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Last update 22 Dec 2025

Tagraxofusp-ERZS

Last update 22 Dec 2025

Overview

R&D Status

Clinical Result

Translational Medicine

Overview

Basic Info

Drug Type

Fusion protein

Synonyms

Diphtheria toxin-il-3 fusion protein targeting IL-3 receptor, Diphtheria-toxin-interleukin-3-fusion-protein, Tagraxofusp

+ [10]

Target

CD123

Action

inhibitors

Mechanism

CD123 inhibitors(Interleukin-3 receptor subunit alpha inhibitors)

Therapeutic Areas

NeoplasmsImmune System DiseasesHemic and Lymphatic Diseases+ [1]

Active Indication

Blastic Plasmacytoid Dendritic Cell NeoplasmCD123 Positive Acute Myeloid LeukemiaAcute Myeloid Leukemia+ [7]

Inactive Indication

Acute Promyelocytic LeukemiaAdult Lymphoblastic LymphomaMultiple Myeloma+ [6]

Originator Organization

Texas A&M University

Active Organization

Stemline Therapeutics, Inc.Menarini Von Heyden GmbH

The University of Texas MD Anderson Cancer Center

+ [1]

Inactive Organization

The University of Texas Southwestern Medical Center

License Organization

A. Menarini Industrie Farmaceutiche Riunite Srl

Nippon Shinyaku Co., Ltd.Menarini Von Heyden GmbH

Drug Highest PhaseApproved

First Approval Date

United States (21 Dec 2018),

Blastic Plasmacytoid Dendritic Cell Neoplasm

RegulationBreakthrough Therapy (United States), Orphan Drug (United States), Orphan Drug (Japan), Orphan Drug (Australia)

Structure/Sequence

Sequence Code 453129448

Source: *****

Clinical Trials associated with Tagraxofusp-ERZS

NCT07148180

/ RecruitingPhase 1/2IIT

A Multi-Site Break Through Cancer Trial: Targeting Measurable Residual Disease in Patients With Acute Myeloid Leukemia: A Phase 1/2 Study of Tagraxofusp, Azacitidine, and Venetoclax

The purpose of this research study is to test the safety and efficacy of a new drug combination with three agents, azacitidine, venetoclax and tagraxofusp. Leftover (residual) leukemia disease that is not visible by eye can be increase the chance of disease recurrence. This research study is to determine if the combination therapy can safely help to control residual Acute Myeloid Leukemia (AML) and to prevent disease recurrence.
The names of the study drugs involved in this study are:
* Tagraxofusp (a type of CD123-directed cytotoxin)
* Azacitidine (a type of standard of care cytidine nucleoside analog)
* Venetoclax (a type of standard of care BCL-2 inhibitor)

Start Date01 Feb 2026

Sponsor / Collaborator

Dana-Farber Cancer Institute, Inc.

[+1]

NCT07007052

/ Not yet recruitingPhase 2IIT

Open Label Phase II Study Evaluating the Efficacy and Safety of the Combination of Tagraxofusp and Venetoclax in Treatment-naive Blastic Plasmacytoid Dendritic Cell Neoplasm Patients

The goal of this clinical trial is to study the efficacy of the tagraxofusp + venetoclax combination in treatment-naive blastic plasmacytoid dendritic cell neoplasm adult patients.
The main question is to verify the response in patients after 3 cycles of tagraxofusp+venetolax and to demonstrate if the combination of tagraxofusp + venetoclax increases the rate of complete remission, assessed after 3 months of treatment.
Patients will receive a ramp-up phase of venetoclax during 3 days and at least 3 cycles of venetoclax. After, the investigators will evaluate the response, and depending on the response observed, patients may receive additional cycles of treatment for a maximum of 24 cycles, or receive an allograft or discontinue the treatment in the case of therapeutic failure.

Start Date01 Dec 2025

Sponsor / Collaborator

Stemline Therapeutics, Inc.

[+1]

CTIS2024-511102-22-00

/ RecruitingPhase 2

Phase II study of standard of care therapy azacitidine (AZA) combined with venetoclax (VEN) and Tagraxofusp (TAG) in patients with higher-Risk chronic myelomonocytic Leukemia (CMML): the PATROL Study

Start Date13 May 2025

Sponsor / Collaborator

GCP Service International Ltd & Co KG

100 Clinical Results associated with Tagraxofusp-ERZS

100 Translational Medicine associated with Tagraxofusp-ERZS

100 Patents (Medical) associated with Tagraxofusp-ERZS

229

Literatures (Medical) associated with Tagraxofusp-ERZS

01 Nov 2025·Blood neoplasia

Tagraxofusp, a CD123-targeted therapy, for chronic myelomonocytic leukemia: final results of a phase 1/2 study

Article

Author: Badar, Talha ; Ali, Haris ; Lindsay, Ross ; Yacoub, Abdulraheem ; Talpaz, Moshe ; Wang, Eunice S ; Garcia-Manero, Guillermo ; Gupta, Vikas ; Galleu, Antonio ; Lasho, Terra L ; Foran, James M ; Mangaonkar, Abhishek A ; Pemmaraju, Naveen ; Stevens, Don Ambrose ; Wall, Sarah ; Patnaik, Mrinal M ; Gupta, Ira ; Schiller, Gary J

