Ascendis Pharma A/S（ASND）のニュースとイベントASNDAscendis Pharma A/S

Ascendis Pharma Reports TransCon PTH Kidney Function Improvement Data

Ascendis Pharma announced new data demonstrating early, sustained, and clinically meaningful improvements in kidney function in adults with hypoparathyroidism treated with TransCon PTH for up to five years in the company's Phase 2 PaTH Forward and Phase 3 PaTHway trials. Results from the pooled analysis were shared in a poster presented by nephrologist Elvira Gosmanova, Professor of Medicine at Albany Medical College, during Kidney Week 2026, the annual meeting of the American Society of Nephrology. Increases in mean estimated glomerular filtration rate, or eGFR, from baseline were statistically significant across patients from Week 26 of treatment through trial completion. Mean improvement in eGFR for all patients from baseline to Week 26 was 7.2 mL/min/1.73 m2 and to Week 52 was 9.6 mL/min/1.73 m2. By Week 52, clinically meaningful improvements in eGFR iv,v were observed in 68.9% of patients, and 49.6% of patients achieved an increase in eGFR of greater than10 mL/min /1.73 m2; eGFR remained stable through the end of the trials. From Week 52 to Week 266, the mean eGFR slope was 0.28 mL/min/1.73 m2/year, indicating clinically meaningful early improvements in eGFR sustained through up to 5 years, in contrast to the expected typical age-related decline in eGFR in adults. Patients with baseline eGFR values less than 60 mL/min/1.73 m2 had a numerically greater increase in mean eGFR than those with a baseline eGFR greater than or equal to 60 mL/min/1.73 m2. Twenty-four urine calcium excretion normalized by Week 26 from mean 396 mg/day at baseline to mean 206 mg/day at Week 26 and further decreased from Week 26 to mean 170 mg/day at Week 52, with improvements sustained through the end of the trials. Overall, 98% of patients achieved independence from active vitamin D, defined as taking no calcitriol or alfacalcidol, at final trial visit. Overall, 95% of patients achieved independence from therapeutic doses of calcium, defined as taking less than600 mg/day of calcium, at final trial visit. Mean serum calcium increased within the normal range to 8.9 mg/dL, with 88% of patients having normal albumin-adjusted serum calcium levels at final trial visit. In both trials, TransCon PTH was generally well tolerated, with no new safety signals identified. Most treatment-emergent adverse events, or TEAEs, were mild to moderate. The PaTH Forward and PaTHway trials included a combined total of 141 adults with hypoparathyroidism. The pooled kidney data analysis covers the 139 patients who received at least one dose of TransCon PTH. Of these, 129 completed the final trial visit. The PaTH Forward and PaTHway trials comprised randomized, double-blind, placebo-controlled periods through Weeks 4 and 26 and open-label extension periods through Weeks 266 and 182, respectively. An eGFR greater than or equal to30 mL/min/1.73 m2 was required for trial eligibility. Mean baseline eGFR for the pooled analysis was 69 mL/min/1.73 m2. The pooled data were analyzed post-hoc and included evaluation of the impact of TransCon PTH on renal function, as assessed by, change from baseline in eGFR and proportion of patients with an increase in eGFR greater than or equal to5 mL/min/1.73 m2, as well as 24-hour urinary calcium.

Ascendis Pharma Recommended for EU Marketing Authorization of TransCon CNP

Ascendis Pharma announced that the Committee for Medicinal Products for Human Use, or CHMP, of the European Medicines Agency, or EMA, has adopted an opinion recommending marketing authorization for once-weekly TransCon CNP in the European Union for the treatment of achondroplasia in children two years of age and older whose epiphyses are not closed. The final European Commission, or EC, decision on Ascendis Pharma's Marketing Authorization Application, or MAA, is expected during Q4.

Ascendis Pharma Shares Drop 12.8% to $231.00

Ascendis Pharma is down -12.8%, or -$33.92 to $231.00.