RARE Daily

Ascendis Reports Positive Phase 3 Results of Human Growth Hormone

December 19, 2023

Rare Daily Staff

Ascendis Pharma reported positive topline results from its phase 3 foresiGHt study of its human growth hormone in adults with growth hormone deficiency.

Growth hormone plays an essential role in the health of children and adults, promoting normal growth in children and maintenance of normal body composition and cardiometabolic health throughout adulthood. In adults, growth hormone boosts protein production, promotes fat utilization, enhances muscle mass, and helps regulate blood sugar levels.

Adult GHD is a condition in which an individual’s body does not produce enough growth hormone. Symptoms and morbidity can include central obesity, metabolic syndrome, decreased bone density, alterations in lipid profile and markers of cardiovascular risk, fatigue, general weakness, lack of muscle tone, and psychological symptoms such as cognitive impairment, social isolation, lack of motivation, and depression.

TransCon hGH is a prodrug of somatropin administered once weekly, designed to provide sustained release of active, unmodified somatropin. The unmodified, unbound somatropin released from TransCon hGH has the identical 191 amino-acid sequence and size as endogenous growth hormone. TransCon hGH is approved and marketed as Skytrofa in the United States and the European Union as a once-weekly treatment for children and adolescents with GHD.

The phase 3 randomized, parallel-arm, placebo-controlled (double-blind) and active-controlled (open-label) trial compared the efficacy and safety of TransCon hGH with placebo and daily hGH (human growth hormone, somatropin) in adults with 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 of a 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 of a change from baseline in total body lean mass.

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.”

Photo: Jan Mikkelsen, Ascendis Pharma’s president and CEO

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