Ryvu Initiates RIVER-52 Phase II Trial with First Patient Dosed in RVU120 Monotherapy for r/r AML and HR-MDS Treatment

Polish pharmaceutical company Ryvu Therapeutics has commenced a Phase II clinical trial for its drug candidate RVU120, which is being developed as a standalone treatment for individuals suffering from relapsed or refractory acute myeloid leukemia (AML) and high-risk myelodysplastic syndromes (MDS). The trial, known as RIVER-52, marks a significant advancement in Ryvu's mission to address unmet needs in oncology with innovative small molecule therapies.

The primary objective of the RIVER-52 study is to assess the safety and effectiveness of RVU120 in a broader patient group characterized by specific genetic AML subtypes, such as those with NPM1 mutations, as well as in patients with HR-MDS. The dosage for the trial, set at 250 mg every other day, is based on findings from a previous Phase Ib study that indicated promising clinical activity.

Initially, the trial will be conducted at medical centers in Poland and Italy, with plans to extend to approximately 80 clinical sites across the European Union and beyond. The study is expected to enroll around 140 patients in total, which aligns with Ryvu's financial strategy and development roadmap, as outlined in October 2023.

RVU120 stands out as a first-of-its-kind dual CDK8/19 kinase inhibitor, a novel approach to treating various hematological malignancies. In the Phase Ib study, RVU120 demonstrated significant clinical benefits for 50% of the patients evaluated, including complete responses and substantial improvements in hematologic measures.

Hendrik Nogai, the Chief Medical Officer at Ryvu Therapeutics, expressed enthusiasm about the initiation of the Phase II study, highlighting the drug's safety and efficacy observed in earlier trials. Kamil Sitarz, the Chief Operating Officer, emphasized the company's commitment to efficient patient enrollment and the timely progression of the RIVER-52 study, with the aim of accelerating the regulatory approval process for RVU120.

The RIVER-52 study is structured in two parts: the first will gauge the anti-tumor activity in genetically defined AML subtypes and HR-MDS patients, while the second part will delve deeper into the safety and efficacy profile in a larger, more responsive patient group. Approval from regulatory bodies and ethics committees in Poland and Italy has already been secured, paving the way for patient recruitment.

Ryvu Therapeutics, established in 2007 and based in Kraków, is dedicated to pioneering cancer treatments. The company's pipeline features a range of internally discovered candidates that leverage the latest insights in cancer biology. In addition to RVU120, Ryvu has other clinical programs, such as SEL24 (MEN1703), which targets kinase pathways and has been licensed to the Menarini Group. Ryvu has also established partnerships with major pharmaceutical companies like BioNTech and Exelixis.

Ryvu Therapeutics is publicly traded on the Warsaw Stock Exchange and is part of the mWIG40 index, reflecting its position as a significant player in the pharmaceutical sector. The company's ongoing clinical trials and development efforts underscore its dedication to bringing forward new treatment options for patients with hematological diseases.