Chronic myelomonocytic leukemia (CMML) is an aggressive hematologic neoplasm characterized by an expansion of CD123⁺ monocytes and plasmacytoid dendritic cells (pDCs). pDC bone marrow clusters in CMML have been associated with higher rates of acute myeloid leukemia transformation. We evaluated tagraxofusp, a CD123-targeted therapy, in a phase 1/2 trial for patients with CMML. There were no dose-limiting toxicities. At the recommended phase 2 dose of 12 μg/kg per day, 37 patients were treated: 15 were treatment naïve; 22 had relapsed/refractory disease (median number of previous therapies, 1 [range, 1-7]). Common nonhematologic treatment-emergent adverse events (AEs) included fatigue (49%), hypoalbuminemia (46%), nausea, hypokalemia, and decreased appetite (44% each). Capillary leak syndrome occurred in 9 patients (23%; grade 3-4, 13%), whereas tumor lysis syndrome was seen in 13%. Hematologic grade 3/4 treatment-related AEs included thrombocytopenia (28%), anemia (13%), leukocytosis (15%), and neutropenia (13%). No complete or partial responses were observed. One patient each in the treatment-naïve and relapsed/refractory groups achieved complete cytogenetic remission with marrow response. Stable disease and clinical benefit were achieved by 40% and 27% of treatment-naïve patients and by 59% and 23% of relapsed/refractory patients, respectively. After a median follow-up of 43.7 months, overall survival was 11.2 months in treatment-naïve and 15.6 months in relapsed/refractory patients. Exploratory analysis showed stable CD123⁺ blast frequency, mutational variant allele frequencies, and monocyte subsets with treatment. Tagraxofusp demonstrated a manageable safety profile with limited clinical efficacy in CMML. This trial was registered at www.ClinicalTrials.gov as #NCT02268253.

12 Sep 2025·Hematology/oncology and stem cell therapy

Allogeneic hematopoietic cell transplantation as the standard of care for blastic plasmacytoid dendritic cell neoplasm in first complete remission.

Article

Author: Kharfan-Dabaja, Mohamed A

Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare hematologic disorder derived from precursors of plasmacytoid dendritic cells, with a generally aggressive clinical course. Tagraxofusp, a first-in-class CD123-directed antibody conjugate comprising human interleukin-3 and truncated diphtheria toxin, represents a welcome addition to the treatment armamentarium for BPDCN. Allogeneic hematopoietic cell transplantation (allo-HCT) is a treatment modality with curative potential in patients with BPDCN, based on data derived from registry studies, as well as multicenter and single-center retrospective studies. Several studies and a systematic review/meta-analysis have shown superior survival when patients undergo allo-HCT in first complete remission (CR1). In younger or fit patients, a myeloablative conditioning (MAC) allo-HCT regimen is the preferred approach whenever an allograft is indicated. Incorporating total body irradiation within the MAC regimen has been shown to improve progression-free survival, disease-free survival, and overall survival. However, MAC regimens have limited applicability in older, frail, or less fit patients, for whom reduced-intensity conditioning is the most appropriate approach. Several questions remain unanswered-namely, the potential benefit of post-transplant consolidation or maintenance strategies to mitigate the risk of relapse or progression, and the role of next-generation sequencing as a prognostic tool and for better defining depth of remission and measurable residual disease, among others. We believe that further improvement in the prognosis of BPDCN will require large collaborative efforts, considering the rarity of this disease.

01 Sep 2025·Chinese Journal of Integrative Medicine

Suppression of Hepatocellular Carcinoma through Apoptosis Induction by Total Alkaloids of Gelsemium elegans Benth.

Article

Author: Zhao, Yu-Qian ; Zhao, Xiang-Pei ; Li, Yan-Ping ; Zhou, Huan-Si ; Lu, Chun-Hua ; Qin, Mei-Jing ; Yang, Mei ; Jin, Ming-Jing

OBJECTIVE:

To evaluate the anti-hepatocellular carcinoma (HCC) activity of total alkaloids from Gelsemium elegans Benth. (TAG) in vivo and in vitro and to elucidate their potential mechanisms of action through transcriptomic analysis.

METHODS:

TAG extraction was conducted, and the primary components were quantified using high-performance liquid chromatography (HPLC). The effects of TAG (100, 150, and 200 µg/mL) on various tumor cells, including SMMC-7721, HepG2, H22, CAL27, MCF7, HT29, and HCT116, were assessed. Effects of TAG on HCC proliferation and apoptosis were detected by colony formation assays and cell stainings. Caspase-3, Bcl-2, and Bax protein levels were detected by Western blotting. In vivo, a tumor xenograft model was developed using H22 cells. Totally 40 Kunming mice were randomly assigned to model, cyclophosphamide (20 mg/kg), TAG low-dose (TAG-L, 0.5 mg/kg), and TAG high-dose (TAG-H, 1 mg/kg) groups, with 10 mice in each group. Tumor volume, body weight, and tumor weight were recorded and compared during 14-day treatment. Immune organ index were calculated. Tissue changes were oberseved by hematoxylin and eosin staining and immunohistochemistry. Additionally, transcriptomic and metabolomic analyses, as well as quatitative real-time polymerase chain reaction (RT-qPCR), were performed to detect mRNA and metabolite expressions.

RESULTS:

HPLC successfully identified the components of TAG extraction. Live cell imaging and analysis, along with cell viability assays, demonstrated that TAG inhibited the proliferation of SMMC-7721, HepG2, H22, CAL27, MCF7, HT29, and HCT116 cells. Colony formation assays, Hoechst 33258 staining, Rhodamine 123 staining, and Western blotting revealed that TAG not only inhibited HCC proliferation but also promoted apoptosis (P<0.05). In vivo experiments showed that TAG inhibited the growth of solid tumors in HCC in mice (P<0.05). Transcriptomic analysis and RT-qPCR indicated that the inhibition of HCC by TAG was associated with the regulation of the key gene CXCL13.

CONCLUSION:

TAG inhibits HCC both in vivo and in vitro, with its inhibitory effect linked to the regulation of the key gene CXCL13.

