Prader-Willi Syndrome (PWS) - Pipeline Review, H1 2016

Published: Feb, 2016 | Pages: 70 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

Prader-Willi Syndrome (PWS) - Pipeline Review, H1 2016

Summary


Global Markets Direct's, 'Prader-Willi Syndrome (PWS) - Pipeline Review, H1 2016', provides an overview of the Prader-Willi Syndrome (PWS) pipeline landscape. 

The report provides comprehensive information on the therapeutics under development for Prader-Willi Syndrome (PWS), 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 Prader-Willi Syndrome (PWS) 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 Prader-Willi Syndrome (PWS)
- The report reviews pipeline therapeutics for Prader-Willi Syndrome (PWS) 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 Prader-Willi Syndrome (PWS) therapeutics and enlists all their major and minor projects
- The report assesses Prader-Willi Syndrome (PWS) 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 Prader-Willi Syndrome (PWS)

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 Prader-Willi Syndrome (PWS)
- 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 Prader-Willi Syndrome (PWS) 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

 Table of Contents

Table of Contents 2
List of Tables 5
List of Figures 5
Introduction 6
Global Markets Direct Report Coverage 6
Prader-Willi Syndrome (PWS) Overview 7
Therapeutics Development 8
Pipeline Products for Prader-Willi Syndrome (PWS) - Overview 8
Pipeline Products for Prader-Willi Syndrome (PWS) - Comparative Analysis 9
Prader-Willi Syndrome (PWS) - Therapeutics under Development by Companies 10
Prader-Willi Syndrome (PWS) - Therapeutics under Investigation by Universities/Institutes 11
Prader-Willi Syndrome (PWS) - Pipeline Products Glance 12
Late Stage Products 12
Clinical Stage Products 13
Early Stage Products 14
Prader-Willi Syndrome (PWS) - Products under Development by Companies 15
Prader-Willi Syndrome (PWS) - Products under Investigation by Universities/Institutes 16
Prader-Willi Syndrome (PWS) - Companies Involved in Therapeutics Development 17
Alize Pharma SAS 17
Chong Kun Dang Pharmaceutical Corp. 18
Essentialis, Inc. 19
Ferring International Center S.A. 20
Helsinn Healthcare S.A. 21
LG Life Science LTD. 22
P2D Bioscience 23
Rhythm Pharmaceuticals, Inc. 24
Prader-Willi Syndrome (PWS) - Therapeutics Assessment 25
Assessment by Monotherapy Products 25
Assessment by Target 26
Assessment by Mechanism of Action 28
Assessment by Route of Administration 30
Assessment by Molecule Type 32
Drug Profiles 34
AZP-531 - Drug Profile 34
Product Description 34
Mechanism of Action 34
R&D Progress 34
beloranib - Drug Profile 36
Product Description 36
Mechanism of Action 36
R&D Progress 36
betahistine hydrochloride - Drug Profile 39
Product Description 39
Mechanism of Action 39
R&D Progress 39
carbetocin - Drug Profile 40
Product Description 40
Mechanism of Action 40
R&D Progress 40
diazoxide - Drug Profile 41
Product Description 41
Mechanism of Action 41
R&D Progress 41
diazoxide choline CR - Drug Profile 42
Product Description 42
Mechanism of Action 42
R&D Progress 42
HM-04 - Drug Profile 44
Product Description 44
Mechanism of Action 44
R&D Progress 44
K-671 - Drug Profile 45
Product Description 45
Mechanism of Action 45
R&D Progress 45
setmelanotide - Drug Profile 46
Product Description 46
Mechanism of Action 46
R&D Progress 46
Small Molecule to Activate snRNP-N for Prader-Willi Syndrome - Drug Profile 48
Product Description 48
Mechanism of Action 48
R&D Progress 48
somatropin - Drug Profile 49
Product Description 49
Mechanism of Action 49
R&D Progress 49
Prader-Willi Syndrome (PWS) - Recent Pipeline Updates 50
Prader-Willi Syndrome (PWS) - Dormant Projects 60
Prader-Willi Syndrome (PWS) - Product Development Milestones 61
Featured News & Press Releases 61
Jan 20, 2016: Zafgen's Pivotal Phase 3 Trial of Beloranib in Prader-Willi Syndrome Achieves Co-Primary Efficacy Endpoints 61
Jan 07, 2016: Rhythm Receives Orphan Drug Designation for Setmelanotide for the Treatment of Prader-Willi Syndrome 63
Dec 02, 2015: Zafgen Announces Beloranib IND Placed on Complete Clinical Hold 63
Dec 02, 2015: Zafgen Provides Clinical Update on Beloranib 63
Oct 22, 2015: Zafgen Announces Beloranib Program Update 64
Oct 14, 2015: Zafgen Issues Statement 65
Sep 25, 2015: Essentialis Presents Results From Clinical Study PC025 Evaluating DCCR in the Treatment of Prader-Willi Syndrome at Annual Meeting of the Foundation for Prader-Willi Research 65
Sep 24, 2015: FDA Office of Orphan Products Development Awards Rhythm $1 Million Grant to Support Phase 2 Study of Setmelanotide in Prader-Willi Syndrome 66
Sep 09, 2015: Essentialis Announces the Dosing of the First Prader-Willi Syndrome Patients in a Long-Term Extension to Clinical Trial PC025 67
Jun 04, 2015: Rhythm Initiates Two Phase 2 Clinical Trials of Setmelanotide in Rare Genetic Disorders of Obesity Caused by MC4 Pathway Deficiencies 67
Appendix 69
Methodology 69
Coverage 69
Secondary Research 69
Primary Research 69
Expert Panel Validation 69
Contact Us 69
Disclaimer 70

