Spinocerebellar Ataxia (SCA) – Pipeline Review, H2 2016

Global Markets Direct’s, ‘Spinocerebellar Ataxia (SCA) – Pipeline Review, H2 2016’, 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 Limited

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

Therapeutics Development 8

Pipeline Products for Spinocerebellar Ataxia (SCA) - Overview 8

Pipeline Products for Spinocerebellar Ataxia (SCA) - Comparative Analysis 9

Spinocerebellar Ataxia (SCA) - Therapeutics under Development by Companies 10

Spinocerebellar Ataxia (SCA) - Therapeutics under Investigation by Universities/Institutes 11

Spinocerebellar Ataxia (SCA) - Pipeline Products Glance 12

Late Stage Products 12

Clinical Stage Products 13

Early Stage Products 14

Spinocerebellar Ataxia (SCA) - Products under Development by Companies 15

Spinocerebellar Ataxia (SCA) - Products under Investigation by Universities/Institutes 16

Spinocerebellar Ataxia (SCA) - Companies Involved in Therapeutics Development 17

Bio Blast Pharma Ltd 17

Biohaven Pharmaceutical Holding Company Limited 18

Ionis Pharmaceuticals Inc 19

Shionogi & Co Ltd 20

Shire Plc 21

Spark Therapeutics Inc 22

Vybion Inc 23

Spinocerebellar Ataxia (SCA) - Therapeutics Assessment 24

Assessment by Monotherapy Products 24

Assessment by Target 25

Assessment by Mechanism of Action 27

Assessment by Route of Administration 29

Assessment by Molecule Type 31

Drug Profiles 33

2AU-2 - Drug Profile 33

Product Description 33

Mechanism Of Action 33

R&D Progress 33

Antisense Oligonucleotides to Inhibit ATXN2 for Spinocerebellar Ataxia - Drug Profile 34

Product Description 34

Mechanism Of Action 34

R&D Progress 34

Antisense RNAi Oligonucleotide to Inhibit Ataxin-1 for Spinocerebellar Ataxia - Drug Profile 35

Product Description 35

Mechanism Of Action 35

R&D Progress 35

Antisense RNAi Oligonucleotide to Inhibit Ataxin-7 for Spinocerebellar Ataxia Type 7 - Drug Profile 36

Product Description 36

Mechanism Of Action 36

R&D Progress 36

BHV-4157 - Drug Profile 37

Product Description 37

Mechanism Of Action 37

R&D Progress 37

epoetin alfa - Drug Profile 39

Product Description 39

Mechanism Of Action 39

R&D Progress 39

immune globulin (human) - Drug Profile 40

Product Description 40

Mechanism Of Action 40

R&D Progress 40

INT-41 - Drug Profile 44

Product Description 44

Mechanism Of Action 44

R&D Progress 44

JOT-105 - Drug Profile 45

Product Description 45

Mechanism Of Action 45

R&D Progress 45

riluzole - Drug Profile 46

Product Description 46

Mechanism Of Action 46

R&D Progress 46

RNAi Gene Therapy to Inhibit Ataxin-3 for Machado-Joseph Disease - Drug Profile 49

Product Description 49

Mechanism Of Action 49

R&D Progress 49

rovatirelin - Drug Profile 50

Product Description 50

Mechanism Of Action 50

R&D Progress 50

Stem Cell Therapy for Osteoarthritis, Diabetes Mellitus, Acute Liver Failure and Neurodegenerative Diseases - Drug Profile 51

Product Description 51

Mechanism Of Action 51

R&D Progress 51

trehalose - Drug Profile 52

Product Description 52

Mechanism Of Action 52

R&D Progress 52

Spinocerebellar Ataxia (SCA) - Dormant Projects 56

Spinocerebellar Ataxia (SCA) - Product Development Milestones 57

Featured News & Press Releases 57

Jul 20, 2016: Biohaven Reports Dosing Of First Subject In Pharmacokinetic And Safety Trial Of BHV-4157 57

Jul 05, 2016: Biohaven Receives FDA Investigational New Drug Application (IND) Approval for BHV-4157 57

