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 that the United
States Food and Drug Administration (FDA) has granted orphan drug
designation for ACE-083, the Company’s locally acting “Myostatin+”
muscle agent, for the treatment of patients with
facioscapulohumeral muscular dystrophy (FSHD).
“We are pleased to receive orphan drug designation for ACE-083,
which has shown the potential to address an area of high unmet
medical need,” said Robert K. Zeldin, M.D., Chief Medical Officer
of Acceleron. “We believe that ACE-083 could become an important
new treatment for patients with FSHD whose muscle weakness
negatively affects their functional abilities. We presented
positive preliminary data from Part 1 of our Phase 2 trial in
patients with FSHD earlier this year and look forward to sharing
Part 2 results later next year.”
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.
In May of 2018, FDA granted ACE-083 Fast Track designation for
FSHD, which could facilitate its development and potentially
expedite its review. ACE-083 is currently being evaluated in two
Phase 2 trials: one in FSHD and one in Charcot-Marie-Tooth (CMT)
disease. The final Part 1 results from both Phase 2 trials are
expected in the second of half of 2018. Preliminary results from
Part 2 of the trial in patients with FSHD are expected in the
second half of 2019.
About ACE-083
ACE-083 is a locally-acting therapeutic candidate, based on the
naturally-occurring protein follistatin, which utilizes the
"Myostatin+" approach to inhibit multiple TGF-beta ligands. It is
designed to have a concentrated effect along targeted muscles to
maximize growth and strength selectively in the muscles into which
the drug is administered. Acceleron is developing ACE-083 for
disorders such as Charcot-Marie-Tooth (CMT) disease and
facioscapulohumeral muscular dystrophy (FSHD), in which improved
muscle strength in target muscles may provide a clinical benefit
and enhance quality of life. For more information, please visit
www.clinicaltrials.gov.
About Facioscapulohumeral Muscular Dystrophy (FSHD)
FSHD is a rare genetic muscle disorder affecting approximately
20,000 people in the United States for which there are currently no
approved treatments. The primary clinical presentation of FSHD is
debilitating skeletal muscle weakness and loss. The symptoms of
FSHD develop in a descending pattern, beginning with the face and
upper body and progressing to the lower body in a "muscle by
muscle" fashion. The disease is typically diagnosed by a
characteristic pattern of muscle weakness and other clinical
symptoms, as well as through genetic testing.
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 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/20180712005183/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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