– Multiple patients continue on treatment with
clinically meaningful increases in hemoglobin and reduction in red
blood cell transfusions surpassing 3 years –
– Top-line results from the MEDALIST Phase 3
trial are on track for the middle of 2018 –
– COMMANDS Phase 3 trial in first-line,
lower-risk MDS to be initiated in Q3 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 Phase 2 trials of luspatercept in patients with lower-risk
myelodysplastic syndromes (MDS) at the American Society of Clinical
Oncology (ASCO) 2018 Annual Meeting in Chicago. Luspatercept is
being developed as part of a global collaboration between Acceleron
and Celgene.
“The ongoing Phase 2 trials continue to provide important
insights into luspatercept’s potential to deliver long-term benefit
to thousands of patients with lower-risk MDS,” said Habib Dable,
President and Chief Executive Officer of Acceleron. “With multiple
patients on treatment for more than three years, we are
increasingly confident in luspatercept’s novel mechanism as an
erythroid maturation agent to address a significant unmet medical
need in lower-risk MDS. We look forward to sharing top-line results
from the MEDALIST Phase 3 trial over the next few months.”
Patients with MDS suffer from insufficient production of red
blood cells, resulting in chronic anemia that can lead to
debilitating fatigue, diminished quality of life and increased
mortality. Because MDS-related chronic anemia often fails to
respond to unapproved therapies which include
erythropoiesis-stimulating agents, many patients require frequent
red blood cell transfusions.
Phase 2 Results
A total of 101 patients with lower-risk MDS have been treated
with luspatercept (dose levels ≥ 0.75 mg/kg) in the Phase 2
trials.
- 55% (55 of 101 patients) achieved a
clinically meaningful erythroid improvement (IWG HI-E
criteria).
- 44% (30 of 68 patients) with a red
blood cell (RBC) transfusion burden at baseline achieved RBC
transfusion independence (RBC-TI) for at least 8 weeks.
- The mean duration of treatment for
RBC-TI responders was 18.3 months (n=30, ongoing).
- Multiple patients continue on treatment
through 40 months, and continue to sustain a clinically meaningful
increase in hemoglobin and reduction in transfusion burden.
Phase 2 Safety Summary
The majority of adverse events (AEs) were Grade 1 or 2. Grade 3
non-serious AEs possibly related to study drug were ascites, blood
bilirubin increase, bone pain, hypertension, mucosal inflammation,
platelet count increase, and transformation to AML (previously
reported as a blast cell count increase). The Grade 3 non-serious
AEs occurred in one patient each, with the exception of
hypertension in 2 patients.
Serious AEs (SAEs) possibly related to study drug were general
physical health deterioration, muscular weakness, musculoskeletal
pain, and myalgia. The four SAEs occurred in three individual
patients.
The ASCO MDS poster presentation is available 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 Ongoing MDS Phase 2 Trials
Data from two Phase 2 trials were presented at the 2018 ASCO
Annual Meeting: the base study in which patients with lower-risk
MDS received treatment with luspatercept for three months and the
long-term extension study in which patients who completed the base
study 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/20180604005451/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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