On December 13, 2021 Ryvu Therapeutics (WSE: RVU) a clinical-stage drug discovery and development company focusing on novel small molecule therapies that address emerging targets in oncology, reported updated clinical and preclinical data demonstrating the single-agent activity of its two lead oncology drug candidates, RVU120 and SEL24 (MEN1703) at the 63rd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, held December 11 – December 14, 2021, in Atlanta, as well as at the 44th Annual San Antonio Breast Cancer Symposium (SABCS) held December 7 – December 10, 2021 (Press release, Ryvu Therapeutics, DEC 13, 2021, View Source [SID1234596993]).

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Presented data included updated clinical results for RVU120, a selective CDK8/19 inhibitor being developed for the treatment of hematological malignancies and solid tumors. The first-in-human (FIH) Phase 1b dose-escalation trial (CLI120-001), which is currently enrolling patients with relapsed/refractory (R/R) acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (HR-MDS), has thus far demonstrated an acceptable safety profile and preliminary signs of efficacy for RVU120.

With a data cutoff of November 16, 2021, data highlights include:

A Complete Remission (CR) in an AML patient harboring mutations in DNMT3A and NPM1. Supportive translational data were also presented demonstrating the anti-leukemic activity of RVU120 in PDX AML models bearing DNMT3A and NPM1 mutations.
An erythroid response (ER) in a HR-MDS patient who had relapsed after several lines of previous treatment; as of the data cutoff, this patient remains on study treatment with stable disease for more than 15 months. Supportive translational data were also presented demonstrating that RVU120 induces erythroid differentiation in (Lin-) CD34+ cells.
Acceptable safety profile in 6 patients completing safety evaluations for cycle 1. None of these patients experienced dose-limiting toxicity (DLT). A total of 12 serious adverse events (SAEs) have been reported – none were deemed to be related to the study drug.
An additional poster on the potential efficacy of RVU120 in hormone-negative breast cancer models was presented at the 2021 San Antonio Breast Cancer Symposium, which showed that oral administration of RVU120 demonstrated strong anticancer activity in a TNBC xenograft model.

Ryvu licensee Menarini Group presented updated Phase 2 data for SEL24 (MEN1703) (DIAMOND-01, ClinicalTrials.gov identifier: NCT03008187), showing pharmacodynamics (PD) and genomic profiling in the First-in-Human Diamond-01 trial.

"We are delighted to present data update at the ASH (Free ASH Whitepaper) and SABCS conferences, including clinical responses in the Phase Ib trial of RVU120, and exciting translational data for this project", said Pawel Przewiezlikowski, Chief Executive Officer of Ryvu Therapeutics. "We have managed to achieve several important development milestones across our pipeline in 2021, highlighting our commitment to challenge current treatment paradigms and develop therapeutics that address clinical limitations of current treatments in oncology. In 2022, we expect to achieve additional clinical milestones for our two lead oncology drug candidates, RVU120 and SEL24, as we remain focused on developing new and innovative therapeutics for patients suffering from cancer."

Posters presented at the 63rd ASH (Free ASH Whitepaper) Annual Meeting & Exposition included:

CLI120-001 Phase Ib Study of RVU120 (SEL120) in Patients with AML and High-Risk MDS: Updated Safety/Efficacy Results from Initial Dose Escalation (Publication Number: 3418)
RVU120 (SEL120) CDK8/19 Inhibitor – a Drug Candidate for the Treatment of MDS Can Induce Erythroid Differentiation (Publication Number: 1518)
Inhibition of Cyclin Dependent Kinase 8 (CDK8): A Novel Approach to Target the Leukemia Initiating Cells (LICs) in T-Cell Acute Lymphoblastic Leukemia (T-ALL) (Publication Number: 2250)
Preclinical and Clinical Signs of Efficacy of RVU120 (SEL120), a Specific CDK8/19 Inhibitor in DNMT3A-Mutated AML (Publication Number: 2371)
SEL24(MEN1703) Inhibits PIM/FLT3 Downstream Target in Acute Myeloid Leukemia (AML) Patients: Results of the Pharmacodynamics (PD) Assay and Genomic Profiling in the First-in-Human Diamond-01 Trial (Publication Number: 3436)
Selective CDK8/CDK19 inhibitor RVU120 demonstrates efficacy against hormone-independent breast cancer cells in vitro and in vivo (#1766), presented at the 2021 San Antonio Breast Cancer Symposium.