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Acceleron Presents Preclinical Data for ACE-083 and ACE-2494 at the 20th International Annual Congress of the World Muscle Society

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CAMBRIDGE, Mass.--(BUSINESS WIRE)--

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.

Acceleron Pharma Inc.
Todd James, 617-649-9393
Senior Director, Corporate Communications
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Media:
Suda Communications LLC
Maureen L. Suda, 585-387-9248





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