Ascendis Pharma announces top-line results from Phase 3 heiGHt trial
Ascendis Pharma announced top-line results from its pivotal Phase 3 heiGHt Trial were presented at the Endocrine Society's annual meeting, ENDO 2019, in New Orleans. Trial results demonstrated that TransCon Growth Hormone administered once-weekly to children with pediatric growth hormone deficiency had comparable safety and tolerability to daily Genotropin, with a significantly greater increase in annualized height velocity over the one-year study period. TransCon hGH has been designed to provide sustained release of unmodified hGH, the same growth hormone used in daily therapies, at a predictable rate over one week. The heiGHt Trial evaluated 161 treatment-naive children with GHD randomized in a 2:1 ratio to receive either once-weekly TransCon hGH or daily Genotropin for 52 weeks. Top-line results showed that once-weekly TransCon hGH was superior to once-daily hGH on the primary endpoint of annualized height velocity at 52 weeks. In the primary analysis of the intent-to-treat population using ANCOVA, TransCon hGH demonstrated an AHV of 11.2 cm/year compared to 10.3 cm/year for the daily hGH. The treatment difference was 0.86 cm/year with a 95% confidence interval of 0.22 to 1.50 cm/year. The AHV for TransCon hGH was significantly greater than the daily hGH. In addition, the incidence of poor responders was 4% and 11% in the TransCon hGH and daily hGH arms, respectively. In the trial, no serious adverse events related to study drug were observed in either arm. No treatment-emergent adverse events leading to discontinuation of study drug were observed in either arm. Adverse events leading to dose reduction occurred twice in the TransCon hGH arm and once in the daily hGH arm. Two subjects in each treatment arm experienced mild injection site reactions that were considered adverse events. Two subjects, one from each arm, withdrew from the trial prior to the final visit.