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Spinocerebellar Ataxias-Pipeline Review, H1 2015

Spinocerebellar Ataxias-Pipeline Review, H1 2015


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Executive Summary

Spinocerebellar Ataxias-Pipeline Review, H1 2015

Summary

Global Markets Direct's, 'Spinocerebellar Ataxias-Pipeline Review, H1 2015', provides an overview of the Spinocerebellar Ataxias's therapeutic pipeline.

This report provides comprehensive information on the therapeutic development for Spinocerebellar Ataxias, 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 Spinocerebellar Ataxias 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 Spinocerebellar Ataxias

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 Spinocerebellar Ataxias and enlists all their major and minor projects

The report summarizes all the dormant and discontinued pipeline projects

A review of the Spinocerebellar Ataxias 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 Spinocerebellar Ataxias 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 Spinocerebellar Ataxias

Plan mergers and acquisitions effectively by identifying key players of the most promising pipeline

Devise corrective measures for pipeline projects by understanding Spinocerebellar Ataxias 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 Ataxias Overview 6

Therapeutics Development 7

Pipeline Products for Spinocerebellar Ataxias-Overview 7

Pipeline Products for Spinocerebellar Ataxias-Comparative Analysis 8

Spinocerebellar Ataxias-Therapeutics under Development by Companies 9

Spinocerebellar Ataxias-Therapeutics under Investigation by Universities/Institutes 10

Spinocerebellar Ataxias-Pipeline Products Glance 11

Late Stage Products 11

Clinical Stage Products 12

Early Stage Products 13

Spinocerebellar Ataxias-Products under Development by Companies 14

Spinocerebellar Ataxias-Products under Investigation by Universities/Institutes 15

Spinocerebellar Ataxias-Companies Involved in Therapeutics Development 16

Baxter International Inc. 16

Bioblast Pharma Ltd. 17

Kissei Pharmaceutical Co., Ltd. 18

Vybion, Inc. 19

Spinocerebellar Ataxias-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

epoetin alfa-Drug Profile 29

Product Description 29

Mechanism of Action 29

R&D Progress 29

immune globulin (human)-Drug Profile 30

Product Description 30

Mechanism of Action 30

R&D Progress 30

INT-41-Drug Profile 33

Product Description 33

Mechanism of Action 33

R&D Progress 33

KPS-0373-Drug Profile 35

Product Description 35

Mechanism of Action 35

R&D Progress 35

RNAi Gene Therapy for Machado-Joseph Disease-Drug Profile 36

Product Description 36

Mechanism of Action 36

R&D Progress 36

Stem Cell Therapy for Osteoarthritis, Diabetes Mellitus, Acute Liver Failure, Post Cardiopulmonary Resuscitation and Neurodegenerative Diseases-Drug Profile 37

Product Description 37

Mechanism of Action 37

R&D Progress 37

trehalose-Drug Profile 38

Product Description 38

Mechanism of Action 38

R&D Progress 38

Spinocerebellar Ataxias-Recent Pipeline Updates 39

Spinocerebellar Ataxias-Dormant Projects 43

Spinocerebellar Ataxias-Dormant Projects 43

Spinocerebellar Ataxias-Product Development Milestones 44

Featured News & Press Releases 44

Mar 30, 2015: BioBlast Pharma Announces Positive in vivo Preclinical Proof-of-Concept Results of Its Cabaletta Platform in Spinocerebellar Ataxia Type 3 44

Nov 19, 2014: Bio Blast Pharma Receives Orphan Drug Designation From U.S. FDA for Cabaletta for the Treatment of Spinocerebellar Ataxia Type 3 44

Appendix 46

Methodology 46

Coverage 46

Secondary Research 46

Primary Research 46

Expert Panel Validation 46

Contact Us 46

Disclaimer 47

List of Tables

Number of Products under Development for Spinocerebellar Ataxias, H1 2015 7

Number of Products under Development for Spinocerebellar Ataxias-Comparative Analysis, H1 2015 8

Number of Products under Development by Companies, H1 2015 9

Number of Products under Investigation by Universities/Institutes, H1 2015 10

Comparative Analysis by Late Stage Development, H1 2015 11

Comparative Analysis by Clinical Stage Development, H1 2015 12

Comparative Analysis by Early Stage Development, H1 2015 13

Products under Development by Companies, H1 2015 14

Products under Investigation by Universities/Institutes, H1 2015 15

Spinocerebellar Ataxias-Pipeline by Baxter International Inc., H1 2015 16

Spinocerebellar Ataxias-Pipeline by Bioblast Pharma Ltd., H1 2015 17

Spinocerebellar Ataxias-Pipeline by Kissei Pharmaceutical Co., Ltd., H1 2015 18

Spinocerebellar Ataxias-Pipeline by Vybion, Inc., H1 2015 19

Assessment by Monotherapy Products, H1 2015 20

Number of Products by Stage and Target, H1 2015 22

Number of Products by Stage and Mechanism of Action, H1 2015 24

Number of Products by Stage and Route of Administration, H1 2015 26

Number of Products by Stage and Molecule Type, H1 2015 28

Spinocerebellar Ataxias Therapeutics-Recent Pipeline Updates, H1 2015 39

Spinocerebellar Ataxias-Dormant Projects, H1 2015 43

List of Figures

Number of Products under Development for Spinocerebellar Ataxias, H1 2015 7

Number of Products under Development for Spinocerebellar Ataxias-Comparative Analysis, H1 2015 8

Number of Products under Development by Companies, H1 2015 9

Number of Products under Investigation by Universities/Institutes, H1 2015 10

Comparative Analysis by Clinical Stage Development, H1 2015 12

Comparative Analysis by Early Stage Products, H1 2015 13

Assessment by Monotherapy Products, H1 2015 20

Number of Products by Top 10 Targets, H1 2015 21

Number of Products by Stage and Top 10 Targets, H1 2015 21

Number of Products by Top 10 Mechanism of Actions, H1 2015 23

Number of Products by Stage and Top 10 Mechanism of Actions, H1 2015 23

Number of Products by Top 10 Routes of Administration, H1 2015 25

Number of Products by Stage and Top 10 Routes of Administration, H1 2015 25

Number of Products by Top 10 Molecule Types, H1 2015 27

Number of Products by Stage and Top 10 Molecule Types, H1 2015 27

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Baxter International Inc.

Bioblast Pharma Ltd.

Kissei Pharmaceutical Co., Ltd.

Vybion, Inc.

Spinocerebellar Ataxias Therapeutic Products under Development, Key Players in Spinocerebellar Ataxias Therapeutics, Spinocerebellar Ataxias Pipeline Overview, Spinocerebellar Ataxias Pipeline, Spinocerebellar Ataxias Pipeline Assessment


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