List of Tables

Number of Products under Development for Prader-Willi Syndrome (PWS), H1 2016 8
Number of Products under Development for Prader-Willi Syndrome (PWS) - Comparative Analysis, H1 2016 9
Number of Products under Development by Companies, H1 2016 10
Number of Products under Investigation by Universities/Institutes, H1 2016 11
Comparative Analysis by Late Stage Development, H1 2016 12
Comparative Analysis by Clinical Stage Development, H1 2016 13
Comparative Analysis by Early Stage Development, H1 2016 14
Products under Development by Companies, H1 2016 15
Products under Investigation by Universities/Institutes, H1 2016 16
Prader-Willi Syndrome (PWS) - Pipeline by Alize Pharma SAS, H1 2016 17
Prader-Willi Syndrome (PWS) - Pipeline by Chong Kun Dang Pharmaceutical Corp., H1 2016 18
Prader-Willi Syndrome (PWS) - Pipeline by Essentialis, Inc., H1 2016 19
Prader-Willi Syndrome (PWS) - Pipeline by Ferring International Center S.A., H1 2016 20
Prader-Willi Syndrome (PWS) - Pipeline by Helsinn Healthcare S.A., H1 2016 21
Prader-Willi Syndrome (PWS) - Pipeline by LG Life Science LTD., H1 2016 22
Prader-Willi Syndrome (PWS) - Pipeline by P2D Bioscience, H1 2016 23
Prader-Willi Syndrome (PWS) - Pipeline by Rhythm Pharmaceuticals, Inc., H1 2016 24
Assessment by Monotherapy Products, H1 2016 25
Number of Products by Stage and Target, H1 2016 27
Number of Products by Stage and Mechanism of Action, H1 2016 29
Number of Products by Stage and Route of Administration, H1 2016 31
Number of Products by Stage and Molecule Type, H1 2016 33
Prader-Willi Syndrome (PWS) Therapeutics - Recent Pipeline Updates, H1 2016 50
Prader-Willi Syndrome (PWS) - Dormant Projects, H1 2016 60