Luspatercept: Additional Phase II data

Acceleron reported data from 59 evaluable patients with beta thalassemia in an open-label, international Phase II trial evaluating subcutaneous luspatercept given every 3 weeks for up to 5 cycles. In 28 transfusion-dependent patients, 22 achieved an erythroid response, the primary endpoint. For transfusion-dependent patients, erythroid response was defined as a >=20% reduction in red blood cell transfusion burden over 12 weeks of treatment compared to the 12 weeks prior to treatment. Additionally, 21 and 16 patients had >=33% and >=50% reductions in red blood cell transfusion burden, respectively. Of 8 transfusion dependent patients with >=5 mg/g dry weight LIC at baseline, 4 had a >=2 mg/g dry weight reduction. All 14 transfusion dependent patients with <5 mg/g dry weight LIC at baseline maintained an LIC of <5 mg/g dry weight. ...