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

SL-401 in Combination With Azacitidine or Azacitidine/Venetoclax in Acute Myeloid Leukemia (AML), High-Risk Myelodysplastic Syndrome (MDS) or Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) (clinicaltrials.gov) - P1 | N=72 | Recruiting | Sponsor: Dana-Farber Cancer Institute | Trial completion date: May 2026 ➔ May 2027 | Trial primary completion date: May 2025 ➔ May 2026

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

HOSPITALIZATION TIME AND TREATMENT COSTS FOR ADULT PATIENTS WITH BLASTIC PLASMACYTOID DENDRITIC CELL NEOPLASM (BPDCN). A SPANISH PETHEMA STUDY (EHA 2025) - "The different regimens were: clinical trial with immunotherapy (8 patients, 21%), 3+7 (7 patients, 18%), HYPERCVAD (6 patients, 15%), tagraxofusp (6 patients, 15%), CHOP (5 patients, 13%), FLAG-IDA (3 patients, 8%), LDAC (1 patient, 3%), FLUGA (1 patient, 3%), SMILE (1 patient, 3%)... Hospitalization time and costs in BPDCN are high, mainly due to hospitalizations and HSCT. Studies are needed to compare chemotherapy vs. CD123 inhibitors for cost-effectiveness."

Clinical • Treatment costs • Bone Marrow Transplantation • Oncology • CD123 • IL3RA

OUTCOMES IN PATIENTS YOUNGER THAN 50 YEARS OLD WITH TREATMENT-NAÏVE BLASTIC PLASMACYTOID DENDRITIC CELL NEOPLASM TREATED WITH TAGRAXOFUSP: SUBANALYSIS OF A PHASE 1/2 TRIAL (EHA 2025) - P1/2 | "In treatment-naïve BPDCN patients <50 years old, including adolescents and young adults, TAG, a chemotherapy-free option, induced high rates of CR/CRc (70%) and allowed all patients who achieved CR/CRc to bridge to SCT. TAG was well tolerated with no cumulative AEs or cumulative myelosuppression. No investigator-assessed CLS was observed in these younger patients."

Clinical • P1/2 data • Hematological Malignancies • Oncology • Pediatrics • Thrombocytopenia • CD123 • IL3RA

BLASTIC PLASMACYTOID DENDRITIC CELL NEOPLASM (BPDCN): PROGNOSTIC FACTORS AND TREATMENT OUTCOMES IN 69 MAYO CLINIC PATIENTS (EHA 2025) - "When censored for SCT, the 1y OS for patients receiving an ALL regimen, tagraxofusp, or other regimen was 82%, 67%, and 47%, respectively (p=0.15).For the 17 patients undergoing allogenic SCT, the most common conditioning regimen was fludarabine/busulfan (n=6) followed by cyclophosphamide/total body irradiation (TBI) (n=3). Newly-diagnosed BPDCN patients treated with an ALL regimen or tagraxofusp followed by SCT had longer OS compared to those treated with all other regimens. WBC >11 x109/L and abnormal karyotype were independent predictors of inferior survival whereas SCT was beneficial."

Biomarker • Clinical • Graft versus Host Disease • Hematological Malignancies • Immunology • Oncology • ASXL1 • NRAS • SRSF2 • TET2

SUBGROUP ANALYSIS BY FITNESS CRITERIA OF PATIENTS WITH BLASTIC PLASMACYTOID DENDRITIC CELL NEOPLASM TREATED WITH FIRST-LINE TAGRAXOFUSP (EHA 2025) - P1/2 | "First-line 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 patients with high risk possibly ineligible for intensive chemotherapy, as TAG is not associated with prolonged myelosuppression seen with intensive chemotherapy. These results affirm TAG as the standard of care in first-line treatment for the majority of patients with BPDCN."

