PULSAR trial expands Acceleron’s pipeline into
pulmonary disease
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 the initiation
of the PULSAR Phase 2 trial of sotatercept for the treatment of
patients with pulmonary arterial hypertension (PAH), a chronic and
life-changing disease that can lead to heart failure.
“There is a real need for new treatment options that have the
potential to improve survival for PAH patients,” said David
Badesch, M.D., the Director of the Pulmonary Hypertension Program
at the University of Colorado and Chair of the PULSAR Trial
Steering Committee. “Based on its novel mechanism and preclinical
results, sotatercept, particularly in combination with
standard-of-care therapies, could be an important advancement in
the future treatment of PAH. I look forward to the results of the
trial.”
“The initiation of the PULSAR trial is an important milestone in
our newly expanded research and development efforts in pulmonary
disease,” said Habib Dable, Chief Executive Officer of Acceleron.
“We believe that treatment with sotatercept could restore a vital
signaling pathway that is known to be deficient in PAH patients.
This is an extremely important trial, as we hope to bring an
innovative medicine to the thousands of patients who are suffering
from PAH.”
The Company plans to report preliminary results from the 6-month
primary treatment period of the PULSAR Phase 2 trial in the first
half of 2020.
About the PULSAR Trial
The PULSAR Phase 2 trial is a randomized, double-blind,
placebo-controlled study designed to evaluate the efficacy and
safety of sotatercept in PAH patients. The primary endpoint of the
trial is the change from baseline in pulmonary vascular resistance
(PVR) over a 24-week treatment period. The key secondary endpoint
is change from baseline in six-minute walk distance (6MWD). A total
of 90 patients will be randomized 1:1:1 into three treatment arms
with standard-of-care vasodilator therapies in combination with
sotatercept or placebo. Following the 6-month double-blind
treatment period, participants in the trial will be eligible to
enroll into the 18-month extension period.
For additional information about this clinical trial, please
visit clinicaltrials.gov, identifier NCT03496207.
About Sotatercept
Sotatercept acts as a ligand trap for members of the
transforming growth factor-beta superfamily, including those
directly involved in the BMP pathway proven critical for
maintaining healthy pulmonary vasculature. In multiple preclinical
studies in PAH, sotatercept significantly decreased pulmonary
vessel muscularization, improved pulmonary arterial pressures, and
decreased indicators of right heart failure. Sotatercept is
currently being evaluated in the PULSAR Phase 2 trial in PAH.
About Pulmonary Arterial Hypertension (PAH)
Pulmonary Arterial Hypertension (PAH) is a rare, chronic, and
rapidly progressing disorder characterized by the narrowing of
small pulmonary arteries and elevated blood pressure in the
pulmonary circulation. PAH results in significant and progressive
strain on the right side of the heart, often leading to limited
physical activity, heart failure, and reduced life expectancy. The
5-year survival rate for patients with PAH is approximately 57%.
Available therapies primarily act by promoting the dilation of
pulmonary vessels without addressing the underlying cause of the
disease. As a result, PAH often progresses rapidly for many
patients despite standard-of-care treatment. A growing body of
research has implicated imbalances in BMP and TGF-beta signaling as
a primary driver of PAH in all forms of the disease.
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/20180522005145/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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