Luspatercept increased hemoglobin levels and
generated transfusion independence in patients with lower risk
myelodysplastic syndromes
Celgene Corporation (NASDAQ:CELG) and Acceleron Pharma Inc.
(NASDAQ:XLRN) today announced preliminary results from an ongoing
long-term Phase 2 extension study in patients with lower risk
myelodysplastic syndromes (MDS) at the 57th American Society of
Hematology (ASH) Annual Meeting and Exposition. Results highlighted
in an oral presentation showed that patients with lower risk MDS
treated with luspatercept in the long-term extension study achieved
and maintained increased hemoglobin levels and transfusion
independence. Celgene and Acceleron are jointly developing
luspatercept.
“These results for longer-term luspatercept treatment in lower
risk MDS patients are very exciting,” said Aristoteles Giagounidis,
M.D., Ph.D., Head of the Department of Oncology, Haematology, and
Palliative Care at Marien Hospital in Düsseldorf, Germany. “There
is substantial unmet medical need for patients who do not respond
or become refractory to treatment with erythropoiesis stimulating
agents or who are considered ineligible to receive them.
Luspatercept has shown very encouraging activity in these patients
which provides the basis for the Phase 3 MEDALIST study.”
Luspatercept Data Presented at
ASH
A total of 32 low- or intermediate-1 risk MDS patients were
treated with luspatercept in this long-term 24-month open-label
extension study. Luspatercept was administered subcutaneously once
every 3 weeks at a starting dose level of 1.0 mg/kg. The dose level
could be increased to 1.75 mg/kg. Of these 32 patients, 13 had a
low transfusion burden (<4 units RBC/8 weeks) and 19 had a high
transfusion burden (≥4 units RBC/8 weeks). 59% of patients had been
treated previously with erythropoiesis stimulating agents (ESA) and
19% of patients had previously been treated with lenalidomide. 91%
of the patients were ring sideroblast positive (more than 15% of
erythroid cells in the bone marrow were ring sideroblasts).
Patients who were refractory or intolerant to prior ESA
treatment
- 63% (12 of 19) of HTB and LTB patients
with prior ESA treatment achieved an International Working Group
(IWG) Hematologic Improvement Erythroid (HI-E) response of a
reduction of ≥4 units RBC over 8 weeks or a hemoglobin increase
≥1.5 g/dL for ≥8 weeks during their luspatercept treatment
period
- 50% (7 of 14) achieved transfusion
independence
Patients who are considered ESA ineligible because they had
elevated erythropoietin levels between 200 and 500 U/L
- 71% (5 of 7) achieved an HI-E response
and 50% (2 of 4) achieved transfusion independence
High Transfusion Burden (HTB) Patients
- 68% (13 of 19) of the HTB patients
achieved the IWG HI-E criteria of a reduction of ≥4 units RBC over
8 weeks
- 42% (8 of 19) of the HTB patients
achieved transfusion independence for ≥8 weeks
Low Transfusion Burden (LTB) Patients
- 69% (9 of 13) of the LTB patients
achieved the IWG HI-E response criteria of a hemoglobin increase
≥1.5 g/dL for ≥8 weeks
- The mean increase in hemoglobin reached
levels between 2.0 and 2.5 g/dL and was maintained for the 9 months
for which data are available
Safety
- Adverse events at least possibly
related to study drug that occurred in patients during the
extension study included bone pain, headache, hypotonia, myalgia
and nausea. No serious or grade 3 or 4 adverse events related to
study drug have been reported during the ongoing extension
study.
Celgene and Acceleron are in the process of initiating a global
Phase 3 study in low risk, ring sideroblast positive MDS
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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version on businesswire.com: http://www.businesswire.com/news/home/20151205005048/en/
For Celgene:Investors:(908)
673-9628 investors@celgene.comorMedia:(908) 673-2275
media@celgene.comorFor
AcceleronInvestors:Todd James, 617-649-9393Senior Director,
Corporate CommunicationsorMedia:BMC CommunicationsBrad Miles,
917-570-7340
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