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A new phase III trial has found that luspatercept reduced the severity of anemia in patients with transfusion-refractory, lower-risk myelodysplastic syndromes with ring sideroblasts. Results from the MEDALIST trial, which included data from 65 sites in 11 countries, were published recently in the New England Journal of Medicine.

Ruxolitinib plus low-dose pegylated interferon-a2 improved peripheral blood cell counts, bone marrow cellularity and fibrosis, and symptom burden with acceptable toxicity in patients with polycythemia vera or proliferative myelofibrosis, according to the 2-year, end-of-study results of the phase II COMBI study, which were recently published in Haematologica.

CPI-0610, a selective and potent oral bromodomain and extra-terminal domain inhibitor, induced spleen and symptom responses as early as 12 weeks in combination with the JAK inhibitor ruxolitinib in patients with JAK inhibitor-naïve myelofibrosis, according to the preliminary findings from the phase II MANIFEST trial presented at the 2019 ASH Annual Meeting.

A multi-antigen off-the-shelf chimeric antigen receptor natural killer cell therapy has been included in the ASH annual meeting spotlight due to exciting preclinical evidence. An investigational new drug application was approved in September 2019 for the therapy, labeled as FT596, developed by Fate Therapeutics, and human trials are scheduled to start in the first quater of 2020.