Investigational drug shows promising results in Phase II study of aggressive, often fatal blood disorder with no approved therapies

(University of Texas M. D. Anderson Cancer Center) A Phase I/II study, led by investigators at The University of Texas MD Anderson Cancer Center, reports an investigational drug called tagraxofusp has demonstrated high response rates in patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN), a rare but highly aggressive -- and often fatal bone marrow and blood disorder -- for which there are no existing approved therapies.