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 plans to
deliver oral and poster presentations on ACE-083 from the ongoing
Phase 2 trial in patients with Charcot-Marie-Tooth (CMT) disease at
the Peripheral Nerve Society (PNS) annual meeting held in
Baltimore, Maryland, July 22-25, 2018.
Oral
presentation
Title:
Preliminary Phase 2 Results for
ACE-083, Local Muscle Therapeutic, in Patients with CMT1 and
CMTX
Session: Oral Abstract Session II Date: Monday, July 23rd Time:
9:00 - 9:15 a.m. EDT (Renaissance Baltimore Harborplace Hotel)
Poster
presentation
Title:
Preliminary Phase 2 Results for
ACE-083, Local Muscle Therapeutic, in Patients with CMT1 and
CMTX
Session: Poster Session II Date: Monday, July 23rd Time: 1:00 -
2:00 p.m. EDT (Renaissance Baltimore Harborplace Hotel)
The ACE-083 clinical presentations will be available immediately
following the presentation at the conference in the “Science”
section on Acceleron’s website, www.acceleronpharma.com.
ACE-083 is currently being evaluated in two Phase 2 trials: one
in facioscapulohumeral muscular dystrophy (FSHD) and one in CMT
disease. The final Part 1 results from both Phase 2 trials are
expected in the second of half of 2018. Part 2 of the CMT trial is
expected to be initiated by the end of 2018.
CMT Phase 2 Trial Design
The two-part Phase 2 clinical trial is designed to evaluate
ACE-083 in CMT patients with muscle weakness in the tibialis
anterior (TA), a muscle in the lower leg involved in foot
dorsiflexion (raising the foot at the ankle). Part 1 is an
open-label, dose-escalation study, with ACE-083 administered by
injection into the TA muscle once every 3 weeks in up to 18
patients to evaluate safety and increases in muscle volume over a
3-month treatment period. Part 2 is a randomized, double-blind,
placebo-controlled study using the optimal dose level selected in
Part 1. A total of 40 patients will be randomized in Part 2 to
receive either placebo or ACE-083 and will be evaluated for changes
in muscle volume, fat fraction, strength, function and safety over
a 6-month primary treatment period, followed by a 6-month
open-label treatment period.
For additional information about this clinical trial, please
visit www.clinicaltrials.gov, identifier NCT03124459.
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.
About Charcot-Marie-Tooth Disease (CMT)
CMT is one of the most common inherited neurologic diseases
estimated to affect more than 125,000 people in the United States.
The primary clinical manifestations of CMT include muscle weakness
in the lower legs and arms. The lower leg muscle weakness can
result in foot drop leading to a high-stepped gait and frequent
tripping or falls. The disease is typically diagnosed by the
presence of a characteristic pattern of muscle weakness, nerve
conduction studies, and genetic testing. There are no FDA approved
drug therapies for CMT.
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
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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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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