Relief Therapeutics Reports Positive Results from RLF-OD032 Proof-of-Concept Clinical Study for Phenylketonuria
Relief Therapeutics reported positive topline results from its proof-of-concept clinical study evaluating RLF-OD032, a concentrated liquid formulation of sapropterin dihydrochloride, for treating phenylketonuria (PKU). The four-way crossover pilot study compared RLF-OD032's pharmacokinetics with KUVAN® powder for oral solution. Topline data showed that RLF-OD032 administered under fed conditions, with or without water, achieved similar peak and total exposure of sapropterin dihydrochloride as KUVAN® under fed conditions with water, meeting FDA bioequivalence study requirements.
Relief plans to advance RLF-OD032 into a pivotal bioequivalence trial as part of the 505(b)(2) NDA submission process in the United States, following FDA pre-IND meeting guidance. The company believes RLF-OD032's potential for administration without water and low-volume dosage offers a unique, portable solution for PKU patients.
Relief Therapeutics ha riportato risultati preliminari positivi dal suo studio clinico di prova di concetto che valuta RLF-OD032, una formulazione liquida concentrata di diidrocloruro di sapropterina, per il trattamento della fenilchetonuria (PKU). Lo studio pilota con crossover a quattro vie ha confrontato la farmacocinetica di RLF-OD032 con quella della polvere KUVAN® per soluzione orale. I dati preliminari hanno mostrato che RLF-OD032, somministrato in condizioni di alimentazione, con o senza acqua, ha raggiunto esposizioni massime e totali di diidrocloruro di sapropterina simili a quelle di KUVAN® in condizioni di alimentazione con acqua, soddisfacendo i requisiti dello studio di bioequivalenza della FDA.
Relief prevede di portare avanti RLF-OD032 in un studio di bioequivalenza cruciale come parte del processo di presentazione della NDA 505(b)(2) negli Stati Uniti, seguendo le indicazioni della riunione pre-IND della FDA. L'azienda crede che il potenziale di RLF-OD032 per la somministrazione senza acqua e il dosaggio a basso volume offra una soluzione unica e portatile per i pazienti con PKU.
Relief Therapeutics informó sobre resultados preliminares positivos de su estudio clínico de prueba de concepto que evalúa RLF-OD032, una formulación líquida concentrada de dihidrocloruro de sapropterina, para el tratamiento de fenilcetonuria (PKU). El estudio piloto de cruce de cuatro vías comparó la farmacocinética de RLF-OD032 con el polvo KUVAN® para solución oral. Los datos preliminares mostraron que RLF-OD032 administrado bajo condiciones alimenticias, con o sin agua, alcanzó una exposición máxima y total de dihidrocloruro de sapropterina similar a la de KUVAN® bajo condiciones alimenticias con agua, cumpliendo con los requisitos del estudio de bioequivalencia de la FDA.
Relief planea avanzar RLF-OD032 a un ensayo de bioequivalencia pivotal como parte del proceso de presentación de NDA 505(b)(2) en los Estados Unidos, siguiendo la orientación de la reunión pre-IND de la FDA. La compañía cree que el potencial de RLF-OD032 para la administración sin agua y el dosificación de bajo volumen ofrece una solución única y portátil para los pacientes con PKU.
Relief Therapeutics는 긍정적인 초기 결과를 보고했습니다. 이는 RLF-OD032, 일종의 다이하이드로염화스프로펜의 농축 액체 제형에 대한 개념 증명 임상 연구 결과입니다. 이 네 방향 교차 파일럿 연구는 RLF-OD032의 약물 동태와 경구용 KUVAN® 분말을 비교했습니다. 초기 데이터는 RLF-OD032가 식사 조건에서 물과 함께 또는 없이 투여되었을 때, KUVAN®이 물과 함께 식사 조건에서 도달한 것과 유사한 최고 및 총 스프로펜 노출을 달성하여 FDA 생물등가성 연구 요건을 충족했음을 보여주었습니다.
Relief는 FDA의 사전 IND 회의 지침에 따라 RLF-OD032를 미국에서 NDA 505(b)(2) 제출 프로세스의 일환으로 중요한 생물등가성 시험으로 진행할 계획입니다. 이 회사는 물 없이 투여할 수 있는 RLF-OD032의 가능성과 저용량 제형이 PKU 환자에게 독특하고 휴대 가능한 솔루션을 제공할 것이라고 믿고 있습니다.
Relief Therapeutics a rapporté des résultats préliminaires positifs de son étude clinique de preuve de concept évaluant RLF-OD032, une formulation liquide concentrée de dihydrochlorure de sapropterine, pour traiter la phénylcétonurie (PKU). L'étude pilote croisée à quatre voies a comparé la pharmacocinétique de RLF-OD032 avec celle de la poudre KUVAN® pour solution orale. Les données préliminaires ont montré que RLF-OD032 administré dans des conditions nourries, avec ou sans eau, a atteint une exposition maximale et totale au dihydrochlorure de sapropterine similaire à celle de KUVAN® dans des conditions nourries avec de l'eau, satisfaisant les exigences de l'étude de bioéquivalence de la FDA.
