Celgene and Acceleron Report Positive Results for Beta-Thalassemia Drug; Sets Stage for Filing for Approval

July 10, 2018

Rare Daily Staff

Celgene and Acceleron Pharma reported that a late-stage clinical trial of their experimental drug luspatercept for beta-thalassemia, a rare blood disorder, showed it was an effective treatment.

Based on the success of the trial, the companies plan to submit applications for luspatercept for marketing approval in the United States and Europe in the first half of 2019.

Beta-thalassemia is caused by mutations in the beta-globin gene that leads to reduced or absent production of hemoglobin, the protein in red blood cells that carries oxygen to cells throughout the body. The disorder causes the destruction of red blood cells, which results in severe anemia and reduced oxygen transport to various tissues in the body.

Luspatercept is a first-in-class erythroid maturation agent that is believed to regulate late-stage red blood cell maturation. Acceleron and Celgene are jointly developing luspatercept as part of a global collaboration.

The study evaluated the efficacy and safety of luspatercept plus best supportive care versus placebo plus best supportive care in adults with transfusion-dependent beta-thalassemia. Luspatercept demonstrated a statistically significant improvement in the primary endpoint of erythroid response, which was defined as at least a 33 percent reduction from baseline in red blood cell transfusion burden with a reduction of at least 2 units during the protocol-defined period of 12 consecutive weeks, from week 13 to week 24, compared to placebo.

Luspatercept also met all key secondary endpoints of demonstrating statistically significant improvements in red blood cell transfusion burden from baseline of at least a 33 percent reduction during the period from week 37 to week 48, at least a 50 percent reduction during the period from week 13 to week 24, at least a 50 percent reduction during the period from week 37 to week 48, and a mean change in transfusion burden from week 13 to week 24.

Adverse events observed in the study were generally consistent with previously reported data.

“For decades, the management of beta-thalassemia in adults has been limited to transfusions and iron chelation,” said Jay Backstrom, chief medical officer for Celgene. “Reduction of transfusion burden represents an important step forward for patients with this rare and debilitating blood disease.”
July 10, 2018
Photo: Jay Backstrom, chief medical officer for Celgene

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