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

  • ID: 3495974
  • Report
  • 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 from company/university sites and industry-specific third party sources. Drug profiles/records featured in the report undergoes periodic updates following a stringent set of processes that ensures that all the profiles are updated with the latest set of information. Additionally, processes including live news & deals tracking, browser based alert-box and clinical trials registries tracking ensure that the most recent developments are captured on a real time basis.

The report enhances decision making capabilities and help to create effective counter strategies to gain competitive advantage. It strengthens R&D pipelines by identifying new targets and MOAs to produce first-in-class and best-in-class products.

Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data for the indicated disease.

Scope

- The report provides a snapshot of the global therapeutic landscape of Acid Sphingomyelinase Deficiency (Niemann-Pick Disease)
- The report reviews key pipeline products under drug profile section which includes, product description, MoA and R&D brief, licensing and collaboration details & other developmental activities
- The report reviews key players involved in the therapeutics development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) and enlists all their major and minor projects
- The report summarizes all the dormant and discontinued pipeline projects
- A review of the Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) products under development by companies and universities/research institutes based on information derived from company and industry-specific sources
- Pipeline products coverage based on various stages of development ranging from pre-registration till discovery and undisclosed stages
- A detailed assessment of monotherapy and combination therapy pipeline projects
- Coverage of the Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) pipeline on the basis of target, MoA, route of administration and molecule type
- Latest news and deals relating related to pipeline products

Reasons to buy

- Provides strategically significant competitor information, analysis, and insights to formulate effective R&D development strategies
- Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage
- Develop strategic initiatives by understanding the focus areas of leading companies
- Identify and understand important and diverse types of therapeutics under development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease)
- Plan mergers and acquisitions effectively by identifying key players of the most promising pipeline
- Devise corrective measures for pipeline projects by understanding Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) 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
- Modify the therapeutic portfolio by identifying discontinued projects and understanding the factors that drove them from pipeline
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Note: Product cover images may vary from those shown
2 of 4

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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