Summary
Global Markets Direct’s, ‘Phenylketonuria (PKU) - Pipeline Review, H2 2016’, provides an overview of the Phenylketonuria (PKU) pipeline landscape.
The report provides comprehensive information on the therapeutics under development for Phenylketonuria (PKU), 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 Phenylketonuria (PKU) 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 Phenylketonuria (PKU)
- The report reviews pipeline therapeutics for Phenylketonuria (PKU) 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 Phenylketonuria (PKU) therapeutics and enlists all their major and minor projects
- The report assesses Phenylketonuria (PKU) 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 Phenylketonuria (PKU)
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 Phenylketonuria (PKU)
- 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 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
Companies Mentioned
American Gene Technologies International Inc.
BioMarin Pharmaceutical Inc.
Codexis, Inc.
Dimension Therapeutics, Inc.
Erytech Pharma SA
SOM Innovation Biotech SL
Synthetic Biologics, Inc.
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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
Phenylketonuria (PKU) - Therapeutics under Development by Companies 8
Phenylketonuria (PKU) - Pipeline Products Glance 9
Late Stage Products 9
Early Stage Products 10
Phenylketonuria (PKU) - Products under Development by Companies 11
Phenylketonuria (PKU) - Companies Involved in Therapeutics Development 12
American Gene Technologies International Inc. 12
BioMarin Pharmaceutical Inc. 13
Codexis, Inc. 14
Dimension Therapeutics, Inc. 15
Erytech Pharma SA 16
SOM Innovation Biotech SL 17
Synthetic Biologics, Inc. 18
Phenylketonuria (PKU) - Therapeutics Assessment 19
Assessment by Monotherapy Products 19
Assessment by Target 20
Assessment by Mechanism of Action 22
Assessment by Route of Administration 24
Assessment by Molecule Type 26
Drug Profiles 28
DTX-501 - Drug Profile 28
Product Description 28
Mechanism Of Action 28
R&D Progress 28
Gene Therapy for Phenylketonuria - Drug Profile 29
Product Description 29
Mechanism Of Action 29
R&D Progress 29
pegvaliase - Drug Profile 30
Product Description 30
Mechanism Of Action 30
R&D Progress 30
Proteins to Activate Phenylalanine Hydroxylase for Phenylketonuria - Drug Profile 33
Product Description 33
Mechanism Of Action 33
R&D Progress 33
Recombinant Enzyme for Phenylketonuria - Drug Profile 34
Product Description 34
Mechanism Of Action 34
R&D Progress 34
Recombinant Phenylalanine-4-Hydroxylase Replacement for Phenylketonuria - Drug Profile 35
Product Description 35
Mechanism Of Action 35
R&D Progress 35
Recombinant Phenylalanine-4-Hydroxylase Replacement for Phenylketonuria - Drug Profile 36
Product Description 36
Mechanism Of Action 36
R&D Progress 36
SOM-7400 - Drug Profile 37
Product Description 37
Mechanism Of Action 37
R&D Progress 37
SYN-200 - Drug Profile 38
Product Description 38
Mechanism Of Action 38
R&D Progress 38
SYNB-2010 - Drug Profile 39
Product Description 39
Mechanism Of Action 39
R&D Progress 39
Phenylketonuria (PKU) - Dormant Projects 40
Phenylketonuria (PKU) - Product Development Milestones 41
Featured News & Press Releases 41
Mar 21, 2016: BioMarin Phase 3 Study of Pegvaliase for Phenylketonuria Meets Primary Endpoint of Blood Phenylalanine Reduction (p0.0001) 41
Jun 05, 2013: BioMarin Initiates Phase III Trial For PEG-PAL For Treatment Of Phenylketonuria 43
Sep 26, 2012: BioMarin Pharma To Initiate Phase III Program For PEG-PAL In Second Quarter Of 2013 44
Aug 02, 2010: BioMarin Reports Encouraging Preliminary Safety And Efficacy Trends In Phase II Clinical Study Of PEG-PAL In Phenylketonuria 45
Sep 22, 2009: BioMarin Initiates Phase II Clinical Study Of PEG-PAL In PKU 46
Jun 09, 2009: Results From Phase 1 Clinical Study Of PEG-PAL In PKU And Update On Phase 2 Clinical Study 46
May 20, 2008: BioMarin Initiates Phase 1 Clinical Study Of PEG-PAL In PKU 47
Nov 27, 2007: BioMarin Files Investigational New Drug Application For PEG-PAL For The Treatment Of PKU 48
May 16, 2005: BioMarin And Serono Form Strategic Alliance For The Development And Commercialization Of Phenoptin And Phenylase 48
Appendix 50
Methodology 50
Coverage 50
Secondary Research 50
Primary Research 50
Expert Panel Validation 50
Contact Us 50
Disclaimer 51
List of Tables
Number of Products under Development for Phenylketonuria (PKU), H2 2016 7
Number of Products under Development by Companies, H2 2016 8
Comparative Analysis by Late Stage Development, H2 2016 9
Comparative Analysis by Early Stage Development, H2 2016 10
Products under Development by Companies, H2 2016 11
Phenylketonuria (PKU) - Pipeline by American Gene Technologies International Inc., H2 2016 12
Phenylketonuria (PKU) - Pipeline by BioMarin Pharmaceutical Inc., H2 2016 13
Phenylketonuria (PKU) - Pipeline by Codexis, Inc., H2 2016 14
Phenylketonuria (PKU) - Pipeline by Dimension Therapeutics, Inc., H2 2016 15
Phenylketonuria (PKU) - Pipeline by Erytech Pharma SA, H2 2016 16
Phenylketonuria (PKU) - Pipeline by SOM Innovation Biotech SL, H2 2016 17
Phenylketonuria (PKU) - Pipeline by Synthetic Biologics, Inc., H2 2016 18
Assessment by Monotherapy Products, H2 2016 19
Number of Products by Stage and Target, H2 2016 21
Number of Products by Stage and Mechanism of Action, H2 2016 23
Number of Products by Stage and Route of Administration, H2 2016 25
Number of Products by Stage and Molecule Type, H2 2016 27
Phenylketonuria (PKU) - Dormant Projects, H2 2016 40
List of Figures
Number of Products under Development for Phenylketonuria (PKU), H2 2016 7
Number of Products under Development by Companies, H2 2016 8
Comparative Analysis by Early Stage Products, H2 2016 10
Assessment by Monotherapy Products, H2 2016 19
Number of Products by Targets, H2 2016 20
Number of Products by Stage and Targets, H2 2016 20
Number of Products by Mechanism of Actions, H2 2016 22
Number of Products by Stage and Mechanism of Actions, H2 2016 22
Number of Products by Routes of Administration, H2 2016 24
Number of Products by Stage and Routes of Administration, H2 2016 24
Number of Products by Molecule Types, H2 2016 26
Number of Products by Stage and Molecule Types, H2 2016 26
