DelveInsight’s, “Phenylketonuria Pipeline Insight 2023” report provides comprehensive insights about 18+ companies and 20+ 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.
In the Phenylketonuria Pipeline 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.
Key takeaways from the Phenylketonuria Pipeline Report
- DelveInsight’s Phenylketonuria Pipeline report depicts a robust space with 10+ active players working to develop 15+ pipeline therapies for Phenylketonuria.
- The leading Phenylketonuria Companies include BioMarin Pharmaceutical, Homology Medicine, Jnana Therapeutics, Nestlé Health Science, Moderna, SOM Biotech, Agios Pharmaceuticals, APR Applied Pharma Research, American Gene Technologies, Generation Bio, PTC Therapeutics, and others.
- Promising Phenylketonuria Pipeline Therapies include Kuvan, SYNB1618, SYNB1934, CDX 6114, Sapropterin dihydrochloride, and others.
- The Phenylketonuria Companies and academics are working to assess challenges and seek opportunities that could influence Phenylketonuria R&D. The Phenylketonuria pipeline therapies under development are focused on novel approaches to treat/improve Phenylketonuria.
To explore more information on the latest breakthroughs in the Phenylketonuria Pipeline treatment landscape of the report, click here @ Phenylketonuria Pipeline Outlook
Phenylketonuria Overview
Phenylketonuria as known as PKU is a metabolic disorder that can adversely affect the body’s natural homeostatic or steady state and lead to chemical imbalances and severe pathological conditions. Phenylketonuria is such an example in which the normal conversion of the dietary amino acid phenylalanine to tyrosine is blocked.
Recent Developmental Activities in the Phenylketonuria Treatment Landscape
- 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).
For further information, refer to the detailed Phenylketonuria Unmet Needs, Phenylketonuria Market Drivers, and Phenylketonuria Market Barriers, click here for Phenylketonuria Ongoing Clinical Trial Analysis
Phenylketonuria Emerging Drugs Profile
- Sepiapterin: PTC Therapeutics
Sepiapterin (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. Previous preclinical and healthy volunteer studies showed that an oral dose of CNSA-001 increased target tissue and plasma tetrahydrobiopterin more effectively than equivalent oral doses of sapropterin dihydrochloride. Since sepiapterin is a more stable molecule and is transported more efficiently across cells, it has the potential to be more effective than Kuvan in boosting PAH activity, reducing phenylalanine levels, and lessening symptoms in PKU patients. In May 2020, PTC Therapeutics announced that it agreed to acquire Censa Pharmaceuticals, focused on developing CNSA-001 (sepiapterin). Sepiapterin is currently being investigated in Phase III stage of development for the treatment of Phenylketonuria.
- SYNB1618: Synlogic
SYNB1618 is an orally administered Synthetic BioticTM medicines being developed as potential treatments for phenylketonuria (PKU). It is an engineered strain of the microorganism E. coli Nissle that encodes phenylalanine ammonia lyase (PAL), an enzyme that breaks down Phe. It is intended to address the needs of patients of all age groups through the consumption of Phe in the gastrointestinal tract, which has the potential to lower blood Phe levels and enable the consumption of more natural protein in the diet. SYNB1618 has received both Orphan Drug and Fast Track designations by the US Food and Drug Administration (FDA) and orphan medicinal product designation by the European Medicines Agency. It is currently being evaluated in Phase II clinical trial to treat patients with phenylketonuria.
- HMI-102: Homology Medicine
HMI-102 is an investigational gene therapy in clinical development for the treatment of phenylketonuria (PKU) in adults. HMI-102 is designed to encode the PAH gene, which is 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 U.S. Food and Drug Administration (FDA), and orphan drug designation from the European Medicines Agency (EMA). The company began the Phase I/II study of HMI-102 in 2019. The FDA held back the study over elevated liver function tests by a few months in 2022. In August 2022, the Company paused enrollment in the pheNIX gene therapy trial evaluating HMI-102, however the drug is currently active in the company’s pipeline in Phase I/II stage of development.
Phenylketonuria Pipeline Therapeutics Assessment
There are approx. 18+ 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 III include, PTC Therapeutics.
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Scope of the Phenylketonuria Pipeline Report
- Coverage- Global
- Phenylketonuria Companies- BioMarin Pharmaceutical, Homology Medicine, Jnana Therapeutics, Nestlé Health Science, Moderna, SOM Biotech, Agios Pharmaceuticals, APR Applied Pharma Research, American Gene Technologies, Generation Bio, PTC Therapeutics, and others.
- Phenylketonuria Pipeline Therapies- Kuvan, SYNB1618, SYNB1934, CDX 6114, Sapropterin dihydrochloride, and others.
- Phenylketonuria Pipeline Segmentation: Product Type, Molecule Type, Mechanism of Action, Route of Administration
Dive deep into rich insights for drugs for Phenylketonuria Market Drivers and Phenylketonuria Market Barriers, click here @ Phenylketonuria Unmet Needs and Analyst Views
Table of Content
- Introduction
- Executive Summary
- Phenylketonuria: Overview
- Pipeline Therapeutics
- Therapeutic Assessment
- Phenylketonuria– DelveInsight’s Analytical Perspective
- Late Stage Products (Phase III)
- Sepiapterin: PTC Therapeutics
- Drug profiles in the detailed report…..
- Mid Stage Products (Phase II)
- SYNB1618: Synlogic
- Drug profiles in the detailed report…..
- Early Stage Products (Phase I)
- Drug name : Company name
- Drug profiles in the detailed report…..
- Preclinical and Discovery Stage Products
- Drug name : Company name
- Drug profiles in the detailed report…..
- Inactive Products
- 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
- Appendix
Got Queries? Find out the related information on Phenylketonuria Mergers and acquisitions, Phenylketonuria Licensing Activities @ Phenylketonuria Emerging Drugs, and Recent Trends
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