Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline Review, H1 2016

Published: Apr, 2016 | Pages: 63 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline Review, H1 2016

Summary

Global Markets Direct's, 'Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline Review, H1 2016', provides an overview of the Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C pipeline landscape. 

The report provides comprehensive information on the therapeutics under development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C, 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 Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C 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 Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C
- The report reviews pipeline therapeutics for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C 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 Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C therapeutics and enlists all their major and minor projects
- The report assesses Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C 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 Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C

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 Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C
- 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 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 Content 2
List of Tables 5
List of Figures 5
Introduction 6
Global Markets Direct Report Coverage 6
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C Overview 7
Therapeutics Development 8
Pipeline Products for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Overview 8
Pipeline Products for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Comparative Analysis 9
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Therapeutics under Development by Companies 10
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Therapeutics under Investigation by Universities/Institutes 11
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
CTD Holdings, Inc. 16
La Jolla Pharmaceutical Company 17
Merck & Co., Inc. 18
Okklo Life Sciences BV 19
Orphazyme ApS 20
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - 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
Antibodies for Metabolic Disorders - Drug Profile 30
Product Description 30
Mechanism of Action 30
R&D Progress 30
arimoclomol - Drug Profile 31
Product Description 31
Mechanism of Action 31
R&D Progress 31
Bryostatin-1 - Drug Profile 33
Product Description 33
Mechanism of Action 33
R&D Progress 33
Delta-tocopherol - Drug Profile 35
Product Description 35
Mechanism of Action 35
R&D Progress 35
HPGCD - Drug Profile 36
Product Description 36
Mechanism of Action 36
R&D Progress 36
LJPC-0712 - Drug Profile 37
Product Description 37
Mechanism of Action 37
R&D Progress 37
ML-SA1 - Drug Profile 38
Product Description 38
Mechanism of Action 38
R&D Progress 38
OKL-1014 - Drug Profile 39
Product Description 39
Mechanism of Action 39
R&D Progress 39
OR-0005 - Drug Profile 40
Product Description 40
Mechanism of Action 40
R&D Progress 40
PERL-101 - Drug Profile 41
Product Description 41
Mechanism of Action 41
R&D Progress 41
Small Molecules for Niemann-Pick Type C Disease - Drug Profile 42
Product Description 42
Mechanism of Action 42
R&D Progress 42
Trappsol Cyclo - Drug Profile 43
Product Description 43
Mechanism of Action 43
R&D Progress 43
vorinostat - Drug Profile 44
Product Description 44
Mechanism of Action 44
R&D Progress 44
VTS-270 - Drug Profile 48
Product Description 48
Mechanism of Action 48
R&D Progress 48
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Recent Pipeline Updates 50
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Dormant Projects 55
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Product Development Milestones 56
Featured News & Press Releases 56
Feb 29, 2016: CTD Holdings Clinical Program to Move Forward in the U.S. 56
Feb 18, 2016: Parent Advocates Join CTD Holdings Family and Physicians Listening Circle on NPC Treatment 56
Dec 14, 2015: CTD Holdings' Clinical Trial Design Receives Positive Responses From U.K.'s Medicines and Healthcare Products Regulatory Agency 57
Jun 24, 2015: Neurotrope to Conduct Study on Niemann-Pick Type C, a Devastating Rare Pediatric Disease 58
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 & Christa Parseghian Scientific Conference" for Niemann-Pick Type C 58
Apr 15, 2015: Orphazyme Forms Scientific Advisory Board and Confirms the Enrolment Initiation for the Clinical Programme 59
Jan 26, 2015: FDA grants orphan drug designation for Arimoclomol 60
Jan 26, 2015: Orphazyme to present at the Gordon Research Conference on Lysosomal Diseases 15-19 March in Galveston, TX 60
Oct 20, 2014: COMP issues positive opinion on orphan drug application for Arimoclomol 60
Oct 06, 2014: Orphazyme in final preparation stages for a Phase II/III clinical trial 61
Appendix 62
Methodology 62
Coverage 62
Secondary Research 62
Primary Research 62
Expert Panel Validation 62
Contact Us 62
Disclaimer 63

List of Tables

Number of Products under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C, H1 2016 8
Number of Products under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Comparative Analysis, H1 2016 9
Number of Products under Development by Companies, H1 2016 10
Number of Products under Investigation by Universities/Institutes, H1 2016 11
Comparative Analysis by Clinical Stage Development, H1 2016 12
Comparative Analysis by Early Stage Development, H1 2016 13
Products under Development by Companies, H1 2016 14
Products under Investigation by Universities/Institutes, H1 2016 15
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by CTD Holdings, Inc., H1 2016 16
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by La Jolla Pharmaceutical Company, H1 2016 17
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by Merck & Co., Inc., H1 2016 18
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by Okklo Life Sciences BV, H1 2016 19
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by Orphazyme ApS, H1 2016 20
Assessment by Monotherapy Products, H1 2016 21
Number of Products by Stage and Target, H1 2016 23
Number of Products by Stage and Mechanism of Action, H1 2016 25
Number of Products by Stage and Route of Administration, H1 2016 27
Number of Products by Stage and Molecule Type, H1 2016 29
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C Therapeutics - Recent Pipeline Updates, H1 2016 50
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Dormant Projects, H1 2016 55