The Danish biotechnology company Ascendis Pharma A/S has announced positive interim results from its ongoing Phase 2 pediatric study.
The study regards evaluating once-weekly TransCon Growth Hormone in children with growth hormone deficiency, or GHD.
“It has been demonstrated that patients who do not comply with the daily injection regimen of currently available growth hormone therapies suffer from suboptimal treatment outcomes,” commented Paul Saenger, M.D., Professor Emeritus of Pediatrics, Albert Einstein College of Medicine. Dr. Saenger continued, “The interim results from the Phase 2 pediatric study suggest that TransCon Growth Hormone has thus far demonstrated an impressive safety and efficacy profile comparable to that of existing daily growth hormone therapies, and may enable once-weekly dosing to improve patient compliance and treatment outcomes.”
Key conclusions from the interim analysis include mean annualized height velocities ranged from 13.0 cm to 14.1 cm for the three weekly dose levels of TransCon Growth Hormone, which were comparable to the active comparator, daily injections of Genotropin; there have been no reports of serious or unexpected adverse events; injection site reactions were generally mild and transient and occurred in only a few patients; thus far, there have been no observations of injection site nodule formation or lipoatrophy; and a dose-proportional increase in IGF-I levels was observed following dosing of the three TransCon Growth Hormone doses.