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Last update 06 Jun 2025

Tagraxofusp-ERZS

Last update 06 Jun 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

+ [8]

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+ [6]

Inactive Indication

Acute Promyelocytic LeukemiaAdult Lymphoblastic LymphomaMultiple Myeloma+ [7]

Originator Organization

Texas A&M University

Active Organization

Stemline Therapeutics, Inc.

The University of Texas MD Anderson Cancer CenterMenarini Von Heyden GmbH

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 (Australia)

Structure/Sequence

Sequence Code 453129448

Source: *****

Clinical Trials associated with Tagraxofusp-ERZS

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 Date30 Sep 2025

Sponsor / Collaborator

Stemline Therapeutics, Inc.

[+1]

NCT06414681

/ Not yet recruitingEarly Phase 1

An Open Label Pilot Trial of the Combination of Tagraxofusp with Pacritinib in Patients with Intermediate-1 or Higher Myelofibrosis, Who Have Had Prior Therapy with the Approved JAK Inhibitors or in Which Therapy with the Approved JAK Inhibitors is Not Appropriate, Contraindicated or Declined

The goal of this open-label, single-center, pilot trial is to test the combination of Tagraxofusp (TAG) with Pacritinib (PAC) in patients with intermediate-II or higher myelofibrosis (MF), who have had prior therapy with the approved JAK1/2 inhibitor or in which therapy with the approved JAK1/2 inhibitors is not appropriate, contraindicated or declined by the subjects.
The Primary Objective is to:
1. Characterized efficacy of the combination of Tagraxofusp and Pacritinib.
The Secondary Objective is to:
1. characterize the safety profile of the combination Tagraxofusp and Pacritinib.
2, Characterize the feasibility of the combination Tagraxofusp and Pacritinib. 3. Characterize hematologic improvement with the combination Tagraxofusp and Pacritinib.
4. Evaluate and compare the effect of Tagraxofusp and Pacritinib on participant reports of MF symptoms.
Exploratory:
Pharmacokinetic (PK) testing of Tagraxofusp and Pacritinib to assess clinical predictors of response.
Next Generation Sequencing (NGS) Testing to define the number and the allele burden of pathological mutations, as well as the changes over the course of therapy, both in regard to progression and response.
Blood will be collected and stored at KU BRCF for future study related PK analysis

Start Date28 Mar 2025

Sponsor / Collaborator

University of Kansas Medical Center

NCT06561152

/ RecruitingPhase 1/2IIT

Tagraxofusp and Low-Intensity Chemotherapy for the Treatment of CD123-Positive Relapsed or Refractory Acute Myeloid Leukemia (AML)

To determine the efficacy of the combination of tagraxofusp, cladribine, and cytarabine.

Start Date10 Feb 2025

Sponsor / Collaborator

Stanford University

[+1]

100 Clinical Results associated with Tagraxofusp-ERZS

100 Translational Medicine associated with Tagraxofusp-ERZS

100 Patents (Medical) associated with Tagraxofusp-ERZS

226

Literatures (Medical) associated with Tagraxofusp-ERZS

01 Jul 2025·PROSTAGLANDINS LEUKOTRIENES AND ESSENTIAL FATTY ACIDS

The single-dose kinetics of [1–14C]-labelled EPA and DHA, administered to male rats as TAG, phosphatidylcholine (PC), and lyso-phosphatidylcholine (LPC), is structurally similar across lipid forms and can be described using the same compartmental models

Article

Author: Hals, Petter-Arnt ; Harris, Stephen ; Scott, Grace ; Hoem, Nils

To investigate the systemic kinetics of EPA and DHA across different lipid classes, male rats were administered [1-¹⁴C]-radiolabelled EPA and DHA as triglycerides (TAG), phosphatidylcholine (PC), or lyso-phosphatidylcholine (LPC) by gavage. LPC was also administered intravenously. Plasma and whole blood concentration-time profiles were recorded from 0 to 168 hours, while cumulative radioactivity in expired air, faeces, and urine was recorded for up to 336 hours. Non-compartmental analysis and compartmental modelling demonstrated overall first-order radiotracer kinetics for both fatty acids, with comparable terminal half-lives. The primary difference was in maximum concentration (Cmax ((µg-eq/g)/(mg/kg)): DHA = 0.18± 0.089, EPA = 0.24± 0.103; P < 0.0001). Between TAG and PC, only time to maximum concentration (Tmax (h)) differed (PC = 3.23 ± 0.94, TAG = 2.55 ± 0.77; P = 0.0004). LPC showed significant differences from TAG and PC in area under the curve (AUC0-inf), Cmax, Tmax, and total clearance (CL/F (mL/(kg h))). Cumulative radioactivity levels in expired air and faeces were consistent with blood and plasma kinetics. As suggested by early-phase (0 to 48 hours) radioactivity accumulation, which deviated from first-order behaviour, TAG and PC, but not LPC, exhibited some faecal loss without systemic absorption. The compartmental models developed performed equally well for radiolabelled EPA and DHA, regardless of whether administered as TAG, PC, or LPC. The model can be adapted to handle non-zero endogenous baselines and was successfully applied to non-radiolabelled EPA, docosapentaenoic acid (DPA), and DHA, quantified via LC-MS/MS. These models can be applied to both radioactive and stable isotopes and adapted to include organ-specific kinetics, as well as those of EPA, DPA, and DHA in other species, including humans.

