Menarini Group to Present New ELZONRIS Data at ASH 2025, Including Promising Triplet Therapy Results for BPDCN
核心洞察
Menarini Group (搜索) will present five abstracts on ELZONRIS (tagraxofusp-erzs) at the American Society of Hematology Annual Meeting in December, including two oral presentations focusing on blastic plasmacytoid dendritic cell neoplasm (搜索) treatment.
A phase 2 trial demonstrated that triplet therapy combining tagraxofusp, azacitidine, and venetoclax showed high response rates and successful bridge-to-transplant outcomes with good tolerability in BPDCN (搜索) patients.
Additional presentations will include real-world survival data comparing tagraxofusp to venetoclax and subgroup analyses examining treatment outcomes independent of baseline skin burden in treatment-naïve BPDCN (搜索) patients.
The Menarini Group (搜索) and its wholly-owned subsidiary Stemline Therapeutics (搜索) announced they will present new clinical data on ELZONRIS (tagraxofusp-erzs) at the 67th American Society of Hematology Annual Meeting and Exposition in Orlando, December 6-9, 2025. The presentations span five abstracts, including two oral presentations highlighting important findings for patients with blastic plasmacytoid dendritic cell neoplasm (搜索) (BPDCN (搜索)).
Triplet Therapy Shows Promise in Phase 2 Trial
The most significant presentation will feature results from a phase 2 trial examining a triplet therapy combining tagraxofusp, azacitidine, and venetoclax (TAG-AZA-VEN). According to the company, this combination demonstrated both efficacy with high response and bridge to transplant rates, as well as tolerability in individuals with BPDCN (搜索). The oral presentation is scheduled for December 7, 2025, from 5:30 PM to 5:45 PM.
"At Menarini Stemline, our commitment to transforming the lives of people living with difficult-to-treat cancers is unwavering," said Elcin Barker Ergun, CEO of the Menarini Group (搜索). "These data demonstrate that tagraxofusp plays an important role in both monotherapy and combination settings for the treatment of BPDCN (搜索), and that it also has potential to help patients living with other aggressive hematologic malignancies, where more effective treatment options are desperately needed."
Real-World Survival Analysis and Additional Studies
The presentations will also include a real-world analysis comparing survival outcomes between tagraxofusp and venetoclax in BPDCN (搜索) patients. This poster presentation, scheduled for December 7, will examine longer survival with tagraxofusp versus venetoclax based on real-world data.
Another key presentation will focus on favorable outcomes in treatment-naïve BPDCN (搜索) patients treated with tagraxofusp, including post-transplant survival data that appears independent of baseline skin burden. This subgroup analysis of a pivotal trial will be presented as a poster on December 8.
Understanding BPDCN and Current Treatment Landscape
BPDCN (搜索) is a highly aggressive, orphan hematologic malignancy that primarily affects skin, bone marrow, and blood. The disease can also impact lymph nodes, spleen, liver, and central nervous system. Approximately 85% to 90% of patients develop skin lesions as the first sign of BPDCN.
The global prevalence of BPDCN (搜索) is estimated at 0.4 to 0.5 per 100,000 people annually. While the disease affects both men and women of all ages, 75% of cases occur in men, with a median age at onset of 60-70 years. A key characteristic of BPDCN cancer cells is their high expression of CD123 (搜索) protein, which serves as both a crucial diagnostic marker and a prime target for precision therapies.
ELZONRIS Regulatory Status and Safety Profile
ELZONRIS was approved by the FDA in December 2018 for treating BPDCN (搜索) in adult and pediatric patients two years and older, covering both treatment-naïve and previously-treated populations. The European Commission granted approval in January 2021.
The drug carries a boxed warning for capillary leak syndrome (搜索) (CLS), which may be life-threatening or fatal. In clinical trials, the overall incidence of CLS was 53% (65/122 patients), with four fatalities (3%). The median time to onset was 4 days, with most events occurring in Cycle 1.
Other significant adverse reactions include hypersensitivity reactions in 43% of patients and hepatotoxicity with ALT elevations in 79% and AST elevations in 76% of patients. The most common adverse reactions (≥30% incidence) are capillary leak syndrome (搜索), nausea, fatigue, pyrexia, peripheral edema, and weight increase.