- Five abstracts highlight RVU120 in acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), and myelofibrosis (MF).
- One abstract on RVU120 activity in MDS patient-derived cells was accepted for oral presentation.
- Four abstracts were accepted for poster presentations covering the Phase II RIVER-52, RIVER-81, and POTAMI-61 studies and translational data supporting the RIVER-81 study.
Krakow, Poland – May 14, 2025 – Ryvu Therapeutics (WSE: RVU), a clinical-stage drug discovery and development company focusing on novel therapies that address emerging targets in oncology, today announced it will present RVU120 data, including one oral presentation and four poster presentations, at the 2025 European Hematology Association Congress (EHA), June 12-15, 2025 in Milan, Italy.
Hendrik Nogai, M.D., Chief Medical Officer of Ryvu Therapeutics, said: We are pleased to have an oral presentation this year on RVU120 translational data in MDS patient-derived cells and have updates across our Phase II program. The early signs of clinical activity described in the EHA abstracts supported our decision in the first quarter of 2025 to prioritize three Phase II clinical studies: RIVER-81 in AML in combination with venetoclax, POTAMI-61 both as a single agent and in combination with ruxolitinib in MF, and REMARK in LR-MDS.
Details on the abstract presentations are as follows (cut-off date February 2025):
Abstract Title (oral presentation): RVU120 enhances erythroid potential in MDS patient-derived cells: preclinical mechanistic insights into CDK8/CDK19 inhibition and potential patient stratification
Session date and time: 12 June 2025, 5:00pm – 6:15pm CEST
Session title: s450 MDS cellular and molecular therapeutic targeting
RVU120, a selective inhibitor of CDK8 and CDK19, demonstrates significant potential in enhancing erythroid differentiation in MDS patient-derived cells confirmed by transcriptomic and functional analysis. Data show that RVU120 promotes erythropoiesis in CD34+ bone marrow cells derived from MDS patients, particularly benefiting those with differentiation defects. Results from multiple patient-derived samples indicate potential patient stratification based on ASXL1 mutations. These findings support RVU120 as a promising therapeutic candidate in the REMARK Phase II clinical study in patients with low-risk myelodysplastic syndromes (LR-MDS).
Abstract Title (poster presentation): Preliminary results from RIVER-81, a Phase 2 study of RVU120+VEN in patients with AML failing first-line VEN+HMA
Session date and time: 14 June 2025, 6:30pm – 7:30pm CEST
Poster Number: PS1509
Preliminary results from the open-label RIVER-81 Phase II clinical study demonstrate that RVU120, when combined with venetoclax (VEN), shows promising anti-leukemic activity in patients with relapsed or refractory acute myeloid leukemia (r/r AML) who failed first-line VEN-based treatment. As of February 25, 2025, 36 patients had been treated. 20 patients were evaluable for response across Parts 1 and 2. One out of two evaluable patients from Cohort 2 achieved a complete remission (CR). Three out of thirteen (23%) evaluable patients from stage 1 of Part 2 achieved a complete remission with incomplete count recovery (CRi), suggesting that RVU120 may help overcome VEN resistance. The study, which continues enrolling patients, supports further exploration of RVU120+VEN as a potential therapeutic strategy for AML with poor prognosis. The combination has been well tolerated, with nausea as the most common adverse event.
Abstract Title (poster presentation): An Open-Label Clinical Trial of RVU120 as Monotherapy and in Combination with Ruxolitinib in Patients with Intermediate or High-Risk, Primary or Secondary Myelofibrosis (POTAMI-61)
Session date and time: 13 June 2025, 6:30pm – 7:30pm CEST
Poster Number: PF861
The open-label POTAMI-61 Phase II clinical trial evaluates RVU120, a selective CDK8/19 inhibitor, as a monotherapy and in combination with ruxolitinib (RUX) for patients with intermediate or high-risk myelofibrosis (MF). As of February 6, 2025, 12 patients were treated. The median time on treatment was four weeks, and no patient had met the follow-up for the primary endpoint due to insufficient time on the study. The ongoing trial aims to assess spleen volume reduction, symptom burden, and safety over 24 weeks. Early results show that RVU120, alone or with RUX, is well tolerated, with three patients showing early signs of clinical improvement, including improved hematologic parameters and an indication of a reduced spleen size. These early findings support further investigation of RVU120 as a potential treatment for MF for patients who have not responded adequately to JAK inhibitors.
