FDA Fast Track Designation Granted to Luspatercept for the Treatment of Patients with Lower-Risk Myelodysplastic Syndromes
07 Diciembre 2015 - 6:00AM
Business Wire
Celgene Corporation (NASDAQ:CELG) and Acceleron Pharma Inc.
(NASDAQ:XLRN) today announced that the United States Food and Drug
Administration (FDA) has granted Fast Track Designation to
luspatercept for the treatment of anemia in patients with
lower-risk myelodysplastic syndromes (MDS). The Fast Track program
of the FDA is designed to facilitate the development, and expedite
the review, of new drugs that are intended to treat serious or
life-threatening conditions and that demonstrate the potential to
address unmet medical needs.
Celgene and Acceleron are developing luspatercept to treat
patients with rare blood disorders, including MDS and
beta-thalassemia. For people with MDS, a type of blood cancer,
anemia is a challenging clinical complication of the disease.
Patients receive chronic red blood cell transfusions as standard of
care to manage the anemia. Luspatercept is designed to
increase red blood cell levels in a way that is fundamentally
distinct from existing therapies, and has the potential to treat
anemia in patients whose disease is not adequately addressed by the
treatments available today.
“The FDA’s Fast Track Designation for luspatercept recognizes
the serious unmet medical needs of patients with MDS,” said
Jacqualyn A. Fouse, President, Hematology/Oncology for Celgene.
“Celgene and Acceleron are working to initiate a Phase 3 clinical
program to investigate the treatment of patients with low- and
intermediate- risk MDS before the end of the year.”
The Companies previously announced FDA Fast Track Designation
for luspatercept to treat beta-thalassemia.
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-beta) superfamily involved in the late
stages of erythropoiesis (red blood cell production). Luspatercept
is thought to regulate 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. Acceleron and Celgene are
initiating phase 3 clinical trials that are designed to evaluate
the safety and efficacy of luspatercept in patients with
myelodysplastic syndromes (the "MEDALIST" study) and in patients
with beta-thalassemia (the "BELIEVE" study). For more information,
please visit www.clinicaltrials.gov.
About Myelodysplastic Syndromes
MDS comprise 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 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.
About Celgene
Celgene Corporation, headquartered in Summit, New Jersey, is an
integrated global pharmaceutical company engaged primarily in the
discovery, development and commercialization of innovative
therapies for the treatment of cancer and inflammatory diseases
through gene and protein regulation. For more information, please
visit www.celgene.com. Follow Celgene on Social Media:
@Celgene, Pinterest, LinkedIn and YouTube.
Forward-Looking Statements
Acceleron:
Cautionary Note on Forward-Looking Statements
This press release includes forward-looking statements about
Acceleron's strategy, future plans and prospects, including
statements regarding the development of luspatercept and
Acceleron’s TGF-beta superfamily program generally, the timeline
for clinical development and regulatory approval of luspatercept,
the expected timing for the reporting of data from ongoing trials,
and the structure of Acceleron’s planned or pending clinical
trials. Each forward-looking statement is subject to risks and
uncertainties that could cause actual results to differ materially
from those expressed or implied in such statement. Applicable risks
and uncertainties include 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
data may not be available when we expect it to be, that Acceleron
or Celgene will be unable to successfully complete the clinical
development of its compounds, that the development of Acceleron's
compounds will take longer or cost more than planned, that
Acceleron or Celgene may be delayed in initiating or completing any
clinical trials, and that Acceleron's compounds will not receive
regulatory approval or become commercially successful products.
Other risks and uncertainties include those identified under the
heading "Risk Factors" included in Acceleron's Annual Report on
Form 10-K which was filed with the Securities and Exchange
Commission (SEC) on March 2, 2015, 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 reflect
Acceleron’s current views with respect to future events, and
Acceleron does not undertake and specifically disclaims any
obligation to update any forward-looking statements.
Celgene:
This press release contains forward-looking statements, which
are generally statements that are not historical facts.
Forward-looking statements can be identified by the words
"expects," "anticipates," "believes," "intends," "estimates,"
"plans," "will," “outlook” and similar expressions. Forward-looking
statements are based on management’s current plans, estimates,
assumptions and projections, and speak only as of the date they are
made. We undertake no obligation to update any forward-looking
statement in light of new information or future events, except as
otherwise required by law. Forward-looking statements involve
inherent risks and uncertainties, most of which are difficult to
predict and are generally beyond our control. Actual results or
outcomes may differ materially from those implied by the
forward-looking statements as a result of the impact of a number of
factors, many of which are discussed in more detail in our Annual
Report on Form 10-K and our other reports filed with the Securities
and Exchange Commission.
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For
Celgene:Investors:908-673-9628investors@celgene.comorMedia:908-673-2275media@celgene.comorFor
Acceleron:Investors:Todd James, 617-649-9393Senior Director,
Corporate CommunicationsorMedia:BMC CommunicationsBrad Miles,
917-570-7340
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