Clinical TrialJun 11, 2026, 04:02 PM
Ascendis Pharma TransCon PTH shows sustained 5-year efficacy in Phase 2 trial
AI Summary
Ascendis Pharma announced positive 5-year (Week 266) data from its Phase 2 PaTH Forward Trial for TransCon PTH (palopegteriparatide) in adults with hypoparathyroidism. The trial demonstrated sustained efficacy and safety, replicating endogenous PTH actions with beneficial impacts on kidney, small intestine, CNS, and bone. Patients showed significant improvements in serum calcium, kidney function, bone mineral density, and quality of life, while achieving independence from conventional active vitamin D and calcium therapy. The treatment was generally well-tolerated with no new safety signals.
Key Highlights
- 82% of patients were responders for the multi-component endpoint.
- 95% of patients completed five years of treatment in the trial.
- 88% of patients had normal albumin-adjusted serum calcium levels.
- 96% of patients achieved independence from active vitamin D.
- 95% of patients achieved independence from therapeutic doses of calcium.
- Mean eGFR increased by 9.4 mL/min/1.73 m2 from baseline, sustained over five years.
- Mean 24-hour urine calcium normalized within 26 weeks and remained normal.
- Improvements in symptoms and health-related quality of life were maintained.
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