Ascendis Pharma (ASND) Study result presentation summary

Background and therapy rationale

• PTH regulates calcium and phosphate homeostasis, acting on bone, kidney, and intestine.

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

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

TransCon PTH product profile

• TransCon PTH is a once-daily prodrug delivering active PTH within physiological range for 24 hours.

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

PaTHway Phase 3 trial design and population

• 82 adults randomized 3:1 to TransCon PTH or placebo, followed by a 156-week open-label extension.

• Key endpoints: normocalcemia, independence from conventional therapy, safety, renal, skeletal, and HRQoL assessments.

• Baseline characteristics were balanced between groups, with most participants having acquired hypoparathyroidism.