Acceleron Pharma Inc. (NASDAQ:XLRN), a leading biopharmaceutical
company in the discovery and development of TGF-beta therapeutics
to treat serious and rare diseases, today announced that the
journal Blood has published results from the Phase 2 study of
luspatercept in patients with red blood cell (RBC)
transfusion-dependent and non-transfusion-dependent
beta-thalassemia. Luspatercept is an investigational therapy that
is part of a global collaboration between Acceleron and
Celgene.
In this open-label, Phase 2 study (a three-month initial stage
followed by a five-year extension stage), patients were treated
with luspatercept subcutaneously once every three weeks. The
primary outcome measures were a reduction in RBC transfusion
burden—compared with pretreatment—in transfusion-dependent
patients, and changes in hemoglobin levels from baseline in
non-transfusion-dependent patients. The extension study is ongoing
and evaluates the long-term safety and tolerability of luspatercept
for up to 5 years.
“Current treatment options for patients with beta-thalassemia
are essentially limited to supportive therapy, including red blood
cell transfusions, which leads to iron overload,” said Antonio
Piga, M.D., Professor, Department of Clinical and Biological
Sciences at Turin University and lead author of the newly published
study. “Luspatercept has demonstrated an ability to improve
hemoglobin levels and reduce transfusion burden through apparent
restoration of the late-stage red blood cell maturation process,
which is known to be inhibited in these patients. Based on these
results and those of subsequent studies, luspatercept has the
potential to address significant unmet medical needs for patients
suffering from beta-thalassemia.”
The article, entitled “Luspatercept improves hemoglobin levels
and blood transfusion requirements in a study of patients with
beta-thalassemia,” is now available online and will be published in
a future print edition of Blood.
“We’re pleased that these important results are now fully
available to the greater hematology community,” said Robert K.
Zeldin, M.D., Chief Medical Officer of Acceleron. “This study
provided key information on the efficacy and safety profile of
luspatercept and informed the design of the Phase 3 trial, known as
BELIEVE, for which positive results in transfusion-dependent
patients with beta-thalassemia were presented last month at the
American Society of Hematology (ASH) Annual Meeting and were
included in the ‘Best of ASH’ session at that meeting.”
Luspatercept is being evaluated in ongoing studies in patients
with non-transfusion-dependent beta-thalassemia (the Phase 2 BEYOND
trial) as well as in patients with lower-risk myelodysplastic
syndromes (MDS) (the Phase 2 PACE-MDS trial and the Phase 3
MEDALIST and COMMANDS trials) and in a Phase 2 trial in patients
with myelofibrosis.
Luspatercept is an investigational therapy that is not approved
for any use in any country. Celgene and Acceleron are
planning submission of marketing applications for luspatercept
in the United States and Europe in
the first half of 2019.
About Luspatercept
Luspatercept is a first-in-class erythroid maturation agent that
is believed to regulate late-stage red blood cell maturation.
Acceleron and Celgene are jointly developing luspatercept
as part of a global collaboration. Phase 3 clinical trials continue
to evaluate the safety and efficacy of luspatercept in patients
with MDS (the MEDALIST trial) and in patients with beta-thalassemia
(the BELIEVE trial). A COMMANDS Phase 3 trial in first-line,
lower-risk, MDS patients, the BEYOND Phase 2 trial in
non-transfusion-dependent beta-thalassemia, and a Phase 2 trial in
myelofibrosis are ongoing. For more information, please
visit www.clinicaltrials.gov.
About Acceleron
Acceleron is a Cambridge-based, clinical-stage
biopharmaceutical company dedicated to the discovery, development,
and commercialization of therapeutics to treat serious and rare
diseases. The Company's leadership in the understanding of TGF-beta
biology and protein engineering generates innovative compounds that
engage the body's ability to regulate cellular growth and
repair.
Acceleron focuses its research and development efforts in
hematologic, neuromuscular, and pulmonary diseases. In hematology,
the Company and its global collaboration partner, Celgene, are
developing luspatercept for the treatment of chronic anemia in
myelodysplastic syndromes, beta-thalassemia, and myelofibrosis.
Acceleron is also advancing its neuromuscular franchise with two
distinct Myostatin+ agents, ACE-083 and ACE-2494, and a phase 2
pulmonary program with sotatercept in pulmonary arterial
hypertension.
For more information, please visit www.acceleronpharma.com.
Follow Acceleron on Social
Media: @AcceleronPharma and LinkedIn.
Forward-Looking Statements
This press release contains forward-looking statements about
Acceleron’s strategy, future plans and prospects, including
statements regarding the development of Acceleron’s compounds, the
timeline for clinical development and regulatory approval of
Acceleron’s compounds and the expected timing for reporting of data
from ongoing clinical trials. The words "anticipate," "believe,"
"could," "estimate," "expect," “goal,” "intend," "may," "plan,"
"potential," "project," "should," "target," "will," "would," and
similar expressions are intended to identify forward-looking
statements, although not all forward-looking statements contain
these identifying words.
Actual results could differ materially from those included in
the forward-looking statements due to various risks and
uncertainties, including, but not limited to, that preclinical
testing of Acceleron’s compounds and data from clinical trials may
not be predictive of the results or success of ongoing or later
clinical trials, that the development of Acceleron’s compounds will
take longer and/or cost more than planned, that Acceleron or its
collaboration partner, Celgene, will be unable to successfully
complete the clinical development of Acceleron’s compounds, that
the Company or Celgene may be delayed in initiating,
enrolling or completing any clinical trials, and that Acceleron’s
compounds will not receive regulatory approval or become
commercially successful products. These and other risks and
uncertainties are identified under the heading "Risk Factors"
included in Acceleron’s most recent Annual Report on Form 10-K, and
other filings that Acceleron has made and may make with
the SEC in the future.
The forward-looking statements contained in this press release
are based on management’s current views, plans, estimates,
assumptions and projections with respect to future events, and
Acceleron does not undertake and specifically disclaims any
obligation to update any forward-looking statements.
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version on businesswire.com: https://www.businesswire.com/news/home/20190110005131/en/
Acceleron Pharma Inc.Todd James, IRC, (617) 649-9393Vice
President, Investor Relations and Corporate
CommunicationsOrMedia:Matt Fearer, (617) 301-9557Director,
Corporate Communications
Acceleron Pharma (NASDAQ:XLRN)
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