News (Medical) associated with Tagraxofusp-ERZS

11 Dec 2025

·www.prnewswire.com

Five Leading Cancer Centers Unite to Target CD123 and Eradicate Hidden Leukemia Cells

A multi-site trial pairs targeted therapy with next-generation MRD testing to track residual disease and uncover vulnerabilities in leukemia cells CAMBRIDGE, Mass., Dec. 11, 2025 /PRNewswire/ -- Acute myeloid leukemia (AML) is an aggressive blood cancer, with overall survival remaining approximately 30% at five years despite advances in therapy. Even when patients achieve remission, the disease often returns, fueled by a small number of leukemia cells that survive initial treatment, known as measurable residual disease (MRD). These remaining cells are major drivers of relapse and are linked with poorer outcomes, yet no standard therapy exists to eliminate them. Addressing MRD has become one of the most urgent clinical challenges in AML, and the focus of the 'Eradicating Measurable Residual Disease in AML' Break Through Cancer TeamLab. Continue Reading "MRD is like the bulk of an iceberg hidden beneath the surface. We can treat the disease we see, but the real challenge is determining how much remains unseen" said Jacqueline Garcia, assistant professor of medicine at Harvard Medical School and medical oncologist at Dana-Farber Cancer Institute and lead investigator on the trial. "We have billions of blood cells in our body, and the questions are: How much disease is truly left over? How do we measure it? And how do we quantify our patient's risk?" Five cancer centers unite to target CD123 and eliminate residual leukemia cells driving relapse in AML patients Post this Why Target CD123 CD123, a leukemia cell-surface marker present in more than 80% of cases, is involved in cancer cell growth and survival and is especially elevated in high-risk subtypes. "We wanted a study that could have broad reach across more subgroups of patients," said Garcia. "CD123 is present on the surface of AML cells and early leukemia stem cells which makes it a meaningful way to target residual disease." This multi-site study will evaluate targeting CD123 in combination with two established AML therapies. Tagraxofusp (Stemline), a CD123-directed immunotoxin already FDA-approved for another blood cancer, "binds to cells expressing CD123, and delivers a diptheria toxin payload directly into the cell," Garcia explained. Work led by fellow TeamLab investigator Dr. Andrew Lane, co-leader of the Dana-Farber/Harvard Cancer Center Leukemia Program, showed that azacitidine and venetoclax can boost sensitivity and enhance anti-leukemic activity in this combination, forming the scientific rationale for combining all three therapies. "By adding two approved treatments for active AML, we're testing if this combination can safely eradicate leftover disease and convert patients from MRD positive to negative" Garcia noted. Early clinical signals reinforce this strategy. In a recent phase 1b study in patients with newly diagnosed AML, this combination achieved a 69% response rate, and among patients in remission, nearly 71% achieved MRD-negativity. A Trial Designed to Learn as well as Treat This phase 1/2 trial (NCT07148180) is now active at Dana-Farber Cancer Institute and will be jointly conducted across four of Break Through Cancer's clinical centers including Memorial Sloan Kettering Cancer Center, The University of Texas MD Anderson Cancer Center, and the Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins. Harnessing the deep expertise within the Eradicating Measurable Residual Disease in AML TeamLab across the four clinical sites and MIT's Koch Institute for Integrative Cancer Research, this study will generate an unusually rich scientific dataset. Blood and bone marrow samples collected throughout treatment will be analyzed using state-of-the-art approaches such as circulating tumor DNA profiling, single-cell sequencing and immunophenotyping, and advanced proteomic analyses. "Alongside routine clinical tests, we are also developing new ultrasensitive assays to detect low levels of residual disease and running research assays to learn why certain leukemia cells persist," added Garcia. Together, these integrated analyses are designed to monitor how patients respond, reveal why MRD occurs, how it evolves, and which vulnerabilities may be exploited to improve future treatment strategies. By pairing deep biological discovery with clinical testing, the TeamLab aims to both eradicate MRD today and reshape how residual disease is detected and addressed in AML going forward. "This study is part of Break Through Cancer's broader work addressing minimal residual disease," said Tyler Jacks, PhD, president of Break Through Cancer. "Eliminating every last cancer cell is a formidable challenge, but one we can meaningfully advance when apply to best of science and work together." About Break Through Cancer Founded in 2021, Break Through Cancer empowers outstanding researchers and physicians to both intercept and find cures for several of the deadliest cancers by stimulating radical collaboration among outstanding cancer research institutions, including its founding partners: Dana-Farber Cancer Institute, Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Memorial Sloan Kettering Cancer Center, MIT's Koch Institute for Integrative Cancer Research, and The University of Texas MD Anderson Cancer Center. The Foundation is supported by a Board of Directors from the five partner institutions and a Scientific Advisory Board of U.S. cancer experts. The Foundation was launched with an extraordinary challenge pledge of $250 million from Mr. and Mrs. William H. Goodwin, Jr. and their family, and the estate of William Hunter Goodwin III. For further information, please visit the Foundation's website at . Media Contact: Soracha Ward [email protected] SOURCE Break Through Cancer 21% more press release views with Request a Demo

Clinical ResultPhase 1Immunotherapy

08 Dec 2025

·www.globenewswire.com

Menarini Group Presents Elacestrant (ORSERDU®) Phase 2 Combination Data in Patients with ER+, HER2- Metastatic Breast Cancer (mBC) at the 2025 San Antonio Breast Cancer Symposium

