Spinocerebellar Ataxia (SCA) - Pipeline Review, H1 2016

Published: May, 2016 | Pages: 55 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

Spinocerebellar Ataxia (SCA) - Pipeline Review, H1 2016

Summary

Global Markets Direct's, 'Spinocerebellar Ataxia (SCA) - Pipeline Review, H1 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

 Table of Contents

Table of Contents 2
List of Tables 4
List of Figures 4
Introduction 5
Global Markets Direct Report Coverage 5
Spinocerebellar Ataxia (SCA) Overview 6
Therapeutics Development 7
Pipeline Products for Spinocerebellar Ataxia (SCA) - Overview 7
Pipeline Products for Spinocerebellar Ataxia (SCA) - Comparative Analysis 8
Spinocerebellar Ataxia (SCA) - Therapeutics under Development by Companies 9
Spinocerebellar Ataxia (SCA) - Therapeutics under Investigation by Universities/Institutes 10
Spinocerebellar Ataxia (SCA) - Pipeline Products Glance 11
Late Stage Products 11
Clinical Stage Products 12
Early Stage Products 13
Spinocerebellar Ataxia (SCA) - Products under Development by Companies 14
Spinocerebellar Ataxia (SCA) - Products under Investigation by Universities/Institutes 15
Spinocerebellar Ataxia (SCA) - Companies Involved in Therapeutics Development 16
Baxalta Incorporated 16
Bioblast Pharma Ltd. 17
Kissei Pharmaceutical Co., Ltd. 18
Vybion, Inc. 19
Spinocerebellar Ataxia (SCA) - Therapeutics Assessment 20
Assessment by Monotherapy Products 20
Assessment by Target 21
Assessment by Mechanism of Action 23
Assessment by Route of Administration 25
Assessment by Molecule Type 27
Drug Profiles 29
Antisense RNAi Oligonucleotide to Inhibit Ataxin-7 for Spinocerebellar Ataxia Type 7 - Drug Profile 29
Product Description 29
Mechanism of Action 29
R&D Progress 29
epoetin alfa - Drug Profile 30
Product Description 30
Mechanism of Action 30
R&D Progress 30
immune globulin (human) - Drug Profile 31
Product Description 31
Mechanism of Action 31
R&D Progress 31
INT-41 - Drug Profile 34
Product Description 34
Mechanism of Action 34
R&D Progress 34
riluzole - Drug Profile 36
Product Description 36
Mechanism of Action 36
R&D Progress 36
RNAi Gene Therapy for Machado-Joseph Disease - Drug Profile 38
Product Description 38
Mechanism of Action 38
R&D Progress 38
rovatirelin - Drug Profile 39
Product Description 39
Mechanism of Action 39
R&D Progress 39
Stem Cell Therapy for Osteoarthritis, Diabetes Mellitus, Acute Liver Failure and Neurodegenerative Diseases - Drug Profile 40
Product Description 40
Mechanism of Action 40
R&D Progress 40
trehalose - Drug Profile 42
Product Description 42
Mechanism of Action 42
R&D Progress 42
Spinocerebellar Ataxia (SCA) - Recent Pipeline Updates 43
Spinocerebellar Ataxia (SCA) - Dormant Projects 50
Spinocerebellar Ataxia (SCA) - Product Development Milestones 51
Featured News & Press Releases 51
Mar 03, 2016: Portage Biohaven orphan drug designation request granted for the treatment of spinocerebellar ataxia 51
Sep 14, 2015: BioBlast Granted Patent From USPTO for Cabaletta for the Treatment of Spinocerebellar Ataxia 52
Jun 29, 2015: BioBlast Pharma Receives Orphan Drug Designation From the European Commission for Cabaletta for the Treatment of Spinocerebellar Ataxia 52
Mar 30, 2015: BioBlast Pharma Announces Positive in vivo Preclinical Proof-of-Concept Results of Its Cabaletta Platform in Spinocerebellar Ataxia Type 3 52
Nov 19, 2014: Bio Blast Pharma Receives Orphan Drug Designation From U.S. FDA for Cabaletta for the Treatment of Spinocerebellar Ataxia Type 3 53
Appendix 54
Methodology 54
Coverage 54
Secondary Research 54
Primary Research 54
Expert Panel Validation 54
Contact Us 54
Disclaimer 55

List of Tables

Number of Products under Development for Spinocerebellar Ataxia (SCA), H1 2016 7
Number of Products under Development for Spinocerebellar Ataxia (SCA) - Comparative Analysis, H1 2016 8
Number of Products under Development by Companies, H1 2016 9
Number of Products under Investigation by Universities/Institutes, H1 2016 10
Comparative Analysis by Late Stage Development, H1 2016 11
Comparative Analysis by Clinical Stage Development, H1 2016 12
Comparative Analysis by Early Stage Development, H1 2016 13
Products under Development by Companies, H1 2016 14
Products under Investigation by Universities/Institutes, H1 2016 15
Spinocerebellar Ataxia (SCA) - Pipeline by Baxalta Incorporated, H1 2016 16
Spinocerebellar Ataxia (SCA) - Pipeline by Bioblast Pharma Ltd., H1 2016 17
Spinocerebellar Ataxia (SCA) - Pipeline by Kissei Pharmaceutical Co., Ltd., H1 2016 18
Spinocerebellar Ataxia (SCA) - Pipeline by Vybion, Inc., H1 2016 19
Assessment by Monotherapy Products, H1 2016 20
Number of Products by Stage and Target, H1 2016 22
Number of Products by Stage and Mechanism of Action, H1 2016 24
Number of Products by Stage and Route of Administration, H1 2016 26
Number of Products by Stage and Molecule Type, H1 2016 28
Spinocerebellar Ataxia (SCA) Therapeutics - Recent Pipeline Updates, H1 2016 43
Spinocerebellar Ataxia (SCA) - Dormant Projects, H1 2016 50