Ryvu Therapeutics to present clinical and preclinical data on RVU120 at the 2024 European Hematology Association Congress

• Updated data from the Phase I trial of RVU120 in patients with relapsed/refractory acute myeloid leukemia (r/r AML) or high-risk myelodysplastic syndromes (HR-MDS) demonstrate promising clinical activity.
• Preclinical data support the synergistic combination of RVU120 and venetoclax in patients with AML, including RVU120's potential to overcome resistance to venetoclax treatment.
• Combination data of RVU120 with other drug candidates in vitro support potentially new therapeutic options for patients with myeloproliferative neoplasm (MPN). In vivo data further support CDK8 inhibition as a potential novel therapeutic strategy in MPNs, particularly myelofibrosis (MF).
• A webinar with Professor Raajit Rampal covering the myelofibrosis landscape, latest data, and potential of RVU120 in MF. The webinar will be held on Friday, May 17, at 3:00 PM (CET). Registration is available at: https://ryvu.clickmeeting.com/pre-eha2024-webinar-rvu120-progress-and-opportunity-in-mf/register

KRAKOW, Poland, May 14, 2024 /PRNewswire/ -- Ryvu Therapeutics (WSE: RVU), a clinical-stage drug discovery and development company focusing on novel small molecule therapies that address emerging targets in oncology, will present clinical and preclinical data from RVU120 at the 2024 European Hematology Association Congress (EHA), June 13-16, Madrid, Spain.

"At this year's EHA conference, we proudly unveil the latest advancements in RVU120 research and clinical development, illuminating its potential in addressing the unmet need in hematologic malignancies. RVU120 has demonstrated clinical activity as a single agent in 15 of 29 evaluable patients with AML and HR-MDS at tolerated doses. Furthermore, our preclinical data demonstrate the synergistic potential of RVU120 in combination with venetoclax, representing a promising strategy against resistance in AML. We are also excited about the emerging data in MPN models, where combination regimens with RVU120 could represent a new treatment paradigm." – Dr. Hendrik Nogai, Chief Medical Officer, Ryvu Therapeutics.

Based on these data and in line with prior guidance, Ryvu's development plan for RVU120 includes four Phase II studies: the RIVER-52 study in patients with AML/HR-MDS as monotherapy, the RIVER-81 study in combination with venetoclax in patients with r/r AML, the POTAMI-61 study in patients with myelofibrosis, and the REMARK study, an investigator-initiated trial in patients with LR-MDS.

Poster highlights:

Abstract Title: RVU120, a first-in-class CDK8 inhibitor for the treatment of relapsed/refractory AML and high-risk MDS: preliminary results from two ongoing studies.

Number: 6466 
Session date and time: Friday, June 14 (18:00-19:00 CEST)

The abstract includes data on 29 evaluable patients out of 38 total dosed patients.

In the Phase I trial CLI120-001, RVU120 shows promising clinical activity in patients with AML or HR-MDS. 15 out of 38 patients experienced clinical benefit with RBC transfusion independence and/or blast reduction. A complete remission (CR) was achieved in a patient with NPM1 and DNMT3A mutations as well as 3 marrow CRs in patients with HR-MDS. Relevant target inhibition was achieved at a dose of 110 mg or higher, supporting a recommended Phase II dose (RP2D) of 250mg.  The follow-on Phase II RIVER-52 study investigates RVU120 as a monotherapy in patients with genetically defined AML or HR-MDS and is currently recruiting. 

Abstract Title: Synergistic potential of RVU120, a first-in-class CDK8/CDK19 inhibitor, with venetoclax in AML: preclinical and initial clinical insights.

Abstract Number: 6720
Session date and time: Friday, June 14 (18:00-19:00 CEST)

The abstract presents updated preclinical data supporting the synergistic combination of RVU120 and venetoclax in AML, including RVU120's potential to overcome resistance to venetoclax treatment. Ongoing preclinical research and an ongoing Phase II clinical study seek to refine patient stratification and enhance therapeutic outcomes, with initial safety profiles appearing favorable. The Phase II RIVER-81 study investigates RVU120 in combination with venetoclax in patients with AML and is currently recruiting.

Abstract Title: CDK8/19 Inhibition: A Promising Therapeutic Strategy in Myeloproliferative Neoplasms.

Poster Number:  P1018

Session date and time:  Friday, June 14 (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 as a monotherapy and in combination with JAK inhibitors and other drug candidates for the treatment of myeloproliferative neoplasms (MPN). Analysis of the effects from combinations of RVU120 with other compounds in vitro has revealed potentially new therapeutic options for patients with MPN sensitivity and resistance to ruxolitinib (RUX). In vivo data further support CDK8 inhibition as a potential novel therapeutic strategy in MPNs. Additional mechanistic work will potentially elucidate disease mechanisms and therapeutic action independent of JAK-STAT attenuation. This work has led to a Phase II clinical study (POTAMI-61) for RVU120 as a monotherapy in patients with myelofibrosis patients not eligible for RUX or in combination with RUX for those patients with myelofibrosis and suboptimal RUX response. 

The final posters that will be presented on June 14th will contain additional information.

Investor Event:

Ryvu will host a webinar on Friday, May 17, at 3:00 PM CET with Professor Raajit Rampal, who will present the myelofibrosis landscape, the latest data and the potential of RVU120 in MF.

To join the webcast, please register here: https://ryvu.clickmeeting.com/pre-eha2024-webinar-rvu120-progress-and-opportunity-in-mf/register 

About Ryvu Therapeutics 

Ryvu Therapeutics is a clinical-stage drug discovery and development company focused on novel small-molecule therapies that address emerging targets in oncology. Internally discovered pipeline candidates use diverse therapeutic mechanisms driven by emerging knowledge of cancer biology, including small molecules directed at kinase, synthetic lethality, and immuno-oncology targets.

Ryvu's most advanced programs include RVU120, a selective CDK8/CDK19 kinase inhibitor with the potential to treat hematological malignancies and solid tumors, currently in Phase II development (i) as a monotherapy for the treatment of patients with relapsed/refractory acute myeloid leukemia (r/r AML) and high-risk myelodysplastic syndromes (HR-MDS) as well as (ii) in combination with venetoclax for the treatment of patients with r/r AML. Another clinical program, SEL24 (MEN1703), is a dual PIM/FLT3 kinase inhibitor licensed to the Menarini Group. Ryvu Therapeutics has signed multiple partnering and licensing deals with global companies, including BioNTech and Exelixis.

The Company was founded in 2007 and is headquartered in Kraków, Poland. Ryvu is listed on the Warsaw Stock Exchange and is a component of the mWIG40 index. For more information, please see www.ryvu.com. 

SOURCE Ryvu Therapeutics