Ascendis Pharma's Hypoparathyroidism Trial Meets Primary, Secondary Endpoints

Ascendis Pharma A/S ASND announced topline data from its Phase 3 PaTHway Trial of TransCon PTH in adults with hypoparathyroidism (HP), a condition in which the body produces abnormally low levels of parathyroid hormone (PTH).

• 78.7% of TransCon PTH-treated patients achieved serum calcium levels in the normal range and independence from therapeutic levels of conventional therapy, compared to 4.8% for patients in the control group.
• The data also exhibited a statistically significant decrease in patient-reported, disease-specific physical and cognitive symptoms compared to patients in the control group.
• At Week 26, 95% of TransCon PTH-treated patients could discontinue conventional treatments with therapeutic levels of calcium supplements and active vitamin D.
• Also See: Ascendis Pharma's Growth Hormone Deficiency Med Scores European Approval.
• TransCon PTH-treated patients demonstrated increased levels of bone turnover markers at Week 26.
• TransCon PTH was generally well tolerated, with no discontinuations related to the study drug. 
• One death in the TransCon PTH arm was deemed unrelated to the study drug.

Next Steps:

• Ascendis plans to submit a U.S. application for TransCon PTH for adults with hypoparathyroidism during Q3 of 2022 and a European application during Q4 of 2022.
• Topline results for the Phase 3 PaTHway Japan Trial are expected in Q3 of 2022. 
• Ascendis also plans to initiate a pediatric hypoparathyroidism trial of TransCon PTH in Q4 of 2022.
• Price Action: ASND shares closed 0.30% lower at $102.18 on Friday.