SHERIDAN, WYOMING – July 21, 2025 – Bristol Myers Squibb’s (BMS) leading anemia therapy, Reblozyl, has failed to meet its primary endpoint in a pivotal Phase III trial for myelofibrosis-associated anemia, highlighting the ongoing challenges in addressing this difficult-to-treat condition.
The INDEPENDENCE study, designed to evaluate Reblozyl's ability to reduce patients’ dependence on red blood cell transfusions, did not achieve statistical significance on its primary objective—independence from transfusions for 12 consecutive weeks within the first 24 weeks of treatment. The company disclosed the outcome in a news release last Friday but withheld detailed data on transfusion independence rates.
Continued Confidence Despite Missed Endpoint
Despite the trial miss, BMS expressed confidence in Reblozyl’s clinical potential for myelofibrosis-related anemia. The company emphasized the positive clinical signals observed in the study, even though they did not meet statistical thresholds.
“We observed a numerical and clinically meaningful improvement in transfusion independence among patients treated with Reblozyl,” BMS stated in its announcement. The company also reported benefits in key secondary outcomes, such as reduced transfusion burden and improved hemoglobin levels in patients who remained transfusion-independent for at least 12 weeks.
BMS noted that the safety profile of Reblozyl in the INDEPENDENCE study aligned with findings from previous trials, maintaining its established risk-benefit balance.
Analysts Underscore Market Challenges
Market analysts viewed the trial results as a reflection of the persistent therapeutic gaps in myelofibrosis-related anemia. BMO Capital Markets commented, “Friday’s readout highlights lack of effective treatments in myelofibrosis anemia.”
However, they also flagged the absence of detailed quantitative data in BMS’ disclosure, stating, “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.”
Strategic Next Steps with Regulatory Agencies
Looking ahead, BMS intends to engage regulatory bodies in both the United States and Europe to discuss potential marketing applications for Reblozyl in this indication. Anne Kerber, Head of Hematology, Oncology, and Cell Therapy Development at BMS, reaffirmed the company’s commitment, saying, “The totality of these results support the potential to address an unmet need in patients who have few treatment options.”
Reblozyl’s Expanding Commercial Footprint
Since its initial FDA approval in 2019 for transfusion-dependent anemia in adults with beta thalassemia, Reblozyl has expanded its indications to include anemia in patients with very low- to intermediate-risk myelodysplastic syndromes or myeloproliferative neoplasms. The drug, administered via subcutaneous injection, enhances the production and quality of mature red blood cells as a recombinant fusion protein.
Reblozyl has emerged as a key growth driver for BMS. In 2024, its global sales surged 76% year-over-year, reaching $1.77 billion. The momentum continued into 2025, with first-quarter revenues climbing 35% to $478 million compared to the same period last year.
Addressing a High-Unmet Need Market
The mixed trial outcome underscores both the complexity of treating myelofibrosis-associated anemia and the high unmet need in this therapeutic area. With few effective options available, BMS’ continued development efforts signal its commitment to expanding treatment possibilities for patients facing this serious condition.
Learn more at www.bms.com