Celgene scores at ASH18 with double luspatercept data

A pair of clinical trial wins for Celgene’s luspatercept at the American Society of Haematology (ASH) meeting have set up a filing for the new drug in mid-2019. The MEDALIST trial in second-line myelodysplastic syndrome (MDS) and BELIEVE study in transfusion-dependent thalassemia (TDT) both met their primary objective of a reduced need for red blood cell transfusions compared to placebo, backing up Celgene’s assertion that luspatercept could in time become a $2 billion product.
Luspatercept works by accelerating the maturation of red blood cells, boosting their numbers, and works in a different way to current drugs such as erythropoiesis-stimulating agents (ESAs). It’s being developed for a range of diseases characterized by severe anemia that requires regular blood transfusions. In MEDALIST, 38% of MDS patients treated with luspatercept were able to go for eight weeks without needing a transfusion, the primary outcome measure, compared to 13% of the placebo group. Meanwhile, the drug also hit the main objective in the BELIEVE trial – a 33% or greater reduction in transfusions during weeks 13 to 24 – in 21% of TDT patients, versus 4.5% of the placebo group.