Ascendis Pharma released 3.5-year data from its Phase 3 PaTHway Trial for TransCon PTH (palopegteriparatide) in adults with hypoparathyroidism. The trial showed continued positive effects on biochemistries, kidney function, and quality of life, irrespective of the cause of hypoparathyroidism. The data, presented at ENDO 2025, reveals sustained improvements for patients through Week 156.
This extended data is crucial for endocrinologists and patients managing hypoparathyroidism, a condition often resulting from thyroid surgery or autoimmune issues. The long-term efficacy demonstrated by TransCon PTH offers a potential solution for the chronic management of this disease, addressing significant unmet needs related to calcium regulation and kidney function. Specifically, the sustained improvements in kidney function, a key concern in hypoparathyroidism, highlight the potential of TransCon PTH to prevent long-term renal complications. This offers a substantial clinical advantage over current standard therapies which often struggle to maintain long-term biochemical control and improve kidney function.
In the 156-week data, 88% of patients achieved normal albumin-adjusted serum calcium levels, and 96% were independent from conventional therapies like calcium and vitamin D supplements. Mean estimated glomerular filtration rate (eGFR), a measure of kidney function, improved significantly, particularly for those with initially impaired kidney function. Patient-reported outcomes also indicated sustained improvements in symptoms and quality of life. Importantly, the treatment remained well-tolerated, with no new safety concerns emerging over the extended period.
These long-term results strengthen the case for TransCon PTH as a viable, long-term treatment for hypoparathyroidism, potentially changing the treatment landscape. The positive data across multiple endpoints, coupled with the sustained safety profile, could lead to wider adoption and improved outcomes for patients living with this challenging endocrine disorder. Further research and real-world evidence will be key to confirming the long-term benefits and positioning of TransCon PTH within the evolving treatment paradigm.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
