- Ascendis Pharma (ASND, Financial) reports 4-year data showing sustained efficacy of TransCon PTH in hypoparathyroidism treatment.
- 98% of patients maintained normal serum calcium, 93% became independent of conventional therapy.
- 67.8% of patients showed significant improvement in kidney function.
Ascendis Pharma (ASND) has announced positive long-term data from its Phase 2 PaTH Forward Trial evaluating TransCon PTH (palopegteriparatide) in adults with hypoparathyroidism. After 214 weeks, the study reported a 95% patient retention rate, with 56 of the original 59 patients continuing in the trial.
The trial results are encouraging, demonstrating that 98% of participants maintained normal albumin-adjusted serum calcium levels, and 93% achieved independence from conventional therapy, which typically involves calcium and active vitamin D supplementation. This achievement reduces the risk of complications like hypercalciuria and kidney stones associated with traditional treatment.
Furthermore, a significant 67.8% of the patients experienced clinically meaningful improvements in renal function, as indicated by a noticeable increase in estimated glomerular filtration rate (eGFR). Bone health also showed positive results, with bone turnover markers stabilizing above baseline and bone mineral density remaining within normal ranges throughout the study.
Regarding safety, TransCon PTH was well tolerated, featuring mostly mild to moderate adverse events, and no serious treatment-related adverse events or discontinuations were observed. This strong safety profile, coupled with the efficacy data, enhances the long-term commercial viability of TransCon PTH for treating hypoparathyroidism.
Dr. Aimee Shu, Executive Vice President of Endocrine & Rare Disease Medical Science at Ascendis Pharma, stated, "We set out to address the underlying cause of disease and are pleased to see this long-term data reinforcing the safety profile and durability of response in patients treated with TransCon PTH, including sustained normalization of skeletal dynamics and significant and sustained improvements in kidney function."
