– Oral presentation to highlight preliminary
results of ACE-083’s Phase 2 trial in patients with
facioscapulohumeral muscular dystrophy –
– Part 2 of the ongoing Phase 2 trial in
patients with facioscapulohumeral muscular dystrophy has been
initiated –
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
preliminary results from the ongoing Phase 2 trial of ACE-083 in
facioscapulohumeral muscular dystrophy (FSHD) are being presented
at the American Academy of Neurology (AAN) 70th Annual Meeting in
Los Angeles, California on April 26, 2018.
The oral presentation will include preliminary findings from
cohorts 1 and 2 in the open-label, dose escalation Part 1 of the
ongoing Phase 2 trial of ACE-083 in patients with FSHD.
Oral Presentation:
Title:
Preliminary Results from a Phase 2
Study to Evaluate ACE-083, a Local Muscle Therapeutic, in Patients
with Facioscapulohumeral Muscular Dystrophy
(Abstract #S38.001) Session: S38 “Best of” Session:
Clinical Trial Updates in Neuromuscular Disorders Presenter: Dr.
Jeffrey Statland, University of Kansas Medical Center Date:
Thursday, April 26th
Time: 8:00 a.m. PDT (Los Angeles Convention Center, Room 408B)
The ACE-083 AAN presentation is available in the “Science”
section on Acceleron’s website, www.acceleronpharma.com.
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.
The randomized, double-blind, placebo controlled Part 2 of the
FSHD study has been initiated, with results expected in the second
half of 2019. Part 2 of the CMT trial is expected to be initiated
by the end of 2018.
About ACE-083
ACE-083 is a 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
diseases such as facioscapulohumeral muscular dystrophy (FSHD) and
Charcot-Marie-Tooth (CMT) disease, in which improved muscle
strength in targeted muscles may provide a clinical benefit and
enhance quality of life.
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 pulmonary
program with a planned Phase 2 trial of 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/20180426005535/en/
Acceleron Pharma Inc.Todd James, IRC, 617-649-9393Vice
President, Investor Relations and Corporate CommunicationsOrCandice
Ellis, 617-649-9226Manager, Investor Relations and Corporate
CommunicationsMedia:BMC CommunicationsBrad Miles, 646-513-3125
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