Ascendis Pharma's Innovative Trial Results in Turner Syndrome

Breakthrough Trial Results for TransCon™ hGH in Turner Syndrome

In a significant advancement for treating Turner syndrome, Ascendis Pharma A/S recently announced the outcomes of its pivotal New InsiGHTS trial. This Phase 2 study focused on the effectiveness and safety of once-weekly TransCon hGH, known as lonapegsomatropin, compared to traditional daily somatropin treatments. Conducted in a randomized and open-label format, the trial involved prepubertal children diagnosed with Turner syndrome.

Positive Findings at Week 26

The topline results from the study revealed that all cohorts receiving TransCon hGH displayed a safety and tolerability profile comparable to those treated with daily somatropin. This outcome is particularly encouraging as it suggests that TransCon hGH can be administered less frequently while maintaining similar efficacy in promoting growth. The study encompassed 49 children, aged 1 to 10, ensuring a robust sample size to support these findings.

Moreover, after 26 weeks, the annualized height velocity in children treated with TransCon hGH was on par with those receiving daily somatropin. This improvement highlights the potential of TransCon hGH as a viable alternative for managing growth in children with this condition. The data affirmed the therapy's capability to enhance growth metrics, independent of the starting dose utilized.

Child-Friendly Treatment Option

“The data collected at Week 26 indicates that once-weekly TransCon hGH can significantly benefit children with Turner syndrome, similar to the well-established daily somatropin,” stated Jan Mikkelsen, President and Chief Executive Officer of Ascendis Pharma. The findings pave the way for future clinical exploration, specifically the upcoming basket trial planned for 2025, which aims to broaden the drug's market label.

Understanding Turner Syndrome

Turner syndrome is a genetic condition affecting females, characterized by the absence or alteration of one of the two X chromosomes. This syndrome occurs in roughly 1 in every 2,000 to 2,500 live female births and may lead to various health issues, including short stature and possible organ dysfunctions. The condition can pose severe health risks, including cardiovascular complications and endocrine disorders, emphasizing the need for effective treatment options.

About Ascendis Pharma A/S

Ascendis Pharma is at the forefront of biopharmaceutical innovation, utilizing its proprietary TransCon technology platform. This platform aims to develop advanced therapies across multiple disease states, demonstrating a strong commitment to enhancing patients' lives. Based in Copenhagen, Denmark, Ascendis also has operational facilities in both Europe and the United States, reinforcing its global reach in health care solutions.

Looking Ahead

As Ascendis Pharma continues its clinical journey, they anticipate the Week 52 results that will further examine the long-term effects and benefits of lonapegsomatropin against traditional therapies. Such findings could significantly influence the treatment landscape for Turner syndrome and potentially expand patient access to innovative therapies.

Frequently Asked Questions

What is TransCon hGH?

TransCon hGH, or lonapegsomatropin, is a novel growth hormone treatment designed for pediatric patients, specifically approved for managing growth hormone deficiency.

What were the primary results of the trial?

The trial demonstrated that TransCon hGH has comparable safety and growth-promoting effects to daily somatropin after 26 weeks of treatment.

What is Turner syndrome?

Turner syndrome is a genetic disorder in females that leads to a variety of developmental and health challenges, primarily characterized by short stature.

When will further trial results be available?

Ascendis Pharma expects to release Week 52 results in the near future, providing more insights on the long-term effectiveness of TransCon hGH.

What is Ascendis Pharma's focus?

Ascendis Pharma aims to develop innovative and best-in-class therapies through its advanced TransCon technology platform, enhancing treatment options for patients worldwide.

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