AEZS 130 (Aeterna Zentaris) safe and effective in Growth Hormone Deficiency

Final Phase III results for its oral ghrelin agonist, AEZS 130 (macimorelin), from Aeterna Zentaris, show that the drug is safe and effective in diagnosing adult Growth Hormone Deficiency (AGHD). This multicenter open label study was originally designed as a cross-over trial of AEZS 130 vs growth hormone-releasing hormone (GHRH)+L-Arginine (ARG) in AGHD patients and in controls, matched for body mass index (BMI), estrogen status, gender and age. After 43 AGHD patients and 10 controls had been tested, GHRH became unavailable. The study was completed by testing 10 more AGHD patients and 38 controls with AEZS 130 alone. Of the 53 AGHD subjects enrolled, 52 received AEZS 130, and 50 who had confirmed AGHD prior to study entry were included in this analysis, along with 48 controls. Mean peak growth hormone (GH) levels in AGHD patients and controls following AEZS 130 administration were 2.36ng/mL (range 0.03-33) and 17.71ng/mL (range 10.5-94), respectively. The receiver operating characteristic (ROC) plot analysis yielded an optimal GH cut-point of 2.7ng/mL, with 82% sensitivity, 92% specificity and a 13% misclassification rate. Obesity (BMI>30) was present in 58% of cases and controls, and peak GH levels were inversely associated with BMI in controls. Adverse events (AE) were seen in 37% of AGHD patients and in 21% of controls. Results were presented at the 94th ENDO Annual Meeting and Expo.