Luspatercept Passes Phase 3 Clinical Trial for Rare Blood Disease

July 3, 2018  Source: FierceBiotech 160

A type of blood cancer named myelodysplastic syndromes occurs in people where the blood-forming cells in the bone marrow become abnormal, leading to the depletion of one of more types of blood cells.

Acceleron Pharma and its partner Celgene have revealed that luspatercept hit its primary and secondary endpoints in phase 3 clinical trial of patients affected with chronic anemia associated with a rare blood disease. The need for red blood transfusions is decreased by luspatercept for at least 8-12 consecutive weeks in patients affected with chronic anemia and myelodysplastic syndromes (MDS), where erythropoietin-stimulating agent (ESA) drugs do not work. Luspatercept increases red blood cell production by targeting the transforming growth factor-beta (TGF-beta) proteins, which helps in red cell differentiation and maturation.

Jay Backstrom, M.D., Chief Medical Officer from Celgene said, “This result from the phase III MEDALIST trial demonstrates the potential clinical benefit of luspatercept as an erythroid maturation agent for the treatment of chronic anemia in patients with low-to-intermediate risk MDS.”

Luspatercept is in a late-stage trial in beta-thalassemia. The companies aims to file the drug for approval in the U.S. and Europe during the initial half of 2019 and proceed for human trials in MDS patients who were not treated with ESA before.

By Ddu
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