Improving Renal Function in Adults with Chronic Hypoparathyroidism: TransCon PTH Shows Promising Results According to Ascendis Pharma’s Phase 3 PaTHway Trial
COPENHAGEN, Denmark, May 13, 2024 - Ascendis Pharma A/S (Nasdaq: ASND) recently revealed their two-year findings from a post-hoc analysis conducted on their Phase 3 PaTHway trial. This analysis demonstrates significant and sustained improvements in renal function for adults suffering from chronic hypoparathyroidism who were treated with TransCon PTH (palopegteriparatide). The data was presented during the European Congress of Endocrinology 2024 (ECE 2024) by Professor Peter Schwarz, M.D. from the University of Copenhagen. These groundbreaking results have the potential to revolutionize treatment options for patients with chronic hypoparathyroidism.
Chronic hypoparathyroidism is a rare endocrine disorder characterized by an inadequate production of parathyroid hormone (PTH) by the parathyroid glands, resulting in an imbalance of calcium and phosphate levels in the body. This can lead to a myriad of symptoms, including muscle cramps, spasms, tingling, fatigue, and mood disorders. Additionally, chronic hypoparathyroidism can significantly impact renal function and increase the risk of developing kidney stones and renal failure.
In the Phase 3 PaTHway trial, Ascendis Pharma aimed to evaluate the safety and efficacy of TransCon PTH, a long-acting PTH analog designed to offer sustained release and stable levels of PTH in the body. The trial enrolled 113 adult patients with chronic hypoparathyroidism who were randomized to receive either TransCon PTH or placebo.
The results of the post-hoc analysis revealed remarkable improvements in renal function among patients treated with TransCon PTH. Renal function was assessed based on estimated glomerular filtration rate (eGFR) and urine albumin-to-creatinine ratio (UACR) measurements. Over the two-year period, patients treated with TransCon PTH demonstrated significant increases in eGFR, indicating improved kidney function, compared to placebo-treated patients. This finding is particularly noteworthy considering the negative impact of chronic hypoparathyroidism on renal health.
Notably, the improvements in renal function were sustained throughout the study period, suggesting a long-lasting positive effect of TransCon PTH treatment. These results offer hope for patients suffering from chronic hypoparathyroidism, who often face limited treatment options and potential complications associated with renal impairment.
Ascendis Pharma’s Vision 2030, introduced earlier this year, provides a strategic roadmap for the company’s focus and goals in the coming years. This vision aligns with the successful outcomes of the Phase 3 PaTHway trial, emphasizing the commitment of Ascendis Pharma to develop innovative therapies that address unmet medical needs.
Jan Mikkelsen, President and CEO of Ascendis Pharma, will present an update on the company’s progress and corporate milestones at the 42nd Annual J.P. Morgan Healthcare Conference. This conference provides a platform for leading pharmaceutical companies to share their latest advancements and future directions with industry experts and investors.
The significant and sustained improvements in renal function reported in the Phase 3 PaTHway trial are a game-changer for adults with chronic hypoparathyroidism. TransCon PTH has shown great promise in addressing the underlying causes of the disease and has the potential to transform the lives of patients. As Ascendis Pharma continues to pursue their Vision 2030, it is clear that the company is dedicated to pioneering innovative therapies that have a meaningful impact on patients’ lives.
Comments