EHA 2021 | Phase I trial of CA-4948 in AML or MDS
Uwe Platzbecker, MD, University of Leipzig, Leipzig, Germany, discusses a novel oral IRAK4 inhibitor therapy for patients with relapsed/refractory high-risk myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML). CA-4948 works by interfering with the signaling pathways involving IRAK4, and its efficacy is still being explored. The data presented is preliminary, however, the Phase I study (NCT042787688) aims to determine the safety profile and tolerance of this novel agent in enrolled patients. Prof. Platzbecker reports that the study seems promising, with one patient achieving complete remission with full hematologic recovery so far. The results from this Phase I trial may lead to the investigation of utilizing CA-4948 as a combination therapy in future trials. This interview took place at the virtual European Hematology Association (EHA) Congress 2021.
Transcript (edited for clarity)
This trial actually investigates a novel agent, an IRAK4 inhibitor, for patients with relapsed/refractory high-risk MDS and AML patients. It’s an oral formulation, and the rationale behind is actually that, for instance, splices of mutated leukemic cells actually alter certain pathways, including the IRAK4 pathway; and by doing so, the, this pathway may become druggable by a specific IRAK4 inhibitor...
This trial actually investigates a novel agent, an IRAK4 inhibitor, for patients with relapsed/refractory high-risk MDS and AML patients. It’s an oral formulation, and the rationale behind is actually that, for instance, splices of mutated leukemic cells actually alter certain pathways, including the IRAK4 pathway; and by doing so, the, this pathway may become druggable by a specific IRAK4 inhibitor. IRAK4 is also important for FLT3 signaling, and therefore, this clinical trial is exploring this single agent treatment in this patient population.
The study is still ongoing. Therefore, the data presented will still be preliminary. But I think most important, because it’s a Phase I study, is that the agent is safe and is well-tolerated in the majority of the patients, and I think this was also the major result of the first couple of patients that we had. Among those, we had some very good responders, so even a CR was achieved in some patients, and I think this is pretty reassuring also for the study rationale, and I think makes the agent pretty interesting also for consecutive trials as a combination partner.
But I think still, so far, the study is ongoing, and the is data presented are I think preliminary, and so I think when the study matures, we will get a full impact, and of course a full, better view on the general results, and also what this agent may play a role for in the armamentarium of patients with high-risk MDS and AML.
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Disclosures
Uwe Platzbecker, MD, has received research support and honoraria from Curis, BMS, Amgen, Novartis and Abbvie.
