Despite the failure, BMS remains “encouraged” by Reblozyl’s clinical activity in myelofibrosis-associated anemia and will approach regulators to discuss potential submissions for this indication, for which few treatment options exist.
Bristol Myers Squibb’s top-selling anemia drug Reblozyl, first approved in 2019, failed a late stage trial in patients with myelofibrosis, unable to significantly ease the need for red blood cell transfusions.
The pharma did not provide specific data in its news release on Friday, revealing only that Reblozyl failed to meet the Phase III INDEPENDENCE study’s primary endpoint—independence from red blood cell transfusions for 12 consecutive weeks starting in the first 24 weeks of treatment.
Writing to investors on Friday morning, analysts at BMO Capital Markets said that Friday’s readout “highlights lack of effective treatments in myelofibrosis anemia.”
Despite missing statistical significance, BMS insisted that patients treated with Reblozyl did have a “numerical and clinically meaningful improvement” in transfusion independence. The pharma also noted that many “important” secondary outcomes of INDEPENDENCE showed “clinically meaningful benefit favoring Reblozyl,” including transfusion burden and increases in hemoglobin levels while remaining transfusion-independent for at least 12 consecutive weeks.
BMS did not say whether any of these secondary endpoints reached statistical significance. “No quantitative measures were provided for transfusion independence rates, limiting clarity on the true impact of [Reblozyl] treatment on both anemia improvement via serological measures and functional improvement in transfusion independence,” the BMO analysts wrote on Friday.
Still, Anne Kerber, head of Hematology, Oncology, and Cell Therapy development at BMS, called Friday’s findings “promising,” focusing on the “clinically relevant improvement” in patients treated with Reblozyl. The “totality of these results,” Kerber continued, “support the potential to address an unmet need in patients who have few treatment options.”
The company said that safety findings were consistent with previous trials for the drug.
BMS plans to approach the FDA and European Medicines Agency to discuss submitting marketing applications for Reblozyl in myelofibrosis anemia.
Designed to be delivered via a subcutaneous injection, Reblozyl is a recombinant fusion protein that works by improving the number and quality of mature red blood cells. The drug was approved by the FDA in 2019 for anemia in adult patients with beta thalassemia who need regular transfusions. Reblozyl has since been approved to address anemia in those with very low- to intermediate-risk myelodysplastic syndromes or myeloproliferative neoplasms.
Reblozyl is one of BMS’ top-selling assets. In 2024, the drug surged 76% year-on-year to bring in $1.77 billion in sales. Reblozyl has maintained its strong market performance into this year, bringing in $478 million in the first quarter of 2025, representing a 35% growth from the same period last year.
