- Second Phase 2 study for ACE-083 expected to
begin 2H 2017 -
- Company to host educational webinar with
leading clinical researcher on April 3rd at 11:00 a.m. EDT -
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,” 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 www.acceleronpharma.com.
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-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
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 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 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.
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version on businesswire.com: http://www.businesswire.com/news/home/20170316005230/en/
Acceleron Pharma Inc.Todd James, IRC, 617-649-9393Senior
Director, Investor Relations and Corporate
CommunicationsorMedia:BMC CommunicationsBrad Miles,
646-513-3125
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