Luspatercept-AAMT

Bristol Myers Squibb (NYSE: BMY) today announced positive top-line results from the ongoing, ex-US, Phase 2 registrational study (NCT05664737) evaluating Reblozyl® (luspatercept-aamt) versus placebo for anemia in adults with Alpha (α)-Thalassemia. The non‑transfusion‑dependent (NTD) and transfusion‑dependent (TD) cohorts of the study met their respective primary endpoints, with Reblozyl demonstrating a statistically significant and clinically meaningful increase in hemoglobin levels in NTD patients with α‑thalassemia, and a statistically significant and clinically meaningful decrease in red blood cell (RBC) transfusion burden in TD patients with α‑thalassemia. The study also met all key secondary endpoints. Safety findings were consistent with the known pro Reblozyl in thalassemia. “These positive data further support the potential of Reblozyl for patients around the world,” said Cristian Massacesi, Executive Vice President, Chief Medical Officer and Head of Development, Bristol Myers Squibb. “This is the first and only registrational Phase 2 trial specifically designed to address the needs of patients, especially in China, with alpha-Thalassemia, a lifelong disease with limited treatment options and the potential for serious long‑term complications.” The data will be presented at an upcoming medical congress and will be discussed with the Center for Drug Evaluation in China. The Phase 2 trial is evaluating the efficacy and safety of luspatercept plus best supportive care versus placebo for anemia in adults and adolescents with α-thalassemia, including both red blood cell (RBC) transfusion-dependent (TD) and non-transfusion-dependent (NTD) cohorts. The primary endpoint of the NTD cohort measures an increase from baseline of ≥ 1 grams (g)/deciliter (dL) in mean hemoglobin (Hb) values over the continuous 12-week interval from Week 13 to Week 24 in the absence of RBC transfusion. The primary endpoint of the TD cohort measures ≥ 50% reduction from baseline in RBC transfusion burden with a reduction of at least 2 units during any continuous 12 weeks during Week 13-48 compared to 12-week interval immediately prior to date of first dose. The adolescent NTD and TD cohorts are ongoing. Reblozyl, a first-in-class therapeutic option, promotes late-stage red blood cell maturation in animal models. Reblozyl is being developed and commercialized through a global collaboration with Merck as of November 2021. Reblozyl is indicated in the U.S. for the treatment of: anemia in adult patients with beta thalassemia who require regular red blood cell (RBC) transfusions, and anemia without previous erythropoiesis stimulating agent use (ESA-naïve) in adult patients with very low- to intermediate-risk myelodysplastic syndromes (MDS) who may require regular red blood cell (RBC) transfusions. anemia failing an erythropoiesis stimulating agent and requiring 2 or more red blood cell (RBC) units over 8 weeks in adult patients with very low- to intermediate-risk myelodysplastic syndrome with ring sideroblasts (MDS-RS) or with myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T). Reblozyl is not indicated for use as a substitute for RBC transfusions in patients who require immediate correction of anemia. In the U.S., Reblozyl is not indicated for use in patients with non-transfusion-dependent beta thalassemia. At Bristol Myers Squibb, our mission is to discover, develop and deliver innovative medicines that help patients prevail over serious diseases. We are pursuing bold science to define what's possible for the future of medicine and the patients we serve. The content above comes from the network. if any infringement, please contact us to modify.