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

ANTI-TUMOR ACTIVITY AND RESTORATION OF HEMATOPOIESIS ACHIEVED WITH TAGRAXOFUSP IN PATIENTS WITH TREATMENT-NAÏVE BLASTIC PLASMACYTOID DENDRITIC CELL NEOPLASM (BPDCN): PIVOTAL TRIAL SUBANALYSIS (EHA 2025) - P1/2 | "The rapid and durable responses associated with TAG in the pivotal trial coupled with its safety profile, lack of cumulative myelosuppression, and restoration of normal hematopoiesis support 1L TAG monotherapy for BPDCN. The on-target effect and unique BM-sparing profile of TAG is paramount in treating myelosuppressive CD123+ hematologic disease and support TAG as a combination partner for CD123+ hematologic malignancies."

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

Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN): 2025 Update on Diagnosis, Pathophysiology, Risk Assessment, and Management. (PubMed, Am J Hematol) - "The median overall survival is 18-24 months. Potential risk factors for shortened survival include older age, overt bone marrow involvement, and genetic abnormalities."

Journal • Dermatology • Hematological Disorders • Hematological Malignancies • Infectious Disease • Oncology • Transplantation • CD123 • CD4 • IL3RA • NCAM1

TAGVEN: Phase II Study Evaluating the Efficacy and Safety of the Combination of Tagraxofusp and Venetoclax in Treatment-naive Blastic Plasmacytoid Dendritic Cell Neoplasm Patients (clinicaltrials.gov) - P2 | N=33 | Not yet recruiting | Sponsor: French Innovative Leukemia Organisation

New P2 trial • Oncology

Blastic plasmacytoid dendritic cell neoplasm (BPDCN): Prognostic factors and outcomes in 68 Mayo Clinic patients. (ASCO 2025) - "Newly-diagnosed BPDCN patients treated with an ALL regimen or tagraxofusp followed by SCT had longer OS compared to those treated with all other regimens. WBC >11 x109/L and abnormal karyotype were independent predictors of inferior survival whereas SCT was beneficial."

Biomarker • Clinical • Hematological Malignancies • Oncology • ASXL1 • NRAS • SRSF2 • TET2

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

Tagraxofusp and low-intensity chemotherapy for the treatment of CD123-positive relapsed or refractory acute myeloid leukemia. (ASCO 2025) - P1/2 | "Funded by No funding sources reported Clinical Trial Registration Number: NCT06561152 Background: The combination of venetoclax and a hypomethylating agent (Ven/HMA) is the standard frontline (1L) therapy for patients with acute myeloid leukemia (AML) who are ineligible for intensive chemotherapy (IC)...Cladribine (CLAD) with LDAC has previously been shown to be well-tolerated and effective in IC-ineligible patients with newly diagnosed AML...The study began enrolling patients in January 2025 and is actively recruiting. Clinical Trial Registration NCT06561152."

IO biomarker • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • BCL2 • 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

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 | "In treatment-naïve BPDCN pts <50 yo, including AYAs, TAG, a chemotherapy-free option, induced high rates of CR/CRc (70%) and allowed all pts who achieved CR/CRc to bridge to SCT. TAG was well tolerated with no cumulative AEs or cumulative myelosuppression. No investigator-assessed CLS was observed in these younger pts."

Clinical • P1/2 data • Hematological Malignancies • Oncology • Pediatrics • Thrombocytopenia • CD123 • IL3RA

New leads to enhance tagraxofusp efficacy in pDC-AML. (PubMed, Blood Neoplasia) - No abstract available

Journal • Acute Myelogenous Leukemia

GO-TAG: Tagraxofusp-erzs, an IL-3 Diphtheria Fusion Protein, in Combination With Gemtuzumab Ozogamicin in Patients With Relapsed/Refractory AML (clinicaltrials.gov) - P1 | N=36 | Active, not recruiting | Sponsor: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins | Recruiting ➔ Active, not recruiting

Enrollment closed • Acute Myelogenous Leukemia • Hematological Malignancies • Infectious Disease • Leukemia • Oncology • CD33 • IL3RA

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) - P2 | "Part 2 will investigate TAG at the selected dose combined with VEN/AZA; 48 additional patients will enroll in 2 cohorts by TP53 mutation status. An interim futility analysis will occur after 12 patients (including part 1 patients treated at selected dose) are enrolled in each cohort."

Clinical • P2 data • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • CD123 • IL3RA • TP53

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

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