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

May 25, 2016: Portage Announces Orphan Drug Designation Request Granted to Biohaven for BHV-4157 59

Mar 03, 2016: Portage Biohaven orphan drug designation request granted for the treatment of spinocerebellar ataxia 60

Sep 14, 2015: BioBlast Granted Patent From USPTO for Cabaletta for the Treatment of Spinocerebellar Ataxia 61

Jun 29, 2015: BioBlast Pharma Receives Orphan Drug Designation From the European Commission for Cabaletta for the Treatment of Spinocerebellar Ataxia 61

Mar 30, 2015: BioBlast Pharma Announces Positive in vivo Preclinical Proof-of-Concept Results of Its Cabaletta Platform in Spinocerebellar Ataxia Type 3 62

Nov 19, 2014: Bio Blast Pharma Receives Orphan Drug Designation From U.S. FDA for Cabaletta for the Treatment of Spinocerebellar Ataxia Type 3 62

Appendix 64

Methodology 64

Coverage 64

Secondary Research 64

Primary Research 64

Expert Panel Validation 64

Contact Us 64

Disclaimer 65

List of Tables

List of Tables

Number of Products under Development for Spinocerebellar Ataxia (SCA), H2 2016 8

Number of Products under Development for Spinocerebellar Ataxia (SCA) – Comparative Analysis, H2 2016 9

Number of Products under Development by Companies, H2 2016 10

Number of Products under Investigation by Universities/Institutes, H2 2016 11

Comparative Analysis by Late Stage Development, H2 2016 12

Comparative Analysis by Clinical Stage Development, H2 2016 13

Comparative Analysis by Early Stage Development, H2 2016 14

Products under Development by Companies, H2 2016 15

Products under Investigation by Universities/Institutes, H2 2016 16

Spinocerebellar Ataxia (SCA) – Pipeline by Bio Blast Pharma Ltd, H2 2016 17

Spinocerebellar Ataxia (SCA) – Pipeline by Biohaven Pharmaceutical Holding Company Limited, H2 2016 18

Spinocerebellar Ataxia (SCA) – Pipeline by Ionis Pharmaceuticals Inc, H2 2016 19

Spinocerebellar Ataxia (SCA) – Pipeline by Shionogi & Co Ltd, H2 2016 20

Spinocerebellar Ataxia (SCA) – Pipeline by Shire Plc, H2 2016 21

Spinocerebellar Ataxia (SCA) – Pipeline by Spark Therapeutics Inc, H2 2016 22

Spinocerebellar Ataxia (SCA) – Pipeline by Vybion Inc, H2 2016 23

Assessment by Monotherapy Products, H2 2016 24

Number of Products by Stage and Target, H2 2016 26

Number of Products by Stage and Mechanism of Action, H2 2016 28

Number of Products by Stage and Route of Administration, H2 2016 30

Number of Products by Stage and Molecule Type, H2 2016 32

Spinocerebellar Ataxia (SCA) – Dormant Projects, H2 2016 56

List of Figures

List of Figures

Number of Products under Development for Spinocerebellar Ataxia (SCA), H2 2016 8

Number of Products under Development for Spinocerebellar Ataxia (SCA) – Comparative Analysis, H2 2016 9

Number of Products under Development by Companies, H2 2016 10

Number of Products under Investigation by Universities/Institutes, H2 2016 11

Comparative Analysis by Late Stage Development, H2 2016 12

Comparative Analysis by Clinical Stage Development, H2 2016 13

Comparative Analysis by Early Stage Products, H2 2016 14

Assessment by Monotherapy Products, H2 2016 24

Number of Products by Targets, H2 2016 25

Number of Products by Stage and Targets, H2 2016 25

Number of Products by Mechanism of Actions, H2 2016 27

Number of Products by Stage and Mechanism of Actions, H2 2016 27

Number of Products by Routes of Administration, H2 2016 29

Number of Products by Stage and Routes of Administration, H2 2016 29

Number of Products by Molecule Types, H2 2016 31

Number of Products by Stage and Molecule Types, H2 2016 31

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