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

A PHASE II TRIAL OF TAGRAXOFUSP, HYPER-CVAD, AND VENETOCLAX FOR PATIENTS WITH NEWLY DIAGNOSED OR RELAPSED/REFRACTORY BPDCN (EHA 2026) - P2 | "Methods Adults ( > 18 years [yrs]) w/ ND or R/R BPDCN received: Cycle [C] 1: TAG days [d] 1-5; C2, 4, 6, 8: TAG d1 – 5 w/ VEN d2 – 14 (C2) and d1 – 7 (C4, 6, 8); C3, 7: VEN d1 – 7 w/ Hyper-CVAD or mini-hyper-CVD; C5: VEN d1 – 7 w/ high-dose methotrexate/cytarabine or mini-methotrexate/cytarabine...This abstract was accepted and previously presented at the 2026 ASCO Annual Meeting. Overall Survival in Frontline (A) and Relapsed/Refractory (B)."

Clinical • P2 data • Hematological Malignancies • Infectious Disease • Myelodysplastic Syndrome • Respiratory Diseases • Septic Shock • ASXL1 • CD123 • DNMT3A • IL3RA • KRAS • NRAS • TET2

Tagraxofusp, Azacitidine, and Venetoclax in Blastic Plasmacytoid Dendritic Cell Neoplasm. (PubMed, Blood) - P1 | "A high proportion of patients proceeded to allogeneic stem cell transplantation in remission (63% 1L, including 10 of 11 patients age 75 or younger; and 55% R/R). TAG-AZA-VEN is highly active in both untreated and relapsed BPDCN with a predictable and manageable safety profile, supporting its use as a new therapeutic option."

IO biomarker • Journal • Hematological Disorders • Hematological Malignancies • Oncology • Transplantation • BCL2 • CD123 • IL3RA

Longer Survival With Tagraxofusp vs Venetoclax in Patients With Blastic Plasmacytoid Dendritic Cell Neoplasm: Results of a Real-World Analysis (SOHO 2026) - "Treatment with a TAG-based regimen was associated with clinically meaningful longer median OS vs VEN-based regimens; this was consistent across patients receiving monotherapy or HSCT. Importantly, more patients received a subsequent LOT after TAGbased regimens. Results support first-line TAG as standard of care for eligible patients with BPDCN."

Clinical • Real-world • Real-world evidence • Hematological Malignancies • Oncology • CD123 • IL3RA

Hematopoietic cell transplantation for blastic plasmacytoid dendritic cell neoplasm: clinical practice guidelines from the American Society for Transplantation and Cellular Therapy. (PubMed, Transplant Cell Ther) - "Treatment of BPDCN has evolved over the years from conventional chemotherapy to novel targeted therapies including anti-CD123 antibody drug conjugates, namely tagraxofusp and pivekimab sunirine, and the BCL-2 inhibitor, namely venetoclax. Post-HCT intrathecal chemotherapy was also recommended regardless of involvement of the central nervous system. Panelists recognized that other unique clinical scenarios not included in these recommendations might demand individualized treatment approaches."

Clinical guideline • Journal • Hematological Disorders • Hematological Malignancies • Oncology • Transplantation • CD123 • IL3RA

Novel Use of Tagraxofusp-erzs/Elzonris in CD123-Positive Gamma/Delta Cutaneous T-Cell Lymphoma (SOHO 2026) - "First-line treatment with brentuximab vedotin–based chemotherapy resulted in unclear benefit, and subsequent brentuximab vedotin monotherapy produced only a transient response. Disease progression continued despite additional therapy with romidepsin, which was discontinued due to intolerance...It also highlights the potential role of targeted therapies in refractory PCGDTCL. Further investigation is warranted to determine the clinical significance of CD123 expression and evaluate the efficacy of CD123-directed treatments in this aggressive malignancy."

