friedreich Ataxia-Pipeline Review, H1 2015


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Executive Summary

Friedreich Ataxia-Pipeline Review, H1 2015

Summary

Global Markets Direct's, 'Friedreich Ataxia-Pipeline Review, H1 2015', 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 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

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AAVLife

Bioblast Pharma Ltd.

BioMarin Pharmaceutical Inc.

Biovista Inc.

Catabasis Pharmaceuticals, Inc.

Edison Pharmaceuticals, Inc.

Horizon Pharma plc

MI.TO. Technology S.r.L.

Reata Pharmaceuticals, Inc.

Shire Plc

STATegics, Inc.

Varinel, Inc.

Friedreich Ataxia Therapeutic Products under Development, Key Players in Friedreich Ataxia Therapeutics, Friedreich Ataxia Pipeline Overview, Friedreich Ataxia Pipeline, Friedreich Ataxia Pipeline Assessment


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