Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline Review, H2 2016

Published: Oct, 2016 | Pages: 72 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline Review, H2 2016

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Acid Sphingomyelinase Deficiency Type C - Pipeline Review, H2 2016, provides an overview of the Acid Sphingomyelinase Deficiency Type C (Genetic Disorders) pipeline landscape.

Niemann-Pick C disease is one of a group of lysosomal storage diseases that affect metabolism and that are caused by genetic mutations. It involves the accumulation of sphingolipids in cells throughout the body, particularly reticuloendothelial cells (the mononuclear phagocyte system). Symptoms include enlarged liver, brain damage, difficulty walking and swallowing, increased sensitivity to touch, difficulty speaking, loss of muscle tone, learning difficulties. 

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Acid Sphingomyelinase Deficiency Type C - Pipeline Review, H2 2016, provides comprehensive information on the therapeutics under development for Acid Sphingomyelinase Deficiency Type C (Genetic Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.

The Acid Sphingomyelinase Deficiency Type C (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase III, Phase II, IND/CTA Filed, Preclinical and Discovery stages are 2, 1, 1, 7 and 1 respectively for Similarly, the Universities portfolio in Preclinical and Discovery stages comprises 2 and 2 molecules, respectively for Acid Sphingomyelinase Deficiency Type C.

Acid Sphingomyelinase Deficiency Type C (Genetic Disorders) pipeline guide 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. The guide 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. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

Scope

- The pipeline guide provides a snapshot of the global therapeutic landscape of Acid Sphingomyelinase Deficiency Type C (Genetic Disorders).
- The pipeline guide reviews pipeline therapeutics for Acid Sphingomyelinase Deficiency Type C (Genetic Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources. 
- The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.
- The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.
- The pipeline guide reviews key companies involved in Acid Sphingomyelinase Deficiency Type C (Genetic Disorders) therapeutics and enlists all their major and minor projects.
- The pipeline guide evaluates Acid Sphingomyelinase Deficiency Type C (Genetic Disorders) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.
- The pipeline guide encapsulates all the dormant and discontinued pipeline projects. 
- The pipeline guide reviews latest news related to pipeline therapeutics for Acid Sphingomyelinase Deficiency Type C (Genetic Disorders)

Reasons To Buy

- Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
- Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.
- Find and recognize significant and varied types of therapeutics under development for Acid Sphingomyelinase Deficiency Type C (Genetic Disorders).
- Classify potential new clients or partners in the target demographic.
- Develop tactical initiatives by understanding the focus areas of leading companies.
- Plan mergers and acquisitions meritoriously by identifying key players and it's most promising pipeline therapeutics.
- Formulate corrective measures for pipeline projects by understanding Acid Sphingomyelinase Deficiency Type C (Genetic Disorders) 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.
- Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.

