Recent data on RVU120 warrant further investigation in AML and HR-MDS

Ryvu Therapeutics has released updated clinical data from the dose escalation phase of its ongoing Phase Ib study RVU120 as monotherapy in patients with Acute Myleoid Leukemia (AML) or High-Risk Myelodysplastic Syndrome (HR) -MDS), which show clinical benefit and a favorable safety profile, the company said. The dose escalation phase continues and patients are currently being recruited to the 175 mg cohort. In addition, in vivo data indicate a synergistic effect of RVU120 in combination therapy with Ruxolitinib (RUX) in the treatment of myeloproliferative neoplasms (MPNs).

“Recent data from 24 previously heavily treated AML and HR-MDS patients enrolled and evaluated in the dose escalation phase of the ongoing Phase Ib study of RVU120 as monotherapy show a favorable safety profile and promising evidence of anti-leukaemic activity of RVU120, supported by the clinical benefit observed in 11 out of 24 patients studied. At higher doses of RVU120, we assume a further increase in the level of inhibition of our molecular target, which we anticipate should further enhance the clinical benefits seen in AML and HR-MDS patients as dose escalation progresses. At the same time, we observed that RVU120 therapy induces erythropoiesis, which supports further studies in anemic patients. In addition, we are pleased that RVU120, as a monotherapy, continues to be well tolerated at all doses. These data confirm our belief that RVU120 can become a valuable therapy for patients with AML and HR-MDS,” said Medical Director, Board Member Hendrik Nogai, quoted in the release.

“We also investigated the potential synergistic effects of RVU120 with Ruxolitinib (RUX) in myeloproliferative disease (MPN) models. Our data suggest that the combination of RVU120 with RUX leads to a significant reduction in disease symptoms in in vivo and in vitro models. We are impressed by the level of reduction in bone marrow fibrosis that is not seen with currently available therapies. The results of these studies indicate the potential of combining a CDK8/19 inhibitor with a JAK1/2 inhibitor in the targeted treatment of myeloproliferative neoplasms” – he added.


Ryvu Therapeutics was founded in 2007 (as Selvita). It is a Polish biotechnology company developing innovative small molecule compounds with therapeutic potential in oncology. Ryvu Therapeutics is listed on the main market of the Warsaw Stock Exchange.


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