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

Global Markets Direct’s, ‘Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline Review, H2 2017’, 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

CTD Holdings Inc

Merck & Co Inc

Okklo Life Sciences BV

Orphazyme ApS

Sucampo Pharmaceuticals Inc

Table of Contents

Table of Contents

Table of Contents 2

List of Tables 5

List of Figures 5

Introduction 6

Global Markets Direct Report Coverage 6

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Overview 7

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Therapeutics Development 8

Pipeline Overview 8

Pipeline by Companies 9

Pipeline by Universities/Institutes 10

Products under Development by Companies 11

Products under Development by Universities/Institutes 12

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Therapeutics Assessment 13

Assessment by Target 13

Assessment by Mechanism of Action 15

Assessment by Route of Administration 17

Assessment by Molecule Type 19

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Companies Involved in Therapeutics Development 21

CTD Holdings Inc 21

Merck & Co Inc 21

Okklo Life Sciences BV 22

Orphazyme ApS 22

Sucampo Pharmaceuticals Inc 23

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Drug Profiles 24

arimoclomol - Drug Profile 24

Product Description 24

Mechanism Of Action 24

R&D Progress 24

Bryostatin-1 - Drug Profile 28

Product Description 28

Mechanism Of Action 28

R&D Progress 28

Delta-tocopherol - Drug Profile 34

Product Description 34

Mechanism Of Action 34

R&D Progress 34

Gene Therapy to Activate NPC1 for Niemann-Pick Type C1 Disease - Drug Profile 35

Product Description 35

Mechanism Of Action 35

R&D Progress 35

HPGCD - Drug Profile 36

Product Description 36

Mechanism Of Action 36

R&D Progress 36

MLSA-1 - Drug Profile 37

Product Description 37

Mechanism Of Action 37

R&D Progress 37

Monoclonal Antibodies to Inhibit oxLDL for Metabolic Disorders - Drug Profile 38

Product Description 38

Mechanism Of Action 38

R&D Progress 38

OKL-1014 - Drug Profile 39

Product Description 39

Mechanism Of Action 39

R&D Progress 39

OR-0005 - Drug Profile 40

Product Description 40

Mechanism Of Action 40

R&D Progress 40

PERL-101 - Drug Profile 41

Product Description 41

Mechanism Of Action 41

R&D Progress 41

RND-001 - Drug Profile 42

Product Description 42

Mechanism Of Action 42

R&D Progress 42

Small Molecule for Niemann-Pick Type C Disease and Atherosclerosis - Drug Profile 43

Product Description 43

Mechanism Of Action 43

R&D Progress 43

Small Molecules for Niemann-Pick Type C Disease - Drug Profile 44

Product Description 44

Mechanism Of Action 44

R&D Progress 44

Small Molecules to Target Cholesterol for Niemann-Pick Type C Disease - Drug Profile 45

Product Description 45

Mechanism Of Action 45

R&D Progress 45

Trappsol Cyclo - Drug Profile 46

Product Description 46

Mechanism Of Action 46

R&D Progress 46

vorinostat - Drug Profile 51

Product Description 51

Mechanism Of Action 51

R&D Progress 51

VTS-270 - Drug Profile 56

Product Description 56

Mechanism Of Action 56

R&D Progress 56

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Dormant Projects 60

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Product Development Milestones 61

Featured News & Press Releases 61

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 61

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 62

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 63

Jul 13, 2017: CTD Holdings to Present at 25th Annual Family Conference of the National Niemann-Pick Disease Foundation 63

Jun 27, 2017: CTD Holdings Ships Additional Trappsol Cyclo to Brazil 64

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 65

Jun 14, 2017: CTD Holdings Announces Appointments of Two Patient Liaisons to Enhance Outreach to Niemann-Pick Disease Type C Families and Caregivers 65

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 66

May 11, 2017: Orphazyme Completes Enrolment Of Patients For Phase 3 Clinical Trial In Niemann-Pick Type C Disease 66

Apr 19, 2017: CTD Holdings Corrects Mis-Statements about Its Product Made in On-Line Publication 67

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 68

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 68

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 69

Jan 17, 2017: CTD Holdings Receives FDA Fast Track Designation for Development of Trappsol Cyclo to Treat Niemann-Pick Disease Type C 70

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 71

Appendix 72

Methodology 72

Coverage 72

Secondary Research 72

Primary Research 72

Expert Panel Validation 72

Contact Us 72

Disclaimer 73

List of Tables

List of Tables

Number of Products under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C, H2 2017 8

Number of Products under Development by Companies, H2 2017 9

Number of Products under Development by Universities/Institutes, H2 2017 10

Products under Development by Companies, H2 2017 11

Products under Development by Universities/Institutes, H2 2017 12

Number of Products by Stage and Target, H2 2017 14

Number of Products by Stage and Mechanism of Action, H2 2017 16

Number of Products by Stage and Route of Administration, H2 2017 18

Number of Products by Stage and Molecule Type, H2 2017 20

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by CTD Holdings Inc, H2 2017 21

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Merck & Co Inc, H2 2017 21

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Okklo Life Sciences BV, H2 2017 22

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Orphazyme ApS, H2 2017 22

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Pipeline by Sucampo Pharmaceuticals Inc, H2 2017 23

Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C – Dormant Projects, H2 2017 60

List of Figures

List of Figures

Number of Products under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C, H2 2017 8

Number of Products under Development by Companies, H2 2017 9

Number of Products under Development by Universities/Institutes, H2 2017 10

Number of Products by Top 10 Targets, H2 2017 13

Number of Products by Stage and Top 10 Targets, H2 2017 13

Number of Products by Top 10 Mechanism of Actions, H2 2017 15

Number of Products by Stage and Top 10 Mechanism of Actions, H2 2017 15

Number of Products by Routes of Administration, H2 2017 17

Number of Products by Stage and Routes of Administration, H2 2017 17

Number of Products by Molecule Types, H2 2017 19

Number of Products by Stage and Molecule Types, H2 2017 19

    Pricing

Discounts available for multiple purchases.

reportstore@globalmarketsdirect.com
+44 20 7947 2745

Saved reports