Ascendis Pharma A/S (ASND) has reported five-year Phase 2 data showing that its TransCon PTH (palopegteriparatide) consistently replicated the systemic actions of natural parathyroid hormone in adults with hypoparathyroidism, sustaining efficacy and safety across multiple organ systems.
The PaTH Forward trial demonstrated that TransCon PTH normalized and stabilized calcium levels, improved kidney function, bone mineral density, and quality of life measures, while enabling patients to achieve independence from conventional therapies such as active vitamin D and high-dose calcium supplements.
At Week 266, 82% of patients met the multi-component endpoint of maintaining normal serum calcium without active vitamin D and with less than 600 mg/day of calcium. Nearly all participants achieved independence from vitamin D and therapeutic calcium, and 95% completed the full five years of treatment. Improvements in kidney function were sustained, with mean eGFR increasing from baseline, countering the expected age-related decline.
Patients also reported rapid and lasting improvements in physical and cognitive symptoms, daily functioning, and overall health-related quality of life, as measured by validated scales such as HPES and SF-36. Bone mineral density scores corrected early and remain stable through five years.
The therapy was generally well tolerated, with no new safety signals identified. Adverse events were mostly mild or moderate, and no discontinuations were linked to the drug. Only one patient developed transient, non-neutralizing anti-PTH antibodies without impact on efficacy or safety.
Presented at the European Congress of Endocrinology 2026, these results reinforce TransCon PTH's potential to become a new standard of care for chronic hypoparathyroidism, moving beyond symptom management to address the underlying hormone deficiency.
ASND has traded between $160.86 and $250.74 over the past year. The stock closed Thursday's trading at $215.60, up 2.77%. During after-hours trading the stock is at $219.87, up 1.98%.
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