Phenylketonuria (PKU) - Pipeline Review, H2 2014

Published: Dec, 2014 | Pages: 45 | Publisher: Global Markets Direct
Industry: Healthcare | Report Format: Electronic (PDF)

Phenylketonuria (PKU) - Pipeline Review, H2 2014

Summary

Global Markets Direct's, 'Phenylketonuria (PKU) - Pipeline Review, H2 2014', provides an overview of the Phenylketonuria (PKU)'s therapeutic pipeline.

This report provides comprehensive information on the therapeutic development for Phenylketonuria (PKU), 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 Phenylketonuria (PKU) and special features on late-stage 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, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources, put together by Global Markets Direct's team. Drug profiles/records featured in the report undergoes periodic updation 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 Phenylketonuria (PKU)
- 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 Phenylketonuria (PKU) and enlists all their major and minor projects
- The report summarizes all the dormant and discontinued pipeline projects 
- A review of the Phenylketonuria (PKU) 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 Phenylketonuria (PKU) 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 Phenylketonuria (PKU)
- Plan mergers and acquisitions effectively by identifying key players of the most promising pipeline
- Devise corrective measures for pipeline projects by understanding Phenylketonuria (PKU) 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 4
List of Figures 4
Introduction 5
Global Markets Direct Report Coverage 5
Phenylketonuria (PKU) Overview 6
Therapeutics Development 7
Pipeline Products for Phenylketonuria (PKU) - Overview 7
Pipeline Products for Phenylketonuria (PKU) - Comparative Analysis 8
Phenylketonuria (PKU) - Therapeutics under Development by Companies 9
Phenylketonuria (PKU) - Pipeline Products Glance 10
Late Stage Products 10
Early Stage Products 11
Phenylketonuria (PKU) - Products under Development by Companies 12
Phenylketonuria (PKU) - Companies Involved in Therapeutics Development 13
BioMarin Pharmaceutical Inc. 13
Erytech Pharma SA 14
Promethera Biosciences S.A. 15
SOM Innovation Biotech SL 16
Phenylketonuria (PKU) - Therapeutics Assessment 17
Assessment by Monotherapy Products 17
Assessment by Route of Administration 18
Assessment by Molecule Type 20
Drug Profiles 22
BMN-165 - Drug Profile 22
Product Description 22
Mechanism of Action 22
R&D Progress 22
Cell therapy for Phenylketonuria - Drug Profile 24
Product Description 24
Mechanism of Action 24
R&D Progress 24
HepaStem - Drug Profile 25
Product Description 25
Mechanism of Action 25
R&D Progress 25
sapropterin dihydrochloride - Drug Profile 27
Product Description 27
Mechanism of Action 27
R&D Progress 27
SOM-7400 - Drug Profile 29
Product Description 29
Mechanism of Action 29
R&D Progress 29
Phenylketonuria (PKU) - Recent Pipeline Updates 30
Phenylketonuria (PKU) - Dormant Projects 33
Phenylketonuria (PKU) - Product Development Milestones 34
Featured News & Press Releases 34
Sep 24, 2014: FDA Receives Paragraph IV Notice Letter for KUVAN (sapropterin dihydrochloride) Tablets 34
Sep 03, 2014: Merck Announces Detailed 26-Week Results from Phase IIIb Study with Kuvan in Children with PKU Below 4 Years of Age 34
Apr 14, 2014: FDA Extends Market Exclusivity Six Months for BioMarin's Rare Disease Therapy KUVAN (sapropterin dihydrochloride) Powder for Oral Solution and Tablets 36
Jun 05, 2013: BioMarin Initiates Phase III Trial For PEG-PAL For Treatment Of Phenylketonuria 37
Feb 19, 2013: BioMarin Announces Kuvan Significantly Improves Inattentiveness In Phenylketonuria Patients 37
Sep 26, 2012: BioMarin Pharma To Initiate Phase III Program For PEG-PAL In Second Quarter Of 2013 38
Jun 30, 2011: Merck Serono Initiates Phase IIIb European Study SPARK In Children Younger Than Four Years, Suffering From Phenylketonuria 40
Mar 18, 2011: BioMarin Announces Preliminary Results From ADAPT Study 40
Sep 01, 2010: BioMarin Presents Results From Investigator-Sponsored Trials On Kuvan At SSIEM Annual Conference 42
Aug 17, 2010: BioMarin Initiates Phase IIIb Study To Evaluate Effects Of Kuvan On Neurophychiatric Symptoms In Subjects With Phenylketonuria 43
Appendix 44
Methodology 44
Coverage 44
Secondary Research 44
Primary Research 44
Expert Panel Validation 44
Contact Us 45
Disclaimer 45

List of Tables
Number of Products under Development for Phenylketonuria (PKU), H2 2014 7
Number of Products under Development for Phenylketonuria (PKU) - Comparative Analysis, H2 2014 8
Number of Products under Development by Companies, H2 2014 9
Comparative Analysis by Late Stage Development, H2 2014 10
Comparative Analysis by Early Stage Development, H2 2014 11
Products under Development by Companies, H2 2014 12
Phenylketonuria (PKU) - Pipeline by BioMarin Pharmaceutical Inc., H2 2014 13
Phenylketonuria (PKU) - Pipeline by Erytech Pharma SA, H2 2014 14
Phenylketonuria (PKU) - Pipeline by Promethera Biosciences S.A., H2 2014 15
Phenylketonuria (PKU) - Pipeline by SOM Innovation Biotech SL, H2 2014 16
Assessment by Monotherapy Products, H2 2014 17
Number of Products by Stage and Route of Administration, H2 2014 19
Number of Products by Stage and Molecule Type, H2 2014 21
Phenylketonuria (PKU) Therapeutics - Recent Pipeline Updates, H2 2014 30
Phenylketonuria (PKU) - Dormant Projects, H2 2014 33