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Phenylketonuria (PKU) (Metabolic Disorder) - Drugs in Development, 2021

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    Drug Pipelines

  • March 2021
  • Region: Global
  • Global Markets Direct
  • ID: 5331255
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The publisher's latest Pharmaceutical and Healthcare disease pipeline guide Phenylketonuria - Drugs in Development, 2021, provides an overview of the Phenylketonuria (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


The publisher's Pharmaceutical and Healthcare latest pipeline guide Phenylketonuria - Drugs in Development, 2021, provides comprehensive information on the therapeutics under development for Phenylketonuria (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 (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, Phase II, Phase I, Preclinical and Discovery stages are 1, 4, 2, 11 and 4 respectively.

Phenylketonuria (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 the publisher’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. 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 (Metabolic Disorders).
  • The pipeline guide reviews pipeline therapeutics for Phenylketonuria (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 (Metabolic Disorders) therapeutics and enlists all their major and minor projects.
  • The pipeline guide evaluates Phenylketonuria (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 (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 (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 it’s most promising pipeline therapeutics.
  • Formulate corrective measures for pipeline projects by understanding Phenylketonuria (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

Introduction
  • 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
  • APR Applied Pharma Research SA
  • BioMarin Pharmaceutical Inc
  • Evox Therapeutics Ltd
  • Generation Bio Co
  • Homology Medicines Inc
  • MipSalus ApS
  • Moderna Inc
  • Nestle Health Science SA
  • Peg-Bio BioPharma Co Ltd
  • Pluvia AS
  • Prosit Sole Biotechnology (Beijing) Co Ltd
  • PTC Therapeutics Inc
  • Sangamo Therapeutics Inc
  • SOM Biotech SL
  • Synlogic Inc
  • Ultragenyx Pharmaceutical Inc
  • Vera Therapeutics Inc

Phenylketonuria (PKU) - Drug Profiles
AGT-324 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress

APROD-031 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress

Biologic for Phenylketonuria - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress

Biologics for Phenylketonuria - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress

BMN-307 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress

CDX-6114 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress

Gene Therapy for Phenylketonuria and Progressive Familial Intrahepatic Cholestasis - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress

HMI-102 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress

HMI-103 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress

mRNA-3283 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress

Oligonucleotides to Activate PAH for Phenylketonuria - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress

pegvaliase - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress

Phelimin - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress

PJ-008 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress

PSP-007 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress

PTC-923 - 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

Small Molecules to Target PAH for Phenylketonuria - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress

SOM-1311 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress

ST-101 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress

SYNB-1618 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress

UX-501 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress

Phenylketonuria (PKU) - Dormant Projects
  • Phenylketonuria (PKU) - Discontinued Products

Phenylketonuria (PKU) - Product Development Milestones
  • Featured News & Press Releases
  • Oct 07, 2020: BioMarin, pioneer in rare disease treatments for Phenylketonuria (PKU), receives FDA approval of label expansion to allow maximum dose of 60 mg for Palynziq (pegvaliase-pqpz) injection for treatment of adults with PKU
  • Mar 26, 2020: FDA grants Orphan Drug Designation to APR-OD031 for the treatment of phenylketonuria
  • Mar 10, 2020: American Gene Technologies to present at 2020 Inborn Errors of Metabolism Drug Development Summit in Boston
  • Dec 11, 2019: Synlogic bridging study data with solid oral formulation of SYNB1618, a synthetic biotic approach to treat Phenylketonuria, demonstrate improved tolerability over early liquid formulation and guide to next stage of clinical development
  • Dec 03, 2019: Censa Pharmaceuticals announces CNSA- 001 met primary and secondary endpoints in phase 2 trial in patients with PKU
  • Sep 04, 2019: Synlogic presents data from phase 1/2a study of SYNB1618 at the Annual Symposium of the Society for the Study of Inborn Errors of Metabolism (SSIEM)
  • Jul 15, 2019: Synlogic reports positive top-line data from phase 1/2a study of SYNB1618 in patients with phenylketonuria and guides to next phase of development
  • May 06, 2019: European Commission approves Palynziq (pegvaliase injection) for treatment of phenylketonuria (PKU) in patients aged 16 years or older
  • Apr 30, 2019: Synlogic presents data describing a Solid Oral Formulation Process for Synthetic Biotic Medicine SYNB1618 for the treatment of PKU at the 22nd Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT)
  • Apr 15, 2019: Synlogic to present data from new solid oral formulation process at the 22nd Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT)
  • Mar 01, 2019: BioMarin Receives Positive CHMP Opinion in Europe for Palynziq (pegvaliase Injection) for Treatment of Patients with Phenylketonuria (PKU) Aged 16 and Older
  • Jan 07, 2019: Retrophin provides update on phenylketonuria drug CNSA-001
  • Jan 07, 2019: BioMarin highlights Palynziq at 37th Annual J.P. Morgan Healthcare Conference in San Francisco
  • Jan 07, 2019: Homology Medicines advances first gene editing development candidate into IND-enabling studies for pediatric patients with phenylketonuria
  • Jan 03, 2019: Synlogic provides update on synthetic biotic medicine SYNB-1618

