en.Wedoany.com Reported - Danish biopharmaceutical company Ascendis Pharma A/S (Nasdaq: ASND) announced 182-week data from the Phase 3 PaTHway trial of its core drug TransCon PTH (palopegteriparatide) in adult patients with hypoparathyroidism. Over the three-and-a-half-year treatment period, the drug demonstrated sustained efficacy and safety. By replicating the systemic effects of endogenous parathyroid hormone (PTH), it improved patients' quality of life, normalized and stabilized urinary calcium, serum calcium, serum phosphorus, and bone mineral density, while enabling patients to discontinue routine treatment with active vitamin D and calcium supplements.
Dr. Aliya Khan, Professor of Medicine at McMaster University in Canada, stated that TransCon PTH, through its ability to replicate the systemic effects of endogenous PTH, successfully alleviated the physical and psychological burden on patients. The Phase 3 data reinforce the rapid and sustained benefits observed throughout the clinical trial.
The trial enrolled 82 adult patients with hypoparathyroidism (85% post-surgical, 15% non-surgical), including a 26-week randomized, double-blind, placebo-controlled period and a 156-week open-label extension period. Results showed that 86% of patients achieved a response on the composite endpoint of multiple measures, defined as serum calcium within the normal range, no use of active vitamin D, and daily calcium intake not exceeding 600 mg. Among these, 89% had normal albumin-adjusted serum calcium levels, with a mean of 8.8 mg/dL; 100% achieved independence from active vitamin D; and 96% achieved independence from therapeutic doses of calcium supplements (daily calcium intake <600 mg). Regarding renal function, the mean (standard error) estimated glomerular filtration rate (eGFR) at Week 182 was 80.2 (1.8) mL/min/1.73 m², representing a mean increase of 11.0 (1.4) mL/min/1.73 m² from baseline. The eGFR increase was sustained, contrasting with the expected age-related decline in adults. 24-hour urinary calcium excretion normalized within 26 weeks and was maintained through Week 182. Bone mineral density Z-scores (matched for age and sex), which were elevated at baseline, were corrected by Week 26 and remained above 0 at Week 182. According to measurements from the Hypoparathyroidism Patient Experience Scale (HPES) and the 36-Item Short Form Health Survey (SF-36v2), patients reported rapid and clinically meaningful improvements in all domain symptoms and health-related quality of life, sustained through Week 182. No new safety signals were identified during treatment; adverse events were mostly mild or moderate, with no discontinuations due to the study drug, and no anti-PTH antibodies were detected over the three-and-a-half-year period.
TransCon PTH is a once-daily prodrug of PTH (1-34) designed to provide stable, physiological levels of active PTH over 24 hours. It is approved under the brand name YORVIPATH in the United States, the European Union, the European Economic Area, and other specified jurisdictions for the treatment of adult hypoparathyroidism. (Guppy M et al. BMJ Open. 2024;14(11):e089783)
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