Elzonris (tagraxofusp-erzs) / Menarini 
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 17 Diseases   11 Trials   11 Trials   835 News 


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  • ||||||||||  Immune Therapy Approaches in MDS (Level 4, Grand Ballroom G-L) -  Jul 26, 2022 - Abstract #SOHO2022SOHO_65;    
    Its aberrant expression on leukemic blasts stem cells (LSCs) and blasts but not on that of normal hematopoietic stem cells make this an attractive therapeutic target.5,6 Binding of the high affi nity anti-TIM3 IgG4 antibody sabatolimab to TIM3 results in phagocytic killing of LSCs and blasts and in combination with DNMT inhibitors have demonstrated safety, minimal immune toxicity and importantly early evidence of durable remissions in the phase 1b setting.7 Toxicities were predominantly expected cytopenias and mild GI intolerance (constipation, nausea)...An on-going phase 1/2 trial is evaluating tagraxofusp monotherapy in chronic myelomonocytic leukemia (N=38) reported tolerability including a 21% capillary leak syndrome (CLS) rate (11% grade 2 and 11% grade 3) and encouraging median overall survival of 15.6 months (95% CI, 8.1-17.5; range, 0.36-40.77).11 Based on strong preclinical evidence demonstrating tagraxofusp resistance was due to decreased expression of DPH1 which could be restored with azacitidine, a phase 1b trial of azacitidine and tagraxofusp in MDS/AML was launched...Targeting immunological dysfunctional pathways is a well-studied approach in MDS with prior successes with immunosuppressive therapies (ATG), immunomodulatory agents such as lenalidomide in 5q deletion MDS and luspatercept which limits TGF-beta signaling...Notably, among 7 patients with splicing mutated higher risk MDS there was 57% marrow CR rate including 1 with HI.15 This trial has plans to assess CA-4948 in combination with azacitidine or venetoclax...It remains a challenge to interpret small single arm studies and mixing populations that are truly resistant (HMA refractory) and treatment naïve, and different formulations of the same class of drugs and thus reliance on large phase 2/3 trials and importantly randomized trials will be most informative. A focus on identifying select disease subpopulations in these early trials (‘signal fi nding’ i.e. magrolimab with TP53 mutations or splicing mutations with IRAK4 inhibition) is an opportunity we cannot afford to miss.
  • ||||||||||  Elzonris (tagraxofusp) / Menarini, Nippon Shinyaku
    Review, Journal:  Blastic plasmacytoid dendritic cell neoplasm (BPDCN): A promising future in the era of targeted therapeutics. (Pubmed Central) -  Jul 24, 2022   
    The CD123-targeted agent, tagraxofusp, was the first Food and Drug Administration-approved monotherapy in the treatment of BPDCN...Although relapsed/refractory disease and central nervous system disease both remain formidable areas of research, there are several promising therapeutic approaches that could have a significant impact on the trajectory of treatment. This review will provide detailed insight on the novel drugs currently in use and those being explored in the management of BPDCN.
  • ||||||||||  Elzonris (tagraxofusp) / Menarini, Nippon Shinyaku
    Review, Journal:  Clinical Insights into the Management of Blastic Plasmacytoid Dendritic Cell Neoplasm. (Pubmed Central) -  Jul 8, 2022   
    In December 2018, the FDA approved tagraxofusp-erzs for adults and pediatric patients older than 2 years who have either treatment-naïve or relapsed/refractory BPDCN...Our induction regimen also includes intrathecal chemotherapy for central nervous system prophylaxis. Patients with poor performance status who are treatment-naïve or patients with relapsed/refractory disease have limited therapeutic options, and we strongly recommend enrollment in clinical trials; several novel agents and combinations are currently under clinical investigation for both treatment-naïve and relapsed/refractory BPDCN.
  • ||||||||||  Elzonris (tagraxofusp) / Menarini, Nippon Shinyaku
    Journal:  Characteristics and Outcomes of Patients with Blastic Plasmacytoid Dendritic Cell Neoplasm Treated with Frontline HCVAD. (Pubmed Central) -  May 22, 2022   
    We conducted a retrospective analysis of patients with BPDCN (n = 100), evaluating complete remission (CR) and median overall survival (OS) among 3 groups: those who received frontline HCVAD-based therapy (n = 35), SL-401 (n = 37), or 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.
  • ||||||||||  Elzonris (tagraxofusp) / Menarini, Nippon Shinyaku
    Review, Journal:  CD123 and More: How to Target the Cell Surface of Blastic Plasmacytoid Dendritic Cell Neoplasm. (Pubmed Central) -  May 15, 2022   
    Recently, the first-in-class CD123-targeting therapy, tagraxofusp, was approved for treatment of BPDCN...Here, we report on novel and innovative therapies in development to target surface molecules in BPDCN currently in clinical trials or in preclinical stages. We also discuss new cell surface targets that may have implications for future BPDCN treatment.
  • ||||||||||  Elzonris (tagraxofusp-erzs) / Menarini, Nippon Shinyaku
    Trial completion date, Trial initiation date, Trial primary completion date, Post-transplantation:  Study of Tagraxofusp for Post-Transplant Maintenance for Patients With CD 123+ AML, MF and CMML (HSCT 002) (clinicaltrials.gov) -  May 5, 2022   
    P1,  N=44, Not yet recruiting, 
    Treatment with TAG demonstrated promising efficacy, with responses that enabled 5 of 8 pediatric pts, including 3 1L and 2 R/R, to be bridged to stem cell transplant. Trial completion date: Jul 2026 --> Oct 2026 | Initiation date: Mar 2022 --> Jun 2022 | Trial primary completion date: Apr 2024 --> Jul 2024
  • ||||||||||  Review, Journal:  Novel treatments for myelofibrosis: beyond JAK inhibitors. (Pubmed Central) -  Apr 30, 2022   
    Specifically, we discuss agents that target epigenetic modification (pelabresib, bomedemstat), apoptosis (navitoclax, navtemdalin), signaling pathways (parsaclisib), bone marrow fibrosis (AVID200, PRM-151), in addition to other targets including telomerase (imetelstat), selective inhibitor of nuclear transport (selinexor), CD123 (tagraxofusp) and erythroid maturation (luspatercept). We end by providing commentary on the ongoing and future therapeutic development in myelofibrosis.
  • ||||||||||  Elzonris (tagraxofusp) / Menarini, Nippon Shinyaku
    Clinical, Journal:  Blastic plasmacytoid dendritic-cell neoplasia: a challenging case report. (Pubmed Central) -  Mar 8, 2022   
    Treatment with the recently approved CD123-cytotoxin Tagraxofusp showed initially a very good response. This case reflects diagnostic and therapeutic difficulties in BPDCN as very rare, easily misdiagnosed neoplasia and the need for precise diagnostic care.
  • ||||||||||  Elzonris (tagraxofusp-erzs) / Menarini
    Enrollment open, Combination therapy:  Tagraxofusp and Decitabine for the Treatment of Chronic Myelomonocytic Leukemia (clinicaltrials.gov) -  Mar 7, 2022   
    P1/2,  N=64, Recruiting, 
    This case reflects diagnostic and therapeutic difficulties in BPDCN as very rare, easily misdiagnosed neoplasia and the need for precise diagnostic care. Not yet recruiting --> Recruiting