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Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Pipeline Review, H2 2015

  • ID: 3495974
  • October 2015
  • 61 pages
  • Global Markets Direct
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FEATURED COMPANIES

  • Genzyme Corporation
  • Merck & Co., Inc.
  • Okklo Life Sciences BV
  • Orphazyme ApS
  • MORE

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Pipeline Review, H2 2015

Summary

The report ‘Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Pipeline Review, H2 2015’, provides an overview of the Acid Sphingomyelinase Deficiency (Niemann-Pick Disease)’s therapeutic pipeline.

This report provides comprehensive information on the therapeutic development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease), complete with comparative analysis at various stages, therapeutics assessment by drug target, mechanism of action (MoA), route of administration (RoA) and molecule type, along with latest updates, and featured news and press releases. It also reviews key players involved in the therapeutic development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) and special features on late-stage and discontinued projects.

The report features investigational drugs from across globe covering over 20 therapy areas and nearly 3,000 indications. The report is built using data and information sourced from proprietary databases, Company/University websites, SEC filings, investor presentations and featured press releases READ MORE >

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

  • Genzyme Corporation
  • 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) Overview

Therapeutics Development

Pipeline Products for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Overview

Pipeline Products for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Comparative Analysis

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Therapeutics under Development by Companies

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Therapeutics under Investigation by Universities/Institutes

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Pipeline Products Glance

Clinical Stage Products

Early Stage Products

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Products under Development by Companies

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Products under Investigation by Universities/Institutes

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Companies Involved in Therapeutics Development

Genzyme Corporation

La Jolla Pharmaceutical Company

Merck & Co., Inc.

Okklo Life Sciences BV

Orphazyme ApS

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Therapeutics Assessment

Assessment by Monotherapy Products

Assessment by Target

Assessment by Mechanism of Action

Assessment by Route of Administration

Assessment by Molecule Type

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

LJPC-0712 - Drug Profile

Product Description

Mechanism of Action

R&D Progress

ML-SA1 - Drug Profile

Product Description

Mechanism of Action

R&D Progress

OKL-1014 - Drug Profile

Product Description

Mechanism of Action

R&D Progress

olipudase alfa - Drug Profile

Product Description

Mechanism of Action

R&D Progress

OR-0005 - Drug Profile

Product Description

Mechanism of Action

R&D Progress

Stem Cell Therapy for Cancer, Pain, Retinal Degeneration and Niemann-Pick Disease - 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) - Recent Pipeline Updates

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Dormant Projects

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Product Development Milestones

Featured News & Press Releases

Jun 24, 2015: Neurotrope to Conduct Study on Niemann-Pick Type C, a Devastating Rare Pediatric Disease

Jun 15, 2015: Genzyme Initiates Phase 1/2 Clinical Trial to Evaluate Olipudase Alfa in Pediatric Patients

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

Jun 04, 2015: FDA Grants Breakthrough Therapy Designation for Genzyme’s Olipudase Alfa

Apr 15, 2015: Orphazyme Forms Scientific Advisory Board and Confirms the Enrolment Initiation for the Clinical Programme

Feb 12, 2015: Genzyme Presents Data from its Phase 1b Program for Niemann-Pick Type B at the Lysosomal Disease Network’s WORLD Symposium 2015

Jan 26, 2015: FDA grants orphan drug designation for Arimoclomol

Jan 26, 2015: Orphazyme to present at the Gordon Research Conference on Lysosomal Diseases 15-19 March in Galveston, TX

Oct 20, 2014: COMP issues positive opinion on orphan drug application for Arimoclomol

Oct 06, 2014: Orphazyme in final preparation stages for a Phase II/III clinical trial

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), H2 2015

Number of Products under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Comparative Analysis, H2 2015

Number of Products under Development by Companies, H2 2015

Number of Products under Investigation by Universities/Institutes, H2 2015

Comparative Analysis by Clinical Stage Development, H2 2015

Comparative Analysis by Early Stage Development, H2 2015

Products under Development by Companies, H2 2015

Products under Investigation by Universities/Institutes, H2 2015

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Pipeline by Genzyme Corporation, H2 2015

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Pipeline by La Jolla Pharmaceutical Company, H2 2015

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Pipeline by Merck & Co., Inc., H2 2015

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Pipeline by Okklo Life Sciences BV, H2 2015

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Pipeline by Orphazyme ApS, H2 2015

Assessment by Monotherapy Products, H2 2015

Number of Products by Stage and Target, H2 2015

Number of Products by Stage and Mechanism of Action, H2 2015

Number of Products by Stage and Route of Administration, H2 2015

Number of Products by Stage and Molecule Type, H2 2015

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Therapeutics - Recent Pipeline Updates, H2 2015

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Dormant Projects, H2 2015

List of Figures

Number of Products under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease), H2 2015

Number of Products under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) - Comparative Analysis, H2 2015

Number of Products under Development by Companies, H2 2015

Number of Products under Investigation by Universities/Institutes, H2 2015

Comparative Analysis by Clinical Stage Development, H2 2015

Comparative Analysis by Early Stage Products, H2 2015

Assessment by Monotherapy Products, H2 2015

Number of Products by Top 10 Targets, H2 2015

Number of Products by Stage and Top 10 Targets, H2 2015

Number of Products by Top 10 Mechanism of Actions, H2 2015

Number of Products by Stage and Top 10 Mechanism of Actions, H2 2015

Number of Products by Top 10 Routes of Administration, H2 2015

Number of Products by Stage and Top 10 Routes of Administration, H2 2015

Number of Products by Top 10 Molecule Types, H2 2015

Number of Products by Stage and Top 10 Molecule Types, H2 2015

Note: Product cover images may vary from those shown
3 of 4

Genzyme Corporation
La Jolla Pharmaceutical Company
Merck & Co., Inc.
Okklo Life Sciences BV
Orphazyme ApS

Note: Product cover images may vary from those shown
4 of 4
Note: Product cover images may vary from those shown

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