ACE-083 can prevent muscle shrinkage or atrophy that cause the debilitating symptoms of CMT disease by blocking the transforming growth factor (TGF) beta, a signaling protein that triggers the reduction of muscle fibers through a series of reactions. When injected directly into the affected muscles, ACE-083 has the potential to increase muscle strength, helping to delay CMT disease progression.
Encouraging preliminary findings have been reported from a Phase 2 trial (NCT03124459) evaluating the efficacy of ACE-083 in CMT patients. The researchers reported an increase in muscle and a favorable safety profile in patients treated with ACE-083.
The study is ongoing and still recruiting patients. The team hopes to see functional improvement in the second part of the trial, adding to the promising results already reported.
According to the release, initial results for the second part of the study are expected by the end of 2019.
“We’re pleased that the FDA has granted this designation for ACE-083,” said Robert K. Zeldin, MD, chief medical officer of Acceleron.
“Patients with CMT currently have no approved therapies. To date, results from our Phase 2 trials have shown that patients treated with ACE-083 experience robust increases in muscle volume. If our ongoing clinical studies show that ACE-083 also improves functional outcomes and confirm the favorable safety profile observed thus far, the Fast Track process could help us work with the FDA to deliver it to patients as quickly as possible,” Zeldin added.
In addition to CMT, Acceleron is also testing the effectiveness of ACE-083 in patients with facioscapulohumeral muscular dystrophy (FSHD), another condition in which patients would benefit from an increase in muscle strength. Earlier this year, ACE-083 received fast track designation for the treatment of FSHD.
The Phase 2 trial (NCT02927080) evaluating ACE-083 in FSHD patients is also a two-part trial, and the results are anticipated in the second half of 2019.
The fast track designation expedites the FDA’s therapy review process by enabling frequent communication between the company and the FDA, especially in the initial stages of development. It helps resolve any issues at the earliest stage possible, which increases the chances of getting the therapy approving sooner, making it available to patients in need.