Relief Therapeutics Completes Clinical Phase in RLF-OD032 Proof-of-Concept Study; Topline Results Expected in October 2024
Relief Therapeutics (SIX:RLF, OTCQB:RLFTF, OTCQB:RLFTY) has completed the clinical phase of its proof-of-concept study for RLF-OD032, a treatment for phenylketonuria (PKU). The study aims to compare RLF-OD032 with an existing sapropterin dihydrochloride product, focusing on bioavailability under different conditions. Topline results are expected in October 2024, which will guide further development towards a pivotal trial and potential regulatory submission in the US.
RLF-OD032 is a highly concentrated liquid formulation of sapropterin dihydrochloride, designed to lower blood phenylalanine in PKU patients. It offers a more patient-friendly solution by reducing the required medication volume, potentially improving compliance, especially among pediatric patients. If approved, RLF-OD032 would be the first portable, ready-to-use liquid formulation of sapropterin dihydrochloride.
Relief Therapeutics (SIX:RLF, OTCQB:RLFTF, OTCQB:RLFTY) ha completato la fase clinica del suo studio di prova di concetto per RLF-OD032, un trattamento per la fenilchetonuria (PKU). Lo studio mira a confrontare RLF-OD032 con un prodotto esistente di diidrocloruro di sapropterina, concentrandosi sulla biodisponibilità in diverse condizioni. I risultati preliminari sono attesi per ottobre 2024, e guideranno ulteriori sviluppi verso uno studio pivotale e una potenziale presentazione regolatoria negli Stati Uniti.
RLF-OD032 è una formulazione liquida altamente concentrata di diidrocloruro di sapropterina, progettata per ridurre la fenilalanina nel sangue nei pazienti affetti da PKU. Offre una soluzione più adatta ai pazienti riducendo il volume di medicazione necessaria, potenzialmente migliorando l'aderenza, specialmente tra i pazienti pediatrici. Se approvato, RLF-OD032 sarebbe la prima formulazione liquida portatile e pronta all'uso di diidrocloruro di sapropterina.
Relief Therapeutics (SIX:RLF, OTCQB:RLFTF, OTCQB:RLFTY) ha completado la fase clínica de su estudio de prueba de concepto para RLF-OD032, un tratamiento para la fenilcetonuria (PKU). El estudio tiene como objetivo comparar RLF-OD032 con un producto existente de dihidrocloruro de sapropterina, centrándose en la biodisponibilidad bajo diferentes condiciones. Se esperan resultados preliminares en octubre de 2024, los cuales guiarán el desarrollo adicional hacia un ensayo pivotal y una posible presentación regulatoria en los EE. UU.
RLF-OD032 es una formulación líquida altamente concentrada de dihidrocloruro de sapropterina, diseñada para reducir la fenilalanina en sangre en pacientes con PKU. Ofrece una solución más amigable para los pacientes reduciendo el volumen de medicación necesario, lo que podría mejorar la adherencia, especialmente entre los pacientes pediátricos. Si se aprueba, RLF-OD032 sería la primera formulación líquida portátil y lista para usar de dihidrocloruro de sapropterina.
Relief Therapeutics (SIX:RLF, OTCQB:RLFTF, OTCQB:RLFTY)는 임상 단계를 완료했습니다 RLF-OD032에 대한 개념 증명 연구를 페닐케톤뇨증 (PKU) 치료제로서 수행했습니다. 이 연구는 RLF-OD032와 기존의 다이하이드로클로라이드 사프로테리니 제품을 비교하고, 다양한 조건에서의 생체이용률을 중점적으로 살펴봅니다. 핵심 결과는 2024년 10월에 발표될 예정입니다, 이는 미국에서의 주요 시험과 잠재적인 규제 제출을 위한 추가 개발의 방향을 안내할 것입니다.
