(RTTNews) – Bristol Myers Squibb (BMY) and Acceleron Pharma Inc. (XLRN) announced Friday the first data from the Phase 2 BEYOND study evaluating Reblozyl (luspatercept-aamt), a first-in-class erythroid maturation agent, plus best supportive care in adult patients with non-transfusion dependent or NTD beta thalassemia.
The results showed that treatment with Reblozyl plus best supportive care improved anemia in 77% of patients compared to placebo.
The results were presented at the European Hematology Association or EHA 2021 Virtual Congress as part of its Presidential Symposium.
BEYOND is a Phase 2, randomized, double-blind, placebo-controlled multi-center study to determine the efficacy and safety of Reblozyl versus placebo in adults with NTD beta thalassemia.
Beta thalassemia is an inherited blood disorder caused by a genetic defect in hemoglobin. NTD beta thalassemia is a term used to describe patients who do not require lifelong regular red blood cell transfusions for survival, although they may require occasional or even frequent transfusions, usually for defined periods of time.
Reblozyl is the first and only erythroid maturation agent approved in the European Union, United States and Canada to address anemia-associated beta thalassemia and lower-risk myelodysplastic syndromes.
The companies noted that 77.7% of patients treated with Reblozyl in the trial achieved a hemoglobin increase compared to 0% of patients in the placebo arm. Changes in patient-reported outcomes also correlated with increases in hemoglobin.
Ali Taher, of American University of Beirut and BEYOND study investigator, said, “Patients with non-transfusion dependent beta thalassemia experience chronic anemia and iron overload, which may lead to a range of clinical complications, and treatment options are greatly needed. Results from the BEYOND study show the clinical potential of luspatercept to sustain the elevation of hemoglobin levels in a majority of patients regardless of their baseline hemoglobin status, and improvements were noted in quality of life outcomes in adults with non-transfusion dependent beta thalassemia.”