- Mean total muscle volume increases of more
than 12% seen in the tibialis anterior –
- Company plans to initiate Part 2 of the CMT
Phase 2 trial in the third quarter of 2018 –
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 positive
preliminary results from Part 1 of the Phase 2 clinical trial with
ACE-083 in patients with Charcot-Marie-Tooth (CMT) disease at the
Peripheral Nerve Society (PNS) annual meeting held in Baltimore,
Maryland. CMT is one of the most common inherited neurological
diseases and is associated with significant focal muscle weakness.
The Company plans to initiate Part 2 of the ACE-083 CMT Phase 2
trial in the third quarter of 2018.
“Preliminary Phase 2 results of ACE-083 in patients with CMT
show robust mean increases in total and contractile muscle volume,
reductions in fat fraction, and an encouraging safety profile,”
said Robert K Zeldin, M.D., Chief Medical Officer of Acceleron. “We
now look forward to initiating the randomized, placebo-controlled
portion of the Phase 2 trial in which we will evaluate ACE-083’s
potential to improve function over a six-month treatment
period.”
Part 1 of the trial was an open-label, dose-escalation study
that enrolled a total of 18 patients (in three cohorts of six
patients each) with CMT1 or CMTX who received ACE-083 at dose
levels of 150 mg, 200 mg, or 240 mg. ACE-083 was administered by
injection into the tibialis anterior (TA) muscle bilaterally once
every three weeks for three months to evaluate safety and increases
in muscle volume. The TA, which is located in the lower leg, is the
primary muscle responsible for ankle dorsiflexion, or the ability
to lift the front of the foot when taking a step. TA weakness can
result in foot drop and increased risk of falls.
Muscle volume was measured by magnetic resonance imaging (MRI)
three weeks after the last injection of ACE-083. Muscle volume and
fat fraction are represented as the changes from baseline averaged
for each side.
- Mean total muscle volume (TMV)
increases ranged from 12.6% to 14.2%.
- Mean absolute decreases (improvement)
in fat fraction ranged from 1.7% to 3.5%.
In addition, treatment with ACE-083 resulted in mean increases
from baseline in contractile muscle volume (an MRI-derived
calculation to measure viable, functional muscle volume as part of
the TMV), ranging from 15.8% to 19.6%.
The most common adverse events—injection-site reactions, muscle
spasms, and myalgia—were mild or moderate (grades 1-2).
“There are currently no FDA-approved therapies for patients with
CMT with muscle weakness. The muscle volume and fat fraction
changes demonstrated in Part 1 of the ACE-083 trial are
encouraging,” said Florian P. Thomas, M.D., neurologist and
principal investigator at Hackensack Meridian School of Medicine at
Seton Hall University. “We’re hopeful that ACE-083 may become an
important option for patients.”
The 2018 PNS ACE-083 Part 1 oral and poster presentations are
available in the “Science” section on Acceleron’s
website, www.acceleronpharma.com.
The double-blind, placebo-controlled Part 2 of the CMT Phase 2
trial will enroll approximately 40 patients who will be randomized
(1:1) to receive either placebo or ACE-083. Patients will be
evaluated for changes in muscle volume, fat fraction, strength,
function and safety over a six-month primary treatment period,
followed by a six-month open-label treatment period. Preliminary
results are expected by the end of 2019.
For additional information on this clinical trial, please visit
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 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 Charcot-Marie-Tooth Disease (CMT)
CMT is one of the most common inherited neurologic diseases. It
is 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 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/20180723005176/en/
For Acceleron:Investors: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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