Luspatercept increased hemoglobin levels,
reduced transfusion burden, improved health-related quality of life
measures and had beneficial effects on liver iron concentration in
patients with beta-thalassemia
Celgene Corporation (NASDAQ:CELG) and Acceleron Pharma Inc.
(NASDAQ:XLRN) today announced preliminary results from two Phase 2
clinical trials of luspatercept in patients with beta-thalassemia
were presented at the 57th American Society of Hematology (ASH)
Annual Meeting and Exposition. Results highlighted in an oral
presentation showed that luspatercept increased hemoglobin levels,
reduced transfusion burden, improved health-related quality of life
measures and had beneficial effects on liver iron concentration in
patients with beta-thalassemia. Celgene and Acceleron are jointly
developing luspatercept.
“These luspatercept results are very exciting as they show
positive effects across a range of clinically challenging
complications of beta-thalassemia,” 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 the anemia,
complications of beta-thalassemia, such as iron overload, and
improve measures of health-related quality of life would be a huge
advance to help address the substantial unmet need of patients with
beta-thalassemia.”
Luspatercept Data Presented at
ASH
Luspatercept was evaluated in a Phase 2, multicenter, open-label
study in adults with non-transfusion-dependent (NTD) and
transfusion-dependent (TD) beta-thalassemia patients. The primary
objectives were to assess the proportion of NTD patients that
achieved a hemoglobin increase ≥ 1.5 g/dL from baseline and the
proportion of TD patients that achieved at least a 20% reduction in
transfusion burden. A total of 64 patients, of which 59 were
efficacy evaluable (31 NTD and 28 TD), were enrolled in the dose
escalation and expansion stages of the Phase 2 clinical trial. In
this study, patients received up to 5 doses via subcutaneous
injection once every 3 weeks. 51 of the 64 patients from this
3-month study enrolled in the long-term Phase 2 extension trial in
which these patients may receive up to two years of treatment with
luspatercept. Data was presented from both the 3-month study and
the long-term extension study.
Improvement of anemia and transfusion burden:
- Of the 17 NTD patients that received at
least 5 cycles of luspatercept at dose levels of 0.8 mg/kg or
higher
- 65% (11/17) increased hemoglobin levels
≥ 1.0 g/dL over a 12-week period
- 47% (8/17) increased hemoglobin levels
≥ 1.5 g/dL over a 12-week period
- Data presented showed sustained
increases in hemoglobin with the longest-treated patients having
received nearly six months of luspatercept
- Of the 28 TD patients
- 79% (22/28) patients had ≥ 20%
reduction of transfusion burden
- 75% (21/28) patients had ≥ 33%
reduction of transfusion burden
- 57% (16/28) patients had ≥ 50%
reduction of transfusion burden
Changes in iron overload:
- Liver iron concentration (LIC), a
measure of iron overload, was maintained or reduced in both
non-transfusion dependent and transfusion-dependent patients
- 50% (4/8) TD patients with baseline LIC
≥ 5 mg/g dry weight (dw) had decrease in LIC ≥ 2 mg/g dw
- 100% (14/14) TD patients with baseline
LIC < 5 mg/g dw maintained LIC < 5 mg/g dw
- 36% (5/14) NTD patients with baseline
LIC ≥ 5 mg/g dw had decrease in LIC ≥ 2 mg/g dw
- 100% (14/14) NTD patients with baseline
LIC < 5 mg/g dw maintained LIC < 5 mg/g dw
Improvement in health-related quality of life (QoL) measures in
NTD patients:
- Improved health-related QoL (FACT-An
anemia subscore) correlated with increases in hemoglobin in NTD
Patients
Safety:
- The most common related adverse events
were bone pain, myalgia, headache, arthralgia, musculoskeletal
pain, asthenia, injection site pain, back pain and pain in jaw
- Related grade 3 adverse events included
headache, bone pain, asthenia, and myalgia
- There were no drug-related serious
adverse events
Celgene and Acceleron are in the process of initiating a global
Phase 3 study in regularly transfused beta-thalassemia
patients.
Luspatercept is an investigational product that is not approved
for any use in any country.
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. 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
therapeutic candidates that regulate cellular growth and repair.
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 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.
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 Statement
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-beta 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 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 the Company expects 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 or Celgene 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 2, 2015, and
other filings that the Company has made and 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
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
AcceleronInvestors:Todd James, 617-649-9393Senior Director,
Corporate CommunicationsorMedia:BMC CommunicationsBrad Miles,
917-570-7340Bmiles@bmccommunications.com
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