|
|
|
|
 |
Macimorelin acetate (AEZS-130) Diagnostic in adult growth hormone deficiency (endocrinology)
Therapeutic area
Endocrine Therapy
Target indication
Diagnostic test for growth hormone deficiencies
Development stage
Phase 3
Description
Macimorelin acetate (AEZS-130) is a novel synthetic small molecule, acting as a ghrelin agonist, that is orally active and stimulates the secretion of growth hormone (GH). In addition to the diagnostic indication, macimorelin acetate (AEZS-130), based on results of Phase 1 studies, has potential applications for the treatment of cachexia, a condition frequently associated with severe chronic diseases such as cancer, chronic obstructive pulmonary disease and AIDS. Mode of action
Growth hormone secretagogues (GHS) are a potent regulator of lipid, sugar and protein metabolism, that directly stimulate growth hormone (GH) secretion from the pituitary gland without the involvement of Growth Hormone Releasing Hormone (GH-RH) or somatostatin.
Trial results
Phase 3 results as a diagnostic test for adult growth hormone deficiency (AGHD)showed that macimorelin acetate (AEZS-130) reached its primary endpoint demonstrating >90% area-under-the-curve (AUC) of the Receiver Operating Characteristic (ROC) curve, which determines the level of specificity and sensitivity of the product. The parameters of the study were achieved as agreed to with the FDA under our Special Protocol Assessment (SPA). Importantly, the primary efficacy parameters showed that the study achieved both specificity and sensitivity at a level of 90% or greater. The use of macimorelin acetate (AEZS-130) was shown to be safe and well tolerated overall throughout the completion of this trial. For more details, please refer to the following link: http://www.aezsinc.com/en/page.php?p=60&q=453
Development plan
Following the successfull Phase 3 trial results in AGHD, for which orphan-drug status had been granted by the FDA in 2007, the Company expects to file a new drug application (NDA) in 2Q-2013.
|
 |
|
|
 |
