Elzonris (tagraxofusp-erzs) / Menarini, Nippon Shinyaku - LARVOL DELTA

Subgroup analysis by fitness criteria of patients (pts) with blastic plasmacytoid dendritic cell neoplasm (BPDCN) treated with first-line (1L) tagraxofusp (TAG). (ASCO 2025) - P1/2 | "Clinical Trial Registration Number: NCT02113982 The abstract will be released to the public on May 22, 2025 at 5:00 PM EDT"

Clinical • Oncology

Outcomes in patients (pts) younger than 50 years old (yo) with treatment-naïve blastic plasmacytoid dendritic cell neoplasm (BPDCN) treated with tagraxofusp (TAG): Subanalysis of a phase 1/2 trial. (ASCO 2025) - P1/2 | "Clinical Trial Registration Number: NCT02113982 The abstract will be released to the public on May 22, 2025 at 5:00 PM EDT"

Clinical • P1/2 data • Oncology

Tagraxofusp and low-intensity chemotherapy for the treatment of CD123-positive relapsed or refractory acute myeloid leukemia. (ASCO 2025) - P1/2 | "Clinical Trial Registration Number: NCT06561152 The abstract will be released to the public on May 22, 2025 at 5:00 PM EDT"

Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • CD123 • IL3RA

Empowering Nurses: Essential Knowledge and Skills for Tagraxofusp-erzs Administration (ONS 2025) - "Through an education inservice and a "train the trainer" model, nurses were able to safely administer Tagraxofusp-erzs and monitor patients. In the future, we plan to educate new nurses on the drug during chemotherapy certification."

Hematological Malignancies • Leukemia • Oncology

Tagraxofusp plus venetoclax and azacitidine in patients ineligible for intensive chemotherapy with previously untreated CD123+ acute myeloid leukemia: A phase 2 multicenter trial (AACR 2025) - "Abstract is embargoed at this time."

Clinical • P2 data • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • CD123 • IL3RA

Immunotherapies Targeting CD123 and CD303: A New Frontier in Treating Blastic Plasmacytoid Dendritic Cell Neoplasm. (PubMed, Int J Mol Sci) - "Tagraxofusp, the first FDA-approved CD123-targeted therapy, has significantly improved patient outcomes...This review explores the latest developments in BPDCN treatment, emphasizing the potential of CD123 and CD303 as targets for precision medicine interventions. The ongoing evolution of targeted immunotherapies holds promise for improving patient survival and redefining treatment paradigms in hematologic malignancies."

IO biomarker • Journal • Review • Hematological Disorders • Hematological Malignancies • Oncology • CD123 • CLEC4C • IL3RA

Tagraxofusp and Low-Intensity Chemotherapy for the Treatment of CD123 Positive Relapsed or Refractory Acute Lymphoblastic Leukemia or Lymphoblastic Lymphoma (clinicaltrials.gov) - P1/2 | N=4 | Terminated | Sponsor: M.D. Anderson Cancer Center | Trial completion date: Jul 2025 ➔ Jul 2024 | Active, not recruiting ➔ Terminated | Trial primary completion date: Jul 2025 ➔ Jul 2024; Administratively Complete 75%< Participants

Trial completion date • Trial primary completion date • Trial termination • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • Hematological Malignancies • Leukemia • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • T Acute Lymphoblastic Leukemia • IL3RA

Diagnostic approach to blastic plasmacytoid dendritic cell neoplasm: historical perspectives and current understanding. (PubMed, J Clin Exp Hematop) - "The emergence of tagraxofusp, a CD123-directed cytotoxin, and other novel therapies has underscored the importance of accurately diagnosing 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."

Journal • Review • Acute Myelogenous Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Oncology • CD123 • IL3RA • RUNX1

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 (clinicaltrials.gov) - P1 | N=20 | Not yet recruiting | Sponsor: University of Kansas Medical Center | Trial primary completion date: Dec 2025 ➔ Dec 2026

Trial primary completion date • Myelofibrosis

Efficacy of first-line tagraxofusp in blastic plasmacytoid dendritic cell neoplasm with prior or concomitant hematologic malignancy: subgroup analysis of a pivotal trial. (PubMed, Leuk Lymphoma) - No abstract available

Journal • Hematological Disorders • Hematological Malignancies • Oncology

Tagraxofusp and Azacitidine for Maintenance Treatment in Patients With CD123 Positive AML and MDS Following Donor Hematopoietic Cell Transplant (clinicaltrials.gov) - P1 | N=43 | Recruiting | Sponsor: City of Hope Medical Center | Trial completion date: Jan 2027 ➔ Apr 2027 | Trial primary completion date: Jan 2027 ➔ Apr 2027

Trial completion date • Trial primary completion date • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • Transplantation

Venetoclax and hypomethylating agents versus tagraxofusp in older patients with blastic plasmacytoid dendritic cell neoplasm. (PubMed, Ann Hematol) - "In conclusion, this large-scale, retrospective database analysis suggests that VEN + HMA is an effective therapeutic alternative to tagraxofusp in older patients for the management of BPDCN. Future studies are needed to prospectively validate these findings."

