Friedreich Ataxia – Pipeline Review, H2 2019

“Global Markets Direct’s, ‘Friedreich Ataxia – Pipeline Review, H2 2019’, 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

Companies mentioned

“Adverum Biotechnologies Inc

Agios Pharmaceuticals Inc

Amylyx Pharmaceutical Corp

BioElectron Technology Corp

Biointaxis SL

BioMarin Pharmaceutical Inc

Biovista Inc

Cardero Therapeutics Inc

Chondrial Therapeutics LLC

Exicure Inc

Fratagene Therapeutics Srl

Fulcrum Therapeutics Inc

Ixchel Pharma LLC

Jupiter Orphan Therapeutics Inc

Metro International Biotech LLC

Minoryx Therapeutics sl

Panorama Research Inc

Pfizer Inc

PTC Therapeutics Inc

Reata Pharmaceuticals Inc

Retrotope Inc

Seelos Therapeutics, Inc.

STATegics Inc

Voyager Therapeutics Inc”

Table of Contents

Table of Contents

List of Tables

List of Figures

Introduction

Global Markets Direct Report Coverage

Friedreich Ataxia Overview

Friedreich Ataxia Therapeutics Development

Pipeline Overview

Pipeline by Companies

Pipeline by Universities/Institutes

Products under Development by Companies

Products under Development by Universities/Institutes

Friedreich Ataxia Therapeutics Assessment

Assessment by Target

Assessment by Mechanism of Action

Assessment by Route of Administration

Assessment by Molecule Type

Friedreich Ataxia Companies Involved in Therapeutics Development

Adverum Biotechnologies Inc

Agios Pharmaceuticals Inc

BioElectron Technology Corp

BioMarin Pharmaceutical Inc

Biovista Inc

Horizon Pharma Plc

Ixchel Pharma LLC

Pfizer Inc

PTC Therapeutics Inc

Reata Pharmaceuticals Inc

STATegics Inc

Takeda Pharmaceutical Co Ltd

Voyager Therapeutics Inc

Friedreich Ataxia Drug Profiles

(sodium phenylbutyrate + tauroursodeoxycholic acid) Drug Profile

Product Description

Mechanism Of Action

R&D Progress

ADVM-063 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

AGIL-FA Drug Profile

Product Description

Mechanism Of Action

R&D Progress

BMN-290 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

BVA-202 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

BVA-203 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Cell Therapy for Central Nervous System and Neurodegenerative Diseases Drug Profile

Product Description

Mechanism Of Action

R&D Progress

CTI-1601 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

dextro epicatechin Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Gene Therapy to Activate Frataxin for Friedreich’s Ataxia Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Gene Therapy to Activate Frataxin for Friedreich’s Ataxia Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Gene Therapy to Activate Frataxin for Friedrich's Ataxia Drug Profile

Product Description

Mechanism Of Action

R&D Progress

interferon gamma-1b Drug Profile

Product Description

Mechanism Of Action

R&D Progress

IXC-103 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

JOT-101 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

omaveloxolone Drug Profile

Product Description

Mechanism Of Action

R&D Progress

RT-001 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

RT-002 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Small Molecules for Friedreich Ataxia Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Small Molecules to Activate Frataxin for Friedreich Ataxia Drug Profile

Product Description

Mechanism Of Action

R&D Progress

STSE-15 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

TAK-831 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

vatiquinone Drug Profile

Product Description

Mechanism Of Action

R&D Progress

VYFXN-01 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Friedreich Ataxia Dormant Projects

Friedreich Ataxia Discontinued Products

Friedreich Ataxia Product Development Milestones

Featured News & Press Releases

May 18, 2018: Voyager Therapeutics Announces Additional Data at the American Society of Gene and Cell Therapy 2018 Annual Meeting

Aug 22, 2017: Jupiter Orphan Therapeutics Receives Orphan Drug Designation for its Trans-Resveratrol Product JOTROL for Treatment of Friedreich's Ataxia

Aug 03, 2017: Chondrial Announces FDA Orphan Drug Designation for CTI-1601, a Novel Investigational Technology for the Treatment of Friedreich's Ataxia

Dec 08, 2016: Horizon Pharma Announces Top line Results from Phase 3 Study of ACTIMMUNE (interferon gamma-1b) in Friedreich's Ataxia

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

Aug 05, 2015: Key Patent Granted for AAVLife’s Gene-Therapy Program to Treat Cardiomyopathy in Friedreich’s Ataxia

Jun 05, 2015: Horizon Pharma Initiates Phase 3 Trial of ACTIMMUNE for the Treatment of People With Friedreich's Ataxia

Apr 10, 2015: Horizon Pharma Receives FDA Fast Track Designation for ACTIMMUNE in the Treatment of Friedreich's Ataxia

Feb 13, 2015: Horizon Pharma Submits Investigational New Drug Application for ACTIMMUNE in the Treatment of Friedreich's Ataxia

