Batten Disease - Pipeline Review, H2 2016

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Date: 31-Aug-2016
No. of pages: 72
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Batten Disease - Pipeline Review, H2 2016

Summary

Global Markets Direct's, ‘Batten Disease - Pipeline Review, H2 2016', provides an overview of the Batten Disease pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Batten Disease, 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 Batten Disease 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 Batten Disease

- The report reviews pipeline therapeutics for Batten Disease 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 Batten Disease therapeutics and enlists all their major and minor projects

- The report assesses Batten Disease 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 Batten Disease

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 Batten Disease

- 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 Batten Disease 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

Batten Disease - Pipeline Review, H2 2016

Table of Contents
Table of Contents 2
List of Tables 5
List of Figures 6
Introduction 7
Global Markets Direct Report Coverage 7
Batten Disease Overview 8
Therapeutics Development 9
Pipeline Products for Batten Disease - Overview 9
Pipeline Products for Batten Disease - Comparative Analysis 10
Batten Disease - Therapeutics under Development by Companies 11
Batten Disease - Therapeutics under Investigation by Universities/Institutes 12
Batten Disease - Pipeline Products Glance 13
Late Stage Products 13
Clinical Stage Products 14
Early Stage Products 15
Unknown Stage Products 16
Batten Disease - Products under Development by Companies 17
Batten Disease - Products under Investigation by Universities/Institutes 18
Batten Disease - Companies Involved in Therapeutics Development 19
Abeona Therapeutics, Inc. 19
BioMarin Pharmaceutical Inc. 20
CereSpir Incorporated 21
Evotec AG 22
Ionis Pharmaceuticals, Inc. 23
Mitochon Pharmaceuticals, Inc. 24
RegenxBio Inc. 25
Retrotope, Inc 26
Spark Therapeutics, Inc. 27
Batten Disease - Therapeutics Assessment 28
Assessment by Monotherapy Products 28
Assessment by Target 29
Assessment by Mechanism of Action 31
Assessment by Route of Administration 33
Assessment by Molecule Type 35
Drug Profiles 37
ABO-201 - Drug Profile 37
Product Description 37
Mechanism Of Action 37
R&D Progress 37
Antisense Oligonucleotides to Activate CLN3 for Batten Disease - Drug Profile 38
Product Description 38
Mechanism Of Action 38
R&D Progress 38
cerliponase alfa - Drug Profile 39
Product Description 39
Mechanism Of Action 39
R&D Progress 39
CSP-1103 - Drug Profile 41
Product Description 41
Mechanism Of Action 41
R&D Progress 41
DUOC-01 - Drug Profile 43
Product Description 43
Mechanism Of Action 43
R&D Progress 43
Gene Therapy to Activate CLN1 and CLN2 for Batten Disease - Drug Profile 44
Product Description 44
Mechanism Of Action 44
R&D Progress 44
Gene Therapy to Activate CLN6 for Batten Disease - Drug Profile 45
Product Description 45
Mechanism Of Action 45
R&D Progress 45
Gene Therapy to Activate Palmitoyl Protein Thioesterase 1 for Batten Disease - Drug Profile 46
Product Description 46
Mechanism Of Action 46
R&D Progress 46
Gene Therapy to Activate Tripeptidyl Peptidase 1 for Batten Disease - Drug Profile 47
Product Description 47
Mechanism Of Action 47
R&D Progress 47
INI-0602 - Drug Profile 49
Product Description 49
Mechanism Of Action 49
R&D Progress 49
MP-101 - Drug Profile 50
Product Description 50
Mechanism Of Action 50
R&D Progress 50
RT-002 - Drug Profile 51
Product Description 51
Mechanism Of Action 51
R&D Progress 51
Small Molecule for Batten Disease - Drug Profile 52
Product Description 52
Mechanism Of Action 52
R&D Progress 52
Small Molecules for Juvenile Batten Disease - Drug Profile 53
Product Description 53
Mechanism Of Action 53
R&D Progress 53
Small Molecules to Inhibit CDC42 for Batten Disease - Drug Profile 54
Product Description 54
Mechanism Of Action 54
R&D Progress 54
SNB-2401 - Drug Profile 55
Product Description 55
Mechanism Of Action 55
R&D Progress 55
SNB-4050 - Drug Profile 56
Product Description 56
Mechanism Of Action 56
R&D Progress 56
SPKTPP-1 - Drug Profile 57
Product Description 57
Mechanism Of Action 57
R&D Progress 57
XN-001 - Drug Profile 58
Product Description 58
Mechanism Of Action 58
R&D Progress 58
Batten Disease - Dormant Projects 59
Batten Disease - Discontinued Products 60
Batten Disease - Product Development Milestones 61
Featured News & Press Releases 61
Jul 27, 2016: FDA Accepts BLA for BioMarins Cerliponase Alfa for CLN2 Disease, Form of Batten Disease 61
May 03, 2016: BioMarin Announces EMA Grants Accelerated Assessment for Cerliponase Alfa, Experimental Treatment for a Form of Batten Disease 61
Mar 02, 2016: Abeona Therapeutics Highlights New Preclinical Juvenile Neuronal Ceroid Lipofuscinosis (JNCL) Data at WORLDSymposium 2016 62
Mar 02, 2016: BioMarin Announces Positive Data From Cerliponase Alfa Program for Treatment of CLN2 Disease, a Form of Batten Disease, at 12th Annual WORLDSymposium 2016 63
Nov 11, 2015: Spark Therapeutics Announces SPK-TPP1, Its First Program Targeting Neurodegenerative Disease 66
Jan 12, 2015: BioMarin Provides Preliminary Data From Ongoing Phase 1/2 Pivotal Study of BMN 190 for Treatment of CLN2 Disorder, a Form of Batten Disease 66
Sep 23, 2013: BioMarin Doses First Patient in Phase 1/2 Trial With BMN 190 for the Treatment of Neuronal Ceroid Lipofuscinosis Type 2, a Form of Batten Disease 68
Apr 01, 2013: BioMarin Pharma Submits CTA For BMN-190 For Batten Disease 68
Mar 15, 2012: Combination treatment in mice shows promise for fatal neurological disorder in kids 69
Appendix 71
Methodology 71
Coverage 71
Secondary Research 71
Primary Research 71
Expert Panel Validation 71
Contact Us 71
Disclaimer 72

