Acceleron  

ACE-083

for Muscle Loss

ACE-083 is an investigational protein therapeutic that has been designed for local administration to increase muscle mass and strength in specific muscles and muscle groups.  Acceleron is developing ACE-083 for diseases in which improved muscle strength may provide a clinical benefit, such as facioscapulohumeral muscular dystrophy (FSHD) or certain other forms of muscular dystrophy.

 

  • 650

    Number of muscles in the human body

Mechanism of Action

Select proteins in the TGF-β protein superfamily normally function to modulate muscle growth.  ACE-083 has been designed to block those proteins and, in doing so, can increase muscle mass and strength.  ACE-083 has been engineered to exert this action only within the muscle or muscle group treated. In preclinical animal studies, ACE-083 has shown selective and dose-dependent increases in muscle mass in treated muscles with no effects in untreated muscles.

Disease Overview

There are a number of rare diseases where, unlike other more generalized muscle-wasting diseases, only a limited number of muscles are impacted and suffer a loss of size and function. Patients with these conditions, such as facioscapulohumeral muscular dystrophy (FSHD) or certain other forms of muscular dystrophy, suffer from a severe clinical course that may ultimately lead to the inability to walk or perform other daily tasks, such as eating or getting dressed, without assistance.

There is currently no effective treatment for many such diseases involving the loss of muscle mass and strength.

 

Clinical Need

ACE-083 is designed to promote muscle growth selectively in those muscles in which the drug is administered.  In conditions where muscle loss or atrophy is limited to specific muscle groups, a locally-acting agent that increases muscle size and function could provide significant clinical benefit.

Clinical Trials

Acceleron is conducting a phase 1 clinical trial of ACE-083 in healthy volunteers.  For more information on the clinical trial, please click here.