Acceleron Abandons CMT Program

Cambridge, Massachusetts-based Acceleron Pharma announced that its Phase II clinical trial of ACE-083 in patients with Charcot-Marie-Tooth disease (CMT) failed to show functional improvement. The company indicated the drug did show a statistically significant increase in mean total muscle volume, which was the primary endpoint of the trial, but it didn’t lead to statistically significant improvements in function or quality of life secondary endpoints. Acceleron says it is discontinuing development of the drug. CMT is one of the most common inherited neurological disorders. It is made up of a group of disorders that affect peripheral nerves, which is to say, those outside the brain and spinal cord. It affects motor and sensory nerves and includes weakness of the foot and lower leg muscles. There are often foot deformities, such as high arches and hammertoes because of weakness of the small muscles in the feet. It affects about one in 2,500 people in the U.S. ACE-083 was the company’s lead product candidate for its neuromuscular therapeutic program. It is designed to bind to and inhibit specific proteins in the TGF-beta protein superfamily that reduce muscle growth, such as activins and myostatins (GDF8). “Unfortunately, over the course of multiple clinical trials, our myostatin-plus hypothesis has not resulted in the functional outcomes necessary to provide clinically meaningful benefits for patients with neuromuscular diseases,” said Habib Dable, president and chief executive officer of Acceleron. “We wish to thank all the patients, families, caregivers, and investigators for their support and participation in this research. I also want to acknowledge our team’s hard work and commitment to executing robust Phase II trials that have provided us the data necessary to make the difficult but informed investment decision to discontinue the program.” The company plans to refocus its resources on hematology with its Reblozyl in anemia and on pulmonary with sotatercept in pulmonary arterial hypertension (PAH).