In addition, the expansive elacestrant clinical development program updates reinforce its potential as a backbone endocrine therapy in combination settings, as well as in both metastatic and early stage breast cancer. FLORENCE, Italy and NEW YORK, Dec. 08, 2025 (GLOBE NEWSWIRE) -- The Menarini Group (“Menarini”), a leading international pharmaceutical and diagnostics company, and Stemline Therapeutics, Inc. (“Stemline”), a wholly-owned subsidiary of the Menarini Group, focused on bringing transformational oncology treatments to cancer patients, will present updated meaningful median progression-free survival (mPFS) efficacy results from two combination regimens of the Phase 2 ELEVATE study in patients with estrogen receptor-positive (ER+), HER2-negative (HER2-) locally advanced or metastatic breast cancer (mBC). The ELEVATE study was designed to evaluate the safety and efficacy of oral-oral combination treatment options to overcome different resistance mechanisms observed in ER+/HER2- mBC with the goal of improving patient outcomes. These data will be presented at the 2025 San Antonio Breast Cancer Symposium (SABCS), December 9-12. “The encouraging progression-free survival data increase our confidence in the role elacestrant could play as an endocrine therapy backbone in the combination setting,” said Virginia Kaklamani, MD, DSc, Professor of Medicine in the Division of Hematology/Oncology at UT Health San Antonio, and Leader of the Breast Cancer Program at UT Health San Antonio MD Anderson Cancer Center. “The safety profile of elacestrant in combination with everolimus or abemaciclib is consistent with the known safety profiles of each drug. No new safety signals have been observed.” The ELEVATE data to be presented at SABCS demonstrate that elacestrant in combination with everolimus or with abemaciclib shows a consistent PFS benefit, irrespective of ESR1 mutation status in patients with ER+/HER2- mBC, who experience disease progression on endocrine therapy (ET), with or without prior exposure to CDK4/6 inhibitors. These updated results also show that the safety of the combinations are consistent with the known safety profiles of each targeted therapy plus standard of care endocrine therapy. Phase 2 mPFS in months (95% CI) in all patients and subgroups Patient Population Elacestrant 345 mg QD+ Everolimus 7.5mg QD(n=50)Elacestrant 345 mg QD+ Abemaciclib 150 mg BID(n=60)All patients 8.3 [4.0 – 10.2]14.3 [7.3-16.6]Visceral disease 7.7 [3.8 – 9.4]14.3 [7.4-16.6]No prior fulvestrant 8.3 [4.2 – 12.9]14.8 [8.7-NR]No primary endocrine resistance 8.3 [4.0-12.9]14.3 [7.3-16.6]ESR1mut 8.7 [3.5 – 12.9]*ESR1wt 9.0 [4.2 – 12.7]*PIK3CAmut 8.3 [3.6 - 10.2]*PIK3CAwt 9.6 [5.6 - NR]* *Maturity not reached for PFS (95% CI) for genomic subgroups (ESR1 / PIK3CA) in the elacestrant + abemaciclib cohort. “The extensive evidence for elacestrant spans the monotherapy setting in our pivotal EMERALD study, now backed by two recent real-world data publications1, 2, and in its growing potential in combination regimens, as highlighted by the data presented at SABCS,” said Elcin Barker Ergun, CEO of the Menarini Group. “We remain deeply committed to fully exploring elacestrant’s potential benefit across multiple ongoing trials in both early stage and metastatic breast cancer.” Additionally, other elacestrant updates will be presented at SABCS, investigating its potential across the spectrum of breast cancer: Presentation Title: Elacestrant in combination with everolimus or abemaciclib in patients with ER+/HER2-locally advanced or metastatic breast cancer (mBC): phase 2 results from ELEVATE, an open-label, umbrella studyAbstract Number: 1255Presentation Date & Time: Thursday, December 11, 2025, 1:00 – 2:00 PM Location: Hemisfair1-2Presenting Author: Hope S. Rugo Presentation Title: Elacestrant alone or in combination with triptorelin in premenopausal women with ER+/HER2-early breast cancer: primary analysis from the phase 2 SOLTI-2104-PremiÈRe trialAbstract Number: 1123Presentation Date & Time: Friday, December 12, 2025, 7:30 – 7:33 AMLocation: 301 ABCPresenting Author: Mertixell Bellet Presentation Title: ELEGANT: Elacestrant Versus Standard Endocrine Therapy (ET) in Women and Men With Node-positive, Estrogen Receptor-positive (ER+), HER2-negative (HER2-), Early Breast Cancer (eBC) With High Risk of Recurrence in a Global, Multicenter, Randomized, Open-label Phase 3 Study.Abstract Number: 1276Presentation Date & Time: Thursday, December 11, 2025, 12:30 – 2:00 PMLocation: Henry B. Convention CenterPresenting Author: Aditya Bardia Presentation Title: The ADELA study: A Double-blind, Placebo-controlled, Randomized Phase 3 Trial of Elacestrant (ELA)+ Everolimus (EVE) Versus ELA + Placebo (PBO) in ER+/HER2-Advanced Breast Cancer (aBC) Patients with ESR1-mutated Tumors Progressing on Endocrine Therapy (ET) + CDK4/6iAbstract Number: 1141Presentation Date & Time: Wednesday, December 10, 2025, 12:30 – 2:00 PMLocation: Henry B. Convention CenterPresenting Author: Antonio Llombart-Cussac Presentation Title: ERADICATE: A phase Ib/II study of elacestrant plus trastuzumab deruxtecan in patients with CDK4/6 inhibitor and endocrine-resistant HR+/HER2-low or HER2-ultralow metastatic breast cancerAbstract Number: 2119Presentation Date & Time: Friday, December 12, 2025, 12:30 – 2:00 PMLocation: Henry B. Convention CenterPresenting Author: Sara L. Sammons Presentation Title: Hormonal receptor (HR)-positive HER2 negative breast cancer patients treated with preoperative Elacestrant and PULSAR adaptive radiotherapy: a phase II study (HELP Trial)Abstract Number: 1071Presentation Date & Time: Friday, December 12, 2025, 12:30 – 2:00 PMLocation: Henry B. Convention CenterPresenting Author: Luca Visani About The Elacestrant Clinical Development ProgramElacestrant is also being investigated in several company-sponsored clinical trials in metastatic breast cancer disease, alone or in combination with other therapies. ELEVATE (NCT05563220) is a phase 1b/2 clinical trial evaluating the safety and efficacy of elacestrant combined with alpelisib, everolimus, capivasertib, palbociclib, ribociclib or abemaciclib. ELECTRA (NCT05386108) is an open-label phase 1b/2, multicenter study evaluating elacestrant in combination with abemaciclib in