Relief Therapeutics announced final results from a clinical trial evaluating PKU GOLIKE for the treatment of phenylketonuria (PKU). The study demonstrated superior metabolic control during prolonged fasting periods compared to standard amino acid protein substitutes, marking a significant advancement in PKU management.
The randomized, crossover, controlled trial, conducted at Birmingham Children's Hospital, UK, focused on pediatric patients with classical PKU. The results indicated that PKU GOLIKE, when administered as the last daily dose, led to a statistically significant reduction in blood phenylalanine (Phe) levels and an increase in tyrosine (Tyr) levels, both crucial for brain function and metabolic health.
Key Findings of the PKU GOLIKE Trial
At the end of the one-week treatment period, patients receiving PKU GOLIKE showed a statistically significant reduction in blood Phe levels (P=0.0002) and a statistically significant increase in blood Tyr levels (P=0.0113) compared to those receiving standard amino acid substitutes. Specifically, the PKU GOLIKE group achieved an average 17.8% reduction in blood Phe levels (P=0.0484) and an average 33.8% increase in blood Tyr levels (P=0.0008) upon awakening after the overnight fasting period, compared to baseline levels.
In contrast, patients treated with standard amino acid protein substitutes experienced an average 27.6% increase in blood Phe levels (P=0.0063) and no significant improvement in blood Tyr levels. These results underscore the potential of PKU GOLIKE to provide more stable metabolic control, particularly during the critical overnight fasting period.
Clinical Significance and Expert Commentary
Prof. Anita MacDonald, principal investigator and leading dietitian in inherited metabolic disorders at Birmingham Children's Hospital, emphasized the clinical significance of these findings: "Giving one dose of PKU GOLIKE as the final daily dose of protein substitute resulted in consistently better metabolic control in our cohort of patients with PKU... They all had classical PKU and were a particularly challenging group to control."
Implications for PKU Management
These results confirm that PKU GOLIKE's prolonged-release profile offers superior metabolic control during extended fasting periods compared to standard amino acid protein substitutes. The company anticipates that these findings will encourage greater adoption of PKU GOLIKE among healthcare providers and within the PKU community, potentially improving the lives of individuals with PKU by providing a more effective means of managing their condition.
The study findings are scheduled for presentation in a poster titled "A Prolonged-Release Formula Has a Positive Impact on Morning Phenylalanine and Tyrosine Fluctuations in Patients with Classical Phenylketonuria" at the 2025 ACMG Annual Clinical Genetics Meeting, March 18-22, in Los Angeles.
