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Celgene and Acceleron's blood disorder drug smashes endpoints at Phase 3

Published on 11/07/18 at 10:53am

Celgene and Acceleron have lifted the curtain on new Phase 3 data for their erythroid maturation agent (EMA) luspatercept, confirming that it demonstrated a “highly statistically significant improvement” in erythroid response in combination with best supportive care in the treatment of transfusion-dependent beta-thalassemia, meeting the trial’s primary endpoint.

This improvement was defined as a reduction of at least 33% from baseline in red blood cell (RBC) transfusion burden with a reduction of at least two units during the period of 12 consecutive weeks from week 13 to week 24 of treatment compared to placebo.

All secondary endpoints of the trial were also met, with luspatercept continuing to show at least a 50% reduction in RBC transfusion burden from baseline from weeks 13 to 24 and weeks 37 to 48,a s well as a mean change from weeks 13 to 24.

Beta-thalassemia is a disorder of the blood which causes a reduction in haemoglobin, and can lead to a lack of oxygen around the body.

“For decades, the management of beta-thalassemia in adults has been limited to transfusions and iron chelation. Reduction of transfusion burden represents an important step forward for patients with this rare and debilitating blood disease,” said Dr Jay Backstrom, Chief Medical Officer for Celgene. “We thank the patients, as well as their families and physicians, for their participation in the BELIEVE study.”

Habib Dable, President and Chief Executive Officer of Acceleron, added: “The BELIEVE study marks the second positive phase III study for luspatercept and underscores the potential of this erythroid maturation agent to impact a range of diseases associated with chronic anemia. We continue to explore luspatercept across our broader development programs, including non-transfusion dependent beta-thalassemia in the ongoing BEYOND study.”

Luspatercept is not currently authorised for use in any indication, but the two companies now plan to submit regulatory applications in the US and Europe in the first half of 2019.

Matt Fellows

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