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news.ACE-031, said to be well tolerated at all dose levels
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Acceleron Pharma (Acceleron) has provided preliminary results from the ACE-031 phase 1 single dose clinical trial, demonstrating that ACE-031 increased lean body mass and muscle volume.
The results from the randomized, placebo-controlled study revealed that ACE-031 was well tolerated at all dose levels. It also demonstrated a linear pharmacokinetic profile with an average half-life ranging from 10-15 days, single doses of ACE-031 at 1mg/kg and 3mg/kg produced dose-dependent increases in lean body mass measured by dual energy X-ray absorptiometry (DXA) as early as day 15 that were sustained through day 57.
Results also include subjects given single doses of placebo had a 0.2% decrease in lean body mass at day 57 compared to a 2.4% increase in subjects receiving 1mg/kg ACE-031, and a 2.6% increase in subjects receiving 3mg/kg ACE-031 and ACE-031 favorably affected biomarkers of fat mass and bone formation and resorption and decreased C-terminal type 1 collagen telopeptide (CTX) at doses of 1 and 3mg/kg.
ACE-031, a soluble fusion protein based on the activin receptor type IIB (ActRIIB), is a biologic therapeutic that inhibits signaling through the ActRIIB receptor. By preventing signaling though ActRIIB, ACE-031 increases muscle mass and strength. In numerous and varied animal models of disease, ACE-031 significantly increased muscle mass and muscle strength.
Matthew Sherman, CMO at Acceleron, said: “We are thrilled that this ACE-031 phase 1 clinical trial generated a substantial amount of encouraging safety data as well as clear signs of biological activity on muscle, bone and fat all following just a single dose of ACE-031. ACE-031 produced rapid, sustained and dose-dependent increases in lean body mass and muscle volume, increased biomarkers of bone formation and positively altered biomarkers of fat mass. Based on these results, we’ve initiated a phase 1 multiple dose study and look forward to further development of ACE-031 for the treatment of patients suffering from neuromuscular diseases.”