01 May 2025·Blood Neoplasia

New leads to enhance tagraxofusp efficacy in pDC-AML

Article

Author: Garnache-Ottou, Francine ; Belakri, Imane ; Boichut, Morgane ; Adotevi, Olivier ; Fredon, Maxime ; Biichlé, Sabeha ; Renosi, Florian ; Roussel, Xavier ; Brooks, Christopher ; Angelot Delettre, Fanny ; Poussard, Margaux ; Boidot, Romain

28 Mar 2025·Journal of Clinical and Experimental Hematopathology

Diagnostic approach to blastic plasmacytoid dendritic cell neoplasm: historical perspectives and current understanding

Review

Author: Takeuchi, Kengo ; Sakamoto, Kana

Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare hematologic malignancy composed of immature cells that exhibit plasmacytoid dendritic cell (pDC) differentiation. The diagnosis of BPDCN is often challenging due to its rarity and morphologic and phenotypic overlap with other hematologic malignancies, such as acute myeloid leukemia (AML). The emergence of tagraxofusp, a CD123-directed cytotoxin, and other novel therapies has underscored the importance of accurately diagnosing BPDCN. This review initially outlined the clinical and histopathological features of BPDCN, including patients with immunoblastoid morphology. Various proposed diagnostic criteria based on flow cytometry and immunohistochemistry findings were presented, highlighting critical points of caution in the diagnostic process. Strategies for detecting minimal residual disease or microinvasion in BPDCN, a significant clinical issue, were also discussed. Additionally, we reviewed the recurrent 8q24 (MYC) and MYB rearrangements observed in BPDCN, which can aid in diagnosis. Furthermore, we explored mature plasmacytoid dendritic cell proliferation (MPDCP) associated with myeloid neoplasm, which is characterized by a clonal proliferation of pDCs in cases with a defined myeloid neoplasm and may also serve as a potential differential diagnosis for BPDCN. Lastly, we discussed pDC-AML, characterized by pDC proliferation in AML cases, which can also be part of MPDCP and is often associated with frequent RUNX1 mutations. Overall, this review provides insights into BPDCN diagnosis and highlights the current challenges in its detection and differential diagnosis.

News (Medical) associated with Tagraxofusp-ERZS

22 May 2025

·www.einpresswire.com

Menarini Group Presents Updated Data Underscoring the Combinability of Elacestrant (ORSERDU®) in Patients with ER+, HER2- Metastatic Breast Cancer (mBC) at the ASCO 2025 Annual Meeting

