Luspatercept increased hemoglobin levels,
reduced transfusion burden and improved measures of iron overload
in beta-thalassemia patients
Celgene Corporation (NASDAQ: CELG) and Acceleron Pharma Inc.
(NASDAQ: XLRN) today announced the presentation of preliminary data
from the luspatercept phase 2 clinical trial in patients with
beta-thalassemia at the 56th American Society of Hematology (ASH)
Annual Meeting and Exposition. This presentation was one of four
selected by ASH to be highlighted in this morning’s press briefing
on emerging anemia treatments. Dr. Antonio Piga will present the
full set of data in an oral session, showing that luspatercept
increased hemoglobin levels, reduced transfusion burden, and
improved measures of iron overload in beta-thalassemia patients.
Celgene and Acceleron are jointly developing luspatercept.
“These results in beta-thalassemia are extremely exciting,” 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. “A therapy that could
potentially treat both the anemia and complications of
beta-thalassemia, such as iron overload, is unprecedented in this
disease and could potentially benefit most patients, regardless of
the genetic background.”
In the phase 2 study, luspatercept was evaluated in
transfusion-dependent (TD) and non-transfusion-dependent (NTD)
beta-thalassemia patients.
A total of 30 patients were treated in the dose escalation stage
of this study, in which luspatercept was administered
subcutaneously, once every 3 weeks for up to 5 doses (16 weeks) to
cohorts of 6 patients each at dose levels of 0.2, 0.4, 0.6, 0.8, or
1.0 mg/kg. Of these 30 patients, 23 were non-transfusion dependent
and 7 were transfusion dependent.
Improvement of anemia and transfusion
burden:
- 9 of 12 patients (75%) treated with
dose levels of 0.8 or 1.0 mg/kg of luspatercept met the study
primary endpoint of an erythroid response
- 6 of 6 (100%) transfusion dependent
patients achieved a reduction in transfusion burden of at least 60%
over a 12 week period
- 3 of 6 (50%) non-transfusion dependent
patients had a sustained hemoglobin increase of at least 1.5 g/dL
for ≥ 2 weeks
Reduction in iron overload:
- Reductions in liver iron concentration
(LIC), a measure of iron overload, were observed in both
non-transfusion dependent and transfusion-dependent patients
- In non-transfusion dependent patients
with baseline LIC ≥5 mg/g dry weight, 8 of 12 (67%) patients had a
reduction in LIC of ≥1 mg/g dry weight in this 16 week study
- In transfusion dependent patients with
baseline LIC ≥5 mg/g dry weight, 4 of 5 (80%) patients had
reductions in LIC ranging from 0.7 to 4.7 mg/g dry weight
- Transfusion dependent patients also had
reductions in serum ferritin, another marker of iron overload,
ranging from 12-60%
Improvement in disease
complications:
- 2 of 2 patients who presented with
persistent leg ulcers, a complication of beta-thalassemia,
experienced rapid healing of the ulcers following treatment with
luspatercept
The most common adverse events were bone pain, headache,
myalgia, asthenia, influenza, macule and pain in extremity. There
were no drug-related serious adverse events and no patient
developed anti-drug antibodies on treatment. 3 patients
discontinued early due to adverse events; 1 patient (0.6 mg/kg)
with occipital headache, 1 patient (0.8 mg/kg) with ankle pain, and
1 patient (0.8 mg/kg) with back pain.
About Beta-thalassemia
Beta-thalassemia is an inherited disease involving mutations in
the beta globin gene leading to deficient 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. These abundant RBC
precursors fail to properly mature into functional RBCs, which 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 as well as the repeated RBC
transfusions to address the anemia. Iron overload can lead to heart
failure, liver fibrosis, and diabetes, among other consequences.
Current clinical management for beta-thalassemia includes regular
RBC transfusions and daily iron chelation therapy, which is
associated with toxicities. There are no drugs approved to treat
beta-thalassemia leaving healthcare providers with few options for
patients.
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 Acceleron
Acceleron is a clinical stage biopharmaceutical company focused
on the discovery, development and commercialization of novel
protein therapeutics for cancer and rare diseases. The company is a
leader in understanding the biology of the Transforming Growth
Factor-Beta (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 protein 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 the Company's website at www.celgene.com. Follow us on
Twitter @Celgene as well.
Forward-Looking Statements
Acceleron: Cautionary Note on Forward-Looking Statements
This press release includes forward-looking statements about the
Company's strategy, future plans and prospects, including
statements regarding the development of the Company's compounds,
including luspatercept, and the Company's TGF-β superfamily program
generally, the timeline for clinical development and regulatory
approval of the Company's compounds, the expected timing for the
reporting of data from ongoing trials, and the structure of the
Company's planned or pending clinical trials. The words
"anticipate," "appear," "believe," "estimate," "expect," "intend,"
"may," "plan," "predict," "project," "target," "potential," "will,"
"would," "could," "should," "continue," and similar expressions are
intended to identify forward-looking statements, although not all
forward-looking statements contain these identifying words. 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 the risks that the Company's cash position
will be insufficient to fund operations into the second half of
2017, that preclinical testing of the Company's compounds and
preliminary 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 the Company or
its collaboration partner, Celgene, will be unable to successfully
complete the clinical development of its compounds, that the
development of the Company's compounds will take longer or cost
more than planned, that the Company may be delayed in initiating or
completing any clinical trials, and that the Company'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 the
Company's Annual Report on Form 10-K which was filed with the
Securities and Exchange Commission (SEC) on March 17, 2014, and
other filings that the Company may make with the SEC in the future.
The forward-looking statements contained in this press release
reflect the Company's current views with respect to future events,
and the Company 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
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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
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For CelgeneInvestors:
908-673-9628investors@celgene.comorMedia:
908-673-2275media@celgene.comorFor
AcceleronInvestors:Acceleron PharmaSteven Ertel,
617-649-9234Chief Business OfficerorMedia:Suda Communications
LLCMaureen L. Suda, 585-387-9248
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