Cutaneous T-cell Lymphoma • Hematological Malignancies • Lymphoma • Oncology • T Cell Non-Hodgkin Lymphoma • CD123 • CD5 • CD7 • IL3RA • KEAP1 • TNFRSF8

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 • 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

A phase II trial of tagraxofusp, hyper-CVAD, and venetoclax for patients with newly diagnosed or relapsed/refractory BPDCN. (ASCO 2026) - P2 | " Adults (≥18 years [yrs]) w/ ND or R/R BPDCN received: Cycle [C] 1: TAG days [d] 1-5; C2, 4, 6, 8: TAG d1 – 5 w/ VEN d2 – 14 (C2) and d1 – 7 (C4, 6, 8); C3, 7: VEN d1 – 7 w/ Hyper-CVAD or mini-hyper-CVD; C5: VEN d1 – 7 w/ high-dose methotrexate/cytarabine or mini-methotrexate/cytarabine... As frontline therapy, TAG with Hyper-CVAD, and VEN shows promising safety with no early mortality or high-grade CLS, with high response rates enabling frequent SCT consolidation and no CNS relapse."

Clinical • P2 data • Hematological Malignancies • Infectious Disease • Oncology • Respiratory Diseases • Septic Shock • ASXL1 • CD123 • DNMT3A • IL3RA • KRAS • NRAS • TET2

Phase 1b trial of tagraxofusp in combination with azacitidine with or without venetoclax in acute myeloid leukemia. (PubMed, Blood Adv) - "Median overall survival and progression-free survival were 14 months (95% CI, 9.5-NA) and 8.5 months (95% CI, 5.1-NA), respectively. In summary, TAG-AZA-VEN shows encouraging safety and activity in high-risk AML, including TP53-mutated disease, supporting further clinical development of TAG combinations."

Combination therapy • IO biomarker • Journal • P1 data • Acute Myelogenous Leukemia • Hematological Malignancies • Infectious Disease • Leukemia • Myelodysplastic Syndrome • Oncology • CD123 • IL3RA • TP53

Tagraxofusp, azacitidine, and venetoclax (TAG-AZA-VEN) triplet therapy shows efficacy, tolerability, and transplant potential in patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN): Results of a phase 2 trial (ASH 2025) - P1 | "Triplet therapy with TAG-AZA-VEN is effective and feasible in patients with previouslyuntreated or R/R BPDCN, including in an older patient population. The safety profile is consistent withprior studies of TAG and AZA/VEN, and triplet therapy does not add new AEs not seen with TAG orAZA/VEN. A high proportion of patients (1L and R/R) proceeded to transplant in remission."

Clinical • IO biomarker • P2 data • Atrial Fibrillation • Cardiovascular • CNS Disorders • Diabetes • Febrile Neutropenia • Hematological Malignancies • Infectious Disease • Neutropenia • Renal Disease • Thrombocytopenia • Transplantation • BCL2 • CD123 • IL3RA

Successful Salvage of Early-Relapsed BPDCN With CD123-Directed Therapy and Azacitidine/Venetoclax (SOHO 2026) - P2 | "Recent evidence demonstrates that cluster of differentiation 123 (CD123)-targeted immunotherapy, particularly Tagraxofusp, achieves improved outcomes compared to chemotherapy-based regimens2,3. BPDCN is a rare and aggressive hematodermic malignancy that has shown clinical response to CD123 immunotherapy. In this case, using a triple-therapy was safe and effective in managing an early relapse, allowing a successful bridge to allogeneic stem cell transplant. The triple-therapy protocol is currently under investigation (NCT06456463)."

Oncology • CD123 • CD4 • IL3RA • NCAM1

Longer survival and favorable healthcare resource utilization of tagraxofusp versus venetoclax in patients with blastic plasmacytoid dendritic cell neoplasm: results of a real-world analysis. (PubMed, Front Oncol) - "In this retrospective claims-based analysis, tagraxofusp was associated with longer survival versus venetoclax, including across treatment contexts. These real-world results are consistent with the established role of tagraxofusp as the first-line standard of care for patients with BPDCN, irrespective of transplant eligibility, and support continued evaluation of tagraxofusp-based regimens as a backbone for future combinations."

