Relief Therapeutics Announces Final Readout of PKU GOLIKE Clinical Trial
Relief Therapeutics (RLFTY) announced final results from a clinical trial evaluating PKU GOLIKE for treating phenylketonuria (PKU). The study demonstrated superior metabolic control during prolonged fasting periods compared to standard amino acid protein substitutes. Key findings showed PKU GOLIKE achieved:
- 17.8% reduction in blood phenylalanine (Phe) levels (P=0.0484)
- 33.8% increase in blood tyrosine (Tyr) levels (P=0.0008)
The randomized, crossover, controlled trial was conducted at Birmingham Children's Hospital, UK, on pediatric patients with classical PKU. The results will be presented at the 2025 ACMG Annual Clinical Genetics Meeting in Los Angeles.
Relief Therapeutics (RLFTY) ha annunciato i risultati finali di uno studio clinico che valuta PKU GOLIKE per il trattamento della fenilchetonuria (PKU). Lo studio ha dimostrato un controllo metabolico superiore durante periodi prolungati di digiuno rispetto ai sostituti proteici standard a base di aminoacidi. Risultati chiave hanno mostrato che PKU GOLIKE ha raggiunto:
- Riduzione del 17,8% dei livelli di fenilalanina (Phe) nel sangue (P=0,0484)
- Aumento del 33,8% dei livelli di tirosina (Tyr) nel sangue (P=0,0008)
Lo studio randomizzato, in crossover e controllato è stato condotto presso il Birmingham Children's Hospital, Regno Unito, su pazienti pediatrici con PKU classica. I risultati saranno presentati alla Conferenza Annuale di Genetica Clinica ACMG del 2025 a Los Angeles.
Relief Therapeutics (RLFTY) anunció los resultados finales de un ensayo clínico que evalúa PKU GOLIKE para el tratamiento de la fenilcetonuria (PKU). El estudio demostró un control metabólico superior durante períodos prolongados de ayuno en comparación con los sustitutos proteicos estándar a base de aminoácidos. Hallazgos clave mostraron que PKU GOLIKE logró:
- Reducción del 17.8% en los niveles de fenilalanina (Phe) en sangre (P=0.0484)
- Aumento del 33.8% en los niveles de tirosina (Tyr) en sangre (P=0.0008)
El ensayo controlado, aleatorizado y cruzado se llevó a cabo en el Birmingham Children's Hospital, Reino Unido, en pacientes pediátricos con PKU clásica. Los resultados se presentarán en la Reunión Anual de Genética Clínica de ACMG 2025 en Los Ángeles.
Relief Therapeutics (RLFTY)는 페닐케톤뇨(PKU) 치료를 위한 PKU GOLIKE의 임상시험 최종 결과를 발표했습니다. 이 연구는 표준 아미노산 단백질 대체물에 비해 장기간 금식 기간 동안 우수한 대사 조절을 보여주었습니다. 주요 발견으로는 PKU GOLIKE가:
- 혈중 페닐알라닌(Phe) 수치를 17.8% 감소시킴 (P=0.0484)
- 혈중 티로신(Tyr) 수치를 33.8% 증가시킴 (P=0.0008)
이 무작위 교차 대조 시험은 영국 버밍엄 아동 병원에서 고전 PKU를 가진 소아 환자를 대상으로 실시되었습니다. 결과는 2025년 로스앤젤레스에서 열리는 ACMG 연례 임상 유전학 회의에서 발표될 예정입니다.
Relief Therapeutics (RLFTY) a annoncé les résultats finaux d'un essai clinique évaluant PKU GOLIKE pour le traitement de la phénylcétonurie (PKU). L'étude a démontré un contrôle métabolique supérieur pendant des périodes de jeûne prolongées par rapport aux substituts de protéines par acides aminés standard. Résultats clés ont montré que PKU GOLIKE a atteint :
- Une réduction de 17,8 % des niveaux de phénylalanine (Phe) dans le sang (P=0,0484)
- Une augmentation de 33,8 % des niveaux de tyrosine (Tyr) dans le sang (P=0,0008)
L'essai contrôlé, randomisé et croisé a été réalisé à l'hôpital pour enfants de Birmingham, au Royaume-Uni, sur des patients pédiatriques atteints de PKU classique. Les résultats seront présentés lors de la réunion annuelle de la Génétique Clinique de l'ACMG en 2025 à Los Angeles.
Relief Therapeutics (RLFTY) hat die endgültigen Ergebnisse einer klinischen Studie zur Evaluierung von PKU GOLIKE zur Behandlung von Phenylketonurie (PKU) bekannt gegeben. Die Studie zeigte eine überlegene metabolische Kontrolle während längerer Fastenperioden im Vergleich zu Standard-Protein-Aminosäure-Ersatzstoffen. Wesentliche Ergebnisse zeigten, dass PKU GOLIKE:
- Eine Reduzierung des Blutpegel von Phenylalanin (Phe) um 17,8% erreichte (P=0,0484)
- Eine Erhöhung des Blutspiegels von Tyrosin (Tyr) um 33,8% erreichte (P=0,0008)
Die randomisierte, kontrollierte Cross-Over-Studie wurde am Birmingham Children's Hospital, UK, mit pädiatrischen Patienten mit klassischer PKU durchgeführt. Die Ergebnisse werden auf der ACMG Jahrestagung für Klinische Genetik 2025 in Los Angeles präsentiert.