RLF-OD032는 고농축 액상 제형의 다이하이드로클로라이드 사프로테리니로, PKU 환자의 혈중 페닐알라닌 수치를 낮추는 데 설계되었습니다. 이를 통해 필요한 약물 용량을 줄여 환자 친화적인 솔루션을 제공하고, 특히 소아 환자들 사이에서 순응도를 향상시킬 수 있습니다. 승인될 경우, RLF-OD032는 다이하이드로클로라이드 사프로테리니의 첫 번째 휴대 가능하고 사용 준비가 완료된 액상 제형이 될 것입니다.
Relief Therapeutics (SIX:RLF, OTCQB:RLFTF, OTCQB:RLFTY) a achevé la phase clinique de son étude de preuve de concept pour RLF-OD032, un traitement pour la phénylcétonurie (PKU). L'étude vise à comparer RLF-OD032 avec un produit existant de dyhydrochlorure de sapropterine, en se concentrant sur la biodisponibilité dans différentes conditions. Les résultats préliminaires sont attendus en octobre 2024, ce qui guidera le développement ultérieur vers un essai pivot et une soumission réglementaire potentielle aux États-Unis.
RLF-OD032 est une formulation liquide hautement concentrée de dyhydrochlorure de sapropterine, conçue pour réduire la phénylalanine dans le sang des patients atteints de PKU. Il offre une solution plus adaptée aux patients en réduisant le volume de médicament nécessaire, améliorant potentiellement l'observance, en particulier chez les patients pédiatriques. S'il est approuvé, RLF-OD032 serait la première formulation liquide portable et prête à l'emploi de dyhydrochlorure de sapropterine.
Relief Therapeutics (SIX:RLF, OTCQB:RLFTF, OTCQB:RLFTY) hat die klinische Phase abgeschlossen für seine Machbarkeitsstudie zu RLF-OD032, einer Behandlung von Phenylketonurie (PKU). Die Studie hat zum Ziel, RLF-OD032 mit einem bestehenden Di-Hydrochlorid-Sapropterin-Produkt zu vergleichen und sich auf die Bioverfügbarkeit unter verschiedenen Bedingungen zu konzentrieren. Die ersten Ergebnisse werden für Oktober 2024 erwartet, die eine weitere Entwicklung hin zu einer entscheidenden Studie und einer möglichen behördlichen Einreichung in den USA leiten werden.
RLF-OD032 ist eine hochkonzentrierte flüssige Formulierung von Di-Hydrochlorid-Sapropterin, die entwickelt wurde, um den Blut-Phenylalaninspiegel bei PKU-Patienten zu senken. Es bietet eine patientenfreundlichere Lösung, indem es das erforderliche Medikamentenvolumen reduziert, was die Compliance insbesondere bei pädiatrischen Patienten verbessern kann. Bei Genehmigung wäre RLF-OD032 die erste tragbare, verzehrfertige flüssige Formulierung von Di-Hydrochlorid-Sapropterin.
- Completion of clinical phase for RLF-OD032 proof-of-concept study
- Potential for improved patient compliance due to reduced medication volume
- Possibility of being the first portable, ready-to-use liquid formulation of sapropterin dihydrochloride
- Advancement towards a pivotal trial and potential regulatory submission
- Topline results not available until October 2024
- Uncertainty regarding the study outcomes and comparison with existing treatments
GENEVA, SWITZERLAND / ACCESSWIRE / September 18, 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 that it has completed dosing in its proof-of-concept clinical study of RLF-OD032 for the treatment of phenylketonuria (PKU), a rare inherited disorder affecting phenylalanine metabolism.
The primary objectives of the study include the comparison of RLF-OD032 to a currently marketed sapropterin dihydrochloride product, with a focus on bioavailability under fed and fasting conditions. The Company expects topline results in October 2024. These results will inform further development of RLF-OD032 toward a pivotal trial and potential regulatory submission under the 505(b)(2) NDA pathway in the United States.
RLF-OD032, an innovative and highly concentrated liquid formulation of sapropterin dihydrochloride, is designed to lower blood phenylalanine 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 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 Holdings AG
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