Journal • Hematological Disorders • Hematological Malignancies • Hepatology • Oncology • CD123 • IL3RA

Tagraxofusp and Low-Intensity Chemotherapy for CD123-Positive Relapsed or Refractory AML (clinicaltrials.gov) - P1/2 | N=20 | Recruiting | Sponsor: Stanford University | Not yet recruiting ➔ Recruiting | Trial primary completion date: Oct 2026 ➔ Oct 2027

Enrollment open • Trial primary completion date • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

Orphan Drug Designations and Approvals (FDA) - Generic Name: Tagraxofusp-erzs, Trade Name: ELZONRIS, Date Designated: 06/06/2013, Orphan Designation: Treatment of blastic plasmacytoid dendritic cell neoplasm, Orphan Designation Status: Designated/Approved

Orphan drug • Acute Myelogenous Leukemia

Stemline Therapeutics Receives Breakthrough Therapy Designation from U.S. Food and Drug Administration for SL-401 (PM360) - "Stemline Therapeutics, Inc. (Nasdaq:STML) announced today that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to SL-401, a targeted therapy directed to the interleukin-3 receptor (CD123), for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN)."

Breakthrough therapy • Hematological Malignancies

Tagraxofusp in Patients With CD123+ or With BPDCN-IPh-like Acute Myeloid Leukemia (clinicaltrials.gov) - P2 | N=25 | Terminated | Sponsor: Gruppo Italiano Malattie EMatologiche dell'Adulto | N=50 ➔ 25 | Recruiting ➔ Terminated; poor enrollment

Enrollment change • Trial termination • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • CD4 • IL3RA • NCAM1

Blastic plasmacytoid dendritic cell neoplasm: a Swiss case series of a very rare disease and a structured review of the literature. (PubMed, Swiss Med Wkly) - "Our study confirms that BPDCN is a very rare and difficult-to-treat disease. Underdiagnosis and underreporting in the literature pose further challenges. Symptoms at presentation seem to differ slightly between sexes and reaching a complete remission after first-line treatment remains crucial for a prolonged overall survival. Effective treatment protocols in first line include transplantation regimens (mainly allogenic, potentially also autologous) as well as ALL protocols. In order to understand the significance of tagraxofusp as a bridge to transplant or as a continuous monotherapy in elderly patients, further evaluation with longer follow-up periods is required. In general, analysis of the Swiss patients confirmed the results from the worldwide cohort."

Journal • Review • Bone Marrow Transplantation • Hematological Malignancies • Lymphoma • Oncology • Rare Diseases • Transplantation • CD123 • IL3RA

A Multidisciplinary Approach to Diagnosing Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN): Practical Recommendations and Insights from Countries of the Gulf Cooperation Council. (PubMed, Cancers (Basel)) - "BPDCN is frequently misidentified, but early, accurate diagnosis is critical to extending patient survival using tagraxofusp, a first-in-class CD123-targeted therapy, and increasing their chances of receiving a potentially curative stem cell transplantation. Cases of BPDCN in countries of the Gulf Cooperation Council are lower than the extrapolated incidence from other geographic locations due to lack of awareness of key diagnostic features, lack of consensus on the minimal phenotype for diagnosis, and lack of local immunohistochemistry testing facilities, contributing to underdiagnosis in this region. Practical recommendations, a streamlined diagnostic panel, and suggested multidisciplinary approaches based on expert experience regarding diagnostic and clinical challenges specific to this region, and a review of the literature are presented here to facilitate diagnosis of BPDCN in this region by primary care physicians, dermatologists, and hematologists."

