Aeterna Zentaris has reported favorable top-line results of its completed phase III study with AEZS-130 as the first oral diagnostic test for Adult Growth Hormone Deficiency (AGHD). The results show that AEZS-130 reached its primary endpoint demonstrating >90% area-under-the-curve (AUC) of the Receiver Operating Characteristic curve, which determines the level of specificity and sensitivity of the product. The company is currently proceeding with further detailed analyses of the data and preparing for a pre-New Drug Application (NDA) meeting with the FDA in the upcoming months, which would be followed by the filing of a NDA for the registration of AEZS-130 in the United States.
The parameters of the study were achieved as agreed to with FDA under Special Protocol Assessment. The primary efficacy parameters show that the study achieved both specificity and sensitivity at a level of 90% or greater. In addition, 8 of the 10 newly enrolled AGHD patients were correctly classified by a pre-specified peak GH threshold level. The use of AEZS-130 was shown to be safe and well tolerated overall throughout the completion of this trial.