Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline Review, H1 2014

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline Review, H1 2014

Summary

Global Markets Direct’s, ‘Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline Review, H1 2014’, provides an overview of the Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C’s therapeutic pipeline.

This report provides comprehensive information on the therapeutic development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C, 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) Type C 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) Type C
- 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) Type C 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) Type C 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) Type C 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) Type C
- 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) Type C 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
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C Overview 6
Therapeutics Development 7
Pipeline Products for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Overview 7
Pipeline Products for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Comparative Analysis 8
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Therapeutics under Development by Companies 9
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Therapeutics under Investigation by Universities/Institutes 10
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline Products Glance 12
Clinical Stage Products 12
Early Stage Products 13
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Products under Development by Companies 14
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Products under Investigation by Universities/Institutes 15
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Companies Involved in Therapeutics Development 16
La Jolla Pharmaceutical Company 16
Orphazyme ApS 17
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Therapeutics Assessment 18
Assessment by Monotherapy Products 18
Assessment by Target 19
Assessment by Mechanism of Action 21
Assessment by Route of Administration 23
Assessment by Molecule Type 25
Drug Profiles 27
Hydroxypropyl Beta Cyclodextrin - Drug Profile 27
Product Description 27
Mechanism of Action 27
R&D Progress 27
OR-0005 - Drug Profile 28
Product Description 28
Mechanism of Action 28
R&D Progress 28
LJPC-0712 - Drug Profile 29
Product Description 29
Mechanism of Action 29
R&D Progress 29
Drug For Niemann-Pick C Disease - Drug Profile 30
Product Description 30
Mechanism of Action 30
R&D Progress 30
ML-SA1 - Drug Profile 31
Product Description 31
Mechanism of Action 31
R&D Progress 31
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Recent Pipeline Updates 32
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Dormant Projects 33
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Product Development Milestones 34
Featured News & Press Releases 34
Jul 17, 2013: La Jolla Pharmaceutical Company Receives Orphan Designation From FDA 34
Apr 03, 2013: Old Drug Offers New Treatment For Niemann-Pick Type C, Study Finds 34
Feb 21, 2013: Orphazyme Receives Orphan Drug Designation For OR0005 In EU 36
Jan 23, 2013: NIH Clinical Trial Begins For Treatment Of Niemann-Pick Disease Type C1 36
Appendix 38
Methodology 38
Coverage 38
Secondary Research 38
Primary Research 38
Expert Panel Validation 38
Contact Us 39
Disclaimer 39

List of Tables
Number of Products under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C, H1 2014 7
Number of Products under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Comparative Analysis, H1 2014 8
Number of Products under Development by Companies, H1 2014 9
Number of Products under Investigation by Universities/Institutes, H1 2014 11
Comparative Analysis by Clinical Stage Development, H1 2014 12
Comparative Analysis by Early Stage Development, H1 2014 13
Products under Development by Companies, H1 2014 14
Products under Investigation by Universities/Institutes, H1 2014 15
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by La Jolla Pharmaceutical Company, H1 2014 16
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by Orphazyme ApS, H1 2014 17
Assessment by Monotherapy Products, H1 2014 18
Number of Products by Stage and Target, H1 2014 20
Number of Products by Stage and Mechanism of Action, H1 2014 22
Number of Products by Stage and Route of Administration, H1 2014 24
Number of Products by Stage and Molecule Type, H1 2014 26
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C Therapeutics - Recent Pipeline Updates, H1 2014 32
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Dormant Projects, H1 2014 33

List of Figures
Number of Products under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C, H1 2014 7
Number of Products under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Comparative Analysis, H1 2014 8
Number of Products under Investigation by Universities/Institutes, H1 2014 10
Comparative Analysis by Early Stage Products, H1 2014 13
Assessment by Monotherapy Products, H1 2014 18
Number of Products by Top 10 Target, H1 2014 19
Number of Products by Stage and Top 10 Target, H1 2014 20
Number of Products by Top 10 Mechanism of Action, H1 2014 21
Number of Products by Stage and Top 10 Mechanism of Action, H1 2014 22
Number of Products by Top 10 Route of Administration, H1 2014 23
Number of Products by Stage and Top 10 Route of Administration, H1 2014 24
Number of Products by Top 10 Molecule Type, H1 2014 25
Number of Products by Stage and Top 10 Molecule Type, H1 2014 26