Phenylketonuria Pipeline Shows Rapid Progress in Clinical Trials | DelveInsight

Los Angeles, USA, Dec. 07, 2021 (GLOBE NEWSWIRE) -- Phenylketonuria Pipeline Shows Rapid Progress in Clinical Trials | DelveInsight

There are approximately 15+ key companies developing the Phenylketonuria therapies. In the upcoming years, many new products such as CNSA-001/PTC923 (PTC Therapeutics), HMI-102 (Homology Medicines), and SYNB1618 (Synlogic) are also expected to enter the Phenylketonuria market. PTC-923 is expected to be the most promising drug among all the upcoming therapies.

DelveInsight’s “Phenylketonuria Pipeline Insight” report provides comprehensive insights about 15+ companies and 15+ pipeline drugs in the Phenylketonuria pipeline landscapes. It comprises Phenylketonuria pipeline drug profiles, including clinical and non-clinical stage products. It also includes the Phenylketonuria therapeutics assessment by product type, stage, route of administration, and molecule type and further highlights the inactive Phenylketonuria pipeline products.     

Some of the key takeaways from the Phenylketonuria Pipeline Report  

• Major companies such as Synlogic, Homology Medicines, PTC therapeutics, Rubius Therapeutics, NestlÃ, BioMarin, SOM Biotech, Evox Therapeutics, Moderna Therapeutics, Orpharma, Generation Bio, Erydel, Janana therapeutics, American gene technologies, Applied Pharma Research and others are developing potential drug candidates to improve the Phenylketonuria treatment scenario. 
• HMI-102 is investigational gene therapy in clinical development for the treatment of phenylketonuria (PKU) in adults. Homology has received Fast Track Designation and orphan drug designation from the FDA and orphan drug designation from the European Medicines Agency (EMA). 
• In August 2021, Janana Therapeutics announced the closing of a $50 million Series B financing To Advance Lead PKU Program And Small Molecule Pipeline Based On Next-Gen Chemoproteomic Platform.
• In October 2020, the Food and Drug Administration (FDA) granted Fast Track designation to the investigational gene therapy BMN 307 (BioMarin Pharmaceutical) for the treatment of patients with phenylketonuria (PKU).
• In May 2020, PTC Therapeutics announced that it agreed to acquire Censa Pharmaceuticals, focused on the development of CNSA-001 (sepiapterin), a clinical-stage investigational therapy for orphan metabolic diseases, including phenylketonuria (PKU) and other diseases.
• APR-OD031, being developed by Applied Pharma Research, is an orally-delivered, extended-release amino acid mix engineered with a patented drug delivery technology enabling the secure physiological absorption of the delivered amino acids in the gut. The US FDA has granted Orphan Drug Designation (ODD) to APR-OD031 to treat phenylketonuria (PKU).
• Pfizer is making a $60 million equity investment in the gene therapy company Homology Medicines, developing a "potentially transformational" therapeutic option for phenylketonuria patients.

Get an overview of pipeline landscape @ Phenylketonuria Clinical Trials Analysis 

Phenylketonuria is an inborn error of metabolism that is detectable during the first days of life via routine newborn screening.

Phenylketonuria Emerging Drugs

• Sepiapterin: PTC therapeutics

PTC923 is an oral formulation of synthetic sepiapterin, a precursor to intracellular tetrahydrobiopterin, an enzymatic cofactor involved in the metabolism and synthesis of numerous metabolic products. Results from a Phase II clinical trial of CNSA-001 demonstrated significant and clinically relevant reductions in phenylalanine levels compared to current first-line treatment. The Food and Drug Administration (FDA) has granted Orphan Drug designation to PTC923 to treat patients with hyperphenylalaninemia. In May 2020, PTC Therapeutics announced that it agreed to acquire Censa Pharmaceuticals, focused on developing CNSA-001 (sepiapterin). The drug is currently being evaluated in the Phase III stage of development to treat phenylketonuria.

• 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. Synlogic has determined that SYNB1618 can effectively break down phenylalanine in the GI tract of both healthy volunteers and patients. The drug is currently being evaluated in the Phase II stage of development to treat Phenylketonuria.

• HMI-102: Homology Medicines

HMI-102 is an investigational gene therapy in clinical development to treat phenylketonuria (PKU) in adults. HMI-102 is designed to encode the PAH gene, mutated in people with PKU and delivered via the liver-tropic AAVHSC15 vector. Homology has received Fast Track Designation and orphan drug designation for HMI-102 from the US FDA and orphan drug designation from the European Medicines Agency (EMA). Homology is conducting the Phase II dose expansion portion of a randomised, concurrently controlled, clinical trial (the pheNIX study).

For further information, refer to the detailed report @ Phenylketonuria Pipeline Therapeutics 

Scope of Phenylketonuria Pipeline Drug Insight    

• Coverage: Global 
• Major Players: 15+ Key Players
• Prominent Players: Synlogic, Homology Medicines, PTC therapeutics, Rubius Therapeutics, NestlÃ, BioMarin, SOM Biotech, Evox Therapeutics, Moderna Therapeutics, Orpharma, Generation Bio, Erydel, Janana therapeutics, American gene technologies, Applied Pharma Research and many others.   
• Key Drugs Profiles: 15+ Products
• Phases:  

·       Phenylketonuria Therapies Late-stage (Phase III)  
·       Phenylketonuria Therapies Mid-stage (Phase II)
·       Phenylketonuria Therapies Early-stage (Phase I) 
·       Phenylketonuria Preclinical stage and Discovery candidates     
·       Discontinued and Inactive candidates  

• Mechanism of Action:

·      Phenylalanine ammonia-lyase customised replacements
·      Mixed function oxygenase modulators
·      Gastrointestinal microbiome modulators
·      SLC6A19 protein inhibitors

• Molecule Types:   

·     Peptides
·     Monoclonal antibodies
·    Small molecules 
·    Gene therapy

• Route of Administration:

·     Parenteral 
·      Intravenous
·      Oral
·      Subcutaneous
·      Topical

• Product Types:

·       Monotherapy
·       Combination
·       Mono/Combination 

Key Questions regarding Current Phenylketonuria Treatment Landscape and Emerging Therapies Answered in the Pipeline Report  

• What are the current options for Phenylketonuria treatment?
• How many companies are developing therapies for the treatment of Phenylketonuria? 
• How many are Phenylketonuria emerging therapies in the early-stage, mid-stage, and late development stages to treat Phenylketonuria? 
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, and significant licensing activities that will impact the Phenylketonuria market? 
• Which are the dormant and discontinued products and the reasons for the same?
• What is the unmet need for current therapies for the treatment of Phenylketonuria?  
• What are the current novel therapies, targets, mechanisms of action, and technologies developed to overcome the limitation of existing Phenylketonuria therapies? 
• What are the critical designations that have been granted for the emerging therapies for Phenylketonuria? 
• How many patents are granted and pending for the emerging therapies to treat Phenylketonuria?   

Table of Contents

Get a customised pipeline report @ Phenylketonuria Drugs Pipeline Report  

Other Reports  

• Phenylketonuria (PKU)- Market 
• Phenylketonuria (PKU) - Epidemiology
• Phenylketonuria (PKU) - Global API Manufacturers, Marketed and Phase III Drugs Landscape, 2020

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