At the European Hematology Association (EHA) 2026 Congress, updated results from the Phase Ia RevSTAR-123 trial were presented on June 13, 2026. The study evaluated AvenCell Therapeutics’ AVC-201-01, a first-in-class switchable allogeneic chimeric antigen receptor (CAR)-T cell therapy, in patients with interleukin-3 receptor alpha chain (CD123)-positive relapsed/refractory (R/R) or minimal residual disease (MRD)-positive acute myeloid leukemia (AML).

AVC-201-01 is differentiated by two key design features. First, it is an allogeneic product manufactured from healthy donor T cells, which could help address the urgency of R/R AML, where patients often cannot wait for autologous manufacturing and may have poor-quality T cells after multiple prior lines of therapy. Second, it uses AvenCell’s RevCAR platform, in which CAR-T activation depends on a separately infused CD123 targeting module. This creates a pharmacological on-and-off switch, allowing CAR-T activity to be modulated by controlling the targeting module rather than relying on an irreversible, continuously active CD123 CAR-T. This is clinically important because CD123 is attractive in AML but biologically difficult, as it is expressed on leukemic blasts and leukemic stem cells, while also being present on normal hematopoietic progenitors and endothelial cells.

In the RevSTAR-123 study, AvenCell’s AVC-201-01 generated an early but meaningful signal in the high-risk AML population, supporting the initial validation of its switchable allogeneic CD123 CAR-T platform. Among 17 treated patients, the population was heavily pretreated with a median of four prior therapies. Specifically, 10 patients had received prior allogeneic hematopoietic stem cell transplant (allo-HSCT), and nine patients classified as European LeukemiaNet (ELN) adverse risk. Safety was manageable, with cytokine release syndrome (CRS) occurring in 13 patients, only one of which was a grade 3. There was no immune effector cell-associated neurotoxicity syndrome (ICANS), no graft-versus-host disease (GvHD), no treatment-related mortality, and one dose-limiting toxicity (DLT) at the highest dose. Efficacy was concentrated at higher doses, with 3/8 evaluable patients at dose level (DL) 12/ DL15 achieving complete remission/complete remission with partial hematologic recovery (CR/CRh), including two MRD-negative complete remissions.

If successfully developed and approved, AVC-201-01 would enter the R/R AML market, which has been shaped by biomarker-selected targeted therapies and emerging cellular approaches. A key commercial benchmark is Astellas’ Xospata (gilteritinib), an oral FMS-like tyrosine kinase 3 (FLT3) inhibitor approved for FLT3-mutated R/R AML, which benefits from outpatient administration and Phase III survival validation, but is limited to a molecularly defined patient subgroup. In contrast, AVC-201-01 could offer broader applicability through interleukin-3 receptor alpha chain (CD123) expression, particularly for patients without actionable mutations, although it will need to show durable MRD conversion, manageable toxicity, and effective bridging to transplant in order to justify a more complex cellular therapy approach. AbbVie’s Decnupaz (pivekimab sunirine), a CD123-directed antibody-drug conjugate that has been approved for blastic plasmacytoid dendritic cell neoplasm and is under evaluation in AML through the Phase III REVIVAL study, further reinforces CD123 as a clinically relevant myeloid target. AVC-201-01 is differentiated by its switchable allogeneic CAR-T design, and its value will depend on delivering deeper and more durable remissions than non-cellular CD123-directed approaches.

Commercially, AVC-201-01 is attractive but remains at high risk. Its allogeneic format could shorten manufacturing timelines and improve scalability in rapidly progressing R/R AML, while its switchable CD123 targeting module may offer an important safety and dosing advantage in a narrow therapeutic window. However, its adoption will depend on clear evidence of deeper responses, MRD negativity, durable remission, manageable CRS and cytopenias, and successful bridging to transplant. AVC-201-01 should therefore be viewed as an early validation of a next-generation AML CAR-T concept, not yet a practice-changing therapy.