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PKU Treatment

Phenylketonuria (PKU)

CommercialActive

Key Facts

Indication

Phenylketonuria (PKU)

Phase

Commercial

Status

Active

Company

Cycle Pharmaceuticals

About Cycle Pharmaceuticals

Cycle Pharmaceuticals is a patient-centric, commercial-stage biopharma company specializing in rare and underserved diseases. It leverages a hybrid model of developing/acquiring treatments and wrapping them in its proprietary Cycle Vita™ patient support ecosystem, which provides clinical, dietary, and access services. The company is actively expanding its U.S. footprint through new product launches and strategic acquisitions, as evidenced by its 2026 tender offer for Applied Therapeutics, positioning it for growth in the high-need, high-value rare disease market.

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Therapeutic Areas

Metabolic Neurological Immunological Oncology

Other Phenylketonuria (PKU) Drugs

Drug	Company	Phase	Status
SYNB1934	Synlogic	Phase 3	Completed; Did Not Meet Primary Endpoint
NB-20X	Nerai Biosciences	Pre-clinical	Active
NGGT002	NGGT	Phase 1/2 (inferred)	Active
SOM1311	SOM Biotech	Preclinical	Active
Repinatrabit	Otsuka	Open-Label Extension	Active
PER303	Perseo Pharma	Preclinical	Active
Engineered Native Bacteria Platform	Endure Biotherapeutics	Pre-clinical	Active
JNT-517	JCR Pharmaceuticals	Phase 1	Active
PALYNZIQ (pegvaliase-pqpz)	BioMarin Pharmaceutical	Approved/Marketed	Active (Label expansion)
PTC923 (sepiapterin)	PTC Therapeutics	Phase 3	Active