patients with ER+, HER2- breast cancer. The phase 2 portion evaluates this treatment regimen in patients with brain metastases. ELCIN (NCT05596409) is a phase 2 trial evaluating the efficacy of elacestrant in patients with ER+, HER2- advanced/metastatic breast cancer who received one or two prior hormonal therapies and no prior CDK4/6 inhibitors in the metastatic setting. ADELA (NCT06382948) is a phase 3 randomized, double-blinded trial evaluating elacestrant in combination with everolimus in patients with ER+, HER2- mBC with ESR1-mut tumors. Elacestrant is also being evaluated in additional investigator-led trials, in trials conducted in collaboration with other companies, in metastatic breast cancer as well as in early disease. About ORSERDU (elacestrant)U.S. Indication: ORSERDU (elacestrant), 345 mg tablets, is indicated for the treatment of postmenopausal women or adult men with estrogen receptor (ER)-positive, human epidermal growth factor receptor 2 (HER2)-negative, ESR1-mutated advanced or metastatic breast cancer with disease progression following at least one line of endocrine therapy. Full prescribing information for the U.S. can be found at www.orserdu.com. Important Safety InformationWarning and Precautions Dyslipidemia: Hypercholesterolemia and hypertriglyceridemia occurred in patients taking ORSERDU at an incidence of 30% and 27%, respectively. The incidence of Grade 3 and 4 hypercholesterolemia and hypertriglyceridemia were 0.9% and 2.2%, respectively. Monitor lipid profile prior to starting and periodically while taking ORSERDU.Embryo-Fetal Toxicity: Based on findings in animals and its mechanism of action, ORSERDU can cause fetal harm when administered to a pregnant woman. Advise pregnant women and females of reproductive potential of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment with ORSERDU and for 1 week after the last dose. Advise male patients with female partners of reproductive potential to use effective contraception during treatment with ORSERDU and for 1 week after the final dose. Adverse Reactions Serious adverse reactions occurred in 12% of patients who received ORSERDU. Serious adverse reactions in >1% of patients who received ORSERDU were musculoskeletal pain (1.7%) and nausea (1.3%). Fatal adverse reactions occurred in 1.7% of patients who received ORSERDU, including cardiac arrest, septic shock, diverticulitis, and unknown cause (one patient each).The most common adverse reactions (>10%), including laboratory abnormalities, of ORSERDU were musculoskeletal pain (41%), nausea (35%), increased cholesterol (30%), increased AST (29%), increased triglycerides (27%), fatigue (26%), decreased hemoglobin (26%), vomiting (19%), increased ALT (17%), decreased sodium (16%), increased creatinine (16%), decreased appetite(15%), diarrhea(13%), headache (12%), constipation (12%), abdominal pain (11%), hot flush (11%), and dyspepsia (10%). Drug interactions Concomitant use with CYP3A4 Inducers and/or inhibitors: Avoid concomitant use of strong or moderate CYP3A4 inhibitors with ORSERDU. Avoid concomitant use of strong or moderate CYP3A4 inducers with ORSERDU. Use in specific populations Lactation: Advise lactating women to not breastfeed during treatment with ORSERDU and for 1 week after the last dose.Hepatic Impairment: Avoid use of ORSERDU in patients with severe hepatic impairment (Child-Pugh C). Reduce the dose of ORSERDU in patients with moderate hepatic impairment (Child-Pugh B).The safety and effectiveness of ORSERDU in pediatric patients have not been established. To report SUSPECTED ADVERSE REACTIONS, contact Stemline Therapeutics, Inc. at 1-877-332-7961 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. About The Menarini GroupThe Menarini Group is a leading international pharmaceutical and diagnostics company, with a turnover of $5 billion and over 17,000 employees. Menarini is focused on therapeutic areas with high unmet needs with products for cardiology, oncology, pneumology, gastroenterology, infectious diseases, diabetology, inflammation, and analgesia. With 18 production sites and 9 Research and Development centers, Menarini’s products are available in 140 countries worldwide. For further information, please visit www.menarini.com. About Stemline Therapeutics Inc.Stemline Therapeutics, Inc. (“Stemline”), a wholly-owned subsidiary of the Menarini Group, is a commercial-stage biopharmaceutical company focused on bringing transformational oncology treatments to patients. Stemline commercializes elacestrant, an oral endocrine therapy indicated for the treatment of postmenopausal women or adult men with estrogen receptor (ER)-positive, human epidermal growth factor receptor 2 (HER2)-negative, ESR1-mutated advanced or metastatic breast cancer with disease progression following at least one line of endocrine therapy, in the U.S., Europe, and other global regions. Stemline also commercializes tagraxofusp-erzs, a novel targeted therapy directed to CD123, for patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN), an aggressive hematologic cancer, in the United States, Europe, and other global regions. In addition, Stemline commercializes selinexor, an XPO1 inhibitor for multiple myeloma, in Europe. The company is also conducting multiple label-expansion studies with elacestrant and tagraxofusp in breast and hematologic cancer indications, respectively, and has an extensive clinical pipeline of additional drug candidates in various stages of development for a host of solid and hematologic cancers. Media ContactsThe Menarini GroupValeria Speroni CardiEmail: pressoffice@menarini.com Stemline Therapeutics, Inc.Cheya PopeEmail: media@menarinistemline.com 1 Lloyd et al. Clinical and genomic factors associated with elacestrant outcomes in ESR1-mutant metastatic breast cancer. Clin Cancer Res (2025). https://aacrjournals-org.libproxy1.nus.edu.sg/clincancerres/article/doi/10.1158/1078-0432.CCR-25-3033/768341/Clinical-and-Genomic-Factors-Associated-with 2 Rugo et al. Real-World Outcomes of Elacestrant in ER+, HER2-, ESR1-mutant Metastatic Breast Cancer. Clin Cancer Res, 2025. CCR-25-3040R1. https://aacrjournals-org.libproxy1.nus.edu.sg/clincancerres/article/doi/10.1158/1078-0432.CCR-25-3040/768340/Real-World-Outcomes-of-Elacestrant-in-ER-HER2-ESR1

