The first patient has been dosed in a Phase 2 clinical trial of ACE-083as a treatment for the muscle impairment inCharcot-Marie-Tooth disease, according to the therapys developer,Acceleron Pharma.
Acceleron is already evaluating ACE-083 in another Phase 2 trial this one as a treatment forfacioscapulohumeral muscular dystrophy.
People diagnosed with CMT currently have no drug therapy options to address the major consequences of their disease, such as impaired walking and falls due to progressive muscle weakness in the lower leg, Dr. Colin Quinn, the new Phase 2 trials principal investigator,said in a press release. He is anassistant professor of clinical neurology at the University of Pennsylvanias Perelman School of Medicine.
ACE-083 has the potential to increase muscle growth and strength in the lower leg muscles we are targeting, and could improve patients ability to walk, Quinn said.
The therapy inhibits agents that impair muscle growth and strength.
Acceleron is developing it for muscular dystrophies with what scientists call focal muscle loss. That kind of loss occurs in a muscle or group of muscles in a specific part of the body.
A Phase 1 clinical trial (NCT02257489) showed that injecting ACE-083 into the legs of healthy volunteers increased their muscle volume. Researchers made the injections inthe rectus femoris a muscle in the upper leg or tibialis anterior a muscle in the lower leg that involves the ankle. The injections led to a 14.5 percent increase in muscle volume in the upper leg and an 8.9 percent increase in the lower leg.
No serious toxicities related to treatment were reported, indicating that ACE-083 was safe.
The positive results prompted Acceleron to move forward with its plans for the two-part Phase 2 clinical trial of ACE-083 in CMT. The study (NCT03124459) will assess ACE-083s ability to reduce weakness in the tibialis anterior, the legs largest muscle.
Acceleron hopes to recruit 42adults with CMT type1 or X-linked CMT for the trial, which will be held at 18 locations in the United States.
In the first part of the trial, 18 patients will receive ascending doses of ACE-083 in their tibialis anterior muscles once every three weeks for up to five doses.
In the second part, 24 patients will randomly receive the optimal dose level identified in Part 1 of the trial, or a placebo. Thesame treatment regimen used in Part 1 will apply to the second stage as well.
Researchers will assess the drugs effectiveness in the Part 2 portion of the trial by looking at changes in muscle volume, strength, and function. They will also evaluate its safety.
We are proud to have advanced ACE-083 into a second Phase 2 clinical trial, said Dr. Matthew Sherman, Accelerons executive vice president and chief medical officer. We designed our clinical development strategy for ACE-083 to explore its activity in diseases with weakness in specific muscles due to an underlying neurological or muscle disorder. With Phase 2 trials now under way in both CMT andfacioscapulohumeral muscular dystrophy,we will be able to evaluate ACE-083s effect on both muscle strength and function across a range of neuromuscular diseases.
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