Spinocerebellar Ataxia (SCA) – Pipeline Review, H2 2017

Global Markets Direct’s, ‘Spinocerebellar Ataxia (SCA) – Pipeline Review, H2 2017’, provides an overview of the Spinocerebellar Ataxia (SCA) pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Spinocerebellar Ataxia (SCA), 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 Spinocerebellar Ataxia (SCA) 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 Spinocerebellar Ataxia (SCA)

The report reviews pipeline therapeutics for Spinocerebellar Ataxia (SCA) 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 Spinocerebellar Ataxia (SCA) therapeutics and enlists all their major and minor projects

The report assesses Spinocerebellar Ataxia (SCA) 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 Spinocerebellar Ataxia (SCA)

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 Spinocerebellar Ataxia (SCA)

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 Spinocerebellar Ataxia (SCA) 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

Bio Blast Pharma Ltd

Biohaven Pharmaceutical Holding Company Ltd

Cadent Therapeutics

Ionis Pharmaceuticals Inc

Shionogi & Co Ltd

Shire Plc

Spark Therapeutics Inc

Vybion Inc

Table of Contents

Table of Contents

Table of Contents 2

List of Tables 5

List of Figures 5

Introduction 6

Global Markets Direct Report Coverage 6

Spinocerebellar Ataxia (SCA) - Overview 7

Spinocerebellar Ataxia (SCA) - Therapeutics Development 8

Pipeline Overview 8

Pipeline by Companies 9

Pipeline by Universities/Institutes 10

Products under Development by Companies 11

Products under Development by Universities/Institutes 12

Spinocerebellar Ataxia (SCA) - Therapeutics Assessment 13

Assessment by Target 13

Assessment by Mechanism of Action 15

Assessment by Route of Administration 17

Assessment by Molecule Type 19

Spinocerebellar Ataxia (SCA) - Companies Involved in Therapeutics Development 21

Bio Blast Pharma Ltd 21

Biohaven Pharmaceutical Holding Company Ltd 21

Cadent Therapeutics 21

Ionis Pharmaceuticals Inc 22

Shionogi & Co Ltd 22

Shire Plc 23

Spark Therapeutics Inc 23

Vybion Inc 24

Spinocerebellar Ataxia (SCA) - Drug Profiles 25

2AU-2 - Drug Profile 25

Product Description 25

Mechanism Of Action 25

R&D Progress 25

Antisense Oligonucleotides to Inhibit ATXN3 for Spinocerebellar Ataxia Type 3 - Drug Profile 26

Product Description 26

Mechanism Of Action 26

R&D Progress 26

Antisense RNAi Oligonucleotide to Inhibit Ataxin-1 for Spinocerebellar Ataxia - Drug Profile 27

Product Description 27

Mechanism Of Action 27

R&D Progress 27

Antisense RNAi Oligonucleotide to Inhibit Ataxin-7 for Spinocerebellar Ataxia Type 7 - Drug Profile 28

Product Description 28

Mechanism Of Action 28

R&D Progress 28

ASO-7 - Drug Profile 29

Product Description 29

Mechanism Of Action 29

R&D Progress 29

BHV-4157 - Drug Profile 30

Product Description 30

Mechanism Of Action 30

R&D Progress 30

CAD-1883 - Drug Profile 33

Product Description 33

Mechanism Of Action 33

R&D Progress 33

epoetin alfa - Drug Profile 34

Product Description 34

Mechanism Of Action 34

R&D Progress 34

immune globulin (human) - Drug Profile 35

Product Description 35

Mechanism Of Action 35

R&D Progress 35

INT-41 - Drug Profile 40

Product Description 40

Mechanism Of Action 40

R&D Progress 40

riluzole - Drug Profile 42

Product Description 42

Mechanism Of Action 42

R&D Progress 42

RNAi Gene Therapy to Inhibit Ataxin-3 for Machado-Joseph Disease - Drug Profile 46

Product Description 46

Mechanism Of Action 46

R&D Progress 46

rovatirelin - Drug Profile 47

Product Description 47

Mechanism Of Action 47

R&D Progress 47

Stem Cell Therapy for Diabetes Mellitus, Acute Liver Failure, Osteoarthritis and Neurodegenerative Diseases - Drug Profile 48

