Acceleron Receives Two FDA Orphan Designations for ACE-536

  Acceleron Receives Two FDA Orphan Designations for ACE-536

ACE-536 granted rare disease designation for the treatment of beta-thalassemia
           and for the treatment of myelodysplastic syndromes (MDS)

Business Wire

CAMBRIDGE, Mass. -- March 26, 2013

Acceleron Pharma, Inc., a biopharmaceutical company developing protein
therapeutics for cancer and orphan diseases, today announced that the United
States Food and Drug Administration (FDA) granted orphan designation for
ACE-536 for the treatment of beta-thalassemia and for the treatment of
myelodysplastic syndromes (MDS), two rare blood disorders characterized by
severe and chronic anemia (low levels of red blood cells). Patients with
beta-thalassemia and MDS suffer from ineffective erythropoiesis, a defect in
which red blood cell precursors are prevented from maturing into healthy,
functional red blood cells resulting in severe anemia. Anemia in these
patients is often unresponsive to current therapies and many patients are
ultimately dependent on frequent red blood cell transfusions. ACE-536 is an
investigational protein therapeutic that increases red blood cells through a
novel mechanism and is being developed by Acceleron as part of a global
collaboration with Celgene Corporation (NASDAQ: CELG).

“ACE-536 has shown the potential to address a significant unmet medical need
in the treatment of these rare hematological disorders,” said Matthew Sherman,
M.D., Chief Medical Officer of Acceleron. “We are excited about the prospect
of bringing a new therapeutic option to these underserved patient populations
and continue to work closely with our clinical investigators on both of our
recently initiated phase 2 clinical studies of ACE-536, one in patients with
beta-thalassemia and a second in patients with MDS.”

Orphan designation is granted by the FDA Office of Orphan Products Development
to advance the evaluation and development of safe and effective therapies for
the treatment of rare diseases or conditions affecting fewer than 200,000
people in the U.S. Under the Orphan Drug Act, the FDA may provide grant
funding towards clinical trial costs, tax advantages, FDA user-fee benefits,
and seven years of market exclusivity in the United States following marketing
approval by the FDA. The granting of an orphan designation request does not
alter the standard regulatory requirements and process for obtaining marketing
approval. For more information about orphan designation, please visit the FDA
website at

About Beta-Thalassemia

Beta-thalassemia is a rare, 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 that fail to properly mature into functional
RBCs, a condition referred to as “ineffective erythropoiesis.” This often
leads to bone deformities, decreased bone mineral density and bone strength,
and pathologic fractures. Beyond the severe anemia and bone effects, 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

About Myelodysplastic Syndromes

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 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 (AML). Nearly all MDS patients suffer
from anemia. The anemia in MDS is characterized by high endogenous levels of
erythropoietin (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 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 beta-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
dose-dependent increases in red blood cell counts and hemoglobin levels.
Acceleron and Celgene are jointly developing ACE-536. ACE-536 is currently in
phase 2 clinical trials in patients with beta-thalassemia and in patients with
myelodysplastic syndromes. For more information, please visit

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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