Phase 2Phase 3Clinical Result

24 Nov 2025

·www.globenewswire.com

Menarini Group Announces New Data on ELZONRIS® (tagraxofusp-erzs) to be Presented at the 67th American Society of Hematology Annual Meeting and Exposition

Data spans five abstracts including two oral presentations with important findings for patients living with blastic plasmacytoid dendritic cell neoplasm (BPDCN).BPDCN is a highly aggressive, orphan hematologic malignancy that primarily affects skin, bone marrow, and blood. FLORENCE, Italy and NEW YORK, Nov. 24, 2025 (GLOBE NEWSWIRE) -- The Menarini Group (“Menarini”), a leading international pharmaceutical and diagnostics company, and Stemline Therapeutics, Inc. (“Stemline”), a wholly-owned subsidiary of the Menarini Group, focused on bringing transformational oncology treatments to cancer patients, today announced that the company will present new data related to ELZONRIS® (tagraxofusp-erzs) at the American Society of Hematology (ASH) Annual Meeting and Exposition in Orlando, December 6-9th. The presentations include an oral session highlighting results from a triplet therapy combining tagraxofusp, azacitidine, and venetoclax, which demonstrated both efficacy (high response and bridge to transplant rates) and tolerability in individuals with blastic plasmacytoid dendritic cell neoplasm (BPDCN). “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. “These data demonstrate that tagraxofusp plays an important role in both monotherapy and combination settings for the treatment of BPDCN, and that it also has potential to help patients living with other aggressive hematologic malignancies, where more effective treatment options are desperately needed.” See below for full details of the Menarini Group/Stemline Therapeutics' upcoming presentations and publication: American Society of Hematology (ASH) Annual Meeting 2025 Lead AuthorTitle and Publication NumberSession NamePresentation DetailsNavarro Vicente I, et al.Disease Presentation and Immunophenotype Characteristics in 257 Patients with Blastic Plasmacytoid Dendritic Cell Neoplasm: a PETHEMA/PALG Study*Publication Number: 218618. Acute Myeloid Leukemias: Biomarkers and Molecular Markers in Diagnosis and Prognosis: Redefining AML: Genetic, Phenotypic and Response-Based InsightsOral PresentationDecember 6, 20252:15 PM - 2:30 PM OCCC - W414ABLane A, et al.Tagraxofusp, azacitidine, and venetoclax (TAG-AZA-VEN) triplet therapy shows efficacy, tolerability, and transplant potential in patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN): Results of a phase 2 trial*Publication Number: 653616. Acute Myeloid Leukemias: Investigational Drug and Cellular Therapies: Immunotherapy and chemotherapy combinations in AMLOral PresentationDecember 7, 20255:30 PM - 5:45 PMOCCC - Chapin Theater (W320)Stein A, et al.Longer Survival with Tagraxofusp versus Venetoclax in Patients with Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN): Results of A Real-World AnalysisPublication Number: 4612908. Outcomes Research: Myeloid Malignancies: Poster IIPoster PresentationDecember 7, 20256:00 PM - 8:00 PMOCCC - West Halls B3-B4Wang E, et al.Favorable outcomes including post-transplant survival are seen independent of baseline skin burden in treatment-naïve patients (pts) with blastic plasmacytoid dendritic cell neoplasm (BPDCN) treated with tagraxofusp (TAG): Subgroup analysis of a pivotal trialPublication Number: 5221617. Acute Myeloid Leukemias: Commercially Available Therapies: Poster IIIPoster presentationDecember 8, 20256:00 PM - 8:00 PMOCCC - West Halls B3-B4Cho W, et al,Tagraxofusp and Low-Intensity Chemotherapy for the Treatment of CD123-Positive Relapsed or Refractory Acute Myeloid Leukemia* Online-only publication *Denotes investigator sponsored research or collaborative research ELZONRIS was approved by the FDA in December 2018 for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN) in adult and pediatric patients two years and older, in both treatment-naïve and previously-treated populations. In January 2021, ELZONRIS was approved by the European Commission. About BPDCNBlastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN), is a highly aggressive, orphan hematologic malignancy that primarily affects skin, bone marrow and blood. It can also affect other parts of the body, such as the lymph nodes, spleen, liver and central nervous system. Approximately 85% to 90% of patients develop skin lesions as a first sign of BPDCN. BPDCN affects all people of all races and geographic locations, with the global prevalence likely in the range of 0.4 – 0.5 per 100,000 people annually. While BPDCN 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 a protein called CD123, which can be observed on the surface of the cells. This makes CD123 a crucial diagnostic marker as well as a prime target for precision therapies to strike the cancer directly. About ELZONRIS® (tagraxofusp-erzs)U.S. Indication: ELZONRIS (tagraxofusp-erzs) is a prescription medicine used to treat blastic plasmacytoid dendritic cell neoplasm (BPDCN) in adults and pediatric patients 2 years and older. Full prescribing information for the U.S. can be found at www.elzonris.com