Eton Pharmaceuticals announces final readout of PKU Golike clinical trial

Eton Pharmaceuticals (ETON) announced the full readout and compelling results from the clinical trial evaluating PKU Golike as a protein substitute for the treatment of phenylketonuria – PKU – in patients during prolonged fasting periods. The study demonstrated that PKU Golike, administered as the last daily dose and compared to standard amino acid protein substitutes, improved metabolic control by reducing harmful phenylalanine levels and increasing beneficial tyrosine levels, both essential for brain function and metabolic health. PKU patients often experience significant fluctuations in blood Phe levels during prolonged fasting periods, particularly at night, when protein breakdown causes Phe concentrations to peak in the early morning. These fluctuations are associated with cognitive difficulties and overall health impacts, making nighttime metabolic control an important focus in PKU management. At the end of the one-week treatment period, 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 and a statistically significant increase in blood Tyr levels. Compared to baseline levels measured prior to the start of treatment, the PKU Golike group achieved an average 17.8% reduction in blood Phe levels and an average 33.8% increase in blood Tyr levels upon awakening after the overnight fasting period. In comparison, when treated with standard amino acid protein substitutes, the same patients experienced an average 27.6% increase in blood Phe levels and no significant improvement in blood Tyr levels. Eton expects these findings to support the adoption of PKU Golike among healthcare providers and within the PKU community.