HEOR • Journal • Real-world evidence • Oncology • Transplantation • CD123 • IL3RA

Two Blastic Plasmacytoid Dendritic Cell Neoplasms treated with the CD123-targeted agent Tagraxofusp presenting divergent outcomes. (PubMed, J Dtsch Dermatol Ges) - No abstract available

Journal • Oncology • CD123 • IL3RA

Decreased TXNRD1 is associated with resistance to tagraxofusp in blastic plasmacytoid dendritic cell neoplasms, as seen in phase II. (PubMed, Leukemia) - P1/2 | "Missense and truncating mutants displayed reduced sensitivities to hypomethylating agents and prolonged S-phase stasis. These results suggest that the levels of TXNRD1 interact with intrinsic TET2 truncating mutations within the bone marrow to modulate patient response to tagraxofusp."

Journal • P2 data • Infectious Disease • Oncology • CD123 • IL3RA • TET2

Central nervous system management remains an evidence gap in first-line tagraxofusp therapy for blastic plasmacytoid dendritic cell neoplasm. Comment on: "First-line tagraxofusp leads to durable responses and prolonged survival in adults with blastic plasmacytoid dendritic cell neoplasm regardless of fitness level". (PubMed, Haematologica) - "Not available."

Journal • Oncology

Diagnosis and Treatment of AML-Type Blastic Plasmacytoid Dendritic Cell Neoplasm (SOHO 2026) - "In these rare neoplasms, diagnosis and differential diagnosis are crucial. For BPDCN, tagraxofusp (CD123-directed cytotoxin) is primarily recommended as induction therapy. However, as tagraxofusp is unavailable in Turkey, 3+7 therapy is given for AML-type BPDCN."

Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Chronic Myelomonocytic Leukemia • Hematological Malignancies • Leukemia • Oncology • ASXL1 • CD123 • CD33 • CD4 • CDKN2A • FLT3 • IL3RA • KIT • NCAM1 • RUNX1 • TET2 • TP53

Characteristics and Outcomes of Patients with Blastic Plasmacytoid Dendritic Cell Neoplasm Treated with Frontline HCVAD. (PubMed, Blood Adv) - "In this setting, we sought to analyze outcomes of the cytotoxic chemotherapy backbone regimen hyperfractionated cyclophosphamide, vincristine, adriamycin, and dexamethasone (HCVAD) in BPDCN. We conducted a retrospective analysis of patients with BPDCN (n=100), evaluating complete remission (CR) and median overall survival (OS) among three groups: those who received frontline HCVAD-based (n=35) vs SL-401 (n=37) vs other regimens (n=28)...These results suggest a continued important role for HCVAD-based chemotherapy for BPDCN, even in the modern targeted-therapy era, with high CR rates in the frontline setting. Further studies must establish the clinical activity, feasibility, and safety, of doublet/triplet combinations of targeted therapies plus cytotoxic agents and addition of CNS prophylaxis, with ultimate goal of durable long-term remissions for patients with BPDCN."

Journal • Bone Marrow Transplantation • Hematological Malignancies • Oncology • Transplantation • BCL2 • CD123

Tagraxofusp in Blastic Plasmacytoid Dendritic-Cell Neoplasm. (PubMed, N Engl J Med) - P1/2; "In adult patients with untreated or relapsed BPDCN, the use of tagraxofusp led to clinical responses. Serious adverse events included capillary leak syndrome; hepatic dysfunction and thrombocytopenia were common. (Funded by Stemline Therapeutics and the Leukemia and Lymphoma Society Therapy Acceleration Program; ClinicalTrials.gov number, NCT02113982.)."

Journal • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Lymphoma • Oncology • Transplantation

Long-Term Benefits of Tagraxofusp for Patients With Blastic Plasmacytoid Dendritic Cell Neoplasm. (PubMed, J Clin Oncol) - "In first-line patients with BPDCN, TAG monotherapy resulted in high and durable responses, allowing many to bridge to stem-cell transplant. TAG was generally well-tolerated with a predictable and manageable safety profile."

