- 88% of patients achieved normal calcium levels and 96% became independent from conventional treatment.
- Significant kidney function improvement noted with mean eGFR increase of 8.76 mL/min/1.73 m².
- High retention rate of 89% over the 3.5-year trial period with a favorable safety profile observed.
Ascendis Pharma (ASND, Financial) has reported promising results from its three-year Phase 3 PaTHway clinical trial of TransCon PTH (palopegteriparatide) for treating adults with hypoparathyroidism. The data shows a durable and sustained efficacy with significant clinical improvements.
Results demonstrated that 88% of the patients achieved normal levels of calcium while 96% no longer required conventional therapy by Week 156. The phase included a double-blind, placebo-controlled initial period, followed by an open-label extension, and is noted for its high retention rate of 89%.
One of the significant outcomes of the trial was the improvement in kidney function across the participants. The mean estimated Glomerular Filtration Rate (eGFR) increased by 8.76 mL/min/1.73 m² overall, with notable improvements seen in patients with lower baseline eGFR, where the increase was 13.98 mL/min/1.73 m². The therapy also maintained normalized calcium excretion in urine, addressing a critical complication.
Safety profile of TransCon PTH was favorable, with no new safety concerns identified. The treatment-emergent adverse events were predominantly mild to moderate, and no serious adverse events related to the drug were reported.
Aimee Shu, M.D., Executive Vice President of Endocrine & Rare Disease Medical Science and Chief Medical Officer at Ascendis Pharma, stated that these results indicate the long-term benefits of TransCon PTH for adults with hypoparathyroidism, regardless of the disease's etiology.
