Eton Pharmaceuticals Announces Final Readout of PKU GOLIKE® Clinical Trial
Eton Pharmaceuticals (ETON) announced compelling results from a clinical trial evaluating PKU GOLIKE for treating phenylketonuria (PKU) during prolonged fasting periods. The randomized, crossover study demonstrated that PKU GOLIKE, when administered as the last daily dose, significantly improved metabolic control compared to standard amino acid protein substitutes.
Key findings showed PKU GOLIKE achieved a 17.8% reduction in blood phenylalanine (Phe) levels and a 33.8% increase in blood tyrosine (Tyr) levels after overnight fasting. In contrast, standard treatments led to a 27.6% increase in Phe levels with no significant Tyr improvement. The study was conducted at Birmingham Children's Hospital, UK, on pediatric patients with classical PKU.
Eton Pharmaceuticals (ETON) ha annunciato risultati eclatanti da uno studio clinico che valuta PKU GOLIKE per il trattamento della fenilchetonuria (PKU) durante periodi di digiuno prolungati. Lo studio randomizzato e crossover ha dimostrato che PKU GOLIKE, somministrato come ultima dose giornaliera, ha migliorato significativamente il controllo metabolico rispetto ai normali sostituti proteici a base di aminoacidi.
I principali risultati hanno mostrato che PKU GOLIKE ha raggiunto una riduzione del 17,8% dei livelli di fenilalanina (Phe) nel sangue e un incremento del 33,8% dei livelli di tirosina (Tyr) nel sangue dopo un digiuno notturno. Al contrario, i trattamenti standard hanno portato a un aumento del 27,6% dei livelli di Phe senza un miglioramento significativo dei livelli di Tyr. Lo studio è stato condotto presso il Birmingham Children's Hospital, Regno Unito, su pazienti pediatrici con PKU classica.
Eton Pharmaceuticals (ETON) anunció resultados convincente de un ensayo clínico que evalúa PKU GOLIKE para el tratamiento de la fenilcetonuria (PKU) durante períodos prolongados de ayuno. El estudio aleatorizado y cruzado demostró que PKU GOLIKE, cuando se administró como la última dosis diaria, mejoró significativamente el control metabólico en comparación con los sustitutos de proteínas de aminoácidos estándar.
Los hallazgos clave mostraron que PKU GOLIKE logró una reducción del 17,8% en los niveles de fenilalanina (Phe) en sangre y un incremento del 33,8% en los niveles de tirosina (Tyr) en sangre después de un ayuno nocturno. En contraste, los tratamientos estándar provocaron un aumento del 27,6% en los niveles de Phe sin una mejora significativa en los niveles de Tyr. El estudio se llevó a cabo en el Hospital Infantil de Birmingham, Reino Unido, en pacientes pediátricos con PKU clásica.
이튼 제약 (ETON)이 장기간 금식 기간 동안 페닐케톤뇨증 (PKU) 치료를 위한 PKU GOLIKE의 임상 시험 결과를 발표했습니다. 무작위 교차 연구에서 PKU GOLIKE를 마지막 하루 복용량으로 투여했을 때, 표준 아미노산 단백질 대체물에 비해 신진대사 조절이 유의미하게 개선되었음을 보여주었습니다.
주요 결과는 PKU GOLIKE가 혈중 페닐알라닌 (Phe) 수치를 17.8% 감소시키고, 혈중 티로신 (Tyr) 수치를 33.8% 증가시켰음을 나타냅니다. 반면, 표준 치료는 Phe 수치를 27.6% 증가시키고 Tyr에 대한 유의미한 개선이 없었습니다. 이 연구는 영국 버밍엄 아동병원에서 고전적인 PKU를 가진 소아 환자를 대상으로 진행되었습니다.
