Relief Therapeutics Announces Final Results of PKU GOLIKE Trial

A recent clinical trial has revealed promising outcomes for PKU GOLIKE, a protein substitute developed for individuals with phenylketonuria (PKU), especially during extended fasting periods. Conducted by the Inherited Metabolic Disorders Unit at Birmingham Children's Hospital, UK, the study involved pediatric patients with classical PKU, known as the most severe manifestation of the disorder. This trial was organized by RELIEF THERAPEUTICS, a biopharmaceutical company focusing on pioneering treatments for rare diseases.

PKU GOLIKE was tested against conventional amino acid protein substitutes to evaluate its effectiveness in managing metabolic health during prolonged fasting, particularly overnight. PKU patients often struggle with rising blood phenylalanine (Phe) levels at night due to protein breakdown, leading to morning peaks that can cause cognitive and health issues. Thus, controlling these fluctuations is a critical aspect of managing PKU.

The study employed a randomized, crossover, and controlled design. It demonstrated that PKU GOLIKE, when taken as the last protein substitute of the day, resulted in significant metabolic improvements. Specifically, patients showed a notable reduction in blood Phe levels and an increase in beneficial tyrosine (Tyr) levels, both vital for brain function and overall metabolic balance. The outcomes indicated a statistically significant decrease in blood Phe levels by 17.8% and an increase in blood Tyr levels by 33.8% in the PKU GOLIKE group compared to baseline measurements. In contrast, patients using standard amino acid substitutes experienced an increase in Phe levels and no significant change in Tyr levels.

Blood samples collected at three early morning intervals across the two groups revealed no significant variation in peak Phe levels upon awakening. However, the overall metabolic control was superior in patients using PKU GOLIKE, as highlighted by Prof. Anita MacDonald, the lead investigator and an expert dietitian in metabolic disorders. She noted the consistent metabolic improvements in the patient cohort, who were particularly challenging to manage due to their classical PKU condition.

The results demonstrate that PKU GOLIKE's prolonged-release formulation offers better metabolic control during extended fasting compared to traditional substitutes. The findings are expected to encourage healthcare providers and the PKU community to adopt PKU GOLIKE more widely.

These significant findings are slated for presentation at the 2025 ACMG Annual Clinical Genetics Meeting in Los Angeles. Further details about the study can be accessed through clinicaltrials.gov under the identifier NCT05487378.

Phenylketonuria (PKU) is a genetic disorder resulting from the absence of an enzyme necessary to break down phenylalanine, causing a potentially toxic accumulation of Phe from protein or aspartame intake. Individuals with PKU need lifelong management to prevent severe neurological and developmental issues. Standard treatment involves a Phe-restricted diet supplemented with medical foods to ensure adequate protein intake without Phe.

PKU GOLIKE products are classified as Foods for Special Medical Purposes (FSMPs) for managing PKU in both children and adults. Developed using Relief's proprietary Physiomimic Technology™ drug delivery platform, these products represent the first prolonged-release amino acid FSMPs. They offer physiological absorption similar to natural proteins and mask the unpleasant taste and odor typically associated with amino acids. PKU GOLIKE is marketed in the U.S. through an exclusive agreement with Eton Pharmaceuticals Inc. and in various other regions through Relief's licensing and distribution partners.

Relief Therapeutics, headquartered in Geneva, is dedicated to advancing treatments for rare diseases, focusing on improving efficacy, safety, and convenience. The company maintains a balanced portfolio of marketed products and patented technologies, concentrating on skin, metabolic, and respiratory diseases.