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Last update 22 May 2025

Tagraxofusp-ERZS

Last update 22 May 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

Adult Lymphoblastic LymphomaMyelofibrosisRecurrent B Acute Lymphoblastic Leukemia+ [6]

Originator Organization

Texas A&M University

Active Organization

Stemline Therapeutics, Inc.Menarini Von Heyden GmbH

The University of Texas MD Anderson Cancer Center

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

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]

NCT06498973

/ RecruitingPhase 1IIT

CD123 Antibody Toxin Congregate (CD123 ATC; Tagraxofusp) Combined With Azacitidine for Maintenance Therapy Post Allogeneic Hematopoietic Cell Transplantation for Patients With CD123-Positive Malignant

This phase Ib trial tests the safety, side effects, best dose and effectiveness of tagraxofusp in combination with azacitidine as maintenance therapy in treating patients with CD123 positive acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) after a donor (allogeneic) hematopoietic cell transplant. An allogeneic hematopoietic cell transplant (HCT) is a type of transplant where the cancer patient receives cells from another person. Maintenance therapy is given after the transplant to prevent the cancer from coming back. Tagraxofusp is a drug that targets cells that have CD123 on their surface in order to kill the cancer cells to help prevent the cancer from coming back. Azacitidine is in a class of medications called demethylation agents. It works by helping the bone marrow to produce normal blood cells and by killing abnormal cells. Giving tagraxofusp in combination with azacitidine may be safe, tolerable and/or effective maintenance therapy in patients with CD123 positive AML and MDS after an allogeneic HCT.

Start Date28 Jan 2025

Sponsor / Collaborator

City of Hope National Medical Center

[+1]

100 Clinical Results associated with Tagraxofusp-ERZS

100 Translational Medicine associated with Tagraxofusp-ERZS

100 Patents (Medical) associated with Tagraxofusp-ERZS

223

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.

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.

01 Mar 2025·Food Science of Animal Resources

Physicochemical and Sensory Characteristics of Hybrid Flexitarian Pork Loin Steak Combined with Different Plant Ingredients

Article

Author: Kim, Chan-Jin ; Alam, Amm Nurul ; Joo, Seon-Tea ; Lee, Eun-Yeong ; Kim, So-Hee ; Hossain, Md Jakir ; Hwang, Young-Hwa

This study intended to assess the impacts of the following plant ingredients as treatment named 2.5% alginate paste (TAG), carrot (TRC), red cabbage (TCA), chickpea (Cicer arietinum; TCP), and included in pork loin meat to produce hybrid meat steak (HBM) to provide healthy meat products suitable for the flexitarian consumers. Plant ingredients were added to the treatment groups at 30%. Results illustrate that HBM steaks had higher (p<0.05) moisture content than HBM steaks. In raw HBM steaks, pH, CIE L*, and CIE a* were increased than pork steak. Pork steak and TCP demonstrated the highest (p<0.05) cooking loss followed by TRC, TCA, and TAG, whereas, TRC and TCA had higher (p<0.05) releasing water content followed by pork, TCP, and TAG. HBM steaks demonstrated a lower (p<0.05) Warner-Bratzler shear force value than pork steaks. According to the sensory panel pork steak and TAG had overall higher acceptance (p<0.05) followed by TRC, TCP, and least for TCA, which was suggested as especially suitable for the elderly or kids. The electronic tongue system revealed higher (p<0.05) umami intensity and bitterness in HBM steaks than in pork steak. The richness value was higher in pork steak followed by TAG, TCP, and TRC, and lowest in TCA. Therefore, the current study demonstrated that plant ingredients can successfully be incorporated into pork loin to make suitable flexitarian steak to keep eating meat but in a lower concentration.

News (Medical) associated with Tagraxofusp-ERZS

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

·www.prnewswire.com

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

12 Dec 2024

·www.prnewswire.com

Menarini Group and MEDSIR Present the Phase III Study ADELA: A New Therapeutic Strategy for Advanced Breast Cancer