Preliminary efficacy analysis from the elacestrant plus everolimus and ribociclib cohorts of the ELEVATE study, along with updated safety data from additional cohorts of elacestrant plus targeted therapy combination arms, will be presented. These data highlight elacestrant’s potential role as an endocrine therapy backbone in combination with various targeted agents for patients with ER+/HER2- mBC. The robust elacestrant clinical development program across wide-ranging studies further solidifies its potential in monotherapy and combination settings, in mBC and in early breast cancer. FLORENCE, Italy and NEW YORK, May 22, 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 preliminary efficacy and safety results from the Phase 1b/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 and improve patient outcomes. Additionally, various other trial-in-progress updates will be presented, investigating elacestrant’s potential to become an endocrine therapy (ET) backbone across the spectrum of breast cancer. These data will be presented at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting. The ELEVATE study is comprised of six treatment regimens evaluating elacestrant in combination with CDK4/6 inhibitors (palbociclib, abemaciclib and ribociclib) and with inhibitors of the PI3K/AKT/mTOR pathway (everolimus, alpelisib and capivasertib). ELEVATE results reported at ASCO 2025 (abstract 1070/49) include updated efficacy data which demonstrate favorable preliminary progression-free survival (PFS) from the elacestrant plus ribociclib and the elacestrant plus everolimus cohorts. A recommended phase 2 dose (RP2D) was determined to be elacestrant 345 mg plus ribociclib 400 mg. The RP2D of elacestrant 345 mg plus everolimus 7.5 mg was previously reported. “It is encouraging to see the positive preliminary efficacy and safety results when everolimus and ribocilib, respectively, are combined with elacestrant. These findings are consistent with the promising elacestrant plus abemaciclib cohort data from the same study that was presented last December, which also demonstrated favorable preliminary efficacy and safety in this setting,” said Hope S. Rugo, MD, Director, Women's Cancers Program and Division Chief, Breast Medical Oncology, City of Hope Comprehensive Cancer Center. “As the progression-free survival data and safety data continue to mature across the various cohorts of the ELEVATE study, we are encouraged by elacestrant’s potential to become an endocrine therapy backbone in combination regimens for the treatment of metastatic breast cancer.” Additional data reported separately (abstract 1079/58) provided updated Phase 1b/2 safety results from four cohorts of the ELEVATE study, including elacestrant plus ribociclib, everolimus, alpelisib, and capivasertib. These updated preliminary results show that the combinations are consistent with the known safety profiles of each targeted therapy plus standard of care endocrine therapy. “These data continue to underscore the potential value of elacestrant as a combination partner in the ER+/HER2- metastatic breast cancer treatment landscape,” said Elcin Barker Ergun, CEO of the Menarini Group. “We are also exploring the potential of elacestrant in other patient populations, including our currently enrolling ELEGANT study, which is designed to assess its potential benefit in early breast cancer patients with high risk of recurrence.” In addition, the company will be presenting other data at the ASCO Annual Meeting. See below for a complete list of Menarini Stemline abstracts. Menarini Stemline Abstracts: Presentation Title: Elacestrant (Ela) combinations with ribociclib (Ribo) and everolimus (Eve) in patients (pts) with ER+/HER2- locally advanced or metastatic breast cancer (mBC): Update from ELEVATE, a phase (Ph) 1b/2, open-label, umbrella study. Abstract Number: 1070 Presentation Date & Time: Monday, June 2, 9:00 AM – 12:00 PM CT Location: Poster Bd 49 Presenting Author: Hope S. Rugo Presentation Title: Elacestrant combinations in patients (pts) with ER+/HER2- locally advanced or metastatic breast cancer (mBC): Safety update from ELEVATE, a phase (Ph) 1b/2, open-label, umbrella study. Abstract Number: 1079 Presentation Date & Time: Monday, June 2, 9:00 AM – 12:00 PM CT Location: Poster Bd 58 Presenting Author: Nancy Chan Presentation Title: ADELA: 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) + CDK4/6i. Abstract Number: TPS1129 Presentation Date & Time: Monday, June 2, 9:00 AM – 12:00 PM CT Location: Poster Bd 103b Presenting Author: Antonio Llombart-Cussac Presentation Title: ELCIN: Elacestrant in women and men with CDK4/6 Inhibitor (CDK4/6i)-naïve estrogen receptor-positive (ER+), HER2-negative (HER2-) metastatic breast cancer (mBC): an open-label multicenter phase 2 study. Abstract Number: TPS1127 Presentation Date & Time: Monday, June 2, 9:00 AM – 12:00 PM CT Location: Poster Bd 102b Presenting Author: Virginia G. Kaklamani 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: TPS619 Presentation Date & Time: Monday, June 2, 9:00 AM – 12:00 PM CT Location: Poster Bd 210a Presenting Author: Aditya Bardia Presentation Title: EORTC-2129-BCG: Elacestrant for treating ER+/HER2- breast cancer patients with ctDNA relapse (TREAT ctDNA) Abstract Number: TPS620 Presentation Date & Time: Monday, June 2, 9:00 AM – 12:00 PM CT Location: Poster Bd 210b Presenting Author: Michail Ignatiadis About The Elacestrant Clinical Development Program Elacestrant 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 Information Warning 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 Group The 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 Contacts The Menarini Group Valeria Speroni Cardi Email: pressoffice@menarini.com Stemline Therapeutics, Inc. Cheya Pope Email: media@menarinistemline.com Legal Disclaimer: EIN Presswire provides this news content "as is" without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.

