Bristol Myers Squibb Announces Positive Phase II Results For Reblozyl In Alpha-Thalassemia

Bristol Myers Squibb announced positive top-line results from an ongoing ex-US Phase II registrational study (NCT05664737) evaluating Reblozyl® (luspatercept-aamt) for the treatment of anaemia in adults with alpha (α)-thalassemia. The trial compared Reblozyl to placebo across both non-transfusion-dependent (NTD) and transfusion-dependent (TD) patient populations.

Both cohorts achieved their respective primary endpoints. In non-transfusion-dependent patients, Reblozyl demonstrated a statistically significant and clinically meaningful increase in haemoglobin levels. In transfusion-dependent patients, the therapy significantly reduced red blood cell transfusion burden compared with placebo. All key secondary endpoints were met, and the safety profile remained consistent with Reblozyl's previous experience in thalassemia.

“These positive data further support the potential of Reblozyl for patients around the world,” said Cristian Massacesi, Executive Vice President, Chief Medical Officer and Head of Development, Bristol Myers Squibb. “This is the first and only registrational Phase 2 trial specifically designed to address the needs of patients, especially in China, with alpha-Thalassemia, a lifelong disease with limited treatment options and the potential for serious long‑term complications.”

The company plans to present the full dataset at an upcoming medical congress and will engage with the Centre for Drug Evaluation in China regarding the results, supporting potential regulatory discussions in the region.