Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline Review, H2 2017

Published: Oct, 2017 | Pages: 73 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Acid Sphingomyelinase Deficiency Type C - Pipeline Review, H2 2017, 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 2017, 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, Phase I, Preclinical and Discovery stages are 2, 2, 1, 5 and 2 respectively. Similarly, the Universities portfolio in Preclinical and Discovery stages comprises 3 and 2 molecules, respectively.

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
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Therapeutics Development 8
Pipeline Overview 8
Pipeline by Companies 9
Pipeline by Universities/Institutes 10
Products under Development by Companies 11
Products under Development by Universities/Institutes 12
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Therapeutics Assessment 13
Assessment by Target 13
Assessment by Mechanism of Action 15
Assessment by Route of Administration 17
Assessment by Molecule Type 19
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Companies Involved in Therapeutics Development 21
CTD Holdings Inc 21
Merck & Co Inc 21
Okklo Life Sciences BV 22
Orphazyme ApS 22
Sucampo Pharmaceuticals Inc 23
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Drug Profiles 24
arimoclomol - Drug Profile 24
Product Description 24
Mechanism Of Action 24
R&D Progress 24
Bryostatin-1 - Drug Profile 28
Product Description 28
Mechanism Of Action 28
R&D Progress 28
Delta-tocopherol - Drug Profile 34
Product Description 34
Mechanism Of Action 34
R&D Progress 34
Gene Therapy to Activate NPC1 for Niemann-Pick Type C1 Disease - 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
MLSA-1 - Drug Profile 37
Product Description 37
Mechanism Of Action 37
R&D Progress 37
Monoclonal Antibodies to Inhibit oxLDL for Metabolic Disorders - 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
RND-001 - Drug Profile 42
Product Description 42
Mechanism Of Action 42
R&D Progress 42
Small Molecule for Niemann-Pick Type C Disease and Atherosclerosis - Drug Profile 43
Product Description 43
Mechanism Of Action 43
R&D Progress 43
Small Molecules for Niemann-Pick Type C Disease - Drug Profile 44
Product Description 44
Mechanism Of Action 44
R&D Progress 44
Small Molecules to Target Cholesterol for Niemann-Pick Type C Disease - Drug Profile 45
Product Description 45
Mechanism Of Action 45
R&D Progress 45
Trappsol Cyclo - Drug Profile 46
Product Description 46
Mechanism Of Action 46
R&D Progress 46
vorinostat - Drug Profile 51
Product Description 51
Mechanism Of Action 51
R&D Progress 51
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 60
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Product Development Milestones 61
Featured News & Press Releases 61
Sep 28, 2017: CTD Holdings Enrolls First Patient in US Phase I Clinical Trial of Trappsol Cyclo for Treatment of Niemann-Pick Disease Type C 61
Sep 18, 2017: CTD Holdings Announces Approval of Clinical Trial Application to Conduct Phase I/II Clinical Study of Trappsol Cyclo in Patients with Niemann-Pick Type C Disease in Israel 62
Jul 19, 2017: CTD Holdings Announces First Patient Dosed in Phase I/II Clinical Trial to Evaluate Trappsol Cyclo in Niemann-Pick Disease Type C 63
Jul 13, 2017: CTD Holdings to Present at 25th Annual Family Conference of the National Niemann-Pick Disease Foundation 63
Jun 27, 2017: CTD Holdings Ships Additional Trappsol Cyclo to Brazil 64
Jun 21, 2017: CTD Holdings Enrolls First Patient in European Phase I/II Clinical Trial of Trappsol Cyclo for Treatment of Niemann-Pick Disease Type C 65
Jun 14, 2017: CTD Holdings Announces Appointments of Two Patient Liaisons to Enhance Outreach to Niemann-Pick Disease Type C Families and Caregivers 65
May 16, 2017: CTD Holdings to Host Webinar to Provide Update on Trappsol Cyclo Drug Development Program for Treatment of Niemann-Pick Disease Type C 66
May 11, 2017: Orphazyme Completes Enrolment Of Patients For Phase 3 Clinical Trial In Niemann-Pick Type C Disease 66
Apr 19, 2017: CTD Holdings Corrects Mis-Statements about Its Product Made in On-Line Publication 67
Mar 23, 2017: CTD Holdings Initiates Recruitment for U.S. Phase I Clinical Study of Trappsol Cyclo for Treatment of Niemann-Pick Disease Type C 68
Mar 21, 2017: CTD Holdings Initiates Recruitment for Phase I/II Clinical Study in Europe of Trappsol Cyclo for Treatment of Niemann-Pick Disease Type C 68
Feb 16, 2017: CTD Holdings Presents Compassionate Use Data on Trappsol Cyclo to Treat Niemann-Pick Disease Type C and Provides Update on Progress of U.S. and EU Clinical Trials at 13th Annual WORLDSymposium 69
Jan 17, 2017: CTD Holdings Receives FDA Fast Track Designation for Development of Trappsol Cyclo to Treat Niemann-Pick Disease Type C 70
Dec 21, 2016: CTD Holdings Files Clinical Trial Application with Italian Drug Agency to Continue Advancing Trappsol Cyclo Drug Development Program to Treat Niemann-Pick Disease Type C 71
Appendix 72
Methodology 72
Coverage 72
Secondary Research 72
Primary Research 72
Expert Panel Validation 72
Contact Us 72
Disclaimer 73

List of Tables

Number of Products under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C, H2 2017 8
Number of Products under Development by Companies, H2 2017 9
Number of Products under Development by Universities/Institutes, H2 2017 10
Products under Development by Companies, H2 2017 11
Products under Development by Universities/Institutes, H2 2017 12
Number of Products by Stage and Target, H2 2017 14
Number of Products by Stage and Mechanism of Action, H2 2017 16
Number of Products by Stage and Route of Administration, H2 2017 18
Number of Products by Stage and Molecule Type, H2 2017 20
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by CTD Holdings Inc, H2 2017 21
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by Merck & Co Inc, H2 2017 21
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by Okklo Life Sciences BV, H2 2017 22
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by Orphazyme ApS, H2 2017 22
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by Sucampo Pharmaceuticals Inc, H2 2017 23
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Dormant Projects, H2 2017 60