Oct 09, 2014: Horizon Pharma Announces Presentation of ACTIMMUNE Phase 2 Data in Friedreich's Ataxia

Oct 03, 2014: Horizon Pharma Receives Orphan-Drug Designation for ACTIMMUNE (Interferon gamma-1b) in Friedreich's Ataxia

Mar 17, 2014: FDA Awards Fast Track Status to Edison Pharmaceuticals' EPI-743 for Friedreich's Ataxia

Feb 04, 2014: FDA Grants Edison Pharmaceuticals' EPI-743 Orphan Status for Friedreich's Ataxia

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

Jan 08, 2013: STATegics Announces a New Grant from Friedreich’s Ataxia Research Alliance

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer

List of Tables

“List of Tables

Table 1: Number of Products under Development for Friedreich Ataxia, H2 2019

Table 2: Number of Products under Development by Companies, H2 2019

Table 3: Number of Products under Development by Universities/Institutes, H2 2019

Table 4: Products under Development by Companies, H2 2019

Table 5: Products under Development by Companies, H2 2019 (Contd..1), H2 2019

Table 6: Products under Development by Universities/Institutes, H2 2019

Table 7: Number of Products by Stage and Target, H2 2019

Table 8: Number of Products by Stage and Mechanism of Action, H2 2019

Table 9: Number of Products by Stage and Route of Administration, H2 2019

Table 10: Number of Products by Stage and Molecule Type, H2 2019

Table 11: Friedreich Ataxia – Pipeline by Adverum Biotechnologies Inc, H2 2019

Table 12: Friedreich Ataxia – Pipeline by Agios Pharmaceuticals Inc, H2 2019

Table 13: Friedreich Ataxia – Pipeline by Amylyx Pharmaceutical Corp, H2 2019

Table 14: Friedreich Ataxia – Pipeline by BioElectron Technology Corp, H2 2019

Table 15: Friedreich Ataxia – Pipeline by Biointaxis SL, H2 2019

Table 16: Friedreich Ataxia – Pipeline by BioMarin Pharmaceutical Inc, H2 2019

Table 17: Friedreich Ataxia – Pipeline by Biovista Inc, H2 2019

Table 18: Friedreich Ataxia – Pipeline by Cardero Therapeutics Inc, H2 2019

Table 19: Friedreich Ataxia – Pipeline by Chondrial Therapeutics LLC, H2 2019

Table 20: Friedreich Ataxia – Pipeline by Exicure Inc, H2 2019

Table 21: Friedreich Ataxia – Pipeline by Fratagene Therapeutics Srl, H2 2019

Table 22: Friedreich Ataxia – Pipeline by Fulcrum Therapeutics Inc, H2 2019

Table 23: Friedreich Ataxia – Pipeline by Ixchel Pharma LLC, H2 2019

Table 24: Friedreich Ataxia – Pipeline by Jupiter Orphan Therapeutics Inc, H2 2019

Table 25: Friedreich Ataxia – Pipeline by Metro International Biotech LLC, H2 2019

Table 26: Friedreich Ataxia – Pipeline by Minoryx Therapeutics sl, H2 2019

Table 27: Friedreich Ataxia – Pipeline by Panorama Research Inc, H2 2019

Table 28: Friedreich Ataxia – Pipeline by Pfizer Inc, H2 2019

Table 29: Friedreich Ataxia – Pipeline by PTC Therapeutics Inc, H2 2019

Table 30: Friedreich Ataxia – Pipeline by Reata Pharmaceuticals Inc, H2 2019

Table 31: Friedreich Ataxia – Pipeline by Retrotope Inc, H2 2019

Table 32: Friedreich Ataxia – Pipeline by Seelos Therapeutics, Inc., H2 2019

Table 33: Friedreich Ataxia – Pipeline by STATegics Inc, H2 2019

Table 34: Friedreich Ataxia – Pipeline by Voyager Therapeutics Inc, H2 2019

Table 35: Friedreich Ataxia – Dormant Projects, H2 2019

Table 36: Friedreich Ataxia – Discontinued Products, H2 2019”

List of Figures

“List of Figures

Figure 1: Number of Products under Development for Friedreich Ataxia, H2 2019

Figure 2: Number of Products under Development by Companies, H2 2019

Figure 3: Number of Products under Development by Universities/Institutes, H2 2019

Figure 4: Number of Products by Targets, H2 2019

Figure 5: Number of Products by Stage and Targets, H2 2019

Figure 6: Number of Products by Top 10 Mechanism of Actions, H2 2019

Figure 7: Number of Products by Stage and Top 10 Mechanism of Actions, H2 2019

Figure 8: Number of Products by Routes of Administration, H2 2019

Figure 9: Number of Products by Stage and Routes of Administration, H2 2019

Figure 10: Number of Products by Molecule Types, H2 2019

Figure 11: Number of Products by Stage and Molecule Types, H2 2019”

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