Abstract Title (poster presentation): RIVER-52: A Multicenter, Open-Label Clinical Trial of RVU120 in Patients with Relapsed or Refractory High-Risk Myelodysplastic Syndrome or Acute Myeloid Leukemia
Session date and time: 13 June 2025, 6:30pm – 7:30pm CEST
Poster Number: PF548
The open-label RIVER-52 Phase II clinical study evaluated RVU120 monotherapy in patients with AML or relapsed or refractory high-risk MDS (HR-MDS). 39 patients received RVU120 (27 AML and 12 HR-MDS patients). RVU120 demonstrated a manageable safety profile, with gastrointestinal and infectious adverse events being the most common. While two patients with NPM1 and DNMT3A mutations showed >50% reduction in bone marrow blasts, no CRs were observed. As predefined efficacy criteria were not met, the trial did not advance to Part 2, and enrollment was suspended.
Abstract Title (poster presentation): Overcoming venetoclax resistance: synergistic potential of RVU120, a CDK8/CDK19 inhibitor, in combination treatment
Session date and time: 13 June 2025, 6:30pm – 7:30pm CEST
Poster Number: PF415
RVU120 demonstrates strong synergy when combined with venetoclax (VEN) to overcome resistance to VEN in the treatment of AML. Preclinical studies reveal that RVU120+VEN effectively targets key resistance pathways, including IL6/JAK/STAT3, TGF-β, and PI3K/AKT/mTOR. The combination also retains efficacy in models of bone marrow stroma-mediated resistance, a common mechanism of therapy failure. These findings support the ongoing Phase II RIVER-81 trial, exploring RVU120+VEN in patients with AML who have failed prior VEN-based treatments. This research underscores RVU120’s potential to improve treatment outcomes by overcoming venetoclax resistance in AML.
All abstracts are now available online and can be obtained from the conference site:
https://ehaweb.org/congress/eha2025-congress/
About Ryvu Therapeutics
Ryvu Therapeutics is a clinical-stage drug discovery and development company focused on novel oncology therapies that address emerging targets in oncology. Internally discovered pipeline candidates at Ryvu use diverse therapeutic mechanisms driven by emerging knowledge of cancer biology, including small molecules and antibody-drug conjugates directed at kinases, synthetic lethality, and immuno-oncology targets.
Ryvu’s most advanced program is RVU120, a selective CDK8/CDK19 kinase inhibitor with the potential to treat hematological malignancies. RVU120 is currently in Phase II development (i) in combination with venetoclax for the treatment of patients with r/r AML – the RIVER-81 study, (ii) as a monotherapy for the treatment of patients with lower-risk myelodysplastic syndromes (LR-MDS) – the REMARK study, (iii) as a monotherapy and in combination with ruxolitinib for the treatment of patients with myelofibrosis (MF) – the POTAMI-61 study. Dapolsertib (MEN1703, SEL24) is a dual PIM/FLT3 kinase inhibitor licensed to the Menarini Group that is currently being investigated in a Phase II study in diffuse large B-cell lymphoma (DLBCL) – the JASPIS-01 study. RVU305, a potentially best-in-class, brain-permeable PRMT5 inhibitor aiming to treat multiple solid tumors, is currently in IND/CTA-enabling studies. Ryvu Therapeutics is also engaged in oncology collaborations with BioNTech and Exelixis.
Ryvu was founded in 2007 and is headquartered in Kraków, Poland. It is listed on the Warsaw Stock Exchange and is a component of the sWIG80 index.
For more information, please visit www.ryvu.com
Contact:
Anna Wilk
Ryvu Therapeutics
+48 532 698 425