Acceleron Announces New Luspatercept Phase 2 Clinical Results at the 20th Congress of the European Hematology Association

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Acceleron Pharma Inc.
XLRN
, a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of novel therapeutic candidates that regulate cellular growth and repair, today announced that new preliminary results from the ongoing phase 2 clinical trials of luspatercept in patients with lower risk myelodysplastic syndromes (MDS) and in patients with beta-thalassemia were highlighted in oral presentations at the 20th Congress of the European Hematology Association in Vienna, Austria. Acceleron and its collaboration partner, Celgene Corporation, are jointly developing luspatercept. "We have previously shown that with just 3 months of treatment, luspatercept can increase hemoglobin levels and achieve transfusion independence in lower risk MDS patients," said Matthew Sherman, Executive Vice President and Chief Medical Officer of Acceleron. "These new results show that longer-term treatment with luspatercept can produce sustained hemoglobin increases and transfusion independence in some patients now exceeding 6 months. Additionally, in our beta-thalassemia study, luspatercept has demonstrated positive effects on multiple complications of the disease by increasing hemoglobin levels, reducing transfusion burden and reducing iron overload in the majority of patients." MDS Phase 2 Study: For the first time, data from the 12-month extension study of low and intermediate-1 risk myelodysplastic syndromes patients were presented. The preliminary results show encouraging longer-term, durable responses with luspatercept. Patients who completed the initial 3-month study may have been eligible to enroll in the 12-month extension study. Twenty-two patients were evaluable for efficacy analyses; 9 low transfusion burden patients and 13 high transfusion burden patients. For the low transfusion burden patients, the mean hemoglobin increase at one month was approximately 2 g/dL, increased to between 2.5 and 3.0 g/dL and was maintained for the 6-month period for which data are available. For transfused patients, 43% achieved transfusion independence with several patients maintaining this transfusion independence for more than 6 months with the longest ongoing transfusion independent patient at nearly 8 months. All of these patients remain on study. Beta-Thalassemia Phase 2 Study: Data were presented for both non-transfusion dependent (NTD) and transfusion dependent (TD) beta-thalassemia patients. The preliminary results demonstrate that luspatercept, by addressing the underlying ineffective erythropoiesis, can increase hemoglobin levels, reduce transfusion burden and reduce liver iron concentrations in iron overloaded patients. Non-transfusion dependent patients For patients who received luspatercept 0.8–1.25 mg/kg, 38% had a mean hemoglobin increase ≥ 1.5 g/dL maintained for at least 9 weeks. For 8 of the 12 iron overloaded NTD patients, luspatercept reduced liver iron concentration by at least 1 and up to 4.6 mg/g dry weight over the 16 week period. Transfusion dependent patients All ten evaluable patients who received luspatercept 0.6–1.25 mg/kg had more than a 40% reduction in transfusion burden. For 2 of the 3 iron overloaded TD patients, luspatercept reduced liver iron concentration by 1.96 and 4.7 mg/g dry weight, respectively. The slides from these two oral presentations will be available on Acceleron's website (www.acceleronpharma.com) under the Publications tab.
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