Acceleron Pharma Inc. (NASDAQ:XLRN), a leading biopharmaceutical
company in the discovery and development of TGF-beta superfamily
therapeutics to treat serious and rare diseases, today announced
that the United States Food and Drug Administration (FDA) has
granted Orphan Drug designation to sotatercept for the treatment of
patients with pulmonary arterial hypertension (PAH).
“We’re pleased that the FDA has granted this designation for
sotatercept,” said Janethe de Oliveira Pena, MD, PhD, Vice
President, Pulmonary Medical Research at Acceleron. “In preclinical
studies, sotatercept has demonstrated an ability to target the
underlying mechanisms of PAH, which is a rare disease of high unmet
medical need. We believe that if similar effects are seen in a
clinical setting, sotatercept has the potential to become an
important addition to the standard of care in PAH.”
Orphan designation is granted by the FDA Office of Orphan
Products Development to advance the evaluation and development of
safe and effective therapies for the treatment of rare diseases or
conditions affecting fewer than 200,000 people in the U.S. Under
the Orphan Drug Act, the FDA may provide grant funding toward
clinical trial costs, tax advantages, FDA user-fee benefits, and
seven years of market exclusivity in the United States following
marketing approval by the FDA. The granting of an orphan
designation request does not alter the standard regulatory
requirements and process for obtaining marketing approval. For more
information about orphan designation, please visit the FDA website
at www.fda.gov.
Sotatercept is being evaluated in two Phase 2 trials in patients
with PAH: the PULSAR trial, which completed its target enrollment
in June of this year and the SPECTRA exploratory trial, which is
currently enrolling. The Company expects to report top-line results
from the PULSAR trial during the first quarter of 2020.
Sotatercept is an investigational therapy that is not approved
for any use in any country.
About Sotatercept
Sotatercept is an investigational agent designed to be a
selective ligand trap for members of the TGF-beta superfamily to
rebalance BMPR2 signaling, which is a key molecular driver of PAH.
In preclinical studies of PAH, sotatercept reversed pulmonary
vessel muscularization and improved indicators of right heart
failure. Sotatercept is currently being evaluated in the PULSAR and
SPECTRA Phase 2 trials in PAH. For more information, please visit
www.clinicaltrials.gov.
About PAH
PAH is a rare and chronic, rapidly progressing disorder
characterized by the constriction of small pulmonary arteries and
elevated blood pressure in the pulmonary circulation. PAH results
in significant strain on 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 generally 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 familial, idiopathic, and acquired forms
of the disease.
About Acceleron
Acceleron is a 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 superfamily 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 program with ACE-083,
a locally-acting Myostatin+ agent in Phase 2 development in
facioscapulohumeral muscular dystrophy and Charcot-Marie-Tooth
disease and is conducting a Phase 2 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 factors, 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 results of any clinical trial may not be
predictive of the results or success of other clinical trials of
the same product candidate, that the development of the Company's
compounds will take longer and/or cost more than planned, that the
Company will be unable to successfully complete the clinical
development of the Company’s compounds, that the Company 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/20190909005250/en/
Acceleron Pharma Inc.
Investors: Todd James, IRC, 617-649-9393 Vice President,
Investor Relations and Corporate Communications
Ed Joyce, 617-649-9242 Director, Investor Relations
Media: Matt Fearer, 617-301-9557 Director, Corporate
Communications
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