This “Phenylketonuria - Pipeline Insight, 2021,” report provides comprehensive insights about 15+ companies and 15+ pipeline drugs in Phenylketonuria pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
Phenylketonuria (PKU) is an inborn error of metabolism that is detectable during the first days of life via routine newborn screening. PKU is inherited in an autosomal recessive pattern. PKU is characterized by absence or deficiency of an enzyme called phenylalanine hydroxylase (PAH), responsible for processing the amino acid phenylalanine. Infants with PKU typically appear normal at birth. With early screening and dietary treatment, affected individuals may never show symptoms of PKU. Symptoms may include vomiting, irritability, and/or a red skin rash with small pimples. Neurological symptoms are present in some untreated patients with PKU, including seizures, abnormal muscle movements, tight muscles, increased reflexes, involuntary movements, or tremor. The goal of treatment for PKU is to keep plasma phenylalanine levels within 120-360 umol/L (2-6 mg/dL). This is generally achieved through carefully planned and monitored diet.
"Phenylketonuria - Pipeline Insight, 2021" report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Phenylketonuria pipeline landscape is provided which includes the disease overview and Phenylketonuria treatment guidelines. The assessment part of the report embraces, in depth Phenylketonuria commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Phenylketonuria collaborations, licensing, mergers and acquisition, funding, designations and other product related details.
The companies and academics are working to assess challenges and seek opportunities that could influence Phenylketonuria R&D. The therapies under development are focused on novel approaches to treat/improve Phenylketonuria.
This segment of the Phenylketonuria report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.
SYNB1618: Synlogic
SYNB1618 is an oral investigational Synthetic Biotic medicine designed to break down phenylalanine in the GI tract as a potential treatment for patients with PKU. A solid oral lyophilized powder formulation was evaluated in a Phase 1 study in healthy volunteers. The drug is currently being evaluated in Phase II stage of development for the treatment of Phenylketonuria.
HMI-102: Homology Medicines
HMI-102 is a gene therapy that uses a harmless adeno-associated viral vector called AAVHSC15, developed from human stem cells, to deliver a functional copy of the PAH gene to cells in the liver. It is designed to produce a lasting effect after a single intravenous (into-the-vein) dose. The drug is in Phase I/II clinical trials for phenylketonuria.
There are approx. 15+ key companies which are developing the therapies for Phenylketonuria. The companies which have their Phenylketonuria drug candidates in the most advanced stage, i.e. Phase II include, Synlogics.
The drugs have been categorized under various product types like Mono, Combination and Mono/Combination.
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Phenylketonuria therapeutic drugs key players involved in developing key drugs.
The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Phenylketonuria drugs.
Geography Covered
- Global coverage
Phenylketonuria Understanding
Phenylketonuria: Overview
Phenylketonuria (PKU) is an inborn error of metabolism that is detectable during the first days of life via routine newborn screening. PKU is inherited in an autosomal recessive pattern. PKU is characterized by absence or deficiency of an enzyme called phenylalanine hydroxylase (PAH), responsible for processing the amino acid phenylalanine. Infants with PKU typically appear normal at birth. With early screening and dietary treatment, affected individuals may never show symptoms of PKU. Symptoms may include vomiting, irritability, and/or a red skin rash with small pimples. Neurological symptoms are present in some untreated patients with PKU, including seizures, abnormal muscle movements, tight muscles, increased reflexes, involuntary movements, or tremor. The goal of treatment for PKU is to keep plasma phenylalanine levels within 120-360 umol/L (2-6 mg/dL). This is generally achieved through carefully planned and monitored diet.
"Phenylketonuria - Pipeline Insight, 2021" report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Phenylketonuria pipeline landscape is provided which includes the disease overview and Phenylketonuria treatment guidelines. The assessment part of the report embraces, in depth Phenylketonuria commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Phenylketonuria collaborations, licensing, mergers and acquisition, funding, designations and other product related details.
Report Highlights
The companies and academics are working to assess challenges and seek opportunities that could influence Phenylketonuria R&D. The therapies under development are focused on novel approaches to treat/improve Phenylketonuria.
Phenylketonuria Emerging Drugs Chapters
This segment of the Phenylketonuria report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.
Phenylketonuria Emerging Drugs
SYNB1618: Synlogic
SYNB1618 is an oral investigational Synthetic Biotic medicine designed to break down phenylalanine in the GI tract as a potential treatment for patients with PKU. A solid oral lyophilized powder formulation was evaluated in a Phase 1 study in healthy volunteers. The drug is currently being evaluated in Phase II stage of development for the treatment of Phenylketonuria.
HMI-102: Homology Medicines
HMI-102 is a gene therapy that uses a harmless adeno-associated viral vector called AAVHSC15, developed from human stem cells, to deliver a functional copy of the PAH gene to cells in the liver. It is designed to produce a lasting effect after a single intravenous (into-the-vein) dose. The drug is in Phase I/II clinical trials for phenylketonuria.
