Spinocerebellar Ataxia (SCA) – Pipeline Review, H1 2018

Global Markets Direct’s, ‘Spinocerebellar Ataxia (SCA) – Pipeline Review, H1 2018’, 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

Spark Therapeutics Inc

Vybion Inc

WAVE Life Sciences Ltd

Table of Contents

Table of Contents

List of Tables

List of Figures

Introduction

Global Markets Direct Report Coverage

Spinocerebellar Ataxia (SCA) Overview

Spinocerebellar Ataxia (SCA) Therapeutics Development

Pipeline Overview

Pipeline by Companies

Pipeline by Universities/Institutes

Products under Development by Companies

Products under Development by Universities/Institutes

Spinocerebellar Ataxia (SCA) Therapeutics Assessment

Assessment by Target

Assessment by Mechanism of Action

Assessment by Route of Administration

Assessment by Molecule Type

Spinocerebellar Ataxia (SCA) Companies Involved in Therapeutics Development

Bio Blast Pharma Ltd

Biohaven Pharmaceutical Holding Company Ltd

Cadent Therapeutics

Ionis Pharmaceuticals Inc

Shionogi & Co Ltd

Spark Therapeutics Inc

Vybion Inc

WAVE Life Sciences Ltd

Spinocerebellar Ataxia (SCA) Drug Profiles

2AU-2 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

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

Product Description

Mechanism Of Action

R&D Progress

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

Product Description

Mechanism Of Action

R&D Progress

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

Product Description

Mechanism Of Action

R&D Progress

ASO-7 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

BHV-4157 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

CAD-1883 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

epoetin alfa Drug Profile

Product Description

Mechanism Of Action

R&D Progress

INT-41 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Oligonucleotides to Inhibit ATXN3 for Spinocerebellar Ataxia Type 3 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

riluzole Drug Profile

Product Description

Mechanism Of Action

R&D Progress

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

Product Description

Mechanism Of Action

R&D Progress

rovatirelin Drug Profile

Product Description

Mechanism Of Action

R&D Progress

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

Product Description

Mechanism Of Action

R&D Progress

trehalose Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Spinocerebellar Ataxia (SCA) Dormant Projects

Spinocerebellar Ataxia (SCA) Product Development Milestones

Featured News & Press Releases

Oct 02, 2017: Biohaven Reports Negative Topline Data from Spinocerebellar Ataxia Phase 2/3 Trial

Aug 07, 2017: Biohaven Completes Randomization In Phase 2/3 Trial In Spinocerebellar Ataxia: Expected Topline Data Advanced To Fourth Quarter Of 2017

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

May 30, 2017: Biohaven Completes Enrollment In Spinocerebellar Ataxia Clinical Trial With Trigriluzole

May 15, 2017: Biohaven's Trigriluzole Receives Fast Track Designation From U.S. FDA

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

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

Dec 22, 2016: Biohaven Enrolls First Patient in Pivotal Trial of BHV-4157 in Patients With Hereditary Spinocerebellar Ataxia

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

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

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

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

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

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

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

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer

List of Tables

List of Tables

Number of Products under Development for Spinocerebellar Ataxia (SCA), H1 2018

Number of Products under Development by Companies, H1 2018

Number of Products under Development by Universities/Institutes, H1 2018

Products under Development by Companies, H1 2018

Products under Development by Universities/Institutes, H1 2018

Number of Products by Stage and Target, H1 2018

Number of Products by Stage and Mechanism of Action, H1 2018

Number of Products by Stage and Route of Administration, H1 2018

Number of Products by Stage and Molecule Type, H1 2018

Spinocerebellar Ataxia (SCA) Pipeline by Bio Blast Pharma Ltd, H1 2018

Spinocerebellar Ataxia (SCA) Pipeline by Biohaven Pharmaceutical Holding Company Ltd, H1 2018

Spinocerebellar Ataxia (SCA) Pipeline by Cadent Therapeutics, H1 2018

Spinocerebellar Ataxia (SCA) Pipeline by Ionis Pharmaceuticals Inc, H1 2018

Spinocerebellar Ataxia (SCA) Pipeline by Shionogi & Co Ltd, H1 2018

Spinocerebellar Ataxia (SCA) Pipeline by Spark Therapeutics Inc, H1 2018

Spinocerebellar Ataxia (SCA) Pipeline by Vybion Inc, H1 2018

Spinocerebellar Ataxia (SCA) Pipeline by WAVE Life Sciences Ltd, H1 2018

Spinocerebellar Ataxia (SCA) Dormant Projects, H1 2018

List of Figures

List of Figures

Number of Products under Development for Spinocerebellar Ataxia (SCA), H1 2018

Number of Products under Development by Companies, H1 2018

Number of Products under Development by Universities/Institutes, H1 2018

Number of Products by Targets, H1 2018

Number of Products by Stage and Targets, H1 2018

Number of Products by Mechanism of Actions, H1 2018

Number of Products by Stage and Mechanism of Actions, H1 2018

Number of Products by Routes of Administration, H1 2018

Number of Products by Stage and Routes of Administration, H1 2018

Number of Products by Molecule Types, H1 2018

Number of Products by Stage and Molecule Types, H1 2018

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