BackgroundThis study assessed the efficacy and safety of the once-daily glucagon-like peptide-1 receptor agonist, lixisenatide, in Asian patients with type 2 diabetes mellitus inadequately controlled on metforminsulfonylurea. MethodsIn this 24-week, double-blind, placebo-controlled, multinational study, patients were randomized to lixisenatide 20 mu g once daily or placebo. The primary endpoint was absolute change in glycated haemoglobin (HbA(1c)) from baseline to week 24. ResultsA total of 391 patients were randomized. Lixisenatide significantly reduced HbA(1c) levels compared with placebo (LS mean difference: -0.36%, p=0.0004). A significantly higher proportion of lixisenatide-treated patients achieved HbA(1c) targets of <7% (p=0.003) and 6.5% (p=0.001) versus placebo. Lixisenatide was associated with a statistically significant reduction in 2-h postprandial plasma glucose after a standardized breakfast versus placebo (LS mean difference: -4.28mmol/L, p<0.0001) and a significant reduction in fasting plasma glucose (p=0.0109). There was no difference in weight loss versus placebo, with a modest reduction in body weight reported for both groups (lixisenatide: -1.50kg, placebo: -1.24kg; p=0.296). The incidence of treatment-emergent adverse events (TEAEs) was 64.3% with lixisenatide versus 47.4% with placebo, with serious TEAEs reported in 1.5% versus 2.1% of patients, respectively. The most common TEAE in the lixisenatide group was nausea (16.3% vs 2.6% with placebo). The incidence of symptomatic hypoglycaemia was 5.6% with lixisenatide treatment and 2.6% with placebo (p=0.1321), with no severe symptomatic hypoglycaemia events reported. ConclusionsIn Asian patients with type 2 diabetes mellitus insufficiently controlled on metformin +/- sulfonylurea, lixisenatide significantly improved glycaemic control and was well tolerated during the 24-week study. (c) 2014 The Authors. Diabetes/Metabolism Research and Reviews published by John Wiley & Sons, Ltd.