Clinical ResultASCOPhase 2Phase 3

20 Mar 2025

·www.globenewswire.com

Menarini Group Announces Collaboration with VisualDx to Aid in Identifying People Who May Have Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)

BPDCN is an aggressive hematologic malignancy with a historically poor prognosisVisualDx is a clinical decision support system used by more than 2,300 hospitals, clinics, and medical schools around the world VisualDx’s system seeks to enhance identification of people who may have BPDCN, leveraging actual images of BPDCN skin lesions and artificial intelligence/machine learning (AI/ML) technologies incorporated into the VisualDx platform FLORENCE, Italy and NEW YORK, March 20, 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 they are collaborating with VisualDx to enhance identification of people who may have BPDCN. This is an example of innovative companies working together to bring artificial intelligence/machine learning (AI/ML) tools to help identify BPDCN as a possible early differential diagnosis. The project includes leveraging actual images of BPDCN skin lesions and AI/ML technologies incorporated into the VisualDx platform. The AI model is now live within VisualDx. VisualDx is a physician-led company committed to improving medical decision-making, medical education and research. The VisualDx tool is a clinical decision support system used by more than 2,300 hospitals, clinics, and medical schools around the world. The software includes a comprehensive database of clinical images, submitted through partnerships with learning institutions and others, and vetted by clinicians, to assist healthcare professionals to identify skin concerns across all skin types. Through the AI image search, clinicians can better understand different skin conditions with the goal of supporting patients to get more accurate diagnoses throughout their care journeys. BPDCN is an aggressive orphan hematologic malignancy with a historically poor prognosis (approximately 8.7 to 14 months post diagnosis1) that typically presents skin lesions and can also involve the bone marrow, blood, central nervous system, lymph nodes and viscera. Dermatologists may be the first to recognize the signs of BPDCN and biopsy suspicious lesions. Pathologists can then test for BPDCN by testing for certain biomarkers which are highly expressed on BPDCN cells. Tagraxofusp-erzs is the only approved treatment for patients with BPDCN, and the first and only approved CD123-targeted therapy, in the United States, Europe and other global regions. "BPDCN is a rare and clinically aggressive hematologic malignancy, often presenting with cutaneous lesions. Due to its aggressive nature and the immature cell involvement, the prognosis can be poor if not treated promptly,” said Marina Konopleva, MD, Phd, Professor, Molecular Pharmacology, Director, Leukemia Program and Co-Director, Blood Cancer Institute, at Montefiore Einstein. “Early diagnosis plays a critical role in improving patient outcomes, and emerging AI/ML technologies may offer valuable support in the differential diagnosis and early identification of BPDCN." “BPDCN often first presents as a skin lesion and usually has a poor prognosis. There is a dire need for early diagnosis so that patients may access appropriate treatment options,” said Elcin Barker Ergun, CEO of the Menarini Group. “We are delighted to collaborate with VisualDx to provide healthcare teams with a tool using the latest artificial intelligence/machine learning (AI/ML) technology to help interpret challenging skin lesions.” VisualDx does not store any images uploaded by clinicians taking a picture of their patients’ skin exams. This helps maintain the patient’s privacy. About BPDCNBPDCN, formerly blastic NK-cell lymphoma, is an aggressive, orphan hematologic malignancy, often with cutaneous manifestations, with historically poor outcomes. BPDCN typically presents skin lesions and may also involve bone marrow, blood, central nervous system, lymph nodes and viscera. The BPDCN cell of origin is the plasmacytoid dendritic cell (pDC) precursor. The diagnosis of BPDCN is based on the immunophenotypic diagnostic triad of CD123, CD4, and CD56, as well as other markers. The World Health Organization (WHO) termed this disease “BPDCN” in 2008; previous names included blastic NK cell lymphoma and CD4+/CD56+ hematodermic neoplasm. About ELZONRIS® (Tagraxofusp-erzs)U.S. Indication: ELZONRIS 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 Precautions Capillary 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 over $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 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. About VisualDxVisualDx is a company dedicated to improving medical decisions through augmented thinking and timely visualization. It is committed to reducing disparities in medicine and believes technology can bridge gaps in knowledge to bring about more equitable care. VisualDx has become the standard professional resource at more than 2,300 hospitals, clinics and medical schools worldwide by combining problem oriented clinical search with the world's best curated medical image library, plus medical knowledge from experts and sophisticated machine learning algorithms to help with differential diagnosis, variation, treatment, and patient communication. Learn more at www.visualdx.com Media ContactsThe Menarini GroupValeria Speroni CardiEmail: pressoffice@menarini.com Stemline Therapeutics, Inc.Cheya PopeEmail: media@menarinistemline.com 1 Pagano L, et al Haematological. 2013;98 (2): 239-246 and Pemmaraju N Curr Hematol Malig Rep. 2017; 12(6): 510-512

