COPENHAGEN, Denmark — November 7, 2025 — Leads & Copy —
Ascendis Pharma A/S (Nasdaq: ASND) has announced new data from a pooled analysis of its Phase 2 PaTH Forward and Phase 3 PaTHway trials, demonstrating sustained and clinically meaningful improvements in renal function in adults with hypoparathyroidism treated with TransCon PTH (palopegteriparatide) through Year 3.
The findings, which corroborate individual trial results presented earlier in the year, were shared at Kidney Week 2025 in a poster presentation led by Dr. Elvira Gosmanova, a nephrologist at Albany Medical College and Albany VAMC (New York).
The PaTH Forward and PaTHway trials included 141 adults with hypoparathyroidism, with the pooled analysis representing 139 of these individuals, with a mean age of 49 years. The trials featured randomized, double-blind, placebo-controlled periods through Weeks 4 and 26, respectively, and open-label extension periods through Weeks 266 and 182, respectively. Participants were required to have an eGFR ≥30 mL/min/1.73 m2 for trial eligibility. The three-year data underwent post-hoc analysis to evaluate the long-term impact of TransCon PTH on renal function, assessed via eGFR. The baseline eGFR in the trials was 69 (17) mL/min/1.73 m2.
The analysis included 41 patients with a baseline eGFR of less than 60 mL/min/1.73 m2 and 98 patients with a baseline eGFR of 60 mL/min/1.73 m2 or greater. Safety was evaluated based on 24-hour urine calcium excretion and treatment-emergent adverse events (TEAEs).
At Year 3, over 91% of palopegteriparatide recipients in both trials were independent of conventional therapy, defined as no active vitamin D intake and calcium intake of 600 mg/day or less. Additionally, at least 84% of patients had normocalcemia (8.3-10.6 mg/dL). Sustained improvements in eGFR (≥ 5 mL/ min / 1.73 m2) were seen in 70.3% of patients, with more significant improvements in those with lower baseline eGFR.
The most considerable eGFR increases occurred in the first six months of TransCon PTH treatment, followed by a continued upward trend. Mean (SD) eGFR increased from baseline to Year 3 by 9.8 (10.9) mL/min/1.73 m2 in PaTH Forward and by 8.8 (11.9) mL/min/1.73 m2 in PaTHway.
Reductions in 24-hour urinary calcium excretion remained within the normal range through Year 3.
TransCon PTH was generally well-tolerated in both trials, with no new safety concerns. Most TEAEs were mild to moderate, and no patients discontinued treatment due to treatment-related TEAEs.
Dr. Aimee Shu, Executive Vice President and Chief Medical Officer at Ascendis Pharma, stated that the analysis of a larger patient group showed the reproducibility of eGFR improvements over time. She emphasized the importance of replicating normal PTH physiology with TransCon PTH in adults with hypoparathyroidism. Dr. Shu added that this was Ascendis Pharma’s first presentation at a nephrology-focused meeting, and they anticipate further discussion on the significance of PTH replacement for overall kidney health.
TransCon PTH, approved as YORVIPATH® in the United States, European Union, and European Economic Area, is a once-daily prodrug of PTH (1-34) designed to provide active PTH within the physiological range for 24 hours/day, for the treatment of adults with hypoparathyroidism.
Hypoparathyroidism is an endocrine disorder resulting from insufficient parathyroid hormone (PTH) levels, which are crucial for calcium and phosphate balance. Individuals with this condition may face severe complications, including neuromuscular irritability, renal issues, extra-skeletal calcifications, and cognitive impairment. Post-surgical hypoparathyroidism is the most common cause, accounting for 70-80% of cases.
Ascendis Pharma A/S is focused on using its TransCon technology platform to create a difference for patients, with core values that include Patients, Science, and Passion. The company develops new therapies that aim to address unmet medical needs. Ascendis Pharma is headquartered in Copenhagen, Denmark, with additional facilities in Europe and the United States.
Investor Contacts:
Chad Fugere
Ascendis Pharma
ir@ascendispharma.com
Media Contact:
Melinda Baker
Ascendis Pharma
media@ascendispharma.com
Patti Bank
ICR Healthcare
+1 (415) 513-1284
patti.bank@icrhealthcare.com
Source: Ascendis Pharma A/S
