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Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Pipeline Review, H2 2014

Published: Dec-2014 | Format: PDF | Global Markets Direct | Number of pages: 55 | Code: MRS - 18564


Global Markets Directs, Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Pipeline Review, H2 2014, 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 Directs 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 Directs 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 Directs 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.

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 Content

Table of Contents 2
List of Tables 4
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 & 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 Combination Products 22
Assessment by Target 23
Assessment by Mechanism of Action 25
Assessment by Route of Administration 27
Assessment by Molecule Type 29
Drug Profiles 31
arimoclomol - Drug Profile 31
Product Description 31
Mechanism of Action 31
R&D Progress 31
cyclodextrins + delta-tocopherol - Drug Profile 33
Product Description 33
Mechanism of Action 33
R&D Progress 33
GZ-402665 - Drug Profile 34
Product Description 34
Mechanism of Action 34
R&D Progress 34
LJPC-0712 - Drug Profile 35
Product Description 35
Mechanism of Action 35
R&D Progress 35
ML-SA1 - Drug Profile 36
Product Description 36
Mechanism of Action 36
R&D Progress 36
OKL-1014 - Drug Profile 37
Product Description 37
Mechanism of Action 37
R&D Progress 37
OR-0005 - Drug Profile 38
Product Description 38
Mechanism of Action 38
R&D Progress 38
Small Molecule to Target Cholesterol for Niemann-Pick Disease Type C - Drug Profile 39
Product Description 39
Mechanism of Action 39
R&D Progress 39
Stem Cell Therapy for Cancer, Pain, Retinal Degeneration and Niemann-Pick Disease - Drug Profile 40
Product Description 40
Mechanism of Action 40
R&D Progress 40
vorinostat - Drug Profile 41
Product Description 41
Mechanism of Action 41
R&D Progress 41
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Recent Pipeline Updates 45
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Dormant Projects 47
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Product Development Milestones 48
Featured News & Press Releases 48
Jul 17, 2013: La Jolla Pharmaceutical Company Receives Orphan Designation From FDA 48
Apr 03, 2013: Old Drug Offers New Treatment For Niemann-Pick Type C, Study Finds 48
Feb 21, 2013: Orphazyme Receives Orphan Drug Designation For OR0005 In EU 50
Jan 23, 2013: NIH Clinical Trial Begins For Treatment Of Niemann-Pick Disease Type C1 50
Mar 13, 2012: U-M biologists Find Potential Drug That Speeds Cellular Recycling 51
Sep 19, 2001: Recombinant Human Acid Sphingomyelinase For The Treatment Of Niemann-Pick Disease, Type B Receives Orphan Drug Designation From Committee For Orphan Medicinal Products 53

Appendix 54
Methodology 54
Coverage 54
Secondary Research 54
Primary Research 54
Expert Panel Validation 54
Contact Us 55
Disclaimer 55

List of Tables

Number of Products under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease), H2 2014 8
Number of Products under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Comparative Analysis, H2 2014 9
Number of Products under Development by Companies, H2 2014 10
Number of Products under Investigation by Universities/Institutes, H2 2014 11
Comparative Analysis by Clinical Stage Development, H2 2014 12
Comparative Analysis by Early Stage Development, H2 2014 13
Products under Development by Companies, H2 2014 14
Products under Investigation by Universities/Institutes, H2 2014 15
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Pipeline by Genzyme Corporation, H2 2014 16
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Pipeline by La Jolla Pharmaceutical Company, H2 2014 17
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Pipeline by Merck & Co., Inc., H2 2014 18
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Pipeline by Okklo Life Sciences BV, H2 2014 19
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Pipeline by Orphazyme ApS, H2 2014 20
Assessment by Monotherapy Products, H2 2014 21
Assessment by Combination Products, H2 2014 22
Number of Products by Stage and Target, H2 2014 24
Number of Products by Stage and Mechanism of Action, H2 2014 26
Number of Products by Stage and Route of Administration, H2 2014 28
Number of Products by Stage and Molecule Type, H2 2014 30
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Therapeutics - Recent Pipeline Updates, H2 2014 45
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Dormant Projects, H2 2014 47

List of Figures

Number of Products under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease), H2 2014 8
Number of Products under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Comparative Analysis, H2 2014 9
Number of Products under Development by Companies, H2 2014 10
Number of Products under Investigation by Universities/Institutes, H2 2014 11
Comparative Analysis by Clinical Stage Development, H2 2014 12
Comparative Analysis by Early Stage Products, H2 2014 13
Assessment by Monotherapy Products, H2 2014 21
Number of Products by Top 10 Targets, H2 2014 23
Number of Products by Stage and Top 10 Targets, H2 2014 23
Number of Products by Top 10 Mechanism of Actions, H2 2014 25
Number of Products by Stage and Top 10 Mechanism of Actions, H2 2014 25
Number of Products by Top 10 Routes of Administration, H2 2014 27
Number of Products by Stage and Top 10 Routes of Administration, H2 2014 27
Number of Products by Top 10 Molecule Types, H2 2014 29
Number of Products by Stage and Top 10 Molecule Types, H2 2014 29

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