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

The primary-in-class antibody-drug conjugate (ADC) pivekimab sunirine (PVEK) demonstrated promising efficacy and excessive response charges for sufferers with blastic plasmacytoid dendritic cell neoplasm (BPDCN), in line with information from a Part I/II research led by researchers from The College of Texas MD Anderson Most cancers Heart.

These outcomes counsel that PVEK ought to be thought-about a possible new customary therapy possibility for sufferers with BPDCN, a uncommon, aggressive blood most cancers that entails the pores and skin, bone marrow and lymph nodes.

Findings from the CADENZA research had been introduced on the 2025 American Society of Scientific Oncology (ASCO) Annual Assembly by Naveen Pemmaraju, who led the trial along with Naval Daver.

As a frontline therapy in 33 newly identified sufferers with BPDCN, researchers noticed an total response charge of 85%, together with a whole response charge of 70% with a median total survival of 16.6 months.

“Sufferers with BPDCN are in want of improved frontline therapies to deal with their illness, so we’re very excited to see this trial going extraordinarily nicely when it comes to security and efficacy,” Pemmaraju stated. “The responses now we have noticed make PVEK a powerful candidate as a standard-of-care therapy.”

The present standard-of-care therapy for sufferers with BPDCN is tagraxofusp-erzs, which targets CD123 discovered at excessive ranges on sure most cancers cells, together with BPDCN. MD Anderson researchers helped advance tagraxofusp-erzs towards Meals and Drug Administration approval in 2018.

PVEK represents a subsequent era of CD123-targeted therapy. As an ADC, it really works by delivering a drug on to most cancers cells by focusing on CD123 on the floor of BPDCN cells, leading to demise of the most cancers cells.

The worldwide multi-center trial enrolled a complete of 84 grownup sufferers with CD123-positive BPDCN. Of those sufferers, 33 sufferers had been newly identified, and 51 sufferers had relapsed or refractory BPDCN with one, two or three prior traces of remedy. Members obtained the remedy intravenously on day one among a 21-day cycle in an outpatient setting.

The commonest negative effects included peripheral edema, which was reversible and located to be manageable.

Given the outcomes of this trial, there’s potential for investigating mixture therapies of CD123-targeted agent PVEK with different therapies energetic in BPDCN in future scientific trials, Pemmaraju defined.

The scientific trial was funded by AbbVie. Pemmaraju has served on the advisory board and as a guide for AbbVie. Daver has obtained analysis assist and has served on the advisory board and as a guide for AbbVie.