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

  • ID: 4529315
  • Drug Pipelines
  • 78 pages
  • Global Markets Direct
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FEATURED COMPANIES

  • CTD Holdings Inc
  • MallInckrodt Plc
  • Merck & Co Inc
  • Okklo Life Sciences BV
  • Orphazyme A/S
  • Perlara PBC
  • MORE
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline Review, H1 2018

Summary:

This 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:

This 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 the researcher'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 (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 for Purchase:
  • 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 it’s 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.
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Note: Product cover images may vary from those shown
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FEATURED COMPANIES

  • CTD Holdings Inc
  • MallInckrodt Plc
  • Merck & Co Inc
  • Okklo Life Sciences BV
  • Orphazyme A/S
  • Perlara PBC
  • MORE
Introduction

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 C

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 cell’s 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
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  • CTD Holdings Inc
  • MallInckrodt Plc
  • Merck & Co Inc
  • Okklo Life Sciences BV
  • Orphazyme A/S
  • Perlara PBC
Note: Product cover images may vary from those shown
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Note: Product cover images may vary from those shown
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