– Clinical Data from Ongoing Phase 1b Dose-Escalation Study of RVU120
in Patients with AML or High-Risk MDS

– Poster presentation to highlight updated data from Phase 1/2 study of SEL24 (MEN1703), in Patients with IDH1/2-Mutated AML

 

Krakow, Poland – May 12, 2022 – 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 the data from the Phase 1b dose-escalation study of RVU120 (SEL120) in patients with AML or high-risk myelodysplastic syndromes (HR-MDS) and the Phase 1/2 study of SEL24 (MEN1703) in Patients with IDH1/2-Mutated AML will be presented at the Annual European Hematology Association (EHA) 2022 Hybrid Congress, June 9-17 in Vienna, Austria and online.

“The data presented at this year’s EHA Congress demonstrate Ryvu’s commitment to providing first-in-class and effective treatment options for people with hematologic malignancies,” said Hendrik Nogai, M.D., Chief Medical Officer at Ryvu Therapeutics. “We are excited to share promising results from our ongoing Phase 1b dose-escalation study in AML and HR-MDS, in which we have seen single-agent activity of RVU120. Notably, our translational data confirm the proposed MoA in a molecularly-defined subset of patients with DNMT3A and NPM1 mutations. Based on the encouraging data, we plan to further advance the clinical development of RVU120 in both biomarker-driven AML patients as well as in the unselected broader AML population.

We are also proud that the SEL24 (MEN1703) program, developed in collaboration with our partner Menarini, is making progress in the clinic. We look forward to future updates from our clinical programs throughout 2022, as we continue to advance new and innovative therapeutics for patients suffering from cancer.”

 

Details on the poster presentations are as follows:

RVU120: orally available CDK8/19 inhibitor

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 10 at 16:30 – 17:45 CEST

Preclinical data demonstrate differential sensitivity to RVU120 treatment in DNMT3 and NPM1 mutated AML Patient-Derived Cells (PDCs) in vitro and in vivo. Anti-cancer efficacy of RVU120 was associated with transcriptomic reprogramming involving inhibition of homeobox genes and induction of lineage commitment markers. Preliminary evidence of clinical response to RVU120 has also been shown in relapsed/refractory (R/R) AML and HR-MDS patients positive for DNMT3A and NPM1 mutations. Both mutations cooperate and define molecular subset of AML, characterized by elevated expression of homeobox genes. Further molecular studies confirming molecular mechanism of action and potential stratification markers are ongoing in more patients treated with RVU120.

 

Abstract Title: “CLI120-001 Phase1b Dose Escalation Study of RVU120 in Patients with AML or High-Risk MDS Safety and Efficacy Data Update”

Abstract Number: #P501

Session date and time: Friday, June 10 at 16:30 – 17:45 CEST

The presentation includes preliminary results from the first six patient cohorts, which demonstrated a favorable safety and a predictable pharmacokinetic (PK) profile of RVU120. Meaningful pharmacodynamic (PD) activity and clinical efficacy have been observed at the 50 and 75 mg doses. Enrollment in the trial continues with highest dose cohort now receiving 85 mg of RVU120 (NCT04021368).

 

SEL24/MEN1703: orally available dual PIM/FLT3 inhibitor

Abstract Title: “Phase 1/2 Study of SEL24/MEN1703, a First-In-Class Dual PIM/FLT3 Kinase Inhibitor, in Patients with IDH1/2-Mutated Acute Myeloid Leukemia: The DIAMOND-01 Trial”

Abstract Number: #P520

Session date and time: Friday, June 10 at 16:30 – 17:45 CEST

Ryvu’s partner Menarini Group reports the updated safety and efficacy results from an additional expansion cohort of the DIAMOND-01 trial which enrolled patients with relapsed or refractory (R/R) IDHm AML treated with the dual PIM/FLT3 inhibitor SEL24 (MEN1703). SEL24/MEN1703 demonstrated a manageable safety profile and single-agent activity in patients with R/R IDHm AML.

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