Acceleron Pharma Inc. (NASDAQ:XLRN), a leading biopharmaceutical company in the discovery and development of TGF-beta therapeutics to treat serious and rare diseases, today announced that the journal Blood has published results from the Phase 2 study of luspatercept in patients with red blood cell (RBC) transfusion-dependent and non-transfusion-dependent beta-thalassemia. Luspatercept is an investigational therapy that is part of a global collaboration between Acceleron and Celgene.
In this open-label, Phase 2 study (a three-month initial stage followed by a five-year extension stage), patients were treated with luspatercept subcutaneously once every three weeks. The primary outcome measures were a reduction in RBC transfusion burdencompared with pretreatmentin transfusion-dependent patients, and changes in hemoglobin levels from baseline in non-transfusion-dependent patients. The extension study is ongoing and evaluates the long-term safety and tolerability of luspatercept for up to 5 years.
Current treatment options for patients with beta-thalassemia are essentially limited to supportive therapy, including red blood cell transfusions, which leads to iron overload, said Antonio Piga, M.D., Professor, Department of Clinical and Biological Sciences at Turin University and lead author of the newly published study. Luspatercept has demonstrated an ability to improve hemoglobin levels and reduce transfusion burden through apparent restoration of the late-stage red blood cell maturation process, which is known to be inhibited in these patients. Based on these results and those of subsequent studies, luspatercept has the potential to address significant unmet medical needs for patients suffering from beta-thalassemia.
The article, entitled Luspatercept improves hemoglobin levels and blood transfusion requirements in a study of patients with beta-thalassemia, is now available online and will be published in a future print edition of Blood.
Were pleased that these important results are now fully available to the greater hematology community, said Robert K. Zeldin, M.D., Chief Medical Officer of Acceleron. This study provided key information on the efficacy and safety profile of luspatercept and informed the design of the Phase 3 trial, known as BELIEVE, for which positive results in transfusion-dependent patients with beta-thalassemia were presented last month at the American Society of Hematology (ASH) Annual Meeting and were included in the Best of ASH session at that meeting.
Luspatercept is being evaluated in ongoing studies in patients with non-transfusion-dependent beta-thalassemia (the Phase 2 BEYOND trial) as well as in patients with lower-risk myelodysplastic syndromes (MDS) (the Phase 2 PACE-MDS trial and the Phase 3 MEDALIST and COMMANDS trials) and in a Phase 2 trial in patients with myelofibrosis.
Luspatercept is an investigational therapy that is not approved for any use in any country. Celgene and Acceleron are planning submission of marketing applications for luspatercept in the United States and Europe in the first half of 2019.