Ascendis Pharma’s TransCon Growth Hormone Passes Phase III With Flying Colors

Ascendis Pharma, based in Copenhagen, Denmark, announced that its once-weekly TransCon Growth Hormone (HGH) hit its primary endpoints in its Phase III height Trial in children with pediatric growth hormone deficiency (GHD). The trial compared Ascendis’ TransCon hGH to a daily growth hormone, Genotropin. The trial met its primary objective, showing that TransCon hGH was non-inferior to Genotropin as well as superior to the daily hGH in terms of annualized height velocity (AHV) at 52 weeks.
Pediatric GHS is a serious orphan disease caused by the pituitary gland not producing enough growth hormone. GHD patients are characterized by short stature, but also have metabolic abnormalities, psychosocial challenges, cognitive deficiencies and poor quality of life. The standard of care has been a daily subcutaneous injection of hGH. This improves growth and metabolic effects, but there are adherence problems due to the daily injections. The AHV was higher for TransCon hGH than the daily hGH at each visit. At 26 weeks and after, the treatment demonstrated statistical significance. Poor responder incidence was 4 percent and 11 percent in the TransCon hGH and daily hGH arms, respectively.