– ACE-083 did not achieve statistically
significant improvements in functional endpoints relative to
placebo –
– Acceleron to discontinue development of
ACE-083 in facioscapulohumeral muscular dystrophy –
– Topline results from the Phase 2 trial of
ACE-083 in Charcot-Marie-Tooth disease expected in Q1 2020 -
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 treatment with ACE-083 in patients with facioscapulohumeral
muscular dystrophy (FSHD) did not achieve functional secondary
endpoints in the Phase 2 trial.
Although ACE-083 demonstrated a robust, statistically
significant increase in mean total muscle volume, the primary
endpoint of the trial, the increase failed to translate to
statistically significant improvements in functional tests. As a
result, Acceleron will not conduct further clinical trials of
ACE-083 in FSHD.
“We are certainly disappointed with these results. As we have
stated consistently, for ACE-083 to become an important new therapy
for patients with FSHD, it would have to deliver a meaningful
functional benefit on top of an ability to grow muscle,” said Habib
Dable, President and Chief Executive Officer of Acceleron.
“Unfortunately, in this case, the data show no evidence of such a
benefit and, therefore, do not support further development of
ACE-083 for FSHD. We’re grateful to the patients, families,
caregivers, and investigators who participated in this
research.”
Dable added: “We now look toward the first quarter of next year,
when we expect topline results from the placebo-controlled Phase 2
trial of ACE-083 in patients with Charcot-Marie-Tooth disease—a
neuromuscular disorder of different pathophysiology.”
In this Phase 2 trial in patients with FSHD, ACE-083 was
generally well tolerated. Adverse events were mostly mild to
moderate (Grade 1 or 2) and injection-site related. Acceleron
expects to present results at a future medical meeting.
ACE-083 is an investigational therapy that is not approved for
any use in any country.
FSHD Phase 2 Trial Design
The two-part Phase 2 clinical trial was designed to evaluate
ACE-083 in FSHD patients with muscle weakness in the biceps brachii
(BB) and the tibialis anterior (TA), a muscle in the lower leg
involved in foot dorsiflexion (raising the foot at the ankle). Part
1 was an open-label, dose-escalation study, with ACE-083
administered by injection into the BB or TA muscle to evaluate
safety and increases in muscle volume over a 3-month treatment
period. Part 2 was a randomized, double-blind, placebo-controlled
study using the optimal dose level selected in Part 1. A total of
56 patients were randomized in Part 2 to receive either placebo or
ACE-083 and were evaluated for changes in muscle volume, fat
fraction, strength, function, quality of life, 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.
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 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
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 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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version on businesswire.com: https://www.businesswire.com/news/home/20190916005830/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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