ADELA, an international phase III study being conducted across multiple countries, combines elacestrant with everolimus to treat advanced ER+/HER2- breast cancer with ESR1 mutations, aiming to delay disease progression. The study was presented by the Menarini Group and MEDSIR at the San Antonio Breast Cancer Symposium 2024 (SABCS 2024), one of the world's most prestigious events in oncology. MEDSIR's participation at SABCS 2024 solidifies its position as a global leader in oncology research and underscores its commitment to innovation and the development of more personalized and effective therapies. FLORENCE, Italy and BARCELONA, Spain, Dec. 12, 2024 /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, along with MEDSIR, a leading global independent clinical research company in oncology and part of Oncoclínicas & Co., the largest specialized oncology treatment group in Latin America, presented research on the pioneering clinical trial ADELA. This important research addresses therapeutic resistance in advanced ER+/HER2- breast cancer. Presented at the San Antonio Breast Cancer Symposium 2024 (SABCS), the study represents a key milestone in the quest for more effective and personalized treatment options for patients with disease progression. The standard first-line treatment for advanced ER+/HER2- breast cancer combines endocrine therapy with CDK4/6 inhibitors. ESR1 mutations develop as a result of prior exposure to endocrine therapy during metastatic treatment, and up to 50% of ER+, HER2- advanced or metastatic breast cancers will develop these mutations. ESR1 mutations cause the tumors to become resistant to endocrine therapy, in turn causing the cancer to progress; therefore, it is important to test for ESR1 whenever mBC progresses. Longer exposure to endocrine therapy during first-line treatment increases the chance of a patient's tumor developing an ESR1 mutation. With the goal of addressing this unmet medical need, the ADELA phase III clinical trial investigates a new therapeutic option combining elacestrant, a next generation, oral selective estrogen receptor degrader, with everolimus, an mTORC1 inhibitor. This combination is being evaluated in patients with advanced ER+/HER2- breast cancer that harbors ESR1 mutations, and who have experienced progression after standard first-line treatment. Results from the phase III EMERALD study were the basis for elacestrant's approval. Meanwhile, everolimus has shown efficacy in inhibiting other resistance mechanisms in this type of cancer. The elacestrant and everolimus combination has demonstrated preliminary efficacy with a manageable safety profile in the phase 1b/2 ELEVATE study (NCT05563220). "We at Menarini Stemline are delighted to announce the collaboration with MEDSIR to continue advancing the clinical research to explore the combination therapy with elacestrant," said Nassir Habboubi, MD, Chief Medical Officer of Stemline Therapeutics. "We are committed to driving innovation in cancer treatment by delivering transformational therapies aiming to extend the lives of people living with cancer." The primary objective of this international, randomized, double-blind trial is to evaluate whether the combination of elacestrant and everolimus offers greater efficacy in delaying disease progression compared to elacestrant monotherapy. Additionally, it investigates other crucial aspects, such as overall survival, toxicity profile, and the impact on patients' quality of life. The ADELA study represents a critical step in understanding how to overcome tumor resistance challenges in patients with ESR1 mutations, with the goal of advancing towards more effective and safer treatments. "At MEDSIR, we understand innovation not only as achieving clinical results but as the ability to transform patients' lives on a global scale. With ADELA, we take a decisive step toward accomplishing less invasive and more accessible treatments, aiming to offer new hope to those facing the most complex forms of the disease. This advancement reinforces our commitment to increasingly personalized and patient-centered medicine, a fundamental pillar in shaping the future of oncology," said Dr. Antonio Llombart-Cussac, Senior Scientific Leader at MEDSIR. The phase III study not only has significant clinical objectives, but also holds the potential to pave the way for regulatory approval of this therapeutic combination, enabling its use in a broader population of patients with advanced breast cancer. Moreover, the international scope of the study, which includes participation from multiple countries, including Spain, Italy, France, Austria, the Czech Republic, Greece, Germany, and the United Kingdom, underscores the study's global importance and relevance in the scientific community. The presentation of the ADELA study at an event as prominent as SABCS 2024 reinforces MEDSIR's leadership in excellence-driven oncology research and highlights its focus on addressing unmet needs in breast cancer treatment. The ADELA study is active and already recruiting patients. 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 . 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 . 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 . 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 Europe, 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 U.S. and E.U. 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 MEDSIR   Established in 2012, MEDSIR prides itself on working closely with its strategic partners to drive innovation in oncology research. Operating in Spain and the United States, the company provides end-to-end clinical trial management, from study design to publication, with an extensive global network of experts and integrated technology to streamline the process. The company offers proof-of-concept support and a strategic approach that enables research partners to benefit from the best of both worlds: industry clinical research and investigator-driven trials.   With the aim of promoting independent research worldwide, MEDSIR has formed a strategic alliance with Oncoclínicas, the leading oncology group in Brazil, which offers outstanding research potential in South America.  For further information:     About Oncoclínicas & CO   Oncoclínicas&Co is the largest group dedicated to cancer treatment in Latin America, with a specialized and innovative model focused on the entire oncology care journey, combining operational efficiency, humanized care, and high specialization through a medical team composed of over 2,700 specialist physicians with an emphasis on oncology. With its mission to democratize cancer treatment, it offers a comprehensive system that integrates outpatient clinics with high-complexity cancer centers. The company operates 145 units across 39 Brazilian cities, allowing high-quality access in all regions it serves, aligned with world-class standards. Focusing on technology, precision medicine, and genomics, Oncoclínicas performed approximately 635,000 treatments in 2023. It is the exclusive partner in Brazil of the Dana Farber Cancer Institute, affiliated with Harvard Medical School, one of the world's leading cancer research and treatments centers. The company also owns Boston Lighthouse Innovation, a bioinformatics firm based in Cambridge, United States, and holds shares in Medsir, a company dedicated to the development and management of clinical trials for independent cancer research, based in Barcelona, Spain. Recently, Oncoclínicas expanded its operations to Saudi Arabia through a joint venture with the Al Faisaliah Group, bringing its mission to beat cancer to a new continent and providing advanced oncology care on a global scale by combining oncological hyperspecialization with innovative treatment approaches. The company is part of the IDIVERSA index, launched by B3, highlighting companies committed to gender and racial diversity. For more information, visit: .    Logo - WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 3Drug 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
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
Primary Myelofibrosis	Phase 2	Italy	Menarini Von Heyden GmbH	30 Jan 2022