IMPORTANT SAFETY INFORMATION, ELZONRIS®Boxed WARNING: CAPILLARY LEAK SYNDROME Capillary Leak Syndrome (CLS) which may be life-threatening or fatal, can occur in patients receiving ELZONRIS. Monitor for signs and symptoms of CLS and take actions as recommended. Warnings and PrecautionsCapillary Leak Syndrome Capillary leak syndrome (CLS), including life-threatening and fatal cases, has been reported among patients treated with ELZONRIS. In patients receiving ELZONRIS in clinical trials, the overall incidence of CLS was 53% (65/122), including Grade 1 or 2 in 43% (52/122) of patients, Grade 3 in 7% (8/122) of patients, Grade 4 in 1% (1/122) of patients, and four fatalities (3%). The median time to onset was 4 days (range - 1 to 46 days), and all but 5 patients experienced an event in Cycle 1. Before initiating therapy with ELZONRIS, ensure that the patient has adequate cardiac function and serum albumin is greater than or equal to 3.2 g/dL. During treatment with ELZONRIS, monitor serum albumin levels prior to the initiation of each dose of ELZONRIS and as indicated clinically thereafter, and assess patients for other signs or symptoms of CLS, including weight gain, new onset or worsening edema, including pulmonary edema, hypotension or hemodynamic instability. Hypersensitivity Reactions ELZONRIS can cause severe hypersensitivity reactions. In patients receiving ELZONRIS in clinical trials, hypersensitivity reactions were reported in 43% (53/122) of patients treated with ELZONRIS and were Grade ≥ 3 in 7% (9/122). Manifestations of hypersensitivity reported in ≥ 5% of patients include rash, pruritus, and stomatitis. Monitor patients for hypersensitivity reactions during treatment with ELZONRIS. Interrupt ELZONRIS infusion and provide supportive care as needed if a hypersensitivity reaction should occur. Hepatotoxicity Treatment with ELZONRIS was associated with elevations in liver enzymes. In patients receiving ELZONRIS in clinical trials, elevations in ALT occurred in 79% (96/122) and elevations in AST occurred in 76% (93/122). Grade 3 ALT elevations were reported in 26% (32/122) of patients. Grade 3 AST elevations were reported in 30% (36/122) and Grade 4 AST elevations were reported in 3% (4/122) of patients. Elevated liver enzymes occurred in the majority of patients in Cycle 1 and were reversible following dose interruption. Monitor alanine aminotransferase (ALT) and aspartate aminotransferase (AST) prior to each infusion with ELZONRIS. Withhold ELZONRIS temporarily if the transaminases rise to greater than 5 times the upper limit of normal and resume treatment upon normalization or when resolved. Adverse Reactions Most common adverse reactions (incidence ≥ 30%) are capillary leak syndrome, nausea, fatigue, pyrexia, peripheral edema, and weight increase. Most common laboratory abnormalities (incidence ≥ 50%) are decreases in albumin, platelets, hemoglobin, calcium, and sodium, and increases in glucose, ALT and AST. Please see full Prescribing Information, including Boxed WARNING.To report SUSPECTED ADVERSE REACTIONS, contact Stemline Therapeutics, Inc. at 1-877-332-7961 or contact the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. About The Menarini GroupThe Menarini Group is a leading international pharmaceutical and diagnostics company, with a turnover of $4.7 billion and over 17,000 employees. Menarini is focused on therapeutic areas with high unmet needs with products for cardiology, oncology, pneumology, gastroenterology, infectious diseases, diabetology, inflammation, and analgesia. With 18 production sites and 9 Research and Development centers, Menarini’s products are available in 140 countries worldwide. For further information, please visit www.menarini.com.About Stemline Therapeutics Inc.Stemline Therapeutics, Inc. (“Stemline”), a wholly-owned subsidiary of the Menarini Group, is a commercial-stage biopharmaceutical company focused on bringing transformational oncology treatments to patients. Stemline commercializes elacestrant, an oral endocrine therapy indicated for the treatment of postmenopausal women or adult men with estrogen receptor (ER)-positive, human epidermal growth factor receptor 2 (HER2)-negative, ESR1-mutated advanced or metastatic breast cancer with disease progression following at least one line of endocrine therapy, in the U.S., Europe, and other global regions. Stemline also commercializes tagraxofusp-erzs, a novel targeted therapy directed to CD123, for patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN), an aggressive hematologic cancer, in the United States, Europe, and other global regions. In addition, Stemline commercializes selinexor, an XPO1 inhibitor for multiple myeloma, in Europe. The company is also conducting multiple label-expansion studies with elacestrant and tagraxofusp in breast and hematologic cancer indications, respectively, and has an extensive clinical pipeline of additional drug candidates in various stages of development for a host of solid and hematologic cancers. Media ContactsThe Menarini GroupValeria Speroni CardiEmail: pressoffice@menarini.com Stemline Therapeutics, Inc.Cheya PopeEmail: media@menarinistemline.com

