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Multiple Ascending Dose Phase 1 Study ‘ A study in healthy postmenopausal women (A031-02), evaluating multiple doses of ACE-031, is currently ongoing. For more information on the study design, click here. Acceleron has presented preliminary results from this study. Click here for the poster presentation of these results and click here for the accompanying press release. As this study is not complete, final results are not yet available. A031-02 randomized 60 subjects in 6 cohorts of 10 subjects each to receive ACE-031 or placebo (8:2) at dose levels ranging from 0. Subjects were followed for 12 weeks after their last dose. Although this safety study was not powered to assess statistically significant changes in endpoints, multiple exploratory statistical analyses were performed on the data. Consistent with the findings of the single dose study (A031-01), multiple doses of ACE-031 given to healthy postmenopausal women were generally well tolerated and resulted in rapid and sustained effects on muscle, bone and fat. The most common adverse events were injection site reactions, headache and nosebleeds. The majority of adverse events were mild and transient. ACE-031 had a half-life of approximately 12 days, supportive of dosing every 2-4 weeks. Mean total body lean mass measured by DXA at day 36 increased by 5. This was statistically significant compared to the 0, gp test u 250 mg injectable steroids nebido. Significant increases were also seen at doses of 2 and 3 mg/kg every 4 weeks. Mean thigh muscle volume measured by MRI at day 36 increased by 4. Significant increases were also seen at 2 mg/kg given every 4 weeks. No statistically significant changes were observed in grip strength or functional tests in this healthy volunteer safety study. Total Whole Body Lean Body Mass (DXA) % Change (Mean +/- SE) Thigh Muscle Volume (MRI) % Change (Mean +/- SE) ACE-031 treatment led to a rapid and significant increase in a biomarker of bone formation (BSAP) and decrease in a biomarker of bone resorption (CTX) versus placebo. Consistent with these effects, lumbar spine bone mineral density by DXA increased up to 3. ACE-031 treatment also led to significantly altered biomarkers of fat metabolism (increased adiponectin and decreased leptin) by day 8. Total body fat mass measured by DXA decreased up to 8. Acceleron is developing ACE-031 for the treatment of patients with neuromuscular diseases, such as Duchenne Muscular Dystrophy (DMD), with the goal of improving strength and preserving physical function. By affecting the muscle directly, ACE-031 may one day offer hope to patients suffering from these debilitating diseases. A mutation in the myostatin gene increases muscle mass and enhances racing performance in heterozygote dogs, Mosher DS et al.
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