Pharma Fridays – December 22

  • Dec 2023
An Endocrine News roundup of the week’s pharmaceutical news, breakthroughs, and general information. *

TransCon™ hGH (lonapegsomatropin) Achieved Statistically Superior Outcomes Compared to Placebo in Phase 3 foresiGHt Trial of Adults with Growth Hormone Deficiency

On December 19, Ascendis Pharma 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 lonapegsomatropin with placebo and daily hGH (human growth hormone, somatropin) in adults with growth hormone deficiency (GHD). Ascendis is marketing the drug as TransCon Hgh.

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,” Jan Mikkelsen, Ascendis Pharma’s president and CEO said in a statement. “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.”

*Inclusion in Pharma Fridays does not suggest an endorsement by Endocrine News or the Endocrine Society.

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