Treatment with the novel CD123-targeting antibody-drug conjugate pivekimab sunirine led to a high rate of complete and durable responses in patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN), especially among patients being treated in the front-line setting. The antibody-drug conjugate also showed a manageable safety profile. New results from the phase I/II CADENZA trial were published in the Journal of Clinical Oncology.
“These strong, durable response results offer hope to patients [with BPDCN] with limited treatment options,” said principal investigator and corresponding study author Naveen Pemmaraju, MD, Professor of Leukemia, The University of Texas MD Anderson Cancer Center. “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].”
Study Methods
CADENZA is an open-label, multicenter, international phase I/II clinical trial evaluating the efficacy of pivekimab sunirine in adults with front-line or relapsed or refractory BPDCN. In the study, front-line was defined as receiving no prior systemic therapy as well as de novo BPDCN or a coexisting blood cancer.
Treatment was administered at 0.045 mg/kg once every 3 weeks.
The primary outcome measure was composite complete response rate, consisting of complete responses and clinical complete responses, in the primary analysis population (patients with front-line de novo BPDCN who enrolled following a protocol amendment to define the de novo population).
Prior results from the study were presented at the 2025 ASH Annual Meeting & Exposition (Abstract 5195) as well as at the 2025 ASCO Annual Meeting (Abstract 6502).
Key Findings
The study enrolled 84 patients, including 33 in the front-line group (of whom 20 were de novo and considered the primary analysis population) and 51 in the relapsed/refractory group.
The composite complete response rate was 75% (95% confidence interval [CI] = 51%–91%), with a median duration of response of 10.6 months (95% CI = 3.8 to not reached). The median overall survival was 16.6 months (95% CI = 7.2 to not reached). Among 15 eligible patients, 8 underwent stem cell transplantation.
In patients with relapsed/refractory disease, the complete response rate was 14% (95% CI = 6%–26%), with a median duration of response of 9.2 months (95% CI = 2.4 to not reached). The median overall survival was 5.8 months (95% CI = 3.9–8.4).
The most common adverse events were peripheral edema (54%), fatigue (26%), and infusion-related reactions (26%). Grade 3 or higher events included neutropenia (16%), thrombocytopenia (14%), and peripheral edema (12%). Serious adverse events of pneumonia were reported in 6% and febrile neutropenia in 5%. Additionally, two cases of on-treatment veno-occlusive disease were reported, but both were reversible.
“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," said senior author Naval Daver, MD, Professor of Leukemia, The University of Texas MD Anderson Cancer Center.
DISCLOSURE: This research was supported by AbbVie. For full disclosures of the study authors, visit ascopubs.org.
