Phase 1 Trial of Acceleron’s CMT Therapy ACE-083 Shows Positive Results in Healthy People

Phase 1 Trial of Acceleron’s CMT Therapy ACE-083 Shows Positive Results in Healthy People
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ACE-083, an investigative therapy for the treatment of Charcot-Marie-Tooth (CMT) disease, was well tolerated and resulted in increased targeted muscle growth in healthy volunteers in a Phase 1 clinical trial, results show.

The study, “Locally Acting ACE-083 Increases Muscle Volume in Healthy Volunteers,” was published in the journal Muscle & Nerve.

Acceleron‘s ACE-083 is a locally-acting drug that binds to a protein called myostatin and other muscle regulators, and works by increasing the growth of muscles. One of the benefits of ACE-083 is that it acts in a targeted manner in muscles where it is administered.

ACE-083 has also been shown to increase muscle mass and strength in mice with neuromuscular disease.

Researchers conducted a Phase 1 randomized, placebo-controlled trial (NCT02257489) to test single and multiple doses of ACE-083 as a local injection into selected skeletal muscles of healthy postmenopausal women ages 45-75.

Researchers tested three single doses 50 mg, 100 mg, and 200 mg and multiple doses of 100 mg, 150 mg, and 200 mg doses.

The study’s primary objective — its endpoint  was to assess the therapy’s safety and tolerability. Additional, or secondary, endpoints tested its effectiveness by measuring muscle strength and volume compared to placebo.

Participants received a range of 50-200 mg ACE-083 or placebo that was administered into either the rectus femoris a muscle in the middle of the front of the thigh, or the tibialis anterior, which is a muscle located primarily near the shin. Participants received either 1 or two doses three weeks apart.

Among the 58 women enrolled, 42 received ACE-083 and 16 received placebo.

Safety results showed that there were no serious adverse events, dose-limiting toxicities, or discontinuations due to adverse events.

The maximum mean increase in muscle volume for the rectus femoris muscle was 14.5%, and 8.9% for the tibialis anterior muscle. However, researchers failed to detect significant changes in mean muscle strength.

“These unprecedented increases in total muscle volume produced in the Phase 1 trial of ACE-083 were critical in leading us to rapidly advance into two Phase 2 trials in two distinct diseases of focal muscle weakness,” Matthew Sherman, MD, chief medical officer of Acceleron, said in a press release.

ACE-083 is now being tested in two Phase 2 clinical trials one for CMT (NCT03124459), currently enrolling participants, and one for facioscapulohumeral dystrophy (FSHD).

Preliminary results from Part 1 of the FSHD trial showed there was a mean muscle total volume increase of over 12 percent in patients who received ACE-083 in the tibialis anterior and biceps brachii  both muscles in the upper arm.

The final results from Part 1 of both Phase 2 trials are expected in the second half of 2018. Part 2 of the FSHD trial is expected to begin soon, with results expected in 2019. Part 2 of the CMT trial is expected to begin by the end of 2018.

“We believe ACE-083 could have a significant impact in increasing muscle mass, strength, and function in patients with neuromuscular disorders,” Sherman said.

Iqra holds a MSc in Cellular and Molecular Medicine from the University of Ottawa in Ottawa, Canada. She also holds a BSc in Life Sciences from Queen’s University in Kingston, Canada. Currently, she is completing a PhD in Laboratory Medicine and Pathobiology from the University of Toronto in Toronto, Canada. Her research has ranged from across various disease areas including Alzheimer’s disease, myelodysplastic syndrome, bleeding disorders and rare pediatric brain tumors.
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Iqra holds a MSc in Cellular and Molecular Medicine from the University of Ottawa in Ottawa, Canada. She also holds a BSc in Life Sciences from Queen’s University in Kingston, Canada. Currently, she is completing a PhD in Laboratory Medicine and Pathobiology from the University of Toronto in Toronto, Canada. Her research has ranged from across various disease areas including Alzheimer’s disease, myelodysplastic syndrome, bleeding disorders and rare pediatric brain tumors.
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