Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline Review, H1 2020

Global Markets Direct’s, ‘Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline Review, H1 2020’, provides an overview of the Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C and features dormant and discontinued projects.

Global Markets Direct’s 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 Global Markets Direct’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. Drug profiles featured in the report undergoes periodic review following a stringent set of processes to ensure that all the profiles are updated with the latest set of information. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

The report 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.

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

Scope

– The report provides a snapshot of the global therapeutic landscape of Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C

– The report reviews pipeline therapeutics for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C by companies and universities/research institutes based on information derived from company and industry-specific sources

– The report covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages

– The report features descriptive drug profiles for the pipeline products which includes, product description, descriptive MoA, R&D brief, licensing and collaboration details & other developmental activities

– The report reviews key players involved Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C therapeutics and enlists all their major and minor projects

– The report assesses Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C therapeutics based on drug target, mechanism of action (MoA), route of administration (RoA) and molecule type

– The report summarizes all the dormant and discontinued pipeline projects

– The report reviews latest news related to pipeline therapeutics for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C

Reasons to buy

- Gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies

- Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage

- Identify and understand important and diverse types of therapeutics under development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C

- Identify potential new clients or partners in the target demographic

- Develop strategic initiatives by understanding the focus areas of leading companies

- Plan mergers and acquisitions effectively by identifying key players and it’s most promising pipeline therapeutics

- Devise corrective measures for pipeline projects by understanding Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C 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

Companies mentioned

Amicus Therapeutics Inc

Cyclo Therapeutics Inc

E-scape Bio Inc

Endece LLC

Evox Therapeutics Ltd

IntraBio Ltd

Jewell Laboratories LLC

Lauren Sciences LLC

Mallinckrodt Plc

Merck & Co Inc

Neurotrope Bioscience Inc

Okklo Life Sciences BV

Oraxion Therapeutics Inc

Orphazyme A/S

Perlara PBC

Prevacus Inc

SOM Biotech SL

StrideBio Inc

Table of Contents

Table of Contents

Introduction

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Overview

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Therapeutics Development

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

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

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

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

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

Appendix

List of Tables

List of Tables

Number of Products under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C, H1 2020

Number of Products under Development by Companies, H1 2020

Number of Products under Development by Universities/Institutes, H1 2020

Products under Development by Companies, H1 2020

Products under Development by Companies, H1 2020 (Contd..1), H1 2020

Products under Development by Universities/Institutes, H1 2020

Number of Products by Stage and Target, H1 2020

Number of Products by Stage and Mechanism of Action, H1 2020

Number of Products by Stage and Route of Administration, H1 2020

Number of Products by Stage and Molecule Type, H1 2020

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Amicus Therapeutics Inc, H1 2020

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Cyclo Therapeutics Inc, H1 2020

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by E-scape Bio Inc, H1 2020

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Endece LLC, H1 2020

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Evox Therapeutics Ltd, H1 2020

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by IntraBio Ltd, H1 2020

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Jewell Laboratories LLC, H1 2020

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Lauren Sciences LLC, H1 2020

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Mallinckrodt Plc, H1 2020

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Merck & Co Inc, H1 2020

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Neurotrope Bioscience Inc, H1 2020

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Okklo Life Sciences BV, H1 2020

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Oraxion Therapeutics Inc, H1 2020

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Orphazyme A/S, H1 2020

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Perlara PBC, H1 2020

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Prevacus Inc, H1 2020

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by SOM Biotech SL, H1 2020

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by StrideBio Inc, H1 2020

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Dormant Projects, H1 2020

List of Figures

List of Figures

Number of Products under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C, H1 2020

Number of Products under Development by Companies, H1 2020

Number of Products under Development by Universities/Institutes, H1 2020

Number of Products by Top 10 Targets, H1 2020

Number of Products by Stage and Top 10 Targets, H1 2020

Number of Products by Top 10 Mechanism of Actions, H1 2020

Number of Products by Stage and Top 10 Mechanism of Actions, H1 2020

Number of Products by Routes of Administration, H1 2020

Number of Products by Stage and Routes of Administration, H1 2020

Number of Products by Top 10 Molecule Types, H1 2020

Number of Products by Stage and Top 10 Molecule Types, H1 2020

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