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Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline Review, H1 2018

Published By :

Global Markets Direct

Published Date : Apr 2018

Category :

Pharmaceutical

No. of Pages : 78 Pages

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline Review, H1 2018

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline Review, H1 2018, provides an overview of the Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) 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 (Niemann-Pick Disease) Type C - Pipeline Review, H1 2018, provides comprehensive information on the therapeutics under development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) 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 (Niemann-Pick Disease) 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, 6 and 2 respectively. Similarly, the Universities portfolio in Phase 0, Preclinical and Discovery stages comprises 1, 2 and 2 molecules, respectively.

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) 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 Directs 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 (Niemann-Pick Disease) Type C (Genetic Disorders).
- The pipeline guide reviews pipeline therapeutics for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) 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 (Niemann-Pick Disease) Type C (Genetic Disorders) therapeutics and enlists all their major and minor projects.
- The pipeline guide evaluates Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) 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 (Niemann-Pick Disease) 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 (Niemann-Pick Disease) 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 its most promising pipeline therapeutics.
- Formulate corrective measures for pipeline projects by understanding Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) 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
List of Tables
List of Figures
Introduction
Global Markets Direct Report Coverage
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Overview
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Therapeutics Development
Pipeline Overview
Pipeline by Companies
Pipeline by Universities/Institutes
Products under Development by Companies
Products under Development by Universities/Institutes
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Therapeutics Assessment
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Companies Involved in Therapeutics Development
CTD Holdings Inc
MallInckrodt Plc
Merck & Co Inc
Okklo Life Sciences BV
Orphazyme A/S
Perlara PBC
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Drug Profiles
acetylleucine - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
adrabetadex - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
arimoclomol - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Bryostatin-1 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Delta-tocopherol - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate NPC1 for Niemann-Pick Type C1 Disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
MLSA-1 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Monoclonal Antibodies to Inhibit oxLDL for Metabolic Disorders - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
OKL-1014 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
ORX-301 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
PERL-101 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
RND-001 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecule for Atherosclerosis and Niemann-Pick Type C Disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecules for Niemann-Pick Type C Disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecules to Target Cholesterol for Niemann-Pick Type C Disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Trappsol Cyclo - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
vorinostat - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Dormant Projects
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Product Development Milestones
Featured News & Press Releases
Mar 29, 2018: CTD Holdings Conducts Initial Review of Safety Data for its Phase I and Phase I/II Trials of Trappsol Cyclo to Treat Niemann-Pick Disease Type C
Jan 19, 2018: Arimoclomol For NPC Receives Rare Pediatric Disease Designation
Dec 06, 2017: CTD Holdings Receives Rare Pediatric Disease Designation for Trappsol Cyclo
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
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
Aug 10, 2017: Study Results Published in The Lancet Demonstrate Safety and Efficacy of Intrathecal VTS-270 in Niemann-Pick Disease Type C1
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
Jul 17, 2017: NIH scientists find rare disease clues in cells recycling system
Jul 13, 2017: CTD Holdings to Present at 25th Annual Family Conference of the National Niemann-Pick Disease Foundation
Jun 27, 2017: CTD Holdings Ships Additional Trappsol Cyclo to Brazil
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
Jun 14, 2017: CTD Holdings Announces Appointments of Two Patient Liaisons to Enhance Outreach to Niemann-Pick Disease Type C Families and Caregivers
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
May 11, 2017: Orphazyme Completes Enrolment of Patients For Phase 3 Clinical Trial In Niemann-Pick Type C Disease
Apr 19, 2017: CTD Holdings Corrects Mis-Statements about Its Product Made in On-Line Publication
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer

List of Tables
Table 1: Number of Products under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C, H1 2018
Table 2: Number of Products under Development by Companies, H1 2018
Table 3: Number of Products under Development by Universities/Institutes, H1 2018
Table 4: Products under Development by Companies, H1 2018
Table 5: Products under Development by Universities/Institutes, H1 2018
Table 6: Number of Products by Stage and Target, H1 2018
Table 7: Number of Products by Stage and Mechanism of Action, H1 2018
Table 8: Number of Products by Stage and Route of Administration, H1 2018
Table 9: Number of Products by Stage and Molecule Type, H1 2018
Table 10: Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by CTD Holdings Inc, H1 2018
Table 11: Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by MallInckrodt Plc, H1 2018
Table 12: Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by Merck & Co Inc, H1 2018
Table 13: Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by Okklo Life Sciences BV, H1 2018
Table 14: Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by Orphazyme A/S, H1 2018
Table 15: Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by Perlara PBC, H1 2018
Table 16: Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Dormant Projects, H1 2018

List of Figures
Figure 1: Number of Products under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C, H1 2018
Figure 2: Number of Products under Development by Companies, H1 2018
Figure 3: Number of Products under Development by Universities/Institutes, H1 2018
Figure 4: Number of Products by Top 10 Targets, H1 2018
Figure 5: Number of Products by Stage and Top 10 Targets, H1 2018
Figure 6: Number of Products by Top 10 Mechanism of Actions, H1 2018
Figure 7: Number of Products by Stage and Top 10 Mechanism of Actions, H1 2018
Figure 8: Number of Products by Routes of Administration, H1 2018
Figure 9: Number of Products by Stage and Routes of Administration, H1 2018
Figure 10: Number of Products by Molecule Types, H1 2018
Figure 11: Number of Products by Stage and Molecule Types, H1 2018

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