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Phenylketonuria (PKU)-Pipeline Review, H1 2015

Phenylketonuria (PKU)-Pipeline Review, H1 2015


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Executive Summary

Phenylketonuria (PKU)-Pipeline Review, H1 2015

Summary

Global Markets Direct's, 'Phenylketonuria (PKU)-Pipeline Review, H1 2015', 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

Codexis, Inc. 14

Erytech Pharma SA 15

Promethera Biosciences S.A. 16

SOM Innovation Biotech SL 17

Phenylketonuria (PKU)-Therapeutics Assessment 18

Assessment by Monotherapy Products 18

Assessment by Target 19

Assessment by Route of Administration 20

Assessment by Molecule Type 22

Drug Profiles 24

BMN-165-Drug Profile 24

Product Description 24

Mechanism of Action 24

R&D Progress 24

Cell therapy for Phenylketonuria-Drug Profile 25

Product Description 25

Mechanism of Action 25

R&D Progress 25

HepaStem-Drug Profile 26

Product Description 26

Mechanism of Action 26

R&D Progress 26

Recombinant Enzyme to Replace Phenylalanine Hydroxylase for Phenyketonuria-Drug Profile 28

Product Description 28

Mechanism of Action 28

R&D Progress 28

sapropterin dihydrochloride-Drug Profile 29

Product Description 29

Mechanism of Action 29

R&D Progress 29

SOM-7400-Drug Profile 31

Product Description 31

Mechanism of Action 31

R&D Progress 31

Phenylketonuria (PKU)-Recent Pipeline Updates 32

Phenylketonuria (PKU)-Dormant Projects 35

Phenylketonuria (PKU)-Product Development Milestones 36

Featured News & Press Releases 36

Sep 24, 2014: FDA Receives Paragraph IV Notice Letter for KUVAN (sapropterin dihydrochloride) Tablets 36

Sep 03, 2014: Merck Announces Detailed 26-Week Results from Phase IIIb Study with Kuvan in Children with PKU Below 4 Years of Age 36

Apr 14, 2014: FDA Extends Market Exclusivity Six Months for BioMarin's Rare Disease Therapy KUVAN (sapropterin dihydrochloride) Powder for Oral Solution and Tablets 38

Jun 05, 2013: BioMarin Initiates Phase III Trial For PEG-PAL For Treatment Of Phenylketonuria 39

Feb 19, 2013: BioMarin Announces Kuvan Significantly Improves Inattentiveness In Phenylketonuria Patients 39

Sep 26, 2012: BioMarin Pharma To Initiate Phase III Program For PEG-PAL In Second Quarter Of 2013 40

Jun 30, 2011: Merck Serono Initiates Phase IIIb European Study SPARK In Children Younger Than Four Years, Suffering From Phenylketonuria 42

Mar 18, 2011: BioMarin Announces Preliminary Results From ADAPT Study 42

Sep 01, 2010: BioMarin Presents Results From Investigator-Sponsored Trials On Kuvan At SSIEM Annual Conference 44

Aug 17, 2010: BioMarin Initiates Phase IIIb Study To Evaluate Effects Of Kuvan On Neurophychiatric Symptoms In Subjects With Phenylketonuria 45

Appendix 46

Methodology 46

Coverage 46

Secondary Research 46

Primary Research 46

Expert Panel Validation 46

Contact Us 47

Disclaimer 47

List of Tables

Number of Products under Development for Phenylketonuria (PKU), H1 2015 7

Number of Products under Development for Phenylketonuria (PKU)-Comparative Analysis, H1 2015 8

Number of Products under Development by Companies, H1 2015 9

Comparative Analysis by Late Stage Development, H1 2015 10

Comparative Analysis by Early Stage Development, H1 2015 11

Products under Development by Companies, H1 2015 12

Phenylketonuria (PKU)-Pipeline by BioMarin Pharmaceutical Inc., H1 2015 13

Phenylketonuria (PKU)-Pipeline by Codexis, Inc., H1 2015 14

Phenylketonuria (PKU)-Pipeline by Erytech Pharma SA, H1 2015 15

Phenylketonuria (PKU)-Pipeline by Promethera Biosciences S.A., H1 2015 16

Phenylketonuria (PKU)-Pipeline by SOM Innovation Biotech SL, H1 2015 17

Assessment by Monotherapy Products, H1 2015 18

Number of Products by Stage and Target, H1 2015 19

Number of Products by Stage and Route of Administration, H1 2015 21

Number of Products by Stage and Molecule Type, H1 2015 23

Phenylketonuria (PKU) Therapeutics-Recent Pipeline Updates, H1 2015 32

Phenylketonuria (PKU)-Dormant Projects, H1 2015 35

List of Figures

Number of Products under Development for Phenylketonuria (PKU), H1 2015 7

Number of Products under Development for Phenylketonuria (PKU)-Comparative Analysis, H1 2015 8

Number of Products under Development by Companies, H1 2015 9

Comparative Analysis by Late Stage Development, H1 2015 10

Comparative Analysis by Early Stage Products, H1 2015 11

Assessment by Monotherapy Products, H1 2015 18

Number of Products by Top 10 Routes of Administration, H1 2015 20

Number of Products by Stage and Top 10 Routes of Administration, H1 2015 21

Number of Products by Top 10 Molecule Types, H1 2015 22

Number of Products by Stage and Top 10 Molecule Types, H1 2015 23

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BioMarin Pharmaceutical Inc.

Codexis, Inc.

Erytech Pharma SA

Promethera Biosciences S.A.

SOM Innovation Biotech SL

Phenylketonuria (PKU) Therapeutic Products under Development, Key Players in Phenylketonuria (PKU) Therapeutics, Phenylketonuria (PKU) Pipeline Overview, Phenylketonuria (PKU) Pipeline, Phenylketonuria (PKU) Pipeline Assessment


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