By Mark Terry
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.
No serious side effects were observed in either arm of the trial. One serious adverse event was observed in each arm but was determined to be unrelated to the study drug.
“The height Trial results announced today represent a potential breakthrough for patients and future treatment options for growth hormone deficiency,” stated Jan Mikkelsen, Ascendis Pharma’s president and chief executive officer.
He went on to say, “The height Trial demonstrated that TransCon hGH had superior efficacy, as well as comparable safety and tolerability to daily growth hormone. We believe these results provide a validation of our TransCon technology platform, which forms the basis of our endocrinology pipeline and has potential application in other therapeutic areas.”
The trial looked at 161 treatment-naïve children with GHD. They were randomly split in a two-to-one ratio to either receive the once-weekly TransCon hGH or daily Genotropin for 52 weeks. Of the total group, two children, one from each arm, withdrew from the trial before the final visit.
TransCon hGH delivers unmodified hGH, the same hormone used in daily treatment, at a predictable rate over one week. In the U.S. and Europe, the only GHD treatment option is daily HGH injections.
“Results from the pivotal height Trial demonstrated that TransCon hGH was more effective than daily hGH, with comparable safety and tolerability observed,” stated Jonathan Leff, Ascendis’ chief medical officer.
The company’s TransCon hGH Phase III program includes heiGHht, fliGHt and enliGHten trials. Data from the fliGHt Trial, which evaluates TransCon hGH in patients who switch from daily hGH, are expected in the second quarter of this year. The enliGHten Trial is a long-term extension study which gives patients from the heiGHt and fliGHt Trials the opportunity to continue once-weekly TransCon hGH treatment.
The company plans a clinical database lock for the TransCon hGH Phase III program in this year’s third quarter. It then plans to submit a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) in the first half of 2020.