Ascendis Pharma Announces Positive 5-Year Phase 2 Data for TransCon PTH in Hypoparathyroidism

Ascendis Pharma A/S announced positive five-year (Week 266) data from its Phase 2 PaTH Forward trial evaluating TransCon PTH (palopegteriparatide) in adults with hypoparathyroidism. The results demonstrated sustained efficacy and safety, with 82% of patients meeting the multi-component endpoint of maintaining normal serum calcium while achieving independence from active vitamin D and therapeutic doses of calcium. Notably, 96% of patients were independent of active vitamin D, and 95% were independent of therapeutic calcium (<600 mg/day). Clinical benefits extended to major organ systems, showing stabilized and improved kidney function (mean eGFR increase of 9.4 mL/min/1.73 m2 from baseline) and normalized 24-hour urine calcium levels. Quality of life measures (HPES and SF-36) showed rapid and maintained improvements across physical and cognitive domains. Bone mineral density (BMD) Z-scores also stabilized within the normative range. TransCon PTH was generally well-tolerated over the five-year period with no new safety signals or drug-related discontinuations. Of the original 59 patients, 56 (95%) completed the five-year trial.