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Phenylketonuria (PKU) - Pipeline Review, H1 2017

Published By :

Global Markets Direct

Published Date : Feb 2017

Category :

Pharmaceutical

No. of Pages : 47 Pages

Phenylketonuria (PKU) - Pipeline Review, H1 2017

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Phenylketonuria (PKU) Pipeline Review, H1 2017, provides an overview of the Phenylketonuria (PKU) (Metabolic Disorders) pipeline landscape.

Phenylketonuria (also called PKU), is a rare inherited disorder that causes an amino acid called phenylalanine to build up in body. Symptoms include intellectual disability, psychiatric disorders, hyperactivity, skin rashes, poor bone strength and abnormally small head.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Phenylketonuria (PKU) Pipeline Review, H1 2017, provides comprehensive information on the therapeutics under development for Phenylketonuria (PKU) (Metabolic Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.

The Phenylketonuria (PKU) (Metabolic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Phenylketonuria (PKU) and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase III, Preclinical and Discovery stages are 1, 9 and 1 respectively.

Phenylketonuria (PKU) (Metabolic Disorders) pipeline guide 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. The guide is built using data and information sourced from Global Markets Directs 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. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

Scope

- The pipeline guide provides a snapshot of the global therapeutic landscape of Phenylketonuria (PKU) (Metabolic Disorders).
- The pipeline guide reviews pipeline therapeutics for Phenylketonuria (PKU) (Metabolic Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources.
- The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.
- The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.
- The pipeline guide reviews key companies involved in Phenylketonuria (PKU) (Metabolic Disorders) therapeutics and enlists all their major and minor projects.
- The pipeline guide evaluates Phenylketonuria (PKU) (Metabolic Disorders) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.
- The pipeline guide encapsulates all the dormant and discontinued pipeline projects.
- The pipeline guide reviews latest news related to pipeline therapeutics for Phenylketonuria (PKU) (Metabolic Disorders)

Reasons to buy

- Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
- Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.
- Find and recognize significant and varied types of therapeutics under development for Phenylketonuria (PKU) (Metabolic Disorders).
- Classify potential new clients or partners in the target demographic.
- Develop tactical initiatives by understanding the focus areas of leading companies.
- Plan mergers and acquisitions meritoriously by identifying key players and its most promising pipeline therapeutics.
- Formulate corrective measures for pipeline projects by understanding Phenylketonuria (PKU) (Metabolic Disorders) 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.
- Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.
Table of Contents
List of Tables
List of Figures
Introduction
Global Markets Direct Report Coverage
Phenylketonuria (PKU) - Overview
Phenylketonuria (PKU) - Therapeutics Development
Pipeline Overview
Pipeline by Companies
Products under Development by Companies
Phenylketonuria (PKU) - Therapeutics Assessment
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Phenylketonuria (PKU) - Companies Involved in Therapeutics Development
American Gene Technologies International Inc
BioMarin Pharmaceutical Inc
Codexis Inc
Dimension Therapeutics Inc
Erytech Pharma SA
MipSalus ApS
SOM Biotech SL
Synlogic Inc
Synthetic Biologics Inc
Phenylketonuria (PKU) - Drug Profiles
DTX-501 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy for Phenylketonuria - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
pegvaliase - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Phelimine - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Recombinant Enzyme for Phenylketonuria - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Recombinant Phenylalanine-4-Hydroxylase Replacement for Phenylketonuria - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Recombinant Phenylalanine-4-Hydroxylase Replacement for Phenylketonuria - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecules to Activate Phenylalanine Hydroxylase for Phenylketonuria - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SOM-7400 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SYN-200 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SYNB-2010 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Phenylketonuria (PKU) - Dormant Projects
Phenylketonuria (PKU) - Product Development Milestones
Featured News & Press Releases
Mar 31, 2016: Promising results with breakthrough treatment of orphan disease PKU
Mar 21, 2016: BioMarin Phase 3 Study of Pegvaliase for Phenylketonuria Meets Primary Endpoint of Blood Phenylalanine Reduction (p0.0001)
Jun 05, 2013: BioMarin Initiates Phase III Trial For PEG-PAL For Treatment Of Phenylketonuria
Sep 26, 2012: BioMarin Pharma To Initiate Phase III Program For PEG-PAL In Second Quarter Of 2013
Aug 02, 2010: BioMarin Reports Encouraging Preliminary Safety And Efficacy Trends In Phase II Clinical Study Of PEG-PAL In Phenylketonuria
Sep 22, 2009: BioMarin Initiates Phase II Clinical Study Of PEG-PAL In PKU
Jun 09, 2009: Results From Phase 1 Clinical Study Of PEG-PAL In PKU And Update On Phase 2 Clinical Study
May 20, 2008: BioMarin Initiates Phase 1 Clinical Study Of PEG-PAL In PKU
Nov 27, 2007: BioMarin Files Investigational New Drug Application For PEG-PAL For The Treatment Of PKU
May 16, 2005: BioMarin And Serono Form Strategic Alliance For The Development And Commercialization Of Phenoptin And Phenylase
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer

List of Tables
Number of Products under Development for Phenylketonuria (PKU), H1 2017
Number of Products under Development by Companies, H1 2017
Products under Development by Companies, H1 2017
Number of Products by Stage and Target, H1 2017
Number of Products by Stage and Mechanism of Action, H1 2017
Number of Products by Stage and Route of Administration, H1 2017
Number of Products by Stage and Molecule Type, H1 2017
Phenylketonuria (PKU) - Pipeline by American Gene Technologies International Inc, H1 2017
Phenylketonuria (PKU) - Pipeline by BioMarin Pharmaceutical Inc, H1 2017
Phenylketonuria (PKU) - Pipeline by Codexis Inc, H1 2017
Phenylketonuria (PKU) - Pipeline by Dimension Therapeutics Inc, H1 2017
Phenylketonuria (PKU) - Pipeline by Erytech Pharma SA, H1 2017
Phenylketonuria (PKU) - Pipeline by MipSalus ApS, H1 2017
Phenylketonuria (PKU) - Pipeline by SOM Biotech SL, H1 2017
Phenylketonuria (PKU) - Pipeline by Synlogic Inc, H1 2017
Phenylketonuria (PKU) - Pipeline by Synthetic Biologics Inc, H1 2017
Phenylketonuria (PKU) - Dormant Projects, H1 2017

List of Figures
Number of Products under Development for Phenylketonuria (PKU), H1 2017
Number of Products under Development by Companies, H1 2017
Number of Products by Targets, H1 2017
Number of Products by Stage and Targets, H1 2017
Number of Products by Mechanism of Actions, H1 2017
Number of Products by Stage and Mechanism of Actions, H1 2017
Number of Products by Routes of Administration, H1 2017
Number of Products by Stage and Routes of Administration, H1 2017
Number of Products by Molecule Types, H1 2017
Number of Products by Stage and Molecule Types, H1 2017

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