Antibody-drug conjugate shows promising safety and response rates for patients with rare blood cancer

The first-in-class antibody-drug conjugate (ADC) pivekimab sunirine (PVEK) demonstrated promising efficacy and high response rates for patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN), according to data from a Phase I/II study led by researchers from The University of Texas MD Anderson Cancer Center.

These results suggest that PVEK should be considered a potential new standard treatment option for patients with BPDCN, a rare, aggressive blood cancer that involves the skin, bone marrow and lymph nodes.

Findings from the CADENZA study were presented at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting by Naveen Pemmaraju, who led the trial together with Naval Daver.

As a frontline treatment in 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.

“Patients with BPDCN are in need of improved frontline therapies to treat their disease, so we’re very excited to see this trial going extremely well in terms of safety and efficacy,” Pemmaraju said. “The responses we have observed make PVEK a strong candidate as a standard-of-care treatment.”

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. MD Anderson researchers helped advance tagraxofusp-erzs toward Food and Drug Administration approval in 2018.

PVEK represents a next generation of CD123-targeted treatment. As an ADC, 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.

The global multi-center trial 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 with one, two or three prior lines of therapy. Participants received the therapy intravenously on day one of a 21-day cycle in an outpatient setting.

The most common side effects included peripheral edema, which was reversible and found to be manageable.

Given the results of this trial, there is potential for investigating combination therapies of CD123-targeted agent PVEK with other treatments active in BPDCN in future clinical trials, Pemmaraju explained.

The clinical trial was funded by AbbVie. Pemmaraju has served on the advisory board and as a consultant for AbbVie. Daver has received research support and has served on the advisory board and as a consultant for AbbVie.

The first-in-class antibody-drug conjugate (ADC) pivekimab sunirine (PVEK) demonstrated promising efficacy and high response rates for patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN), according to data from a Phase I/II study led by researchers from The University of Texas MD Anderson Cancer Center.

These results suggest that PVEK should be considered a potential new standard treatment option for patients with BPDCN, a rare, aggressive blood cancer that involves the skin, bone marrow and lymph nodes.

Findings from the CADENZA study were presented at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting by Naveen Pemmaraju, who led the trial together with Naval Daver.

As a frontline treatment in 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.

“Patients with BPDCN are in need of improved frontline therapies to treat their disease, so we’re very excited to see this trial going extremely well in terms of safety and efficacy,” Pemmaraju said. “The responses we have observed make PVEK a strong candidate as a standard-of-care treatment.”

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. MD Anderson researchers helped advance tagraxofusp-erzs toward Food and Drug Administration approval in 2018.

PVEK represents a next generation of CD123-targeted treatment. As an ADC, 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.

The global multi-center trial 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 with one, two or three prior lines of therapy. Participants received the therapy intravenously on day one of a 21-day cycle in an outpatient setting.

The most common side effects included peripheral edema, which was reversible and found to be manageable.

Given the results of this trial, there is potential for investigating combination therapies of CD123-targeted agent PVEK with other treatments active in BPDCN in future clinical trials, Pemmaraju explained.

The clinical trial was funded by AbbVie. Pemmaraju has served on the advisory board and as a consultant for AbbVie. Daver has received research support and has served on the advisory board and as a consultant for AbbVie.