Ascendis Pharma Announces Topline Results From TransCon hGH Achieved Statistically Superior Outcomes Compared To Placebo In Phase 3 foresiGHt Trial Of Adults With Growth Hormone Deficiency

–   On primary efficacy and key secondary endpoints, TransCon hGH demonstrated statistically significant reduction from baseline in trunk fat and increase in total body lean mass at Week 38 compared to placebo

–   TransCon hGH was generally safe and well tolerated, similar to daily hGH, with no discontinuations related to study drug

–   Exploratory post-hoc analysis suggests the same treatment effects on target tissues as daily hGH

COPENHAGEN, Denmark, Dec. 19, 2023 (GLOBE NEWSWIRE) — Ascendis Pharma A/S (NASDAQ:ASND) today announced positive topline results from foresiGHt, its Phase 3 randomized, parallel-arm, placebo-controlled (double-blind) and active-controlled (open-label) trial to compare the efficacy and safety of TransCon hGH (lonapegsomatropin) with placebo and daily hGH (human growth hormone, somatropin) in adults with growth hormone deficiency (GHD).

The foresiGHt Trial evaluated 259 adults with GHD aged 23 to 80 years old, randomized 1:1:1, titrated to receive a target fixed dose of TransCon hGH, placebo, or daily hGH based on age and oral estrogen intake with approximately equivalent hGH mg/week for TransCon hGH and daily hGH.

• 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 -1.67% vs. placebo +0.37%, LS mean difference = -2.04%, p < 0.0001)
• 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, LS mean difference = 1.70 kg, p < 0.0001)

  • Change from baseline in trunk fat mass (TransCon hGH -0.48 kg vs placebo +0.22 kg, LS mean difference = -0.70 kg, p = 0.0053)
• 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 ≤ 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.”