Luspatercept thalassemia

First disclosure from Phase 3 COMMANDS study, selected for both ASCO’s official press program and EHA’s plenary session, highlights potential of Reblozyl (luspatercept-aamt) as first-line treatment of anemia in very low- to intermediate-risk myelodysplastic syndromes First presentation of results from primary analysis of TRANSCEND CLL 004 demonstrates benefit of Breyanzi (lisocabtagene maraleucel) in relapsed or refractory chronic lymphocytic leukemia or small lymphocytic lymphoma New TRIDENT-1 data reinforce potential of precision medicine repotrectinib in patients with locally advanced or metastatic ROS1 fusion-positive non-small cell lung cancer (NSCLC), including those with central nervous system metastases Four-year data in NSCLC from CheckMate -9LA demonstrate durable long-term survival benefit for Opdivo (nivolumab) + Yervoy (ipilimumab) + platinum-based chemotherapy and three-year data from CheckMate -816 continue to demonstrate long-term clinical benefits of Opdivo + chemotherapy in patients with earlier stages of NSCLC Bristol Myers Squibb to Host Virtual Investor Event on Tuesday, June 6, 2023, to Discuss ASCO Highlights PRINCETON, N.J.--(BUSINESS WIRE)-- “We look forward to sharing our research during ASCO, EHA and ICML, demonstrating the diversity of our assets, cutting-edge pipeline, and science-driven strategy focused on shaping the next generation of cancer care,” said Key data being presented by Bristol Myers Squibb at ASCO, EHA and ICML 2023 include: Hema...

Citation Maciej W. Garbowski , Manuel Ugidos , Alberto Risueño , Rajasekhar N.V.S. Suragani , Jeevan K. Shetty , Sadanand Vodala , Anjan Thakurta , Martin Schwickart , John B. Porter; Luspatercept Redistributes Body Iron to the Liver in Transfusion-Dependent-Thalassemia (TDT) during Erythropoietic Response. Blood 2021; 138 (Supplement 1): 761. doi: Download citation file: • • • • • • • • • Introduction: Luspatercept inhibits select ligands of the TGF-β superfamily implicated in thalassemic erythropoiesis and promotes late-stage erythroid maturation (Suragani RN, et al. Nat Med 2014;20:408-414). This leads to greater red blood cell (RBC) output from thalassemic marrow and reduces transfusion dependence in TDT (Cappellini MD, et al. N Engl J Med 2020;382:1219-1231). The underlying mechanisms for this clinical outcome are not well understood in a syndrome involving significant iron overload and cyclical stimulation of erythropoiesis between transfusions. Here we report novel physiological and clinical insights from a BELIEVE trial (NCT02604433) biomarker analysis, which demonstrate that hepcidin and erythropoietic changes in TDT lead to iron redistribution from macrophages to hepatocytes on luspatercept. Methods: 336 TDT patients ≥ 18 years of age who took part in the BELIEVE study, a multicenter, randomized, double-blind, placebo-controlled phase 3 trial (Cappellini et al. 2020), were randomized 2:1 to receive luspatercept or placebo subcutaneously every 21 days for 48 weeks...

Citation Antonio Piga , Silverio Perrotta , Maria Rita Gamberini , Ersi Voskaridou , Angela Melpignano , Aldo Filosa , Vincenzo Caruso , Antonello Pietrangelo , Filomena Longo , Immacolata Tartaglione , Caterina Borgna-Pignatti , Xiaosha Zhang , Abderrahmane Laadem , Matthew L. Sherman , Kenneth M. Attie; Luspatercept improves hemoglobin levels and blood transfusion requirements in a study of patients with β-thalassemia. Blood 2019; 133 (12): 1279–1289. doi: Download citation file: • • • • • • • • • β-thalassemia is a hereditary disorder with limited approved treatment options; patients experience anemia and its complications, including iron overload. The study aim was to determine whether luspatercept could improve anemia and disease complications in patients with β-thalassemia. This open-label, nonrandomized, uncontrolled study consisted of a 24-week dose-finding and expansion stage (initial stage) and a 5-year extension stage, currently ongoing. Sixty-four patients were enrolled; 33 were non–transfusion dependent (mean hemoglobin, <10.0 g/dL; <4 red blood cell [RBC] units transfused per 8 weeks), and 31 were transfusion dependent (≥4 RBC units per 8 weeks). Patients received 0.2 to 1.25 mg/kg luspatercept subcutaneously every 21 days for ≥5 cycles (dose-finding stage) and 0.8 to 1.25 mg/kg (expansion cohort and 5-year extension). The primary end point was erythroid response, defined as hemoglobin increase of ≥1.5 g/dL from baseline for ≥14 consecutive days (without RBC ...