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

  • ID: 4412885
  • Report
  • 73 pages
  • Global Markets Direct
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FEATURED COMPANIES

  • CTD Holdings Inc
  • Merck & Co Inc
  • Okklo Life Sciences BV
  • Orphazyme ApS
  • MORE
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline Review, H2 2017

Summary

Our 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

Our 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 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.
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Note: Product cover images may vary from those shown
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FEATURED COMPANIES

  • CTD Holdings Inc
  • Merck & Co Inc
  • Okklo Life Sciences BV
  • Orphazyme ApS
  • MORE
List of Tables

List of Figures

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

Merck & Co Inc

Okklo Life Sciences BV

Orphazyme ApS

Sucampo Pharmaceuticals Inc

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Drug Profiles

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

HPGCD - 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

OR-0005 - 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 Niemann-Pick Type C Disease and Atherosclerosis - 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

VTS-270 - 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

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

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 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

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

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

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

Jan 17, 2017: CTD Holdings Receives FDA Fast Track Designation for Development of Trappsol Cyclo to Treat Niemann-Pick Disease Type C

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

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer

List of Tables

Number of Products under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C, H2 2017

Number of Products under Development by Companies, H2 2017

Number of Products under Development by Universities/Institutes, H2 2017

Products under Development by Companies, H2 2017

Products under Development by Universities/Institutes, H2 2017

Number of Products by Stage and Target, H2 2017

Number of Products by Stage and Mechanism of Action, H2 2017

Number of Products by Stage and Route of Administration, H2 2017

Number of Products by Stage and Molecule Type, H2 2017

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by CTD Holdings Inc, H2 2017

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by Merck & Co Inc, H2 2017

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by Okklo Life Sciences BV, H2 2017

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by Orphazyme ApS, H2 2017

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline by Sucampo Pharmaceuticals Inc, H2 2017

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Dormant Projects, H2 2017

List of Figures

Number of Products under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C, H2 2017

Number of Products under Development by Companies, H2 2017

Number of Products under Development by Universities/Institutes, H2 2017

Number of Products by Top 10 Targets, H2 2017

Number of Products by Stage and Top 10 Targets, H2 2017

Number of Products by Top 10 Mechanism of Actions, H2 2017

Number of Products by Stage and Top 10 Mechanism of Actions, H2 2017

Number of Products by Routes of Administration, H2 2017

Number of Products by Stage and Routes of Administration, H2 2017

Number of Products by Molecule Types, H2 2017

Number of Products by Stage and Molecule Types, H2 2017
Note: Product cover images may vary from those shown
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CTD Holdings Inc
Merck & Co Inc
Okklo Life Sciences BV
Orphazyme ApS
Sucampo Pharmaceuticals Inc
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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