Acceleron Initiates Phase 2 Study of ACE-536 to Treat Patients with Beta-Thalassemia

  Acceleron Initiates Phase 2 Study of ACE-536 to Treat Patients with

  Novel biologic has potential to treat rare blood disorder with substantial
                              unmet medical need

Business Wire

CAMBRIDGE, Mass. -- March 7, 2013

Acceleron Pharma, Inc., a biopharmaceutical company developing protein
therapeutics for cancer and orphan diseases, today announced the initiation of
a phase 2 study of its novel, investigational protein therapeutic, ACE-536, to
treat patients with beta-thalassemia, a genetic hematologic disorder causing
chronic and life-threatening anemia and serious complications affecting the
spleen, liver and heart. Patients and healthcare providers currently have
limited options for the treatment of beta-thalassemia. This is the second
ongoing Phase 2 trial for ACE-536, which is being developed by Acceleron as
part of a global collaboration with Celgene Corporation (NASDAQ: CELG).

“The unmet medical need in beta-thalassemia is enormous as treatment options
are limited mainly to blood transfusions and iron chelating agents,” said
Professor Antonio Piga, M.D., Ph.D., Director of Pediatrics at San Luigi
Gonzaga University Hospital in Torino, Italy and coordinating principal
investigator of the study. “ACE-536 could address this important unmet need
and I am excited to explore the potential benefits of this innovative
therapeutic in a phase 2 study.”

“We are excited to start our second phase 2 trial for ACE-536,” said Matthew
Sherman, M.D., Chief Medical Officer of Acceleron. “Both the preclinical and
clinical data assembled to date and our understanding of the protein’s novel
mechanism of action suggest that ACE-536 may become an important new treatment
option for those suffering with beta-thalassemia or other diseases that
negatively impact the body’s ability to produce a sufficient number of
functional red blood cells.”

About the Phase 2 Clinical Trial

The phase 2 clinical trial is designed as an open-label study to evaluate the
safety, tolerability and efficacy of ACE-536 in non-transfusion dependent
patients with beta-thalassemia and is being conducted in Europe. Efficacy
measures include increases in hemoglobin and red blood cell levels and
biomarkers of erythropoiesis, hemolysis, iron metabolism, and bone metabolism.
For additional information on this clinical trial, please visit, identifier NCT01749540.

About Beta-Thalassemia

Beta-thalassemia is an inherited disease involving mutations in the beta
globin gene leading to defective hemoglobin production and serious anemia. In
beta-thalassemia patients, there is an over production of red blood cell (RBC)
precursors in the bone marrow, often resulting in bone deformities, decreased
bone mineral density and bone strength, and pathologic fractures, yet these
abundant RBC precursors fail to properly mature into functional red blood
cells. This form of defective red blood cell formation is 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 and the
repeated RBC transfusions to address the anemia. Iron overload can lead to
heart failure, liver fibrosis, and diabetes, among other consequences. Current
treatment for beta-thalassemia includes regular RBC transfusions and daily
iron chelation therapy, which is associated with toxicities.

About ACE-536

ACE-536 is a modified type II activin receptor fusion protein that acts as a
ligand trap for members in the TGF-β superfamily involved in erythropoiesis.
ACE-536 regulates late-stage erythrocyte (red blood cell) precursor cell
differentiation and maturation, distinct from erythropoietin (EPO) which
stimulates the proliferation of early-stage erythrocyte precursor cells. In
diseases of ineffective erythropoiesis, such as myelodysplastic syndromes
(MDS) and thalassemia, in which there is an over-production of early-stage
erythrocyte precursors in the bone marrow, administration of erythropoietin
does not correct the underlying cause of the anemia. By promoting the
differentiation of the precursor cells into mature RBCs, ACE-536 has the
potential to treat the anemia in MDS and beta-thalassemia patients. In a phase
1 clinical study in healthy volunteers, ACE-536 produced a dose-dependent
increase in red blood cell counts and hemoglobin levels. Acceleron and Celgene
are jointly developing ACE-536.

About Acceleron

Acceleron is a privately-held biopharmaceutical company committed to discover,
develop, manufacture and commercialize novel protein therapeutics for orphan
diseases and cancer. Acceleron’s scientific approach takes advantage of its
unique insight to discover first-in-class therapies based on the TGF-β protein
superfamily. Acceleron utilizes proven biotherapeutic technologies and
capitalizes on the company’s internal GMP manufacturing capability to advance
its therapeutic programs rapidly and efficiently. The investors in Acceleron
include Advanced Technology Ventures, Alkermes, Avalon Ventures, Bessemer
Ventures, Celgene, Flagship Ventures, MPM BioEquities, OrbiMed Advisors,
Polaris Ventures, QVT Financial, Sutter Hill Ventures and Venrock. For further
information on Acceleron, please visit


Acceleron Pharma
Steven Ertel, 617-649-9234
Chief Business Officer
Maureen L. Suda (Media)
Suda Communications LLC
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