Ascendis announces TransCon hGH achieved ‘statistically superior’ outcomes

Ascendis Pharma A/S announced positive topline results from foresiGHt, its Phase 3 randomized, parallel-arm, placebo-controlled and active-controlled trial to compare the efficacy and safety of TransCon hGH with placebo and daily hGH in adults with growth hormone deficiency. TransCon hGH demonstrated superiority on its primary efficacy endpoint at Week 38: Change from baseline in trunk percent fat as measured by dual x-ray absorptiometry; TransCon hGH demonstrated superiority on its key secondary efficacy endpoints at Week 38: Change from baseline in total body lean mass – TransCon hGH +1.60 kg vs placebo -0.10 kg, Change from baseline in trunk fat mass – TransCon hGH -0.48 kg vs placebo +0.22 kg; Exploratory post-hoc analysis at Week 38 demonstrated comparable treatment effect of TransCon hGH and daily hGH on target tissues. For patients with IGF-1 SDS levels less than or equal to 1.75 at Week 38: Change from baseline in trunk percent fat – TransCon hGH -2.42% vs. daily hGH -2.59%, Change from baseline in total body lean mass – TransCon hGH +1.70 kg vs daily hGH +1.37 kg, Change from baseline in trunk fat mass – TransCon hGH -0.90 kg vs daily hGH -0.94 kg; TransCon hGH was generally safe and well tolerated, with no discontinuations related to study drug and with comparable safety and tolerability to daily hGH. “TransCon hGH, our once-weekly growth hormone, is designed to deliver unmodified somatropin, thereby preserving the same overall endocrine benefits inherent in endogenous and daily hGH,” said Jan Mikkelsen, Ascendis Pharma’s President and CEO. “Next year, we plan to submit a supplemental Biologics License Application to the U.S. FDA for the adult GHD indication and look forward to reporting data from our Phase 2 program in Turner syndrome.”