- Statistically significant reduction in blood Phe levels (P=0.0002)
- Statistically significant increase in blood Tyr levels (P=0.0113)
- 17.8% reduction in blood Phe levels from baseline
- 33.8% increase in blood Tyr levels from baseline
- None.
Clinical Trial Demonstrates Superior Metabolic Control During Prolonged Fasting in PKU Patients
Results Expected to Promote Awareness and Adoption of PKU GOLIKE ®
GENEVA, SWITZERLAND / ACCESSWIRE / December 16, 2024 / RELIEF THERAPEUTICS Holding SA (SIX:RLF )(OTCQB:RLFTF )(OTCQB:RLFTY ) (Relief, or the Company), a biopharmaceutical company committed to delivering innovative treatment options for select specialty, unmet and rare diseases, today 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 (Phe) levels and increasing beneficial tyrosine (Tyr) 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.
The Company-sponsored, randomized, crossover, controlled clinical study was conducted by the Inherited Metabolic Disorders Unit at Birmingham Children's Hospital, UK, on pediatric patients with classical PKU, the condition's most severe form. The trial compared PKU GOLIKE to standard amino acid protein substitutes in managing metabolic parameters during overnight fasting, the longest fasting period within 24 hours.
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 (P=0.0002) and a statistically significant increase in blood Tyr levels (P=0.0113). Compared to baseline levels measured prior to the start of treatment, the PKU GOLIKE group achieved an average
Highlighting the clinical significance of the findings, Prof. Anita MacDonald, principal investigator and leading dietitian in inherited metabolic disorders at Birmingham Children's Hospital, stated: "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."
These results confirm that PKU GOLIKE's prolonged-release profile provides superior metabolic control during extended fasting periods compared to standard amino acid protein substitutes. The Company expects these findings to support adoption of PKU GOLIKE among healthcare providers and within the PKU community.
The study findings will be presented 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, 2025, in Los Angeles.
For more information on this study (NCT05487378), please visit clinicaltrials.gov .
ABOUT PHENYLKETONURIA
Phenylketonuria (PKU) is a genetic disorder caused by a deficiency of the enzyme needed to break down phenylalanine (Phe), leading to a toxic buildup of Phe from the consumption of foods containing protein or aspartame. Individuals with PKU lack the ability to metabolize Phe, which is present in many foods. Without treatment, PKU can cause severe neurological and developmental issues. The standard treatment involves a lifelong phenylalanine-restricted diet supplemented with amino acid-based, phenylalanine-free medical foods to prevent protein deficiency and optimize metabolic control.
ABOUT PKU GOLIKE ®
PKU GOLIKE products are Foods for Special Medical Purposes (FSMPs) for the dietary management of PKU in children and adults. Developed with Relief's proprietary, patent-protected Physiomimic Technology™ drug delivery platform, PKU GOLIKE products are the first prolonged-release amino acid FSMPs, characterized by a special coating that ensures physiological absorption of the amino acids mirroring that of natural proteins, while also masking the unpleasant taste and odor typically associated with amino acids. PKU GOLIKE products are marketed in the U.S. by Eton Pharmaceuticals Inc. under an exclusive license and supply agreement with Relief, in key European markets by Relief, and in select countries worldwide through licensing and distribution partners.
ABOUT RELIEF
Relief is a commercial-stage biopharmaceutical company committed to advancing treatment paradigms and delivering improvements in efficacy, safety, and convenience to benefit the lives of patients living with select specialty and rare diseases. Relief's portfolio offers a balanced mix of marketed, revenue-generating products, proprietary, globally patented TEHCLO™ and Physiomimic™ platform technologies and a targeted clinical development pipeline consisting of risk-mitigated assets focused in three core therapeutic areas: rare skin diseases, rare metabolic disorders, and rare respiratory diseases. In addition, Relief is commercializing several legacy products via licensing and distribution partners. Headquartered in Geneva, Relief is listed on the SIX Swiss Exchange under the symbol RLF and quoted in the U.S. on OTCQB under the symbols RLFTF and RLFTY. For more information, visit www.relieftherapeutics.com .
CONTACT :
RELIEF THERAPEUTICS Holding SA
Jeremy Meinen
Chief Financial Officer
contact@relieftherapeutics.com
DISCLAIMER
This press release contains forward-looking statements, which may be identified by words such as "believe," "assume," "expect," "intend," "may," "could," "will," or similar expressions. These statements are based on current plans and assumptions and are subject to risks and uncertainties that could cause actual results, financial condition, performance, or achievements to differ materially from those expressed or implied. Such factors include, but are not limited to, changes in economic conditions, market developments, regulatory changes, competitive dynamics, and other risks or changes in circumstances. This communication is provided as of the date hereof, and Relief undertakes no obligation to update any forward-looking statements contained herein as a result of new information, future events or otherwise.
SOURCE: Relief Therapeutics Holding SA
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