Eton Pharma Announces Final Readout Of PKU GOLIKE Study

Eton Pharmaceuticals

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(RTTNews) - Eton Pharmaceuticals, Inc. (ETON), Tuesday announced final readout from the study of PKU GOLIKE as a protein substitute for the treatment of patients with phenylketonuria (PKU) during prolonged fasting periods.

Phenylketonuria is a genetic disorder that increases the levels of a substance called phenylalanine (Phe) in the blood. Untreated PKU can result in developmental delay, intellectual disability, growth failure, hypopigmentation, motor deficits, ataxia and seizures.

The study, conducted at Birmingham Children's Hospital, UK, on pediatric patients with classical PKU, compared PKU GOLIKE to standard amino acid protein substitutes in managing metabolic parameters during overnight fasting. PKU patients often experience significant increase in blood Phe levels during prolonged fasting periods, particularly at night.

At the end of the one-week, patients receiving PKU GOLIKE as the last daily protein substitute dose showed a statistically significant reduction in blood Phe levels compared to those receiving standard amino acid substitutes.

The Results will be presented at the 2025 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting, March 18-22, 2025, in Los Angeles, California.