Eton Pharmaceuticals (ETON) a annoncé des résultats convaincants d'un essai clinique évaluant PKU GOLIKE pour le traitement de la phénylcétonurie (PKU) pendant les périodes de jeûne prolongées. L'étude randomisée et croisée a démontré que PKU GOLIKE, lorsqu'il était administré comme dernière dose quotidienne, améliorait considérablement le contrôle métabolique par rapport aux substituts protéiques à base d'acides aminés standard.
Les résultats clés ont montré que PKU GOLIKE avait atteint une réduction de 17,8 % des niveaux de phénylalanine (Phe) dans le sang et une augmentation de 33,8 % des niveaux de tyrosine (Tyr) dans le sang après un jeûne nocturne. En revanche, les traitements standards ont entraîné une augmentation de 27,6 % des niveaux de Phe, sans amélioration significative des niveaux de Tyr. L'étude a été réalisée à l'hôpital pour enfants de Birmingham, au Royaume-Uni, sur des patients pédiatriques atteints de PKU classique.
Eton Pharmaceuticals (ETON) gab beeindruckende Ergebnisse aus einer klinischen Studie bekannt, die PKU GOLIKE zur Behandlung der Phenylketonurie (PKU) während längerer Fastenzeiten bewertet. Die randomisierte, crossover Studie zeigte, dass PKU GOLIKE, wenn es als letzte tägliche Dosis verabreicht wurde, die metabolische Kontrolle im Vergleich zu standardmäßigen Aminosäure-Protein-Ersatzstoffen erheblich verbesserte.
Wichtige Ergebnisse zeigten, dass PKU GOLIKE eine 17,8%ige Reduktion der Blut-Phe (Phenylalanin)-Spiegel und eine 33,8%ige Erhöhung der Blut-Tyr (Tyrosin)-Spiegel nach einer nächtlichen Fastenzeit erreichte. Im Gegensatz dazu führten Standardbehandlungen zu einem Anstieg der Phe-Spiegel um 27,6% ohne signifikante Verbesserung der Tyr-Werte. Die Studie wurde im Birmingham Children's Hospital, UK, an pädiatrischen Patienten mit klassischer PKU durchgeführt.
- Statistically significant reduction in blood Phe levels (P=0.0002)
- 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
- Results demonstrate superior efficacy compared to standard treatments
- No significant differences in peak Phe levels upon reawakening between groups
Insights
The clinical trial results for PKU GOLIKE represent a significant breakthrough in PKU management. The 17.8% reduction in blood Phe levels and 33.8% increase in Tyr levels during overnight fasting demonstrate superior metabolic control compared to standard treatments. This is particularly noteworthy as the study focused on classical PKU, the most severe form of the condition, where metabolic control is especially challenging.
The prolonged-release profile of PKU GOLIKE addresses a critical unmet need in PKU treatment by maintaining better metabolic control during fasting periods. The statistically significant improvements (P=0.0002 for Phe reduction, P=0.0113 for Tyr increase) strongly support its clinical efficacy. These results could lead to improved standard of care for PKU patients and potentially increased market adoption.
This positive clinical data strengthens Eton Pharmaceuticals' market position in the rare disease space. PKU GOLIKE's integration into Eton's existing metabolic sales force, alongside Carglumic Acid, Betaine and Nitisinone products, creates operational synergies and potential cost efficiencies. The insurance coverage for PKU medical formulas and FDA regulation as medical food products provide a clear path to commercialization.
The compelling clinical results should drive healthcare provider adoption and patient uptake, potentially leading to increased market share in the PKU treatment space. For a company with a
Clinical Trial Demonstrates Clinical and Statistical Improvement in Metabolic Control During Prolonged Fasting in PKU Patients - Results Expected to Promote Awareness and Adoption of PKU GOLIKE®
DEER PARK, Ill., Dec. 17, 2024 (GLOBE NEWSWIRE) -- Eton Pharmaceuticals, Inc. (“Eton” or the “Company”) (Nasdaq: ETON), an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases 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 study was sponsored by Relief Therapeutics Holding SA, and was a randomized, crossover, controlled clinical study 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 clinically and statistically significant improvements in metabolic control during extended fasting periods compared to standard amino acid protein substitutes. Eton expects these findings to support the 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 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting, March 18-22, 2025, in Los Angeles, California.
