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Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Pipeline Review, H2 2015

Summary

Global Markets Direct’s, ‘Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Pipeline Review, H2 2015’, provides an overview of the Acid Sphingomyelinase Deficiency (Niemann-Pick Disease)’s therapeutic pipeline.

This report provides comprehensive information on the therapeutic development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease), 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 Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) 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 Acid Sphingomyelinase Deficiency (Niemann-Pick Disease)
- 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 Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) and enlists all their major and minor projects
- The report summarizes all the dormant and discontinued pipeline projects
- A review of the Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) 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 Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) 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 Acid Sphingomyelinase Deficiency (Niemann-Pick Disease)
- Plan mergers and acquisitions effectively by identifying key players of the most promising pipeline
- Devise corrective measures for pipeline projects by understanding Acid Sphingomyelinase Deficiency (Niemann-Pick 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

Table Of Contents

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Pipeline Review, H2 2015
Table of Contents
Table of Contents 2
List of Tables 5
List of Figures 5
Introduction 6
Global Markets Direct Report Coverage 6
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Overview 7
Therapeutics Development 8
Pipeline Products for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Overview 8
Pipeline Products for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Comparative Analysis 9
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Therapeutics under Development by Companies 10
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Therapeutics under Investigation by Universities/Institutes 11
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Pipeline Products Glance 12
Clinical Stage Products 12
Early Stage Products 13
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Products under Development by Companies 14
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Products under Investigation by Universities/Institutes 15
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Companies Involved in Therapeutics Development 16
Genzyme Corporation 16
La Jolla Pharmaceutical Company 17
Merck and Co., Inc. 18
Okklo Life Sciences BV 19
Orphazyme ApS 20
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Therapeutics Assessment 21
Assessment by Monotherapy Products 21
Assessment by Target 22
Assessment by Mechanism of Action 24
Assessment by Route of Administration 26
Assessment by Molecule Type 28
Drug Profiles 30
arimoclomol - Drug Profile 30
Product Description 30
Mechanism of Action 30
RandD Progress 30
Bryostatin-1 - Drug Profile 32
Product Description 32
Mechanism of Action 32
RandD Progress 32
Delta-tocopherol - Drug Profile 34
Product Description 34
Mechanism of Action 34
RandD Progress 34
LJPC-0712 - Drug Profile 35
Product Description 35
Mechanism of Action 35
RandD Progress 35
ML-SA1 - Drug Profile 36
Product Description 36
Mechanism of Action 36
RandD Progress 36
OKL-1014 - Drug Profile 37
Product Description 37
Mechanism of Action 37
RandD Progress 37
olipudase alfa - Drug Profile 38
Product Description 38
Mechanism of Action 38
RandD Progress 38
OR-0005 - Drug Profile 40
Product Description 40
Mechanism of Action 40
RandD Progress 40
Stem Cell Therapy for Cancer, Pain, Retinal Degeneration and Niemann-Pick Disease - Drug Profile 41
Product Description 41
Mechanism of Action 41
RandD Progress 41
vorinostat - Drug Profile 42
Product Description 42
Mechanism of Action 42
RandD Progress 42
VTS-270 - Drug Profile 46
Product Description 46
Mechanism of Action 46
RandD Progress 46
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Recent Pipeline Updates 48
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Dormant Projects 53
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Product Development Milestones 54
Featured News and Press Releases 54
Jun 24, 2015: Neurotrope to Conduct Study on Niemann-Pick Type C, a Devastating Rare Pediatric Disease 54
Jun 15, 2015: Genzyme Initiates Phase 1/2 Clinical Trial to Evaluate Olipudase Alfa in Pediatric Patients 54
Jun 12, 2015: Neurotrope to Present Findings of In-vitro Studies of Bryostatin's Effects on Niemann Pick Type C1 Cells at the Annual "Michael, Marcia and Christa Parseghian Scientific Conference" for Niemann-Pick Type C 55
Jun 04, 2015: FDA Grants Breakthrough Therapy Designation for Genzyme's Olipudase Alfa 56
Apr 15, 2015: Orphazyme Forms Scientific Advisory Board and Confirms the Enrolment Initiation for the Clinical Programme 56
Feb 12, 2015: Genzyme Presents Data from its Phase 1b Program for Niemann-Pick Type B at the Lysosomal Disease Network's WORLD Symposium 2015 57
Jan 26, 2015: FDA grants orphan drug designation for Arimoclomol 57
Jan 26, 2015: Orphazyme to present at the Gordon Research Conference on Lysosomal Diseases 15-19 March in Galveston, TX 58
Oct 20, 2014: COMP issues positive opinion on orphan drug application for Arimoclomol 58
Oct 06, 2014: Orphazyme in final preparation stages for a Phase II/III clinical trial 58
Appendix 60
Methodology 60
Coverage 60
Secondary Research 60
Primary Research 60
Expert Panel Validation 60
Contact Us 60
Disclaimer 61

List of Tables
Number of Products under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease), H2 2015 8
Number of Products under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Comparative Analysis, H2 2015 9
Number of Products under Development by Companies, H2 2015 10
Number of Products under Investigation by Universities/Institutes, H2 2015 11
Comparative Analysis by Clinical Stage Development, H2 2015 12
Comparative Analysis by Early Stage Development, H2 2015 13
Products under Development by Companies, H2 2015 14
Products under Investigation by Universities/Institutes, H2 2015 15
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Pipeline by Genzyme Corporation, H2 2015 16
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Pipeline by La Jolla Pharmaceutical Company, H2 2015 17
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Pipeline by Merck and Co., Inc., H2 2015 18
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Pipeline by Okklo Life Sciences BV, H2 2015 19
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Pipeline by Orphazyme ApS, H2 2015 20
Assessment by Monotherapy Products, H2 2015 21
Number of Products by Stage and Target, H2 2015 23
Number of Products by Stage and Mechanism of Action, H2 2015 25
Number of Products by Stage and Route of Administration, H2 2015 27
Number of Products by Stage and Molecule Type, H2 2015 29
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Therapeutics - Recent Pipeline Updates, H2 2015 48
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Dormant Projects, H2 2015 53

List of Figures
Number of Products under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease), H2 2015 8
Number of Products under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Comparative Analysis, H2 2015 9
Number of Products under Development by Companies, H2 2015 10
Number of Products under Investigation by Universities/Institutes, H2 2015 11
Comparative Analysis by Clinical Stage Development, H2 2015 12
Comparative Analysis by Early Stage Products, H2 2015 13
Assessment by Monotherapy Products, H2 2015 21
Number of Products by Top 10 Targets, H2 2015 22
Number of Products by Stage and Top 10 Targets, H2 2015 22
Number of Products by Top 10 Mechanism of Actions, H2 2015 24
Number of Products by Stage and Top 10 Mechanism of Actions, H2 2015 24
Number of Products by Top 10 Routes of Administration, H2 2015 26
Number of Products by Stage and Top 10 Routes of Administration, H2 2015 26
Number of Products by Top 10 Molecule Types, H2 2015 28
Number of Products by Stage and Top 10 Molecule Types, H2 2015 28

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