Aeterna Zentaris has reported favorable top-line results of AEZS-130, a novel orally active small molecule that stimulates the secretion of growth hormone.
The company has completed a Phase 3 trial for use as a simple oral diagnostic test for adult growth hormone deficiency (AGHD).
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.
AEZS-130 works by stimulating a patient’s growth hormone secretion, which normally only occurs during sleep, after which a healthcare provider will measure how well the body responds to that stimulation based on the patient’s growth hormone levels over a period of time.
AEZS-130 has been granted orphan-drug designation by the US Food and Drug Administration (FDA) for use as a diagnostic test for growth hormone deficiency.
Aeterna Zentaris owns the worldwide rights to AEZS-130 which, if approved, would become the first orally administered diagnostic test for AGHD.
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 US.