Drug ApprovalClinical Result

10 Jan 2025

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Menarini Group and Insilico Medicine Enter a Second Exclusive Global License Agreement for an AI Discovered Preclinical Asset Targeting High Unmet Needs in Oncology

This is the second asset Menarini Group has inlicensed from Insilico Medicine which was discovered through their generative AI platform, similar to the preclinical stage KAT6 inhibitor (MEN2312) licensed a year ago and which advanced rapidly into the clinical phase. Under the agreement, Menarini Group will be granted global rights to develop and commercialize the asset. The deal includes a $20m upfront payment, and the combined value, including all development, regulatory, and commercial milestones, is over $550 million, followed by tiered royalties. FLORENCE, Italy and CAMBRIDGE, Mass., Jan. 10, 2025 /PRNewswire/ -- 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, and Insilico Medicine ("Insilico"), a clinical stage generative artificial intelligence (AI)-driven biotechnology company, today announced that the companies have entered into an exclusive licensing agreement granting Stemline the global rights to develop and commercialize a preclinical small molecule targeting high unmet needs in oncology. The asset is a highly selective and potentially best-in-class small molecule inhibitor targeting a broad range of solid tumor cancers, developed with the help of Chemistry42, Insilico's generative chemistry engine, and Insilico's drug discovery team. The asset has successfully completed preclinical development and has demonstrated broad anti-tumor activity in selected cancers. "We are thrilled to enter our second collaboration with Insilico Medicine, a leader in the field of generative AI, for a highly selective and potentially best-in-class small molecule targeting a broad range of cancers," said Elcin Barker Ergun, CEO of the Menarini Group. "This asset will help us enter into new areas of high unmet need, expanding the tumor areas where we can help cancer patients with ground-breaking therapies." "Our previous experience with Menarini Stemline proved that the company is efficient, agile, strategic, and committed to rapidly delivering the best novel therapeutic solutions to patients with cancer, maximizing the probability of success of the program," said Alex Zhavoronkov, PhD, Founder and CEO of Insilico Medicine. "Menarini Stemline's strategic visionary management is rapidly re-shaping the field of oncology, and we are very happy to take part in their quest to extend patients' lives around the world." Under the terms of the agreement, Stemline will provide a $20 million upfront payment to Insilico. The combined value of the deal, including all development, regulatory, and commercial milestones, is over $550 million, followed by tiered royalties. Prior to this collaboration, the Menarini Group and Insilico entered an exclusive licensing agreement in January 2024 for MEN2312, an innovative small molecule for breast cancer treatment and other oncology indications. About MEN2312 MEN2312 was designed by Insilico's R&D team with the help of its end-to-end Pharma Generative AI platform to inhibit KAT6 and block endocrine receptor (ER) at the transcriptional level, giving it the potential to overcome resistance to endocrine therapies due to mutation or ligand-independent constitutive activation of ER. In preclinical studies, the molecule has demonstrated potent inhibition against KAT6 in multiple CDX and PDX models with good efficacy and safety. About The Menarini Group The Menarini Group is a leading international pharmaceutical and diagnostics company, with a turnover of over $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 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 the development and commercialization of novel oncology therapeutics. Stemline commercializes ORSERDU® (elacestrant) in the U.S. and in the E.U., 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. Stemline also commercializes ELZONRIS® (tagraxofusp-erzs), a novel targeted treatment directed to CD123 for patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN), an aggressive hematologic cancer, in the United States and Europe, which is the only approved treatment for BPDCN in the US and EU to date. Stemline also commercializes NEXPOVIO® (selinexor) in Europe, an XPO1 inhibitor for multiple myeloma. Stemline also has an extensive clinical pipeline of small molecules and biologics in various stages of development for a host of solid and hematologic cancers. About Insilico Medicine Insilico Medicine, a global clinical stage biotechnology company powered by generative AI, is connecting biology, chemistry, and clinical trials analysis using next-generation AI systems. The company has developed AI platforms that utilize deep generative models, reinforcement learning, transformers, and other modern machine learning techniques for novel target discovery and the generation of novel molecular structures with desired properties. Insilico Medicine is developing breakthrough solutions to discover and develop innovative drugs for cancer, fibrosis, central nervous system diseases, infectious diseases, autoimmune diseases, and aging-related diseases. SOURCE Insilico Medicine WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

