Acceleran presents preclinical data for muscle therapeutics ACE-083 and ACE-2494

Acceleron reported results from preclinical studies of ACE-2494, a systemic muscle therapeutic, and from ACE-083, a locally-acting muscle therapeutic, at the 20th International Annual Congress of the World Muscle Society (WMS).

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 is currently being assessed in a Phase 1 clinical trial

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 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 fibre size rather than altering the kinetic properties of the muscle.

Acceleron has said it 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.