July 18, 2025--PRINCETON, N.J.--(BUSINESS WIRE)-- Bristol Myers Squibb (NYSE: BMY) today announced the Phase 3 INDEPENDENCE trial evaluating Reblozyl® (luspatercept-aamt) with concomitant janus kinase inhibitor (JAKi) therapy in adult patients with myelofibrosis-associated anemia receiving red blood cell (RBC) transfusions did not meet its primary endpoint of RBC transfusion independence during any consecutive 12-week period, starting within the first 24 weeks of treatment, compared to placebo (p=0.0674). Patients saw a numerical and clinically meaningful improvement in RBC transfusion independence favoring Reblozyl, in line with previous results from the Phase 2 trial (NCT03194542).
Several important secondary measures also showed a clinically meaningful benefit favoring Reblozyl, which included a higher number of patients who achieved at least a 50% reduction (and by at least 4 RBC units) in RBC transfusion burden, as well as a higher number of patients achieving a hemoglobin (Hb) level increase by at least 1 g/dL while remaining transfusion independent for at least 12 consecutive weeks.
Frequently observed treatment emergent adverse events were consistent with the known safety profile of Reblozyl previously reported across indications.
The company is encouraged by the clinically meaningful results of the study and will engage with the FDA and EMA to discuss the submission of marketing applications.
“It is promising to see that Reblozyl led to clinically relevant improvement of anemia for patients with myelofibrosis, where patients often become increasingly transfusion dependent over time,” said Anne Kerber, Senior Vice President, Head of Development, Hematology, Oncology, and Cell Therapy for Bristol Myers Squibb. “We remain confident in the ability of Reblozyl to improve outcomes for patients with myelofibrosis-associated anemia and believe the totality of these results, including meaningful improvements in transfusion burden and hemoglobin levels, support the potential to address an unmet need in patients who have few treatment options.”
“Anemia remains a significant challenge in the treatment of myelofibrosis, with many patients still dependent on red blood cell transfusions or suboptimal treatment approaches that can sometimes worsen anemia associated with the disease,” said John Mascarenhas, MD, Professor of Medicine at the Icahn School of Medicine at Mount Sinai and Director of the Center of Excellence for Blood Cancers and Myeloid Disorders at The Tisch Cancer Institute. “Patients with myelofibrosis and anemia are difficult to treat, and these results show that Reblozyl can have an important impact on anemia associated with the disease.”
Reblozyl is a standard of care for the first-line treatment of anemia without previous erythropoiesis stimulating agent use (ESA-naïve) in adult patients with very low- to intermediate-risk myelodysplastic syndromes (MDS) who may require RBC transfusions, treatment of anemia failing an erythropoiesis stimulating agent and requiring 2 or more RBC units over 8 weeks in adult patients with very low- to intermediate-risk myelodysplastic syndromes with ring sideroblasts (MDS-RS) or with myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T), and treatment of anemia in adult patients with beta thalassemia who require RBC transfusions.
Bristol Myers Squibb thanks the patients, investigators and clinical trial sites who participated in the INDEPENDENCE clinical trial.
About the Phase 3 INDEPENDENCE Trial
INDEPENDENCE (NCT04717414) is a Phase 3, double-blind, randomized study to compare the efficacy and safety of luspatercept vs placebo in patients with myeloproliferative neoplasms (MPN)-associated myelofibrosis (MF) on associated JAK2 inhibitor therapy and who require red blood cell (RBC) transfusions. The primary outcome measure is the proportion of patients who became RBC-transfusion-free over any consecutive 12-week period starting within the first 24 weeks. The key secondary endpoint was the proportion of patients who became RBC transfusion independent over any consecutive 16-week period, starting within the first 24 weeks. Additional secondary endpoints measured increase in hemoglobin (Hb) levels, and at least a 50% reduction in RBC transfusion burden.
About Myelofibrosis
Myelofibrosis (MF) is a rare type of blood cancer, with an annual incidence of approximately 0.3 cases per 100,000 individuals in the U.S. It is one of a group of blood cancers called chronic myeloproliferative neoplasms (MPN), in which bone marrow stem cells that produce blood cells develop and function abnormally. MF is characterized by the buildup of scar tissue, called fibrosis, in the bone marrow. As scar tissue increases, the bone marrow cannot make enough healthy blood cells, which can lead to anemia, a condition characterized by not having enough healthy red blood cells to carry oxygen to the body's tissues.
About Reblozyl
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:
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anemia in adult patients with beta thalassemia who require regular red blood cell (RBC) transfusions, and
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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.
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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.
Bristol Myers Squibb: Creating a Better Future for People with Cancer
Bristol Myers Squibb is inspired by a single vision — transforming patients’ lives through science. The goal of the company’s cancer research is to deliver medicines that offer each patient a better, healthier life and to make cure a possibility. Building on a legacy across a broad range of cancers that have changed survival expectations for many, Bristol Myers Squibb researchers are exploring new frontiers in personalized medicine and, through innovative digital platforms, are turning data into insights that sharpen their focus. Deep understanding of causal human biology, innovative capabilities and differentiated research platforms uniquely position the company to approach cancer from every angle.
Cancer can have a relentless grasp on many parts of a patient’s life. As a leader in cancer care, Bristol Myers Squibb is working to empower all people with cancer to have a better future.
About Bristol Myers Squibb: Transforming Patients' Lives Through Science
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.