List of Tables
Number of Products under Development for Batten Disease, H2 2016 9
Number of Products under Development for Batten Disease - Comparative Analysis, H2 2016 10
Number of Products under Development by Companies, H2 2016 11
Number of Products under Investigation by Universities/Institutes, H2 2016 12
Comparative Analysis by Late Stage Development, H2 2016 13
Comparative Analysis by Clinical Stage Development, H2 2016 14
Comparative Analysis by Early Stage Development, H2 2016 15
Comparative Analysis by Unknown Stage Development, H2 2016 16
Products under Development by Companies, H2 2016 17
Products under Investigation by Universities/Institutes, H2 2016 18
Batten Disease - Pipeline by Abeona Therapeutics, Inc. , H2 2016 19
Batten Disease - Pipeline by BioMarin Pharmaceutical Inc., H2 2016 20
Batten Disease - Pipeline by CereSpir Incorporated, H2 2016 21
Batten Disease - Pipeline by Evotec AG, H2 2016 22
Batten Disease - Pipeline by Ionis Pharmaceuticals, Inc., H2 2016 23
Batten Disease - Pipeline by Mitochon Pharmaceuticals, Inc., H2 2016 24
Batten Disease - Pipeline by RegenxBio Inc., H2 2016 25
Batten Disease - Pipeline by Retrotope, Inc, H2 2016 26
Batten Disease - Pipeline by Spark Therapeutics, Inc., H2 2016 27
Assessment by Monotherapy Products, H2 2016 28
Number of Products by Stage and Target, H2 2016 30
Number of Products by Stage and Mechanism of Action, H2 2016 32
Number of Products by Stage and Route of Administration, H2 2016 34
Number of Products by Stage and Molecule Type, H2 2016 36
Batten Disease - Dormant Projects, H2 2016 59
Batten Disease - Discontinued Products, H2 2016 60

List of Figures
Number of Products under Development for Batten Disease, H2 2016 9
Number of Products under Development for Batten Disease - Comparative Analysis, H2 2016 10
Number of Products under Development by Companies, H2 2016 11
Number of Products under Investigation by Universities/Institutes, H2 2016 12
Comparative Analysis by Clinical Stage Development, H2 2016 14
Comparative Analysis by Early Stage Products, H2 2016 15
Assessment by Monotherapy Products, H2 2016 28
Number of Products by Targets, H2 2016 29
Number of Products by Stage and Targets, H2 2016 29
Number of Products by Mechanism of Actions, H2 2016 31
Number of Products by Stage and Mechanism of Actions, H2 2016 31
Number of Products by Routes of Administration, H2 2016 33
Number of Products by Stage and Routes of Administration, H2 2016 33
Number of Products by Molecule Types, H2 2016 35
Number of Products by Stage and Molecule Types, H2 2016 35
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