Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline Review, H2 2019

“Global Markets Direct’s, ‘Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline Review, H2 2019’, 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

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

Ranedis Pharmaceuticals LLC

SOM Biotech SL”

Table of Contents

Table of Contents

List of Tables

List of Figures

Introduction

Global Markets Direct 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

Amicus Therapeutics Inc

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 for Acid Sphingomyelinase Deficiency Type C - 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

LAUR-501 - 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

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

ursodiol - 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

Oct 05, 2018: CTD Holdings establishes second clinical trial site for us phase I clinical study of Trappsol Cyclo for treatment of Niemann-Pick Disease Type C

Oct 03, 2018: CTD announces encouraging top-line data from two ongoing clinical trials of Trappsol Cyclo administered intravenously in Niemann Pick Disease Type C (NPC)

Sep 28, 2018: Orphazyme reports encouraging arimoclomol clinical trial top-line data in Niemann-Pick disease Type C (NPC)

Sep 13, 2018: CTD Holdings to Present at UK Annual Conference for NPC Patients, Families, and Health Professionals

Aug 30, 2018: CTD Holdings discusses its Phase I clinical trial for intravenous use of Trappsol Cyclo in Niemann-Pick Disease Type C

Aug 23, 2018: CTD Holdings' Trappsol Cyclo featured in television program on Niemann-Pick Disease Type C

Aug 13, 2018: CTD Holdings announces expanded access protocol using Intravenous Trappsol Cyclo for Alzheimer’s disease

Jun 27, 2018: CTD to Present at National Niemann-Pick Disease Foundation Annual Conference

Jun 04, 2018: Orphazyme Presents at 2018 Parseghian Scientific Conference for Niemann-Pick Type C Research

Apr 30, 2018: FDA Authorizes CTD to Proceed with Extension Protocol to US Phase I Trappsol Cyclo Trial for Niemann-Pick Disease Type C

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

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer

List of Tables

“List of Tables

Number of Products under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C, H2 2019

Number of Products under Development by Companies, H2 2019

Number of Products under Development by Universities/Institutes, H2 2019

Products under Development by Companies, H2 2019

Products under Development by Companies, H2 2019 (Contd..1), H2 2019

Products under Development by Universities/Institutes, H2 2019

Number of Products by Stage and Target, H2 2019

Number of Products by Stage and Mechanism of Action, H2 2019

Number of Products by Stage and Route of Administration, H2 2019

Number of Products by Stage and Molecule Type, H2 2019

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Amicus Therapeutics Inc, H2 2019

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Dormant Projects, H2 2019”

List of Figures

“List of Figures

Number of Products under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C, H2 2019

Number of Products under Development by Companies, H2 2019

Number of Products under Development by Universities/Institutes, H2 2019

Number of Products by Targets, H2 2019

Number of Products by Stage and Targets, H2 2019

Number of Products by Mechanism of Actions, H2 2019

Number of Products by Stage and Mechanism of Actions, H2 2019

Number of Products by Routes of Administration, H2 2019

Number of Products by Stage and Routes of Administration, H2 2019

Number of Products by Molecule Types, H2 2019

Number of Products by Stage and Molecule Types, H2 2019”

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