New Antibody-Drug Conjugate Shows Promising Results for Rare Blood Cancer Treatment

Researchers at The University of Texas MD Anderson Cancer Center have unveiled encouraging results from a Phase I/II clinical trial of the first-in-class antibody-drug conjugate (ADC) pivekimab sunirine (PVEK) for patients diagnosed with blastic plasmacytoid dendritic cell neoplasm (BPDCN), a rare and aggressive blood cancer affecting the skin, bone marrow, and lymph nodes.

The findings, presented during the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting, indicate that PVEK could serve as a new standard treatment option for BPDCN. In the CADENZA study, which enrolled 84 adult participants with CD123-positive BPDCN, the overall response rate was measured at an impressive 85%, with 70% of patients achieving a complete response. The median overall survival for participants was recorded at 16.6 months.

With current treatment options being limited, the results of this trial are particularly significant. The previous standard of care for BPDCN patients was tagraxofusp-erzs, which targets the CD123 molecule present in high concentrations on certain cancer cells, including those associated with BPDCN. Researchers from MD Anderson played a pivotal role in advancing tagraxofusp-erzs toward FDA approval in 2018.

PVEK represents a next-generation treatment that delivers cytotoxic medication directly to cancer cells by specifically targeting CD123 on BPDCN cells, leading to their destruction. Among the 84 patients involved in the global multi-center trial, 33 were newly diagnosed, while the remaining 51 had experienced relapses or had refractory BPDCN following one to three previous treatment lines. The therapy was administered intravenously in an outpatient setting on the first day of a 21-day treatment cycle.

Notably, the most frequently reported side effect was peripheral edema, which was manageable and reversible. Given the encouraging results of this trial, there is a potential avenue for future investigations into combination therapies that incorporate PVEK alongside other active treatments for BPDCN.

The clinical trial received funding from AbbVie, and it is worth noting that researchers involved in the study have connections with the company, serving in advisory and consultancy roles.