CAMBRIDGE, Mass.--(BUSINESS WIRE)--Acceleron Pharma Inc. (NASDAQ:XLRN), a clinical stage biopharmaceutical
company focused on the discovery, development and commercialization of
innovative therapeutics to treat serious and rare diseases, today
announced that in the second half of 2017 it plans to initiate a Phase 2
clinical trial of ACE-083, the Company’s locally acting muscle agent,
for the treatment of patients with Charcot-Marie-Tooth disease (CMT),
one of the most common inherited neurologic diseases leading to focal
muscle weakness.
“We are pleased to expand our clinical development program for ACE-083
into a second neuromuscular disease”
“We are pleased to expand our clinical development program for ACE-083
into a second neuromuscular disease,” said Matthew Sherman, M.D.,
Executive Vice President and Chief Medical Officer at Acceleron. “We
believe ACE-083 can strengthen the targeted leg muscles in CMT patients
and thereby improve their ability to walk and avoid falls. We look
forward to hosting the educational webinar during which we will describe
CMT and our enthusiasm for the potential of ACE-083 to address the
substantial unmet medical needs of patients.”
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 phase of the
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 phase using the optimal dose level
selected in Part 1. A total of 24 patients will be randomized in Part 2
to receive either placebo or ACE-083 and will be evaluated for increases
in muscle volume, strength, function and safety over a 3-month treatment
period.
Webinar
The Company will host an educational webinar providing an overview of
CMT and a detailed review of the Company's Phase 2 clinical trial. The
webinar will be led by neuromuscular disease expert David Walk, M.D.,
Director of the multidisciplinary CMT and ALS clinics at the University
of Minnesota Medical Center Fairview, and Acceleron management, and will
take place on Monday, April 3, 2017 at 11:00 a.m. EDT.
Conference Call and Webinar Details
Date:
Monday, April 3, 2017
Time:
11:00 a.m. (EDT)
Participants can access the live conference call by
dialing 877-312-5848 (domestic) or 253-237-1155 (international) and
refer to the Acceleron CMT Webinar.
The live webinar can be accessed on the Investors page of the Company's
website at .
A replay of the webinar will be available approximately two hours after
the event on the Acceleron website.
About ACE-083
ACE-083 is a therapeutic candidate that acts as a ligand trap for
members in the transforming growth factor-beta (TGF-ß) 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 such as facioscapulohumeral muscular dystrophy and
Charcot-Marie-Tooth disease in which improved muscle strength in target
muscles may provide a clinical benefit.
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 and a
high-stepped gait leading to frequent tripping or falls. The disease is
typically diagnosed by the presence of a characteristic pattern of
muscle weakness and other clinical signs and symptoms, as well as
through genetic testing. There are no FDA approved drug therapies for
CMT.
About Acceleron
Acceleron is a clinical stage biopharmaceutical company focused on the
discovery, development and commercialization of innovative therapeutics
to treat serious and 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 approach to drug discovery has
generated four therapeutic candidates that are 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 .
Follow Acceleron on social media: @AcceleronPharma and
LinkedIn.
Cautionary Note on 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 compound ACE-083, the
timeline for clinical development and regulatory approval of the
ACE-083, the expected timing for the reporting of data from ongoing
trials, and the structure of the Company'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 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 data
may not be available when the Company expects it to be, that the Company
will be unable to successfully complete the clinical development of the
Company's compounds, that the development of the Company's compounds
will take longer or cost more than planned, 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.
Other risks and uncertainties include those identified under the heading
"Risk Factors" included in the Company's Annual Report on Form 10-K
which was filed with the Securities and Exchange Commission (SEC)
on March 1, 2017, 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 reflect the Company's current views with
respect to future events, and the Company does not undertake and
specifically disclaims any obligation to update any forward-looking
statements.
Acceleron Pharma Inc.Todd James, IRC, 617-649-9393Senior
Director, Investor Relations and Corporate CommunicationsorMedia:BMC
CommunicationsBrad Miles, 646-513-3125