Luspatercept Data Presented at the 13th International Symposium on Myelodysplastic Syndromes
02 Mayo 2015 - 9:30AM
Business Wire
– In lower risk myelodysplastic syndromes
patients, luspatercept increased hemoglobin levels and enabled
transfused patients to become transfusion independent –
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 preliminary data from
the ongoing phase 2 clinical trial of luspatercept in patients with
lower risk myelodysplastic syndromes (MDS). The data showed that
luspatercept increased hemoglobin levels and enabled many patients
to become transfusion independent. The presentation was given this
morning by Uwe Platzbecker, M.D., University Hospital in Dresden,
Germany in an oral session titled “Best of Clinical Trials II” at
the MDS Foundation 13th International Symposium on MDS. Acceleron
and its collaboration partner, Celgene Corporation, are jointly
developing luspatercept.
“We are excited by the results in lower risk MDS patients, which
confirm and extend our previous findings,” said Uwe Platzbecker,
M.D., Professor of Hematology and Head of the MDS program at the
University Hospital in Dresden, Germany and coordinating principal
investigator of the luspatercept PACE-MDS study. “Luspatercept may
be useful early in the treatment of lower risk MDS patients, either
as the initial treatment for anemia or in patients who do not
respond or become refractory to treatment with erythropoiesis
stimulating agents.”
Key preliminary data from the presentation are highlighted
below:
- The ongoing phase 2 clinical trial is
fully enrolled and data from 44 of the 58 patients was available
for efficacy analyses. 17 of these 44 patients were enrolled in the
expansion cohort, using the planned phase 3 dosing regimen, whose
data had not been previously presented.
- Luspatercept was generally
well-tolerated with the majority of adverse events (AE) being mild
to moderate (grade 1 or 2). There were two possibly related serious
adverse events of muscle pain and worsening of general
condition.
- In the higher dose groups (0.75 to 1.75
mg/kg administered subcutaneously every three weeks):
- 54% achieved the International Working
Group (IWG) hematologic improvement-erythroid(HI-E) threshold of
efficacy.
- 36% of patients who received red blood
cell transfusions during the 8 weeks prior to treatment in the
study achieved transfusion independence for at least 8 weeks during
the study.
- Ring sideroblasts (RS) are a type of
abnormal red blood cell (RBC) precursor cell in the bone marrow.
These ring sideroblasts are associated with ineffective
erythropoiesis and anemia. When at least 15% of the cells in an MDS
patient’s bone marrow are ring sideroblasts, this patient is
considered RS positive. At least 30% of all MDS patients are RS
positive, and the proportion is likely even greater within the
lower risk segment of all MDS patients.
- In the RS positive patients in the
higher dose groups treated with luspatercept,
- 63% achieved IWG HI-E
- 39% achieved transfusion
independence
The presentation slides from Dr. Platzbecker’s talk are
available on Acceleron's website (www.acceleronpharma.com) under
the Publications tab.
About Luspatercept
Luspatercept is a modified activin receptor type IIB fusion
protein that acts as a ligand trap for members in the Transforming
Growth Factor-Beta (TGF-β) 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 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 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-β
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 protein 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.
Acceleron Pharma Inc.Kevin F. McLaughlin, 617-649-9204Senior
Vice President and Chief Financial OfficerorMedia:Suda
Communications LLCMaureen L. Suda, 585-387-9248
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