Accelreon Pharma's ACE-031Shown to Increase Muscle Mass in Preclinical Studies
LUCCA, ITALY, May 14, 2007-Acceleron Pharma, Inc., a
biopharmaceutical company developing therapeutics for the treatment
of musculoskeletal, metabolic and cancer-related diseases, today
announced results of a preclinical study with ACE-031 that
demonstrated the ability of the compound to increase muscle mass
and strength in normal mice and in murine models of muscular
dystrophy. ACE-031 inhibits negative regulators of muscle to
increase muscle mass and strength as a potential treatment for
neuromuscular diseases and cancer-related muscle loss. The data was
presented on Sunday, May 13 at the Gordon Research Conference on
Myogenesis in Lucca, Italy. ³
Acceleron is encouraged by the significant outcomes and positive
data generated by ACE-031 spanning numerous preclinical models of
muscle loss², said Jas Seehra, Ph.D., Chief Scientific Officer
of Acceleron. ³ACE-031 and related Acceleron compounds
consistently demonstrate substantial increases in skeletal muscle
mass and strength.² ACE-031, a protein therapeutic based on
the activin receptor type IIB (ActRIIB), is the first of several
compounds Acceleron is developing for the treatment of diseases
involving the loss of muscle mass, strength and function. ActRIIB
binds to myostatin (GDF-8) and other negative regulators of muscle
mass and strength. Over-expression of myostatin has been shown to
cause a loss in muscle mass and strength, and inhibition of
myostatin results in the selective increase in skeletal muscle mass
and strength. In preclinical models, ACE-031 increased skeletal
muscle mass and strength in disease models of amyotrophic lateral
sclerosis (ALS), muscular dystrophy, glucocorticoid-induced muscle
loss and age-related muscle loss (sarcopenia).
ACE-031 offers the potential for an entirely new type of therapy
for the treatment of various muscle wasting conditions. ³The
clinical potential of ACE-031 to selectively increase skeletal
muscle will offer a growing number of patients an excellent
opportunity to benefit from a promising therapy for the otherwise
untreatable muscle loss associated with their disease,² said
Matthew L. Sherman, M.D., Chief Medical Officer of Acceleron.
³
Based on the comprehensive preclinical program currently underway,
Acceleron is working toward advancing ACE-031 into Phase 1 clinical
trials later this year.² Study Findings The presentation,
entitled ³Treatment with a Soluble Activin Receptor IIB Causes
Increased Muscle Mass and Strength in mdx Mice² discussed data
from preclinical studies in normal mice and in a mouse model of
muscular dystrophy. These studies show a substantial increase in
muscle mass and a dose-dependent increase in lean body mass in
treated animals. These effects are significant in just two weeks
following start of therapy and increase further as treatment
continues. In the mdx murine model of muscular dystrophy, treated
animals showed increases in muscle mass which translated into
significantly increased muscle strength.
About Muscle Loss
Loss of muscle mass and strength is a serious clinical consequence
of several types of diseases and conditions: (1) neuromuscular
diseases, such as ALS and muscular dystrophy, in which patients
suffer from the loss of muscle strength and function which
ultimately leads to respiratory failure and death (2)
cancer-cachexia, in which patients can lose muscle mass and body
weight as a result of both their cancer treatments (hormone
deprivation therapy causes loss of muscle) and the cancer itself
(3) other cachexias, in which the muscle loss is associated with
chronic diseases such as congestive heart failure, chronic
obstructive pulmonary disease, and chronic kidney disease and, (4)
age-related muscle loss, known as sarcopenia, in which generalized
muscle loss and frailty leads to frequent falls and morbidity and
mortality from broken bones. A drug candidate that would increase
muscle mass and strength is expected to produce a major clinical
benefit across this broad range of serious medical conditions in
which there are currently no safe and effective therapies that
selectively increase skeletal muscle mass and strength.
About Acceleron Pharma
Acceleron is a privately held biopharmaceutical company committed
to discover, develop, manufacture and commercialize novel
biotherapeutics that modulate the growth of bone, muscle, fat and
the vasculature to treat musculoskeletal, metabolic and
cancer-related diseases. Acceleron¹s scientific approach takes
advantage of its unique insight on the regenerative powers of two
protein families: the Growth and Differentiation Factor (GDF) and
Bone Morphogenetic Protein (BMP) families. The company¹s lead
program is an anabolic bone agent being developed to reverse bone
loss in diseases such as osteoporosis and cancer. Additionally, the
company is advancing several preclinical programs that increase
muscle mass, control angiogenesis and inhibit fat accumulation.
Acceleron utilizes proven biotherapeutic technologies and
capitalizes on the company¹s internal GMP manufacturing
capability to rapidly and efficiently advance its therapeutic
programs. The investors in Acceleron are Advanced Technology
Ventures, Flagship Ventures, OrbiMed Advisors, Polaris Ventures,
Sutter Hill Ventures and Venrock.
Contact: Steven Ertel, Vice President, Business Development,
Acceleron Pharma, tel: 617-520-1334 and Paul Kidwell (Media) Suda
Communications, tel: 617-296-3854 Paul Kidwell 617.296.3854: Office
617.680.1088: Mobile
Posted: May 2007