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


ASH2024	Not Applicable	Blastic Plasmacytoid Dendritic Cell Neoplasm	-	Tagraxofusp	fcprglbdwr(wajrcgymqs) = ykjizmducr dhatqjgtlo (yrbynqktwu ) View more	-	08 Dec 2024
AML-323 (SOHO2024)	Not Applicable	CD123	40	TAG 12 mcg/kg	tttdlxgytk(jdouyfxzzz) = wuvdflnkef bhtjgwtfmv (wyixdeztdo ) View more	Positive	04 Sep 2024
NCT02270463 (CTgov)	Phase 1/2	Acute Myeloid Leukemia \| Residual Neoplasm	16	Tagraxofusp-erzs (Stage 1: Tagraxofusp-erzs 7 µg/kg/Day)	nluseoebjc = boglhgrlpj hyujzgdjzb (dpzzqgrlai, vmjdrbntdh - brznvtwmcn) View more	-	21 Aug 2024
NCT02270463 (CTgov)	Phase 1/2	Acute Myeloid Leukemia \| Residual Neoplasm	16	Tagraxofusp-erzs (Stage 1: Tagraxofusp-erzs 9 µg/kg/Day)	nluseoebjc = sncmyvvlby hyujzgdjzb (dpzzqgrlai, ryawoxwbiw - ccwvjqiicz) View more	-	21 Aug 2024
NCT04317781 (CTgov)	Phase 2	Blastic Plasmacytoid Dendritic Cell Neoplasm	3	Tagraxofusp-erzs	symyiyppmo = pavthngqrq urxdxjeosu (dfomocsmyx, qflphwfgcn - faxomguibp) View more	-	16 Jul 2024
NPP (EHA2024)	Not Applicable	Blastic Plasmacytoid Dendritic Cell Neoplasm CD123	18	TAG 12 mcg/kg	qaaujdffwd(bcryilvayl) = chohwoiygi kpjesbocfo (efwkphqwte ) View more	Positive	14 May 2024
NCT03113643 (Pubmed) Manual	Phase 1	Acute Myeloid Leukemia	56	Tagraxofusp + AzacitidineVenetoclax	topwtzzesj(bzjqgcnieb) = oshzjddpuj fvtdhjddpa (ntcxhuhdvg ) View more	Positive	13 Feb 2024
NCT03113643 (Pubmed) Manual	Phase 1	Acute Myeloid Leukemia	56	Tagraxofusp + Azacitidine + Venetoclax (expansion cohort)	topwtzzesj(bzjqgcnieb) = upablxmxti fvtdhjddpa (ntcxhuhdvg ) View more	Positive	13 Feb 2024
ASH2023	Phase 1	CD123 Positive Acute Myeloid Leukemia	26	Tagraxofusp	alfyopchwp(yswpahvwpi) = waejcoeaob eqttvivnmp (hntpjkieiu, 14.0 - NA) View more	-	11 Dec 2023
ASH2023	Phase 1	CD123 Positive Acute Myeloid Leukemia	26	Azacitidine	zcmfraewxh(brirmbnrrv) = cebusmnzpa xsqhmnaegi (kksbsbafhp )	-	11 Dec 2023
NCT04342962 (GIMEMA AML2020, ASH2023)	Phase 2	CD123 Positive Acute Myeloid Leukemia	23	Tagraxofusp	qnciztsyam(ijrfkkblor) = 1 patient receiving 4 courses achieved PR and died due to CNS bleeding kmholtzuoj (glupvzrurs ) View more	-	10 Dec 2023
ASH2023	Not Applicable	Blastic Plasmacytoid Dendritic Cell Neoplasm First line	22	Tagraxofusp	ehejorcign(skqjmlizlb) = vgookxgmxz muftdoskrc (oczllsdizo, 10 - NR) View more	-	10 Dec 2023

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