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Preliminary results from a Phase 2 clinical trial assessing Acceleron Pharma's (NASDAQ:XLRN) luspatercept in patients with lower risk myelodysplastic syndromes (MDS) show that three month's treatment with luspatercept increased hemoglobin levels and achieved transfusion independence in lower MDS patients. Longer term treatment produced sustained benefit for six months, possibly longer. The data were presented at the 20th Congress of the European Hematology Association in Vienna, Austria.
- Results from the 12-month extension study show that low transfusion burden patients (n=9) experienced a mean hemoglobin increase of ~2 g/dL (normal range: 13.5-17.5 g/dL for men and 12.0-15.5 g/dL for women) at one month which increased as high as 3.0 g/dL and was maintained for the six-month period for which data are available. In transfused patients, 43% achieved transfusion independence with several maintaining independence for more than six months.
- Also on a preliminary basis, data from a Phase 2 trial in beta thalassemia showed that 38% of patients treated with luspatercept experienced a mean increase in hemoglobin of at least 1.5 g/dL for at least nine weeks. In non-transfusion dependent patients who had iron overload (n=12), eight (67%) experienced reduced liver iron concentrations of 1-4.6 mg/g dry weight over a 16-week period. In transfusion dependent patients, 100% (n=10/10) who received luspatercept had more than a 40% reduction in transfusion burden while two of three with iron overload showed reduced liver iron concentration by 1.96 and 4.7 mg/g dry weight, respectively (reference value = ~1.8 mg/g dry weight).
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Luspatercept, being co-developed with Celgene (NASDAQ:CELG) is a protein therapeutic called a ligand trap. It promotes red blood cell formation by inhibiting members of the transforming growth factor beta superfamily, proteins that induce programmed cell death (apoptosis).