Phenylketonuria (PKU) – Pipeline Review, H2 2018

Global Markets Direct’s, ‘Phenylketonuria (PKU) – Pipeline Review, H2 2018’, 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

Agios Pharmaceuticals Inc

American Gene Technologies International Inc

BioMarin Pharmaceutical Inc

Censa Pharmaceuticals Inc

Codexis Inc

Erytech Pharma SA

Homology Medicines, Inc.

MipSalus ApS

Rubius Therapeutics Inc

Synlogic Inc

Synthetic Biologics Inc

Ultragenyx Pharmaceutical Inc

Table of Contents

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

Agios Pharmaceuticals Inc

American Gene Technologies International Inc

BioMarin Pharmaceutical Inc

Censa Pharmaceuticals Inc

Codexis Inc

Erytech Pharma SA

Homology Medicines, Inc.

MipSalus ApS

Rubius Therapeutics Inc

Synlogic Inc

Synthetic Biologics Inc

Ultragenyx Pharmaceutical Inc

Phenylketonuria (PKU) - Drug Profiles

BMN-307 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

CDX-6114 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

CNSA-001 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

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

Gene Therapy for Phenylketonuria - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

HMI-102 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

pegvaliase - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Peptides for Phenylketonuria - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Phelimin - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Recombinant Enzyme for Phenylketonuria - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

RTX-134 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Small Molecules 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

SYN-200 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

SYNB-1618 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Phenylketonuria (PKU) - Dormant Projects

Phenylketonuria (PKU) - Product Development Milestones

Featured News & Press Releases

Jul 09, 2018: Codexis Doses First Subjects in Phase 1a Trial of CDX-6114

May 24, 2018: BioMarin Receives Standard Approval for Palynziq (pegvaliase-pqpz) Injection for Treatment of Adults with Phenylketonuria (PKU), a Rare Genetic Disease

May 18, 2018: Homology Medicines Presents Data from PKU Gene Therapy Program Demonstrating Long-Term Durability of Treatment in IND-Enabling Studies

Apr 30, 2018: Homology Medicines Announces Upcoming Presentations on its Genetic Medicines Platform at the American Society of Gene & Cell Therapy Meeting

Mar 28, 2018: European Medicines Agency Accepts BioMarin's Marketing Application for Pegvaliase MAA for Treatment of Phenylketonuria

Dec 22, 2017: BioMarin Receives Anticipated Notification of PDUFA Extension for Pegvaliase Biologics License Application (BLA) to May 28, 2018

Sep 14, 2017: FDA Not Currently Planning to Hold Advisory Committee Meeting for BioMarins Pegvaliase Biologics License Application (BLA)

Sep 05, 2017: BioMarin To Present Data On Pegvaliase At 13th International Congress of Inborn Errors of Metabolism 2017

Aug 29, 2017: FDA Accepts BioMarins Pegvaliase Biologics License Application and Grants Priority Review Designation

Jul 31, 2017: MipSalus: Promising Results from Preliminary Safety Study

Jun 30, 2017: BioMarin Submits Pegvaliase Biologics License Application to the U.S. FDA for Treatment of Phenylketonuria

Apr 30, 2017: MipSalus: Multi-dose studies with Phelimin show high efficacy and flexibility

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)

Feb 28, 2014: Grant for development of breakthrough treatment of PKU

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer

List of Tables

List of Tables

Number of Products under Development for Phenylketonuria (PKU), H2 2018

Number of Products under Development by Companies, H2 2018

Products under Development by Companies, H2 2018

Number of Products by Stage and Target, H2 2018

Number of Products by Stage and Mechanism of Action, H2 2018

Number of Products by Stage and Route of Administration, H2 2018

Number of Products by Stage and Molecule Type, H2 2018

Phenylketonuria (PKU) – Pipeline by Agios Pharmaceuticals Inc, H2 2018

Phenylketonuria (PKU) – Pipeline by American Gene Technologies International Inc, H2 2018

Phenylketonuria (PKU) – Pipeline by BioMarin Pharmaceutical Inc, H2 2018

Phenylketonuria (PKU) – Pipeline by Censa Pharmaceuticals Inc, H2 2018

Phenylketonuria (PKU) – Pipeline by Codexis Inc, H2 2018

Phenylketonuria (PKU) – Pipeline by Erytech Pharma SA, H2 2018

Phenylketonuria (PKU) – Pipeline by Homology Medicines, Inc., H2 2018

Phenylketonuria (PKU) – Pipeline by MipSalus ApS, H2 2018

Phenylketonuria (PKU) – Pipeline by Rubius Therapeutics Inc, H2 2018

Phenylketonuria (PKU) – Pipeline by Synlogic Inc, H2 2018

Phenylketonuria (PKU) – Pipeline by Synthetic Biologics Inc, H2 2018

Phenylketonuria (PKU) – Pipeline by Ultragenyx Pharmaceutical Inc, H2 2018

Phenylketonuria (PKU) – Dormant Projects, H2 2018

List of Figures

List of Figures

Number of Products under Development for Phenylketonuria (PKU), H2 2018

Number of Products under Development by Companies, H2 2018

Number of Products by Targets, H2 2018

Number of Products by Stage and Targets, H2 2018

Number of Products by Mechanism of Actions, H2 2018

Number of Products by Stage and Mechanism of Actions, H2 2018

Number of Products by Routes of Administration, H2 2018

Number of Products by Stage and Routes of Administration, H2 2018

Number of Products by Molecule Types, H2 2018

Number of Products by Stage and Molecule Types, H2 2018

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