Clinical ResultDrug ApprovalASHPhase 2Immunotherapy

100 Deals associated with Tagraxofusp-ERZS

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KEGG	Wiki	ATC	Drug Bank
-	Tagraxofusp-ERZS	L01XX67	DB14731

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Blastic Plasmacytoid Dendritic Cell Neoplasm	United States	Stemline Therapeutics, Inc.	21 Dec 2018

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Relapsing acute myeloid leukemia	Phase 2	United States	Stemline Therapeutics, Inc.	10 Feb 2025
CD123 Positive Acute Myeloid Leukemia	Phase 2	United States	Stemline Therapeutics, Inc.	14 Jan 2025
CD123 Positive Acute Myeloid Leukemia	Phase 2	Australia	Stemline Therapeutics, Inc.	14 Jan 2025
Acute Myeloid Leukemia	Phase 2	United States	Stemline Therapeutics, Inc.	01 May 2024
Myelodysplastic Syndromes	Phase 2	United States	The University of Texas MD Anderson Cancer Center	04 Mar 2022
Myeloproliferative Disorders	Phase 2	United States	The University of Texas MD Anderson Cancer Center	04 Mar 2022
Adult Lymphoblastic Lymphoma	Phase 2	United States	The University of Texas MD Anderson Cancer Center	17 Feb 2022
Recurrent B Acute Lymphoblastic Leukemia	Phase 2	United States	The University of Texas MD Anderson Cancer Center	17 Feb 2022
Refractory B Acute Lymphoblastic Leukemia	Phase 2	United States	The University of Texas MD Anderson Cancer Center	17 Feb 2022
Refractory T Acute Lymphoblastic Leukemia	Phase 2	United States	The University of Texas MD Anderson Cancer Center	17 Feb 2022

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


ASH2025	Not Applicable	Blastic Plasmacytoid Dendritic Cell Neoplasm First line	94	Tagraxofusp (TAG)	lkuxxlsppd(tuptjttdms) = reyqmjhmuc iqeypivpzv (kgnuqipbjw ) View more	Positive	06 Dec 2025
ASH2025	Not Applicable	Blastic Plasmacytoid Dendritic Cell Neoplasm First line	94	Venetoclax (VEN)	lkuxxlsppd(tuptjttdms) = ttpupbtkjw iqeypivpzv (kgnuqipbjw ) View more	Positive	06 Dec 2025
NCT03113643 (ASH2025)	Phase 2	Blastic Plasmacytoid Dendritic Cell Neoplasm First line	27	Tagraxofusp+azacitidine+venetoclax (1L)	xqinsnfffk(xgswlwinle) = loxvybvnyb ryftpimvln (pnavmjpgvx, 32 - 88) View more	Positive	06 Dec 2025
NCT03113643 (ASH2025)	Phase 2	Blastic Plasmacytoid Dendritic Cell Neoplasm First line	27	Tagraxofusp+azacitidine+venetoclax (relapsed/refractory)	xqinsnfffk(xgswlwinle) = lnftmkvbnr ryftpimvln (pnavmjpgvx, 8 - 65) View more	Positive	06 Dec 2025
ASH2025	Not Applicable	Blastic Plasmacytoid Dendritic Cell Neoplasm	795	Tagraxofusp	ovccclovut(kwgmhenpdq) = Anemia, thrombocytopenia, neutropenia, and edema were common AEs across LoT1 and LoT2 mszgdkqzgb (wlxvakpsag )	Negative	06 Dec 2025
NCT05032183 (CTgov)	Phase 1/2	Recurrent B Acute Lymphoblastic Leukemia \| Refractory T Acute Lymphoblastic Leukemia \| Refractory B Acute Lymphoblastic Leukemia ... View more	4	Pegfilgrastim+Filgrastim-sndz+Methotrexate+Mercaptopurine+Cytarabine+Mesna+Cyclophosphamide+Leucovorin+Prednisone+Dexamethasone+Rituximab+Vincristine+Tagraxofusp-erzs	chsabazcpg(ucmfxkvcwg) = bmoeandhfx djrdlhprah (bstlimainy, bqiwwvqcmm - xajwshbjnd) View more	-	11 Aug 2025
NCT02113982 (ASCO2025)	Phase 1/2	Blastic Plasmacytoid Dendritic Cell Neoplasm CD123	-	Tagraxofusp 12 µg/kg	qdixxsmunh(fpcjqdulyr) = No investigator-assessed CLS was reported, although some pts required dose interruption due to weight gain or hypoalbuminemia bgkavuusms (dgdxlstscf ) View more	Positive	30 May 2025
NCT02113982 (ASCO2025)	Phase 1/2	Blastic Plasmacytoid Dendritic Cell Neoplasm First line CD123	65	tagraxofusp (HCT-CI low)	xovwcznhwp(bapawgqflw) = Two deaths due to CLS occurred in int pts. Grade 3-4 CLS occurred in 0%, 9%, and 4% of pts; all CLS events were in C1 and all grade 1-4 CLS events resolved livlcfkvmc (rjmtuuafhh ) View more	Positive	30 May 2025
NCT02113982 (ASCO2025)	Phase 1/2		65	tagraxofusp (HCT-CI intermediate)		Positive	30 May 2025
NCT02113982 (PB2471, EHA2025)	Phase 1/2	Blastic Plasmacytoid Dendritic Cell Neoplasm First line CD123	-	TAGRAXOFUSP (HCT-CI low)	jsvydzvviy(rchiggwsxp) = Two deaths due to CLS occurred in intermediate patients. Grade 3-4 CLS occurred in 0%, 9%, and 4% of patients; all CLS events were in cycle 1 and all grade 1-4 CLS events resolved. zvthbpmmcm (btqfpuerhh )	Positive	22 May 2025
NCT02113982 (PB2471, EHA2025)	Phase 1/2		-	TAGRAXOFUSP (HCT-CI intermediate)		Positive	22 May 2025
NCT02113982 (PB2470, EHA2025)	Phase 2	Blastic Plasmacytoid Dendritic Cell Neoplasm CD123+	-	Tagraxofusp 12 µg/kg	oycseizxhz(yvytycooqu) = jhrocvoigv pwoenoakqt (zldebniqmh, 1.7) View more	Positive	14 May 2025
ASH2024	Not Applicable	Blastic Plasmacytoid Dendritic Cell Neoplasm	-	Tagraxofusp	uedxwqizzq(fbtsjntqtu) = ljzfcmhpni bslblbusfp (fmkvxvnuaw ) View more	-	08 Dec 2024
AML-323 (SOHO2024)	Not Applicable	CD123	40	TAG 12 mcg/kg	urbszdclqd(argwyshbrm) = oitjtefrrc gdbmqyzmzf (hxqrgaoqnk ) View more	Positive	04 Sep 2024

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