Ascendis Pharma (ASND) Study result presentation summary

Background and therapy rationale

• PTH is essential for calcium and phosphate homeostasis, acting on bone, kidney, and indirectly on the intestine.

• Conventional therapy for hypoparathyroidism alleviates symptoms but does not restore normal PTH physiology.

• PTH replacement aims to normalize serum and urine calcium and phosphate, skeletal health, and quality of life.

TransCon PTH design and regulatory status

• TransCon PTH is a once-daily prodrug of PTH (1-34) providing physiological PTH levels for 24 hours.

• Approved for adult hypoparathyroidism in the US, EU, and other regions.

Phase 2 PaTH Forward trial design and population

• Randomized, double-blind, placebo-controlled trial with a 262-week open-label extension in adults with chronic hypoparathyroidism.

• 59 participants enrolled; 56 completed the 5-year trial.

• Majority were female, white, and had acquired hypoparathyroidism from neck surgery.