Bristol Myers’ Reblozyl Misses Main Goal in Phase 3 Myelofibrosis Trial

Bristol Myers Squibb said its Phase 3 INDEPENDENCE trial testing Reblozyl (luspatercept-aamt) in adults with myelofibrosis-associated anemia who require regular red-blood-cell transfusions failed to reach the study’s primary goal. The placebo-controlled study did not achieve the targeted proportion of patients who became transfusion-free for any consecutive 12-week period during the first 24 weeks of treatment (p = 0.0674). Despite missing statistical significance, the company reported "numerical and clinically meaningful" improvements favoring Reblozyl on several secondary measures, including reductions of at least 50 % in transfusion burden and increases of at least 1 g/dL in hemoglobin levels while maintaining transfusion independence. Adverse events were in line with the drug’s known safety profile. Bristol Myers said it remains encouraged by the overall data set and plans to discuss potential regulatory submissions with the U.S. Food and Drug Administration and the European Medicines Agency. Reblozyl, already approved for certain types of myelodysplastic syndromes and beta thalassemia, is being developed in partnership with Merck as a potential therapy for the hard-to-treat anemia seen in myelofibrosis.