ACE-031
ACE-031 (soluble activin receptor type IIB)
Also known as: Ramatercept
A myostatin/activin decoy studied for muscular dystrophy whose trials were halted over vascular safety signals.
Overview
ACE-031 is a soluble form of the activin receptor type IIB that acts as a decoy, trapping myostatin and related factors to promote muscle growth. It was studied for Duchenne muscular dystrophy, but trials were halted in 2011 after signals including nosebleeds and small vessel changes. It is not approved.
Mechanism of action
Binds and neutralizes myostatin and activin before they can signal, releasing their inhibition of muscle growth.
Key studies & citations
- Human2013
A single ascending-dose study of muscle regulator ACE-031 in healthy volunteers
One dose in 48 postmenopausal women: half-life 10 to 15 days; at 3 mg/kg total lean mass rose 3.3% and thigh muscle volume 5.1% by day 29.
Muscle and Nerve - Human2017
Myostatin inhibitor ACE-031 treatment of ambulatory boys with Duchenne muscular dystrophy: results of a randomized, placebo-controlled clinical trial
The trial was stopped after the second dose level because of epistaxis and telangiectasias; only non-significant trends toward preserved six-minute walk and higher lean mass were seen.
Muscle and Nerve
Frequently asked questions
Why was ACE-031 discontinued?
Its trials were halted after non-muscle safety signals, including nosebleeds and small blood vessel changes, outweighed the muscle benefits.