Appendix
  • Methodology
  • Coverage
  • Secondary Research
  • Primary Research
  • Expert Panel Validation
  • Contact the Publisher
  • Disclaimer

List of Tables
  • Number of Products under Development for Phenylketonuria (PKU), 2021
  • Number of Products under Development by Companies, 2021
  • Products under Development by Companies, 2021
  • Products under Development by Companies, 2021 (Contd..1), 2021
  • Number of Products by Stage and Target, 2021
  • Number of Products by Stage and Mechanism of Action, 2021
  • Number of Products by Stage and Route of Administration, 2021
  • Number of Products by Stage and Molecule Type, 2021
  • Phenylketonuria (PKU) - Pipeline by Agios Pharmaceuticals Inc, 2021
  • Phenylketonuria (PKU) - Pipeline by American Gene Technologies International Inc, 2021
  • Phenylketonuria (PKU) - Pipeline by APR Applied Pharma Research SA, 2021
  • Phenylketonuria (PKU) - Pipeline by BioMarin Pharmaceutical Inc, 2021
  • Phenylketonuria (PKU) - Pipeline by Evox Therapeutics Ltd, 2021
  • Phenylketonuria (PKU) - Pipeline by Generation Bio Co, 2021
  • Phenylketonuria (PKU) - Pipeline by Homology Medicines Inc, 2021
  • Phenylketonuria (PKU) - Pipeline by MipSalus ApS, 2021
  • Phenylketonuria (PKU) - Pipeline by Moderna Inc, 2021
  • Phenylketonuria (PKU) - Pipeline by Nestle Health Science SA, 2021
  • Phenylketonuria (PKU) - Pipeline by Peg-Bio BioPharma Co Ltd, 2021
  • Phenylketonuria (PKU) - Pipeline by Pluvia AS, 2021
  • Phenylketonuria (PKU) - Pipeline by Prosit Sole Biotechnology (Beijing) Co Ltd, 2021
  • Phenylketonuria (PKU) - Pipeline by PTC Therapeutics Inc, 2021
  • Phenylketonuria (PKU) - Pipeline by Sangamo Therapeutics Inc, 2021
  • Phenylketonuria (PKU) - Pipeline by SOM Biotech SL, 2021
  • Phenylketonuria (PKU) - Pipeline by Synlogic Inc, 2021
  • Phenylketonuria (PKU) - Pipeline by Ultragenyx Pharmaceutical Inc, 2021
  • Phenylketonuria (PKU) - Pipeline by Vera Therapeutics Inc, 2021
  • Phenylketonuria (PKU) - Dormant Projects, 2021
  • Phenylketonuria (PKU) - Discontinued Products, 2021

List of Figures
  • Number of Products under Development for Phenylketonuria (PKU), 2021
  • Number of Products under Development by Companies, 2021
  • Number of Products by Top 10 Targets, 2021
  • Number of Products by Stage and Top 10 Targets, 2021
  • Number of Products by Top 10 Mechanism of Actions, 2021
  • Number of Products by Stage and Top 10 Mechanism of Actions, 2021
  • Number of Products by Top 10 Routes of Administration, 2021
  • Number of Products by Stage and Top 10 Routes of Administration, 2021
  • Number of Products by Top 10 Molecule Types, 2021
  • Number of Products by Stage and Top 10 Molecule Types, 2021

Companies Mentioned (Partial List)

A selection of companies mentioned in this report includes, but is not limited to:

  • Agios Pharmaceuticals Inc
  • American Gene Technologies International Inc
  • APR Applied Pharma Research SA
  • BioMarin Pharmaceutical Inc
  • Evox Therapeutics Ltd
  • Generation Bio Co
  • Homology Medicines Inc
  • MipSalus ApS
  • Moderna Inc
  • Nestle Health Science SA
  • Peg-Bio BioPharma Co Ltd
  • Pluvia AS
  • Prosit Sole Biotechnology (Beijing) Co Ltd
  • PTC Therapeutics Inc
  • Sangamo Therapeutics Inc
  • SOM Biotech SL
  • Synlogic Inc
  • Ultragenyx Pharmaceutical Inc
  • Vera Therapeutics Inc