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 Charcot-Marie-Tooth disease (CMT)—an indication for which the U.S. FDA has granted ACE-083 Fast Track and Orphan Drug designations. 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.
We anticipate results from Part 2 of our Phase 2 trial in CMT in 1H 2020.
ACE-083 is an investigational therapy that is not approved for any use in any country.
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.