Ascendis Pharma Announces Positive Top-line Results from a Phase 2 Pediatric Study for the Treatment of Growth Hormone Deficiency

04-Aug-2015 - Denmark
Ascendis Pharma A/S announced positive top-line results from a six-month Phase 2 study to evaluate the safety and efficacy of once-weekly TransCon Growth Hormone in 53 treatment-naïve, pre-pubertal children with growth hormone deficiency, or GHD.
"We are extremely pleased with the top-line results from our Phase 2 pediatric study and believe these data establish once-weekly TransCon Growth Hormone as the potential best-in-class long-acting human growth hormone program," stated Jan Mikkelsen, President and Chief Executive Officer.  Mr. Mikkelsen continued, "Most importantly, these results move us one step closer to fulfilling our goal of developing an innovative therapy that has the potential to improve the real-world treatment outcomes of patients with GHD."
The Phase 2 pediatric study was conducted to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of TransCon Growth Hormone in treatment-naïve pre-pubertal children with growth hormone deficiency, or GHD, who meet internationally recognized diagnostic criteria for GHD. The study enrolled 53 patients into the treatment phase and was a 6-month multi-center, randomized, open-label study comparing three dose levels of TransCon Growth Hormone (0.14; 0.21; and 0.30 mg hGH/kg/week), administered once per week, to the active control Genotropin (0.21 mg hGH/kg/week), administered as a daily injection.
Highlights from the top-line analysis include:
  • mean annualized height velocities among the three dosing levels administered weekly ranged from 11.9 cm for the 0.14 mg/kg/week dose to 13.9 cm for the 0.30 mg/kg/week dose, which were comparable to 11.6 cm for the active comparator, daily injections of Genotropin® at a 0.21 mg/kg/week dose;
  • there were no reports of drug-related serious or unexpected adverse events;
  • injection site reactions were generally mild and transient and were observed at a rate that was similar to the daily hGH control arm;
  • there were no observations of injection site nodule formation or lipoatrophy;
  • low immunogenicity consistent with published data for daily growth hormone;
  • maximum hGH blood concentration was comparable between equivalent weekly doses of TransCon Growth Hormone and daily hGH; and
  • a dose-proportional increase in IGF-I levels was observed following dosing of the three TransCon Growth Hormone dose levels. Consistent with expectations, transient point values of IGF-I standard deviation score > +2 have been observed in a small number of patients and primarily in the high-dose treatment arm.

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