Journal • Bone Marrow Transplantation • Hematological Malignancies • Oncology • Transplantation • CD123

INTERIM ANALYSIS OF A REGISTRATION ENABLING STUDY OF PIVEKIMAB SUNIRINE (PVEK, IMGN632) A CD123-TARGETING ANTIBODY-DRUG CONJUGATE, IN PATIENTS WITH BLASTIC PLASMACYTOID DENDRITIC CELL NEOPLASM (BPDCN) (EHA 2023) - P1/2 | "BPDCN currently has 1 approved therapy, tagraxofusp (TAG), with a median age of 68, CR/CRc rate of 57% and mOS of 15.8 mos (n=65; Pemmaraju JCO 2022). PVEK demonstrates compelling activity in frontline and R/R BPDCN pts, including durable responses in the R/R setting for pts who received prior TAG. PVEK safety was manageable with primarily low-grade IRRs and edema and no new safety signals were observed. Enrollment continues in the pivotal de novo frontline BPDCN cohort (NCT03386513) Antibody targeting, Monoclonal antibody, Myeloid malignancies"

Clinical • Chronic Myelomonocytic Leukemia • Constipation • Fatigue • Gastroenterology • Gastrointestinal Disorder • Hematological Disorders • Hematological Malignancies • Neutropenia • Oncology • Thrombocytopenia • Transplantation • CD123

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

Efficacy and safety of pivekimab sunirine (PVEK) in patients (pts) with blastic plasmacytoid dendritic cell neoplasm (BPDCN) in the CADENZA study. (ASCO 2025) - P1/2 | "Pts with R/R BPDCN had received 1-3 prior systemic therapies; 57% had prior tagraxofusp. PVEK treatment demonstrated promising efficacy, with high and durable CR + CRc responses. PVEK was tolerable at the RP2D. The safety profile was manageable, with no CLS events."

Clinical • Hematological Malignancies • Hepatology • Oncology • CD123 • IL3RA

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 | "1L TAG BPDCN treatment yielded high response rates regardless of HCT-CI fitness, with a similar safety profile across HCT-CI groups. TAG enabled bridge to HCT across all fitness groups, including pts with high risk possibly ineligible for IC, as TAG is not associated with prolonged myelosuppression seen with IC. These results affirm TAG as the SOC in 1L treatment for the majority of pts with BPDCN."

Clinical • Hematological Disorders • Hematological Malignancies • Oncology • Thrombocytopenia • CD123 • IL3RA

EFFICACY AND SAFETY OF PIVEKIMAB SUNIRINE (PVEK) IN PATIENTS (PTS) WITH BLASTIC PLASMACYTOID DENDRITIC CELL NEOPLASM (BPDCN) IN THE CADENZA STUDY (EHA 2025) - P1/2 | "Pts with R/R BPDCN had received 1-3 prior systemic therapies; 57% had prior tagraxofusp (TAG). PVEK treatment demonstrated promising efficacy, with high and durable CR + CRc responses, and was tolerable at the RP2D. The safety profile was manageable, with no CLS events, predominantly low-grade peripheral edema, and very low incidence of resolvable VOD, a potential TEAE important to monitor during treatment. These results support PVEK as a potential new treatment option for adult pts with BPDCN."

Clinical • Fatigue • Hematological Malignancies • Hepatology • Oncology • CD123 • IL3RA

First-line tagraxofusp induces durable responses with prolonged survival in adults younger than 50 years with blastic plasmacytoid dendritic cell neoplasm: Post hoc analysis of a phase 2 trial. (PubMed, Br J Haematol) - No abstract available

Journal • P2 data • Retrospective data • Oncology • Transplantation

Favorable Outcomes, Including Posttransplant Survival, Are Seen Independent of Baseline Skin Burden in Treatment-Naïve Patients With Blastic Plasmacytoid Dendritic Cell Neoplasm Treated With Tagraxofusp: Subgroup Analysis of a Pivotal Trial (SOHO 2026) - P1/2 | "First-line TAG enabled patients with BPDCN to achieve CR/CRc and successfully bridge to HCT, independent of baseline skin disease burden. In both groups, patients who bridged to HCT following TAG-induced remission demonstrated prolonged survival, underscoring TAG's role in potentially curative HCT. These results confirm TAG is an effective first-line therapy irrespective of disease burden or clinical presentation and serves as the standard of care in all eligible patients with BPDCN."

Clinical • Post-transplantation • Oncology • CD123 • IL3RA