Bristol-Myers Squibb Company (NYSE:BMY) and Acceleron Pharma Inc. (NASDAQ:XLRN) today announced that the New England Journal of Medicine (NEJM) has published results from MEDALIST, the pivotal phase 3 study evaluating the use of Reblozyl® (luspatercept-aamt) to treat anemia in patients with very low- to intermediate-risk myelodysplastic syndromes (MDS) who have ring sideroblasts (RS+) and require red blood cell transfusions, and who had failed, were intolerant to, or ineligible for/unlikely to respond to treatment with erythropoiesis-stimulating agents (ESAs).1
“This NEJM publication recognizes the importance and robustness of the MEDALIST study, as well as the impact and potential clinical benefit of Reblozyl in MDS patients who have ring sideroblasts,” said Diane McDowell, M.D., Vice President, Hematology Global Medical Affairs, Bristol-Myers Squibb. “As the first erythroid maturation agent, Reblozyl holds the potential to help these patients address the underlying cause of their disease-related chronic anemia.”
MEDALIST achieved a statistically significant improvement in the primary endpoint of red blood cell transfusion independence (RBC-TI) for 8 or more weeks during the first 24 weeks of the study. The study also met the key secondary endpoint of RBC-TI for 12 or more weeks during the first 24 or 48 weeks of the study, as well as the additional secondary endpoint of hematologic improvement-erythroid (HI-E) for 8 or more weeks as assessed by International Working Group 2006 criteria.1
The most common treatment-emergent adverse events (TEAEs) of any grade in greater than 10% of patients in either the treatment or placebo arm were fatigue, diarrhea, asthenia, nausea, dizziness, and back pain. TEAEs of Grade 3 or 4 were reported in 42.5% (65/153) of patients receiving luspatercept-aamt and 44.7% (34/76) of patients receiving placebo. Progression to acute myeloid leukemia (AML) occurred in three patients (2.0%) receiving luspatercept-aamt and one patient (1.3%) receiving placebo. Five patients receiving luspatercept-aamt (3.3%) and four patients receiving placebo (5.3%) experienced one or more TEAE that resulted in death.1
Results from MEDALIST were initially presented during the Plenary Scientific Session at the 60th American Society of Hematology (ASH) Annual Meeting and Exposition in December 2018. Longer-term analyses from MEDALIST were recently presented at the 61st ASH Annual Meeting in December 2019.
“It is truly an honor to see work that began in Acceleron laboratories more than a decade ago and advanced successfully through a collaboration with Bristol-Myers Squibb now highlighted in the New England Journal of Medicine,” said Habib Dable, President and Chief Executive Officer of Acceleron. “Publication of the MEDALIST trial results in a journal of such eminence will certainly help to raise awareness among physicians of a potential new therapeutic option for treating anemia in certain patients with MDS.”
The U.S. Food and Drug Administration (FDA) is evaluating Reblozyl for the treatment of anemia in adults with very low- to intermediate-risk MDS who have ring sideroblasts and require red blood cell transfusions, and has set a Prescription Drug User Fee Act (PDUFA), or target action, date of April 4, 2020 for this indication. In Europe, Bristol-Myers Squibb’s Marketing Authorization Application for the treatment of anemia in adults with beta thalassemia and MDS is currently under review.
In November 2019, Reblozyl was granted approval by the FDA to treat anemia in adult patients with beta thalassemia who require regular red blood cell transfusions. Reblozyl is not indicated for use as a substitute for red blood cell transfusions in patients who require immediate correction of anemia.
Reblozyl is not approved to treat MDS in any geography.