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Acceleron to Present Luspatercept Clinical Trials at the 20th Congress of the European Hematology Association

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Acceleron Pharma Inc. (NASDAQ: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 data from two ongoing phase 2 clinical trials of luspatercept in patients with beta-thalassemia and myelodysplastic syndromes (MDS) will be given as oral presentations at the 20th Congress of the European Hematology Association in Vienna, Austria.

Beta-thalassemia oral presentation

Title:

Luspatercept increases hemoglobin and decreases transfusion burden and liver iron concentration in adults with beta-thalassemia: preliminary results from a phase 2 study (abstract S136)

Presenter:

Antonio Piga, M.D., Ph.D., San Luigi Gonzaga University Hospital, Torino, Italy

Session:

Red cells: novel clinical aspects

Date:

Friday, June 12

Time:

11:30 – 11:45 AM CEST (Room: Strauss 1)

MDS oral presentation

Title:

Luspatercept increases hemoglobin and reduces transfusion burden in patients with low or int-1 MDS: preliminary results from the phase 2 PACE-MDS study (abstract S509)

Presenter:

Uwe Platzbecker, M.D., University Hospital in Dresden, Germany

Session:

MDS Clinical

Date:

Saturday, June 13

Time:

4:30 – 4:45 PM CEST (Room: C1)

The presentation slides will be available on Acceleron’s website (www.acceleronpharma.com) under the Publications section.

About Luspatercept

Luspatercept is a modified activin receptor type IIB fusion protein that acts as a ligand trap for members of the Transforming Growth Factor-Beta (TGF-beta) superfamily involved in the late stages of erythropoiesis (red blood cell production). Luspatercept regulates late-stage erythrocyte (red blood cell) precursor cell differentiation and maturation. This mechanism of action is distinct from that of erythropoietin (EPO), which stimulates the proliferation of early-stage erythrocyte precursor cells. Acceleron and Celgene Corporation are jointly developing luspatercept as part of a global collaboration. Luspatercept is currently in phase 2 clinical trials in patients with beta-thalassemia and in patients with myelodysplastic syndromes. For more information, please visit www.clinicaltrials.gov.

About Beta-thalassemia

Beta-thalassemia is an inherited disease involving mutations in the beta globin gene leading to deficient hemoglobin levels and defective red blood cell production in the bone marrow known as ineffective erythropoiesis. Beyond the severe anemia, many patients also suffer from multiple organ dysfunction, largely due to excess iron deposits, known as “iron overload,” resulting from the ineffective erythropoiesis as well as the repeated red blood cell (RBC) transfusions to address the anemia. Iron overload can lead to heart failure, liver fibrosis, and diabetes, among other consequences. Current clinical management for beta-thalassemia includes RBC transfusions and iron chelation therapy, which is associated with toxicities. There are no drugs approved to treat beta-thalassemia leaving healthcare providers with few options for patients.

About Myelodysplastic Syndromes

MDS are a heterogeneous group of hematologic malignancies of the bone marrow commonly leading to severe and chronic anemia due to ineffective erythropoiesis. The National Cancer Institute estimates that more than 10,000 people are diagnosed with MDS in the United States each year. Patients with MDS often have a hypercellular bone marrow with various dysplastic changes of the cells that are also seen in peripheral blood, resulting in cytopenias (low blood cell counts) and an increased risk of progression to acute myeloid leukemia. Nearly all MDS patients suffer from anemia. The anemia in MDS is often characterized by high endogenous levels of EPO driving an abundance of early stage red blood cell precursors and an inability of these precursor cells to properly differentiate into healthy, functional red blood cells. Many patients are therefore unresponsive to the administration of erythropoietin to correct the resulting anemia and instead require red blood cell transfusions, which can increase the risk of infection and iron-overload related toxicities.

About Acceleron

Acceleron is a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of novel therapeutic candidates that regulate cellular growth and repair. The company is a leader in understanding the biology of the TGF-beta protein superfamily, a large and diverse group of molecules that are key regulators in the growth and repair of tissues throughout the human body, and in targeting these pathways to develop important new medicines. Acceleron has built a highly productive R&D platform that has generated innovative clinical and preclinical therapeutic candidates with novel mechanisms of action. These therapeutic candidates have the potential to significantly improve clinical outcomes for patients with cancer and rare diseases.

For more information, please visit www.acceleronpharma.com.

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