 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) 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
Late Stage Products 12
Clinical Stage Products 13
Early Stage Products 14
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Products under Development by Companies 15
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Products under Investigation by Universities/Institutes 16
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Companies Involved in Therapeutics Development 17
CTD Holdings, Inc. 17
La Jolla Pharmaceutical Company 18
Merck & Co., Inc. 19
Okklo Life Sciences BV 20
Orphazyme ApS 21
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Therapeutics Assessment 22
Assessment by Monotherapy 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
Antibodies for Metabolic Disorders - Drug Profile 31
Product Description 31
Mechanism Of Action 31
R&D Progress 31
arimoclomol - Drug Profile 32
Product Description 32
Mechanism Of Action 32
R&D Progress 32
Bryostatin-1 - Drug Profile 34
Product Description 34
Mechanism Of Action 34
R&D Progress 34
Delta-tocopherol - Drug Profile 39
Product Description 39
Mechanism Of Action 39
R&D Progress 39
HPGCD - Drug Profile 40
Product Description 40
Mechanism Of Action 40
R&D Progress 40
LJPC-0712 - Drug Profile 41
Product Description 41
Mechanism Of Action 41
R&D Progress 41
MLSA-1 - Drug Profile 42
Product Description 42
Mechanism Of Action 42
R&D Progress 42
OKL-1014 - Drug Profile 43
Product Description 43
Mechanism Of Action 43
R&D Progress 43
OR-0005 - Drug Profile 44
Product Description 44
Mechanism Of Action 44
R&D Progress 44
PERL-101 - Drug Profile 45
Product Description 45
Mechanism Of Action 45
R&D Progress 45
Small Molecule for Niemann-Pick Type C Disease - Drug Profile 46
Product Description 46
Mechanism Of Action 46
R&D Progress 46
Small Molecules for Niemann-Pick Type C Disease - Drug Profile 47
Product Description 47
Mechanism Of Action 47
R&D Progress 47
Small Molecules to Target Cholesterol for Niemann-Pick Type C Disease - Drug Profile 48
Product Description 48
Mechanism Of Action 48
R&D Progress 48
Trappsol Cyclo - Drug Profile 49
Product Description 49
Mechanism Of Action 49
R&D Progress 49
vorinostat - Drug Profile 52
Product Description 52
Mechanism Of Action 52
R&D Progress 52
VTS-270 - Drug Profile 56
Product Description 56
Mechanism Of Action 56
R&D Progress 56
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Dormant Projects 58
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Product Development Milestones 59
Featured News & Press Releases 59
Sep 15, 2016: CTD Holdings Announces MHRA Approval of Clinical Trial Application for Treatment of NPC With Trappsol Cyclo 59
Sep 12, 2016: CTD Holdings Files Clinical Trial Application With Swedish Medical Products Agency to Continue Advancing Its Drug Development Program 60
Sep 06, 2016: CTD Holdings Announces FDA Acceptance of Investigational New Drug Application for Treatment of NPC With Trappsol Cyclo 60
Aug 16, 2016: CTD Holdings Meets With Parent Advocate Groups and Physicians 61
Aug 08, 2016: CTD Holdings Files Investigational New Drug Application With US FDA to Further Advance Its Drug Development Program 62
Aug 01, 2016: CTD Holdings Files Clinical Trial Application with U.K. Regulatory Agency to Advance Its Drug Development Program 63
Jun 28, 2016: CTD Holdings Announces Joint Presentation With UCSF Benioff Children Hospital Oakland on Patient Experience in Compassionate Treatment Program 64
May 03, 2016: CTD Holdings to Present "Management of Niemann-Pick Type C With Intravenous Hydroxypropyl- b -Cyclodextrin" at Ara Parseghian Medical Research Foundation Annual Conference 64
Feb 29, 2016: CTD Holdings Clinical Program to Move Forward in the U.S. 65
Feb 18, 2016: Parent Advocates Join CTD Holdings Family and Physicians Listening Circle on NPC Treatment 66
Dec 14, 2015: CTD Holdings' Clinical Trial Design Receives Positive Responses From U.K.'s Medicines and Healthcare Products Regulatory Agency 66
Sep 16, 2015: N. Scott Fine Named CEO of CTD Holdings 67
Jun 24, 2015: Neurotrope to Conduct Study on Niemann-Pick Type C, a Devastating Rare Pediatric Disease 68
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 69
Jan 27, 2015: FDA Accepts CTD Holdings Drug Master File for Second-Generation Orphan Drug 70
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 Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C, H2 2016 8
Number of Products under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Comparative Analysis, H2 2016 9
Number of Products under Development by Companies, H2 2016 10
Number of Products under Investigation by Universities/Institutes, H2 2016 11
Comparative Analysis by Late Stage Development, H2 2016 12
Comparative Analysis by Clinical Stage Development, H2 2016 13
Comparative Analysis by Early Stage Development, H2 2016 14
Products under Development by Companies, H2 2016 15
Products under Investigation by Universities/Institutes, H2 2016 16
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by CTD Holdings, Inc., H2 2016 17
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by La Jolla Pharmaceutical Company, H2 2016 18
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by Merck & Co., Inc., H2 2016 19
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by Okklo Life Sciences BV, H2 2016 20
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by Orphazyme ApS, H2 2016 21
Assessment by Monotherapy Products, H2 2016 22
Number of Products by Stage and Target, H2 2016 24
Number of Products by Stage and Mechanism of Action, H2 2016 26
Number of Products by Stage and Route of Administration, H2 2016 28
Number of Products by Stage and Molecule Type, H2 2016 30
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Dormant Projects, H2 2016 58