Core Insights - Eton Pharmaceuticals announced positive results from a clinical trial of PKU GOLIKE, a protein substitute for treating phenylketonuria (PKU), showing improved metabolic control during prolonged fasting periods [1][5]. Clinical Trial Details - The study was a randomized, crossover, controlled trial conducted on pediatric patients with classical PKU at Birmingham Children's Hospital, UK, comparing PKU GOLIKE to standard amino acid substitutes [3]. - Results indicated a statistically significant reduction in blood phenylalanine (Phe) levels by an average of 17.8% and an increase in blood tyrosine (Tyr) levels by an average of 33.8% in the PKU GOLIKE group compared to standard substitutes [4]. Clinical Significance - The principal investigator highlighted that administering PKU GOLIKE as the final daily dose led to better metabolic control in patients, confirming its prolonged-release profile's effectiveness [5]. - The findings are expected to support the adoption of PKU GOLIKE among healthcare providers and the PKU community [5]. Product Information - PKU GOLIKE is a food for special medical purposes designed for dietary management of PKU, utilizing a proprietary drug delivery platform that mimics natural protein absorption [12]. - The product line includes various forms, such as flavorless granules and medical formula bars, and has been commercially available in the U.S. since October 2022 [12]. Future Presentation - The study findings will be presented at the 2025 American College of Medical Genetics and Genomics Annual Clinical Genetics Meeting in Los Angeles [6].
Eton Pharmaceuticals Announces Final Readout of PKU GOLIKE® Clinical Trial