The first-in-class antibody-drug conjugate pivekimab sunirine was found to be safe and effective, with high response rates, in patients with blastic plasmacytoid dendric cell neoplasm (BPDCN), according to data from a phase I/II study presented at the 2025 ASCO Annual Meeting (Abstract 6502).
These results, presented by Naveen Pemmaraju, MD, Professor of Leukemia at The University of Texas MD Anderson Cancer Center, suggest that pivekimab sunirine could one day become a new standard treatment option for patients with BPDCN, a rare, aggressive blood cancer that involves the skin, bone marrow, and lymph nodes. Dr. Pemmaraju led the trial together with Naval Daver, MD, Professor of Leukemia at MD Anderson.
Study Details
The global multi-center trial CADENZA study enrolled a total of 84 adult patients with CD123-positive BPDCN. Of these patients, 33 patients were newly diagnosed, and 51 patients had relapsed or refractory BPDCN and had received one, two, or three prior lines of therapy. Participants received pivekimab sunirine intravenously on day 1 of a 21-day cycle in an outpatient setting.
As a front-line treatment in the 33 newly diagnosed patients with BPDCN, researchers observed an overall response rate of 85%, including a complete response rate of 70% with a median overall survival of 16.6 months.
The most common side effects included peripheral edema, which was reversible and found to be manageable.
Next Steps
The current standard-of-care treatment for patients with BPDCN is tagraxofusp-erzs, which targets CD123 found at high levels on certain cancer cells, including BPDCN.
Pivekimab sunirine represents a next generation of CD123-targeted treatment. As an antibody-drug conjugate, it works by delivering a drug directly to cancer cells by targeting CD123 on the surface of BPDCN cells, resulting in death of the cancer cells.
“Patients with BPDCN are in need of improved front-line therapies to treat their disease, so we’re very excited to see this trial going extremely well in terms of safety and efficacy,” Dr. Pemmaraju said. “The responses we have observed make [pivekimab sunirine] a strong candidate as a standard-of-care treatment.”
Given the results of this trial, there is potential for investigating combination therapies of CD123-targeted agent pivekimab sunirine with other treatments active in BPDCN in future clinical trials, Dr. Pemmaraju explained.
Disclosure: This trial was funded by AbbVie. For full disclosures of the study authors, visit coi.asco.org.
