A novel antibody-drug conjugate, pivekimab sunirine (PVEK), has demonstrated high complete response rates and durable responses in patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN), a rare and aggressive blood cancer, according to new data published today in the Journal of Clinical Oncology.
The Phase I/II CADENZA trial, an open-label, multicenter international study, showed a composite complete response rate of 75% (95% confidence interval: 51%-91%) among 20 patients with front-line, de novo BPDCN. The median duration of response was 10.6 months (95% CI: 3.8 to not reached), and the median overall survival was 16.6 months (95% CI: 7.2 to not reached). Researchers at The University of Texas MD Anderson Cancer Center, who led the trial, reported that 8 of 15 eligible patients proceeded to stem cell transplantation following treatment.
“These strong, durable response results offer hope to patients with BPDCN with limited treatment options,” said Naveen Pemmaraju, M.D., Professor of Leukemia at MD Anderson and the study’s principal investigator. “An effective and safe front-line treatment for patients would be practice changing, and these positive results suggest that pivekimab sunirine should be considered a potential standard treatment for patients with BPDCN.”
BPDCN is characterized by the overexpression of the CD123 antigen, which PVEK targets. The antibody-drug conjugate delivers a cytotoxic agent directly to cancer cells, minimizing impact on healthy tissue. The CADENZA trial evaluated PVEK in both front-line and relapsed/refractory settings. Whereas the response rate was lower in patients with relapsed or refractory disease – 14% complete response rate and 35% overall response rate – those who responded experienced a median duration of response of 9.2 months.
The study enrolled a total of 84 patients, with 33 receiving treatment in the front-line setting and 51 with relapsed or refractory disease. Treatment was administered at a dose of 0.045 mg/kg every three weeks.
Adverse events were reported in 99% of patients, with Grade 3 or higher events occurring in 79%. The most common adverse events included peripheral edema (54%), fatigue (26%), and infusion-related reactions (26%). More serious adverse events, such as neutropenia (16%), thrombocytopenia (14%), and pneumonia (6%), were also observed. Two cases of on-treatment veno-occlusive disease were reported, but both resolved.
Naval Daver, M.D., Professor of Leukemia at MD Anderson and senior author of the study, noted the potential for broader applications of the treatment. “We are encouraged by what we are seeing in the treatment of BPDCN, and early results also indicate encouraging efficacy in frontline acute myeloid leukemia, including in combination approaches,” he said.
AbbVie supported the research. A Biologics License Application (BLA) for PVEK in BPDCN was submitted to regulatory authorities in September 2025, based on the CADENZA trial data, potentially positioning the drug as a new standard of care.