Journal • Review • Bone Marrow Transplantation • Dermatology • Hematological Disorders • Hematological Malignancies • Oncology • Transplantation • CD123 • IL3RA

Tagraxofusp and Decitabine for the Treatment of Chronic Myelomonocytic Leukemia (clinicaltrials.gov) - P1/2 | N=64 | Recruiting | Sponsor: M.D. Anderson Cancer Center | Trial completion date: Jan 2025 ➔ Jan 2026 | Trial primary completion date: Jan 2025 ➔ Jan 2026

Trial completion date • Trial primary completion date • Chronic Myelomonocytic Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Myeloproliferative Neoplasm • Oncology

Study 0314: Tagraxofusp (SL-401) in Participants With Chronic Myelomonocytic Leukemia (CMML) and Myelofibrosis (MF) (clinicaltrials.gov) - P1/2 | N=82 | Completed | Sponsor: Stemline Therapeutics, Inc. | Phase classification: P2 ➔ P1/2

Phase classification • Chronic Myelomonocytic Leukemia • Hematological Malignancies • Leukemia • Myelofibrosis • Oncology • JAK2 • PCM1 • PDGFRA • PDGFRB

A Study of Tagraxofusp in Combination With Venetoclax and Azacitidine in Adults With Untreated CD123+ Acute Myeloid Leukemia Who Cannot Undergo Intensive Chemotherapy (clinicaltrials.gov) - P2 | N=76 | Recruiting | Sponsor: Stemline Therapeutics, Inc. | Not yet recruiting ➔ Recruiting

Enrollment open • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

Phase 2 Multicenter Trial of Tagraxofusp in Combination with Venetoclax and Azacitidine in Adults with Previously Untreated CD123+ Acute Myeloid Leukemia Ineligible for Intensive Chemotherapy (ASH 2024) - P2 | "Genomic profiling and CD123 expression will be analyzed at baseline and during study treatment. Part 1 will enroll patients from North America, S Korea & Australia."

Clinical • Combination therapy • P2 data • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Malignancies • Hepatology • Infectious Disease • Leukemia • Oncology • CD123 • IL3RA • TP53

Results of a Phase I/II Study of Tagraxofusp in Combination with Decitabine for Patients with Myelodysplastic/Myeloproliferative Neoplasms and Higher Risk Myelodysplastic Syndromes (ASH 2024) - "One patient (11%) had received prior allogeneic stem-cell transplantation and 1 (11%) had received prior HMA-venetoclax therapy...Both events occurred in patients >75 years of age during cycle 1 of therapy and reverted to baseline (< grade 1) with appropriate management including steroids, albumin and furosemide. One patient received a single dose of tocilizumab...Although TAG at low doses is associated with acceptable safety profile with no new safety signals, CLS and CRS can be observed in older patients. Further follow up and enrollment is ongoing in younger (<75 years of age) patients to confirm the long-term efficacy and safety of this combination."

Clinical • Combination therapy • P1/2 data • Atrial Fibrillation • Bone Marrow Transplantation • Cardiovascular • Chronic Myelomonocytic Leukemia • Constipation • Cough • Febrile Neutropenia • Gastroenterology • Gastrointestinal Disorder • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Myelodysplastic Syndrome • Myeloproliferative Neoplasm • Neutropenia • Oncology • Pneumonia • Respiratory Diseases • CD123 • IL3RA • TP53

Treatment Approaches for Blastic Plasmacytoid Dendritic Cell Neoplasm: Data from the Pethema and PALG Epidemiologic Registry (ASH 2024) - "This includes the CD33 or CD123 targeted molecules, as tagraxofusp, which has demonstrated activity as a front-line therapy...Among intensive treatment group, 45 (34%) received AML-like (34 with 3+7, 9 FLAG-IDA, and 2 HAM); 60 (46%) ALL-like (55 HyperCVAD and 5 with PETHEMA-ALL), and 27 (20%) lymphoma-like (17 CHOP, 7 etoposide-based, 1 ABVD, and 2 bortezomib-based). The non-intensive group included 21 (40%) AML-like regimens (10 AZA, 5 AZA-VEN, 4 LDAC, and 2 fludarabine+LDAC); 18 (34%) CD123 or CD33 targeted molecules, 9 (16%) ALL-like therapies (vincristine and dexamethasone-based), and 5 (10%) lymphoma-like, including 3 with radiotherapy or intralesional chemotherapy...The differences between treatment subgroups, such as age at diagnosis, among others, could influence the differences in OS. Larger studies and observational protocols are needed to identify best upfront regimens for BPDCN."

Clinical • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Lymphoma • Oncology • CD123 • IL3RA

Pilot Phase II Trial of the Combination of Tagraxofusp with Pacritinib in Patients with Myelofibrosis, Tagpac Study (ASH 2024) - P1 | "We expect to enroll the first patient in this trial in August of 2024. We plan to enroll 10 subjects and based on observed efficacy and safety results to expand to 20 subjects."

Clinical • P2 data • Anemia • Chronic Myeloid Leukemia • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Myelofibrosis • Oncology • ACVR1 • CD123 • IL3RA • IRAK1 • JAK2