Tagraxofusp providing good prospects against the rare but fatal BPDCN disorder

The Phase II study has achieved positive results for the application of the investigational drug tagraxofusp in tackling a rare but often fatal bone marrow and blood disorder.

The breakthrough is achieved for a condition with no currently approved therapies

 The Phase II study has achieved positive results for the application of the investigational drug tagraxofusp in tackling a rare but often fatal bone marrow and blood disorder. 

The University of Texas’ MD Anderson Cancer Center has announced an important milestone in the Phase I/II study led by investigators in this center on the investigational drug “tagraxofusp”. This recombinant fusion protein has shown positive response rates on cases of the rare blastic plasmacytoid dendritic cell neoplasm (BPDCN). This bone marrow and blood disorder, as of present have had to approved therapies despite it being a rather highly aggressive and fatal ailment. 

In the context of the 60th American Society of Hematology Annual Meeting in San Diego, USA, Professor Naveen Pemmaraju (Professor of Leukemia, MD Anderson Cancer Center, University of Texas, USA) presented some initial findings. "This was the largest prospectively designed multi-center, multi-cycle clinical trial specifically [for cases of] BPDCN," said the professor, adding that even for frontline-treated patients, the response rate for tagraxofusp has been of 90%. And Prof. Pemmaraju’s optimism is particularly encouraging to BPDCN patients who have had no disorder-specific treatments until present. Historically, these patients had to undergo cytotoxic chemotherapeutic strategies after diagnosis, with low response rates. Complicating things further, BPDCN patients receive instead chemotherapies approved for other types of blood cancers or are ineligible for standard chemotherapeutic approaches.

The phase study selected forty-five patients with 70 years as a median age, enrolled in seven sites and who were administered tagraxofusp as a first-line or relapsed/refractory treatment. While the median study follow-up was 13.8 months, further patients are being enrolled for the clinical trial’s next phase, through which hopefully continuing access to tagraxofusp can be sustained for patients of BPDCN.  

The investigational drug tagraxofusp is targeting in this trial, the CD-123, a cell surface receptor that is expressed in BPDCN and other blood conditions. Aside from BPDCN, tagraxofusp is being tested on clinical trials with chronic myelomonocytic leukemia (CMML) and myelofibrosis patients.

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