Eton Pharmaceuticals Announces Final Readout of PKU GOLIKE庐 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 猫 stato condotto presso il Birmingham Children's Hospital, Regno Unito, su pazienti pediatrici con PKU classica.
Eton Pharmaceuticals (ETON) anunci贸 resultados convincente de un ensayo cl铆nico que eval煤a PKU GOLIKE para el tratamiento de la fenilcetonuria (PKU) durante per铆odos prolongados de ayuno. El estudio aleatorizado y cruzado demostr贸 que PKU GOLIKE, cuando se administr贸 como la 煤ltima dosis diaria, mejor贸 significativamente el control metab贸lico en comparaci贸n con los sustitutos de prote铆nas de amino谩cidos est谩ndar.
Los hallazgos clave mostraron que PKU GOLIKE logr贸 una reducci贸n 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 despu茅s de un ayuno nocturno. En contraste, los tratamientos est谩ndar provocaron un aumento del 27,6% en los niveles de Phe sin una mejora significativa en los niveles de Tyr. El estudio se llev贸 a cabo en el Hospital Infantil de Birmingham, Reino Unido, en pacientes pedi谩tricos con PKU cl谩sica.
鞚错娂 鞝滌暯 (ETON)鞚� 鞛リ赴臧� 旮堨嫕 旮瓣皠 霃欖晥 韼橂嫄旒�韱る嚚歃� (PKU) 旃橂毳� 鞙勴暅 PKU GOLIKE鞚� 鞛勳儊 鞁滍棙 瓴瓣臣毳� 氚滍憸頄堨姷雼堧嫟. 氍挫瀾鞙� 甑愳皑 鞐瓣惮鞐愳劀 PKU GOLIKE毳� 毵堨毵� 頃橂（ 氤奠毄霟夓溂搿� 韴棳頄堨潉 霑�, 響滌 鞎勲雲胳偘 雼氨歆� 雽�觳措鞐� 牍勴暣 鞁犾雽�靷� 臁办爤鞚� 鞙犾潣氙疙晿瓴� 臧滌劆霅橃棃鞚岇潉 氤挫棳欤检棃鞀惦媹雼�.
欤检殧 瓴瓣臣電� PKU GOLIKE臧� 順堨 韼橂嫄鞎岆澕雼� (Phe) 靾橃箻毳� 17.8% 臧愳唽鞁滍偆瓿�, 順堨 韹半鞁� (Tyr) 靾橃箻毳� 33.8% 歃濌皜鞁滌及鞚岇潉 雮橅儉雰呺媹雼�. 氚橂┐, 響滌 旃橂電� Phe 靾橃箻毳� 27.6% 歃濌皜鞁滍偆瓿� Tyr鞐� 雽�頃� 鞙犾潣氙疙暅 臧滌劆鞚� 鞐嗢棃鞀惦媹雼�. 鞚� 鞐瓣惮電� 鞓侁淡 氩勲皪鞐� 鞎勲彊氤戩洂鞐愳劀 瓿犾爠鞝侅澑 PKU毳� 臧�歆� 靻岇晞 頇橃瀽毳� 雽�靸侅溂搿� 歆勴枆霅橃棃鞀惦媹雼�.
Eton Pharmaceuticals (ETON) a annonc茅 des r茅sultats convaincants d'un essai clinique 茅valuant PKU GOLIKE pour le traitement de la ph茅nylc茅tonurie (PKU) pendant les p茅riodes de je没ne prolong茅es. L'茅tude randomis茅e et crois茅e a d茅montr茅 que PKU GOLIKE, lorsqu'il 茅tait administr茅 comme derni猫re dose quotidienne, am茅liorait consid茅rablement le contr么le m茅tabolique par rapport aux substituts prot茅iques 脿 base d'acides amin茅s standard.
Les r茅sultats cl茅s ont montr茅 que PKU GOLIKE avait atteint une r茅duction de 17,8 % des niveaux de ph茅nylalanine (Phe) dans le sang et une augmentation de 33,8 % des niveaux de tyrosine (Tyr) dans le sang apr猫s un je没ne nocturne. En revanche, les traitements standards ont entra卯n茅 une augmentation de 27,6 % des niveaux de Phe, sans am茅lioration significative des niveaux de Tyr. L'茅tude a 茅t茅 r茅alis茅e 脿 l'h么pital pour enfants de Birmingham, au Royaume-Uni, sur des patients p茅diatriques atteints de PKU classique.
Eton Pharmaceuticals (ETON) gab beeindruckende Ergebnisse aus einer klinischen Studie bekannt, die PKU GOLIKE zur Behandlung der Phenylketonurie (PKU) w盲hrend l盲ngerer Fastenzeiten bewertet. Die randomisierte, crossover Studie zeigte, dass PKU GOLIKE, wenn es als letzte t盲gliche Dosis verabreicht wurde, die metabolische Kontrolle im Vergleich zu standardm盲脽igen Aminos盲ure-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 Erh枚hung der Blut-Tyr (Tyrosin)-Spiegel nach einer n盲chtlichen Fastenzeit erreichte. Im Gegensatz dazu f眉hrten 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 p盲diatrischen Patienten mit klassischer PKU durchgef眉hrt.
- 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庐
DEER PARK, Ill., Dec. 17, 2024 (GLOBE NEWSWIRE) -- Eton Pharmaceuticals, Inc. (鈥淓ton鈥� or the 鈥淐ompany鈥�) (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鈥檚 Hospital, UK, on pediatric patients with classical PKU, the condition鈥檚 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鈥檚 Hospital, stated: 鈥淕iving 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.鈥�
These results confirm that PKU GOLIKE鈥檚 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鈥檚 Carglumic Acid, Betaine, and Nitisinone products. PKU patients鈥� 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 .
For more information on this study (NCT05487378), please visit .
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庐, PKU GOLIKE庐, Carglumic Acid, Betaine Anhydrous, and Nitisinone. The Company has three additional product candidates in late-stage development: ET-400, ET-600, and ZENEO庐 hydrocortisone autoinjector. For more information, please visit our website at .
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庐
PKU GOLIKE庐 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鈥檚 proprietary, patent-protected Physiomimic Technology鈩� drug delivery platform, PKU GOLIKE庐 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庐 granules are flavorless and can be mixed with many foods. PKU GOLIKE庐 products contain all 19 amino acids that people with PKU need to maintain neurological and muscular health and PKU GOLIKE PLUS庐 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庐 line of products are available in convenient packets (PKU GOLIKE PLUS庐 3-16 and 16+) and medical formula bars (PKU GOLIKE BAR庐). PKU GOLIKE庐 products have been commercially available in the U.S. since October 2022. For more information, visit . (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 鈥渇orward-looking statements鈥� 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鈥檚 business strategy, Eton鈥檚 plans to develop and commercialize its product candidates, the safety and efficacy of Eton鈥檚 product candidates, Eton鈥檚 plans and expected timing with respect to regulatory filings and approvals, and the size and growth potential of the markets for Eton鈥檚 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 鈥渂elieves,鈥� 鈥渁nticipates,鈥� 鈥減lans,鈥� 鈥渆xpects,鈥� 鈥渋ntends,鈥� 鈥渨ill,鈥� 鈥済oal,鈥� 鈥減otential鈥� and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Eton鈥檚 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鈥檚 development programs and financial position are described in additional detail in Eton鈥檚 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:
Source:聽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?