Eton promotes PKU GOLIKE with its existing metabolic sales force, which also promotes Eton’s Carglumic Acid, Betaine, and Nitisinone products. PKU patients’ care is typically overseen by metabolic geneticists and their support staff of nurse practitioners and registered dieticians. Medical formulas for PKU are frequently covered by insurance and are regulated by the FDA as medical food products. Patients and healthcare professionals seeking additional information or requesting a product sample can visit pkugolike.com.
For more information on this study (NCT05487378), please visit clinicaltrials.gov.
About Eton Pharmaceuticals
Eton is an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases. The Company currently has five commercial rare disease products: ALKINDI SPRINKLE®, PKU GOLIKE®, Carglumic Acid, Betaine Anhydrous, and Nitisinone. The Company has three additional product candidates in late-stage development: ET-400, ET-600, and ZENEO® hydrocortisone autoinjector. For more information, please visit our website at etonpharma.com.
About Phenylketonuria (PKU)
Phenylketonuria (PKU) is caused by a defect 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. Untreated PKU can result in global developmental delay or severe irreversible intellectual disability, as well as growth failure, hypopigmentation, motor deficits, ataxia and seizures. Living with PKU requires a limited diet and very careful management. If left unmanaged, PKU can lead to devastating consequences, such as brain damage. People living with PKU do not have the ability to metabolize Phe, which is found in many foods, and they require supplementation of amino acid-based phenylalanine-free medical formulas as part of an effort to prevent protein deficiency and optimize metabolic control. Medical formulas used in PKU are challenged to provide a range of amino acids slowly and without a medicinal aftertaste.
About PKU GOLIKE®
PKU GOLIKE® products are foods for special medical purposes (FSMPs) for the dietary management of PKU in both children and adults for use under medical supervision. 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. The special coating also masks the unpleasant taste, odor, and aftertaste of the amino acids. PKU GOLIKE PLUS® granules are flavorless and can be mixed with many foods. PKU GOLIKE® products contain all 19 amino acids that people with PKU need to maintain neurological and muscular health and PKU GOLIKE PLUS® granules are fortified with 27 essential vitamins and minerals, including ones normally found in protein-rich foods like iron, calcium and vitamin B12. The PKU GOLIKE® line of products are available in convenient packets (PKU GOLIKE PLUS® 3-16 and 16+) and medical formula bars (PKU GOLIKE BAR®). PKU GOLIKE® products have been commercially available in the U.S. since October 2022. For more information, visit pkugolike.com. (Please note this site is intended for U.S. audiences only).
Forward-Looking Statements
Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements associated with the expected ability of Eton to undertake certain activities and accomplish certain goals and objectives. These statements include but are not limited to statements regarding Eton’s business strategy, Eton’s plans to develop and commercialize its product candidates, the safety and efficacy of Eton’s product candidates, Eton’s plans and expected timing with respect to regulatory filings and approvals, and the size and growth potential of the markets for Eton’s product candidates. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as “believes,” “anticipates,” “plans,” “expects,” “intends,” “will,” “goal,” “potential” and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Eton’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. These and other risks concerning Eton’s development programs and financial position are described in additional detail in Eton’s filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made. Eton undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.
Investor Relations:
Lisa M. Wilson, In-Site Communications, Inc.
T: 212-452-2793
E: lwilson@insitecony.com
Source: Eton Pharmaceuticals, Inc.
FAQ
What were the key results of Eton Pharmaceuticals' PKU GOLIKE clinical trial in December 2024?
How does PKU GOLIKE perform compared to standard amino acid substitutes for PKU treatment?
When will ETON present the PKU GOLIKE trial results?
What is the target patient population for ETON's PKU GOLIKE treatment?
