Ghrelin, a peptide hormone of the gastrointestinal system, has numerous biological activities. In particular, it has a direct effect on the release of growth hormone (GH). We have synthesized a pseudopeptide (JMV1843, Macimorelin, MacrilenTM) capable of stimulating GH secretion. Phase 3 clinical studies have been successfully completed, and a "Marketing Authorization" for application in the diagnosis of growth hormone deficiency in adults has been filed with the FDA in North America. At the same time, this compound is in Phase 2A trials for its effects on cachexia in cancer patients.
In this presentation, the various steps leading to the design of this orally active ghrelin analog and its clinical studies will be described and discussed.