– For the primary composite endpoint, TransCon PTH demonstrated a response rate of 78.7% compared to 4.8% for control (p-value <0.0001).
– TransCon PTH demonstrated statistically significant improvements compared to control on all key secondary endpoints, which included measures evaluating patient-reported disease symptoms and impacts.
– TransCon PTH was generally well-tolerated, with no discontinuations related to study drug.
– On track to submit NDA during the third quarter and MAA during the fourth quarter of 2022.
COPENHAGEN, Denmark, March 13, 2022 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced that top-line data from the randomized, double-blind, placebo-controlled portion of its Phase 3 PaTHway Trial of TransCon PTH in adults with hypoparathyroidism (HP) demonstrated statistically significant improvement with TransCon PTH compared to control on the primary composite endpoint and all key secondary endpoints. The primary endpoint – defined as serum calcium levels in the normal range (8.3–10.6 mg/dL) and independence from conventional therapy (active vitamin D and >600 mg/day of calcium supplements) with no increase in prescribed study drug within the 4 weeks prior to the Week 26 visit – was achieved by 78.7% of TransCon PTH-treated patients (48 of 61), compared to 4.8% for patients (1 of 21) in control group (p-value <0.0001).
“Hypoparathyroidism is an area of major unmet medical need with an estimated 200,000 patients in the United States, Europe, and Japan. Conventional therapy with calcium supplements and active vitamin D is aimed at maintaining serum calcium in the normal range, with the hope of reducing short-term symptoms, and is not able to address the underlying disease. In addition, conventional therapy can lead to long-term complications that include severe diseases such as chronic kidney diseases, liver and basal ganglia calcifications, cardiovascular complications, and bone damage,” said Jan Mikkelsen, Ascendis Pharma’s President and CEO. “This is the first Phase 3 trial where more than three quarters of patients achieved control of their hypoparathyroidism, defined as normalization of serum calcium and independence from conventional therapy. This study also demonstrated statistically significant improvement in all pre-specified patient-reported symptom and quality-of-life domains.”
Highlights of the Phase 3 PaTHway Trial Top-Line Data
The PaTHway Trial is a Phase 3 double-blind, placebo-controlled trial of 82 dosed adults with chronic hypoparathyroidism randomized 3:1 (TransCon PTH:placebo).
Primary Composite Endpoint:
78.7% of TransCon PTH-treated patients (48 of 61) achieved serum calcium levels in the normal range (8.3–10.6 mg/dL) and independence from therapeutic levels of conventional therapy, compared to 4.8% for patients (1 of 21) in control group (p-value = <0.0001).
Key Pre-Specified Secondary Endpoints:
Statistically significant decrease in patient-reported, disease-specific physical and cognitive symptoms compared to patients in control group, as shown on Hypoparathyroidism Patient Experience Scales (HPES) Symptom-Physical domain scores (p-value = 0.0038) and HPES Symptom-Cognitive domain scores (p-value = 0.0055).
Statistically significant reduction in patient-reported disease impact compared to patients in control group, as shown on HPES Impact-Physical Functioning domain scores (p-value = 0.0046) and HPES Impact-Daily Life domain scores (p-value = 0.0061).