Endocrine Therapy

Therapeutic treatment for growth hormone deficiencies

Phase 1

AEZS-130, a growth hormone secretagogue (GHS), is a novel synthetic small molecule, acting as a ghrelin mimetic, that is orally active and stimulates the secretion of growth hormone (GH). Based on results of Phase 1 studies, AEZS-130 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. In addition to the therapeutic application, AEZS-130 is also in a Phase 3 trial as a diagnostic test for growth hormone deficiencies in adults.

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.

An open, randomized, placebo-controlled dose-escalation Phase 1 study has been completed, involving a total of 36 healthy male volunteers. The data demonstrated that between 1-2 hours following drug administration, there was a statistically significant increase in the levels of growth hormone in the blood without any effect on other hormone levels. In all cases, AEZS-130 was well tolerated and no adverse events were reported. AEZS-130, the only orally-administered specific GHS in development, presents a major competitive advantage in terms of ease and convenience of delivery over current treatments which are only available through injections.