-ACE-083 increased muscle volume of the
tibialis anterior muscle by 8.9% in healthy volunteers-
Acceleron Pharma Inc. (NASDAQ:XLRN), a clinical stage
biopharmaceutical company focused on the discovery and development
of novel therapeutic candidates that engage the body’s ability to
rebuild and repair its own cells and tissues, today announced
ACE-083 Phase 1 clinical trial results in healthy volunteers at the
14th International Congress on Neuromuscular Diseases in Toronto,
Canada. ACE-083 is designed to selectively increase muscle mass and
strength in the muscles in which the drug is administered.
Results highlighted in the poster presentation (Abstract #266,
“ACE-083, A Locally-Acting Muscle Agent, Increases Muscle Volume in
Healthy Volunteers”) showed that ACE-083 produced
statistically-significant, dose-dependent increases in muscle
volume, assessed by magnetic resonance imaging (MRI), of the
tibialis anterior muscle. At the highest dose level, ACE-083
generated a mean increase in tibialis anterior muscle volume of
8.9%.
"The new ACE-083 results in the tibialis anterior muscle are
very exciting as they confirm the significant biological activity
that we previously observed in the rectus femoris muscle in healthy
volunteers," said Matthew L. Sherman, M.D., Chief Medical Officer
of Acceleron. "The positive results in the tibialis anterior extend
our previously reported positive findings and provide us with
valuable clinical experience in a target muscle for our planned
Phase 2 trial in facioscapulohumeral muscular dystrophy (FSHD)
patients."
Acceleron intends to advance ACE-083 into a Phase 2 clinical
trial in patients with FSHD in the second half of 2016.
ACE-083 Phase 1 Results
Acceleron previously reported positive results from the Phase 1
randomized, double-blind, placebo-controlled, dose-ranging study in
healthy volunteers for trial cohorts 1 through 5 assessing muscle
volume changes of the rectus femoris muscle in the thigh. Today,
the Company presented positive data from trial cohorts 6 and 7,
which assessed muscle changes of the tibialis anterior muscle in
the shin. In each new cohort, six subjects received ACE-083 (cohort
6: 100 mg and cohort 7: 150 mg) and three subjects received
placebo. The subjects received two intramuscular doses in the
tibialis anterior muscle three weeks apart with MRI evaluations at
baseline, three weeks post last dose, and eight weeks post last
dose.
At three weeks after the last dose, ACE-083 generated
dose-dependent increases in muscle volume, assessed by MRI. In the
placebo, ACE-083 100 mg and ACE-083 150 mg cohorts, tibialis
anterior muscle volume increases were -0.1%, 5.0% (p<0.05 vs
placebo) and 8.9% (p<0.001 vs placebo), respectively.
There were no serious adverse events, dose-limiting toxicities,
or discontinuations due to adverse events. All adverse events were
grade 1 or 2, transient, and most commonly injection-site related,
and there was a similar incidence of adverse events observed in
placebo and ACE-083 treated groups.
The clinical poster is available on Acceleron's website
(www.acceleronpharma.com) under the Science tab.
About ACE-083
ACE-083 is a therapeutic candidate that acts as a ligand trap
for members in the transforming growth factor-beta (TGF-β)
superfamily involved in the regulation of muscle mass and strength.
ACE-083 has been designed to increase muscle mass and strength
selectively in the muscles into which the drug is
administered. Acceleron is developing ACE-083 for diseases in
which improved muscle strength in a specific set of muscles may
provide a clinical benefit, such as facioscapulohumeral muscular
dystrophy. The Phase 1 clinical trial of ACE-083 in healthy
volunteers has been completed. For additional information on this
clinical trial, please visit www.clinicaltrials.gov, identifier
NCT02257489.
About Acceleron
Acceleron discovers and develops novel therapies to treat a wide
range of rare diseases. Its pioneering research platform leverages
the powerful biology behind the body’s ability to rebuild and
repair its own cells and tissues. This innovative approach to drug
discovery has generated four therapeutic candidates currently in
clinical trials. The Company’s lead therapeutic candidate,
luspatercept, is being evaluated in Phase 3 studies for the
treatment of the hematologic diseases, myelodysplastic syndromes
(MDS) and beta-thalassemia under a global partnership with Celgene
Corp. Acceleron is also advancing clinical programs in the fields
of oncology and neuromuscular diseases and has a comprehensive
preclinical research effort targeting fibrotic and other serious
diseases.
For more information, please visit www.acceleronpharma.com.
Follow Acceleron on Social
Media: @AcceleronPharma and LinkedIn.
Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements about
Acceleron's strategy, future plans and prospects, including
statements regarding the development of ACE-083, the timeline for
clinical development and regulatory approval of Acceleron's
compounds, the expected timing for the reporting of data from
ongoing trials, and the structure of Acceleron's planned or pending
clinical trials. The words "anticipate," "appear," "believe,"
"continue," "could," "estimate," "expect," "forecast," "goal,"
"intend," "may," "plan," "potential," "predict," "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.
Each forward-looking statement is subject to risks and
uncertainties that could cause actual results to differ materially
from those expressed or implied in such statement. Applicable risks
and uncertainties include the risks that preclinical testing of
Acceleron's compounds and data from clinical trials may not be
predictive of the results or success of ongoing or later clinical
trials, that data may not be available when Acceleron expects it to
be, that Acceleron will be unable to successfully complete the
clinical development of Acceleron's compounds, that the development
of Acceleron's compounds will take longer or cost more than
planned, that Acceleron may be delayed in initiating or completing
any clinical trials, and that Acceleron's compounds will not
receive regulatory approval or become commercially successful
products.
Other risks and uncertainties include those identified under the
heading "Risk Factors" included in Acceleron's Annual Report on
Form 10-K which was filed with the Securities and Exchange
Commission (SEC) on February 25, 2016, and other filings that
Acceleron has made and may make with the SEC in the future. The
forward-looking statements contained in this press release reflect
Acceleron's current views with respect to future events, and
Acceleron does not undertake and specifically disclaims any
obligation to update any forward-looking statements.
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version on businesswire.com: http://www.businesswire.com/news/home/20160708005127/en/
Acceleron Pharma Inc:Todd James, IRC, 617-649-9393Senior
Director, Investor Relations and Corporate
CommunicationsorMedia:BMC Communications LLCBrad Miles,
646-513-3125
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