• Updated Clinical Data from the Ongoing Phase 1b Dose-Escalation Study of RVU120 in Patients with AML or High-Risk MDS Show Favorable Safety and Promising Signs of Efficacy; 
• Poster Presentation to Highlight Synergistic Effects of RVU120 in Combination with Ruxolitinib in Myeloproliferative Neoplasms;

Krakow, Poland – May 11, 2023 – Ryvu Therapeutics (WSE: RVU), a clinical-stage drug discovery and development company focusing on novel small molecule therapies that address emerging targets in oncology, today announced that updated safety and efficacy data from the Phase 1b dose-escalation study of RVU120 in patients with Acute Myeloid Leukemia (AML) and High-Risk Myelodysplastic Syndromes (HR-MDS) and nonclinical data of RVU120 in combination with JAK1/2 inhibitor Ruxolitinib (RUX) in myeloproliferative neoplasms will be presented at the Annual European Hematology Association (EHA) 2023 Hybrid Congress, taking place June 8-11 in Frankfurt, Germany.

“We are very pleased with the emerging safety and efficacy data from the ongoing Phase 1b dose-escalation trial in these heavily pre-treated AML and HR-MDS patients,” said Hendrik Nogai, M.D., Chief Medical Officer of Ryvu Therapeutics. “Until February 2023, treatment with single-agent RVU120 has led to complete remission in one patient, an increase of hemoglobin and platelets in four patients, and bone marrow blast reduction in five patients, including in a patient with TP53 double-hit. Based on these encouraging clinical benefits, favorable safety, and no dose-limiting toxicities, we continue to dose escalate RVU120 and anticipate a further increase of anti-leukemic and erythroid-stimulating activity at higher doses.”

Dr. Nogai continued, “In addition, our data suggest synergistic effects between RVU120 and RUX in myelofibrosis by demonstrating a significant reduction of disease manifestation in vivo. These data reinforce the potential emerging role of targeting both CDK8/19 and JAK1/2 in myeloproliferative neoplasms. We look forward to sharing our findings at this year’s EHA Congress and to the continued development of RVU120.”

Details on the poster presentations are as follows:

Abstract Title: “Preclinical and Clinical Signs of RVU120 Efficacy, a Specific CDK8/19 Inhibitor in DNMT3A Mutation Positive AML and HR-MDS”
Abstract Number: #P450
Session date and time: Friday, June 9, 18:00 – 19:00 CEST

The clinical abstract presents updated safety and efficacy results from the ongoing Phase 1b dose escalation study of RVU120 for relapsed/refractory AML and high-risk MDS (NCT04021368). Results from patients dosed up to 110 mg have shown a favorable safety profile of RVU120. At the data cut-off of February 28, 2023, 22 pts have been enrolled, and 10 out of 19 evaluable patients showed clinical benefit: 1 pt. with AML treated at 75 mg with CR, 3 pts with AML treated at 100 mg, and 1 pt. with HR-MDS at 75 mg with a significant increase of hemoglobin and platelets, 4 pts with adverse risk cytogenetics treated between 10 and 100 mg with a BM blast reduction and 1 pt. with AML, dosed at 110 mg, showing a BM blast reduction of 70% at the beginning of cycle 4. No DLTs were observed, and no study drug interruptions due to adverse drug reactions occurred. The data warrant further exploration of RVU120 in AML and HR-MDS, and enrollment is ongoing at 135 mg.

 

Abstract Title: “Combination JAK1/2 and CDK8/19 inhibition demonstrates enhanced efficacy in myeloproliferative neoplasms”
Abstract Number: #P986
Session date and time: Friday, June 9, 18:00 – 19:00 CEST

The presentation, prepared in collaboration with Prof. Raajit Rampal’s group from Memorial Sloan Kettering Cancer Center, includes the assessment of RVU120, a highly selective and potent CDK8/19 inhibitor in monotherapy and combination with Ruxolitinib (RUX), a JAK1/2 inhibitor for the treatment of myeloproliferative neoplasms (MPN). Treatment with RVU120 demonstrated single agent efficacy that could be further enhanced by a synergistic combination with RUX in MPN models, both in vitro and in vivo. Treatment with the combination of RUX and RVU120 resulted in significant reductions of disease manifestation – mutated cell fractions, WBC, splenomegaly, and megakaryocyte differentiation when compared to RUX alone. These data nominate CDK8/19 inhibition in combination with JAK1/2 inhibition as a potential novel therapeutic strategy in MPNs.

All abstracts are now available online and can be obtained from the conference site: https://ehaweb.org/.