- Clinically meaningful increases in hemoglobin
and reductions in red blood cell transfusion burden observed
through 36 months –
- Top-line results from the BELIEVE Phase 3
trial are on track for the middle of 2018 -
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 updated results
from the ongoing Phase 2 trials of luspatercept in patients with
beta-thalassemia during an oral presentation at the 23rd Congress
of the European Hematology Association (EHA) in Stockholm, Sweden.
Luspatercept is being developed as part of a global collaboration
between Acceleron and Celgene.
“These results reinforce our enthusiasm for luspatercept’s
potential as a safe, efficacious therapy for beta-thalassemia
patients over the long term,” said Habib Dable, President and Chief
Executive Officer of Acceleron.
Beta-thalassemia is caused by a genetic defect in the production
of hemoglobin, a protein that carries oxygen to red blood cells
throughout the body. Patients suffer from severe, chronic anemia
and often experience fatigue, organ enlargement, and bone
complications. Patients require lifelong therapy that includes
frequent red blood cell transfusions and corresponding treatment
for the iron overload that results.
“We now have patients with both non-transfusion- and
transfusion-dependent beta-thalassemia continuing on treatment for
three years,” Dable continued. “We look forward to sharing top-line
results from the BELIEVE Phase 3 trial over the next few
months.”
Phase 2 Results
A total of 31 non-transfusion-dependent patients have been
treated with luspatercept (dose levels ≥ 0.6 mg/kg) in the
trials.
- 53% (16 of 30) achieved a ≥ 1 gram per
deciliter increase in hemoglobin in the fixed 12-week intervals
including weeks 13 to 24 and weeks 37 to 48 when compared to
baseline.
- At week 48, the mean improvement in
6-minute walk distance was 18.6% compared to baseline (n=9).
- Multiple patients remain on treatment
through 36 months, and continue to sustain clinically meaningful
increases in hemoglobin.
A total of 32 transfusion-dependent patients have been treated
with luspatercept (dose levels ≥ 0.6 mg/kg) in the trials.
- 41% (12 of 29) achieved a reduction in
RBC transfusion burden of at least 33% in the fixed 12-week
intervals including weeks 13 to 24 and weeks 37 to 48 when compared
to baseline.
Phase 2 Safety Summary
The majority of adverse events (AEs) were Grade 1 or 2. Grade 3
AEs possibly or probably related to study drug were bone pain (n=3
patients), asthenia (n=2 patients), bone infarction (n=1 patient),
headache (n=1 patient), and presyncope (n=1 patient). One serious
AE of biliary colic was reported as possibly related to study
drug.
The EHA beta-thalassemia presentation will be available
immediately following the presentation on Saturday, June 16th at
the conference under the Science page of the Company’s website at
www.acceleronpharma.com/.
Luspatercept is an investigational product that is not approved
for any use in any country.
About the Phase 2 Trial
Data from two Phase 2 trials were presented at the 23rd Congress
of the EHA: the base study in which beta-thalassemia patients
received treatment with luspatercept for three months and the
ongoing long-term safety extension study in which patients may
receive treatment with luspatercept for up to an additional five
years.
About Luspatercept
Luspatercept is a first-in-class erythroid maturation agent
(EMA) that regulates late-stage red blood cell maturation.
Acceleron and Celgene are jointly developing luspatercept as part
of a global collaboration. Phase 3 clinical trials are underway 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 Phase 3 trial is being planned in first-line,
lower-risk, MDS patients (the COMMANDS trial). 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 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 the
Company's strategy, future plans and prospects, including
statements regarding the development of the Company's compounds,
the timeline for clinical development and regulatory approval of
the Company’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 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 the development of the Company's compounds
will take longer and/or cost more than planned, that the Company or
its collaboration partner, Celgene, will be unable to successfully
complete the clinical development of the Company’s compounds, that
the Company or Celgene may be delayed in initiating, enrolling or
completing any clinical trials, and that the Company'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 the
Company's most recent Annual Report on Form 10-K, 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
are based on management’s current views, plans, estimates,
assumptions and projections with respect to future events, and the
Company 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/20180615005201/en/
Acceleron Pharma Inc.Todd James, IRC, (617) 649-9393Vice
President, Investor Relations and Corporate CommunicationsorCandice
Ellis, 617-649-9226Manager, Investor Relations and Corporate
CommunicationsorMedia:Matt Fearer, 617-301-9557Director, Corporate
Communications
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