Relief Therapeutics Reports New Study Results for RLF-OD032 and Files Provisional Patents
Relief Therapeutics announced positive clinical study results for RLF-OD032, their investigational drug for phenylketonuria (PKU), and filed provisional patents in the United States. The study revealed that RLF-OD032, a liquid formulation of sapropterin dihydrochloride, showed superior absorption in fasted conditions compared to KUVAN®. Unlike KUVAN®, which requires food and water for optimal absorption, RLF-OD032 demonstrated effective absorption without these requirements. This breakthrough could offer PKU patients more flexible dosing options and improved convenience. The company plans to file a 505(b)(2) NDA in the U.S. by Q3/2025.
Relief Therapeutics ha annunciato risultati positivi dello studio clinico per RLF-OD032, il loro farmaco sperimentale per la fenilchetonuria (PKU), e ha presentato brevetti provvisori negli Stati Uniti. Lo studio ha rivelato che RLF-OD032, una formulazione liquida di sapropterina diidrochloride, mostrava un'assorbimento superiore in condizioni di digiuno rispetto a KUVAN®. A differenza di KUVAN®, che richiede cibo e acqua per un'assorbimento ottimale, RLF-OD032 ha dimostrato un'assorbimento efficace senza tali requisiti. Questa innovazione potrebbe offrire ai pazienti con PKU opzioni di dosaggio più flessibili e maggiore comodità. L'azienda prevede di presentare un NDA 505(b)(2) negli Stati Uniti entro il terzo trimestre del 2025.
Relief Therapeutics anunció resultados positivos de un estudio clínico para RLF-OD032, su medicamento en investigación para la fenilcetonuria (PKU), y presentó patentes provisionales en los Estados Unidos. El estudio reveló que RLF-OD032, una formulación líquida de sapropterina dihidrocloruro, mostró una absorción superior en condiciones de ayuno en comparación con KUVAN®. A diferencia de KUVAN®, que requiere alimentos y agua para una absorción óptima, RLF-OD032 demostró una absorción efectiva sin estos requisitos. Este avance podría ofrecer a los pacientes con PKU opciones de dosificación más flexibles y mayor comodidad. La empresa planea presentar una NDA 505(b)(2) en EE. UU. para el tercer trimestre de 2025.
Relief Therapeutics는 RLF-OD032에 대한 긍정적인 임상 연구 결과를 발표했으며, 이는 페닐케톤뇨증(PKU)에 대한 그들의 실험 약물입니다. 이 약물에 대한 잠정 특허를 미국에서 제출했습니다. 연구 결과 RLF-OD032는 KUVAN®과 비교하여 금식 상태에서 뛰어난 흡수를 보였습니다. KUVAN®는 최적의 흡수를 위해 음식과 물이 필요하지만, RLF-OD032는 이러한 조건 없이도 효과적인 흡수를 보여주었습니다. 이 혁신은 PKU 환자들에게 더 유연한 투약 옵션과 편리함을 제공할 수 있습니다. 회사는 2025년 3분기 내에 미국에서 505(b)(2) NDA를 제출할 계획입니다.
Relief Therapeutics a annoncé des résultats positifs d'étude clinique pour RLF-OD032, leur médicament expérimental pour la phénylcétonurie (PKU), et a déposé des brevets provisoires aux États-Unis. L'étude a révélé que RLF-OD032, une formulation liquide de dihydrochlorure de sapropterine, montrait une absorption supérieure dans des conditions de jeûne par rapport à KUVAN®. Contrairement à KUVAN®, qui nécessite de la nourriture et de l'eau pour une absorption optimale, RLF-OD032 a démontré une absorption efficace sans ces exigences. Cette avancée pourrait offrir aux patients atteints de PKU des options de dosage plus flexibles et une meilleure commodité. L'entreprise prévoit de déposer un NDA 505(b)(2) aux États-Unis d'ici le troisième trimestre 2025.
Relief Therapeutics hat positive Ergebnisse einer klinischen Studie zu RLF-OD032, ihrem experimentellen Medikament gegen Phenylketonurie (PKU), bekannt gegeben und provisorische Patente in den Vereinigten Staaten angemeldet. Die Studie ergab, dass RLF-OD032, eine flüssige Formulierung von Sapropterindihydrochlorid, eine überlegene Absorption unter Fastenbedingungen im Vergleich zu KUVAN® zeigte. Im Gegensatz zu KUVAN®, das für eine optimale Absorption Nahrung und Wasser benötigt, zeigte RLF-OD032 eine wirksame Absorption ohne diese Anforderungen. Dieser Durchbruch könnte PKU-Patienten flexiblere Dosierungsoptionen und mehr Komfort bieten. Das Unternehmen plant, bis zum dritten Quartal 2025 einen 505(b)(2) NDA in den USA einzureichen.
- Superior drug absorption demonstrated in fasted state compared to competitor KUVAN®
- Potential competitive advantage with flexible dosing without food/water requirements
- Patent applications filed to strengthen intellectual property protection
- Clear regulatory pathway with 505(b)(2) NDA filing planned for Q3/2025
- None.
New Study Results Indicate Superior Absorption of RLF-OD032 in Fasted State Compared to KUVAN®, Potentially Enabling Flexible Dosing Options
GENEVA, SWITZERLAND / ACCESSWIRE / October 25, 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 new positive clinical study results for RLF-OD032 and the filing of provisional patent applications in the United States. The patents, based on these new findings, cover additional claims for Relief's investigational drug RLF-OD032, a highly concentrated, novel liquid formulation of sapropterin dihydrochloride, for the treatment of phenylketonuria (PKU).
The patent filings follow the recently announced completion of a pilot, proof-of-concept, four-way crossover study that evaluated the pharmacokinetic profile of RLF-OD032 and its absorption in both fasted and fed conditions. The Company previously reported that RLF-OD032, when administered in fed conditions without water, achieved peak and total exposure of sapropterin dihydrochloride similar to those achieved by the reference product (KUVAN ® Powder) administered with water.
Today, Relief announced additional positive and unexpected results from the study that form the basis of these new patent applications. Specifically, the administration of RLF-OD032 in a fasted state without water resulted in greater absorption of sapropterin dihydrochloride compared to the reference product administered under fed conditions with water. In contrast, KUVAN shows poor absorption when taken with water in a fasted state, as reported in KUVAN's Full Prescribing Information, which recommends that PKU patients take the product exclusively with meals with a large volume of water.
These findings indicate that RLF-OD032 could offer new administration options for PKU patients, providing greater flexibility for dosing without the need for food and water. This may improve patient convenience and compliance, allowing them to take their medication anytime, even while on the go.
The U.S. provisional patent applications strengthen the intellectual property around RLF-OD032 and complement the Company's existing patent estate. Relief is evaluating the development and regulatory implications of these findings as it continues to advance RLF-OD032 through clinical development with the objective of filing a 505(b)(2) NDA in the U.S. by Q3/2025.
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.
KUVAN ® is a registered trademark of BioMarin Pharmaceutical Inc. The use of this trademark in this press release is for reference purposes only, and Relief has no affiliation, sponsorship, or endorsement from BioMarin Pharmaceutical Inc. All references to KUVAN are made solely for comparison of study results and do not imply any relationship between the companies.
SOURCE: Relief Therapeutics Holding SA
View the original press release on accesswire.com
FAQ
What are the key findings of Relief Therapeutics' (RLFTF) new study for RLF-OD032?
When does Relief Therapeutics (RLFTF) plan to file the NDA for RLF-OD032?
What advantage does RLF-OD032 offer over KUVAN® for PKU patients?