License out/inDrug Approval

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


NCT02113982 (ASCO2025)	Phase 1/2	Blastic Plasmacytoid Dendritic Cell Neoplasm CD123	-	Tagraxofusp 12 µg/kg	uvfdvacevy(tdrdqqhydg) = No investigator-assessed CLS was reported, although some pts required dose interruption due to weight gain or hypoalbuminemia fphovkqetw (khyuzckndv ) View more	Positive	30 May 2025
NCT02113982 (ASCO2025)	Phase 1/2	Blastic Plasmacytoid Dendritic Cell Neoplasm First line CD123	65	tagraxofusp (HCT-CI low)	nmfjizwokt(blyahiufxr) = 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 ofszeqzqxu (jupcoxluvp ) View more	Positive	30 May 2025
NCT02113982 (ASCO2025)	Phase 1/2		65	tagraxofusp (HCT-CI intermediate)		Positive	30 May 2025
ASH2024	Not Applicable	Blastic Plasmacytoid Dendritic Cell Neoplasm	-	Tagraxofusp	pqpdaldsdg(udgyngodpg) = odiyclrdqs ofjnqirnbj (latofvcfpi ) View more	-	08 Dec 2024
AML-323 (SOHO2024)	Not Applicable	CD123	40	TAG 12 mcg/kg	bxykqtqlts(iqfmxoabqn) = riofllsezw paddzhavly (mpqwbsylmo ) View more	Positive	04 Sep 2024
NCT02270463 (CTgov)	Phase 1/2	Acute Myeloid Leukemia \| Treatment related acute myeloid leukaemia \| Residual Neoplasm	16	Tagraxofusp-erzs (Stage 1: Tagraxofusp-erzs 7 µg/kg/Day)	xqfrfrneal = dsgfkdywib ivwvqexfzt (lezwtanidx, cshkqnuedg - jtdrmxqjdh) View more	-	21 Aug 2024
NCT02270463 (CTgov)	Phase 1/2		16	Tagraxofusp-erzs (Stage 1: Tagraxofusp-erzs 9 µg/kg/Day)	xqfrfrneal = iveiboyuec ivwvqexfzt (lezwtanidx, iypcyappcg - qvlglewcij) View more	-	21 Aug 2024
NCT04317781 (CTgov)	Phase 2	Blastic Plasmacytoid Dendritic Cell Neoplasm	3	Tagraxofusp-erzs	kmnpikugsj = twvpsmcqrj nmhrlimjnc (eihmzbfgyd, hkjpviggdw - rtfgkskein) View more	-	16 Jul 2024
NPP (EHA2024)	Not Applicable	Blastic Plasmacytoid Dendritic Cell Neoplasm CD123	18	TAG 12 mcg/kg	cbagioeeif(trqcgjzcjc) = rmvucuqwax eshrvnqdok (feriajpjkg ) View more	Positive	14 May 2024
NCT03113643 (Pubmed) Manual	Phase 1	Acute Myeloid Leukemia	56	Tagraxofusp + AzacitidineVenetoclax	lnayfyanwz(cggtwhwvsp) = nmcejxqame ojkyoeobyg (enfuysvgtl ) View more	Positive	13 Feb 2024
NCT03113643 (Pubmed) Manual	Phase 1	Acute Myeloid Leukemia	56	Tagraxofusp + Azacitidine + Venetoclax (expansion cohort)	lnayfyanwz(cggtwhwvsp) = kwrebnxtwr ojkyoeobyg (enfuysvgtl ) View more	Positive	13 Feb 2024
ASH2023	Phase 1	CD123 Positive Acute Myeloid Leukemia	26	Tagraxofusp	pqkhvjryrp(dsuuyjqyzn) = jghxnfjkxg gorknybqxo (ofrimksqji, 14.0 - NA) View more	-	11 Dec 2023
ASH2023	Phase 1	CD123 Positive Acute Myeloid Leukemia	26	Azacitidine	vjqvfneghh(wpejoosoxc) = nymwzysziq eumnlyvkvs (rnvqdcwync )	-	11 Dec 2023
NCT04342962 (GIMEMA AML2020, ASH2023)	Phase 2	CD123 Positive Acute Myeloid Leukemia	23	Tagraxofusp	qlsoiohhpu(kyxtovfcwr) = 1 patient receiving 4 courses achieved PR and died due to CNS bleeding kzzxreghzc (bkpcbqpmxn ) View more	-	10 Dec 2023

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