Relief prévoit d'avancer RLF-OD032 dans un essai de bioéquivalence majeur dans le cadre du processus de soumission NDA 505(b)(2) aux États-Unis, suivant les directives de la réunion pré-IND de la FDA. L'entreprise croit au potentiel de RLF-OD032 pour une administration sans eau et à faible volume, offrant une solution unique et portable pour les patients atteints de PKU.
Relief Therapeutics hat positive vorläufige Ergebnisse aus seiner proof-of-concept-Studie veröffentlicht, die RLF-OD032, eine konzentrierte Flüssigkeitsformulierung von Dihydrochlorid-Sapropterin, zur Behandlung von Phenylketonurie (PKU) evaluiert. Die Pilotstudie mit vier Wege-Crossover verglich die Pharmakokinetik von RLF-OD032 mit KUVAN®-Pulver für eine orale Lösung. Vorläufige Daten zeigten, dass RLF-OD032, das unter Nahrungsaufnahme, mit oder ohne Wasser verabreicht wurde, eine ähnliche maximale und totale Exposition gegenüber Dihydrochlorid-Sapropterin erreichte wie KUVAN® unter Nahrungsaufnahme mit Wasser und die Anforderungen der FDA für Bioäquivalenzstudien erfüllte.
Relief plant, RLF-OD032 in eine entscheidende Bioäquivalenzstudie als Teil des NDA-Einreichungsprozesses 505(b)(2) in den Vereinigten Staaten zu überführen, gemäß den Richtlinien des FDA-Vorab-IND-Meetings. Das Unternehmen glaubt, dass das Potenzial von RLF-OD032 zur Einnahme ohne Wasser und das geringe Volumen eine einzigartige, tragbare Lösung für PKU-Patienten bietet.
- Positive topline results from proof-of-concept clinical study for RLF-OD032
- RLF-OD032 achieved similar peak and total exposure as KUVAN®, meeting FDA bioequivalence requirements
- Potential for RLF-OD032 to be administered without water, offering enhanced convenience for PKU patients
- Plans to advance RLF-OD032 into pivotal bioequivalence trial for NDA submission
- None.
GENEVA, SWITZERLAND / ACCESSWIRE / October 4, 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 reported positive topline results from its proof-of-concept clinical study evaluating RLF-OD032, an innovative and highly concentrated liquid formulation of sapropterin dihydrochloride, for the treatment of phenylketonuria (PKU).
The four-way crossover pilot study compared the pharmacokinetics of Relief's investigational drug RLF-OD032 with the reference listed drug KUVAN ® powder for oral solution. Topline data showed that RLF-OD032 administered under fed conditions, with or without water intake, achieved peak and total exposure of sapropterin dihydrochloride similar to those achieved by KUVAN ® under fed conditions with water, considering the range required by the FDA for bioequivalence studies.
"The pilot study successfully met its objective and provided valuable data for designing the next stages of our development and regulatory strategy. These results significantly increase the likelihood of meeting regulatory requirements, and we believe RLF-OD032 is well-positioned to achieve bioequivalence versus KUVAN ® in an upcoming pivotal bioequivalence trial," commented Giorgio Reiner, Chief Scientific Officer of Relief. "The potential for RLF-OD032 to be administered without water, along with its low-volume dosage, illustrates its value as a unique, ready-to-use, and portable solution, offering enhanced convenience and ease of administration for patients managing PKU."
Relief plans to advance RLF-OD032 into a pivotal bioequivalence trial as part of the 505(b)(2) NDA submission process in the United States and pursuant to the development guidance previously provided by the FDA in a pre-IND meeting.
ABOUT RLF-OD032
RLF-OD032 is an innovative, ready-to-use, portable and highly concentrated formulation of sapropterin dihydrochloride in liquid suspension for oral administration, designed to lower blood phenylalanine levels in adult and pediatric PKU patients. It offers a more patient-friendly solution by significantly reducing the volume of medication required compared to current formulations. This advancement aims to enhance compliance, particularly among pediatric patients, who often struggle with the high volumes associated with existing sapropterin treatments. If approved, RLF-OD032 would be the first and only portable, ready-to-use liquid formulation of sapropterin dihydrochloride.
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. However, this diet is highly restrictive and often creates barriers to social interaction, limiting compliance and increasing the risk of poor disease management. Sapropterin dihydrochloride is the first approved drug for PKU for reducing Phe blood levels and allowing patients to follow a less restrictive diet.
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. Forward-looking statements involve known and unknown risks, uncertainties, including its ability to achieve its corporate, development and commercial goals, and other factors which could cause the actual results, financial condition, performance or achievements of Relief to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. A number of factors, including those described in Relief's filings with the SIX Swiss Exchange and the U.S. Securities and Exchange Commission (SEC), could adversely affect Relief. Copies of Relief's filings with the SEC are available on the SEC EDGAR database at www.sec.gov. Relief does not undertake any obligation to update the information contained herein, which speaks only as of this date.
SOURCE: Relief Therapeutics Holding SA
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