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.
Read Also: Rocket Pharmaceuticals Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation
