Pivekimab Sunirine Yields Enduring Responses in Frontline BPDCN Trial

First-line treatment with pivekimab sunirine demonstrated durable responses and a manageable safety profile among patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN), according to data from the phase 1/2 CADENZA trial (NCT03386513) published in the Journal of Clinical Oncology.¹

Among 20 patients with de novo disease, the complete response (CR) plus clinical CR (CRc) rate was 75% (95% CI, 51%-91%), and the overall response rate (ORR) was 80% (95% CI, 56%-94%). Additionally, data showed a median duration of CR plus CRc of 10.6 months (95% CI, 3.8-not reached [NR]) and a median time to response of 1.4 months (IQR, 1-3). Regarding 33 patients with frontline disease, the CR plus CRc rate was 70% (95% CI, 51%-84%), and the ORR was 85% (95% CI, 68%-95%). In this cohort, the median duration of CR plus CRc was 9.8 months (95% CI, 4.6-NR), with a median time to response of 1.5 months (IQR, 1-3).

Among 51 patients with relapsed/refractory disease, pivekimab sunirine produced a CR plus CRc rate of 14% (95% CI, 6%-26%) and an ORR of 35% (95% CI, 22%-50%). Moreover, the median duration of CR plus CRc was 9.2 months (95% CI, 2.4-NR), and the median time to response was 1.4 months (IQR, 1-2).

Investigators noted responses in each patient group regardless of CD123 levels. The CR plus CRc rates were typically comparable across prespecified subgroups, although patients who received treatment after stem cell transplant (SCT) experienced higher response rates.

“These strong, durable response results offer hope to [patients with] BPDCN with limited treatment options,” lead study author Naveen Pemmaraju, MD, a professor in the Department of Leukemia and the Division of Cancer Medicine at the University of Texas MD Anderson Cancer Center, said in a press release regarding these findings.² “An effective and safe frontline 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.”

The open-label phase 1/2 CADENZA study included 2 prospectively recruited groups of patients: those with de novo BPDCN or with a prior or concomitant hematologic malignancy without prior systemic therapy (n = 33), and those with relapsed/refractory BPDCN who received 1 to 3 prior lines of systemic treatment (n = 51). Patients received pivekimab sunirine at the recommended phase 2 dose of 0.045 mg/kg every 3 weeks.

The trial’s primary end point was the CR plus CRc rate per investigator evaluation. The duration of CR plus CRc in patients with frontline de novo BPDCN was a key secondary end point.

Across an overall population of 84 patients, the median age was 72 years (range, 19-85), and most patients were male (82%) and White (82%). Most of the study population had an ECOG performance status of 1 (61%), disease involved in the skin (77%), normal hepatic function (83%), normal renal function (46%), de novo disease (76%), and 1 prior line of treatment (35%).

Among all evaluable patients, any adverse effect (AE) occurred in 99% of those who received pivekimab sunirine, and 79% experienced grade 3 or higher AEs. Toxicities associated with dose delays, dose reductions, and treatment discontinuation occurred in 25%, 5%, and 13% of patients, respectively. The most common AEs of any grade included peripheral edema (54%), fatigue (26%), infusion-related reactions (26%), nausea (20%), and hypokalemia (20%).

In September 2025, developers submitted a biologics license application (BLA) seeking approval for pivekimab sunirine as a treatment for those with BPDCN.³ Supporting data for the BLA came from the CADENZA trial.

“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,” study author Naval Daver, MD, a professor and director of the Leukemia Research Alliance Program in the Department of Leukemia at MD Anderson Cancer Center, said in the press release.²

References

1. Pemmaraju N, Marconi G, Montesinos P, et al. Pivekimab sunirine in blastic plasmacytoid dendritic cell neoplasm. J Clin Oncol. Published online February 11, 2026. doi:10.1200/JCO-25-02083
2. Antibody-drug conjugate achieves high response rates as frontline treatment in aggressive, rare blood cancer. News release. The University of Texas MD Anderson Cancer Center. February 11, 2026. Accessed February 13, 2026. https://tinyurl.com/2v5p7s3j
3. AbbVie submits biologics license application (BLA) to U.S. FDA for pivekimab sunirine (PVEK) - an investigational antibody-drug conjugate (ADC) to treat rare cancer with limited treatment options. News release. AbbVie. September 30, 2025. Accessed February 13, 2026. https://tinyurl.com/ykzujenf