ACE-083 is the lead product candidate in our neuromuscular therapeutic program. It is a locally active agent that may be useful for diseases of focal muscle loss such as muscular dystrophies, such as facioscapulohumeral muscular dystrophy (FSHD) and Charcot-Marie-Tooth disease (CMT). The U.S. FDA has granted ACE-083 Fast Track and Orphan Drug designations in FSHD and CMT. ACE-083 is designed to increase strength and function in specific target muscles for improved patient function and quality of life.
ACE-083 is designed to bind to and inhibit select proteins in the TGF-beta protein superfamily that negatively regulate (reduce) muscle growth, such as activins and myostatin (GDF8). This “Myostatin +” approach, is believed to increase muscle mass and strength in the muscle where the drug is administered. Untreated muscles or other organs are not affected, reducing the potential for systemic side effects.
Based on encouraging Part 1 results from our two-part Phase 2 trials in FSHD and CMT, we have initiated Part 2 of both of the trials.
ACE-083 is an investigational therapy that is not approved for any use in any country.
There are no FDA approved therapies for FSHD, one of the most common muscular dystrophies, a progressive, degenerative and often debilitating disorder that affects about 20,000 people in the U.S. Many of these patients have trouble walking and running, and develop upper arm muscle weakness, ultimately limiting their ability to perform daily tasks, such as eating or getting dressed. It is estimated that 20 percent of people with FSHD require the use of a wheelchair at least some of the time. Current management for the disease is primarily limited to physical and occupational therapy and certain surgical interventions.
We are conducting a trial of ACE-083 as a potential treatment for CMT, the most common inherited neurological disorder. CMT, for which there is currently no FDA-approved treatment, affects approximately 1 in 2,500—or more than 100,000—people in the U.S.
CMT is a group of disorders that impact certain peripheral nerves, resulting in muscle atrophy and weakness. Symptoms can begin in adolescence or early adulthood with initial signs of weakness in the lower leg muscles, affecting the patient’s ability to walk and causing frequent falls. As the disease progresses, muscle atrophy may occur in the feet and hands. CMT is currently managed with physical and occupational therapy, leg braces, and foot surgery.