Acceleron Presents Preclinical Data for ACE-083 & ACE-2494 at the 20th International Annual Congress of the World Muscle Soci...
01 Octubre 2015 - 6:30AM
Business Wire
-- First presentation of data for new systemic
muscle agent, ACE-2494, demonstrates substantial increases in
muscle mass --
Acceleron Pharma Inc. (NASDAQ: XLRN), a clinical stage
biopharmaceutical company focused on the discovery, development and
commercialization of novel therapeutic candidates that regulate
cellular growth and repair, today reported results from preclinical
studies of ACE-083, a locally-acting muscle therapeutic, and from
ACE-2494, a systemic muscle therapeutic and a new Acceleron program
first introduced today. Data from both programs were presented at
the 20th International Annual Congress of the World Muscle Society
(WMS) held in Brighton, United Kingdom.
“We are excited to present data for the first time from the
ACE-2494 program. This molecule generates substantial increases in
muscle mass and we are encouraged by its potential to become yet
another clinical candidate for Acceleron,” said Ravi Kumar, Ph.D.,
Chief Scientific Officer at Acceleron. “We aim to build a portfolio
of distinct molecules in the area of muscle biology, leveraging
Acceleron’s deep experience and understanding of the TGF-beta
superfamily’s role in regulating muscle mass and strength.”
ACE-2494
ACE-2494 is a ligand trap that inhibits the key negative
regulators of skeletal muscle, thereby systemically increasing
skeletal muscle mass and strength.
- In mice, ACE-2494 generated dose
dependent increases in muscle mass at a magnitude comparable to the
powerful effects of soluble ActRIIB fusion proteins. After 4 weeks
of treatment, ACE-2494 (10 mg/kg twice weekly) generated
substantial increases in muscle mass: 41% (rectus femoris), 53%
(gastrocnemius), and 87% (pectoralis).
- ACE-2494 is engineered to achieve
muscle enhancement by selectively inhibiting negative regulators of
skeletal muscle, while avoiding the effects on regulators of the
vasculature that have been observed with soluble ActRIIB fusion
proteins.
ACE-083
ACE-083 is a locally-acting therapeutic candidate that has been
designed to increase skeletal muscle mass and strength selectively
in only the muscles into which the drug is administered. In this
preclinical study, ACE-083 demonstrated increases in both muscle
mass and strength.
- In mice, ACE-083 was administered by
injection into the right tibialis anterior (TA) muscle twice per
week for 3 weeks.
- ACE-083 produced significant increases
in muscle mass of roughly 75% in the injected muscle with no
observed effect on either the uninjected contralateral muscle or on
whole body mass. These results are consistent with a local effect
only in the injected muscle.
- Increases in muscle mass were
associated with a significant increase in muscle force and power of
roughly 40%. Changes in muscle strength were due to larger fiber
size rather than altering the kinetic properties of the
muscle.
These programs, including preliminary results from the phase 1
trial of ACE-083, will be discussed at Acceleron’s R&D Day on
Friday, October 23, 2015.
Both posters are available on Acceleron’s website
(www.acceleronpharma.com) under the Publications section.
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 and certain other forms of
muscular dystrophy. ACE-083 is currently in a phase 1 clinical
trial in healthy volunteers. For additional information on this
clinical trial, please visit www.clinicaltrials.gov, identifier
NCT02257489.
About ACE-2494
ACE-2494 is a therapeutic candidate that traps ligands in the
Transforming Growth Factor-Beta (TGF-β) superfamily involved in the
regulation of muscle mass and strength. ACE-2494 has been designed
to selectively bind several key negative regulators of muscle mass
and thereby increase muscle mass and strength.
About Acceleron
Acceleron is a clinical stage biopharmaceutical company focused
on the discovery, development and commercialization of novel
therapeutic candidates that regulate cellular growth and repair.
The company is a leader in understanding the biology of the
Transforming Growth Factor-Beta (TGF-β) protein superfamily, a
large and diverse group of molecules that are key regulators in the
growth and repair of tissues throughout the human body, and in
targeting these pathways to develop important new medicines.
Acceleron has built a highly productive R&D platform that has
generated innovative clinical and preclinical therapeutic
candidates with novel mechanisms of action. These therapeutic
candidates have the potential to significantly improve clinical
outcomes for patients with cancer and rare diseases.
For more information, please visit www.acceleronpharma.com.
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Acceleron Pharma Inc.Todd James, 617-649-9393Senior Director,
Corporate CommunicationsorMedia:Suda Communications LLCMaureen L.
Suda, 585-387-9248
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