Bristol Myers Squibb announced that its prominent medicine Reblozyl, in conjunction with another treatment, did not achieve the primary objective of a late-stage study aimed at treating anemia resulting from a bone marrow cancer.
The trial failed to provide statistically meaningful outcomes in facilitating myelofibrosis patients’ independence from transfusions of red blood cells during any 12-week interval within the first 24 weeks of therapy.
The advanced study in myelofibrosis patients evaluated Reblozyl alongside a Janus kinase inhibitor, a category of medications that functions by inhibiting the immune system. Myelofibrosis is a malignancy that induces fibrosis in the bone marrow, obstructing the synthesis of healthy blood cells.
Myelofibrosis (MF) is an uncommon hematological tumor, with a yearly rate of around 0.3 cases per 100,000 persons in the U.S. It is classified as a kind of blood cancer known as chronic myeloproliferative neoplasms (MPN), characterized by the unusual development and activity of bone marrow stem cells that generate blood cells. Myelofibrosis is essentially defined by the accumulation of fibrous scar tissue inside the bone marrow.
Numerous significant secondary measures demonstrated a clinically relevant edge for Reblozyl, including an increased number of individuals attaining a minimum decrease of 50% in RBC transfusion burden, as well as a greater number of people attaining a hemoglobin (Hb) level elevation of at least 1 g/dL whilst staying transfusion independent for a minimum of 12 consecutive weeks.
Commonly observed treatment-related side events aligned with the established safety profile of Reblozyl earlier reported throughout indications.
The firm is optimistic about the clinically significant outcomes of the research and will consult with the FDA and EMA about the filing of marketing applications.
Anne Kerber, who leads development for hematology, oncology, and cell therapy at BMS, emphasized the encouraging results seen with Reblozyl in treating anemia associated with myelofibrosis. She noted that patients with this condition frequently become increasingly dependent on transfusions over time, making the observed clinical improvements especially significant. Kerber said the findings – highlighting reductions in transfusion needs and increased hemoglobin levels – demonstrate Reblozyl’s potential to fill a major treatment gap for patients with limited therapeutic options.
John Mascarenhas, M.D., who teaches medicine at the Icahn School of Medicine at Mount Sinai, stated that anemia continues to pose a serious challenge in managing myelofibrosis. He explained that many patients remain reliant on red blood cell transfusions or therapies that offer limited effectiveness and may even worsen anemia linked to the disease. Mascarenhas emphasized that treating myelofibrosis patients with anemia is particularly difficult, and the latest findings suggest that Reblozyl could offer meaningful improvement for addressing disease-related anemia.
Reblozyl is the standard treatment for primary anemia in adult individuals with very low- to normal-risk myelodysplastic syndromes (MDS) who may need red blood cell transfusions. It is also indicated for those with the same level of MDS with ring sideroblasts (MDS-RS) who have anemia and are unresponsive to erythropoiesis stimulating agents and require two or more RBC units within eight weeks.