Phenylketonuria: Therapeutic Assessment
This segment of the report provides insights about the different Phenylketonuria drugs segregated based on following parameters that define the scope of the report, such as:
Major Players in Phenylketonuria
There are approx. 15+ key companies which are developing the therapies for Phenylketonuria. The companies which have their Phenylketonuria drug candidates in the most advanced stage, i.e. Phase II include, Synlogics.
Phases
This report covers around 15+ products under different phases of clinical development like
- Late stage products (Phase III)
- Mid-stage products (Phase II)
- Early-stage product (Phase I) along with the details of
- Pre-clinical and Discovery stage candidates
- Discontinued & Inactive candidates
- Route of Administration
Phenylketonuria pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as
- Oral
- Parenteral
- Intravenous
- Subcutaneous
- Topical
- Molecule Type
Products have been categorized under various Molecule types such as
- Monoclonal Antibody
- Peptides
- Polymer
- Small molecule
- Gene therapy
- Product Type
The drugs have been categorized under various product types like Mono, Combination and Mono/Combination.
Phenylketonuria: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Phenylketonuria therapeutic drugs key players involved in developing key drugs.
Pipeline Development Activities
The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Phenylketonuria drugs.
Phenylketonuria Report Insights
- Phenylketonuria Pipeline Analysis
- Therapeutic Assessment
- Unmet Needs
- Impact of Drugs
Phenylketonuria Report Assessment
- Pipeline Product Profiles
- Therapeutic Assessment
- Pipeline Assessment
- Inactive drugs assessment
- Unmet Needs
Key Questions Answered
Current Treatment Scenario and Emerging Therapies:
- How many companies are developing Phenylketonuria drugs?
- How many Phenylketonuria drugs are developed by each company?
- How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Phenylketonuria?
- What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Phenylketonuria therapeutics?
- What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
- What are the clinical studies going on for Phenylketonuria and their status?
- What are the key designations that have been granted to the emerging drugs?
Table of Contents
IntroductionExecutive Summary
Phenylketonuria: Overview
- Causes
- Mechanism of Action
- Signs and Symptoms
- Diagnosis
- Disease Management
Pipeline Therapeutics
- Comparative Analysis
Therapeutic Assessment
- Assessment by Product Type
- Assessment by Stage and Product Type
- Assessment by Route of Administration
- Assessment by Stage and Route of Administration
- Assessment by Molecule Type
- Assessment by Stage and Molecule Type
Phenylketonuria - Analytical Perspective
In-depth Commercial Assessment
- Phenylketonuria companies’ collaborations, Licensing, Acquisition -Deal Value Trends
Phenylketonuria Collaboration Deals
- Company-Company Collaborations (Licensing/Partnering) Analysis
- Company-University Collaborations (Licensing/Partnering) Analysis
Late Stage Products (Phase III)
- Comparative Analysis
P2-VP8: PATH/ SK Biosciences
- Product Description
- Research and Development
- Product Development Activities
Mid Stage Products (Phase II)
- Comparative Analysis
SYNB1618: Synlogic
- Product Description
- Research and Development
- Product Development Activities
Early stage products (Phase I/II)
- Comparative Analysis
HMI-102: Homology Medicines, Inc
- Product Description
- Research and Development
- Product Development Activities
Inactive Products
- Comparative Analysis
Phenylketonuria Key Companies
Phenylketonuria Key Products
Phenylketonuria- Unmet Needs
Phenylketonuria- Market Drivers and Barriers
Phenylketonuria- Future Perspectives and Conclusion
Phenylketonuria Analyst Views
Phenylketonuria Key Companies
AppendixList of Tables
Table 1 Total Products for Phenylketonuria
Table 2 Late Stage Products
Table 3 Mid Stage Products
Table 4 Early Stage Products
Table 5 Pre-clinical & Discovery Stage Products
Table 6 Assessment by Product Type
Table 7 Assessment by Stage and Product Type
Table 8 Assessment by Route of Administration
Table 9 Assessment by Stage and Route of Administration
Table 10 Assessment by Molecule Type
Table 11 Assessment by Stage and Molecule Type
Table 12 Inactive ProductsList of Figures
Figure 1 Total Products for Phenylketonuria
Figure 2 Late Stage Products
Figure 3 Mid Stage Products
Figure 4 Early Stage Products
Figure 5 Preclinical and Discovery Stage Products
Figure 6 Assessment by Product Type
Figure 7 Assessment by Stage and Product Type
Figure 8 Assessment by Route of Administration
Figure 9 Assessment by Stage and Route of Administration
Figure 10 Assessment by Molecule Type
Figure 11 Assessment by Stage and Molecule Type
Figure 12 Inactive Products
Companies Mentioned
A selection of companies mentioned in this report includes:
- Synlogic
- Homology Medicines, Inc
- PTC therapeutics
- Rubius Therapeutics
- NestlÃ
- BioMarin
- SOM Biotech
- Evox Therapeutics
- Moderna Therapeutics
- Orpharma
- Generation Bio
