Sotatercept is being jointly developed with partner Celgene for the treatment of diseases in which anemia is common. It is currently being evaluated in three ongoing Phase 2 investigator-sponsored trials in myelofibrosis (MF), a rare bone marrow disorder, as well as multiple myeloma and Diamond-Blackfan Anemia.
Sotatercept works by targeting molecules in the TGF-beta superfamily to increase hemoglobin levels and boost red blood cells. Preliminary results from the Phase 2 study in MF were presented at the 2016 Annual Meeting of the American Society of Hematology (ASH), and demonstrated that treatment with sotatercept can increase hemoglobin and achieve transfusion independence. The investigator-sponsored trial was designed and conducted at MD Anderson and included patients with all three forms of myelofibrosis: primary MF, post polycythemia vera MF, and post essential thrombocythemia MF. All patients were either red blood cell transfusion dependent or had hemoglobin levels less than 10 over a baseline period of at least 12 weeks prior to enrollment in the study. Overall, 5 of 14 patients, or 36% of the evaluable patients, responded to sotatercept. This included patients who are transfusion-dependent or non-transfusion-dependent at baseline.
Myelofibrosis (MF) is a rare bone marrow disorder that disrupts the body’s normal production of red blood cells. Some MF patients see their disease progress to a more serious form of leukemia, and many will develop anemia over the course of their lifetime. There are no approved therapies for anemia in MF patients and currently available therapies only address symptoms. Patients are treated with red blood cell transfusions, erythropoiesis-stimulating agents (ESAs), and immunomodulatory drugs—all of which produce suboptimal results and also carry significant risks.
Acceleron and Celgene are planning to initiate a Phase 2 luspatercept trial in myelofibrosis.