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ASH 2024 | Preliminary safety & efficacy of single-agent emavusertib in patients with HR-MDS

Uwe Platzbecker, MD, University of Leipzig, Leipzig, Germany, comments on the preliminary safety, efficacy, and molecular characterization of single-agent emavusertib in patients with higher-risk myelodysplastic syndromes (HR-MDS). This IRAK4 inhibitor is being investigated in the Phase I/II TakeAim Leukemia trial (NCT04278768), and Prof. Platzbecker highlights that preliminary data confirm its efficacy in achieving a complete response (CR), particularly in patients with FLT3 mutations. This interview took place at the 66th ASH Annual Meeting and Exposition, held in San Diego, CA.

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Transcript (AI-generated)

Now, this is an agent, an orally available agent. It’s an IRAK4 inhibitor. IRAK4 is actually a pathway which is hijacked by myeloid progenitor cells and also upregulated in relapsed/refractory disease. The drug also inhibits FLT3 and therefore preliminary data already showed some activity. So this trial is still ongoing, but the data presented at the ASH, number one, confirm the safety of the agent...

Now, this is an agent, an orally available agent. It’s an IRAK4 inhibitor. IRAK4 is actually a pathway which is hijacked by myeloid progenitor cells and also upregulated in relapsed/refractory disease. The drug also inhibits FLT3 and therefore preliminary data already showed some activity. So this trial is still ongoing, but the data presented at the ASH, number one, confirm the safety of the agent. So there were some concerns about increase of creatine kinase in the early days of the trial, which was actually dose-related. So with the current dose, this is no longer an issue anymore. And number two, the drug is also efficacious in the sense that a considerable amount of patients actually achieved a CR, especially patients with a FLT3 mutation did so, again, supporting the preclinical rationale. The drug is still evaluated in this trial as a monotherapy but it’s also evaluated in other segments of myeloid neoplasm, including as a triplet combination partner together with azacitidine and venetoclax.

 

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Disclosures

Amgen: Consultancy, Research Funding; BMS: Consultancy, Membership on an entity’s Board of Directors or advisory committees, Other: Travel support, Research Funding; MDS Foundation: Membership on an entity’s Board of Directors or advisory committees; Abbvie: Consultancy, Research Funding; Curis: Consultancy, Honoraria, Research Funding; Geron: Consultancy; Janssen: Consultancy, Honoraria, Research Funding; Merck: Research Funding; Novartis: Consultancy, Research Funding.