Product Description 48

Mechanism Of Action 48

R&D Progress 48

trehalose - Drug Profile 49

Product Description 49

Mechanism Of Action 49

R&D Progress 49

Spinocerebellar Ataxia (SCA) - Dormant Projects 54

Spinocerebellar Ataxia (SCA) - Product Development Milestones 55

Featured News & Press Releases 55

Jun 05, 2017: Bioblast Pharma to Seek Strategic Alternatives Including Partnership and Merger Opportunities 55

Jan 26, 2017: Bioblast Pharma to Host Key Opinion Leader Lunch Focused on Orphan Neurological Diseases on February 2 56

Jan 18, 2017: Bioblast Announces Phase 2a Results of Trehalose in Patients with Spinocerebellar Ataxia Type 3 (SCA3) 56

Jun 01, 2016: Scripps Florida Scientists Create Compound that Erases Disease-Causing RNA Defects 58

May 27, 2016: Announcement of Additional Clinical Trial of “KPS-0373 " for the Treatment of Spinocerebellar Ataxia 59

Mar 03, 2016: Portage Biohaven orphan drug designation request granted for the treatment of spinocerebellar ataxia 59

Sep 14, 2015: BioBlast Granted Patent From USPTO for Cabaletta for the Treatment of Spinocerebellar Ataxia 60

Jun 29, 2015: BioBlast Pharma Receives Orphan Drug Designation From the European Commission for Cabaletta for the Treatment of Spinocerebellar Ataxia 60

Mar 30, 2015: BioBlast Pharma Announces Positive in vivo Preclinical Proof-of-Concept Results of Its Cabaletta Platform in Spinocerebellar Ataxia Type 3 61

Nov 19, 2014: Bio Blast Pharma Receives Orphan Drug Designation From U.S. FDA for Cabaletta for the Treatment of Spinocerebellar Ataxia Type 3 61

Appendix 63

Methodology 63

Coverage 63

Secondary Research 63

Primary Research 63

Expert Panel Validation 63

Contact Us 63

Disclaimer 64

List of Tables

List of Tables

Number of Products under Development for Spinocerebellar Ataxia (SCA), H2 2017 8

Number of Products under Development by Companies, H2 2017 9

Number of Products under Development by Universities/Institutes, H2 2017 10

Products under Development by Companies, H2 2017 11

Products under Development by Universities/Institutes, H2 2017 12

Number of Products by Stage and Target, H2 2017 14

Number of Products by Stage and Mechanism of Action, H2 2017 16

Number of Products by Stage and Route of Administration, H2 2017 18

Number of Products by Stage and Molecule Type, H2 2017 20

Spinocerebellar Ataxia (SCA) – Pipeline by Bio Blast Pharma Ltd, H2 2017 21

Spinocerebellar Ataxia (SCA) – Pipeline by Biohaven Pharmaceutical Holding Company Ltd, H2 2017 21

Spinocerebellar Ataxia (SCA) – Pipeline by Cadent Therapeutics, H2 2017 22

Spinocerebellar Ataxia (SCA) – Pipeline by Ionis Pharmaceuticals Inc, H2 2017 22

Spinocerebellar Ataxia (SCA) – Pipeline by Shionogi & Co Ltd, H2 2017 23

Spinocerebellar Ataxia (SCA) – Pipeline by Shire Plc, H2 2017 23

Spinocerebellar Ataxia (SCA) – Pipeline by Spark Therapeutics Inc, H2 2017 24

Spinocerebellar Ataxia (SCA) – Pipeline by Vybion Inc, H2 2017 24

Spinocerebellar Ataxia (SCA) – Dormant Projects, H2 2017 54

List of Figures

List of Figures

Number of Products under Development for Spinocerebellar Ataxia (SCA), H2 2017 8

Number of Products under Development by Companies, H2 2017 9

Number of Products under Development by Universities/Institutes, H2 2017 10

Number of Products by Targets, H2 2017 13

Number of Products by Stage and Targets, H2 2017 13

Number of Products by Mechanism of Actions, H2 2017 15

Number of Products by Stage and Mechanism of Actions, H2 2017 15

Number of Products by Routes of Administration, H2 2017 17

Number of Products by Stage and Routes of Administration, H2 2017 17

Number of Products by Molecule Types, H2 2017 19

Number of Products by Stage and Molecule Types, H2 2017 19

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